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

Maria Grazia D'Angelo, Sandra Gandossini, Filippo Martinelli Boneschi, Clara Sciorati, Sara Bonato, Erika Brighina, Giacomo Pietro Comi, Anna Carla Turconi, Francesca Magri, Giuseppe Stefanoni, Silvia Brunelli, Nereo Bresolin, Dario Cattaneo, Emilio Clementi

Research output: Contribution to journalArticle

Abstract

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.

Original languageEnglish
Pages (from-to)472-479
Number of pages8
JournalPharmacological Research
Volume65
Issue number4
DOIs
Publication statusPublished - Apr 2012

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Nitric Oxide Donors
Muscular Dystrophies
Anti-Inflammatory Agents
Isosorbide Dinitrate
Safety
Ibuprofen
Drug Combinations
Pharmaceutical Preparations
Heart Function Tests
Outcome Assessment (Health Care)
Limb-Girdle Muscular Dystrophies
Respiratory Function Tests
Hematologic Tests
Therapeutics
Hypotension
Signs and Symptoms
Physical Examination
Headache
Steroids
Population

Keywords

  • Anti-inflammatory drug
  • Muscular dystrophies
  • Nitric oxide donor
  • Pharmacological treatment
  • Safety

ASJC Scopus subject areas

  • Pharmacology

Cite this

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abstract = "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.",
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AU - D'Angelo, Maria Grazia

AU - Gandossini, Sandra

AU - Boneschi, Filippo Martinelli

AU - Sciorati, Clara

AU - Bonato, Sara

AU - Brighina, Erika

AU - Comi, Giacomo Pietro

AU - Turconi, Anna Carla

AU - Magri, Francesca

AU - Stefanoni, Giuseppe

AU - Brunelli, Silvia

AU - Bresolin, Nereo

AU - Cattaneo, Dario

AU - Clementi, Emilio

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