Nikles C Jane, Mitchell Geoffrey K, Del Mar Chris B, Clavarino Alexandra, McNairn Norma
The University of Queensland, Herston, Queensland 4006, Australia.
Pediatrics. 2006 Jun;117(6):2040-6. doi: 10.1542/peds.2005-1328.
We sought to describe the clinical use of n-of-1 trials for attention-deficit/hyperactivity disorder in publicly and privately funded family and specialized pediatric practice in Australia.
We used a within-patient randomized, double-blind, crossover comparison of stimulant (dexamphetamine or methylphenidate) versus placebo or alternative stimulant using 3 pairs of treatment periods. Trials were conducted from a central location using mail and telephone communication, with local supervision by the patients' clinicians.
Our study population included children with clinically diagnosed attention-deficit/hyperactivity disorder who were aged 5 to 16 years and previously stabilized on an optimal dose of stimulant. They were selected because treatment effectiveness was uncertain.
Our measures included number of patients recruited, number of doctors who used the service, geographic spread, completion rates, response rate, and post-n-of-1 trial decisions.
Forty-five doctors across Australia requested 108 n-of-1 trials, of which 86 were completed. In 69 drug-versus-placebo comparisons, 29 children responded better to stimulant than placebo. Immediately posttrial, 19 of 25 drug-versus-placebo responders stayed on the same stimulant, and 13 of 24 nonresponders ceased or switched stimulants. In 40 of 63 for which data were available, posttrial management was consistent with the trial results. For all types of n-of-1 trials, management changed for 28 of 64 children for whom information was available.
Attention-deficit/hyperactivity disorder n-of-1 trials can be implemented successfully by mail and telephone communication. This type of trial can be valuable in clarifying treatment effect when it is uncertain, and in this series, they had a noticeable impact on short-term management.
我们试图描述在澳大利亚公共和私人资助的家庭及专科儿科诊所中，单病例试验在注意缺陷多动障碍治疗中的临床应用情况。
我们采用患者内随机、双盲、交叉对照试验，比较兴奋剂（右旋苯丙胺或哌甲酯）与安慰剂或其他兴奋剂，共设置3组治疗期。试验通过邮件和电话沟通在中心地点进行，由患者的临床医生进行当地监督。
我们的研究对象包括临床诊断为注意缺陷多动障碍、年龄在5至16岁且之前已稳定使用最佳剂量兴奋剂的儿童。选择他们是因为治疗效果不确定。
我们的指标包括招募的患者数量、使用该服务的医生数量、地理分布、完成率、反应率以及单病例试验后的决策。
澳大利亚各地的45名医生申请了108项单病例试验，其中86项完成。在69项药物与安慰剂的比较中，29名儿童对兴奋剂的反应优于安慰剂。试验结束后立即进行评估，25名药物与安慰剂比较的反应者中有19名继续使用相同的兴奋剂，24名无反应者中有13名停止或更换了兴奋剂。在有数据的63项中的40项中，试验后的管理与试验结果一致。对于所有类型的单病例试验，在有信息的64名儿童中，有28名的管理方式发生了改变。
注意缺陷多动障碍的单病例试验可以通过邮件和电话沟通成功实施。当治疗效果不确定时，这类试验在明确治疗效果方面可能很有价值，在本系列研究中，它们对短期管理产生了显著影响。
