Allogeneic Hematopoietic Cell Transplantation in Advanced Systemic Mastocytosis: A retrospective analysis of the DRST and GREM registries
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Allogeneic Hematopoietic Cell Transplantation in Advanced Systemic Mastocytosis: A retrospective analysis of the DRST and GREM registries. / 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; Panse, Jens.
in: LEUKEMIA, Jahrgang 38, Nr. 4, 04.2024, S. 810-821.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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TY - JOUR
T1 - Allogeneic Hematopoietic Cell Transplantation in Advanced Systemic Mastocytosis: A retrospective analysis of the DRST and GREM registries
AU - Lübke, Johannes
AU - Christen, Deborah
AU - Schwaab, Juliana
AU - Kaiser, Anne
AU - Naumann, Nicole
AU - Shoumariyeh, Khalid
AU - Jentzsch, Madlen
AU - Sockel, Katja
AU - Schaffrath, Judith
AU - Ayuk, Francis A
AU - Stelljes, Matthias
AU - Hilgendorf, Inken
AU - Sala, Elisa
AU - Kaivers, Jennifer
AU - Schönland, Stefan
AU - Wittke, Christoph
AU - Hertenstein, Bernd
AU - Radsak, Markus
AU - Kaiser, Ulrich
AU - Brückl, Valeska
AU - Kröger, Nicolaus
AU - Brümmendorf, Tim H
AU - Hofmann, Wolf-Karsten
AU - Klein, Stefan
AU - Jost, Edgar
AU - Reiter, Andreas
AU - Panse, Jens
N1 - © 2024. The Author(s).
PY - 2024/4
Y1 - 2024/4
N2 - 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.
AB - 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.
KW - Humans
KW - Mastocytosis, Systemic/genetics
KW - Retrospective Studies
KW - Leukemia, Mast-Cell
KW - Hematopoietic Stem Cell Transplantation
KW - Leukemia, Myeloid, Acute
U2 - 10.1038/s41375-024-02186-x
DO - 10.1038/s41375-024-02186-x
M3 - SCORING: Journal article
C2 - 38448757
VL - 38
SP - 810
EP - 821
JO - LEUKEMIA
JF - LEUKEMIA
SN - 0887-6924
IS - 4
ER -
