Study Summaries
CIBMTR performs ground-breaking research into blood and marrow transplant. This research has a significant impact on the survival and quality of life of thousands of transplant and cellular therapy patients. Here we provide summaries of CIBMTR research in an easy-to-read way. Now you and your family have access to useful information that can help you talk with your doctor about treatment options.
Study summaries are organized by disease / condition and other transplant characteristics. More easy-to-read information about transplant, including diseases treatable by transplant, is available on the NMDP website. You may also visit the NMDP Jason Carter Clinical Trials Search & Support webpage to view additional summaries of completed clinical trials, also called research studies. For help understanding this information, contact the NMDP Patient Support Center at 1 (888) 999-6743 or patientinfo@nmdp.org.
CIBMTR shares data sets from scientific papers with the public, free of charge. CIBMTR removes personal data to protect the privacy of study participants. For more information, visit the Publicly Available Datasets webpage.
Diseases and Conditions
Other Topics related to Transplant |
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Diseases and Conditions
Acute Leukemia
Acute leukemia includes acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL). AML is the most common type of acute leukemia. Nearly 15,000 new cases are found in the United States each year. AML can affect people of any age, but is most common in adults. There are about 6,000 new cases of ALL in the United States each year. It can affect people of any age, but is the most common type of leukemia in children under 15.
| Summary (PDF) | Important Points | Full Article |
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| Given after transplant, medicine helps patients with traces of cancer | Gilteritinib as Post-Transplant Maintenance for Acute Myeloid Leukemia With Internal Tandem Duplication Mutation of FLT3 | |
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A medicine for transplant can raise the chance of infections |
Cyclophosphamide is still helpful; patients should be watched for infections | Post-transplantation cyclophosphamide is associated with increased bacterial infections |
| Older adults with leukemia should get younger, matched donors, if possible | Impact of Donor Age on Allogeneic Hematopoietic Cell Transplantation Outcomes in Older Adults with Acute Myeloid Leukemia | |
| Of adults older than 60 who needed BMT, fewer than 5% got BMT | Age is no barrier for adults undergoing HCT for AML in CR1: contemporary CIBMTR analysis. | |
| Blood and marrow transplant is an option for some people with rare leukemia | Outcomes of allogeneic hematopoietic cell transplantation in T cell prolymphocytic leukemia: A contemporary analysis from the Center for International Blood and Marrow Transplant Research. | |
| Both help people who have acute leukemia but no matched donor | Comparison of haploidentical and umbilical cord blood transplantation after myeloablative conditioning. | |
| For some people older than 50, transplant from young, matched donors is better than half-matched donors | Study looked at ages and types of donors to treat people with acute leukemia | Alternative donor transplantation for acute myeloid leukemia in patients aged ≥50 years: Young HLA-matched unrelated or haploidentical donor? |
| Haplo transplant helps people with leukemia | News may help people of all ethnicities get BMT sooner | Outcomes of haploidentical vs matched sibling transplantation for acute myeloid leukemia in first complete remission. |
| Blood or marrow transplant helps treat leukemia after age 60 | People older than 60 lived longer after getting BMT than chemo alone. | Allogeneic hematopoietic cell transplantation compared to chemotherapy consolidation in older acute myeloid leukemia (AML) patients 60-75 years in first complete remission (CR1): An alliance (A151509), SWOG, ECOG-ACRIN, and CIBMTR study. |
| Experimental medicine treats children with leukemia and MDS | Treosulfan may be safer than a similar medicine before transplant. | Treosulfan, fludarabine and low-dose total body irradiation for children and young adults with acute myeloid leukemia or myelodysplastic syndrome undergoing allogeneic hematopoietic cell transplantation: A prospective phase II trial of the Pediatric Blood and Marrow Transplant Consortium. |
| Chemotherapy prep for transplant works for acute leukemia, but risks of relapse are higher | People with acute leukemia who got only chemotherapy (chemo) to prepare them for transplant lived almost as long as people who got chemo plus radiation, but their risks for leukemia recurrence were higher. People had fewer side effects with chemo-only prep. | Intravenous busulfan compared with total body irradiation pretransplant conditioning for adults with acute lymphoblastic leukemiaIntravenous busulfan compared with total body irradiation pretransplant conditioning for adults with acute lymphoblastic leukemia. |
| Patients with myelodysplastic syndromes or acute myeloid leukemia may benefit from a standard preparative regimen | Patients who got a standard preparative regimen were more likely to be cancer-free a year and a half after transplant than patients who got a reduced-intensity regimen. | Myeloablative versus reduced-intensity hematopoietic cell transplantation for acute myeloid leukemia and myelodysplastic syndromes |
| Treatment helps children and teens with acute leukemia thrive after cord blood transplants | The TCF treatment regimen before cord blood transplant helped young people with acute leukemia live longer. | Umbilical cord blood transplantation in children with acute leukemia: Impact of conditioning on transplantation outcomes |
| Early results suggest people live equally long after half-matched transplants using either peripheral blood or bone marrow | Scientists followed people for two years after transplants for: Acute leukemias, myelodysplastic syndrome, and lymphomas. | Mobilized peripheral blood stem cells versus unstimulated bone marrow as a graft source for T-cell—replete haploidentical donor transplantation using post-transplant cyclophosphamide |
| A transplant from an unrelated donor can treat acute lymphoblastic leukemia |
New research may make it possible for more people with acute lymphoblastic leukemia (ALL) to get life-saving transplants. In the past, a transplant using blood-forming cells from a sibling was the only well-tested transplant option. New research shows that a transplant from an unrelated donor can help people with ALL live equally long as a transplant from a sibling. |
Comparing outcomes of matched related donor and matched unrelated donor hematopoietic cell transplants in adults with B-cell acute lymphoblastic leukemia |
| Less chronic GVHD after cord blood transplant for acute leukemia |
Patients had less chronic GVHD after cord blood transplants. Patients who received ATG had less acute GVHD. |
GVHD after umbilical cord transplantation for acute leukemia: An analysis of risk factors and effect on outcomes |
| Blood or marrow transplant can work well to treat B-cell acute lymphoblastic leukemia for older patients |
Reduced-intensity BMT can work well for older patients with B-cell ALL. For patients who had BMT when the disease was in the 1st complete remission, almost half (45%) were alive 3 years later. |
Reduced intensity conditioned allograft yields favorable survival for older adults with B-Cell acute lymphoblastic leukemia: A CIBMTR analysis |
| Transplant may help older patients with acute myeloid leukemia | Reduced-intensity transplant is a good treatment option for some older patients with AML. | Phase II study of allogeneic transplantation for older patients with acute myeloid leukemia in first complete remission using a reduced-intensity conditioning regimen: Results from Cancer and Leukemia Group B 100103 (Alliance for Clinical Trials in Oncology)/Blood and Marrow Transplant Clinical Trial Network 0502 |
| More teens and young adults with ALL survive after blood or marrow transplant than in the past | 5-year survival increased over time in teens and young adults and in children. | Survival improvements in adolescents and young adults after myeloablative allogeneic transplantation for acute lymphoblastic leukemia |
| Similar survival for leukemia patients receiving transplants from related vs. unrelated donors | Patients with leukemia who get a transplant from a well matched unrelated donor or from an unrelated donor with a single mismatch should have similar survival to patients who have a related donor. | Outcomes after matched unrelated donor versus identical sibling hematopoietic cell transplantation in adults with acute myelogenous leukemia |
| Unrelated umbilical cord blood transplants equal to unrelated bone marrow in helping children with acute leukemia | Children with acute leukemia who receive unrelated umbilical-cord blood transplants have outcomes equal to - or in some cases, better than - those treated with bone marrow. | Outcomes of transplantation of unrelated donor umbilical cord blood and bone marrow in children with acute leukaemia: A comparison study |
Cancer in Children and Teens
The types of cancers that affect children and teens are often different than the types that affect adults. Children and teens also respond to treatments differently than adults. Some cancers that affect children include leukemia, neuroblastoma, Wilms tumor, and lymphoma.
