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Effects of Pharmacotherapy Treatment on Patient-Reported Outcomes in a Narcolepsy and Idiopathic Hypersomnia Cohort.

ABSTRACT: STUDY OBJECTIVE:We aimed to evaluate the association between patient-reported outcomes (PROs) and treatment regimen/standardized dose (STD), a measure of drug burden, in patients with narcolepsy type 1 (NT1)/type 2 (NT2) and idiopathic hypersomnia (IH). METHODS:Patients age 18 years or older with NT1/NT2 and IH with baseline and ? 6-month follow-up during 2008-2010 were included. Changes in PROs (Epworth Sleepiness Scale [ESS], Fatigue Severity Scale [FSS], Patient Health Questionnaire 9 [PHQ-9], total sleep time [TST]) by diagnosis, treatment regimen (monotherapy versus polytherapy, sodium oxybate [SO] use), and STD were assessed by t tests and univariable/multivariable linear regressions, adjusting for patient characteristics. RESULTS:A total of 92 patients (26 [28.3%] NT1, 27 [29.3%] NT2, 39 [42.4%] IH) were included (age 43.8 ± 14.8 years; 66 [71.7%] female). Baseline PROs suggested excessive daytime sleepiness (ESS 14.2 ± 5.2 [74% patients > 10]), significant fatigue (FSS 47.5 ± 12.9), and mild depression (PHQ-9 9.0 [4.0, 14.0] [49.4% ? 10]). At follow-up, ESS and PHQ-9 improved significantly overall and within diagnostic, monotherapy/polytherapy, and SO use groups (all P < .01). FSS improved significantly overall (P = .016), but improvements were not significant for IH, monotherapy, polytherapy, and non-SO using groups. In multivariable models, PRO changes were not significantly different between groups, but baseline STD was associated with worsening PHQ-9 across PHQ-9 change models, and ESS worsened with increasing STD at follow-up (P = .056). CONCLUSIONS:Significant improvements in sleep-related PROs were seen with pharmacotherapy use, regardless of diagnosis or treatment type, highlighting the importance of individualized prescribing decisions for this population.

SUBMITTER: Pascoe M

PROVIDER: S-EPMC7099187 | biostudies-literature | 2019 Dec

REPOSITORIES: biostudies-literature

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Effects of Pharmacotherapy Treatment on Patient-Reported Outcomes in a Narcolepsy and Idiopathic Hypersomnia Cohort.

Pascoe Maeve M Bena James J Foldvary-Schaefer Nancy N

Journal of clinical sleep medicine : JCSM : official publication of the American Academy of Sleep Medicine 20191030 12

<h4>Study objective</h4>We aimed to evaluate the association between patient-reported outcomes (PROs) and treatment regimen/standardized dose (STD), a measure of drug burden, in patients with narcolepsy type 1 (NT1)/type 2 (NT2) and idiopathic hypersomnia (IH).<h4>Methods</h4>Patients age 18 years or older with NT1/NT2 and IH with baseline and ≥ 6-month follow-up during 2008-2010 were included. Changes in PROs (Epworth Sleepiness Scale [ESS], Fatigue Severity Scale [FSS], Patient Health Question ...[more]

PMID: 31855165

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Project description:Narcolepsy and idiopathic hypersomnia are chronic conditions that negatively affect alertness, mental and physical energy, functioning, and quality of life (QoL). Calcium, magnesium, potassium, and sodium oxybates (low-sodium oxybate; LXB) is an oxybate formulation with 92% less sodium than sodium oxybate (SXB; a treatment for narcolepsy) and the same active moiety. LXB is approved in the US for treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age or older with narcolepsy, and idiopathic hypersomnia in adults. In Phase 3 clinical trials, LXB exhibited a safety profile consistent with that of SXB in narcolepsy. Besides continued efficacy in treating symptoms, potential benefits of long-term LXB treatment include flexible optimization of dosing and regimen, improvement of QoL and functioning, weight loss, and (relative to SXB in narcolepsy) health benefits of reduced sodium content. Dosing of LXB is twice nightly (for narcolepsy) or once or twice nightly (for idiopathic hypersomnia) based on patient characteristics and response, and individualized titration can be leveraged over the long term as a patient's life circumstances change. Patients with narcolepsy transitioning from SXB initiate LXB at the same dose, and most patients require no further changes to achieve similar efficacy and tolerability. Improvements in functioning and QoL with LXB treatment could have cascading positive effects in multiple domains, particularly in younger patients. In clinical trials, LXB was associated with weight loss in both narcolepsy (in which obesity is a well-established comorbidity) and idiopathic hypersomnia, only occasionally leading participants to be underweight. As both narcolepsy and idiopathic hypersomnia are associated with increased risk of cardiometabolic and cardiovascular comorbidities, limiting medication-related sodium intake with LXB may have significant health benefits, although this has not yet been verified prospectively due to the prolonged follow-up required. LXB is a promising long-term treatment for narcolepsy and idiopathic hypersomnia.

Project description:Study objectivesBasic experiments support the impact of hypocretin on hyperarousal and motivated state required for increasing drug craving. Our aim was to assess the frequencies of smoking, alcohol and drug use, abuse and dependence in narcolepsy type 1 (NT1, hypocretin-deficient), narcolepsy type 2 (NT2), idiopathic hypersomnia (IH) (non-hypocretin-deficient conditions), in comparison to controls. We hypothesized that NT1 patients would be less vulnerable to drug abuse and addiction compared to other hypersomniac patients and controls from general population.MethodsWe performed a cross-sectional study in French reference centres for rare hypersomnia diseases and included 450 adult patients (median age 35 years; 41.3% men) with NT1 (n = 243), NT2 (n = 116), IH (n = 91), and 710 adult controls. All participants were evaluated for alcohol consumption, smoking habits, and substance (alcohol and illicit drug) abuse and dependence diagnosis during the past year using the Mini International Neuropsychiatric Interview.ResultsAn increased proportion of both tobacco and heavy tobacco smokers was found in NT1 compared to controls and other hypersomniacs, despite adjustments for potential confounders. We reported an increased regular and frequent alcohol drinking habit in NT1 versus controls but not compared to other hypersomniacs in adjusted models. In contrast, heavy drinkers were significantly reduced in NT1 versus controls but not compared to other hypersomniacs. The proportion of patients with excessive drug use (codeine, cocaine, and cannabis), substance dependence, or abuse was low in all subgroups, without significant differences between either hypersomnia disorder categories or compared with controls.ConclusionsWe first described a low frequency of illicit drug use, dependence, or abuse in patients with central hypersomnia, whether Hcrt-deficient or not, and whether drug-free or medicated, in the same range as in controls. Conversely, heavy drinkers were rare in NT1 compared to controls but not to other hypersomniacs, without any change in alcohol dependence or abuse frequency. Although disruption of hypocretin signaling in rodents reduces drug-seeking behaviors, our results do not support that hypocretin deficiency constitutes a protective factor against the development of drug addiction in humans.

Dataset Information

Effects of Pharmacotherapy Treatment on Patient-Reported Outcomes in a Narcolepsy and Idiopathic Hypersomnia Cohort.

Publications

Effects of Pharmacotherapy Treatment on Patient-Reported Outcomes in a Narcolepsy and Idiopathic Hypersomnia Cohort.

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