Fitzhugh Courtney D, Volanakis Emmanuel J, Idassi Ombeni, Duberman Josh A, DeBaun Michael R, Friedman Debra L
Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute, NIH, Building 10, MSC 1589, Bethesda, MD 20814, USA.
Department of Pediatrics, Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease, Vanderbilt University Medical Center, 2525 West End Ave., Suite 750 VIGH, Nashville, TN 37203, USA.
J Clin Med. 2022 May 31;11(11):3118. doi: 10.3390/jcm11113118.
The goal of curing children and adults with sickle cell disease (SCD) is to maximize benefits and minimize intermediate and long-term adverse outcomes so that individuals can live an average life span with a high quality of life. While greater than 2000 individuals with SCD have been treated with curative therapy, systematic studies have not been performed to evaluate the long-term health effects of hematopoietic stem cell transplant (HSCT) in this population. Individuals with SCD suffer progressive heart, lung, and kidney disease prior to curative therapy. In adults, these sequalae are associated with earlier death. In comparison, individuals who undergo HSCT for cancer are heavily pretreated with chemotherapy, resulting in potential acute and chronic heart, lung, and kidney disease. The long-term health effects on the heart, lung, and kidney for children and adults undergoing HSCT for cancer have been extensively investigated. These studies provide the best available data to extrapolate the possible late health effects after curative therapy for SCD. Future research is needed to evaluate whether HSCT abates, stabilizes, or exacerbates heart, lung, kidney, and other diseases in children and adults with SCD receiving myeloablative and non-myeloablative conditioning regimens for curative therapy.
治愈镰状细胞病（SCD）患儿和成人的目标是使益处最大化，并将中期和长期不良后果降至最低，以便患者能够过上平均寿命的高质量生活。虽然已有超过2000名SCD患者接受了治愈性治疗，但尚未进行系统性研究来评估造血干细胞移植（HSCT）对该人群的长期健康影响。在接受治愈性治疗之前，SCD患者会逐渐出现心脏、肺部和肾脏疾病。在成人中，这些后遗症与过早死亡有关。相比之下，因癌症接受HSCT的患者会接受大量化疗预处理，这会导致潜在的急慢性心脏、肺部和肾脏疾病。针对因癌症接受HSCT的儿童和成人的心脏、肺部和肾脏的长期健康影响已进行了广泛研究。这些研究提供了现有最佳数据，可据此推断SCD治愈性治疗后可能出现的晚期健康影响。未来需要开展研究，以评估接受清髓性和非清髓性预处理方案进行治愈性治疗的SCD儿童和成人中，HSCT是否能减轻、稳定或加重心脏、肺部、肾脏及其他疾病。
