PRO Data Collection Protocol
CIBMTR collects longitudinal or cross-sectional PRO data from recipients of cellular therapies who are also enrolled in the Protocol for a Research Database for Hematopoietic Cell Transplantation, Other Cellular Therapies, and Marrow Toxic Injuries (Research Database protocol).
On this page:
Research Goals
The primary purpose of the PRO Protocol is to have a centralized collection of patient-reported symptoms and functioning to supplement traditional outcomes, such as survival or disease relapse, in registration and other studies. These data may be used in research to:
- Assess trajectories of patient symptom and functioning overtime
- Determine and evaluate symptoms and functioning that affect HCT and cellular therapy outcomes
- Assess long-term outcomes after HCT or cellular therapy
Core PRO Domains and Measures
STANDARD
| DOMAIN/CONTENT AREA | ADULT MEASURE | PEDIATRIC MEASURE |
|---|---|---|
| Physical Function | PROMIS Physical Function | PROMIS Mobility |
| Fatigue | PROMIS Fatigue | PROMIS Fatigue |
| Sleep Disturbance | PROMIS Sleep Disturbance | PROMIS Sleep Disturbance |
| Pain Interference | PROMIS Pain Interference | PROMIS Pain Interference |
| Anxiety | PROMIS Anxiety | PROMIS Anxiety |
| Depression | PROMIS Depression | PROMIS Depressive Symptoms |
| Cognitive Function | PROMIS Cognitive Function | PROMIS Cognitive Function |
| Ability to Participate in Social Roles and Activities/Peer Relationships | PROMIS Ability to Participate in Social Roles and Activities | PROMIS Family Relationships/Peer Relationships |
| Sexual Function | PROMIS Sexual Function | |
| Financial Toxicity | COmprehensive Score for Financial Toxicity (COST) | Parent/Guardian reported COST |
| Standard sociodemographics – Core Items | Core Items | Core Items |
| Standard sociodemographics – Expanded Sociodemographics | Expanded Sociodemographics | |
| Standard sociodemographics – Expanded Occupational Functioning | Expanded Occupational Functioning | Expanded Occupational Functioning |
| Standard sociodemographics – Expanded Social Determinants of Health (SDOH) | Expanded SDOH | Expanded SDOH |
| Standard sociodemographics – Caregiver Items | Caregiver Items |
Time Points
The time points for PRO data collection parallel the time points at which treatment centers submit clinical outcomes data from healthcare records, with an additional time point at 30 days post-treatment for cellular therapy patients. Below are the measures and time points collected for adult HCT and CAR T patients:
- Pre-infusion
- Day 30 (CAR T only)
- Day 100
- Day 180
- Year 1
- Annually
Protocol Enrollment
Presented here are the number of patients enrolled in the CIBMTR PRO Protocol by type and year of infusion, as of May 20, 2025.
| Characteristic | Allogeneic Hematopoietic Cell Transplantation | Autologous Hematopoietic Cell Transplantation | Cellular Therapy | Gene Therapy | Total |
|---|---|---|---|---|---|
| Total no. patients1 | 637 | 761 | 310 | 3 | 1711 |
| Year of Infusion - no. (%) | |||||
| 2025 | 12 (2) | 17 (2) | 42 (14) | 3 (100) | 74 (4) |
| 2024 | 120 (19) | 156 (20) | 120 (39) | 0 (0) | 396 (23) |
| 2023 | 131 (21) | 140 (18) | 90 (29) | 0 (0) | 361 (21) |
| 2022 | 221 (35) | 303 (40) | 45 (15) | 0 (0) | 569 (33) |
| 2021 | 120 (19) | 135 (18) | 12 (4) | 0 (0) | 267 (16) |
| 2020 | 31 (5) | 10 (1) | 1 (<1) | 0 (0) | 42 (2) |
| 2019 | 2 (<1) | 0 (0) | 0 (0) | 0 (0) | 2 (<1) |
111 HCTs not included in the table due to lack of infusion type information in FormsNet3.
PRO Data Collected
Presented here are the number of completed PRO surveys available for analysis by infusion-related time point, infusion type, age at transplant, gender, and indication, as of May 20, 2025.
