Gurney James G, Ness Kirsten K, Rosenthal Joseph, Forman Stephen J, Bhatia Smita, Baker K Scott
Department of Pediatrics, Division of General Pediatrics, 300 NIB 6E02 Box 0456, University of Michigan, 300 N. Ingalls Street, Ann Arbor, MI 48109, USA.
Cancer. 2006 Mar 15;106(6):1402-8. doi: 10.1002/cncr.21752.
Because of treatment-related toxicity, research is increasingly being focused on long-term sequelae secondary to hematopoietic stem cell transplantation (HSCT) in survivor populations.
This study describes the incidence of auditory, sensory, motor, and visual impairments, including cataracts, among 235 individuals who were treated with HSCT during childhood or adolescence. Outcomes were compared with 705 siblings of childhood cancer survivors. Participants completed a survey with questions on posttransplant organ system impairments. Approximately half of survivors were transplanted when younger than 10 years of age. The median length of followup was 11 years.
The cumulative incidence of cataracts was 36% at 15 years post-HSCT, although cataracts occurred only in those who received total body irradiation as an HSCT conditioning agent or head irradiation before transplant. Persistent pain was reported by 21% of survivors. Loss of hearing in one or both ears, and legal blindness in one or both eyes, each occurred after transplant in 2% of survivors. Occurrences were uncommon, but survivors were 4.3 times (95% confidence interval [CI]: 2.0-9.4) more likely to report coordination problems, 7.7 times (95% CI: 3.2-18.5) more likely to report chewing or swallowing problems, and 3.5 times (3.5; 95% CI: 1.6-7.9) more likely to report muscle weakness than those in the comparison group. Muscle weakness was strongly associated with positive history of chronic graft-versus-host disease.
Increased risks were found for motor impairments, hearing loss, vision loss, and persistent pain among study participants. Cataracts were a frequent adverse effect, suggesting that close monitoring with appropriate intervention for preservation of vision, particularly among those who received total body irradiation, should be a primary goal in survivors of HSCT performed in childhood.
由于与治疗相关的毒性作用，针对幸存者群体中造血干细胞移植（HSCT）继发的长期后遗症的研究日益受到关注。
本研究描述了235名在儿童期或青少年期接受HSCT治疗的个体中听觉、感觉、运动和视觉障碍（包括白内障）的发生率。将结果与705名儿童癌症幸存者的兄弟姐妹进行比较。参与者完成了一项关于移植后器官系统损伤的问卷调查。大约一半的幸存者在10岁之前接受了移植。中位随访时间为11年。
HSCT后15年白内障的累积发生率为36%，不过白内障仅发生在那些接受全身照射作为HSCT预处理剂或移植前接受头部照射的个体中。21%的幸存者报告有持续性疼痛。移植后，2%的幸存者出现单耳或双耳听力丧失以及单眼或双眼法定失明。这些情况并不常见，但与对照组相比，幸存者报告协调问题的可能性高4.3倍（95%置信区间[CI]：2.0 - 9.4），报告咀嚼或吞咽问题的可能性高7.7倍（95% CI：3.2 - 18.5），报告肌肉无力的可能性高3.5倍（3.5；95% CI：1.6 - 7.9）。肌肉无力与慢性移植物抗宿主病的阳性病史密切相关。
研究参与者中运动障碍、听力丧失、视力丧失和持续性疼痛的风险增加。白内障是一种常见的不良反应，这表明对HSCT幸存者，尤其是那些接受全身照射的幸存者，进行密切监测并采取适当干预措施以保护视力应成为主要目标。
