Damien Luque Paz, Nico Gagelmann, Lina Benajiba, Jérémie Riou, Rachel Salit, Corentin Orvain, Thomas Schroeder, Claire Bories, Carmelo Gurnari, Anita Badbaran, Françoise Boyer, Simona Pagliuca, Christina Rautenberg, Suzanne Tavitian, Victoria Pangiota, Jean-Christophe Ianotto, Felicitas Thol, Emilie Cayssials, Michael Heuser, Marie-Thérèse Rubio, Bruno Cassinat, Rafael Daltro de Oliveira, Craig Sauter, Jaroslaw P Maciejewski, Hans Christian Reinhardt, Bart L Scott, Valérie Ugo, Nicolaus Kröger, Jean-Jacques Kiladjian, Marie Robin
{"title":"分子改变在原发性骨髓纤维化患者移植决定中的作用。","authors":"Damien Luque Paz, Nico Gagelmann, Lina Benajiba, Jérémie Riou, Rachel Salit, Corentin Orvain, Thomas Schroeder, Claire Bories, Carmelo Gurnari, Anita Badbaran, Françoise Boyer, Simona Pagliuca, Christina Rautenberg, Suzanne Tavitian, Victoria Pangiota, Jean-Christophe Ianotto, Felicitas Thol, Emilie Cayssials, Michael Heuser, Marie-Thérèse Rubio, Bruno Cassinat, Rafael Daltro de Oliveira, Craig Sauter, Jaroslaw P Maciejewski, Hans Christian Reinhardt, Bart L Scott, Valérie Ugo, Nicolaus Kröger, Jean-Jacques Kiladjian, Marie Robin","doi":"10.1182/bloodadvances.2024014368","DOIUrl":null,"url":null,"abstract":"<p><strong>Abstract: </strong>The aim of our study was to analyze the potential survival benefit associated with hematopoietic stem cell transplantation (HSCT) according to clinicobiological scores, which incorporate mutation-enhanced international prognostic score system (MIPSS) to facilitate decision-making in this context. One transplant (n = 241) and 1 nontransplant cohort (n = 239) were used to test the hypothesis that patients with primary myelofibrosis with higher risk molecular score benefit from HSCT. A weighted propensity score was applied to balance confounding factors with the transplanted cohort as reference. Weighted Cox proportional hazard models and logistic regression analyses were performed. Overall, 105 patients who did not receive transplant could be matched to the 239 patients who did receive transplants. HSCT was associated with a higher 6-year overall survival rate in intermediate-2 (60.1% vs 41.5%) and high-risk DIPSS patients (44.4% vs 6.55%), high-risk MIPSS70 (46.5% vs 23.9%), high-risk (73.2% vs 39.7%) or very high-risk MIPSS70+V2 (51.8% vs 24%). Patients with intermediate MIPSS70 scores have an advantage of survival with HSCT only when their myelofibrosis transplant scoring system (MTSS) were low or intermediate. Patients who received transplant had an increased mortality risk the first year, but a significant benefit with HSCT after the 1-year landmark was observed in higher risk patients. This study confirms that, similar to DIPSS, MIPSS70 and MIPSS70+V2 risk score in addition to MTSS can be used to determine which patients with primary myelofibrosis have survival benefit from HSCT over non-HSCT strategies.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"797-807"},"PeriodicalIF":7.4000,"publicationDate":"2025-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11869958/pdf/","citationCount":"0","resultStr":"{\"title\":\"Role of molecular alterations in transplantation decisions for patients with primary myelofibrosis.\",\"authors\":\"Damien Luque Paz, Nico Gagelmann, Lina Benajiba, Jérémie Riou, Rachel Salit, Corentin Orvain, Thomas Schroeder, Claire Bories, Carmelo Gurnari, Anita Badbaran, Françoise Boyer, Simona Pagliuca, Christina Rautenberg, Suzanne Tavitian, Victoria Pangiota, Jean-Christophe Ianotto, Felicitas Thol, Emilie Cayssials, Michael Heuser, Marie-Thérèse Rubio, Bruno Cassinat, Rafael Daltro de Oliveira, Craig Sauter, Jaroslaw P Maciejewski, Hans Christian Reinhardt, Bart L Scott, Valérie Ugo, Nicolaus Kröger, Jean-Jacques Kiladjian, Marie Robin\",\"doi\":\"10.1182/bloodadvances.2024014368\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Abstract: </strong>The aim of our study was to analyze the potential survival benefit associated with hematopoietic stem cell transplantation (HSCT) according to clinicobiological scores, which