Quality Measures for the Care of Patients with Narcolepsy

Krahn, Lois E.; Hershner, Shelley; Loeding, Lauren D.; Maski, Kiran; Rifkin, Daniel I.; Selim, Bernardo J.; Watson, Nathaniel F.

doi:10.5664/jcsm.4554

Cited by 36 publications

(28 citation statements)

References 36 publications

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“…Results of the survey also showed a broader range of symptoms associated with narcolepsy. Although clinical and research outcome measures focus on assessments of excess daytime sleepiness and cataplexy, 18 participants reported their most troublesome symptoms included general fatigue and subjective cognitive difficulties (difficulty thinking, remembering, concentrating, or paying attention). Depression was the most common comorbidity with narcolepsy reported by 25% of participants.…”

Section: Discussionmentioning

confidence: 99%

“…This pilot test also helped gauge which questions required free text responses. The survey was accessible to patients from August 26, 2013 to November 15, 2013 on a Unite Narcolepsy website 18 and promoted through patient advocacy websites, social media, web postings, and web advertisements. Interim analysis of the data was presented at the FDA public meeting on September 24, 2014 and available on the Unite Narcolepsy website.…”

Section: Survey Instrumentmentioning

confidence: 99%

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Listening to the Patient Voice in Narcolepsy: Diagnostic Delay, Disease Burden, and Treatment Efficacy

Maski

Steinhart

Williams

et al. 2017

Journal of Clinical Sleep Medicine

111

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Study Objectives: Describe common symptoms, comorbidities, functional limitations, and treatment responsiveness among patients with narcolepsy. Investigate the effect of pediatric onset of narcolepsy symptoms on time to diagnosis of narcolepsy and presence of comorbid depression. Methods: Cross-sectional survey of 1,699 people in the United States with self-reported diagnosis of narcolepsy. We utilized mixed-methods data analyses to report study findings. Results: Most participants reported receiving a diagnosis of narcolepsy more than 1 y after symptom onset. We found that the strongest predictor of this delayed diagnosis was pediatric onset of symptoms (odds ratio = 2.4, p < 0.0005). Depression was the most common comorbidity but we detected no association with pediatric onset of narcolepsy symptoms. Overall, participants reported that fatigue and cognitive difficulties were their most burdensome symptoms in addition to sleepiness and cataplexy. The majority of participants reported residual daytime fatigue and/or sleepiness despite treatment. Most participants reported they could not perform at work or school as well as they would like because of narcolepsy symptoms. Conclusions: This study provides unique insight into the narcolepsy disease experience. The study quantifies the problem of diagnostic delay for narcolepsy patients in the United States and highlights that symptoms are more likely to be missed if they develop before 18 y of age. These results suggest that narcolepsy awareness efforts should be aimed at parents, pediatric health care providers, school professionals, and children/adolescents themselves. Disease burden is high because of problems with fatigue, cognition, and persistence of residual symptoms despite treatment.

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Section: Discussionmentioning

confidence: 99%

Section: Survey Instrumentmentioning

confidence: 99%

Listening to the Patient Voice in Narcolepsy: Diagnostic Delay, Disease Burden, and Treatment Efficacy

Maski

Steinhart

Williams

et al. 2017

Journal of Clinical Sleep Medicine

111

View full text Add to dashboard Cite

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“…Treatment focuses on the following: 1) reduce EDS for fullest return of normal function; 2) minimize nocturnal sleep disruption; 3) treat cataplexy, hypnagogic hallucination (HH), and sleep paralysis (SP); and 4) consider risk–benefit ratio of the drug, cost of medications and ongoing care, and convenience of administration 16. Quality measures formulated for the care of patients with narcolepsy include three outcome measures – 1) reducing EDS, 2) improving diagnostic accuracy, and 3) reducing adverse events 17. Seven process measures were designated based on proportion of patients who met criteria that reflect these outcomes 17.…”