| Summary (PDF) | Important Points | Full Article |
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| Schoolchildren need more support | Return to school practices after hematopoietic cell transplantation: a survey of transplant centers in the United States | |
| About 1 in 7 children end up in ICU within 1 year after transplant | Critical Illness Risk and Long-Term Outcomes Following Intensive Care in Pediatric Hematopoietic Cell Transplant Recipients. | |
| Largest study of children who had acute myeloid leukemia compares treatments | Chronic conditions, late mortality, and health status after childhood AML: a Childhood Cancer Survivor Study report | |
| 40% of children have effects 10 years later; treatments available | Association of Chronic Graft-versus-Host Disease with Late Effects following Allogeneic Hematopoietic Cell Transplantation for Children with Hematologic Malignancy. | |
| Children can return to school after vaccinated for COVID-19 | Return-to-School Practices for Pediatric Hematopoietic Cell Transplantation Recipients during the COVID-19 Pandemic | |
| Carbapenems may seriously affect young people who have leukemia | Broad-Spectrum Antibiotics and Risk of Graft-versus-Host Disease in Pediatric Patients Undergoing Transplantation for Acute Leukemia: Association of Carbapenem Use with the Risk of Acute Graft-versus-Host Disease. | |
| More support and resources are needed for children and families | Neighborhood-Poverty and Pediatric Allogeneic Hematopoietic Cell Transplantation Outcomes: A CIBMTR Analysis. | |
| Tool forecasts how well blood and marrow transplant will work | A validated pediatric disease risk index for allogeneic hematopoietic cell transplantation | |
| Teens and young adults need check-ups for side effects, even years after leukemia treatment | Blood or marrow transplant cures leukemia but may have late side effects | Late effects after ablative allogeneic stem cell transplantation for adolescent and young adult acute myeloid leukemia. |
| Second transplant helps some children with acute leukemia | Those whose leukemia was controlled (in remission) at the time of their second BMT lived longer than those whose leukemia failed to respond to chemo. | Outcomes after second hematopoietic cell transplant for children and young adults with relapsed acute leukemia. |
| What happens when blood cells are used instead of bone marrow in BMT for children and teens with leukemia? | For children and teens with acute leukemia, researchers recommend BMT with bone marrow cells. | Bone marrow versus peripheral blood from unrelated donors for children and adolescents with acute leukemia. |
| Experimental medicine treats children with leukemia and MDS | Treosulfan may be safer than a similar medicine before transplant | Treosulfan, fludarabine and low-dose total body irradiation for children and young adults with acute myeloid leukemia or myelodysplastic syndrome undergoing allogeneic hematopoietic cell transplantation: A prospective phase II trial of the Pediatric Blood and Marrow Transplant Consortium. |
| Childhood transplants linked to slightly higher risk of heart problems and diabetes | Scientists urge checkups for cholesterol and blood pressure. | Late cardiovascular morbidity and mortality following pediatric allogeneic hematopoietic cell transplantation |
| Treatment helps children and teens with acute leukemia thrive after cord blood transplants | The TCF treatment regimen before cord blood transplant helped young people with acute leukemia live longer. | Umbilical cord blood transplantation in children with acute leukemia: Impact of conditioning on transplantation outcomes |
| Common late effects after transplant in very young children |
30% of very young BMT recipients have organ damage or other late effects. Common late effects are delayed growth, cataracts, and hypothyroidism. Full-body radiation increases the chance of getting these late effects. |
Survival and late effects after allogeneic hematopoietic cell transplantation for hematologic malignancy at less than three years of age |
| Some children who donate cells to a brother or a sister have a lower quality of life and need extra support |
About 20% of donors (1 in 5) at each time point had very poor QOL. Parents tended to overestimate their child's QOL. Younger children were more likely to have a lower QOL. |
Health-related quality of life among pediatric hematopoietic stem cell donors |
| Transplant outcomes for children and young adults with chronic myeloid leukemia |
Transplant outcomes are similar in children and young adults with CML. Transplant works better with bone marrow from sibling donors. Taking TKIs before transplant doesn't affect how well transplant works. |
Outcomes of allogeneic hematopoietic cell transplantation in children (<18y) and young adults (18-29y) with chronic myeloid leukemia: A CIBMTR cohort analysis |
| Transplant can help some children with hypodiploid acute lymphoblastic leukemia |
Children with hypodiploid ALL did worse when they had a transplant later (in their 2nd complete remission). Children with 43 or fewer chromosomes in their diseased cells had a higher risk of the leukemia coming back and dying after transplant compared to children with 44 or 45 chromosomes. While more children may live longer because doctors can find better matched donors, disease factors still affect transplant outcomes. |
Transplant outcomes for children with hypodiploid acute lymphoblastic leukemia |
| More teens and young adults with ALL survive after blood or marrow transplant than in the past | 5-year survival increased over time in teens and young adults and in children. | Survival improvements in adolescents and young adults after myeloablative allogeneic transplantation for acute lymphoblastic leukemia |
| Allo transplant helps some children with neuroblastoma |
Only 4% of patients with neuroblastoma (NBL) get an allo transplant. 20% of patients with NBL who get an allo transplant go into remission for at least 5 years. Patients with NBL who only get an allo transplant do better than patients who get an auto transplant first and then an allo transplant. |
Allogeneic hematopoietic cell transplantation for neuroblastoma: The CIBMTR experience |
| Younger sibling donors better for children and teens needing transplants |
Children and teen patients who get transplants from their younger siblings get less GVHD than patients who get transplants from their older siblings. For adult patients, it makes no difference if a sibling donor is younger or older. |
Birth order and transplantation outcome in HLA-identical sibling stem cell transplantations: An analysis on behalf of the Center for International Blood and Marrow Transplantation |
| Unrelated umbilical cord blood transplants equal to unrelated bone marrow in helping children with acute leukemia | Children with acute leukemia who receive unrelated umbilical-cord blood transplants have outcomes equal to - or in some cases, better than - those treated with bone marrow. | Outcomes of transplantation of unrelated donor umbilical cord blood and bone marrow in children with acute leukaemia: A comparison study |
Chronic Leukemia and Myelodysplastic Syndromes
Chronic leukemia includes chronic lymphocytic leukemia (CLL), chronic myeloid leukemia (CML), and myelodysplastic syndromes (MDS). CLL is primarily an adult disease. It is very rare in children and young adults. About 15,000 people are diagnosed each year in the United States. CML is a relatively common form of leukemia. It affects more than 20,000 people in the United States. There are 6,000 new cases each year. Most cases of CML appear in adults. MDS is uncommon before age 50, and the risk increases as a person gets older. It is most commonly diagnosed in people in their 70s. About 19,000 people are diagnosed with MDS in the United States each year.