incorporate mutation-enhanced international prognostic score system (MIPSS) to facilitate decision-making in this context. One transplant (n = 241) and 1 nontransplant cohort (n = 239) were used to test the hypothesis that patients with primary myelofibrosis with higher risk molecular score benefit from HSCT. A weighted propensity score was applied to balance confounding factors with the transplanted cohort as reference. Weighted Cox proportional hazard models and logistic regression analyses were performed. Overall, 105 patients who did not receive transplant could be matched to the 239 patients who did receive transplants. HSCT was associated with a higher 6-year overall survival rate in intermediate-2 (60.1% vs 41.5%) and high-risk DIPSS patients (44.4% vs 6.55%), high-risk MIPSS70 (46.5% vs 23.9%), high-risk (73.2% vs 39.7%) or very high-risk MIPSS70+V2 (51.8% vs 24%). Patients with intermediate MIPSS70 scores have an advantage of survival with HSCT only when their myelofibrosis transplant scoring system (MTSS) were low or intermediate. Patients who received transplant had an increased mortality risk the first year, but a significant benefit with HSCT after the 1-year landmark was observed in higher risk patients. 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Role of molecular alterations in transplantation decisions for patients with primary myelofibrosis.
Abstract: The aim of our study was to analyze the potential survival benefit associated with hematopoietic stem cell transplantation (HSCT) according to clinicobiological scores, which incorporate mutation-enhanced international prognostic score system (MIPSS) to facilitate decision-making in this context. One transplant (n = 241) and 1 nontransplant cohort (n = 239) were used to test the hypothesis that patients with primary myelofibrosis with higher risk molecular score benefit from HSCT. A weighted propensity score was applied to balance confounding factors with the transplanted cohort as reference. Weighted Cox proportional hazard models and logistic regression analyses were performed. Overall, 105 patients who did not receive transplant could be matched to the 239 patients who did receive transplants. HSCT was associated with a higher 6-year overall survival rate in intermediate-2 (60.1% vs 41.5%) and high-risk DIPSS patients (44.4% vs 6.55%), high-risk MIPSS70 (46.5% vs 23.9%), high-risk (73.2% vs 39.7%) or very high-risk MIPSS70+V2 (51.8% vs 24%). Patients with intermediate MIPSS70 scores have an advantage of survival with HSCT only when their myelofibrosis transplant scoring system (MTSS) were low or intermediate. Patients who received transplant had an increased mortality risk the first year, but a significant benefit with HSCT after the 1-year landmark was observed in higher risk patients. This study confirms that, similar to DIPSS, MIPSS70 and MIPSS70+V2 risk score in addition to MTSS can be used to determine which patients with primary myelofibrosis have survival benefit from HSCT over non-HSCT strategies.
期刊介绍:
Blood Advances, a semimonthly medical journal published by the American Society of Hematology, marks the first addition to the Blood family in 70 years. This peer-reviewed, online-only, open-access journal was launched under the leadership of founding editor-in-chief Robert Negrin, MD, from Stanford University Medical Center in Stanford, CA, with its inaugural issue released on November 29, 2016.
Blood Advances serves as an international platform for original articles detailing basic laboratory, translational, and clinical investigations in hematology. The journal comprehensively covers all aspects of hematology, including disorders of leukocytes (both benign and malignant), erythrocytes, platelets, hemostatic mechanisms, vascular biology, immunology, and hematologic oncology. Each article undergoes a rigorous peer-review process, with selection based on the originality of the findings, the high quality of the work presented, and the clarity of the presentation.