Health-related quality of life and functional outcomes from a randomized, controlled study of lisdexamfetamine dimesylate in children and adolescents with attention deficit hyperactivity disorder.
Health-related quality of life and functional outcomes from a randomized, controlled study of lisdexamfetamine dimesylate in children and adolescents with attention deficit hyperactivity disorder.
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DOI：
10.1007/s40263-013-0095-5
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发表时间：
2013-10
期刊：
影响因子：
6
通讯作者：
Coghill, David
中科院分区：
文献类型：
--
作者：
Banaschewski, Tobias;Soutullo, Cesar;Lecendreux, Michel;Johnson, Mats;Zuddas, Alessandro;Hodgkins, Paul;Adeyi, Ben;Squires, Liza A.;Coghill, David
注意缺陷多动障碍（ADHD）的最佳管理不仅旨在改善患者的症状，而且还旨在改善健康相关的生活质量（HRQL）和功能。一项在10个欧洲国家的儿童和青少年中进行的为期7周、随机、双盲、安慰剂对照的关键III期研究表明，兴奋剂前药二甲磺酸利右苯丙胺（LDX）是一种有效且耐受性良好的ADHD症状治疗药物。本研究的目的是分别使用儿童健康和疾病概况-儿童版：父母报告表（CHIP-CE：PRF）和韦斯功能损害评定量表-父母报告（WFIRS-P）评估本临床试验中的HRQL和功能损害结局。将确诊为ADHD且基线ADHD评定量表IV总分≥28分的患者（年龄6-17岁）随机（1：1：1）接受为期7周的双盲治疗，包括每日一次LDX、安慰剂或参比治疗药物哌甲酯缓释口服系统（OROS-MPH）。受试者的父母（或法定授权代表）在基线、第4周和第7周和/或提前终止时完成CHIP-CE：PRF和WFIRS-P问卷。终点定义为具有有效数据（≤ 30%缺失项目）的末次治疗访视。CHIP-CE：PRF成就领域预先指定为主要HRQL结局。全分析集包括317例患者（LDX，n = 104;安慰剂，n = 106; OROS-MPH，n = 107），其中大多数完成了研究（LDX，n = 77;安慰剂，n = 42; OROS-MPH，n = 72）。基线CHIP-CE：5个领域中有4个领域的PRF T评分低于正常值≥1个标准差（美国社区样本）。与安慰剂相比，LDX与这四个领域从基线到终点的统计学显著改善T评分相关，成就效应量为1.280（p < 0.001），风险规避效应量为1.079（p < 0.001），弹性效应量为0.421（p < 0.01），满意度效应量为0.365（p < 0.05）。在LDX治疗的患者中，安慰剂校正的WFIRS-P评分从基线到终点的改善在总分和6个领域中的4个方面具有统计学显著性（p < 0.001），效应量为0.924（总分）、1.249（学习和学校）、0.730（家庭）、0.643（社交活动）和0.640（风险活动）。OROS-MPH治疗在CHIP-CE：PRF和WFIRS-P评分方面显示出从基线到终点的相似改善模式。基线HRQL和功能障碍评分反映了未经治疗的ADHD的负担。ADHD儿童和青少年短期兴奋剂治疗的益处不仅仅是症状缓解，而且对HRQL和日常功能产生积极影响。本文的在线版本（doi：10.1007/s40263-013-0095-5）包含补充材料，可供授权用户使用。
Optimal management of attention deficit hyperactivity disorder (ADHD) aims not only to ameliorate patients’ symptoms, but also to improve health-related quality of life (HRQL) and functioning. A pivotal, 7-week, randomized, double-blind, placebo-controlled, phase III study in children and adolescents in ten European countries demonstrated that the stimulant prodrug lisdexamfetamine dimesylate (LDX) is an effective and generally well-tolerated treatment for symptoms of ADHD. The aim of this study was to assess HRQL and functional impairment outcomes in this clinical trial, using the Child Health and Illness Profile-Child Edition: Parent Report Form (CHIP-CE:PRF) and the Weiss Functional Impairment Rating Scale-Parent Report (WFIRS-P), respectively. Patients (aged 6–17 years) with diagnosed ADHD and a baseline ADHD Rating Scale IV total score ≥28 were randomized (1:1:1) to 7 weeks of double-blind treatment with once-daily LDX, placebo or the reference treatment, osmotic-release oral system methylphenidate (OROS-MPH). Participants’ parents (or legally authorized representatives) completed the CHIP-CE:PRF and WFIRS-P questionnaires at baseline, at weeks 4 and 7, and/or at early termination. Endpoint was defined as the last on-treatment visit with valid data (≤30 % missing items). The CHIP-CE:PRF Achievement domain was pre-specified as the primary HRQL outcome. The full analysis set comprised 317 patients (LDX, n = 104; placebo, n = 106; OROS-MPH, n = 107), the majority of whom completed the study (LDX, n = 77; placebo, n = 42; OROS-MPH, n = 72). Baseline CHIP-CE:PRF T-scores in four of the five domains were ≥1 standard deviation below norms (US community samples). Compared with placebo, LDX was associated with statistically significantly improved T-scores from baseline to endpoint in these four domains, with effect sizes of 1.280 (p < 0.001) in Achievement, 1.079 (p < 0.001) in Risk Avoidance, 0.421 (p < 0.01) in Resilience and 0.365 (p < 0.05) in Satisfaction. In LDX-treated patients, placebo-adjusted improvements from baseline to endpoint in WFIRS-P scores were statistically significant (p < 0.001) for total score and four of the six domains, with effect sizes of 0.924 (total score), 1.249 (Learning and School), 0.730 (Family), 0.643 (Social Activities) and 0.640 (Risky Activities). OROS-MPH treatment showed similar patterns of improvement from baseline to endpoint in both CHIP-CE:PRF and WFIRS-P scores. Baseline HRQL and functional impairment scores reflect the burden of untreated ADHD. The benefits of short-term stimulant treatment in children and adolescents with ADHD extend beyond symptomatic relief and impact positively on HRQL and daily functioning. The online version of this article (doi:10.1007/s40263-013-0095-5) contains supplementary material, which is available to authorized users.
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影响因子：
5.6
作者：
Coghill, David;Banaschewski, Tobias;Squires, Liza
通讯作者：
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