Pediatric growth hormone deficiency: Understanding the patient and caregiver perspectives

IF 1.6 4区 医学 Q4 CELL BIOLOGY Growth Hormone & Igf Research Pub Date : 2024-04-16 DOI:10.1016/j.ghir.2024.101592
Bradley S. Miller , Nicky Kelepouris , Harlan Tenenbaum , Sonia Di Carlo , Philippe Backeljauw
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Abstract

Childhood growth hormone deficiency (GHD) is a rare disorder associated with significant burden on both patients and caregivers. Although previous reports have detailed aspects of the burden experienced by patients and their caregivers, there is a paucity of first-hand information on the patient and caregiver journeys from their respective voices. To address this need, an advisory board meeting was conducted on September 30, 2022, with 4 pediatric patients with GHD and their caregivers to discuss their experiences prior to GHD diagnosis, during the diagnostic process, and during ongoing treatment with recombinant growth hormone. Feedback from patients and caregivers was reviewed by pediatric endocrinologists, who provided their own perspectives on the patient and caregiver journeys based on the information reported. Despite the small sample size, important insights were obtained: patients and caregivers reported remarkable growth benefits achieved with treatment, which provided strong motivation to remain adherent to daily injection regimens. Patient and caregiver accounts reflected wide variability between families in time from suspicion to diagnosis and in treatment challenges faced, ranging from practical issues such as handling and administration of medication to broader concerns about treatment access and continuity, as well as key knowledge gaps among patients, caregivers, and clinicians. Recommendations are provided to enhance the patient and caregiver journeys, including increasing development and availability of educational materials, providing opportunities for patient advocacy by clinicians and health care providers, and encouraging institutional improvements to ensure that patients continue to receive uninterrupted treatment during their critical period of growth.

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小儿生长激素缺乏症:了解患者和护理人员的观点
儿童生长激素缺乏症(GHD)是一种罕见疾病,给患者和照护者都带来了沉重的负担。虽然以往的报告详细介绍了患者及其护理人员所承受的各方面负担,但从患者和护理人员各自的角度出发,了解他们心路历程的第一手资料却非常缺乏。为了满足这一需求,我们于 2022 年 9 月 30 日召开了一次咨询委员会会议,与 4 名儿科 GHD 患者及其护理人员讨论了他们在 GHD 诊断前、诊断过程中以及正在接受重组生长激素治疗期间的经历。儿科内分泌专家对患者和护理人员的反馈意见进行了审查,并根据所报告的信息对患者和护理人员的心路历程提出了自己的看法。尽管样本量较小,但我们还是获得了重要的启示:患者和护理人员报告了治疗对生长的显著益处,这为坚持每日注射提供了强大的动力。患者和照护者的陈述反映出不同家庭从怀疑到确诊的时间以及所面临的治疗挑战存在很大差异,这些挑战既包括处理和用药等实际问题,也包括对治疗机会和持续性的更广泛关注,还包括患者、照护者和临床医生之间的主要知识差距。本报告提出了一些建议,以改善患者和护理人员的治疗过程,包括增加教育材料的开发和供应,为临床医生和医疗服务提供者提供为患者宣传的机会,以及鼓励机构改进以确保患者在其成长的关键时期继续接受不间断的治疗。
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来源期刊
Growth Hormone & Igf Research
Growth Hormone & Igf Research 医学-内分泌学与代谢
CiteScore
3.30
自引率
0.00%
发文量
38
审稿时长
57 days
期刊介绍: Growth Hormone & IGF Research is a forum for research on the regulation of growth and metabolism in humans, animals, tissues and cells. It publishes articles on all aspects of growth-promoting and growth-inhibiting hormones and factors, with particular emphasis on insulin-like growth factors (IGFs) and growth hormone. This reflects the increasing importance of growth hormone and IGFs in clinical medicine and in the treatment of diseases.
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