The Nexus Narcolepsy Registry: methodology, study population characteristics, and patterns and predictors of narcolepsy diagnosis.

Objective/background: The real-world experience of people with narcolepsy is not well understood.

Patients/methods: The Nexus Narcolepsy Registry (NCT02769780) is a longitudinal, web-based patient registry of self-reported data from adults with physician-diagnosed narcolepsy. Surveys were electronically distributed every 6 months; the current analysis reports registry population demographics, narcolepsy diagnosis journey, and predictors of diagnostic delays.

Assessing the impact of sodium oxybate treatment on functioning, productivity, and health-related quality of life in patients with narcolepsy: findings from the Nexus Narcolepsy Registry (waves 1-4).

Background: The aim of this study was to evaluate the impact of different therapy regimens, including sodium oxybate (SXB)-containing regimens, on patient-reported outcomes (PROs) in people with narcolepsy.

Methods: Online surveys were used to collect information from persons with narcolepsy in the Nexus Narcolepsy Registry. Surveys contained questionnaires assessing self-reported sleep quality (SQ; via single question), daytime sleepiness and function (Epworth Sleepiness Scale and Functional Outcomes of Sleep Questionnaire), health-related quality of life (HRQoL; 36-Item Short Form Health Survey [SF-36]), work productivity and impairment (Work Productivity and Activity Impairment: Specific Health Problem), and history of injuries or motor vehicle accidents.

Relationship between sleep efficacy endpoints and measures of functional status and health-related quality of life in participants with narcolepsy or obstructive sleep apnea treated for excessive daytime sleepiness.

This study examined the correlation between improvements in excessive daytime sleepiness in participants with obstructive sleep apnea or narcolepsy and changes in functional status, work productivity and health-related quality of life. Data from two 12-week randomized controlled trials of solriamfetol were analyzed. Participants completed the Epworth Sleepiness Scale, 10-item Functional Outcomes of Sleep Questionnaire, Work Productivity and Activity Impairment questionnaire and 36-Item Short Form Health Survey and performed the Maintenance of Wakefulness Test at baseline and weeks 4, 8 and 12.

Understanding the burden of illness of excessive daytime sleepiness associated with obstructive sleep apnea: a qualitative study.

Background: Obstructive sleep apnea (OSA) is associated with excessive daytime sleepiness (EDS), which may go undiagnosed and can significantly impair a patient's health-related quality of life (HRQOL). This qualitative research examined timing and reasons patients sought medical care for their EDS and OSA symptoms, and the impact of EDS on HRQOL.

Methods: Focus groups were conducted in 3 US cities with 42 participants currently experiencing EDS with OSA.

Healthcare resource utilization in a phase 3 study of CPX-351 in patients with newly diagnosed high-risk/secondary acute myeloid leukemia.

Treatment of acute myeloid leukemia (AML) requires significant healthcare resource utilization (HRU), including lengthy hospitalizations. In a phase 3 study (NCT01696084), CPX-351 (Vyxeos) showed significant benefits to overall survival and complete remission versus conventional 7 + 3 cytarabine/daunorubicin. This analysis evaluated HRU in patients aged 60-75 years with newly diagnosed high-risk/secondary AML treated with CPX-351 versus 7 + 3 in the phase 3 study.

Measures of functional outcomes, work productivity, and quality of life from a randomized, phase 3 study of solriamfetol in participants with narcolepsy.

Objective: Solriamfetol (formerly JZP-110), a dopamine/norepinephrine reuptake inhibitor, is approved in the US to improve wakefulness in adults with excessive daytime sleepiness associated with narcolepsy (75-150 mg/d) or obstructive sleep apnea (37.5-150 mg/d). In a randomized, double-blind, placebo-controlled trial in participants with narcolepsy, effects of solriamfetol on functional status, health-related quality of life (HRQoL), and work productivity were evaluated.

Burden of disease in pediatric narcolepsy: a claims-based analysis of health care utilization, costs, and comorbidities.

Background: This study analyzed a privately insured pediatric population with and without narcolepsy to determine the impact of pediatric narcolepsy on comorbidities, health care utilization, and cost. Additional analyses compared narcolepsy type 1 and type 2.

Methods: This retrospective cross-sectional study identified US patients with narcolepsy <18 years of age with ≥2 claims with a diagnosis code of narcolepsy using Truven MarketScan® data 2011 to 2015.

Consolidation outcomes in CPX-351 versus cytarabine/daunorubicin-treated older patients with high-risk/secondary acute myeloid leukemia.

