Academic Journal
A predictive classifier of poor prognosis in transplanted patients with juvenile myelomonocytic leukemia: a study on behalf of the Société Francophone de Greffe de Moelle et de Thérapie Cellulaire
| العنوان: | A predictive classifier of poor prognosis in transplanted patients with juvenile myelomonocytic leukemia: a study on behalf of the Société Francophone de Greffe de Moelle et de Thérapie Cellulaire |
|---|---|
| المؤلفون: | Meyran, Déborah, Arfeuille, Chloé, Chevret, Sylvie, Neven, Quentin, Caye-Eude, Aurélie, Lainey, Elodie, Petit, Arnaud, Rialland, Fanny, Michel, Gérard, Plantaz, Dominique, Jubert, Charlotte, Theron, Alexandre, Gandemer, Virginie, Ouachée-Chardin, Marie, Paillard, Catherine, Bruno, Bénédicte, Buchbinder, Nimrod, Pochon, Cécile, Calvo, Charlotte, Fahd, Mony, Baruchel, André, Cavé, Hélène, Dalle, Jean-Hugues, Strullu, Marion |
| المساهمون: | Hôpital Robert Debré, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), University of Melbourne, Hématopoïèse normale et pathologique : émergence, environnement et recherche translationnelle Paris ((UMR_S1131 / U1131)), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris Cité (UPCité), Hopital Saint-Louis AP-HP (AP-HP), Université Paris Cité (UPCité), CHU Trousseau APHP, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Sorbonne Université (SU), Centre Hospitalier Universitaire de Nantes = Nantes University Hospital (CHU Nantes), Assistance Publique - Hôpitaux de Marseille (APHM), Centre Hospitalier Universitaire CHU Grenoble (CHUGA), Centre Hospitalier Universitaire de Bordeaux (CHU Bordeaux), Cellules Souches, Plasticité Cellulaire, Médecine Régénératrice et Immunothérapies (IRMB), Centre Hospitalier Régional Universitaire Montpellier (CHRU Montpellier)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université de Montpellier (UM), CHU Montpellier = Montpellier University Hospital, Centre Hospitalier Régional Universitaire Montpellier (CHRU Montpellier), Centre Hospitalier Universitaire de Rennes CHU Rennes = Rennes University Hospital Pontchaillou, Institut d'hématologie et d'oncologie pédiatrique CHU - HCL (IHOPe), Hospices Civils de Lyon (HCL), Hôpital de Hautepierre Strasbourg, Hôpital Jeanne de Flandre Lille, CHU Rouen, Normandie Université (NU), Centre Hospitalier Régional Universitaire de Nancy (CHRU Nancy), Immunologie humaine, physiopathologie & immunothérapie (HIPI (UMR_S_976 / U976)) |
| المصدر: | ISSN: 0390-6078. |
| بيانات النشر: | CCSD Ferrata Storti Foundation |
| سنة النشر: | 2024 |
| مصطلحات موضوعية: | Juvenile myelomonocytic leukemia, hematopoietic stem cell transplant, RAS pathway, monocyte, pediatrics, [SDV.CAN]Life Sciences [q-bio]/Cancer, [SDV.MHEP.HEM]Life Sciences [q-bio]/Human health and pathology/Hematology |
| الوصف: | International audience ; Juvenile myelomonocytic leukemia (JMML) is an aggressive pediatric myeloproliferative neoplasm requiring hematopoietic stem cell transplantation (HSCT) in most cases. We retrospectively analyzed 119 JMML patients who underwent first allogeneic HSCT between 2002 and 2021. The majority (97%) carried a RAS-pathway mutation, and 62% exhibited karyotypic alterations or additional mutations in SETBP1, ASXL1, JAK3 and/or the RAS pathway. Relapse was the primary cause of death, with a 5-year cumulative incidence of 24.6% (95%CI: 17.1-32.9). Toxic deaths occurred in 12 patients, resulting in treatmentrelated mortality (TRM) of 9.0% (95%CI: 4.6-15.3). The 5-year overall (OS) and event-free survival were 73.6% (95%CI: 65.7-82.4) and 66.4% (95%CI: 58.2-75.8), respectively. Four independent adverse prognostic factors for OS were identified: age at diagnosis >2 years, time from diagnosis to HSCT >6 months, monocyte count at diagnosis >7.2x109/L, and the presence of additional genetic alterations. Based on these factors, we proposed a predictive classifier. Patients with three or more predictors (21% of the cohort) had a 5-year OS of 34.2%, whereas those with none (7%) had a 5-year OS of 100%. Our study demonstrates improved transplant outcomes compared to prior published data, which can be attributed to the synergistic impacts of a low TRM and a reduced yet still substantial relapse incidence. By integrating genetic information with clinical and hematological features, we have devised a predictive classifier. This classifier effectively identifies a subgroup of patients who are at a heightened risk of unfavorable post-transplant outcomes who would benefit novel therapeutic agents and post-transplant strategies. |
| نوع الوثيقة: | article in journal/newspaper |
| اللغة: | English |
| Relation: | info:eu-repo/semantics/altIdentifier/pmid/38385260; PUBMED: 38385260 |
| DOI: | 10.3324/haematol.2023.284103 |
| الاتاحة: | https://univ-rennes.hal.science/hal-04645180 https://univ-rennes.hal.science/hal-04645180v1/document https://univ-rennes.hal.science/hal-04645180v1/file/11460-Article%20Text-83464-2-10-20240802.pdf https://doi.org/10.3324/haematol.2023.284103 |
| Rights: | http://creativecommons.org/licenses/by-nc/ ; info:eu-repo/semantics/OpenAccess |
| رقم الانضمام: | edsbas.3B8B48E7 |
| قاعدة البيانات: | BASE |
| DOI: | 10.3324/haematol.2023.284103 |
|---|