recombinant human growth hormone treatment

重组人生长激素治疗
  • 文章类型: Journal Article
    背景:重组人生长激素(rhGH)治疗对Prader-Willi综合征(PWS)儿童在改善身材矮小和新陈代谢方面有益,但是早期rhGH治疗对3岁以下PWS儿童的呼吸和睡眠参数的影响仍然难以捉摸。因此,本研究旨在探讨rhGH治疗对PWS幼儿睡眠相关呼吸障碍(SRBDs)的影响。
    方法:在2018年10月至2023年1月期间,共招募了17名年龄匹配的接受rhGH治疗的PWS患者(rhGH组)和17名未接受rhGH治疗的对照个体(非rhGH组)。收集与多导睡眠图(PSG)相关的数据以及胰岛素样生长因子(IGF-1)和胰岛素样生长因子结合蛋白3(IGFBP-3)的血清水平。
    结果:rhGH组的平均年龄为20.76±9.22个月,与非rhGH组(25.23±13.81个月)相当。治疗52周后,两组的人口统计学和人体测量参数相似。对幼儿施用rhGH不会对阻塞性呼吸暂停低通气指数(OAHI)产生不利影响,中枢呼吸暂停指数(CAI),氧饱和度指数(ODI),平均经皮氧饱和度(SpO2),最低SpO2,当SpO2低于90%时的持续时间,或SpO2低于90%的患者比例。此外,IGF-1z评分和IGFBP-3水平的升高并未使SRBD恶化.
    结论:用rhGH治疗52周对患有PWS的幼儿没有显示出对SRBD的有害影响。这进一步阐明了PWS患者早期开始rhGH治疗的重要性。
    Recombinant human growth hormone (rhGH) therapy is beneficial for children with Prader-Willi syndrome (PWS) in improving short stature and metabolism, but the effect of early rhGH treatment on respiratory and sleep parameters for PWS children under three years old remains elusive. Thus, this study aimed to investigate the impact of rhGH treatment on sleep-related breathing disorders (SRBDs) for toddlers with PWS.
    A total of 17 age-matched PWS patients receiving rhGH treatment (rhGH group) and 17 control individuals not receiving rhGH treatment (non-rhGH group) were recruited for this study between October 2018 and January 2023. Data related to polysomnography-polygraphy (PSG) and serum levels of insulin-like growth factor (IGF-1) and insulin-like growth factor binding protein 3 (IGFBP-3) were collected.
    The mean age in the rhGH group was 20.76 ± 9.22 months, which was comparable to that of the non-rhGH group (25.23 ± 13.81 months). The demographic and anthropometric parameters were similar across the two groups after 52 weeks of treatment. Administration of rhGH to toddlers did not exert adverse effects on the obstructive apnea-hypopnea index (OAHI), central apnea index (CAI), oxygen desaturation index (ODI), mean percutaneous oxygen saturation (SpO2), lowest SpO2, duration when SpO2 is lower than 90%, or proportion of the patients with SpO2 lower than 90%. Furthermore, the increased IGF-1 z-score and IGFBP-3 level did not worsen SRBDs.
    Treatment with rhGH for 52 weeks on young toddlers with PWS showed no deleterious effects on SRBDs. This shed more light on the importance of initiating rhGH therapy early in PWS patients.
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  • 文章类型: Journal Article
    最近的一项系统评价发现,高达71%的生长激素缺乏症儿童及其家人不遵守规定的治疗。更好地理解儿科不遵守的复杂问题的关键方法是探索父母/照顾者的看法和经验。我们的研究是第一个专门研究影响患有这种内分泌紊乱的儿童的父母/照顾者不坚持rhGH治疗的潜在可改变因素的研究。
    进行了十四次半结构化电话访谈,以探索父母/照顾者对孩子的状况和规定治疗的看法和经验,除了他们与医疗保健专业人员的感知关系。对研究结果进行了主题分析和叙述综合,与Braun和Clarke(2006)的定性方法一致。
    影响不坚持生长激素治疗的可能改变的因素分为四个主题:1.设备负担,2.治疗注意事项,3.后勤干扰和4。人际关系的影响。
    我们的探索性研究提出了影响生长激素治疗方式的多种潜在可改变因素。这些发现可以,反过来,用于告知和促进有针对性的发展,以依从性为重点的干预措施,支持生长激素缺乏的儿童及其家庭,并在内分泌临床实践中优化处方生长激素治疗的使用。
    UNASSIGNED: A recent systematic review found that up to 71% of children with growth hormone deficiency and their families are non-adherent to treatment as prescribed. A key way to better understanding the complex issue of pediatric non-adherence is to explore the perceptions and experiences of the parent/caregiver. Our study is the first to look specifically at the potentially modifiable factors that influence non-adherence to rhGH treatment amongst parents/caregivers of children with this endocrine disorder.
    UNASSIGNED: Fourteen semi-structured telephone interviews were conducted to explore parents/caregivers\' perceptions and experiences of their child\'s condition and prescribed treatment, in addition to their perceived relationship with their healthcare professional. The findings were thematically analyzed and narratively synthesized, in line with the qualitative approach of Braun and Clarke (2006).
    UNASSIGNED: Potentially modifiable factors that influence non-adherence to growth hormone treatment were grouped under four themes: 1. Device Burdens, 2. Treatment Considerations, 3. Logistical Interferences and 4. Interpersonal Influences.
    UNASSIGNED: Our exploratory study presents the wide range of potentially modifiable factors that influence the way in which growth hormone treatment is used. These findings can, in turn, be used to inform and promote the development of targeted, adherence-focused interventions, to support growth hormone deficient children and their families and optimize the use of prescribed growth hormone treatment within endocrine clinical practice.
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