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
Ivosidenib as Maintenance Therapy in Transplant-Ineligible IDH1-mutated AML and HR-MDS
This study will explore the efficacy and safety of ivosidenib as maintenance therapy in patients with IDH1-mutated AML and high-risk MDS who are ineligible for transplantation, along with accompanying molecular biomarker research. Patients who meet the eligibility criteria will receive ivosidenib treatment until disease progression or unacceptable toxicity. This study will provide an effective maintenance treatment option for transplant-ineligible patients with IDH1-mutated AML and high-risk MDS.
Thiotepa-Containing Conditioning Regimen for Allogeneic HSCT in Chronic Myelomonocytic Leukemia
This is a prospective, single-arm clinical study. It aims to evaluate the effectiveness and safety of a conditioning regimen containing thiotepa (in combination with busulfan and fludarabine, with or without ATG) of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with chronic myelomonocytic leukemia (CMML) who have an intermediate-2 or high-risk prognosis. The main goal is to evaluate 1 year RFS and OS. Other goals include assessing engraftment, overall survival, transplant-related complications, and side effects. A total of 31 participants will be enrolled.
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