Pabedinskas K, Kobrzynski M, Filler G
Department of Paediatrics, Children's Hospital at the London Health Sciences Centre, London, Ontario, Canada.
Department of Pathology and Laboratory Medicine, University of Western Ontario, London, Ontario, Canada.
Indian J Nephrol. 2017 May-Jun;27(3):237-238. doi: 10.4103/0971-4065.200520.
Tuberous sclerosis complex frequently results in the formation of renal angiomyolipomas (AMLs). Sirolimus (SIR) or everolimus can be used to treat large AMLs, although this treatment has rarely been described in children, particularly for multiple renal AMLs. A 15-year-old girl presented with bilateral severe chronic flank pain coinciding with increased renal size and hundreds of renal AMLs. We opted to treat her with SIR over the course of 3.5 years. Following her treatment, her renal size had substantially decreased and the AMLs had shrunk. The patient's pain subsided as well. Our case, which has never been described in published literature, demonstrates that a child with multiple renal AMLs can be treated with SIR, and suggests that this treatment may be a viable option for preventing the development of secondary morbidities such as chronic pain.
结节性硬化症常导致肾血管平滑肌脂肪瘤（AML）的形成。西罗莫司（SIR）或依维莫司可用于治疗较大的AML，尽管这种治疗在儿童中很少有描述，尤其是对于多发性肾AML。一名15岁女孩出现双侧严重慢性胁腹痛，同时肾脏增大，有数百个肾AML。我们选择在3.5年的时间里用SIR对她进行治疗。治疗后，她的肾脏大小显著减小，AML也缩小了。患者的疼痛也减轻了。我们的病例在已发表的文献中从未有过描述，表明患有多发性肾AML的儿童可以用SIR治疗，并表明这种治疗可能是预防慢性疼痛等继发性疾病发展的可行选择。
