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

Lübke J, Christen D, Schwaab J, Kaiser A, Naumann N, Shoumariyeh K, Jentzsch M, Sockel K, Schaffrath J, Ayuk FA, Stelljes M, Hilgendorf I, Sala E, Kaivers J, Schönland S, Wittke C, Hertenstein B, Radsak M, Kaiser U, Brückl V, Kröger N, Brümmendorf TH, Hofmann WK, Klein S, Jost E, Reiter A, Panse J (2024)

Publication Language: English

Publication Type: Journal article

Publication year: 2024

Journal

Book Volume: 38

Pages Range: 810-821

DOI: 10.1038/s41375-024-02186-x

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 KIT D816V (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.

Involved external institutions

Universitätsklinikum Mannheim Germany (DE) Universitätsklinikum Aachen (UKA) Germany (DE) Universitätsklinikum Carl Gustav Carus Dresden Germany (DE) Martin-Luther-Universität Halle-Wittenberg (MLU) Germany (DE) Universitätsklinikum Jena Germany (DE) Universitätsmedizin Rostock Germany (DE) Universitätsklinikum Freiburg Germany (DE) Universitätsklinikum Hamburg-Eppendorf (UKE) Germany (DE) Universitätsklinikum Ulm Germany (DE) Universitätsklinikum Essen Germany (DE) Universitätsklinikum Heidelberg Germany (DE) Klinikverbund Bremen (Gesundheit Nord) Germany (DE) Universitätsmedizin der Johannes Gutenberg-Universität Mainz Germany (DE) Universitätsklinikum Regensburg Germany (DE) Universitätsklinikum Leipzig Germany (DE) Westfälische Wilhelms-Universität (WWU) Münster Germany (DE)

How to cite

APA:

Lübke, J., Christen, D., Schwaab, J., Kaiser, A., Naumann, N., Shoumariyeh, K.,... Panse, J. (2024). Allogeneic Hematopoietic Cell Transplantation in Advanced Systemic Mastocytosis: A retrospective analysis of the DRST and GREM registries. Leukemia, 38, 810-821. https://doi.org/10.1038/s41375-024-02186-x

MLA:

Lübke, Johannes, et al. "Allogeneic Hematopoietic Cell Transplantation in Advanced Systemic Mastocytosis: A retrospective analysis of the DRST and GREM registries." Leukemia 38 (2024): 810-821.

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