Bone marrow transplantation (stem cell transplant) replaces diseased blood-forming cells with healthy donor or patient-derived stem cells and is the only curative option for many blood cancers and bone marrow failure conditions. Research focuses on reducing toxicity, expanding eligibility, and improving outcomes in patients who relapse after transplant.
What's actually going on in research
Haploidentical transplant — using half-matched family donors — has expanded access dramatically, and post-transplant cyclophosphamide has made it nearly as safe as matched unrelated donor transplant. CAR-T cell therapy before transplant is improving remission depth and post-transplant relapse prevention. Reduced-intensity conditioning regimens allow older and medically fragile patients to receive transplants that would have been prohibitively toxic a decade ago.
Haplo-identical transplant
Half-matched family donors are now a viable option using post-transplant cyclophosphamide, expanding access to patients without matched siblings or unrelated donors in the registry.
Reduced-intensity conditioning
Gentler pre-transplant regimens allow patients in their 60s and 70s and those with organ dysfunction to receive transplants, with trials comparing regimen intensity and outcomes.
Post-transplant maintenance
Azacitidine, targeted drugs, and immune checkpoint inhibitors are being tested as maintenance therapy after transplant to prevent relapse in high-risk blood cancers.
What to know before you search
Eligibility depends on underlying disease and remission status, donor availability, organ function, age, and performance status.
What types of trials are currently open
- Conditioning regimen trials — Comparing myeloablative and reduced-intensity conditioning regimens for safety and relapse prevention.
- Donor type trials — Comparing haploidentical, matched sibling, unrelated, and cord blood donor outcomes.
- GVHD prevention trials — Testing novel prophylaxis approaches to reduce acute and chronic graft-versus-host disease.
- Post-transplant therapy trials — Evaluating maintenance drugs after transplant to lower relapse risk.
- Long-term outcomes studies — Tracking late effects, quality of life, and immune reconstitution years after transplant.
Recently added Bone Marrow Transplantation trials
Low-Dose ATG/PTCy Plus Ivarmacitinib for aGVHD Prevention in Haplo-PBSCT From Parous Female Donors
Graft-versus-host disease (GVHD) is a major complication after allogeneic hematopoietic stem cell transplantation (allo-HSCT), significantly affecting survival and quality of life. Acute GVHD (aGVHD) typically occurs within 100 days post-transplant, commonly involving skin, gastrointestinal tract, and liver. Chronic GVHD (cGVHD) can appear months to years later. Despite prophylaxis with calcineurin inhibitors (e.g., cyclosporine or tacrolimus), methotrexate, mycophenolate mofetil, and post-transplant cyclophosphamide (PTCy), patients receiving haploidentical transplantation from parous female donors remain at high risk for moderate-to-severe aGVHD. JAK1-dependent cytokine signaling (IL-6, IFN-γ) is central to GVHD pathogenesis. Selective JAK1 inhibition may attenuate T cell-mediated inflammation while preserving hematopoiesis. Ivarmacitinib (SHR0302) is a highly selective oral JAK1 inhibitor, showing favorable safety and preliminary efficacy in autoimmune and GVHD settings, making it a candidate for early GVHD prophylaxis.
Cord Blood Transplantation in Children and Young Adults With Blood Cancer
The purpose of this study is to find out whether Cord Blood Transplantation/CBT as the first or second transplant is an effective treatment for children and young adults with blood cancer.
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