Reduced intensity hematopoietic stem cell transplantation for accelerated-phase myelofibrosis
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Reduced intensity hematopoietic stem cell transplantation for accelerated-phase myelofibrosis. / Gagelmann, Nico; Wolschke, Christine; Salit, Rachel B; Schroeder, Thomas; Ditschkowski, Markus; Panagiota, Victoria; Cassinat, Bruno; Thol, Felicitas; Badbaran, Anita; Robin, Marie; Reinhardt, Hans Christian; Ayuk, Francis; Heuser, Michael; Scott, Bart L; Kröger, Nicolaus.
In: BLOOD ADV, Vol. 6, No. 4, 22.02.2022, p. 1222-1231.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Reduced intensity hematopoietic stem cell transplantation for accelerated-phase myelofibrosis
AU - Gagelmann, Nico
AU - Wolschke, Christine
AU - Salit, Rachel B
AU - Schroeder, Thomas
AU - Ditschkowski, Markus
AU - Panagiota, Victoria
AU - Cassinat, Bruno
AU - Thol, Felicitas
AU - Badbaran, Anita
AU - Robin, Marie
AU - Reinhardt, Hans Christian
AU - Ayuk, Francis
AU - Heuser, Michael
AU - Scott, Bart L
AU - Kröger, Nicolaus
N1 - Copyright © 2022 American Society of Hematology.
PY - 2022/2/22
Y1 - 2022/2/22
N2 - Accelerated-phase myelofibrosis, currently defined by circulating blasts 10% to 19%, usually confers very high risk for progression and poor outcome. The outcome of hematopoietic stem cell transplantation for accelerated-phase myelofibrosis has not been evaluated yet. We analyzed the outcome of 349 clinically and genetically annotated patients with primary or secondary myelofibrosis undergoing reduced intensity transplantation, of whom 35 had accelerated-phase myelofibrosis. In comparison with chronic-phase (<10% blasts) myelofibrosis, median leukocyte counts were higher, more patients had constitutional symptoms, and RAS mutations were detected more frequently in the accelerated-phase group. After a median follow-up of 5.9 years, estimated 5-year overall survival was 65% (95% confidence interval [CI], 49% to 81%) vs 64% (95% CI, 59% to 69%) for the chronic-phase group (P = .91), and median overall survival was not reached. In terms of relapse-free survival, estimated 5-year outcome for the accelerated-phase group was 49% (95% CI, 32% to 67%) vs 55% (95% CI, 50% to 61%) for the chronic-phase group (P = .65). Estimated 5-year nonrelapse mortality was 20% (95% CI, 8% to 33%) for the accelerated-phase group vs 30% (95% CI, 24% to 35%; P = .25) for the chronic-phase group. In terms of relapse, 5-year incidence was 30% (95% CI, 14% to 46%) for the accelerated-phase group vs 15% (95% CI, 11% to 19%) for the chronic-phase group (P = .02). Results were confirmed in multivariable analysis and propensity score matching. In conclusion, reduced intensity transplantation showed excellent survival but higher relapse for accelerated-phase myelofibrosis.
AB - Accelerated-phase myelofibrosis, currently defined by circulating blasts 10% to 19%, usually confers very high risk for progression and poor outcome. The outcome of hematopoietic stem cell transplantation for accelerated-phase myelofibrosis has not been evaluated yet. We analyzed the outcome of 349 clinically and genetically annotated patients with primary or secondary myelofibrosis undergoing reduced intensity transplantation, of whom 35 had accelerated-phase myelofibrosis. In comparison with chronic-phase (<10% blasts) myelofibrosis, median leukocyte counts were higher, more patients had constitutional symptoms, and RAS mutations were detected more frequently in the accelerated-phase group. After a median follow-up of 5.9 years, estimated 5-year overall survival was 65% (95% confidence interval [CI], 49% to 81%) vs 64% (95% CI, 59% to 69%) for the chronic-phase group (P = .91), and median overall survival was not reached. In terms of relapse-free survival, estimated 5-year outcome for the accelerated-phase group was 49% (95% CI, 32% to 67%) vs 55% (95% CI, 50% to 61%) for the chronic-phase group (P = .65). Estimated 5-year nonrelapse mortality was 20% (95% CI, 8% to 33%) for the accelerated-phase group vs 30% (95% CI, 24% to 35%; P = .25) for the chronic-phase group. In terms of relapse, 5-year incidence was 30% (95% CI, 14% to 46%) for the accelerated-phase group vs 15% (95% CI, 11% to 19%) for the chronic-phase group (P = .02). Results were confirmed in multivariable analysis and propensity score matching. In conclusion, reduced intensity transplantation showed excellent survival but higher relapse for accelerated-phase myelofibrosis.
U2 - 10.1182/bloodadvances.2021006827
DO - 10.1182/bloodadvances.2021006827
M3 - SCORING: Journal article
C2 - 35051996
VL - 6
SP - 1222
EP - 1231
JO - BLOOD ADV
JF - BLOOD ADV
SN - 2473-9529
IS - 4
ER -
