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Publications

Journal Article Ahead of print

PubMed: 42463828 DOI: 10.1038/s41409-026-02947-5

Update of the decision-making algorithm on selecting transfusion-dependent β-thalassemic patients for gene therapy approaches: joint consensus report on behalf of EHA-specialized working group and EBMT hemoglobinopathies working party.

Bone Marrow Transplant

Authors: Lucia de Franceschi, Mahmoud Aljurf, Donatella Baronciani, Celeste Bento, Christian Chabannon, Selim Corbacioglu, Josu de la Fuente, Gian Luca Forni, Antonis Kattamis, Franco Locatelli, Roland Meisel, Ali Taher, Paul Telfer, Isabelle Thuret, Emanuele Angelucci

Affiliations

  • Department of Engineering for the Innovative Medicine, University of Verona & AOUI, Verona, Italy.
  • Adult Hematology/HSCT, Oncology Center, King Faisal Specialist Hospital & Research Centre, Riyadh, Saudi Arabia.
  • UOC Immunoematologia e Trasfusionale, AORMN, Pesaro-Fano, Italy.
  • Eritropathology and Iron Metabolism Unit, Hematology Department, University Hospital Coimbra, Portugal and Research Centre for Anthropology and Health (CIAS), University of Coimbra, Coimbra, Portugal.
  • Institut Paoli-Calmettes Comprehensive Cancer Centre and Module Biothérapies du Centre d'Investigations Cliniques de Marseille, INSERM-Aix-Marseille Université-AP-HM-IPC, Marseille, France.
  • Department of Paediatric Haematology, Oncology and Stem Cell Transplantation, University of Regensburg, Regensburg, Germany.
  • Imperial College Healthcare NHS Trust, London, UK.
  • Fondazione FORANEMIA, Genova, Italy.
  • Division of Paediatric Haematology-Oncology, First Department of Paediatrics, National and Kapodistrian University of Athens, Athens, Greece.
  • IRCCS Ospedale Pediatrico Bambino Gesù and Catholic University of the Sacred Heart, Rome, Italy.
  • Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology and Clinical Immunology, Medical Faculty, Heinrich-Heine-University, Duesseldorf, Germany.
  • Division of Hematology & Oncology, Department of Internal Medicine American University of Beirut Medical Center (AUBMC), Beirut, Lebanon.
  • Department of Paediatric Haematology and Oncology, St. Bartholomew's and The Royal London Hospitals NHS Trust, London, UK.
  • CHU de Marseille-Hôpital de la Timone, Marseille, France.
  • Azienda Ospedaliera Metropolitana-IRCCS Ospedale Policlinico, San Martino, Genova, Italy.

Abstract

β-thalassemia is a globally distributed hereditary red blood cell disorder. Up to now, clinical management of transfusion-dependent β-thalassemia (TDT) patients is still based on chronic transfusion combined with iron chelation therapy. Allogeneic hematopoietic cell transplantation potentially provides a cure, but few patients have an HLA-identical sibling, and optimal results are reported in patients ≤ 14 years. The European Hematology Association (EHA), through the EHA Scientific Working Group on Red Cells/Iron and the European Bone Marrow Transplantation (EBMT) group, has updated a 2021 EHA decision-making algorithm on evidence and expert consensus with the aim of identifying which patients with TDT could benefit from gene therapy (GT). Indeed, it is important to establish the patient setting for whom it is a priority, particularly in the early phase of real-world use outside experimental trials. Moreover, actual price, limited availability, and resource disposal constitute a further indication of a rational and progressive approach to this innovative treatment. In this expert consensus document, different clinical scenarios have been considered and analyzed for the possible impact on treatment outcome. This expert opinion provides dynamic, updatable, priority-based guidance for physicians taking care of TDT patients.

MeSH

Cells, Genes, Decision Making, Algorithms, Thalassemia, Therapeutics, Hemoglobinopathies