T-cell depleted allogeneic hematopoietic stem cell transplant for the treatment of Fanconi anemia and MDS/AML.

Autor: Satty AM; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA. sattya@mskcc.org., Klein E; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA., Mauguen A; Department of Epidemiology & Biostatistics, Memorial Sloan Kettering Cancer Center, New York, NY, USA., Kunvarjee B; Department of Pharmacy, Memorial Sloan Kettering Cancer Center, New York, NY, USA., Boelens JJ; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.; Department of Pediatrics, Weill Cornell Medicine, New York, NY, USA., Cancio M; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.; Department of Pediatrics, Weill Cornell Medicine, New York, NY, USA., Curran KJ; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.; Department of Pediatrics, Weill Cornell Medicine, New York, NY, USA., Kernan NA; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.; Department of Pediatrics, Weill Cornell Medicine, New York, NY, USA., Prockop SE; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.; Department of Pediatrics, Weill Cornell Medicine, New York, NY, USA.; Dana Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA., Scaradavou A; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.; Department of Pediatrics, Weill Cornell Medicine, New York, NY, USA., Spitzer B; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.; Department of Pediatrics, Weill Cornell Medicine, New York, NY, USA., Tamari R; Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, USA., Ruggiero J; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA., Torok-Castanza J; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA., Mehta PA; Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA., O'Reilly RJ; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.; Department of Pediatrics, Weill Cornell Medicine, New York, NY, USA., Boulad F; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.; Department of Pediatrics, Weill Cornell Medicine, New York, NY, USA.
Jazyk: angličtina
Zdroj: Bone marrow transplantation [Bone Marrow Transplant] 2024 Jan; Vol. 59 (1), pp. 23-33. Date of Electronic Publication: 2023 Sep 29.
DOI: 10.1038/s41409-023-02113-1
Abstrakt: The only curative approach for myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) arising in patients with Fanconi anemia (FA) is allogeneic hematopoietic stem cell transplantation (HCT); however, HCT approaches are inconsistent and limited data on outcomes exist. We retrospectively evaluated outcomes of thirty patients with FA and MDS/AML who underwent first allogeneic HCT with a T-cell depleted (TCD) graft at our institution. Patients were transplanted on successive protocols with stepwise changes in cytoreduction and GVHD prophylaxis. All but two patients (93%) experienced durable hematopoietic engraftment. With median follow-up of 8.7 years, 5-year OS was 66.8% and DFS 53.8%. No significant differences in survival were found in patients with high-risk prognostic features (age ≥20 years, AML diagnosis, alternative donor graft) or when stratified by conditioning regimen. The 5-year cumulative incidences of relapse and NRM were 24.3% and 21.9%, respectively. NRM was higher in patients ≥20 years at HCT but did not otherwise differ. We herein demonstrate promising outcomes following allogeneic HCT for patients with FA and MDS/AML using TCD grafts, particularly in a cohort of high-risk patients with 50% ≥20 years and a majority receiving mismatched grafts. Future prospective studies are needed to compare this approach with other HCT platforms.
(© 2023. The Author(s), under exclusive licence to Springer Nature Limited.)
Databáze: MEDLINE
Externí odkaz: