Kwiatkowski Janet L
Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
Department of Pediatrics, Perelman School of Medicine of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Ann N Y Acad Sci. 2023 Dec;1530(1):105-109. doi: 10.1111/nyas.15070. Epub 2023 Oct 13.
Individuals with transfusion-dependent beta thalassemia require a high burden of care and experience significant morbidity from the underlying disease and its treatment, which negatively impact the quality of life. Allogeneic hematopoietic stem cell transplantation offers the chance for a cure, but donor availability and transplant-related risks, especially in older patients, limit its use. Gene addition utilizing autologous CD34 cells is an alternative, potentially curative, treatment option. Several clinical trials have investigated the use of lentiviral vectors containing a functional beta globin gene, including Lentiglobin BB305, GLOBE, and TNS9.3.55. The efficacy and safety data from these ongoing trials are discussed in this review.
依赖输血的β地中海贫血患者需要承担高额的护理负担，并因基础疾病及其治疗而出现显著的发病率，这对生活质量产生负面影响。异基因造血干细胞移植提供了治愈的机会，但供体可用性和移植相关风险，尤其是在老年患者中，限制了其应用。利用自体CD34细胞进行基因添加是一种替代性的、潜在治愈性的治疗选择。多项临床试验研究了含有功能性β珠蛋白基因的慢病毒载体的应用，包括Lentiglobin BB305、GLOBE和TNS9.3.55。本综述讨论了这些正在进行的试验的疗效和安全性数据。
