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.
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.
E Medea Scientific Institute, Bosisio Parini, Italy. grazia.dangelo@bp.lnf.it
Maria Grazia
MG
D'Angelo
Sandra
S
Gandossini
Filippo
F
Martinelli Boneschi
Clara
C
Sciorati
Sara
S
Bonato
Erika
E
Brighina
Giacomo Pietro
GP
Comi
Anna Carla
AC
Turconi
Francesca
F
Magri
Giuseppe
G
Stefanoni
Silvia
S
Brunelli
Nereo
N
Bresolin
Dario
D
Cattaneo
Emilio
E
Clementi
D000293:Adolescent; D000328:Adult; D000894:Anti-Inflammatory Agents, Non-Steroidal / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D007052:Ibuprofen / Q000627:therapeutic use*; D007548:Isosorbide Dinitrate / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D009136:Muscular Dystrophies / Q000188:drug therapy*; D020030:Nitric Oxide Donors / Q000627:therapeutic use*; D016896:Treatment Outcome; D055815:Young Adult
D000894:Anti-Inflammatory Agents, Non-Steroidal; D020030:Nitric Oxide Donors; D007548:Isosorbide Dinitrate; D007052:Ibuprofen