A phase II trial to assess Haploidentical a/ß T-depleted stem cell transplantation in patients with sickle cell disease with no available sibling donor - Acronym: T-HAPLO-SCT for SCD

一项 II 期试验，用于评估在没有可用同胞供体的镰状细胞病患者中进行单倍相合 a/à T 耗尽干细胞移植 - 缩写：T-HAPLO-SCT for SCD

• 批准号: 375203949
• 负责人: Professor Dr. Selim Corbacioglu
• 金额: --
• 依托单位: Klinik und Poliklinik für Kinder- und Jugendmedizin
• 依托单位国家: 德国
• 项目类别: Clinical Trials
• 财政年份: 2019
• 资助国家: 德国
• 起止时间: 2018-12-31 至 2023-12-31
• 项目状态: 已结题
• 来源: https://gepris.dfg.de/gepris/projekt/375203949?language=en
• 关键词: phase; II; trial; assess; Haploidentical

Sickle cell disease (SCD) represents a worldwide health problem with over 300,000 affected children born each year and remains a disease with high morbidity and mortality. Migration makes SCD an increasingly eminent problem of western countries. The disease is overtaking haemophilia and cystic fibrosis in certain parts of Europe and is a progressive chronic multi-organ disease. Stroke is the major debilitating complication of SCD and the most frequent cause of stroke in children. 30% develop silent strokes leading to cognitive impairment in children and poor quality of life. Matched sibling donor (MSD) hematopoietic stem cell transplantation (HSCT) is the gold standard and the only curative treatment available, with a 90% survival without graft failure at 5 years. But MSD are available for only 20% of patients and HSCT with unrelated donors resulted in unacceptably high rates of graft-versus-host disease (GvHD). T-cell depleted haploidentical HSCT (T-Haplo-HSCT) from relatives, mostly parents, but also haplo-matched siblings and cousins could offer cure to the remaining 80% of affected patients. This trial aims to demonstrate that a haploidentical stem cell transplantation from a relative is equivalent to a sibling donor HSCT with regard to outcome and treatment related morbidity and mortality. Patients with an established indication for HSCT will be stratified and will receive a sibling donor transplantation if available. If not, the patient will receive a transplant from a haploidentical relative. With the exception of the stem cell source, both groups will receive an almost identical treatment. We expect to show that T-Haplo-HSCT is safe and can achieve a comparable outcome to a MSD transplantation with regard to overall, disease-free and GvHD-free survival. The intention is, to offer cure to every patient affected with sickle cell disease.

镰状细胞病（SCD）是一个世界性的健康问题，每年有超过30万名受影响的儿童出生，并且仍然是一种具有高发病率和死亡率的疾病。移民使SCD成为西方国家日益突出的问题。在欧洲某些地区，该病正在超过血友病和囊性纤维化，是一种进行性慢性多器官疾病。卒中是SCD的主要衰弱并发症，也是儿童卒中的最常见原因。30%的人会出现无症状中风，导致儿童认知障碍和生活质量下降。匹配的同胞供体（MSD）造血干细胞移植（HSCT）是金标准，也是唯一可用的治愈性治疗，5年无移植失败的存活率为90%。但是，MSD仅适用于20%的患者，并且与无关供体的HSCT导致不可接受的高移植物抗宿主病（GvHD）发生率。来自亲属（主要是父母）的T细胞耗尽的单倍体相合HSCT（T-Haplo-HSCT），但也可以为其余80%的受影响患者提供治疗。本试验旨在证明亲属单倍相合干细胞移植在结局和治疗相关发病率和死亡率方面等同于同胞供体HSCT。将对具有确定的HSCT适应症的患者进行分层，并将接受同胞供体移植（如可用）。如果没有，患者将接受来自单倍相合亲属的移植。除了干细胞来源外，两组将接受几乎相同的治疗。我们期望证明T-Haplo-HSCT是安全的，并且在总体、无疾病和无GvHD生存率方面可以达到与MSD移植相当的结果。其目的是为每一位患有镰状细胞病的患者提供治疗。