Ji Yi, Yang Kaiying, Chen Siyuan, Peng Suhua, Lu Guoyan, Liu Xingtao
Division of Oncology, Department of Pediatric Surgery, West China Hospital of Sichuan University, Chengdu, China,
Pediatric Intensive Care Unit, Department of Critical Care Medicine, West China Hospital of Sichuan University, Chengdu, China,
Cancer Manag Res. 2018 Sep 7;10:3325-3331. doi: 10.2147/CMAR.S171223. eCollection 2018.
Musculoskeletal complications have been associated with kaposiform hemangioendothelioma (KHE) and can lead to disability and reduced quality of life. We aimed to determine the clinical characteristics of musculoskeletal complication in patients with KHE without Kasabach-Merritt phenomenon (KMP) in order to identify features that may aid clinicians in KHE treatment.
We conducted a cohort study of KHE without KMP associated with musculoskeletal complication between January 2006 and February 2017 at three tertiary medical centers in China. The study included 29 nonthrombocytopenic patients with KHE and musculoskeletal complication.
The mean age at diagnosis of KHE was 4.5 years (range, 0.3-50.0 years). The mean follow-up was 4.1 years (range, 0.5-9.0 years). In most cases (72.4%), decreased range of motion (ROM) appeared within 2 years of KHE onset. Associated chronic pain was reported in 12 patients. Bone-joint changes were common in patients with decreased ROM (75.9%). All the patients received at least one medical therapy including corticosteroids, vincristine, propranolol, and sirolimus. Sirolimus demonstrated the highest efficacy rate, with 94.7% of patients showing improvements in ROM and chronic pain.
Musculoskeletal complication can occur early in the disease course of KHE without KMP. Although no uniformly effective treatment modality was found, sirolimus demonstrated the best response in patients with KHE with decreased ROM and chronic pain.
肌肉骨骼并发症与卡波西样血管内皮瘤（KHE）相关，可导致残疾并降低生活质量。我们旨在确定无卡萨巴赫-梅里特现象（KMP）的KHE患者肌肉骨骼并发症的临床特征，以识别有助于临床医生治疗KHE的特征。
我们于2006年1月至2017年2月在中国的三个三级医疗中心对无KMP且伴有肌肉骨骼并发症的KHE进行了一项队列研究。该研究纳入了29例非血小板减少性KHE并伴有肌肉骨骼并发症的患者。
KHE诊断时的平均年龄为4.5岁（范围0.3 - 50.0岁）。平均随访时间为4.1年（范围0.5 - 9.0年）。在大多数情况下（72.4%），运动范围（ROM）减小出现在KHE发病后2年内。12例患者报告有相关慢性疼痛。ROM减小的患者中骨关节改变很常见（75.9%）。所有患者均接受了至少一种药物治疗，包括皮质类固醇、长春新碱、普萘洛尔和西罗莫司。西罗莫司显示出最高的有效率，94.7%的患者ROM和慢性疼痛有所改善。
无KMP的KHE病程早期可出现肌肉骨骼并发症。虽然未发现统一有效的治疗方式，但西罗莫司对ROM减小和有慢性疼痛的KHE患者反应最佳。
