Comprehensive clinical-molecular transplant scoring system for myelofibrosis undergoing stem cell transplantation
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- Nico Gagelmann
- Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany;
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- Markus Ditschkowski
- Department of Bone Marrow Transplantation, West German Cancer Center, University Hospital of Essen, Essen, Germany;
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- Rashit Bogdanov
- Department of Bone Marrow Transplantation, West German Cancer Center, University Hospital of Essen, Essen, Germany;
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- Swann Bredin
- Service d'Hématologie-Greffe and
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- Marie Robin
- Service d'Hématologie-Greffe and
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- Bruno Cassinat
- Laboratoire de biologie cellulaire, Hôpital Saint-Louis, Assistance Publique Hôpitaux de Paris, Paris, France; and
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- Rabia Shahswar
- Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany
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- Felicitas Thol
- Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany
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- Michael Heuser
- Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany
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- Gerard Socié
- Service d'Hématologie-Greffe and
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- Dietrich Beelen
- Department of Bone Marrow Transplantation, West German Cancer Center, University Hospital of Essen, Essen, Germany;
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- Ioanna Triviai
- Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany;
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- Anita Badbaran
- Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany;
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- Nicolaus Kröger
- Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany;
説明
<jats:title>Abstract</jats:title> <jats:p>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.</jats:p>
収録刊行物
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- Blood
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Blood 133 (20), 2233-2242, 2019-05-16
American Society of Hematology