12 In the open-label extension, patients who enrolled were treated for at least an additional 12 months with active drug regardless of their randomization group in the parent study. This is a post hoc analysis using data from a previously published randomized, placebo-controlled, double-blind, parallel-group trial (GHB-2 N = 136) 11 and its open-label extension (GHB-3 N = 118). These analyses were based on data from previous SXB clinical studies. Therefore, the objective of this analysis was to determine the time to response with SXB for the treatment of EDS and cataplexy in patients with narcolepsy. Knowing the time frame in which, on average, a response can be expected may be helpful to set expectations for patients as well as clinicians. 7 – 10 Patient management with SXB relies on titration to efficacy and tolerability, and as with any of the recommended narcolepsy therapies in clinical practice, there may be variability in the observed response both in terms of the degree of clinical improvement as well as the time needed to achieve a clinical response. Sodium oxybate (SXB) is the only medication approved for the treatment of both EDS and cataplexy in narcolepsy, 6 and clinical trials have demonstrated that SXB results in significant improvement for both of these symptoms relative to placebo. Clinicians should recognize that the time course to initial and maximum response to sodium oxybate may take weeks to months. ![]() Study Impact: Clinically meaningful improvements in excessive daytime sleepiness and cataplexy were seen within 2 months in most patients, and maximum response required a longer period. Since it is important to allow sufficient time to observe a clinically relevant response before changing a treatment strategy, this analysis determined the time to response with sodium oxybate for improvement in excessive daytime sleepiness and cataplexy. 5Ĭurrent Knowledge/Study Rationale: Sodium oxybate is approved for the treatment of excessive daytime sleepiness and cataplexy in patients with narcolepsy. ![]() 4 Thus, these two symptoms represent the main symptoms patients want treated and are the targets of currently approved narcolepsy therapies. 2 By definition, EDS is present in all narcolepsy patients, and although the presence of cataplexy provides confirmation of a narcolepsy diagnosis, it is only present in up to 70% of patients but is considered the most pathognomonic of all the symptoms. Among the pentad, EDS and cataplexy are the two symptoms that are most frequently recognized. ![]() Symptom presentation among narcolepsy patients is variable, since not all symptoms may be present, and when present, may differ in severity and may be described in different ways. Since narcolepsy has no known cure, patient management is driven by the symptomatic response. 3 Symptomatically, the disease is characterized by the pentad of excessive daytime sleepiness (EDS), cataplexy, hypnagogic or hypnopompic hallucinations, sleep paralysis, and disturbed nocturnal sleep. 1, 2Īccumulating evidence supports an autoimmune response for the loss of hypocretin-producing neurons in the brain that is the underlying pathophysiology of narcolepsy. 1 This delay likely results, at least in part, from the reported low awareness among physicians and the general population of narcolepsy symptoms and their associated impact. Despite recent advances in the understanding of the chronic neurological disorder known as narcolepsy, it remains a condition that often has a long delay between symptom onset, symptom recognition and, most importantly, clinical diagnosis and treatment.
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