The effect of methylphenidate on neurofibromatosis type 1: a randomised, double-blind, placebo-controlled, crossover trial.
The effect of methylphenidate on neurofibromatosis type 1: a randomised, double-blind, placebo-controlled, crossover trial.
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DOI：
10.1186/s13023-014-0142-4
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发表时间：
2014-09-10
影响因子：
3.7
通讯作者：
Réseau NF1 Rhône Alpes Auvergne-France
中科院分区：
文献类型：
--
作者：
Lion-François L;Gueyffier F;Mercier C;Gérard D;Herbillon V;Kemlin I;Rodriguez D;Ginhoux T;Peyric E;Coutinho V;Bréant V;des Portes V;Pinson S;Combemale P;Kassaï B;Réseau NF1 Rhône Alpes Auvergne-France
1型神经纤维瘤病（NF 1）是一种常染色体显性遗传疾病，估计患病率约为1/3000，与种族、人种或性别无关。注意缺陷多动障碍（ADHD）样特征经常在NF 1患者中报告。我们假设NF 1儿童的学习障碍与ADHD症状有关。哌甲酯（MPD）治疗通过作用于神经递质改善了ADHD的学习障碍。我们的目的是评估其对神经纤维瘤病1型儿童（7-12岁）ADHD样症状的疗效。这是一项随机、双盲、安慰剂对照和交叉试验，在具有ADHD样症状的NF 1儿童中比较了0.5至0.8 mg/kg/d MPD（适用于ADHD）与安慰剂。7至12岁的儿童如果智商在80至120之间，就有资格参加。总随访时间为9周，包括每个阶段4周和1周洗脱期。需要50例受试者（每个阶段25例）来检验主要研究假设。主要结果是简化的Conners父母评定量表评分的改善。2004年4月至2010年12月期间纳入39例患者。在第一阶段，20名参与者接受了MPD，19名接受了安慰剂。他们都完成了审判。MPD使简化Conners评分降低3.9分（±1.1，p = 0. 001）。0003）。这是第一项随机对照试验，显示了MPD对NF 1儿童简化Conners评分的短期益处。ClinicalTrials.gov NCT00169611。
Neurofibromatosis type 1 (NF1) is an autosomal dominant disorder with an estimated prevalence of about 1/3000, independent of ethnicity, race, or gender. Attention Deficit Hyperactivity like Disorder (ADHD)-like characteristics are often reported in patients with NF1. We hypothesised that learning disabilities in NF1 children were related to ADHD symptoms. Treatment with methylphenidate (MPD) has improved learning disabilities in ADHD by acting on neurotransmitters. Our objective was to evaluate its efficacy on ADHD-like symptoms in neurofibromatosis type 1 children (7–12 years). This was a randomised, double blind, placebo controlled, and crossover trial comparing 0.5 to 0.8 mg/kg/d of MPD as it is indicated for ADHD to placebo in NF1 children with ADHD-like symptoms. Children aged 7 to 12 years were eligible when their IQ was between 80 and 120. The total follow-up was 9 weeks including 4 weeks for each period and 1 week wash out. Fifty subjects (25 for each period) were required for testing the primary study hypothesis. The main outcome was an improvement in scores on the simplified Conners’ Parent Rating Scale. Thirty-nine patients were included between April 2004 and December 2010. Twenty participants received MPD and 19 placebo during the first period. They all completed the trial. MPD decreased the simplified Conners by 3.9 points (±1.1, p = 0. 0003). This is the first randomised controlled trial showing the short-term benefit of MPD on simplified Conners scores in NF1 children. ClinicalTrials.gov NCT00169611.
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DOI：
10.1097/00004583-200405000-00009
发表时间：
2004-05-01
影响因子：
13.3
作者：
Charach, A;Ickowicz, A;Schachar, R
通讯作者：
Schachar, R
影响因子：
5.1
作者：
Johnson, NS;Saal, HM;Schorry, EK
通讯作者：
Schorry, EK
影响因子：
3
作者：
Coude, Francois X;Mignot, Claire;Munnich, Arnold
通讯作者：
Munnich, Arnold
DOI：
10.1097/00004583-199908000-00009
发表时间：
1999-08-01
影响因子：
13.3
作者：
Law, SF;Schachar, RJ
通讯作者：
Schachar, RJ
影响因子：
1.5
作者：
Pride, Natalie A.;Payne, Jonathan M.;North, Kathryn N.
通讯作者：
North, Kathryn N.