Brod Meryl, Beck Jane F, Højbjerre Lise, Bushnell Donald M, Adalsteinsson Johan Erpur, Wilkinson Lars, Rasmussen Michael Højby
The Brod Group, 219 Julia Avenue, Mill Valley, CA, 94941, USA.
Novo Nordisk A/S, Vandtaarnsvej 114, 2860, Soeborg, Denmark.
Pharmacoecon Open. 2019 Mar;3(1):71-80. doi: 10.1007/s41669-018-0082-3.
This study's purpose was to assess the minimal important difference (MID) for the Treatment-Related Impact Measure-Adult Growth Hormone Deficiency (TRIM-AGHD), a patient-reported outcome measure assessing growth hormone deficiency (GHD) impacts. The measure was demonstrated to have adequate psychometric measurement properties, and be reliable and valid. For scores to be interpretable, the TRIM-AGHD must be responsive to treatment benefit and the MID in scores quantified.
A prospective, non-interventional, observational, clinic-based survey study of naïve-to-treatment adult GHD patients (N = 98) was conducted. Key assessments were at baseline and follow-up (between 4 and approximately 8 weeks), with weekly telephone monitoring post-baseline (last n = 34 patients). Responsiveness was evaluated using the effect size of change scores from baseline to follow-up. MID estimates were derived from distribution-based (half standard deviation [0.5 SD], standard error of measurement [SEm]) and anchor-based methods (patient global rating of change [PGRC]) using change scores from baseline to initial report of minimal improvement in GHD severity. Findings from each method were converged to establish an acceptable MID.
Patients were mean age 49.7 years, 65.6% female, and 76.0% Caucasian. The TRIM-AGHD was highly responsive to treatment with the total score effect size being 1.38. For the total score, the 0.5 SD was 8.09 and the SEm was 2.66. The difference found using the PGRC was 20.43. The converged MID value for the total score was 10 points.
The TRIM-AGHD is a highly responsive measure assessing AGHD treatment impacts. A 10-point change score is considered a clinically meaningful improvement.
本研究旨在评估治疗相关影响量表-成人生长激素缺乏症（TRIM-AGHD）的最小重要差异（MID），这是一种患者报告的结局指标，用于评估生长激素缺乏症（GHD）的影响。该量表已被证明具有足够的心理测量学特性，且可靠有效。为了使分数具有可解释性，TRIM-AGHD必须对治疗益处有反应，并对分数的MID进行量化。
对初治的成年GHD患者（N = 98）进行了一项前瞻性、非干预性、观察性的基于门诊的调查研究。关键评估在基线和随访时（4至约8周之间）进行，基线后每周进行电话监测（最后n = 34例患者）。使用从基线到随访的变化分数的效应大小评估反应性。MID估计值来自基于分布的方法（半标准差[0.5 SD]、测量标准误[SEm]）和基于锚定的方法（患者对变化的整体评分[PGRC]），使用从基线到GHD严重程度最小改善的初始报告的变化分数。将每种方法的结果进行汇总，以确定可接受的MID。
患者的平均年龄为49.7岁，女性占65.6%，白种人占76.0%。TRIM-AGHD对治疗反应高度敏感，总分效应大小为1.38。对于总分，0.5 SD为8.09，SEm为2.66。使用PGRC发现的差异为20.43。总分的汇总MID值为10分。
TRIM-AGHD是一种评估AGHD治疗影响的高反应性指标。10分的变化分数被认为是具有临床意义的改善。