Number (%) of patients who have survey completed at each time point
| Characteristic | Baseline | Day 30 | Day 100 | Day 180 | Year 1 | Year 2 | Year 3 | Year 4 | Total |
|---|---|---|---|---|---|---|---|---|---|
| No. of surveys1 | 337 | 170 | 1245 | 1284 | 995 | 632 | 281 | 69 | 5013 |
| No. of centers | 20 | 28 | 42 | 43 | 35 | 25 | 24 | 17 | 59 |
| Sex - no. (%) | |||||||||
| Male | 180 (53) | 109 (64) | 738 (59) | 753 (59) | 578 (58) | 363 (57) | 173 (62) | 39 (57) | 2933 (59) |
| Female | 140 (42) | 60 (35) | 504 (40) | 527 (41) | 415 (42) | 267 (42) | 108 (38) | 30 (43) | 20251 (41) |
| Not reported | 17 (5) | 1 (1) | 3 (<1) | 4 (<1) | 2 (<1) | 2 (<1) | 0 (0) | 0 (0) | 29 (1) |
| Infusion type - no. (%) | |||||||||
| Allo-HCT | 149 (44) | 0 (0) | 400 (32) | 332 (33) | 332 (33) | 223 (35) | 123 (44) | 48 (70) | 1704 (34) |
| Auto-HCT | 131 (39) | 0 (0) | 617 (50) | 637 (50) | 524 (53) | 347 (55) | 151 (54) | 20 (29) | 2427 (48) |
| Cellular therapy | 45 (13) | 169 (99) | 228 (18) | 217 (17) | 139 (14) | 62 (10) | 7 (2) | 1 (1) | 868 (17) |
| Gene therapy | 2 (1) | 1 (1) | 0 (0) | 0 (0) | 0 (0) | 0 (0) | 0 (0) | 0 (0) | 3 (<1) |
| HCT, type not reported | 10 (3) | 0 (0) | 0 (0) | 1 (<1) | 0 (0) | 0 (0) | 0 (0) | 0 (0) | 11 (<1) |
| Age at infusion - no. (%) | |||||||||
| 18-29 | 10 (3) | 7 (4) | 42 (3) | 46 (4) | 32 (3) | 20 (3) | 7 (2) | 6 (9) | 170 (3) |
| 30-39 | 18 (5) | 3 (2) | 65 (5) | 68 (5) | 53 (5) | 31 (5) | 15 (5) | 6 (9) | 259 (5) |
| 40-49 | 31 (9) | 11 (6) | 125 (10) | 123 (10) | 101 (10) | 60 (9) | 22 (8) | 7 (10) | 480 (10) |
| 50-59 | 72 (21) | 35 (21) | 292 (23) | 294 (23) | 235 (24) | 146 (23) | 58 (21) | 13 (19) | 1145 (23) |
| 60-69 | 129 (38) | 50 (29) | 466 (37) | 501 (39) | 390 (39) | 253 (40) | 133 (47) | 29 (42) | 1951 (39) |
| ≥70 | 60 (18) | 63 (37) | 252 (20) | 248 (19) | 182 (18) | 120 (19) | 46 (16) | 8 (12) | 979 (20) |
| Not reported | 17 (5) | 1 (1) | 3 (<1) | 4 (<1) | 2 (<1) | 2 (<1) | 0 (0) | 0 (0) | 29 (1) |
| Primary disease for which the infusion was performed - no. (%) | |||||||||
| Acute myeloid leukemia | 62 (18) | 0 (0) | 179 (14) | 196 (15) | 153 (15) | 95 (15) | 51 (18) | 33 (48) | 769 (15) |
| Acute lymphoblastic leukemia | 17 (5) | 0 (0) | 46 (4) | 47 (4) | 37 (4) | 24 (4) | 19 (7) | 6 (9) | 196 (4) |
| Other acute leukemia | 1 (<1) | 0 (0) | 6 (<1) | 7 (1) | 7 (1) | 5 (1) | 3 (1) | 1 (1) | 30 (1) |
| Chronic myeloid leukemia | 4 (1) | 0 (0) | 8 (1) | 10 (1) | 9 (1) | 6 (1) | 4 (1) | 1 (1) | 42 (1) |
| Myelodysplastic syndrome | 36 (11) | 0 (0) | 102 (8) | 103 (8) | 79 (8) | 58 (9) | 34 (12) | 6 (9) | 418 (8) |
| Myeloproliferative neoplasms | 12 (4) | 0 (0) | 23 (2) | 26 (2) | 20 (2) | 12 (2) | 4 (1) | 0 (0) | 97 (2) |
| Other leukemia | 2 (1) | 0 (0) | 5 (<1) | 6 (<1) | 2 (<1) | 4 (1) | 1 (<1) | 0 (0) | 20 (<1) |
| Hodgkin lymphoma | 7 (2) | 0 (0) | 32 (3) | 32 (2) | 26 (3) | 17 (3) | 6 (2) | 0 (0) | 120 (2) |
