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STEM CELL TRANSPLANTATION

Allogeneic haematopoietic cell transplantation for advanced systemic mastocytosis: Best practice recommendations on behalf of the EBMT Practice Harmonisation and Guidelines Committee

Leukemia volume 38, pages 699–711 (2024)Cite this article

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Abstract

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.

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Fig. 1: Management strategies for potential allo-HCT candidates: Aggressive Systemic Mastocytosis.
Fig. 2: Management strategies for potential allo-HCT candidates: SM-Associated Haematological Neoplasm.
Fig. 3: Management strategies for potential allo-HCT candidates: Mast Cell Leukaemia.

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Author information

Authors and Affiliations

  1. Chair of the Chronic Malignancies Working Party of the EBMT. Department of Haematology and Stem Cell Transplantation, University College Hospital, London, UK

    Donal P. McLornan

  2. Maria Skłodowska-Curie National Research Institute of Oncology, Gliwice, Poland

    Tomasz Czerw

  3. Haematology Institute, Normandy University School of Medicine, Caen, France

    Gandhi Damaj

  4. Department of Haematology, The Royal Marsden NHS Hospital, Sutton, UK

    Mark Ethell

  5. Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy

    Carmelo Gurnari

  6. Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH, USA

    Carmelo Gurnari

  7. Hospital Clínico Universitario-INCLIVA, University of Valencia, Valencia, Spain

    Juan Carlos Hernández-Boluda

  8. Unit of Bone Marrow Transplantation, Division of Hematology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy

    Nicola Polverelli

  9. Department of Hematology and Oncology, University Hospital Mannheim, Heidelberg University, Mannheim, Germany

    Juliana Schwaab & Andreas Reiter

  10. Medical Clinic and Policlinic I, University Hospital Dresden, TU Dresden, Germany

    Katja Sockel

  11. 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

    Greco Raffaella

  12. Co-Chair of the Practice Harmonization and Guidelines Committee of the EBMT. ASST Fatebenefratelli-Sacco-University of Milan, Milano, Italy

    Francesco Onida

  13. Secretary of the Practice Harmonization and Guidelines Committee of EBMT, EBMT Medical Officer, Executive Office, Barcelona, Spain

    Isabel Sánchez-Ortega

  14. Hematology Department, Federico II University of Naples, Naples, Italy

    Giorgia Battipaglia

  15. Department of Hematology Oncology, Foundation IRCCS Policlinico San Matteo, University of Pavia, Pavia, Italy

    Chiara Elena

  16. Division of Hematology, Stanford Cancer Institute/Stanford University School of Medicine, Stanford, CA, USA

    Jason Gotlib

  17. Center National de Référence des Mastocytoses (CEREMAST), Service d’hématologie adulte, Hôpitaux Necker-Enfants Malades et Cochin, Paris, France

    Julien Rossignol

  18. Division of Hematology/Oncology/Cell Therapy, Rush University, Chicago, IL, USA

    Celalettin Ustun

  19. Department of Internal Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria

    Peter Valent

  20. Director of the Ludwig Boltzmann Institute for Hematology and Oncology, Medical University of Vienna, Vienna, Austria

    Peter Valent

  21. Coordinator of the European Competence Network on Mastocytosis (ECNM), Vienna, Austria

    Peter Valent

  22. Chair of the EBMT Practice Harmonization and Guidelines Committee. CHU de Lille, Univ Lille, INSERM U1286, Infinite, 59000, Lille, France

    Ibrahim Yakoub-Agha

  23. Department of Haematology, Guy’s and St. Thomas’ NHS Foundation Trust, London, UK

    Deepti H. Radia

Authors
  1. Donal P. McLornan
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  2. Tomasz Czerw
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  3. Gandhi Damaj
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  4. Mark Ethell
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  5. Carmelo Gurnari
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  6. Juan Carlos Hernández-Boluda
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  7. Nicola Polverelli
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  8. Juliana Schwaab
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  9. Katja Sockel
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  10. Greco Raffaella
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  11. Francesco Onida
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  12. Isabel Sánchez-Ortega
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  13. Giorgia Battipaglia
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  14. Chiara Elena
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  15. Jason Gotlib
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  16. Andreas Reiter
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  19. Peter Valent
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  21. Deepti H. Radia
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Contributions

DPM, DR, ISO, RG, FO and IYA designed the initial project concept. DPM, TC, GD, ME, CG, JHB, NP, JS, KS, IYA, ISO and DHR met for the two-day in-person workshop meeting in Lille. GB, CE, JG, AR, JR, CU and PV gave expert opinions on the first drafts, and wrote sections and summary points of topics raised. All authors contributed to the subsequent consensus development, contributed by writing sections and reviewing the article drafts. All authors reviewed and agreed on the final draft and agree to be accountable for all aspects of the work.

