Transplant helps teens, young adults with sickle cell disease

Longer, larger studies are needed to show best treatment 

A small study showed blood or marrow transplant (BMT) is one good option for treating sickle cell disease. 

Sickle cell disease is an inherited disease or passed down from parents to children. Normal red blood cells are round and flexible. But in sickle cell disease, the cells are stiff and can block the flow of blood. These stiff sickle cells cause episodes of severe pain and can harm the brain, causing strokes. 

BMT can cure sickle cell disease but can have serious effects. 

The standard care for sickle cell disease includes medicines and blood transfusions. These help with short-term relief. People get the standard care more often than BMT. Gene therapy is another option now approved for sickle cell disease. But, very few persons have received gene therapy. 

Researchers wanted to compare standard care versus BMT. About 110 patients with sickle cell disease joined a clinical trial, starting in 2016. Everyone was aged 15-40. Patients who had a matched BMT donor got BMT; patients with no donor got standard care. 

The trial ended early because not enough patients joined, and also the COVID-19 pandemic prevented patients from coming for treatment. 

About 2 years after treatments started, about 9 out of 10 patients were alive, whether they got standard care or matched BMT. However, the trial was not able to determine which treatment helped people live longer. 

Compared to patients who got standard care, the patients who got BMT: 

• Had fewer episodes of severe pain 

• Felt less tired 

• Were more able to be social with friends and family 

Keep In Mind

BMT sometimes has serious effects, such as graft-versus-host disease (GVHD) and organ damage. 

This study was smaller and shorter than planned. Larger, longer studies are needed to show which treatment is best. 

Learn More About

• Sickle cell disease at NMDP.org 

• Clinical trials for sickle cell disease at CTsearchsupport.org 

• More study summaries at CIBMTR.org 

Source

Walters MC, Eapen M, Liu Y, et al. Hematopoietic Cell Transplant compared with Standard Care in Adolescents and Young Adults with Sickle Cell Disease. Blood Advances. 2024. Epub 2024 Oct 29. doi: 10.1182/bloodadvances.2024013926. 

Clinical Trial IDs

BMT CTN 1503; ClinicalTrials.gov NCT02766465

Sponsor

Blood and Marrow Transplant Clinical Trials Network® (BMT CTN®) BMT CTN thanks study participants. BMT CTN, funded by the National Heart, Lung, and Blood Institute and by the National Cancer Institute, is a collaborative effort of 20 core transplant centers/Consortia, the Center for International Blood and Marrow Transplant Research (CIBMTR), NMDP, and The Emmes Company. CIBMTR is a research collaboration of the Medical College of Wisconsin (MCW) and NMDP. You may contact CIBMTR, MCW, Clinical Cancer Center, 9200 W. Wisconsin Ave., Suite C5500, Milwaukee, WI 53226, 414-805-0700.