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一项针对医生建议启动和停止生长激素治疗身材矮小的全国性研究。

A national study of physician recommendations to initiate and discontinue growth hormone for short stature.

机构信息

Weatherhead School of Management, Department of Internal Medicine, MetroHealth Medical Center, School of Medicine, Rainbow Babies & Children's Hospital, Case Western Reserve University, 11100 Euclid Ave, Room 737, Cleveland, OH 44106, USA.

出版信息

Pediatrics. 2010 Sep;126(3):468-76. doi: 10.1542/peds.2009-3609. Epub 2010 Aug 30.

Abstract

OBJECTIVES

Overall growth hormone (GH) use depends on decisions to both initiate treatment and continue treatment. The determinants of both are unclear. We studied how physicians decided to begin GH in idiopathic short stature and how, after an initial course of treatment, they decided to continue, intensify (increase the dose), or terminate treatment.

METHODS

We used a national census study of 727 pediatric endocrinologists involving a structured questionnaires with a factorial experimental design. Main outcome measures were GH recommendations for previously untreated children and those children who were treated with GH for 1 year.

RESULTS

The response rate was 90%. In previously untreated children, recommendations to initiate GH were consistent with guidelines and also influenced by family preferences and physician attitudes (P<.001). In children treated with GH, recommendations on whether to continue GH were influenced by the growth response to therapy (P<.01) but were divided regarding course of action. With identical growth responses to treatment, physician decisions diverged (intensify versus discontinue GH) and were driven by independent, nonphysiologic, and contextual factors (eg, physician attitudes, family preferences, and GH-initiation recommendation; each P<.001). Together, attitudinal and contextual factors exerted more influence on continuation decisions than did the growth response to therapy.

CONCLUSIONS

Physician decisions to initiate GH are largely consistent with evidence-based medicine. However, decisions about continuing GH vary and are strongly influenced by factors other than response to treatment. With a potential market of 500 000 US children and costs exceeding $10 billion per year, changes in GH use may depend on potentially modifiable physician attitudes and family preferences as much as physiologic evidence.

摘要

目的

总体上,生长激素(GH)的使用取决于启动治疗和继续治疗的决策。目前尚不清楚这些决策的决定因素。我们研究了医生在特发性身材矮小症中决定开始使用 GH 的方式,以及在初始治疗疗程后,他们如何决定继续、强化(增加剂量)或终止治疗。

方法

我们使用了一项涉及 727 名儿科内分泌学家的全国性普查研究,该研究采用了带有因子实验设计的结构化问卷。主要观察指标是对从未接受过治疗的儿童和接受 GH 治疗 1 年的儿童的 GH 推荐。

结果

回复率为 90%。对于从未接受过治疗的儿童,启动 GH 的建议与指南一致,也受到家庭偏好和医生态度的影响(P<.001)。对于接受 GH 治疗的儿童,是否继续使用 GH 的建议受到治疗后生长反应的影响(P<.01),但治疗方案存在分歧。对于治疗后的生长反应相同的儿童,医生的决策存在分歧(强化或停止 GH),并且受到独立的、非生理的和背景因素的驱动(例如,医生态度、家庭偏好和 GH 启动建议;每项 P<.001)。总的来说,态度和背景因素对继续治疗的决策的影响大于治疗的生长反应。

结论

医生启动 GH 的决策在很大程度上符合循证医学。然而,关于继续使用 GH 的决策存在差异,并且受到治疗反应以外的因素的强烈影响。在美国,有潜在的 50 万名儿童有此需求,每年的费用超过 100 亿美元,GH 使用的变化可能取决于医生态度和家庭偏好等潜在可改变的因素,而不仅仅是生理证据。

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