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Redefining Allogeneic Transplants & Cellular Therapy: Key Takeaways from EBMT 2025 - Episode 6

EBMT 2025 Spotlight: Long-Term Outcomes With PTCy – Insights From the ALWP-EBMT

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Panelists discuss how long-term outcomes following posttransplant cyclophosphamide (PTCy)-based graft-vs-host disease (GVHD) prophylaxis in allogeneic hematopoietic stem cell transplantation (alloHSCT) show promising survival rates but highlight the ongoing need for optimization in GVHD management and supportive care due to the higher incidence of chronic GVHD and nonrelapse mortality.

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    Summary for Physicians:

    At European Society for Blood and Marrow Transplantation 2025, the Acute Leukemia Working Party presented long-term outcomes following PTCy as part of GVHD prophylaxis in patients undergoing alloHSCT.

    Key Highlights:

    • Survival outcomes: The 2-year leukemia-free survivalrate was approximately 58%, with overall survival at 66%. GVHD-free relapse-free survivalwas around 44%. These outcomes were consistent across donor types, including haploidentical, matched unrelated, and matched sibling donors.
    • GVHD incidence: Acute GVHD grade II to IV occurred in 30.3% of patients, with grade III and IV in 14.3% at day 180. Chronic GVHD developed in 21% of patients at 2 years, with 7% of cases being extensive.
    • Nonrelapse mortality (NRM): The 2-year NRM rate was 19%, with most deaths attributed to disease relapse and infections rather than transplant complications.

    Clinical Implications: These findings affirm the utility of PTCy-based GVHD prophylaxis in various donor settings, showing promising long-term survival and manageable GVHD rates. However, the higher incidence of chronic GVHD and nonrelapse mortality suggest a continued need for optimization in GVHD management and supportive care strategies posttransplant.

    Nelli Bejanyan reports consulting or advisory role for CareDx, Medexus Pharmaceuticals, Orca Biosystems, AlloVir, TScan Therapeutics, and Pfizer; and research funding from CRISPR Therapeutics. Everett Meyer reports sponsored research from Orca Biosciences, Kyverna; and a scientific advisor role and equity holder for GigaMune, Indee, TRACT, Jura Biosciences.Caspian Oliai reports no relevant disclosures (investigator on the Orca T trial funded by Orca Biosciences). Arpita P. Gandhi reports roles with OncLive, MJH Life Sciences, OrcaBio (research), CareDx (Advisory Board). Amandeep Salhotra reports received funding from Rigel, Bristol Myers Squibb and Orca Biosciences; and speakers bureau for Incyte and Sanofi.

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