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
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
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引用次数: 0
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.
镰状细胞病(SCD)仍然与预期寿命缩短和生活质量差有关,尽管在过去几十年中观察到的改善主要与综合护理、使用羟基脲、筛查卒中风险患者以及实施安全输血方案有关。这种疾病的病程变化很大,因此很难预测其严重程度和对治疗的反应。同种异体造血干细胞移植可能提供一种并发症发生率相对较低的治疗方法,但很少有患者有相同hla的兄弟姐妹。在基因治疗研究取得初步优异成果后,患者和医疗保健提供者的希望被提了出来。然而,家庭和患者的高期望与产品的有限供应形成了强烈的对比,该产品技术复杂且非常昂贵。考虑到这一点,以及关于不同基因治疗方法的长期疗效和毒性的有限数据,欧洲血液协会(European Hematology Association Red Cell &;铁专门工作组(EHA SWG)和血红蛋白病工作部分欧洲血液&;骨髓移植(EBMT)小组优先发展推荐选择SCD患者谁是良好的候选基因治疗。决策算法由EHA SWG和EBMT选择的血红蛋白病和/或移植专家小组开发,讨论SCD患者基因治疗的选择,并对相关临床问题进行记录。
期刊介绍:
HemaSphere, as a publication, is dedicated to disseminating the outcomes of profoundly pertinent basic, translational, and clinical research endeavors within the field of hematology. The journal actively seeks robust studies that unveil novel discoveries with significant ramifications for hematology.
In addition to original research, HemaSphere features review articles and guideline articles that furnish lucid synopses and discussions of emerging developments, along with recommendations for patient care.
Positioned as the foremost resource in hematology, HemaSphere augments its offerings with specialized sections like HemaTopics and HemaPolicy. These segments engender insightful dialogues covering a spectrum of hematology-related topics, including digestible summaries of pivotal articles, updates on new therapies, deliberations on European policy matters, and other noteworthy news items within the field. Steering the course of HemaSphere are Editor in Chief Jan Cools and Deputy Editor in Chief Claire Harrison, alongside the guidance of an esteemed Editorial Board comprising international luminaries in both research and clinical realms, each representing diverse areas of hematologic expertise.