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

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.

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Publication Details

Journal
HemaSphere
Published
2025-03-01
DOI
https://doi.org/10.1002/hem3.70089
Citations
4
Primary Topic
Hemoglobinopathies and Related Disorders
Type
article
Field-Weighted Citation Impact
6.50
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article

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

Josu de la Fuente, Mariane de Montalembert, Jean‐Hugues Dalle, Franco Locatelli et al.
4 citations
HemaSphere
Hemoglobinopathies and Related Disorders
6.50
article

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

Josu de la Fuente, Mariane de Montalembert, Jean‐Hugues Dalle, Franco Locatelli, Achille Iolascon, David C. Rees, Lucia De Franceschi, Stefano B. Rivella, Christian Chabannon, Stephan Lobitz, Emanuele Angelucci, Miguel R. Abboud, Pagona Flevari
article en
4 citations

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.

HemaSphereVol. 9(3)
Università Cattolica del Sacro Cuore (IT), University of Verona (IT), Children's Hospital of Philadelphia (US), Imperial College Healthcare NHS Trust (GB), Inserm (FR), King's College London (GB), Université Paris Cité (FR), Aix-Marseille Université (FR), Laboratory of Excellence GR-Ex (FR), Azienda Ospedaliera Universitaria Integrata Verona (IT), Assistance Publique – Hôpitaux de Paris (FR), Laiko General Hospital of Athens (GR), Bambino Gesù Children's Hospital (IT), Penn Center for AIDS Research (US), CEINGE Biotecnologie Avanzate Franco Salvatore (Italy) (IT), Centre de Recherche en Cancérologie de Marseille (FR), Ospedale Policlinico San Martino (IT), Gemeinschaftsklinikum Mittelrhein (DE), King's College Hospital (GB), Imperial College London (GB), University of Naples Federico II (IT), University of Pennsylvania (US), American University of Beirut (LB)
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Openalex Percentile: Top 4%
Hemoglobinopathies and Related Disorders
6.50
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