| Summary (PDF) | Important Points | Full Article |
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For older adults with MDS, transplant preserves quality of life |
Blood and marrow transplant improves survival | Health-related quality of life in reduced-intensity hematopoietic cell transplantation based on donor availability in patients aged 50-75 with advanced myelodysplastic syndrome: BMT CTN 1102 |
| Blood and marrow transplant helps people with myelodysplastic syndromes | Relapse and Disease-Free Survival in Patients With Myelodysplastic Syndrome Undergoing Allogeneic Hematopoietic Cell Transplantation Using Older Matched Sibling Donors vs Younger Matched Unrelated Donors. | |
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Blood and marrow transplants (BMT) help people with advanced myelodysplastic syndromes (MDS) |
Not enough people have access to life-saving transplant |
Biologic Assignment Trial of Reduced-Intensity Hematopoietic Cell Transplantation Based on Donor Availability in Patients 50-75 Years of Age With Advanced Myelodysplastic Syndrome Blood and Marrow Transplant Clinical Trials Network Study 1102 heralds a new era in hematopoietic cell transplantation in high-risk myelodysplastic syndromes: Challenges and opportunities in implementation |
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Half-matched donors help more people with MDS get transplants |
Both matched, unrelated people and half-matched relatives can donate | Alternative donor transplantation for myelodysplastic syndromes: Haploidentical relative and matched unrelated donors |
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New tool shows which people with myelodysplastic syndromes will be most helped by transplant |
The tool calculates how likely it is for the person to be alive and cancer-free for up to 2 years after transplant | A Personalized Prediction Model for Outcomes after Allogeneic Hematopoietic Cell Transplant in Patients with Myelodysplastic Syndromes |
| BMT may help more people with myelofibrosis than previously known | Survival following allogeneic transplant in patients with myelofibrosis. | |
| Transplant can help people aged 65 and older with myelodysplastic syndromes (MDS) | Experts recommend that Medicare pay for transplant |
Comparison of patient age groups in transplantation for myelodysplastic syndrome: The Medicare coverage with evidence development study. |
| Patients with myelodysplastic syndromes or acute myeloid leukemia may benefit from a standard preparative regimen | Patients who got a standard preparative regimen were more likely to be cancer-free a year and a half after transplant than patients who got a reduced-intensity regimen. | Myeloablative versus reduced-intensity hematopoietic cell transplantation for acute myeloid leukemia and myelodysplastic syndromes |
| Cord blood transplant helps some people with myelodysplastic syndromes | If someone with MDS does not have a matched family member or unrelated donor, a cord blood transplant might help. | Outcomes after umbilical cord blood transplantation for myelodysplastic syndromes |
| How to make transplant work best for myelodysplastic syndromes | There are many things to think about before using transplant to treat MDS. Doctors and patients should think about the timing, the donor, the preparative regimen, and the cells. | Transplantation for myelodysplastic syndromes: Who, when, and which conditioning regimens |
| Transplant outcomes for children and young adults with chronic myeloid leukemia |
Transplant outcomes are similar in children and young adults with CML. Transplant works better with bone marrow from sibling donors. Taking TKIs before transplant doesn't affect how well transplant works. |
Outcomes of allogeneic hematopoietic cell transplantation in children (<18y) and young adults (18-29y) with chronic myeloid leukemia: A CIBMTR cohort analysis |
Graft-versus-Host Disease
Graft-versus-host disease (GVHD) is a common (sometimes serious) side effect of an allogeneic transplant. GVHD happens when cells from the donor (the graft) see the patient’s cells (the host) as different and attack them. GVHD can occur along a spectrum that includes acute or chronic GVHD.
| Summary (PDF) | Important Points | Full Article |
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| Post-transplant cyclophosphamide works better than older meds | Improved Patient-Reported Outcomes With Post-Transplant Cyclophosphamide: A Quality-of-Life Evaluation and 2-Year Outcomes of BMT CTN 1703 | |
| Results of clinical trial will change treatments for leukemia and lymphoma | Post-Transplantation Cyclophosphamide-Based Graft-versus-Host Disease Prophylaxis | |
| New medicines allow more people to get treatment, including people of color | Real-World Data Showing Trends and Outcomes by Race and Ethnicity in Allogeneic Hematopoietic Cell Transplantation: A Report from the Center for International Blood and Marrow Transplant Research. | |
| Cyclophosphamide helpful after blood and marrow transplant, study shows | Characteristics of Graft-Versus-Host Disease (GvHD) After PostTransplantation Cyclophosphamide Versus Conventional GvHD Prophylaxis. |
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| Better options needed to prevent graft-versus-host disease | Randomized phase III BMT CTN trial of calcineurin inhibitor-free chronic graft-versus-host disease interventions in myeloablative hematopoietic cell transplantation for hematologic malignancies. | |
| Medicine for acute graft-versus-host disease helps more people get BMT |
Improved overall survival of patients treated with abatacept in combination with a calcineurin inhibitor and methotrexate following allogeneic hematopoietic stem cell transplantation: Analysis of the Center for International Blood and Marrow Transplant Research Database. Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR; 2022. |
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| New tool helps doctors decide when to stop medicines | After BMT, many people need immune suppression for more than 5 years | Factors associated with successful discontinuation of immune suppression after allogeneic hematopoietic cell transplantation. |
| New treatment for GVHD has fewer side effects | Sirolimus treats graft-versus-host disease | Randomized Multicenter Trial of Sirolimus vs. Prednisone as Initial Therapy for Standard Risk Acute GVHD: BMT CTN 1501 |
| Medicines keep away both GVHD and cancer | Three medicines given after BMT work best, according to a clinical trial. | Three prophylaxis regimens (tacrolimus, mycophenolate mofetil, and cyclophosphamide; tacrolimus, methotrexate, and bortezomib; or tacrolimus, methotrexate, and maraviroc) versus tacrolimus and methotrexate for prevention of graft-versus-host disease with haemopoietic cell transplantation with reduced-intensity conditioning: A randomised phase 2 trial with a non-randomised contemporaneous control group (BMT CTN 1203) |
| Transplant prep for follicular lymphoma has less risk | FCR combo before transplant may lower risk of chronic GVHD | Fludarabine and busulfan versus fludarabine, cyclophosphamide, and rituximab as reduced-intensity conditioning for allogeneic transplantation in follicular lymphoma |
| Better survival over time for patients with acute GVHD |
Treatments for patients with moderate to severe acute GVHD have gotten better over time. Tacrolimus helped patients with moderate acute GVHD live longer. |
Improved survival after acute graft vs. host disease diagnosis in the modern era |
| Less chronic GVHD after cord blood transplant for acute leukemia |
Patients had less chronic GVHD after cord blood transplants. Patients who received ATG had less acute GVHD. |
GVHD after umbilical cord transplantation for acute leukemia: An analysis of risk factors and effect on outcomes |
| A new way to measure acute graft-versus-host disease can help doctors predict transplant outcomes |
The Refined Acute GVHD Risk Score is a way for doctors to predict the likelihood that: -A patient will be alive 6 months after starting treatment for acute GVHD. |
A refined risk score for acute graft-versus-host disease that predicts response to initial therapy, survival, and transplant-related mortality |
| New blood test can predict the severity of acute graft-versus-host disease | Biomarkers can predict whether acute GVHD will be mild or severe and whether it is likely to respond to treatment. | A prognostic score for acute graft-versus-host disease based on biomarkers: A multicentre study |
| Risk score for chronic graft-versus-host disease | Researchers created a chronic GVHD risk score. | Chronic GVHD risk score: A Center for International Blood and Marrow Transplant Research analysis |
Lymphoma
Lymphoma includes Hodgkin lymphoma and non-Hodgkin lymphoma (NHL). About 9,000 people are diagnosed with Hodgkin lymphoma in the United States each year. It can occur in both children and adults. But it is more common among young adults and older adults. About 69,000 people are diagnosed with NHL in the United States each year. Most of these people are older than 60.