CPX-351 is a dual-drug liposomal encapsulation of cytarabine/daunorubicin. In a phase 3 study (ClinicalTrials.gov Identifier: NCT01696084), patients aged 60-75 years with newly diagnosed, high-risk/secondary AML received 1-2 induction cycles with CPX-351 or 7 + 3 chemotherapy; those achieving complete remission (including with incomplete platelet or neutrophil recovery) could receive up to 2 consolidation cycles with CPX-351 or 5 + 2 chemotherapy, respectively.

Publisher Correction: Systematic review of defibrotide studies in the treatment of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS).

The original version of this article included some minor typesetting errors in Table 1-a corrected version of which is provided in this correction article and has been corrected in the original article-and one error in text, which has been corrected in the original article. Furthermore, the article was erroneously published with the copyright holders as 'Springer Nature Limited', however, since the article is Open Access, the copyright holders should in fact be 'The Author(s)'. This has also been corrected in the original article.

Systematic review of defibrotide studies in the treatment of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS).

Veno-occlusive disease (VOD), also called sinusoidal obstruction syndrome (SOS), is a potentially life-threatening complication of hematopoietic stem cell transplantation (HSCT) conditioning or high-dose nontransplant chemotherapy. VOD/SOS with multi-organ dysfunction (MOD) is associated with a mortality rate of > 80%. Defibrotide (25 mg/kg/day) is approved to treat hepatic VOD/SOS with renal or pulmonary dysfunction post HSCT in the United States and to treat severe hepatic VOD/SOS in patients > 1 month of age in the European Union.

Comorbidities, Health-Related Quality of Life, and Work Productivity Among People With Obstructive Sleep Apnea With Excessive Sleepiness: Findings From the 2016 US National Health and Wellness Survey.

Study Objectives: Few population-based studies have explored how excessive sleepiness (ES) contributes to burden of illness among patients with obstructive sleep apnea (OSA).

Methods: This study utilized data from the annual, cross-sectional 2016 US National Health and Wellness Survey. Respondents self-reporting an OSA diagnosis were categorized as having ES (Epworth Sleepiness Scale [ESS] score ≥ 11) or not having ES (ESS score < 11).

Indirect costs associated with premature mortality among those with veno-occlusive disease/sinusoidal obstruction syndrome with multiorgan dysfunction post-hematopoietic stem-cell-transplant.

Purpose: The study objective was to develop an economic model to assess projected costs of lost productivity associated with premature deaths due to veno-occlusive disease (VOD)/ sinusoidal obstruction syndrome (SOS) with multiorgan dysfunction (MOD) among patients in the US who underwent hematopoietic stem-cell transplant (HSCT) in 2013.

Methods: Data sources included the US Census Bureau and Department of Health, epidemiologic research organizations, and medical research literature. The model considered only lost productivity associated with premature death, with lifetime salary assumed to reflect productivity.

Risk Score for the Development of Veno-Occlusive Disease after Allogeneic Hematopoietic Cell Transplant.

A risk score identifying patients at high risk for veno-occlusive disease (VOD) may aid efforts to study preventive strategies for this uncommon complication of hematopoietic cell transplantation (HCT). Patients receiving a first allogeneic HCT between 2008 and 2013 as reported to the Center for International Blood and Marrow Transplant Research (N = 13,097) were randomly divided into training and validation sets. Independent prognostic factors for development of VOD by day +100 after HCT were identified with a multivariate logistic regression model.

Changes in Medical Services and Drug Utilization and Associated Costs After Narcolepsy Diagnosis in the United States.

Background: Healthcare utilization and the cost implications associated with undiagnosed and/or misdiagnosed narcolepsy have not been evaluated, and there is scant literature characterizing the newly diagnosed population with narcolepsy with respect to treatment patterns and resource utilization.

Objective: To analyze the changes in medication use, healthcare utilization, and the associated costs after a new diagnosis of narcolepsy.

Methods: In this retrospective cohort study, we used data from the Truven Health Analytics MarketScan Research Databases, between January 2006 and March 2013, to identify patients who had a probable new diagnosis of narcolepsy-defined as a de novo medical claim for a multiple sleep latency test-which was preceded by ≥6 months of continuous insurance and was followed by a de novo diagnosis of narcolepsy.

The Cost of Hematopoietic Stem-Cell Transplantation in the United States.

Background: Hematopoietic stem-cell transplantation (HSCT) requires highly specialized, resource-intensive care. Myeloablative conditioning regimens used before HSCT generally require inpatient stays and are more intensive than other preparative regimens, and may therefore be more costly.

Objective: To estimate the costs associated with inpatient HSCT according to the type of the conditioning regimen used and other potential contributors to the overall cost of the procedure.

Burden of illness associated with sinusoidal obstruction syndrome/veno-occlusive disease in patients with hematopoietic stem cell transplantation.