Section: Managementmentioning

confidence: 99%

New developments in the management of narcolepsy

Abad

Guilleminault

2017

NSS

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Narcolepsy is a life-long, underrecognized sleep disorder that affects 0.02%–0.18% of the US and Western European populations. Genetic predisposition is suspected because of narcolepsy’s strong association with HLA DQB1*06-02, and genome-wide association studies have identified polymorphisms in T-cell receptor loci. Narcolepsy pathophysiology is linked to loss of signaling by hypocretin-producing neurons; an autoimmune etiology possibly triggered by some environmental agent may precipitate hypocretin neuronal loss. Current treatment modalities alleviate the main symptoms of excessive daytime somnolence (EDS) and cataplexy and, to a lesser extent, reduce nocturnal sleep disruption, hypnagogic hallucinations, and sleep paralysis. Sodium oxybate (SXB), a sodium salt of γ hydroxybutyric acid, is a first-line agent for cataplexy and EDS and may help sleep disruption, hypnagogic hallucinations, and sleep paralysis. Various antidepressant medications including norepinephrine serotonin reuptake inhibitors, selective serotonin reuptake inhibitors, and tricyclic antidepressants are second-line agents for treating cataplexy. In addition to SXB, modafinil and armodafinil are first-line agents to treat EDS. Second-line agents for EDS are stimulants such as methylphenidate and extended-release amphetamines. Emerging therapies include non-hypocretin-based therapy, hypocretin-based treatments, and immunotherapy to prevent hypocretin neuronal death. Non-hypocretin-based novel treatments for narcolepsy include pitolisant (BF2.649, tiprolisant); JZP-110 (ADX-N05) for EDS in adults; JZP 13-005 for children; JZP-386, a deuterated sodium oxybate oral suspension; FT 218 an extended-release formulation of SXB; and JNJ-17216498, a new formulation of modafinil. Clinical trials are investigating efficacy and safety of SXB, modafinil, and armodafinil in children. γ-amino butyric acid (GABA) modulation with GABAA receptor agonists clarithromycin and flumazenil may help daytime somnolence. Other drugs investigated include GABAB agonists (baclofen), melanin-concentrating hormone antagonist, and thyrotropin-releasing hormone agonists. Hypocretin-based therapies include hypocretin peptide replacement administered either through an intracerebroventricular route or intranasal route. Hypocretin neuronal transplant and transforming stem cells into hypothalamic neurons are also discussed in this article. Immunotherapy to prevent hypocretin neuronal death is reviewed.

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“…In order to improve the quality of care given to patients with sleep disorders, clinicians must have valid, reliable, and practical tools to evaluate the effects of implementing recommended interventions and tests. The AASM, in consultation with numerous stakeholders, has developed Quality Measures for the diagnosis and management of several sleep disorders [20][21][22][23][24][25] ; many of these measures are based upon previously established clinical practice recommendations. Going forward, strong recommendations for or against a patient-care strategy will form the foundation of new quality measures, or be used to update existing quality measures.…”

Section: Strongmentioning

confidence: 99%

The Evolution of the AASM Clinical Practice Guidelines: Another Step Forward

Morgenthaler

Deriy

Heald

et al. 2016

Journal of Clinical Sleep Medicine

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One of the goals of the American Academy of Sleep Medicine (AASM) is to provide clear, evidence-based recommendations in our clinical practice guidelines. Periodically, the AASM will assess and update the process by which these guidelines are developed so that it is in line with the standards currently being used for guideline development. The AASM is now taking the next step forward by fully adopting GRADE (Grading of Recommendation Assessment, Development and Evaluation) as the methodology used for evaluating evidence and forming clinical practice guidelines recommendations. Starting this year, AASM recommendations will be based on the following four interdependent domains: 1. quality of evidence; 2. balance of desirable and undesirable consequences; 3. patients' values and preferences; and 4. resource use (when known). AASM strengths of recommendations will be dichotomized into two categories: "Strong" and "Weak," either for or against a patient-care strategy. In an effort to provide clarity and transparency, all AASM recommendations will be actionable statements that include the specific patient population for which the patient-care strategy is recommended, and clearly define the comparator against which the patient-care strategy was evaluated. In some recommendations, the comparator will be an alternative patient-care strategy (e.g., a "gold standard" or previously available alternative), while in other recommendations the comparator will be a placebo or no treatment; this is determined by the availability of evidence, and analyses decisions made by the AASM task force. Implementation of the complete GRADE criteria by the AASM allows us the best path forward towards continuing to provide high quality clinical practice guidelines. I NTRO DUCTI O NAmerican Academy of Sleep Medicine clinical practice guidelines (and the prior Practice Parameters and Best Practice Guides) are developed to bring the fruits of clinical and basic research into practice, offering guidance to practicing sleep specialists and other health care professionals who work with patients and families who suffer from sleep disorders. These guidelines have influenced clinical practice in the office and at the bedside, on a national and international level. It is therefore imperative that the very best efforts and techniques be applied to produce guidelines that are accurate, clear, and reliable. This document will describe some recent evolutionary changes to how recommendations will be developed by the AASM and how they will appear in AASM clinical practice guidelines, beginning this year.In 2009, the AASM began adopting GRADE (Grading of Recommendations Assessment, Development, and Evaluation) as the methodology for evaluating evidence and forming clinical practice guideline recommendations.1 The major impetus for this change was that, compared to the prior employed methodologies which heavily emphasized study design, GRADE placed weight on the systematic evaluation of a wider spectrum of characteristics of the evidence (e.g., not only study...

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Quality Measures for the Care of Patients with Narcolepsy

Cited by 36 publications

References 36 publications

Listening to the Patient Voice in Narcolepsy: Diagnostic Delay, Disease Burden, and Treatment Efficacy

Listening to the Patient Voice in Narcolepsy: Diagnostic Delay, Disease Burden, and Treatment Efficacy

New developments in the management of narcolepsy

The Evolution of the AASM Clinical Practice Guidelines: Another Step Forward

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