Role of molecular alterations in transplantation decisions for patients with primary myelofibrosis

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.

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Publication Details

Journal
Blood Advances
Published
2024-11-19
DOI
https://doi.org/10.1182/bloodadvances.2024014368
Citations
8
Primary Topic
Myeloproliferative Neoplasms: Diagnosis and Treatment
Type
article
Field-Weighted Citation Impact
3.50

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article

Role of molecular alterations in transplantation decisions for patients with primary myelofibrosis

Christina Rautenberg, Nicolaus Martin Kröger, Jean‐Jacques Kiladjian, Marie Robin et al.
8 citations
Blood Advances
Myeloproliferative Neoplasms: Diagnosis and Treatment
3.50
article

Role of molecular alterations in transplantation decisions for patients with primary myelofibrosis

Christina Rautenberg, Nicolaus Martin Kröger, Jean‐Jacques Kiladjian, Marie Robin, Jean‐Christophe Ianotto, Valérie Ugo, Jérémie Riou, Hans Christian Reinhardt, Craig S. Sauter, Rachel B. Salit, Lina Benajiba, Anita Badbaran, Françoise Boyer‐Perrard, Michael Heuser, Victoria Panagiota, Nico Gagelmann, Felicitas Thol, Carmelo Gurnari, Simona Pagliuca, Thomas A. Schroeder, Bart L. Scott, Rafael Daltro De Oliveira, Damien Luque Paz, Émilie Cayssials, Jaroslaw Pawel Maciejewski, Corentin Orvain, Suzanne Tavitian, Marie‐Thérèse Rubio, Bruno Cassinat, Claire Bories
article en
8 citations

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.

Blood AdvancesVol. 9(4)
University of Rome Tor Vergata (IT), Centre National de la Recherche Scientifique (FR), Cleveland Clinic (US), Inserm (FR), Universität Hamburg (DE), Université Paris Cité (FR), Medizinische Hochschule Hannover (DE), Fred Hutch Cancer Center (US), Centre de Recherche en Cancérologie et Immunologie Intégrée Nantes Angers (FR), Assistance Publique – Hôpitaux de Paris (FR), Institut universitaire du cancer de Toulouse Oncopole (FR), Centre Hospitalier Régional et Universitaire de Nancy (FR), Centre Hospitalier de Lens (FR), University Medical Center Hamburg-Eppendorf (DE), Centre Hospitalier Universitaire de Poitiers (FR), Centre Hospitalier Universitaire d'Angers (FR), Essen University Hospital (DE), Centre Hospitalier Régional Universitaire de Brest (FR), Hôpital Saint-Louis (FR), University Hospital Cologne (DE), West German Heart and Vascular Center Essen (DE), Université d'Angers (FR), Nantes Université (FR)
Institut National Du Cancer
Good health and well-being
Openalex Percentile: Top 7%
Myeloproliferative Neoplasms: Diagnosis and Treatment
3.50
DOIOpen Access PDFOpenAlex
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