Aims: Sinusoidal obstruction syndrome (SOS) is a life-threatening complication of hematopoietic stem cell transplantation (HSCT) associated with significant morbidity and mortality. Healthcare utilization, costs, and mortality were assessed in HSCT patients diagnosed with SOS, with and without multi-organ dysfunction (MOD).

Materials And Methods: This retrospective observational study identified real-world patients undergoing HSCT between January 1, 2009 and May 31, 2014 using the Premier Healthcare Database.

The budget impact and cost-effectiveness of defibrotide for treatment of veno-occlusive disease with multi-organ dysfunction in patients post-hematopoietic stem cell transplant.

Background: A Phase-3 study of defibrotide compared with historical controls demonstrated a 23% improvement in 100-day survival post-hematopoietic stem cell transplantation (HSCT) among patients with veno-occlusive disease with multi-organ dysfunction (VOD with MOD).

Aim: To estimate the budget impact and cost-effectiveness of introducing defibrotide to a transplant center.

Methods: The authors developed a budget impact model from the perspective of a bone-marrow transplant center.

Risk-Benefit Analysis of Pediatric-Inspired Versus Hyperfractionated Cyclophosphamide, Vincristine, Doxorubicin, and Dexamethasone Protocols for Acute Lymphoblastic Leukemia in Adolescents and Young Adults.

Purpose: To estimate the risk-benefit trade-off of a pediatric-inspired regimen versus hyperfractionated cyclophosphamide, vincristine, doxorubicin, and dexamethasone (hyper-CVAD) for first-line treatment of adolescents/young adult (AYA; ages 16-39 years) patients with Philadelphia-negative acute lymphoblastic leukemia.

Methods: Patient outcomes were simulated using a 6-state Markov model, including complete response (CR), no CR, first relapse, second CR, second relapse, and death. A Weibull distribution was fit to the progression-free survival curve of hyper-CVAD-treated AYA patients from a single-center study, and comparable patient data from a retrospective study of pediatric regimen-treated AYA patients were utilized to estimate a relative progression difference (hazard ratio = 0.

Evaluation of Quality of Life in Patients With Narcolepsy Treated With Sodium Oxybate: Use of the 36-Item Short-Form Health Survey in a Clinical Trial.

Introduction: The present post hoc analysis was designed to evaluate health-related quality of life (HRQoL) using the 36-item Short Form Health Status Survey (SF-36) during an 8-week trial of sodium oxybate (SXB).

Methods: SF-36 was assessed in a phase 3 placebo-controlled trial in patients with narcolepsy (N = 228) randomized to placebo or SXB in doses of 4.5, 6, or 9 g nightly for 8 weeks.

The Humanistic and Economic Burden of Narcolepsy.

Study Objectives: To evaluate the burden of narcolepsy--with respect to psychiatric comorbidities, Health-Related Quality of Life (HRQoL), direct costs for healthcare resource utilization, and indirect costs for reported work loss-through comparison of patients to matched controls.

Methods: This analysis was conducted on data from the 2011, 2012, and 2013 US National Health and Wellness Survey (NHWS; 2011 NHWS n = 75,000, 2012 NHWS n = 71,157, and 2013 NHWS n = 75,000). Patients who reported a narcolepsy diagnosis (n = 437) were matched 1:2 with controls (n = 874) on age, sex, race/ethnicity, marital status, education, household income, body mass index, smoking status, alcohol use, exercise, and physical comorbidity.

The humanistic and economic burden of providing care for a patient with schizophrenia.

Background: Schizophrenia is a debilitating disorder that often requires the affected individual to receive part- or full-time care from a caregiver.

Aims: The purpose of this study was to examine the humanistic and economic burden of caring for an individual with Schizophrenia with regard to the measures of quality of life (QoL), work productivity, healthcare resource use and estimated economic costs.

Methods: Data for this study came from the 2012 US National Health and Wellness Survey (NHWS; n = 71,149).

Negative symptoms and functioning during the first year after a recent onset of schizophrenia and 8 years later.

Background: Understanding the longitudinal course of negative symptoms, especially in relationship to functioning, in the early phase of schizophrenia is crucial to developing intervention approaches. The course of negative symptoms and daily functioning was examined over a 1-year period following a recent onset of schizophrenia and at an 8-year follow-up point.

Methods: The study included 149 recent-onset schizophrenia patients who had a mean age of 23.

Antipsychotic patterns of use in patients with schizophrenia: polypharmacy versus monotherapy.

Background: The objective of this study was to characterize real-world treatment patterns in the prescription of antipsychotic polypharmacy (≥ 2 concurrent antipsychotics) compared with antipsychotic monotherapy for patients with schizophrenia.

Methods: This study was a retrospective claims-based analysis of patients (aged 13-64 years) with schizophrenia belonging to an employer-based health plan. Duration of therapy was measured as the number of treatment days over one year following the initial date of antipsychotic therapy.