| Summary (PDF) | Important Points | Full Article |
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| Treatment for lymphoma may be affected by race and ethnicity | Real-world and clinical trial outcomes in large B-cell lymphoma with axicabtagene ciloleucel across race and ethnicity. | |
| BMT helps some older people who have lymphoma | Scientists say Medicare should pay for BMT for people older than 65 who have non-Hodgkin lymphoma (NHL). | Outcomes of Medicare-age eligible NHL patients receiving RIC allogeneic transplantation: A CIBMTR analysis. |
| Transplant prep for follicular lymphoma has less risk | FCR combo before transplant may lower risk of chronic GVHD | Fludarabine and busulfan versus fludarabine, cyclophosphamide, and rituximab as reduced-intensity conditioning for allogeneic transplantation in follicular lymphoma |
| Two-part transplant helps people with Hodgkin lymphoma | Splitting an autologous transplant into two doses of cells with more medicines may help people live longer than giving one transplant, a review of two studies showed. | Tandem autologous hematopoietic cell transplantation for patients with primary progressive or recurrent Hodgkin lymphoma: A SWOG and Blood and Marrow Transplant Clinical Trials Network phase II Trial (SWOG S0410/BMT CTN 0703) |
| Early results suggest people live equally long after half-matched transplants using either peripheral blood or bone marrow | Scientists followed people for two years after transplants for: Acute leukemias, myelodysplastic syndrome, and lymphomas. | Mobilized peripheral blood stem cells versus unstimulated bone marrow as a graft source for T-cell—replete haploidentical donor transplantation using post-transplant cyclophosphamide |
| Transplant from half-matched donors may be as good as matched donors for people with lymphoma |
People were just as likely to be alive 3 years after transplant whether they had a matched sibling transplant or a haploidentical transplant. Fewer people got GVHD after a haploidentical transplant. |
Reduced-intensity transplantation for lymphomas using haploidentical related donors versus HLA-matched sibling donors: A Center for International Blood and Marrow Transplant Research analysis |
| More older people with non-Hodgkin lymphoma get transplant | Transplant is a treatment option for healthy older people with NHL | Allogeneic hematopoietic cell transplantation as curative therapy for patients with non-Hodgkin lymphoma: Increasingly successful application to older patients |
| Half-matched (haploidentical) transplant for patients with lymphoma |
Patients who had a haploidentical or unrelated donor transplant had about the same 3-year survival. Patients who had a haploidentical transplant had less chronic GVHD. |
Reduced-intensity transplantation for lymphomas using haploidentical related donors versus HLA-matched unrelated donors |
Multiple Myeloma
Multiple myeloma is a cancer of the plasma cells. Plasma cells are a part of the immune system. More than 22,000 people in the United States are diagnosed with multiple myeloma each year. Most people who have the disease are age 65 or older.
| Summary (PDF) | Important Points | Full Article |
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| Emotional and physical therapies may help | Symptom clusters and their impact on quality of life in multiple myeloma survivors: Secondary analysis of BMT CTN 0702 trial | |
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Overweight not linked to relapse or quality of life after transplant |
For adults with multiple myeloma, being overweight is not worrisome | Impact of Visceral Obesity on Clinical Outcome and Quality of Life for Patients with Multiple Myeloma: A Secondary Data Analysis of STaMINA (BMT CTN 0702) Trial |
| People who got early BMT stayed myeloma-free longer than people who got medicines only | Triplet Therapy, Transplantation, and Maintenance until Progression in Myeloma | |
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People who get BMT for multiple myeloma have good quality of life |
However, some patients still have symptoms more than a year later | Trajectories of quality of life recovery and symptom burden after autologous hematopoietic cell transplantation in multiple myeloma |
| Single transplant effective for multiple myeloma | Doctors recommend a single autologous blood or marrow transplant followed by only lenalidomide maintenance, instead of more intensive treatments. | Autologous transplantation, consolidation and maintenance therapy in multiple myeloma |
| Auto transplants are safe and effective for people with multiple myeloma and damaged kidneys |
Auto transplants are safe and work equally well in people with multiple myeloma and damaged kidneys. The treatment can help some people stop dialysis treatments. Higher doses of melphalan can improve results. |
Autologous hematopoietic cell transplantation for multiple myeloma patients with renal insufficiency: A Center for International Blood and Marrow Transplant Research analysis |
Other Diseases
Transplant is also used to treat other diseases, such as multiple sclerosis and sickle cell disease. Multiple sclerosis is a chronic, often disabling disease, that attacks the central nervous system (brain and spinal cord). More than 2.3 million people are affected by MS worldwide. Most are diagnosed between the ages of 20 and 50. Women get MS more often than men. Sickle cell disease is an inherited disease of the red blood cells. In the United States, it affects about 70,000-100,000 people. It is most common among people with African or Hispanic ancestry.
| Summary (PDF) | Important Points | Full Article |
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Half-matched transplant helps adults with sickle cell disease |
Certain medicines make transplant safer | Haploidentical bone marrow transplantation for sickle cell disease |
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Transplant helps teens, young adults with sickle cell disease |
Longer, larger studies are needed | Hematopoietic cell transplant compared with standard care in adolescents and young adults with sickle cell disease |
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Patients live long after transplant for severe aplastic anemia |
Serious effects possible many years after transplant | Long-term survival after unrelated donor marrow transplantation for aplastic anaemia after optimized conditioning regimen: a retrospective multicentre cohort study |
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Most children are alive and well after unrelated donor transplant for sickle cell disease |
Blood or marrow transplant from unrelated donors is an option | Long-term outcomes after unrelated donor transplantation for severe sickle cell disease on the BMT CTN 0601 trial |
| Most people who live for 2 years or longer after their blood or marrow transplant (BMT) are cured of their disease and can expect to live for many more years | Long-term survival after hematopoietic cell transplant for sickle cell disease compared to the United States population | |
| New risk score predicts person's chance that transplant will work to treat sickle cell disease Accessible version | Children who have matched sibling donors are helped most | Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease |
| Certain medicines used for bone marrow transplant (BMT) offer better cure for severe aplastic anemia (SAA) | For people who got BMT from a matched brother or sister, the best medicines were cyclophosphamide together with anti-thymocyte globulin (ATG), given with or without fludarabine. | Choice of conditioning regimens for bone marrow transplantation in severe aplastic anemia. |
| Blood or marrow transplant can help children with severe sickle cell disease | Matched, sibling donor is best | Effect of donor type and conditioning regimen intensity on allogeneic transplantation outcomes in patients with sickle cell disease: A retrospective multicentre, cohort study. |
| BMT can treat blood cancers in people with HIV / AIDS | An early study shows that blood or marrow transplant (BMT) is as safe for people with HIV as for those without it. | Allogeneic Hematopoietic Cell Transplant for HIV Patients with Hematologic Malignancies: the BMT CTN-0903/AMC-080 Trial. |
| BMT may protect IQ in children with sickle cell disease | Blood or marrow transplant from an unrelated donor may preserve intelligence in children with sickle cell disease. | Functional and radiologic assessment of the brain after reduced intensity unrelated donor transplantation for severe sickle cell disease BMT CTN 0601 |
| Reduced-intensity transplant prep helps young people with immune system disorders | In this clinical trial, doctors treated people with 5 rare immune disorders: -Hemophagocytic lymphohistiocytosis (HLH) -Chronic active Epstein-Barr virus (CAEBV) -Chronic granulomatous disease (CGD) -Hyperimmunoglobulin syndrome (HIGM1) -Immune dysregulation, polyendocrinopathy, enteropathy and X-linked syndrome (IPEX) |
Reduced intensity conditioning for hematopoietic cell transplant for HLH and primary immune deficiencies BMT CTN 1204 |
| Doctors say transplants treat systemic sclerosis or scleroderma | Autologous transplants help people with systemic sclerosis live longer and improve their skin, lungs and quality of life. | Systemic sclerosis as an indication for autologous hematopoietic cell transplantation: position statement from the American Society for Blood and Marrow Transplantation |
| Auto transplant helps some people with multiple sclerosis | For almost half of people who got auto transplant, their MS didn't get worse for 5 years after transplant. | Long-term outcomes after autologous hematopoietic stem cell transplantation for multiple sclerosis |
Other Topics Related to Transplant
Access to Transplant
Doctors want to make transplant available to everyone who needs it. Research helps doctors know which patients are healthy enough and would benefit from transplant. It also helps doctors know when people don’t have access to transplant and understand why.
