1. Allogeneic Hematopoietic Cell Transplantation in Advanced Systemic Mastocytosis: A retrospective analysis of the DRST and GREM registries
- Author
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Lübke, Johannes, Christen, Deborah, Schwaab, Juliana, Kaiser, Anne, Naumann, Nicole, Shoumariyeh, Khalid, Jentzsch, Madlen, Sockel, Katja, Schaffrath, Judith, Ayuk, Francis A., Stelljes, Matthias, Hilgendorf, Inken, Sala, Elisa, Kaivers, Jennifer, Schönland, Stefan, Wittke, Christoph, Hertenstein, Bernd, Radsak, Markus, Kaiser, Ulrich, Brückl, Valeska, Kröger, Nicolaus, Brümmendorf, Tim H., Hofmann, Wolf-Karsten, Klein, Stefan, Jost, Edgar, Reiter, Andreas, and Panse, Jens
- Abstract
We identified 71 patients with AdvSM (aggressive SM [ASM], SM with 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 an allogeneic hematopoietic cell transplantation (alloHCT) performed in Germany from 1999–2021. Median overall survival (OS) of ASM/SM-AHN (n= 30, 45%), SM-AML (n= 28, 39%) and MCL ± AHN (n= 13, 19%) was 9.0, 3.3 and 0.9 years (P= 0.007). Improved median OS was associated with response of SM (17/41, 41%; HR 0.4 [0.2–0.9], P= 0.035) and/or of AHN (26/43, 60%, HR 0.3 [0.1–0.7], P= 0.004) prior to alloHCT. Adverse predictors for OS included absence of KITD816V (10/61, 16%, HR 2.9 [1.2–6.5], P< 0.001) and a complex karyotype (9/60, 15%, HR 4.2 [1.8–10.0], P= 0.016). HLA-match, conditioning type or transplantation at centers reporting above-average alloHCTs (≥7) had no impact on OS. Taking into account competing events at years 1, 3 and 5, relapse-related mortality and non-relapse mortality rate were 15%/23%, 20%/30% and 23%/35%, respectively. Irrespective of subtype, subsequent treatment response was achieved in 13/30 (43%) patients and was highest on midostaurin/avapritinib (7/9, 78%). We conclude that outcome of alloHCT in AdvSM is more affected by disease phenotype and treatment response prior to transplant than by transplant characteristics.
- Published
- 2024
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