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New Recommendations from the EBMT Practice Harmonisation and Guidelines (PH&G) Committee

Autoimmune Diseases Working Party (ADWP)
Chronic Malignancies Working Party (CMWP)
Practice Harmonisation Committee

The EBMT is pleased to announce three ADWP and CMWP publications on behalf of the EBMT Practice Harmonisation and Guidelines Committee. See below:

Innovative cellular therapies for autoimmune diseases: expert-based position statement and clinical practice recommendations from the EBMT practice harmonization and guidelines committee

EClinicalMedicineOpen Access - Published: February 09, 2024 - DOI:

Authors: Raffaella Greco, Tobias Alexander, Nicoletta Del Papa, Fabian Müller, Riccardo Saccardi, Fermin Sanchez-Guijo, Georg Schett, Basil Sharrack, John A. Snowden, Karin Tarte, Francesco Onida, Isabel Sánchez-Ortega, Joachim Burman, Cristina Castilla Llorente, Ricard Cervera, Fabio Ciceri, Andrea Doria, Jörg Henes, James Lindsay, Andreas Mackensen, Paolo A. Muraro, Elena Ricart, Montserrat Rovira, Tsila Zuckerman, Ibrahim Yakoub-Agha, Dominique Farge

Autoimmune diseases (ADs) are a heterogeneous group of diseases, characterised by loss of immune tolerance, high chronicity, with substantial morbidity and mortality. Over the last three decades, hematopoietic stem cell transplantation (HCT) has been successfully used to treat specific subgroups of patients affected by severe and refractory ADs. Recently, innovative cellular therapies (CT), including mesenchymal stromal cells (MSC), Chimeric Antigen Receptors T cells (CART) and regulatory T cells (Tregs), have been developed and adopted in ADs.

To gather more insights into this issue, an international expert panel of the European Society for Blood and Marrow Transplantation (EBMT) and the International Society for the Cell and Gene Therapy (ISCT), including representation from the Joint Accreditation Committee of ISCT and EBMT (JACIE) met in a series of virtual conferences, culminating in a 2-day meeting in Lille, France. On the basis of these deliberations, expert-based consensus and recommendations for best practice and quality of patient care were developed to support clinicians, scientists, and their multidisciplinary teams, as well as patients and care providers. These delineate detailed indications, contraindications and areas for caution for each AD, comprehensive recommendations on diagnostic work-up, clinical management and immune monitoring protocols for these innovative CTs.

These recommendations aim to support national and international stakeholders in the field of ADs and local clinical teams delivering such care. They reflect currently available evidence, coupled with expert opinion, and will be regularly updated.


Allogeneic Haematopoietic Cell Transplantation for Advanced Systemic Mastocytosis: Best Practice Recommendations on behalf of the EBMT Practice Harmonisation and Guidelines Committee

Leukemia - To be published soon

Authors: Donal P McLornan, Tomasz Czerw, Gandhi Damaj. Mark Ethell, Carmelo Gurnari, Juan Carlos Hernández-Boluda, Nicola Polverelli, Juliana Schwaab, Katja Sockel, Raffaella Greco, Francesco Onida, Isabel Sánchez-Ortega, Giorgia Battipaglia, Chiara Elena, Jason Gotlib, Andreas Reiter, Julien Rossignol, Celalettin Ustun, Peter Valent,Ibrahim Yakoub-Agha and Deepti H Radia.

Advanced Systemic Mastocytosis (AdvSM) is a complex and heterogeneous disease. Marked advances have been made in understanding this complexity leading to wider availability of novel therapeutic options; in particular implementation of two KIT-targeting kinase inhibitors: midostaurin and avapritinib.

Following formulation of an expert international panel with a specialist interest in allo-HCT and mastocytosis, best practice recommendations were generated according to the European Society for Blood and Marrow Transplantation (EBMT) Practice Harmonisation and guidelines and ECNM methodology.

These guidelines will address all aspects of allo-HCT for Adv SM including detail of which patients should be considered for allo-HCT and optimal timing, the management and optimisation of KIT inhibitors, discussion on how these agents may fit into the transplant algorithm, both pre- and post, to optimise outcomes and what investigations should be performed prior to transplant. In addition, we discuss how best to monitor the mast cell component in the post allo-HCT setting and where maintenance with some of the novel agents should be considered. Overall, we provide a clinically relevant and practical framework to guide clinicians when dealing with allo-HCT in AdvSM in 2024 and beyond.

These guidelines will be regularly updated when new developments occur and when further data is available. We will be discussing this guideline in the Harmonisation session at the Annual Meeting in Glasgow 2024 and hope to see you there!

Management of adult patients with CMML undergoing allogeneic hematopoietic cell transplantation: recommendations from the EBMT Practice Harmonisation and Guidelines Committee

Blood - To be published soon

Authors: Francesco Onida, Nico Gagelmann, Yves Chalandon, Guido Kobbe, Marie Robin, Argiris Symeonidis, Theo de Witte, Raphael Itzykson, Madlen Jentzsch, Uwe Platzbecker, Valeria Santini, Guillermo Sanz, Christof Scheid, Eric Solary, Peter Valent, Raffaela Greco, Isabel Sanchez-Ortega, Ibrahim Yakoub-Agha and Lisa Pleyer.

Chronic myelomonocytic leukemia (CMML) is a heterogeneous disease presenting with either myeloproliferative or myelodysplastic features. Allogeneic hematopoietic cell transplantation (allo-HCT) remains the only potentially curative option, but the inherent toxicity of this procedure makes the decision to proceed to allo-HCT challenging, particularly as patients with CMML are mostly older and comorbid. Therefore, the decision between a non-intensive treatment approach and allo-HCT represents a delicate balance, especially since prospective randomized studies are lacking and retrospective data in the literature is conflicting.

Moreover, in the context of allo-HCT, the absence of prospective data drags behind many uncertainties not only regarding patient selection, but also possible pre-transplant treatment strategies, the timing of allo-HCT, the optimal overall transplant policy and patient management in the post-transplant setting.

Thus, the EBMT Practice Harmonisation and Guidelines Committee assembled a panel of experts in the field to provide the first best practice recommendations on the role of allo-HCT specifically in CMML.

Algorithms for patient selection, timing of allo-HCT during the course of the disease, pre-transplant strategies, allo-HCT modality including donor selection, stem cell source and conditioning intensity as well as post-transplant management for patients with CMML were outlined and a set of best practice recommendations delivered, providing a clinically relevant and practical framework to guide clinicians when dealing with allo-HCT in CMML.

We will be discussing these guidelines during the Practice Harmonisation Day (Tuesday 16 April) at the Annual Meeting in Glasgow 2024. Hope to see you there!