重度原发性胰岛素样生长因子-1缺乏症（IGFD）患儿的管理：IGFD的诊断与管理
Managing the child with severe primary insulin-like growth factor-1 deficiency (IGFD): IGFD diagnosis and management.
作者信息
Cohen Jay, Blethen Sandra, Kuntze Joyce, Smith Susan L, Lomax Kathleen G, Mathew Puthenpurackal M
机构信息
BMG: The Endocrine Clinic, P.C., 5659 South Rex Road, Memphis, TN, 38119, USA,
出版信息
Drugs R D. 2014 Mar;14(1):25-9. doi: 10.1007/s40268-014-0039-7.
Abstract
Growth failure associated with severe primary insulin-like growth factor 1 (IGF-1) deficiency (SPIGFD), a condition defined as basal IGF-1 standard deviation score (SDS) less than or equal to -3 and height SDS less than or equal to -3 in a child with normal or elevated levels of growth hormone, can be successfully treated with the recombinant human IGF-1 mecasermin. In this review, we describe the most safe and effective way to use mecasermin in the treatment of patients with SPIGFD, including how to initiate dosing, key side effects, and how to monitor treatment. Finally, mention of how to reinitiate therapy is made, given the recent drug shortage with mecasermin.
摘要
与严重原发性胰岛素样生长因子1（IGF-1）缺乏症（SPIGFD）相关的生长衰竭，这一病症被定义为生长激素水平正常或升高的儿童中，基础IGF-1标准差评分（SDS）小于或等于 -3且身高SDS小于或等于 -3，可用重组人生长激素美卡舍明成功治疗。在本综述中，我们描述了使用美卡舍明治疗SPIGFD患者的最安全有效的方法，包括如何开始给药、关键副作用以及如何监测治疗。最后，鉴于近期美卡舍明药物短缺，提及了如何重新开始治疗。
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