| Summary (PDF) | Important Points | Full Article |
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| Transplant centers should help patients find care after BMT | The Caregiver Paradigm in Hematopoietic Cell Transplant: Current and Future Directions | |
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White patients more likely to join clinical trials, get transplant |
Black, American Indian, Asian, Hispanic and others under-represented | Representativeness of Blood and Marrow Transplant Clinical Trials Network (BMT CTN) Trial Participants |
| Fewer transplants if people are uninsured, have Medicare, or are Black | Utilization of Autologous Hematopoietic Cell Transplantation Over Time in Multiple Myeloma: A Population-Based Study | |
| Programs help people get treated for blood cancers and disorders | Reducing barriers of access and care related to hematopoietic cell transplantation and cellular therapy: The mission-driven role of the National Marrow Donor Program | |
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Doctors look for ways to help more people get lifesaving care |
Blood or marrow transplant (BMT) and other cellular therapies can cure sickle cell disease, blood cancers such as leukemia and lymphoma, and more | The ASTCT-NMDP ACCESS Initiative: A Collaboration to Address and Sustain Equal Outcomes for All across the Hematopoietic Cell Transplantation and Cellular Therapy Ecosystem. |
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Medicaid does not cover many costs of transplant for people with sickle cell disease |
Survey shows more coverage needed for life-saving treatment | A qualitative analysis of state Medicaid coverage benefits for allogeneic hematopoietic cell transplantation (alloHCT) for patients with sickle cell disease (SCD). |
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New treatment may help more people have a safe match for blood and marrow transplant |
Use of cyclophosphamide after BMT expands access for people of color | A National Marrow Donor Program Sponsored Multi-Center, Phase II Trial of HLA-Mismatched Unrelated Donor Bone Marrow Transplantation Using Post-Transplant Cyclophosphamide. |
| BMT helps some older people who have lymphoma | Scientists say Medicare should pay for BMT for people older than 65 who have non-Hodgkin lymphoma (NHL). | Outcomes of Medicare-age eligible NHL patients receiving RIC allogeneic transplantation: A CIBMTR analysis. |
| Auto transplants are safe and effective for people with multiple myeloma and damaged kidneys |
Auto transplants are safe and work equally well in people with multiple myeloma and damaged kidneys. The treatment can help some people stop dialysis treatments. Higher doses of melphalan can improve results. |
Autologous hematopoietic cell transplantation for multiple myeloma patients with renal insufficiency: A Center for International Blood and Marrow Transplant Research analysis |
| More older people with non-Hodgkin lymphoma get transplant | Transplant is a treatment option for healthy older people with NHL. | Allogeneic hematopoietic cell transplantation as curative therapy for patients with non-Hodgkin lymphoma: Increasingly successful application to older patients |
| Most patients who need an allo transplant can Find a well matched donor |
Most patients can find a well matched or a very well matched available adult donor in the Be The Match Registry. It is important to find the best donor as soon as possible, so the transplant isn't delayed. If a very well matched donor isn't available, it is better to use a well matched donor than to wait to try to find a very well matched one. |
HLA match likelihoods for hematopoietic stem-cell grafts in the U.S. registry |
| A quicker transplant with the best donor available is often the better choice | Patients may have better transplant results if they have the transplant sooner with the best donor available, rather than wait for a perfectly-matched donor while their cancer progresses. | High-resolution donor-recipient HLA matching contributes to the success of unrelated donor marrow transplantation |
| Access to bone marrow and stem cell transplant differs among different groups |
Transplants are used more often to treat leukemia, lymphoma, and multiple myeloma in Whites than in Blacks. Men get transplants more often than women. |
Access to hematopoietic stem cell transplantation: Effect of race and sex Race and outcomes of autologous hematopoietic cell transplantation for multiple myeloma |
New Therapies: CAR T-cells, Gene Therapy, and More
Now, doctors can modify a person’s cells to fight cancer. Chimeric antigen receptor (CAR) T-cells treat some types of leukemia and lymphoma. Also, doctors are studying gene therapies for sickle cell disease, beta thalassemia and hemophilia.
| Summary (PDF) | Important Points | Full Article |
|---|---|---|
| Study included adults whose lymphoma continued after 2 kinds of treatment | Autologous transplant versus chimeric antigen receptor T-cell therapy for relapsed DLBCL in partial remission. | |
| Large study of tisagenlecleucel shows it helps hard-to-treat cases | Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma. | |
| Chimeric antigen receptor T cells, or CAR-Ts, are a new type of cellular therapy | Indications for Hematopoietic Cell Transplantation and Immune Effector Cell Therapy: Guidelines from the American Society for Transplantation and Cellular Therapy | |
| Medicine for COVID-19 does not raise risk of other infections | Tocilizumab is already used in people with cancer after CAR T-cell therapy | Tocilizumab Not Associated With Increased Infection Risk After CAR T - Implications for COVID-19? |
Donor Health and Safety
Doctors are committed to making stem cell donation as safe as possible. More than 99% of donors experience a full recovery.
| Summary (PDF) | Important Points | Full Article |
|---|---|---|
| 7-year study of filgrastim shows it does NOT cause blood disorders | Long-term Outcomes of Peripheral Blood Stem Cell Unrelated Donors Mobilized with Filgrastim | |
| Donating blood-forming cells twice is safe for donors |
Your doctor can help you feel well during a second donation. Donating twice is rare. |
Donor experiences of second marrow or peripheral blood stem cell collection mirror the first, but CD34+ yields are less |
| After donating blood-forming cells, older people report similar quality of life as younger people |
Siblings 60 and older can donate blood-forming cells. Older donors have similar quality of life after donation as younger donors. |
Health-related quality of life among older related hematopoietic stem cell donors (>60 years) is equivalent to that of younger related donors (18 to 60 years): A related donor safety study |
| Some children who donate cells to a brother or a sister have a lower quality of life and need extra support |
About 20% of donors (1 in 5) at each time point had very poor QOL. Parents tended to overestimate their child's QOL. Younger children were more likely to have a lower QOL. |
Health-related quality of life among pediatric hematopoietic stem cell donors |
| How to decide whether a child can safely donate blood-forming cells to a family member | Experts make recommendations to help doctors and parents protect children who might donate blood-forming cells to a sick family member. | Determination of eligibility in related pediatric hematopoietic cell donors: ethical and clinical considerations. Recommendations from a working group of the Worldwide Network for Blood and Marrow Transplantation Association |
| Bone marrow donors have more side effects at hospitals that do fewer bone marrow collections | Bone marrow donors had more side effects at hospitals that did fewer bone marrow collections. | An analysis of the effect of race, socioeconomic status and center size on unrelated NMDP donor outcomes: Donor toxicities are more common at low volume bone marrow collection centers |
| Very few donors have severe side effects from donation |
Less than 1% of donors have severe side effects from donation. Bone marrow donors have severe side effects 3 times more often than blood stem cell donors. Blood stem cell donors who get G-CSF do NOT get cancer, an autoimmune illness, or a stroke more often than those who don't get G-CSF. |
Lower risk of serious adverse events and no increased risk of cancer after PBSC versus bone marrow donation |
Donor Selection
Doctors always try to find the best donor for a person who needs transplant. Research helps doctors know which type of person is best: related vs. unrelated, very well matched vs. half matched, older vs. younger. Research also helps doctors know which stem cell source is best: bone marrow vs. peripheral blood vs. cord blood.
