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Efficacy of modafinil on excessive daytime sleepiness in Prader-Willi syndrome
V Cochen De Cock1, G Diene, C Molinas
1Service de Neurologie, Hôpital Purpan, Toulouse, France.
Insights
Modafinil effectively reduced excessive daytime sleepiness in children with Prader-Willi Syndrome (PWS). This study found the central stimulant well-tolerated and beneficial for improving daily routines disrupted by sleepiness.
Area of Science:
- Pediatric Neurology
- Sleep Medicine
- Genetics
Background:
- Excessive daytime sleepiness is a common and disruptive symptom in Prader-Willi Syndrome (PWS).
- Current treatments for sleepiness in PWS are limited, impacting patient quality of life.
- Understanding sleep patterns and effective interventions is crucial for managing PWS.
Purpose of the Study:
- To evaluate the efficacy of modafinil in treating excessive daytime sleepiness in pediatric patients with PWS.
- To assess the impact of modafinil on sleep latency and overall sleepiness scores.
- To determine the safety and tolerability of modafinil in this population.
Main Methods:
- An open-label pilot study involving nine children and adolescents with confirmed PWS and excessive daytime sleepiness.
- Assessment included the Epworth Sleepiness Scale (ESS) and Multiple Sleep Latency Tests (MSLT) before and after treatment.
- Patients received modafinil, with dosage adjustments as needed; one patient with severe obstructive sleep apnea was excluded.
Main Results:
- All nine participants experienced significant improvement in excessive daytime sleepiness, with ESS scores decreasing from a median of 14 to 4 (P = 0.007).
- MSLT revealed a mean sleep latency of 5 minutes, with six patients exhibiting a narcolepsy-like phenotype (≥2 sleep-onset REM periods).
- Modafinil was well-tolerated with no reported side effects, and body mass index remained stable.
Conclusions:
- Modafinil demonstrates significant efficacy in reducing excessive daytime sleepiness in children and adolescents with Prader-Willi Syndrome.
- The findings suggest modafinil is a safe and well-tolerated treatment option for this symptom in PWS.
- Further research, including larger controlled trials, is warranted to confirm these promising results.
Abstract:
Excessive daytime sleepiness is a frequent and a highly disruptive symptom to the daily routine of children with Prader-Willi Syndrome (PWS) and their families. The objective of the study was to evaluate the efficacy of modafinil, a central stimulant, on excessive daytime sleepiness in children and adolescents with PWS. The efficacy of modafinil was evaluated in this open label pilot study comparing the Epworth sleepiness scale before and after treatment. Ten patients with molecularly confirmed PWS and a complaint of excessive daytime sleepiness underwent a night-time sleep recording and multiple sleep latency tests. One patient was excluded because of severe obstructive sleep apnea syndrome. Nine patients (4 males) with median age of 16 years (8-21) received modafinil at a starting dose of 100 mg/day. We found that all patients had excessive daytime sleepiness with an Epworth sleepiness scale at 14 (11-20) and mean sleep latency on multiple sleep latency tests at 5 (3-6) minutes. Moreover, six patients had at least two sleep-onset rapid eye movement periods showing a narcolepsy-like phenotype. Modafinil significantly improved sleepiness in all patients on the Epworth sleepiness scale from 14 (11-20) to 4 (3-12), (P = 0.007). Body mass index of the patients did not change significantly under treatment. No side effects were reported, and the drug was well-tolerated. We posit that this open label case series shows good efficacy of modafinil in nine children and adolescents with PWS.
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