| Non-Hodgkin lymphoma | 77 (23) | 168 (99) | 350 (28) | 345 (27) | 245 (25) | 135 (21) | 45 (16) | 6 (9) | 1371 (27) |
| Multiple myeloma / plasma cell disorder | 89 (26) | 0 (0) | 466 (37) | 477 (37) | 388 (39) | 257 (41) | 110 (39) | 16 (23) | 1803 (36) |
| Other malignancies | 2 (1) | 0 (0) | 7 (1) | 7 (1) | 5 (1) | 2 (<1) | 1 (<1) | 0 (0) | 24 (<1) |
| Severe aplastic anemia | 4 (1) | 0 (0) | 7 (1) | 11 (1) | 10 (1) | 9 (1) | 2 (1) | 0 (0) | 43 (1) |
| Sickle cell disease | 0 (0) | 1 (1) | 0 (0) | 1 (<1) | 1 (<1) | 0 (0) | 0 (0) | 0 (0) | 3 (<1) |
| Paroxysmal nocturnal hemoglobinuria (PNH) | 0 (0) | 0 (0) | 1 (<1) | 1 (<1) | 1 (<1) | 0 (0) | 0 (0) | 0 (0) | 3 (<1) |
| Autoimmune diseases | 3 (1) | 0 (0) | 7 (1) | 9 (1) | 9 (1) | 5 (1) | 1 (<1) | 0 (0) | 34 (1) |
| Other non-malignant hematologic disorders | 2 (1) | 0 (0) | 1 (<1) | 2 (<1) | 1 (<1) | 1 (<1) | 0 (0) | 0 (0) | 7 (<1) |
| Not reported | 19 (6) | 1 (1) | 5 (<1) | 4 (<1) | 2 (<1) | 2 (<1) | 0 (0) | 0 (0) | 33 (1) |
110 year 5 surveys not included in the table
Recipient and Donor Participation
Any recipient of an unrelated or related HCT or CAR T in a CIBMTR-affiliated center may participate in the PRO protocol. Complete eligibility requirements are outlined in the study protocol.
If the recipient of HCT or cellular therapy does not consent to the Research Database protocol, they may still participate in the PRO protocol, but the use of their data will be limited to CIBMTR and NMDP process improvement analyses or study-specific analysis as laid out in the PRO protocol consent form.
CIBMTR's Survey Research Group identifies, approaches, and obtains consent from eligible recipients. Informed consent is documented in CIBMTR's electronic PRO (ePRO) system. The Survey Research Group centrally collects PRO surveys from participating recipients electronically, on paper, or by phone and follows up with non-responders.
PRO data are collected in the ePRO system and then stored in CIBMTR's database where they can be linked to CIBMTR clinical outcomes data.
IRB Requirements
The PRO protocol is a single-site CIBMTR protocol. All research activities, including enrollment and data collection, are centrally performed within CIBMTR. Centers do not need to submit the PRO protocol or consent form to their local IRB for their patients to participate.
CIBMTR-Approved Protocol and Consent Forms
CIBMTR, through the NMDP IRB, has approved the following protocol and consent form. These are provided for reference only. Centers should not submit these materials for local IRB approval. Centers should not use these materials to consent recipients.
PRO Protocol
PRO IRB Approvals
PRO Consent Forms
Protocol Team Contacts
- General mailbox: Surveys@cibmtr.org
- Elizabeth Fosberg, Clinical Project Manager: efosberg@nmdp.org
- Deborah Mattila, Survey Research Group Manager: dmattila@nmdp.org
- Rachel Cusatis, PhD, Scientific Director of Patient-Centered Research and Survivorship: rcusatis@mcw.edu