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医生对每周一次索马鲁肽与每日一次 rhGH 方案治疗儿童生长激素缺乏症的经验:来自全球 3 期研究的医生的横断面调查。

Physician experience with once-weekly somatrogon versus once-daily rhGH regimen in pediatric patients with growth hormone deficiency: a cross-sectional survey of physicians from the global phase 3 study.

机构信息

Global Medical Affairs, Pfizer Pte Limited, Singapore, Singapore.

Adelphi Values, Patient-Centered Outcomes, Boston, MA, United States.

出版信息

Front Endocrinol (Lausanne). 2023 Oct 17;14:1254424. doi: 10.3389/fendo.2023.1254424. eCollection 2023.

Abstract

INTRODUCTION

The standard of care for pediatric growth hormone deficiency (pGHD) is once-daily recombinant human growth hormone (rhGH). Somatrogon, a long-acting rhGH, requires less frequent, once-weekly, dosing. We describe physicians' preference for, experiences, and satisfaction with once-weekly somatrogon vs once-daily rhGH.

METHODS

English-speaking investigators from somatrogon's global phase III study (NCT02968004) with prior experience using once-daily rhGH were included. Participants answered an online survey containing 14 closed- and open-ended items.

RESULTS

Twenty-four pediatric endocrinologists (41.7% men; 79.2% practiced at public/private hospitals) from 12 countries with 25.8 ± 12.0 years' experience treating pGHD completed the survey. In terms of the time and effort required to explain device instructions, injection regimen, procedure for missed injection, and address patients'/caregivers' concerns, a similar proportion of physicians chose once-weekly somatrogon and once-daily rhGH; 62.5% physicians indicated that once-daily rhGH required greater effort to monitor adherence. Overall, 75% preferred once-weekly somatrogon over once-daily rhGH, 79.2% considered once-weekly somatrogon to be more convenient and less burdensome, and 83.3% were likely to prescribe somatrogon in the future. Overall, 50% felt that once-weekly somatrogon was more beneficial to patients, while 50% chose "No difference". Most physicians (62.5%) felt both regimens were equally likely to support positive long-term growth outcomes and reduce healthcare utilization. More physicians were "very satisfied" with once-weekly somatrogon (62.5%) than with once-daily rhGH (16.7%). Reduced injection frequency, patient and caregiver burden, increased convenience, and improved adherence were reasons for these choices.

CONCLUSION

Physicians had a positive experience with, and perception of, treating pGHD with once-weekly somatrogon.

摘要

简介

儿科生长激素缺乏症(pGHD)的标准治疗方法是每日一次重组人生长激素(rhGH)。长效 rhGH 索马鲁肽需要更频繁的、每周一次的给药。我们描述了医生对每周一次索马鲁肽与每日一次 rhGH 的偏好、经验和满意度。

方法

我们纳入了来自索马鲁肽全球 III 期研究(NCT02968004)的讲英语的调查人员,这些调查人员有使用每日一次 rhGH 的经验。参与者回答了一份包含 14 个封闭式和开放式问题的在线调查。

结果

来自 12 个国家的 24 名儿科内分泌学家(41.7%为男性;79.2%在公立/私立医院执业)完成了这项调查,他们治疗 pGHD 的经验为 25.8±12.0 年。在解释设备说明、注射方案、漏注程序以及解决患者/照顾者的担忧方面所需的时间和精力方面,选择每周一次索马鲁肽和每日一次 rhGH 的医生比例相似;62.5%的医生表示,每日一次 rhGH 更需要努力监测依从性。总体而言,75%的医生更喜欢每周一次索马鲁肽,79.2%的医生认为每周一次索马鲁肽更方便、负担更小,83.3%的医生将来可能会开索马鲁肽。总的来说,50%的医生认为每周一次索马鲁肽对患者更有益,而 50%的医生选择“没有区别”。大多数医生(62.5%)认为两种方案都有可能支持长期积极的生长结果和减少医疗保健的使用。更多的医生对每周一次索马鲁肽“非常满意”(62.5%)而不是每日一次 rhGH(16.7%)。减少注射频率、患者和照顾者负担、提高便利性和改善依从性是做出这些选择的原因。

结论

医生对每周一次索马鲁肽治疗 pGHD 有积极的体验和看法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8cb2/10634585/6ca0dc565e2d/fendo-14-1254424-g001.jpg

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