Non-Myeloablative Conditioning and Bone Marrow Transplantation

Summary

Allogeneic blood or marrow transplantation (alloBMT) is a curative therapy for a variety of hematologic disorders, including sickle cell disease and thalassemia. Even when it is clear that alloBMT can give to these patients an improvement in their disease, myeloablative transplants have important toxicities and mortalities associated. The lack of suitable donors continues to be a limit to access to transplantation. Substantial progress has been made recently in the development of pre-treatment regimens that facilitate the sustained engraftment of donor marrow with reduced toxicity. Most of these regimens incorporate highly immunosuppressive drugs, which allow the reduction or elimination of myeloablative agents or total body irradiation without endangering the sustained engraftment of HLA-identical allogeneic stem cells. Preliminary results of non-myeloablative allogeneic stem cell transplantation suggest that the procedure can be performed in patients who are ineligible for myeloablative alloBMT, and that sustained remissions of several hematologic malignancies can be obtained.

Timeline

Start

2013-05

Primary completion

2023-11

Completion

2024-12-31

Publications

• Alasbali R, Alzahrani M, Wilkerson K, Akhom P, Alotaibi F, Alahmari B, de la Fuente J, DeBaun MR, Rasheed W, Samarkandi H, El Fakih R, Saleh M, Elhassan T, Alzahrani H, Aljurf M, Almohareb F, Kassim AA, AlAhmari A. Excellent outcomes using a novel reduced intensity conditioning with thiotepa and post-transplant cyclophosphamide for HLA-matched related donor transplant in adolescents and adults with sickle cell disease. Bone Marrow Transplant. 2026 Jul;61(7):847-851. doi: 10.1038/s41409-026-02876-3. Epub 2026 Apr 28.
• Kassim AA, de la Fuente J, Nur E, Wilkerson KL, Alahmari AD, Seber A, Bonfim C, Simoes BP, Alzahrani M, Eckrich MJ, Horn B, Hanna R, Dhedin N, Rangarajan HG, Gouveia RV, Almohareb F, Aljurf M, Essa M, Alahmari B, Gatwood K, Connelly JA, Dovern E, Rodeghier M, DeBaun MR. An international learning collaborative phase 2 trial for haploidentical bone marrow transplant in sickle cell disease. Blood. 2024 Jun 20;143(25):2654-2665. doi: 10.1182/blood.2023023301.
• Aumann MA, Richerson W, Song AK, Davis LT, Pruthi S, Davis S, Patel NJ, Custer C, Kassim AA, DeBaun MR, Donahue MJ, Jordan LC. Cerebral hemodynamic changes after haploidentical hematopoietic stem cell transplant in adults with sickle cell disease. Blood Adv. 2024 Feb 13;8(3):608-619. doi: 10.1182/bloodadvances.2023010717.

Drugs

Indications