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
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 t...
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2025-03-01
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| Series: | HemaSphere |
| Online Access: | https://doi.org/10.1002/hem3.70089 |
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| author | 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 |
| author_facet | 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 |
| author_sort | Lucia de Franceschi |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-656f25d7dbbc4ae09e982134af816de7 |
| institution | Kabale University |
| issn | 2572-9241 |
| language | English |
| publishDate | 2025-03-01 |
| publisher | Wiley |
| record_format | Article |
| series | HemaSphere |
| spelling | doaj-art-656f25d7dbbc4ae09e982134af816de72025-08-20T03:42:25ZengWileyHemaSphere2572-92412025-03-0193n/an/a10.1002/hem3.70089Selecting 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 conferenceLucia de Franceschi0Franco Locatelli1David Rees2Christian Chabannon3Jean‐Hugues Dalle4Stefano Rivella5Achille Iolascon6Stephan Lobitz7Miguel R. Abboud8Josu de la Fuente9Pagona Flevari10Emanuele Angelucci11Mariane de Montalembert12Department of Engineering for Innovative Medicine University of Verona Verona ItalyIRCCS Bambino Gesù Children's Hospital Catholic University of the Sacred Heart Rome ItalySchool of Cancer and Pharmaceutical Sciences, Faculty of Life Sciences Medicine, King's College London, and Department of Haematological Medicine King's College Hospital London UKInstitut 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 FrancePediatric Hematology and Immunoloy Department, Robert‐Debré Academic Hospital GHU AP‐HP Nord Université Paris Cité Paris FranceDepartment of Pediatrics Hematology, The Children's Hospital of Philadelphia Philadelphia Pennsylvania USADipartimento di Medicina Molecolare e Biotecnologie Mediche Università degli Studi di Napoli Federico II Naples ItalyPediatric Hematology & Oncology, Gemeinschaftsklinikum Mittelrhein Koblenz GermanyDepartment of Pediatrics and Adolescent Medicine American University of Beirut Beirut LebanonDepartment of Immunology and Inflammation Centre for Haematology, Imperial College London London UKThalassemia Unit—Center of Expertise in Haemoglobinopathies, Laiko General Hospital Athens GreeceUO Ematologia e Terapie Cellulari, IRCCS Ospedale Policlinico San Martino Genova ItalyDepartment 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 FranceABSTRACT 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.https://doi.org/10.1002/hem3.70089 |
| spellingShingle | 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 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 |
| title | 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 |
| title_full | 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 |
| title_fullStr | 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 |
| title_full_unstemmed | 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 |
| title_short | 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 |
| title_sort | 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 |
| url | https://doi.org/10.1002/hem3.70089 |
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