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西罗莫司治疗结节性硬化症儿童的纤维瘤病和多灶性肾细胞癌。

Sirolimus therapy for fibromatosis and multifocal renal cell carcinoma in a child with tuberous sclerosis complex.

机构信息

Division of Hematology-Oncology, Department of Pediatrics, University of Alabama at Birmingham, Birmingham, Alabama, USA.

出版信息

Pediatr Blood Cancer. 2010 Jul 1;54(7):1035-7. doi: 10.1002/pbc.22401.

DOI:10.1002/pbc.22401
PMID:20108343
Abstract

A male with tuberous sclerosis complex (TSC) developed a chest wall fibromatosis and bilateral multifocal renal cell carcinoma (RCC). The fibromatosis tumor was initially resected during infancy but recurred 5 years later. At that time, bilateral RCC was also detected, leading to the resection of the more extensively affected right kidney. In an attempt to avoid bilateral nephrectomies, the patient was treated with the mTOR inhibitor sirolimus. Within 6 months of therapy, the fibromatosis and remaining RCC tumors responded substantially with minimal adverse effects.

摘要

一名男性患有结节性硬化症(TSC),并发了胸壁纤维瘤病和双侧多灶性肾细胞癌(RCC)。该纤维瘤瘤在婴儿期曾被切除,但 5 年后复发。那时,还检测到双侧 RCC,导致更广泛受影响的右肾被切除。为避免双侧肾切除术,该患者接受了 mTOR 抑制剂西罗莫司治疗。在治疗的 6 个月内,纤维瘤病和剩余的 RCC 肿瘤有了显著的反应,且副作用极小。

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