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Publications

Journal Article Epublish 1 citation

PubMed: 40084235 DOI: 10.1002/hem3.70089

Selecting patients with sickle cell disease for gene addition or gene editing-based therapeutic approaches: Report on behalf of a joint EHA Specialized Working Group and EBMT Hemoglobinopathies Working Party consensus conference.

Hemasphere 9(3):e70089

Authors: Lucia de Franceschi, Franco Locatelli, David Rees, Christian Chabannon, Jean-Hugues Dalle, Stefano Rivella, Achille Iolascon, Stephan Lobitz, Miguel R Abboud, Josu de la Fuente, Pagona Flevari, Emanuele Angelucci, Mariane de Montalembert

Affiliations

  • Department of Engineering for Innovative Medicine University of Verona Verona Italy.
  • Azienda Ospedaliera Universitaria integrata di Verona Verona Italy.
  • IRCCS Bambino Gesù Children's Hospital Catholic University of the Sacred Heart Rome Italy.
  • School of Cancer and Pharmaceutical Sciences, Faculty of Life Sciences Medicine, King's College London, and Department of Haematological Medicine King's College Hospital London UK.
  • Institut Paoli-Calmettes Comprehensive Cancer Center and Module Biotherapies du Centre d'Investigations Cliniques de Marseille, INSERM-Aix-Marseille Université AP-HM-IPC CBT-1409 Marseille France.
  • Pediatric Hematology and Immunoloy Department, Robert-Debré Academic Hospital GHU AP-HP Nord Université Paris Cité Paris France.
  • Department of Pediatrics Hematology, The Children's Hospital of Philadelphia Philadelphia Pennsylvania USA.
  • Penn Institute for RNA Innovation University of Pennsylvania Philadelphia Pennsylvania USA.
  • Penn Institute for Regenerative Medicine (IRM) University of Pennsylvania Philadelphia Pennsylvania USA.
  • Dipartimento di Medicina Molecolare e Biotecnologie Mediche Università degli Studi di Napoli Federico II Naples Italy.
  • CEINGE Biotecnologie Avanzate Naples Italy.
  • Pediatric Hematology & Oncology, Gemeinschaftsklinikum Mittelrhein Koblenz Germany.
  • Department of Pediatrics and Adolescent Medicine American University of Beirut Beirut Lebanon.
  • Department of Immunology and Inflammation Centre for Haematology, Imperial College London London UK.
  • Department of Paediatrics Imperial College Healthcare NHS Trust London UK.
  • Thalassemia Unit-Center of Expertise in Haemoglobinopathies, Laiko General Hospital Athens Greece.
  • UO Ematologia e Terapie Cellulari, IRCCS Ospedale Policlinico San Martino Genova Italy.
  • Department of General Pediatrics and Pediatric Infectious Diseases, Sickle Cell Center, Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP) Université Paris Cité Paris France.
  • Laboratory of Excellence GR-Ex Paris France.

Abstract

Sickle cell disease (SCD) remains associated with reduced life expectancy and poor quality of life despite improvements observed in the last decades mostly related to comprehensive care, use of hydroxycarbamide, screening to identify patients at risk of strokes, and implementation of safe transfusion protocols. The course of the disease is highly variable, making it difficult to predict severity and response to therapy. Allogeneic hematopoietic stem cell transplantation potentially provides a cure with a relatively low rate of complications, but few patients have an HLA-identical sibling. The hopes of patients and healthcare providers have been raised after the initial excellent results of gene therapy studies. However, there is a strong contrast between the high expectations of families and patients and the limited availability of the product, which is technically complex and very expensive. In light of this consideration and of the limited data available on the long-term efficacy and toxicity of different gene therapy approaches, the European Hematology Association Red Cell & Iron Specialized Working Group (EHA SWG) and the hemoglobinopathy working part of the European Blood & Marrow Transplant (EBMT) Group have prioritized the development of recommendations for selection of patients with SCD who are good candidates for gene therapy. The decision-making algorithm was developed by a panel of experts in hemoglobinopathies and/or transplantation chosen by EHA SWG and EBMT, to discuss the selection of SCD patients for gene therapy and draw notes on the related clinical problems.

MeSH

Cells, Genes, Anemia, Sickle Cell, Gene Editing, Transplants, Therapeutics, Hemoglobinopathies, Transplantation