The effect of methylphenidate on neurofibromatosis type 1: a randomised, double-blind, placebo-controlled, crossover trial

被引:37
作者
Lion-Francois, Laurence [1 ,17 ,18 ]
Gueyffier, Francois [2 ,3 ]
Mercier, Catherine [4 ,5 ,6 ,7 ]
Gerard, Daniel [8 ]
Herbillon, Vania [1 ]
Kemlin, Isabelle [9 ,10 ]
Rodriguez, Diana [9 ,10 ,11 ]
Ginhoux, Tiphanie [12 ,13 ]
Peyric, Emeline [1 ]
Coutinho, Virginie [9 ,10 ]
Breant, Valentine [14 ]
des Portes, Vincent [1 ,15 ]
Pinson, Stephane [16 ]
Combemale, Patrick [17 ]
Kassai, Behrouz [12 ]
机构
[1] Mother & Child Hosp, Dept Pediat Neurol, 59 Blvd Pinel, F-69677 Bron, France
[2] Univ Lyon, UMR 5558, Dept Clin Pharmacol & Clin Trials, F-69000 Lyon, France
[3] Hop L Pradel, CHU Lyon, F-69000 Lyon, France
[4] Hosp Civils Lyon, Serv Biostat, F-69003 Lyon, France
[5] Univ Lyon, F-69000 Lyon, France
[6] Univ Lyon 1, F-69100 Villeurbanne, France
[7] Equipe Biotatist Sante, Lab Biometrie & Biol Evolut, UMR 5558, F-69100 Villeurbanne, France
[8] CHU Lyon, Grp Hosp Est, Dept Child Psychiat, F-69000 Lyon, France
[9] Paris Univ Hosp East, Armand Trousseau Hosp, AP HP, Dept Pediat Neurol, F-75012 Paris, France
[10] Paris Univ Hosp East, Armand Trousseau Hosp, Neurofibromatosis Reference Ctr, F-75012 Paris, France
[11] Univ Paris 06, F-75012 Paris, France
[12] Univ Lyon, CIC1407, INSERM, F-69000 Lyon, France
[13] Hop L Pradel, CHU Lyon, Dept Clin Pharmacol & Clin Trials, EPICIME, F-69000 Lyon, France
[14] CHU Lyon, Grp Hosp Est, Hosp Civils Lyon, Pharm, F-69000 Lyon, France
[15] Univ Lyon, F-69008 Lyon, France
[16] Hop Edouard Herriot, Human Genet Lab, F-69000 Lyon, France
[17] Reseau NF1 Rhone Alpes Auvergne IHOP, Lyon, France
[18] Hop Femme, Serv Neurol Pediat, Mere, Enfant, F-69677 Bron, France
关键词
Neurofibromatosis; Randomised controlled trials; Methylphenidate; Attention deficit hyperactivity disorder; ATTENTION-DEFICIT DISORDER; CHILDREN; HYPERACTIVITY; ADHD; ADOLESCENTS;
D O I
10.1186/s13023-014-0142-4
中图分类号
Q3 [遗传学];
学科分类号
071007 ; 090102 ;
摘要
Background: 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). Methods: 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. Results: 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). Conclusions: This is the first randomised controlled trial showing the short-term benefit of MPD on simplified Conners scores in NF1 children.
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页数:8
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