Last reviewed 2026-04-23 · How we verify

Haploidentical Bone Marrow Transplant

Haploidentical bone marrow transplant replaces a patient's diseased bone marrow with stem cells from a partially matched donor to restore hematopoietic and immune function.

Haploidentical bone marrow transplant replaces a patient's diseased bone marrow with stem cells from a partially matched donor to restore hematopoietic and immune function. Used for Acute leukemia (AML, ALL), Chronic leukemia (CML, CLL), Lymphoma.

At a glance

Mechanism of action

In this procedure, bone marrow or mobilized peripheral blood stem cells from a haploidentical donor (typically a parent or child with 50% HLA match) are infused into a conditioned recipient. The donor stem cells engraft and reconstitute the patient's bone marrow, restoring production of blood cells and immune cells. This approach is used to treat hematologic malignancies and severe bone marrow disorders where a fully matched donor is unavailable.

Approved indications

• Acute leukemia (AML, ALL)
• Chronic leukemia (CML, CLL)
• Lymphoma
• Myelodysplastic syndrome
• Severe aplastic anemia

Common side effects

• Graft-versus-host disease (GVHD)
• Infection
• Graft failure or delayed engraftment
• Mucositis
• Organ toxicity (hepatic, renal, pulmonary)

Key clinical trials

• Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies (PHASE2)
• A Vaccine (CMV-MVA Triplex Vaccine) for the Enhancement of CMV-Specific Immunity and the Prevention of CMV Viremia in Patients Undergoing Haploidentical Hematopoietic Stem Cell Transplant (PHASE1)
• Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency (PHASE2)
• Allogeneic Hematopoietic Cell Transplantation for Disorders of T-cell Proliferation and/or Dysregulation (PHASE2)
• ²¹¹At-OKT10-B10 and Fludarabine Alone or in Combination With Cyclophosphamide and Low-Dose TBI Before Donor Stem Cell Transplant for the Treatment of Newly Diagnosed, Recurrent, or Refractory High-Risk Multiple Myeloma (PHASE1)
• Reduced Intensity Haploidentical BMT for High Risk Solid Tumors (PHASE2)
• Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders (NA)
• Safety and Efficacy of Allogeneic HPV-specific T Cells in Adults With Recurrent or Metastatic HPV16+ Cancers (PHASE1, PHASE2)

Primary sources

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