Mazindol in narcolepsy and idiopathic and symptomatic hypersomnia refractory to stimulants: A long-term chart review

• Published in: Sleep medicine 2013-01, Vol.14 (1), p.30-36
• Main Authors: Nittur, Nandini, Konofal, Eric, Dauvilliers, Yves, Franco, Patricia, Leu-Semenescu, Smaranda, Cock, Valérie Cochen De, Inocente, Clara O, Bayard, Sophie, Scholtz, Sabine, Lecendreux, Michel, Arnulf, Isabelle
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Aged; Blood Pressure - drug effects; Cataplexy; Cataplexy - drug therapy; Central Nervous System Stimulants - adverse effects; Central Nervous System Stimulants - therapeutic use; Child; Female; Heart Rate - drug effects; Humans; Hypersomnolence, Idiopathic - drug therapy; Idiopathic hypersomnia; Male; Mazindol; Mazindol - adverse effects; Mazindol - therapeutic use; Middle Aged; Narcolepsy; Narcolepsy - drug therapy; Neurology; Observational study; Retrospective Studies; Sleep Medicine; Sleepiness; Wakefulness - drug effects; Young Adult
• ISSN: 1389-9457; 1878-5506
• DOI: 10.1016/j.sleep.2012.07.008

Abstract Objective Mazindol is a tricyclic, anorectic, non-amphetamine stimulant used in narcolepsy and obesity since 1970. This study aimed to evaluate the long-term benefit/risk ratio in drug-resistant hypersomniacs and cataplexy sufferers. Methods By retrospective analysis of the patients’ files in the hospitals of Paris-Salpêtrière ( n = 91), Montpellier ( n = 40) and Lyon ( n = 8), the benefit (Epworth Sleepiness Score (ESS), cataplexy frequency, authorization renewal) and tolerance (side-effects, vital signs, electrocardiogram and cardiac echography) of mazindol were assessed. Results The 139 patients (45% men) aged 36 ± 15 years (range: 9–74) suffered narcolepsy ( n = 94, 66% with cataplexy), idiopathic ( n = 37) and symptomatic hypersomnia ( n = 8) refractory to modafinil, methylphenidate and sodium oxybate. Under mazindol (3.4 ± 1.3 mg/day, 1–6 mg) for an average of 30 months, the ESS decreased from 17.7 ± 3.5 to 12.8 ± 5.1, with an average fall of −4.6 ± 4.7 ( p < 0.0001) and the frequency of cataplexy fell from 4.6 ± 3.1 to 2 ± 2.8 episodes per week. The cataplexy was eliminated in 14.5% of patients, improved in 27.5%, and unchanged in 29% (missing data in 29%). The treatment was maintained long term in 83 (60%) patients, and stopped because of a lack of efficacy (22%) and/or secondary effects (9%). There was no pulmonary hypertension in the 45 patients who underwent a cardiac echography. The most common adverse effects were dry mouth (13%), palpitations (10%, including one with ventricular hyperexcitability), anorexia (6%), nervousness (6%) and headaches (6%). Conclusion Mazindol has a long-term, favorable benefit/risk ratio in 60% of drug-resistant hypersomniacs, including a clear benefit on cataplexy.