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

Bone Marrow Transplantation volume 59, pages 23–33 (2024)Cite this article

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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.

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Fig. 1: Post-HCT outcomes for patients with FA and MDS/AML.
Fig. 2: Overall survival did not differ by patient risk features.
Fig. 3: Cumulative incidence of graft-versus-host disease.
Fig. 4: Immune reconstitution post-HCT.
Fig. 5: Donor chimerism post-HCT.

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Data availability

The datasets generated during and/or analyzed during the current study are not publicly available due patient privacy requirements but are available from the corresponding author on reasonable request.

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Acknowledgements

This research was funded in part through the NIH/NCI Cancer Center Support Grant P30 CA008748. We acknowledge the expert care provided to patients by staff of the Department of Pediatrics at MSK including our nurses, fellows, residents, and administrators. We are grateful for the dedication and support of our patients and their families. We thank Joseph Olechnowicz for editorial assistance.

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Authors and Affiliations

  1. Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY, USA

    Alexandra M. Satty, Elizabeth Klein, Jaap Jan Boelens, Maria Cancio, Kevin J. Curran, Nancy A. Kernan, Susan E. Prockop, Andromachi Scaradavou, Barbara Spitzer, Julianne Ruggiero, Joanne Torok-Castanza, Richard J. O’Reilly & Farid Boulad

  2. Department of Epidemiology & Biostatistics, Memorial Sloan Kettering Cancer Center, New York, NY, USA

    Audrey Mauguen

  3. Department of Pharmacy, Memorial Sloan Kettering Cancer Center, New York, NY, USA

    Binni Kunvarjee

  4. Department of Pediatrics, Weill Cornell Medicine, New York, NY, USA

    Jaap Jan Boelens, Maria Cancio, Kevin J. Curran, Nancy A. Kernan, Susan E. Prockop, Andromachi Scaradavou, Barbara Spitzer, Richard J. O’Reilly & Farid Boulad

  5. Dana Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA, USA

    Susan E. Prockop

  6. Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, USA

    Roni Tamari

  7. Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, USA

    Parinda A. Mehta

  8. Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA

    Parinda A. Mehta

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Contributions

AMS, BK, JJB, MC, KJC, NAK, SEP, ASc, BS, RT, JR, JTC, PAM, RJO, FB performed the research; AMS, EK, AM analyzed the data; AMS and FB wrote the paper. All authors provided substantive review and approval of the manuscript.

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Correspondence to Alexandra M. Satty.

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Competing interests

JJB – consulting Sobi, Omeros, Bluebird Bio, Bluerock, Sanofi, Advanced Clinical, Immusoft; KJC – Serves on the advisory board/consulting for Novartis, Atara Biotherapeutics, and Turn Bio. He receives research funding from Novartis, Celegene, Cellectis. SEP – Receives support for the conduct of clinical trials from Atara Biotherapeutics, AlloVir, and Jasper. Consulting for CellEvolve, Pierre Fabre and Regeneron. IP related to development of adoptive T cell therapies with all rights assigned to MSKCC; ROR received royalties following licensure of the EBV-specific T-cell bank by Atara Biotherapeutics and has subsequently received research support and consultant fees from Atara Biotherapeutics. The other authors declare no competing interests.

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Satty, A.M., Klein, E., Mauguen, A. et al. T-cell depleted allogeneic hematopoietic stem cell transplant for the treatment of Fanconi anemia and MDS/AML. Bone Marrow Transplant 59, 23–33 (2024). https://doi.org/10.1038/s41409-023-02113-1

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