A pilot study proposing an algorithm for pubertal induction in cerebral palsy.

IF 1.3 4区 医学 Q4 ENDOCRINOLOGY & METABOLISM Journal of Pediatric Endocrinology & Metabolism Pub Date : 2024-02-20 Print Date: 2024-03-25 DOI:10.1515/jpem-2024-0013
Anne Trinh, Angelina Lim, Phillip Wong, Justin Brown, Janne Pitkin, Beverley Wollenhoven, Peter Ebeling, Peter Fuller, Frances Milat, Margaret Zacharin
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Abstract

Objectives: To explore delayed puberty in cerebral palsy (CP) and to test the acceptability of an interventional puberty induction algorithm.

Methods: A two phase cohort study in children and adolescents diagnosed with CP who have delayed puberty. Phase 1: Retrospective review of clinical records and interviews with patients who have been treated with sex-steroids and Phase 2: Prospective interventional trial of pubertal induction with a proposed algorithm of transdermal testosterone (males) or oestrogen (females). Phase 1 examined experiences with sex-steroid treatment. Phase 2 collected data on height adjusted bone mineral density (BMAD), fractures, adverse effects, mobility and quality of life over two years during the induction.

Results: Phase 1, treatment was well tolerated in 11/20 treated with sex-steroids; phase 2, using the proposed induction algorithm, 7/10 treated reached Tanner stage 3 by nine months. One participant reached Tanner stage 5 in 24 months. Mean change in BMAD Z-scores was +0.27 % (SD 0.002) in those who could be scanned by dual-energy X-ray absorptiometry (DXA).

Conclusions: Delayed puberty may be diagnosed late. Treatment was beneficial and well tolerated, suggesting all patients with severe pubertal delay or arrest should be considered for sex hormone supplementation.

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一项试点研究,提出了脑瘫患者青春期诱导算法。
目的探讨脑瘫(CP)患者的青春期延迟问题,并测试干预性青春期诱导算法的可接受性:方法:对确诊为CP的青春期延迟儿童和青少年进行两阶段队列研究。第 1 阶段:回顾性审查临床记录,并与接受过性类固醇治疗的患者进行访谈;第 2 阶段:前瞻性干预试验,采用建议的经皮睾酮(男性)或雌激素(女性)算法进行青春期诱导。第 1 阶段研究使用性类固醇治疗的经验。第二阶段收集了青春期诱导期间两年内身高调整后骨矿物质密度(BMAD)、骨折、不良反应、活动能力和生活质量的数据:结果:第一阶段,11/20 接受性类固醇治疗者对治疗耐受良好;第二阶段,采用建议的诱导算法,7/10 接受治疗者在 9 个月前达到 Tanner 3 期。一名受试者在 24 个月内达到 Tanner 5 期。通过双能 X 射线吸收测量法(DXA)扫描的受试者的 BMAD Z 评分的平均变化率为 +0.27%(SD 0.002):结论:青春期延迟的诊断可能较晚。治疗有益且耐受性良好,建议所有青春期严重延迟或停滞的患者都应考虑补充性激素。
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来源期刊
CiteScore
2.70
自引率
7.10%
发文量
176
审稿时长
3-6 weeks
期刊介绍: The aim of the Journal of Pediatric Endocrinology and Metabolism (JPEM) is to diffuse speedily new medical information by publishing clinical investigations in pediatric endocrinology and basic research from all over the world. JPEM is the only international journal dedicated exclusively to endocrinology in the neonatal, pediatric and adolescent age groups. JPEM is a high-quality journal dedicated to pediatric endocrinology in its broadest sense, which is needed at this time of rapid expansion of the field of endocrinology. JPEM publishes Reviews, Original Research, Case Reports, Short Communications and Letters to the Editor (including comments on published papers),. JPEM publishes supplements of proceedings and abstracts of pediatric endocrinology and diabetes society meetings.
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