T-cell depleted allogeneic hematopoietic stem cell transplant for the treatment of Fanconi anemia and MDS/AML Journal Article
| Authors: | Satty, A. M.; Klein, E.; Mauguen, A.; Kunvarjee, B.; Boelens, J. J.; Cancio, M.; Curran, K. J.; Kernan, N. A.; Prockop, S. E.; Scaradavou, A.; Spitzer, B.; Tamari, R.; Ruggiero, J.; Torok-Castanza, J.; Mehta, P. A.; O’Reilly, R. J.; Boulad, F. |
| Article Title: | T-cell depleted allogeneic hematopoietic stem cell transplant for the treatment of Fanconi anemia and MDS/AML |
| Abstract: | 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. |
| Keywords: | adolescent; adult; child; clinical article; controlled study; preschool child; school child; middle aged; retrospective studies; transplantation, homologous; young adult; overall survival; leukemia, myeloid, acute; busulfan; fludarabine; mortality; neutropenia; case report; disease free survival; follow up; t lymphocyte; t-lymphocytes; low drug dose; incidence; cohort analysis; relapse; cyclophosphamide; clinical protocol; hematopoietic stem cell transplantation; retrospective study; high risk patient; chimera; engraftment; myelodysplastic syndrome; whole body radiation; donor; radiation dose fractionation; prophylaxis; graft versus host reaction; transplantation conditioning; allogeneic hematopoietic stem cell transplantation; age distribution; graft rejection; t cell depletion; tacrolimus; donor selection; graft vs host disease; granulocyte colony stimulating factor; alemtuzumab; allotransplantation; thymocyte antibody; fanconi anemia; immune reconstitution; evaluation research; complication; clinical outcome; demographics; acute myeloid leukemia; procedures; non relapse mortality; humans; prognosis; human; male; female; article; transfusion associated graft versus host disease |
| Journal Title: | Bone Marrow Transplantation |
| Volume: | 59 |
| Issue: | 1 |
| ISSN: | 0268-3369 |
| Publisher: | Nature Publishing Group |
| Date Published: | 2024-01-01 |
| Start Page: | 23 |
| End Page: | 33 |
| Language: | English |
| DOI: | 10.1038/s41409-023-02113-1 |
| PUBMED: | 37773270 |
| PROVIDER: | scopus |
| DOI/URL: | |
| Notes: | The MSK Cancer Center Support Grant (P30 CA008748) is acknowledged in the PDF -- Corresponding author is MSK author: Alexandra M. Satty -- Source: Scopus |
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