| Summary (PDF) | Important Points | Full Article |
|---|---|---|
| Patients unlikely to have a fully matched donor should use alternates | Primary Results from BMT CTN 1702 - Clinical Transplant-related Long-term Outcomes of Alternative Donor Allogeneic Transplantation | |
| Black or African American children may have more side effects | Impact of Race and Ethnicity on Outcomes After Umbilical Cord Blood Transplantation | |
| New treatment combination helps children, adults of all races and ethnicities | Haploidentical bone marrow transplantation in patients with relapsed or refractory severe aplastic anaemia in the USA (BMT CTN 1502): a multicentre, single-arm, phase 2 trial | |
| Some people who donate blood or marrow cells twice in 1 year may not be able to give as many cells the second time. | Shorter interdonation interval contributes to lower cell counts in subsequent stem cell donations | |
| Study shows half-matched and cord blood transplants are acceptable | Optimal Donor for African Americans with Hematologic Malignancy: HLA-Haploidentical Relative or Umbilical Cord Blood Transplant | |
| Haploidentical donors slightly better than cord blood in some cases | Double unrelated umbilical cord blood versus HLA-haploidentical bone marrow transplantation (BMT CTN 1101) | |
| Unlicensed cord blood transplants can help people of color | Umbilical cord blood is safe, effective treatment for many diseases | Unlicensed umbilical cord blood units provide a safe and effective graft source for a diverse population: A study of 2456 umbilical cord blood recipients |
| For some people older than 50, transplant from young, matched donors is better than half-matched donors | Study looked at ages and types of donors to treat people with acute leukemia | Alternative donor transplantation for acute myeloid leukemia in patients aged ≥50 years: Young HLA-matched unrelated or haploidentical donor? |
| A transplant from an unrelated donor can treat acute lymphoblastic leukemia |
New research may make it possible for more people with acute lymphoblastic leukemia (ALL) to get life-saving transplants. New research shows that a transplant from an unrelated donor can help people with ALL live equally long as a transplant from a sibling. |
Comparing outcomes of matched related donor and matched unrelated donor hematopoietic cell transplants in adults with B-cell acute lymphoblastic leukemia |
| Transplant may be better when it uses bone marrow compared to peripheral blood stem cells from unrelated donors |
At 5 years after transplant from an unrelated donor, people who got bone marrow had better quality of life than people who got PBSC. People who got bone marrow: -Were more likely to be back at work. |
Comparison of patient-reported outcomes in 5-year survivors who received bone marrow vs peripheral blood unrelated donor transplantation: Long-term follow-up of a randomized clinical trial |
| Comparing 2 types of blood and marrow transplant: Double cord blood and haplo-cord |
For both types of transplant, about half of patients were alive 1 year after transplant. Patients who had a haplo-cord transplant had less GVHD, and the disease came back less often. |
Reduced intensity haplo plus single cord transplant compared to double cord transplant: Improved engraftment and graft-versus-host disease-free, relapse-free survival |
| Younger unrelated donors are better for transplant patients | The best unrelated donors are aged 18-32 years old and closely HLA-matched to the patient. | The effect of donor characteristics on survival after unrelated donor transplantation for hematologic malignancy |
| Half-matched (haploidentical) transplant for patients with lymphoma |
Patients who had a haploidentical or unrelated donor transplant had about the same 3-year survival. Patients who had a haploidentical transplant had less chronic GVHD. |
Reduced-intensity transplantation for lymphomas using haploidentical related donors versus HLA-matched unrelated |
| Most patients who need an allo transplant can find a well matched donor |
Most patients can find a well matched or a very well matched available adult donor in the Be The Match Registry. It is important to find the best donor as soon as possible, so the transplant isn't delayed. If a very well matched donor isn't available, it is better to use a well matched donor than to wait to try to find a very well matched one. |
HLA match likelihoods for hematopoietic stem-cell grafts in the U.S. registry |
| Younger sibling donors better for children and teens needing transplants |
Children and teen patients who get transplants from their younger siblings get less GVHD than patients who get transplants from their older siblings. For adult patients, it makes no difference if a sibling donor is younger or older. |
Birth order and transplantation outcome in HLA-identical sibling stem cell transplantations: An analysis on behalf of the Center for International Blood and Marrow Transplantation |
| SNPs - New information for matching patients and donors for transplant |
Many complications of HLA-mismatched transplantation are the result of genes that are not currently tested in the pre-transplant evaluation process of donors and patients. Scientists use SNPs, which are very small changes in the DNA genetic code, to locate unknown genes. Some SNPs are associated with better results after transplantation than other SNPs. |
Mapping MHC haplotype effects in unrelated donor hematopoietic cell transplantation |
| Similar survival for leukemia patients receiving transplants from related vs. unrelated donors | Patients with leukemia who get a transplant from a well matched unrelated donor or from an unrelated donor with a single mismatch should have similar survival to patients who have a related donor. | Outcomes after matched unrelated donor versus identical sibling hematopoietic cell transplantation in adults with acute myelogenous leukemia |
| Unrelated umbilical cord blood transplants equal to unrelated bone marrow in helping children with acute leukemia | Children with acute leukemia who receive unrelated umbilical-cord blood transplants have outcomes equal to - or in some cases, better than - those treated with bone marrow. | Outcomes of transplantation of unrelated donor umbilical cord blood and bone marrow in children with acute leukaemia: A comparison study |
| A quicker transplant with the best donor available is often the better choice | Patients may have better transplant results if they have the transplant sooner with the best donor available, rather than wait for a perfectly-matched donor while their cancer progresses. | High-resolution donor-recipient HLA matching contributes to the success of unrelated donor marrow transplantation |
HLA Matching and Biologic Factors
HLA matching is used to match patients and donors for transplant. Usually a very close match is best. But sometimes a half-matched transplant is best. Research helps doctors know which match is best.
| Summary (PDF) | Important Points | Full Article |
|---|---|---|
|
Partially matched blood stem cells transplant is safe and effective |
Newer medicines prevent most serious side effects | Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis After Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation |
| Younger, mismatched unrelated donors can help close the gap | Expanding donor options: haploidentical transplant recipients also highly likely to have a 7/8 matched unrelated donor | |
| Fully matched donors still the first choice for blood and marrow transplant | Umbilical cord blood or HLA-haploidentical transplantation: Real-world outcomes versus randomized trial outcomes | |
| Half-matched donors are acceptable if no match is found | HLA-haploidentical vs matched unrelated donor transplants with posttransplant cyclophosphamide-based prophylaxis. | |
| Quick testing for a blood or marrow donor helps people with leukemia | People who had fast-tracked tests (cytogenetics and HLA-typing) were more likely to get a BMT. | Rapid donor identification improves survival in high-risk first-remission patients with acute myeloid leukemia. |
| Transplant from half-matched donors may be as good as matched donors for people with lymphoma |
People were just as likely to be alive 3 years after transplant whether they had a matched sibling transplant or a haploidentical transplant. Fewer people got GVHD after a haploidentical transplant. |
Reduced-intensity transplantation for lymphomas using haploidentical related donors versus HLA-matched sibling donors: A Center for International Blood and Marrow Transplant Research analysis |
Late Effects and Quality of Life
A transplant can have a long-term impact on a person’s health and wellbeing. Late effects are health problems that can happen months or years after treatment. For example, treatment can affect fertility, physical and emotional health, and risk of other cancers.
