Comprehensive clinical-molecular transplant scoring system for myelofibrosis undergoing stem cell transplantation
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Comprehensive clinical-molecular transplant scoring system for myelofibrosis undergoing stem cell transplantation. / Gagelmann, Nico; Ditschkowski, Markus; Bogdanov, Rashit; Bredin, Swan; Robin, Marie; Cassinat, Bruno; Shahswar, Rabia; Thol, Felicitas; Heuser, Michael; Socié, Gerard; Beelen, Dietrich; Triviai, Ioanna; Badbaran, Anita; Kröger, Nicolaus.
In: BLOOD, Vol. 133, No. 20, 16.05.2019, p. 2233-2242.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Comprehensive clinical-molecular transplant scoring system for myelofibrosis undergoing stem cell transplantation
AU - Gagelmann, Nico
AU - Ditschkowski, Markus
AU - Bogdanov, Rashit
AU - Bredin, Swan
AU - Robin, Marie
AU - Cassinat, Bruno
AU - Shahswar, Rabia
AU - Thol, Felicitas
AU - Heuser, Michael
AU - Socié, Gerard
AU - Beelen, Dietrich
AU - Triviai, Ioanna
AU - Badbaran, Anita
AU - Kröger, Nicolaus
N1 - © 2019 by The American Society of Hematology.
PY - 2019/5/16
Y1 - 2019/5/16
N2 - Allogeneic hematopoietic stem cell transplantation is curative in myelofibrosis, and current prognostic scoring systems aim to select patients for transplantation. Here, we aimed to develop a prognostic score to determine prognosis after transplantation itself, using clinical, molecular, and transplant-specific information from a total of 361 patients with myelofibrosis. Of these, 205 patients were used as a training cohort to create a clinical-molecular myelofibrosis transplant scoring system (MTSS), which was then externally validated in a cohort of 156 patients. Multivariable analysis on survival identified age at least 57 years, Karnofsky performance status lower than 90%, platelet count lower than 150 × 109/L, leukocyte count higher than 25 × 109/L before transplantation, HLA-mismatched unrelated donor, ASXL1 mutation, and non-CALR/MPL driver mutation genotype being independent predictors of outcome. The uncorrected concordance index for the final survival model was 0.723, and bias-corrected indices were similar. Risk factors were incorporated into a 4-level MTSS: low (score, 0-2), intermediate (score, 3-4), high (score, 5), and very high (score, >5). The 5-year survival according to risk groups in the validation cohort was 83% (95% confidence interval [CI], 71%-95%), 64% (95% CI, 53%-75%), 37% (95% CI, 17%-57%), and 22% (95% CI, 4%-39%), respectively (P < .001). Increasing score was predictive of nonrelapse mortality (P < .001) and remained applicable to primary (0.718) and post-essential thrombocythemia (ET)/polycythemia vera (PV) myelofibrosis (0.701) improving prognostic ability in comparison with all currently available disease-specific systems. In conclusion, this MTSS predicts outcome of patients with primary and post-ET/PV myelofibrosis undergoing allogeneic stem cell transplantation.
AB - Allogeneic hematopoietic stem cell transplantation is curative in myelofibrosis, and current prognostic scoring systems aim to select patients for transplantation. Here, we aimed to develop a prognostic score to determine prognosis after transplantation itself, using clinical, molecular, and transplant-specific information from a total of 361 patients with myelofibrosis. Of these, 205 patients were used as a training cohort to create a clinical-molecular myelofibrosis transplant scoring system (MTSS), which was then externally validated in a cohort of 156 patients. Multivariable analysis on survival identified age at least 57 years, Karnofsky performance status lower than 90%, platelet count lower than 150 × 109/L, leukocyte count higher than 25 × 109/L before transplantation, HLA-mismatched unrelated donor, ASXL1 mutation, and non-CALR/MPL driver mutation genotype being independent predictors of outcome. The uncorrected concordance index for the final survival model was 0.723, and bias-corrected indices were similar. Risk factors were incorporated into a 4-level MTSS: low (score, 0-2), intermediate (score, 3-4), high (score, 5), and very high (score, >5). The 5-year survival according to risk groups in the validation cohort was 83% (95% confidence interval [CI], 71%-95%), 64% (95% CI, 53%-75%), 37% (95% CI, 17%-57%), and 22% (95% CI, 4%-39%), respectively (P < .001). Increasing score was predictive of nonrelapse mortality (P < .001) and remained applicable to primary (0.718) and post-essential thrombocythemia (ET)/polycythemia vera (PV) myelofibrosis (0.701) improving prognostic ability in comparison with all currently available disease-specific systems. In conclusion, this MTSS predicts outcome of patients with primary and post-ET/PV myelofibrosis undergoing allogeneic stem cell transplantation.
KW - Journal Article
U2 - 10.1182/blood-2018-12-890889
DO - 10.1182/blood-2018-12-890889
M3 - SCORING: Journal article
C2 - 30760453
VL - 133
SP - 2233
EP - 2242
JO - BLOOD
JF - BLOOD
SN - 0006-4971
IS - 20
ER -