Allogeneic Hematopoietic Cell Transplantation in Advanced Systemic Mastocytosis: A retrospective analysis of the DRST and GREM registries

• DOI: 10.1038/s41375-024-02186-x
• Publication Date: 2024-03-06T17:02:17Z
• Authors: Johannes Lübke; Deborah Christen; Juliana Schwaab; Anne Kaiser; Nicole Naumann; Khalid Shoumariyeh; Madlen Jentzsch; Katja Sockel; Judith Schaffrath; Francis A. Ayuk; Matthias Stelljes; Inken Hilgendorf; Elisa Sala; Jennifer Kaivers; Stefan Schönland; Christoph Wittke; Bernd Hertenstein; Markus Radsak; Ulrich Kaiser; Valeska Brückl; Nicolaus Kröger; Tim H. Brümmendorf; Wolf-Karsten Hofmann; Stefan Klein; Edgar Jost; Andreas Reiter; Jens Panse
• Keywords: Midostaurin

Abstract We identified 71 patients with AdvSM (aggressive SM [ASM], an associated hematologic neoplasm [SM-AHN, e.g., acute myeloid leukemia, SM-AML], mast cell leukemia [MCL]) in two national registries (DRST/GREM) who received allogeneic hematopoietic transplantation (alloHCT) performed Germany from 1999–2021. Median overall survival (OS) of ASM/SM-AHN ( n = 30, 45%), SM-AML 28, 39%) and MCL ± AHN 13, 19%) was 9.0, 3.3 0.9 years P 0.007). Improved median OS response (17/41, 41%; HR 0.4 [0.2–0.9], 0.035) and/or (26/43, 60%, 0.3 [0.1–0.7], 0.004) prior to alloHCT. Adverse predictors for included absence KIT D816V (10/61, 16%, 2.9 [1.2–6.5], < 0.001) a complex karyotype (9/60, 15%, 4.2 [1.8–10.0], 0.016). HLA-match, conditioning type or at centers reporting above-average alloHCTs (≥7) had no impact on OS. Taking into account competing events 1, 3 5, relapse-related mortality non-relapse rate were 15%/23%, 20%/30% 23%/35%, respectively. Irrespective subtype, subsequent treatment achieved 13/30 (43%) highest midostaurin/avapritinib (7/9, 78%). conclude that outcome alloHCT is more affected by disease phenotype transplant than characteristics.