| Summary (PDF) | Important Points | Full Article |
|---|---|---|
| People who get a solid organ transplant followed by blood or marrow transplant may have serious problems | Risk of organ failure is higher after BMT | Risk Factors for Solid Organ Graft Failure and Death in Solid Organ Transplant Recipients Undergoing Hematopoietic Cell Transplantation: A Retrospective Center for International Blood and Marrow Transplant Research (CIBMTR) and Organ Procurement and Transplantation Network (OPTN) Study |
| Patients can help researchers design studies | Patient engagement in hematopoietic stem cell transplantation and cell therapy: a survey by the EBMT patient engagement task force & transplantation complications working party | |
| A patient-reported outcome (PRO) is any report of a patient’s health condition that comes directly from the patient, without interpretation by a doctor or anyone else. | Current Trends and Outcomes in Cellular Therapy Activity in the United States, Including Prospective Patient-Reported Outcomes Data Collection in the Center for International Blood and Marrow Transplant Research Registry | |
| Side effects may affect physical and emotional health for years | International Recommendations for Screening and Preventative Practices for Long-Term Survivors of Transplantation and Cellular Therapy: A 2023 Update. | |
| Quality of life. | Incorporating patient-reported outcome data into a predictive calculator for allogeneic hematopoietic cell transplantation recipients. | |
| Patients and caregivers have useful advice | Bringing Patient and Caregivers Voices to the Clinical Trial Chorus: A Report From the BMT CTN Patient and Caregiver Advocacy Task Force | |
|
People aged 60 and older generally have low distress after BMT |
A year after blood or marrow transplant, most cancer survivors have low levels of physical and mental distress. | Health-Related Quality of Life Outcomes in Older Hematopoietic Cell Transplantation Survivors. Transplantation and Cellular Therapy. |
| People aged 18 to 39 have lower quality of life after blood or marrow transplant | Health-Related Quality of Life in Young Adult Survivors of Hematopoietic Cell Transplantation | |
| Correct diagnosis is key to helping patients feel better | Acute GVHD diagnosis and adjudication in a multicenter trial: A report from the BMT CTN 1202 Biorepository Study. | |
| Check-ups and treatments helpful after allogeneic blood or marrow transplant | Male-specific late effects in adult hematopoietic cell transplantation recipients: a systematic review from the Late Effects and Quality of Life Working Committee of the Center for International Blood and Marrow Transplant Research and Transplant Complications Working Party of the European Society of Blood and Marrow Transplantation. | |
| More than 90% of patients and caregivers strongly agreed or agreed that the care guides helped them understand recommended tests at check-up appointments. | Application and evaluation of survivorship care guides for hematopoietic cell transplantation recipients. | |
| In older adults, memory problems may be linked with health after BMT | Geriatric assessment in older alloHCT recipients: Association of functional and cognitive impairment with outcomes. | |
| 87% of transplant centers say return-to-work programs would help patients | Return-to-work guidelines and programs for post-hematopoietic cell transplant survivors: An initial survey. | |
| Teens and young adults need check-ups for side effects, even years after leukemia treatment | Blood or marrow transplant cures leukemia but may have late side effects | Late effects after ablative allogeneic stem cell transplantation for adolescent and young adult acute myeloid leukemia. |
| In year after blood or marrow transplant, 6-8% regret it | Regret was more likely if cancer returned or if the patient felt less support from family and friends prior to transplant | Prevalence of decisional regret among patients who underwent allogeneic hematopoietic stem cell transplantation and associations with quality of life and clinical outcomes. |
| Patients, families and caregivers need transplant info | Blood and marrow transplant (BMT) is a highly complex treatment that can affect health for years afterward. | A conceptual framework and key research questions in educational needs of blood and marrow transplant patients, caregivers and families. |
| Survivorship care plans help transplant survivors | Survivorship care plans helped survivors of blood or marrow transplant have a better quality of life, according to a study. | Randomized controlled trial of individualized treatment summary and survivorship care plans for hematopoietic cell transplantation survivors. |
| Tailoring care plans for life long after transplant | A survivorship care plan includes possible health issues that can happen months and years after BMT plus ways to watch for and prevent them. | Tailoring a survivorship care plan: patient and provider preferences for recipients of hematopoietic cell transplantation. |
| Caregivers and patients may have PTSD after transplant | More caregivers than recipients reported symptoms of post-traumatic stress disorder (PTSD). PTSD can be treated. | Rates and risk factors for post-traumatic stress disorder symptomatology among adult hematopoietic cell transplant recipients and their informal caregivers. |
| Childhood transplants linked to slightly higher risk of heart problems and diabetes | Scientists urge checkups for cholesterol and blood pressure. | Late cardiovascular morbidity and mortality following pediatric allogeneic hematopoietic cell transplantation |
| Quick survey checks your quality of life | Talk with your doctor about how you are feeling physically and emotionally. | PROMIS measures can be used to assess symptoms and function in long-term hematopoietic cell transplantation survivors |
| After transplant, some people have problems focusing | Cognitive changes after BMT may include altered attention, focus, learning, memory and thinking. Your doctor can help. |
Neurocognitive dysfunction in hematopoietic cell transplant recipients: Expert review from the Late Effects and Quality of Life Working Committee of the CIBMTR and Complications and Quality of Life Working party of the EBMT |
| Common late effects after transplant in very young children |
30% of very young BMT recipients have organ damage or other late effects. Common late effects are delayed growth, cataracts, and hypothyroidism. Full-body radiation increases the chance of getting these late effects. |
Survival and late effects after allogeneic hematopoietic cell transplantation for hematologic malignancy at less than three years of age |
| Fertility preservation options before transplant | Tell your doctor right away if you want to talk about fertility preservation. | Clinical guide to fertility preservation in hematopoietic cell transplant recipients |
| Medical guidelines for survivors of stem cell transplantation | This research article describes best practices for follow-up, prevention, and screening of late complications. | Recommended screening and preventive practices for long-term survivors after hematopoietic cell transplantation |
| Long-term survival after transplantation |
Patients are more likely to survive long-term after transplant if: |
Long-term survival and late deaths after allogeneic hematopoietic cell transplantation |
| Spouses, partners share mental distress of cancer | Spouses of cancer patients suffer distress and depression at levels equal to - or greater than - their partners. | Late effects of cancer and hematopoietic stem-cell transplantation on spouses or partners compared with survivors and survivor-matched controls |
| Improving health for long‐term survivors of cancer and transplants | Bone marrow or stem cell transplant survivors should actively protect their health. This can help speed up their recovery and improve the quality of their life. It can also lower their risk of developing health problems in later years. |
The preventive health behaviors of long-term survivors of cancer and hematopoietic stem cell transplantation compared with matched controls |
Social and Financial Aspects of Transplant
Transplant affects more than a person’s physical health. It affects emotional health, ability to work, and finances. Transplant also affects more than the patient. It affects the entire family.
