0603 Novel Treatments Should Be Considered For Patients with Narcolepsy

Abstract

Introduction: Recognized by the National Organization for Rare Disorders, narcolepsy is characterized by debilitating sleepiness (type 1 and type 2) and cataplexy (type 1). Medications for narcolepsy have dangerous side effects and potential for abuse. Patients often have residual symptoms despite treatment. Pitolisant, a selective histamine H3-receptor modulator, recently became available for the treatment of sleepiness and cataplexy. We hypothesized that many patients with narcolepsy have residual symptoms and may benefit from treatment with pitolisant. Methods: We conducted a retrospective, electronic chart review using ICD-9-CM and ICD-10-CM narcolepsy-related diagnostic codes (347.01; G47.411; 347.00; G47.419; 347.10; 347.11) of outpatients evaluated at Rush University Medical Center between June 2011 and December 2018. Records were queried for demographics, medical comorbidities, polysomnography (PSG) and multiple sleep latency tests (MSLT), symptoms (sleepiness, cataplexy, hypnopompic/hypnogogic hallucinations, sleep paralysis, sleep fragmentation), and medication use. Results: Of the 97 patients analyzed, patients were predominantly white (56.2%), middle aged (39 years, SD=15.64), overweight (BMI: 28.22, SD= 8.03 kg/m2) and female (58%). A minority of patients had narcolepsy type 1 (35%). On MSLT, the average mean sleep latency and number of SOREMPs was 4.8 minutes (SD=3.9 min) and 2.24 (SD=1.5), respectively. The most common medical comorbidity was obstructive sleep apnea (38.1%), followed by depression (24.7%) and hypertension (19.6%). Only 16.5% of patients reported insufficient sleep (Total sleep time <7 hours). Residual sleepiness and sleep fragmentation were reported in 64.9% and 29.9% of patients, respectively. Among patients with narcolepsy type 1, 59% reported residual cataplexy. Overall, 75.3% of patients reported at least one residual symptom. Modafinil was most commonly prescribed (41.2%), followed by amphetamines (32%), antidepressants (25.8%), and sodium oxybate (21.6%). Many patients were taking at least two medications (26.8%) and some were taking three medications (10.3%). Conclusion: At a large tertiary care center, over three quarters of patients with narcolepsy reported residual symptoms. Recognizing patients at risk leads to increased access to new treatments, including pitolisant. More research is needed to assess impact of pitolisant access on patient outcomes.