Leung Wing, Ahn Hyunah, Rose Susan R, Phipps Sean, Smith Teresa, Gan Kwan, O'Connor Madeline, Hale Gregory A, Kasow Kimberly A, Barfield Raymond C, Madden Renee M, Pui Ching-Hon
From Department of Hematology-Oncology (WL, HA, TS, KG, MOC, GAH, KAK, RCB, RMM, CHP) and Division of Behavioral Medicine (SP), St. Jude Children's Research Hospital, Memphis, Tennessee; and Department of Pediatrics (WL, GAH, KAK, RCB, RMM, CHP) and Division of Endocrinology (SRR), University of Tennessee College of Medicine, Memphis, Tennessee.
Medicine (Baltimore). 2007 Jul;86(4):215-224. doi: 10.1097/MD.0b013e31812f864d.
As survivors of pediatric allogeneic hematopoietic stem cell transplantations (HSCTs) increase in number, it is increasingly important to evaluate their well-being. We conducted this prospective cohort study to evaluate the cumulative incidence and risk factors for late sequelae of HSCT. Comprehensive surveillance tests were performed annually on every participant, regardless of signs and symptoms, to obtain accurate information on the time-of-onset of each late event to allow hazard function analyses. All participants included in this report had been followed for at least 3 years after HSCT. With a median follow-up of 9 years and a current age of 18.5 years, only 20 of the 155 participants (13%) had no late sequelae; 18 survivors (12%) had 1 chronic health condition, 71 (46%) had 2-4 conditions, and 46 (30%) had 5-9 conditions. Risk factors for increasing number of chronic conditions included young age at the time of HSCT, female sex, high radiation dose, and history of chronic graft-versus-host disease. The cumulative incidence at 10 years for common late events was as follows (ordered by the median time-of-onset): osteonecrosis 13.8%, chronic renal insufficiency 26.8%, hypothyroidism 45.1%, growth hormone deficiency 31.2%, female hypogonadism 57.4%, osteopenia 47.7%, cataracts 43.4%, pulmonary dysfunction 63.2%, and male hypogonadism 20.3%. Coexistence of multiple late sequelae was common in HSCT survivors. Our findings provide a basis for more effective patient counseling, optimal surveillance, and early intervention.
随着小儿异基因造血干细胞移植（HSCT）幸存者数量的增加，评估他们的健康状况变得越来越重要。我们进行了这项前瞻性队列研究，以评估HSCT晚期后遗症的累积发生率和风险因素。每年对每位参与者进行全面的监测检查，无论有无症状和体征，以获取每个晚期事件发病时间的准确信息，以便进行风险函数分析。本报告纳入的所有参与者在HSCT后至少随访了3年。中位随访时间为9年，当前年龄为18.5岁，155名参与者中只有20名（13%）没有晚期后遗症；18名幸存者（12%）有一种慢性健康状况，71名（46%）有2 - 4种状况，46名（30%）有5 - 9种状况。慢性病数量增加的风险因素包括HSCT时年龄小、女性、高辐射剂量以及慢性移植物抗宿主病病史。常见晚期事件在10年时的累积发生率如下（按中位发病时间排序）：骨坏死13.8%、慢性肾功能不全26.8%、甲状腺功能减退45.1%、生长激素缺乏31.2%、女性性腺功能减退57.4%、骨质减少47.7%、白内障43.4%、肺功能障碍63.2%、男性性腺功能减退20.3%。多种晚期后遗症共存在HSCT幸存者中很常见。我们的研究结果为更有效的患者咨询、优化监测和早期干预提供了依据。
