医学
多发性内分泌肿瘤2型
降钙素
甲状腺癌
癌胚抗原
内科学
胃肠病学
背景(考古学)
不利影响
甲状腺髓样癌
进行性疾病
肿瘤科
甲状腺
内分泌学
疾病
癌症
古生物学
生物化学
化学
种系突变
生物
突变
基因
作者
Ananth Shankar,Tom Kurzawinski,Emma J. Ross,Sara Stoneham,Tim Beale,Ian Proctor,Tony Hulse,Kathleen Simpson,Mark Gaze,Elene Cattaneo,Evelien Gevers,Lynley V. Marshall,Johnathan Hubbard,Caroline Brain
标识
DOI:10.1016/j.ejca.2021.09.012
摘要
Medullary thyroid carcinoma (MTC) in the context of multiple endocrine neoplasia type 2 (MEN2) is caused by mutations in the RET proto-oncogene. Therefore, in children with MEN2 and advanced MTC, the RET tyrosine kinase (TK) pathway is a target for treatment with selpercatinib, a selective RET TK inhibitor.A retrospective review of the clinical, genetic, biochemical (calcitonin and carcinoembryonic antigen [CEA]) and imaging data of six medically untreated children with MEN2 and recurrent and or progressive MTC. The main parameters were safety and objective treatment response to selpercatinib.Six children (three males and three females, aged 3-12 years), four with MEN2B and two MEN2A, are reported. All had initial total thyroidectomy and extensive neck dissections but subsequently developed recurrent and progressive disease. All experienced an improvement in clinical symptoms with a concomitant biochemical response evidenced by significant fall in serum calcitonin and CEA concentrations. The fall in serum calcitonin was evident within 2 weeks of the start of selpercatinib, and responses were ongoing at a median follow-up of 13 months (range, 11-22 months). Four children with measurable radiological disease had good volume reduction. The most common adverse effects were transient but reversible grade 1 or 2 increase in alanine aminotransferase, serum bilirubin and constipation. No child required a dose modification or had to discontinue selpercatinib because of a drug-related adverse event.Selpercatinib has shown excellent therapeutic efficacy with minimal toxicity in children with MEN2 and progressive metastatic RET-mutated MTC.
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