| Summary (PDF) | Important Points | Full Article |
|---|---|---|
|
1 year after transplant or cell therapy, patients’ social lives return |
Social functioning returns to same levels as before treatment | Social Functioning after Transplantation and Cellular Therapy: Initial Patient-Reported Outcomes Results from the CIBMTR. Poster presentation at Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR; 2023-02-16. |
|
Support from family and friends helps patients at cellular level |
Social support affects the immune system in transplant | Unfavorable transcriptome profiles and social disadvantage in hematopoietic cell transplantation: a CIBMTR analysis. |
| Blood or marrow transplant helps people with lymphoma, leukemia | Cost-Effectiveness of Unrelated Umbilical Cord Blood Transplantation versus HLA-Haploidentical Related Bone Marrow Transplantation: Evidence from BMT CTN 1101 | |
| 3 years after blood or marrow transplant, 60% of young adults are working | Return to work among young adult survivors of allogeneic hematopoietic cell transplantation in the United States. | |
| 87% of transplant centers say return-to-work programs would help patients | Return-to-work guidelines and programs for post-hematopoietic cell transplant survivors: An initial survey. | |
| BMT in childhood linked with higher jobless rate in adulthood | In the U.S., survivors had unemployment rates of about 15%. Survivors who had BMT outside the U.S. had even higher rates. | Post‐transplantation employment status of adult survivors of childhood allogeneic hematopoietic cell transplant: A report from the Center for International Blood and Marrow Transplant Research (CIBMTR). |
| Caregivers and patients may have PTSD after transplant | More caregivers than recipients reported symptoms of post-traumatic stress disorder (PTSD). PTSD can be treated. | Rates and risk factors for post-traumatic stress disorder symptomatology among adult hematopoietic cell transplant recipients and their informal caregivers. |
| New consent form helps people talk about treatment options | Easy-to-read consent form leads to better discussions between people and their doctors. | Easy-to-Read Informed Consent Form for Hematopoietic Cell Transplantation Clinical Trials: Results from Blood and Marrow Transplant Clinical Trials Network 1205 Trial |
| Transplant often affects family finances |
At 2 years after transplant: -Most patients and caregivers thought transplant affected their family finances a medium or large amount. |
Financial impact of allogeneic hematopoietic cell transplantation on patients and families over 2 years: Results from a multicenter pilot study |
| A survey that asks patients about their physical health before transplant may predict how they’ll do after transplant |
How patients feel physically before and after transplant affects transplant outcomes. |
Patient-reported physical functioning predicts the success of hematopoietic cell transplantation (BMT CTN 0902) |
| Spouses, partners share mental distress of cancer | Spouses of cancer patients suffer distress and depression at levels equal to - or greater than - their partners. | Late effects of cancer and hematopoietic stem-cell transplantation on spouses or partners compared with survivors and survivor-matched controls |
Trends in Transplant
Transplant treatment and outcomes change over time as doctors learn more about what works best.
| Summary (PDF) | Important Points | Full Article |
|---|---|---|
|
Transplant patients should get vaccinated for COVID-19 early |
Shots helpful even when given in the first 4 months after blood or marrow transplant | SARS-CoV-2 vaccination in the first year after hematopoietic cell transplant or chimeric antigen receptor T cell therapy: A prospective, multicenter, observational study |
| 1 year after BMT, it’s not clear whether the vaccine stops cancer from worsening | Randomized Trial of a Personalized Dendritic Cell Vaccine after Autologous Stem Cell Transplant for Multiple Myeloma | |
| Newer treatment for people who have leukemia, lymphoma, other blood cancers | Three-Year Outcomes in Recipients of Mismatched Unrelated Bone Marrow Donor Transplants Using Post-Transplantation Cyclophosphamide: Follow-Up from a National Marrow Donor Program-Sponsored Prospective Clinical Trial. Transplantation and Cellular Therapy. | |
| Ideal circumstances were most common for Japanese patients | Race and survival in unrelated hematopoietic cell transplantation | |
| Blood and marrow transplant is underused in people older than 60 | Physicians’ perceptions on candidacy for allogeneic hematopoietic cell transplantation in older adults. | |
| Be The Match Registry overcomes COVID-19 challenges | Meeting the Demand for Unrelated Donors in the Midst of the COVID-19 Pandemic: Rapid Adaptations by the National Marrow Donor Program and Its Network Partners Ensured a Safe Supply of Donor Products | |
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People who had a blood or marrow transplant (BMT) have higher risk dying from COVID-19 |
Largest study so far shows that most patients survive | Clinical characteristics and outcomes of COVID-19 in haematopoietic stem cell transplantation recipients |
| Experts advise new safety measures for patients and donors | Real-World Issues and Potential Solutions in Hematopoietic Cell Transplantation during the COVID-19 Pandemic: Perspectives from the Worldwide Network for Blood and Marrow Transplantation and Center for International Blood and Marrow Transplant Research Health Services and International Studies Committee | |
| For people with aplastic anemia, fresh cells rather than frozen are better for blood and marrow transplant | During the pandemic, the National Marrow Donor Program (NMDP) / Be The Match urges doctors to use frozen cells. However, people with aplastic anemia can still receive fresh cells. | Hematopoietic cell transplantation with cryopreserved grafts for severe aplastic anemia |
| Freezing cells for blood and marrow transplant saves lives | Study helps people with cancer get life-saving transplants during pandemic; people who got fresh or frozen cells were equally likely to be alive and recovering. |
Graft cryopreservation does not impact overall survival allogeneic hematopoietic cell transplantation using post-transplant cyclophosphamide for GVHD prophylaxis. |
| More radiation not necessarily better | Irradiation therapy helps most people with leukemia before blood or marrow transplant | Comparison of high doses of total body irradiation in myeloablative conditioning prior to hematopoietic cell transplantation |
| Intense treatment before transplant linked to infections | More people with leukemia who had intense treatment just before blood or marrow transplant (BMT) got a bacterial infection, compared to people who had less intense treatment | Increased overall and bacterial infections following myeloablative allogeneic HCT for patients with AML in CR1 |
| Better transplant strategies needed for babies | Transplant may cure disease but has side effects | Survival trends in infants undergoing allogeneic hematopoietic cell transplant. |
| Doctors say transplants treat systemic sclerosis or scleroderma | Autologous transplants help people with systemic sclerosis live longer and improve their skin, lungs and quality of life. | Systemic sclerosis as an indication for autologous hematopoietic cell transplantation: position statement from the American Society for Blood and Marrow Transplantation |
| Online tool helps predict liver problems after transplant prep | Researchers at CIBMTR created a free, online VOD Risk Calculator for doctors. Veno-occlusive disease (VOD) is a rare liver problem that may happen after preparation for blood or marrow transplant (BMT). |
Risk score for development of veno-occlusive disease after allogeneic hematopoietic cell transplant. |
| Treatment helps children and teens with acute leukemia thrive after cord blood transplants | The TCF treatment regimen before cord blood transplant helped young people with acute leukemia live longer. | Umbilical cord blood transplantation in children with acute leukemia: Impact of conditioning on transplantation outcomes |
| The number of allogeneic transplants for people age 70 years and older are increasing, and outcomes are improving |
More older people got BMT between 2000 and 2013. BMT results for older people have gotten better between 2000 and 2013. |
Increasing use of allogeneic hematopoietic cell transplantation in patients aged 70 years and older in the United States |
| Better survival over time for patients with acute GVHD |
Treatments for patients with moderate to severe acute GVHD have gotten better over time. Tacrolimus helped patients with moderate acute GVHD live longer. |
Improved survival after acute graft vs. host disease diagnosis in the modern era |