Corresponding author

Correspondence to Donal P. McLornan.

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Competing interests

There was no external funding associated with this consensus-based expert opinion paper. For each individual author, declarations of interest are as below: Donal P McLornan: Advisory boards and research funding from Novartis. Educational events on behalf of Novartis. Tomasz Czerw: No relevant disclosures. Gandhi Damaj: Travel expenses from Novartis and speakers bureau/ advisory boards on behalf of Blueprint Medicines Corporation. Mark Ethell: No relevant disclosures. Carmelo Gurnari: No relevant disclosures. Juan Carlos Hernández-Boluda: No relevant disclosures. Nicola Polverelli: No relevant disclosures. Juliana Schwaab: No relevant disclosures. Katja Sockel: Advisory boards and received lecture fees from Novartis, BMS/Celgene and Blueprint Medicines Corporation. Raffaella Greco: Discloses speaking honoraria from Biotest, Pfizer, Medac, Neovii and Magenta. Francesco Onida: No relevant disclosures. Isabel Sánchez-Ortega: No relevant disclosures. Giorgia Battipaglia: No relevant disclosures. Chiara Elena: Advisory Boards for Cogent Biosciences, Blueprint Medicines Corporation, Istituto Gentili. Jason Gotlib: Incyte: Consultancy, Honoraria, Research Funding; Deciphera: Consultancy, Honoraria, Research Funding; PharmaEssentia: Consultancy, Honoraria, Research Funding; Blueprint Medicines Corporation: Consultancy, Honoraria, Research Funding; Kartos: Honoraria, Advisory committees, Research Funding; BMS: Research Funding; Abbvie: Membership on an entity’s Board of Directors or advisory committees, Research Funding; Novartis: Consultancy, Honoraria, Membership on an entity’s Board of Directors or advisory committees, Research Funding; Cogent Biosciences: Consultancy, Research Funding; Allakos: Consultancy, Membership on an entity’s Board of Directors or advisory committees, Research Funding. Andreas Reiter: AbbVie: Consultancy, Honoraria, Research Funding; Blueprint Medicines Corporation: Consultancy, Honoraria, Research Funding; AOP Orphan Pharmaceuticals: Consultancy, Honoraria, Research Funding, Incyte Corporation: Consultancy, Honoraria, Research Funding; BMS: Consultancy, Honoraria, Research Funding; GSK: Consultancy, Honoraria, Research Funding; Novartis: Consultancy, Honoraria, Membership on an entity’s Board of Directors or advisory committees, Research Funding. Julien Rossignol: served on an advisory board for Blueprint Medicines Corporation; and has received research grants from Blueprint Medicines Corporation, BMS, and Novartis. Celalettin Ustun: Speaker Bureau honoraria: Takeda and Blueprint Medicines Corporation. Peter Valent: Project-Related: none. Project-Independent but Related to Mastocytosis in General: Ad-Board Honoraria: Novartis, Blueprint Medicines Corporation, Cogent. Ibrahim Yakoub-Agha: No relevant disclosures. Deepti H Radia: Clinical advisory board/study steering group member (EXPLORER/PATHFINDER) for Blueprint Medicines Corporation; a study steering committee member for Cogent Biosciences; and involved in educational events and advisory boards for Novartis. Author fees for Medscape cases. Royalties for Fast Facts: Systemic Mastocytosis.

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McLornan, D.P., Czerw, T., Damaj, G. et al. Allogeneic haematopoietic cell transplantation for advanced systemic mastocytosis: Best practice recommendations on behalf of the EBMT Practice Harmonisation and Guidelines Committee. Leukemia 38, 699–711 (2024). https://doi.org/10.1038/s41375-024-02182-1

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