Nitric oxide donor and non steroidal anti inflammatory drugs as a therapy for muscular dystrophies: Evidence from a safety study with pilot efficacy measures in adult dystrophic patients

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• 作者：Maria Grazia D’ ; Angelo^a ; ^{grazia.dangelo@bp.lnf.it} ; Sandra Gandossini^a ; Filippo Martinelli Boneschi^b ; Clara Sciorati^b ; Sara Bonato^a ; Erika Brighina^a ; Giacomo Pietro Comi^c ; Anna Carla Turconi^a ; Francesca Magri^c ; Giuseppe Stefanoni^a ; Silvia Brunelli^d ; Nereo Bresolin^a ; ^c ; Dario Cattaneo^e ; ^{cattaneo.dario@hsacco.it} ; Emilio Clementi^a ; ^e ; ^{emilio.clementi@unimi.it}
• 关键词：Muscular dystrophies ; Pharmacological treatment ; Nitric oxide donor ; Anti-inflammatory drug ; Safety
• 刊名：Pharmacological Research
• 出版年：2012
• 期刊代码：54_10436618
• 类别：pha
• 出版时间：April, 2012
• 卷：65
• 期：4
• 页码：472-479
• 文件大小：422 K

摘要

This open-label, single centre pilot study was designed to evaluate safety and tolerability of the combination of the drugs isosorbide dinitrate, a nitric oxide donor, and ibuprofen, a non steroid anti-inflammatory drug, in a cohort of adult dystrophic patients (Duchenne, Becker and Limb-Girdle Muscular Dystrophy). Seventy-one patients were recruited: 35, treated with the drug combination for 12 months, and 36 untreated. Safety and adverse events were assessed by reported signs and symptoms, physical examinations, blood tests, cardiac and respiratory function tests. Exploratory outcomes measure, such as the motor function measure scale, were also applied.

Good safety and tolerability profiles of the long-term co-administration of the drugs were demonstrated. Few and transient side effects (i.e. headache and low blood pressure) were reported. Additionally, exploratory outcomes measures were feasible in all the disease population studied and evidenced a trend towards amelioration that reached statistical significance in one dimension of the MFM scale. Systemic administration of ibuprofen and isosorbide dinitrate provides an adequate safety margin for clinical studies aimed at assessing efficacy.