دورية أكاديمية
Allogeneic haematopoietic cell transplantation for advanced systemic mastocytosis: Best practice recommendations on behalf of the EBMT Practice Harmonisation and Guidelines Committee.
| العنوان: | Allogeneic haematopoietic cell transplantation for advanced systemic mastocytosis: Best practice recommendations on behalf of the EBMT Practice Harmonisation and Guidelines Committee. |
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| المؤلفون: | McLornan DP; Chair of the Chronic Malignancies Working Party of the EBMT. Department of Haematology and Stem Cell Transplantation, University College Hospital, London, UK. donal.mclornan@nhs.net., Czerw T; Maria Skłodowska-Curie National Research Institute of Oncology, Gliwice, Poland., Damaj G; Haematology Institute, Normandy University School of Medicine, Caen, France., Ethell M; Department of Haematology, The Royal Marsden NHS Hospital, Sutton, UK., Gurnari C; Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy.; Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH, USA., Hernández-Boluda JC; Hospital Clínico Universitario-INCLIVA, University of Valencia, Valencia, Spain., Polverelli N; Unit of Bone Marrow Transplantation, Division of Hematology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy., Schwaab J; Department of Hematology and Oncology, University Hospital Mannheim, Heidelberg University, Mannheim, Germany., Sockel K; Medical Clinic and Policlinic I, University Hospital Dresden, TU Dresden, Germany., Raffaella G; Co-Chair of the Practice Harmonization and Guidelines Committee of EBMT and Chair of the ADWP of the EBMT. Haematology and Bone Marrow Transplant Unit, IRCCS San Raffaele Hospital, Milano, Italy., Onida F; Co-Chair of the Practice Harmonization and Guidelines Committee of the EBMT. ASST Fatebenefratelli-Sacco-University of Milan, Milano, Italy., Sánchez-Ortega I; Secretary of the Practice Harmonization and Guidelines Committee of EBMT, EBMT Medical Officer, Executive Office, Barcelona, Spain., Battipaglia G; Hematology Department, Federico II University of Naples, Naples, Italy., Elena C; Department of Hematology Oncology, Foundation IRCCS Policlinico San Matteo, University of Pavia, Pavia, Italy., Gotlib J; Division of Hematology, Stanford Cancer Institute/Stanford University School of Medicine, Stanford, CA, USA., Reiter A; Department of Hematology and Oncology, University Hospital Mannheim, Heidelberg University, Mannheim, Germany., Rossignol J; Center National de Référence des Mastocytoses (CEREMAST), Service d'hématologie adulte, Hôpitaux Necker-Enfants Malades et Cochin, Paris, France., Ustun C; Division of Hematology/Oncology/Cell Therapy, Rush University, Chicago, IL, USA., Valent P; Department of Internal Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.; Director of the Ludwig Boltzmann Institute for Hematology and Oncology, Medical University of Vienna, Vienna, Austria.; Coordinator of the European Competence Network on Mastocytosis (ECNM), Vienna, Austria., Yakoub-Agha I; Chair of the EBMT Practice Harmonization and Guidelines Committee. CHU de Lille, Univ Lille, INSERM U1286, Infinite, 59000, Lille, France., Radia DH; Department of Haematology, Guy's and St. Thomas' NHS Foundation Trust, London, UK. |
| المصدر: | Leukemia [Leukemia] 2024 Apr; Vol. 38 (4), pp. 699-711. Date of Electronic Publication: 2024 Mar 12. |
| نوع المنشور: | Journal Article; Review |
| اللغة: | English |
| بيانات الدورية: | Publisher: Nature Publishing Group, Specialist Journals Country of Publication: England NLM ID: 8704895 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5551 (Electronic) Linking ISSN: 08876924 NLM ISO Abbreviation: Leukemia Subsets: MEDLINE |
| أسماء مطبوعة: | Publication: 2000- : London : Nature Publishing Group, Specialist Journals Original Publication: [Baltimore, Md.] : Williams & Wilkins, [c1987- |
| مواضيع طبية MeSH: | Mastocytosis, Systemic*/therapy , Mastocytosis, Systemic*/drug therapy , Antineoplastic Agents*/therapeutic use , Mastocytosis*/therapy , Leukemia, Mast-Cell*/drug therapy , Hematopoietic Stem Cell Transplantation*, Humans ; Proto-Oncogene Proteins c-kit/genetics ; Mast Cells |
| مستخلص: | Systemic Mastocytosis (SM) is a multifaceted clinically heterogeneous disease. Advanced SM (AdvSM) comprises three entities: aggressive SM (ASM), mast cell leukaemia (MCL) and SM with an associated hematologic neoplasm (SM-AHN), the latter accounting for 60-70% of all AdvSM cases. Detection of a disease-triggering mutation in the KIT gene (esp. KIT D816V) in >90% of the patients with ASM or SM-AHN has led to a significant improvement in therapeutic options by the implementation of two KIT-targeting kinase inhibitors: midostaurin and avapritinib. Although complete remissions have been reported, neither of these targeted agents is 'curative' in all patients and the duration of responses varies. The median overall survival, depending on the WHO subtype and scoring result, is approximately 1 to 4 years. Although the European Competence Network on Mastocytosis (ECNM) and American Initiative in Mast Cell Diseases (AIM) consensus groups recommend allogeneic haematopoietic cell transplantation (allo-HCT) in drug-resistant and other high-risk patients, there is a relative lack of information to guide clinicians on which patients with AdvSM should be considered for transplant, and how KIT inhibitors may fit into the transplant algorithm, including their use pre- and post-transplant to optimise outcomes. Following the generation of an expert panel with a specialist interest in allo-HCT and mastocytosis, these best practice recommendations were generated according to the European Society for Blood and Marrow Transplantation (EBMT) Practice Harmonisation and guidelines and ECNM methodology. We aim to provide a practical, clinically relevant and up-to-date framework to guide allo-HCT in AdvsM in 2024 and beyond. (© 2024. The Author(s), under exclusive licence to Springer Nature Limited.) |
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| المشرفين على المادة: | 0 (Antineoplastic Agents) EC 2.7.10.1 (Proto-Oncogene Proteins c-kit) |
| تواريخ الأحداث: | Date Created: 20240313 Date Completed: 20240408 Latest Revision: 20240408 |
| رمز التحديث: | 20240408 |
| DOI: | 10.1038/s41375-024-02182-1 |
| PMID: | 38472477 |
| قاعدة البيانات: | MEDLINE |
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| DOI: | 10.1038/s41375-024-02182-1 |