Global perspectives on cellular therapy for children with sickle cell disease.
Global perspectives on cellular therapy for children with sickle cell disease.
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DOI：
10.1097/moh.0000000000000738
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发表时间：
2022-11-01
影响因子：
3.2
通讯作者：
Tubman, Venee N.
中科院分区：
文献类型：
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作者：
John, Tami D.;Namazzi, Ruth;Chirande, Lulu;Tubman, Venee N.
低收入和中等收入国家 (LMIC)，主要是撒哈拉以南非洲 (SSA)，在全世界范围内承受着镰状细胞病 (SCD) 的负担。急性和慢性并发症的高频率导致医疗保健的利用率增加，这给脆弱的卫生系统带来了负担。医疗服务有限的儿童死亡率仍然高得惊人。基于细胞的疗法，例如同种异体造血干细胞移植 (HSCT)，越来越多地在资源丰富的环境中用作 SCD 的治疗方法。 SSA 中 SCD 的广泛治疗将极大地改变该疾病的全球影响。目前，基于细胞的疗法在中低收入国家的应用受到成本、人员以及 HSCT 特定技术和支持护理的可用性的限制。尽管面临挑战，针对 SCD 的 HSCT 仍在中低收入国家取得进展。备受期待的基因修饰疗法最近在资源丰富的国家的临床试验中被证明是安全可行的，但获得途径仍然极其有限。针对 SCD 的基于细胞的治愈性疗法的转化应优先考虑到中低收入国家，因为这些国家的疾病负担和非治疗性治疗的成本很高，而且长期生活质量很差。专注于对当前和未来疗法进行深思熟虑的修改，以满足中低收入国家（尤其是SSA）的需求，将特别具有影响力。
Low- and middle-income countries (LMICs), primarily in sub-Saharan Africa (SSA), experience the burden of sickle cell disease (SCD) worldwide. High frequency of acute and chronic complications leads to increased utilization of healthcare, which burdens fragile health systems. Mortality for children with limited healthcare access remains alarmingly high. Cellular-based therapies such as allogeneic hematopoietic stem cell transplant (HSCT) are increasingly used in resource-rich settings as curative therapy for SCD. Broad access to curative therapies for SCD in SSA would dramatically alter the global impact of the disease. Currently, application of cellular-based therapies in LMICs is limited by cost, personnel, and availability of HSCT-specific technologies and supportive care. Despite the challenges, HSCT for SCD is moving forward in LMICs. Highly-anticipated gene modification therapies have recently proven safe and feasible in clinical trials in resource-rich countries, but access remains extremely limited. Translation of curative cellular-based therapies for SCD should be prioritized to LMICs where the disease burden and cost of non-curative treatments is high, and long-term quality of life is poor. Focus on thoughtful modifications of current and future therapies to meet the need in LMICs, especially in SSA, will be especially impactful.