Bone marrow transplantation, also called hematopoietic stem cell transplantation, replaces damaged or diseased bone marrow with healthy stem cells. It's used to treat blood cancers like leukemia and lymphoma, inherited blood disorders like sickle cell disease, and immune system conditions. The procedure has become safer over the past two decades, with better donor matching, infection control, and management of graft-versus-host disease.
What's actually going on in research
Trials are testing ways to make transplant safer and more accessible — expanding donor options through half-matched family members, reducing transplant-related complications, and improving outcomes for older patients. Studies are examining CAR T-cell therapy as an alternative to transplant in some blood cancers, post-transplant maintenance drugs to prevent relapse, and new approaches to graft-versus-host disease that preserve the transplant's anti-cancer effect while reducing tissue damage.
Haploidentical transplant
Using half-matched family donors makes transplant possible for patients who lack a fully matched donor. New techniques using post-transplant cyclophosphamide have made these transplants nearly as successful as matched transplants.
GVHD prevention
Trials are testing drugs like abatacept and ruxolitinib given early after transplant to prevent graft-versus-host disease. The goal is to reduce GVHD without eliminating the transplant's ability to fight cancer.
Reduced-intensity conditioning
Lower doses of chemotherapy before transplant allow older patients and those with other health conditions to undergo the procedure. Studies are refining which patients benefit most from this approach.
What to know before you search
Eligibility depends on the underlying disease, disease status at time of transplant, prior treatments, organ function, and availability of a suitable donor.
What types of trials are currently open
- Conditioning regimen trials — Testing different chemotherapy or radiation doses given before transplant to prepare the body for donor cells while minimizing toxicity.
- GVHD prevention trials — Testing drugs or cell manipulation techniques to prevent graft-versus-host disease while preserving the transplant's anti-cancer effect.
- Donor source trials — Comparing outcomes using different donor types — matched siblings, unrelated donors, half-matched family members, or cord blood.
- Post-transplant maintenance — Testing drugs given after transplant to prevent cancer relapse, particularly in high-risk leukemia and myeloma.
- Supportive care trials — Testing treatments for transplant complications like infections, organ damage, and late effects on quality of life.
Recently added Bone Marrow Transplantation trials
A Study of LBP-MARCO and Ruxolitinib in People With Graft-Versus-Host Disease (GVHD)
The researchers are doing this study to find out whether the combination of LBP-MARCO (Microbiota Augmentation to Reestablish Commensal Organisms) and ruxolitinib is a safe treatment for people with graft-versus-host disease (GVHD) after receiving a stem cell (allogeneic hematopoietic cell transplantation).
An Exploratory Study on the Use of Gecacitinib in Patients With Myeloid Malignancies and Myelofibrosis During the Peri-transplant Period Following Allogeneic Hematopoietic Stem Cell Transplantation
This is a single-arm, single-center exploratory study evaluating the safety and efficacy of gecacitinib administered in the peri-transplant period in patients with myeloid neoplasms and myelofibrosis (MF) who are scheduled to undergo allogeneic hematopoietic stem cell transplantation (allo-HSCT).Approximately 10 eligible patients will receive oral gecacitinib starting 13 weeks before transplantation. Treatment will continue through the peri-transplant period and for up to 24 weeks after hematopoietic reconstitution. The primary endpoint is the engraftment rate at day +28 post-transplant. Secondary endpoints include poor graft function rate, grade III-IV acute GVHD rate, overall survival, infection rate, and bone marrow fibrosis grade changes.
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