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Comparative effectiveness of oral prostacyclin pathway drugs on hospitalization in patients with pulmonary hypertension in the United States: a retrospective database analysis.

ABSTRACT: Two oral medications targeting the prostacyclin pathway are available to treat pulmonary arterial hypertension in the United States: oral treprostinil and selexipag. We compared real-world hospitalization in patients receiving these medications. A retrospective administrative claims study was conducted using the Optum® Clinformatics® Data Mart database. Patients with pulmonary hypertension were identified using diagnostic codes. Cohort inclusion required age???18 years, first oral treprostinil or selexipag prescription between 1 January 2015 and 30 September 2017 (index date), and continuous enrollment in the prior ?6 months. Patients who switched index drug were excluded. Follow-up was from index date until the first of end of index drug exposure, end of continuous enrollment, death, or 31 December 2017. Multivariable Cox proportional hazard and Poisson regression were used to compare risk and rate, respectively, of hospitalization associated with oral treprostinil vs. selexipag, adjusting for potential confounders. The study cohort included 99 patients receiving oral treprostinil and 123 receiving selexipag. Mean age was 61 years, and most patients were females (71%). Compared with oral treprostinil, selexipag was associated with a 46% lower risk of all-cause hospitalization (hazard ratio 0.54, 95% confidence interval 0.31, 0.92; P?=?0.02), a 47% lower risk of pulmonary hypertension-related hospitalization (hazard ratio 0.53, 95% confidence interval 0.31, 0.93; P?=?0.03), a 42% lower all-cause hospitalization rate (rate ratio 0.58, 95% confidence interval 0.39, 0.87; P?=?0.01), and a 46% lower pulmonary hypertension-related hospitalization rate (rate ratio 0.54, 95% confidence interval 0.35, 0.82; P?=?0.004). This study suggests that selexipag is associated with lower hospitalization risk and rate than oral treprostinil.

SUBMITTER: McConnell JW

PROVIDER: S-EPMC7675886 | biostudies-literature | 2020 Oct-Dec

REPOSITORIES: biostudies-literature

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Comparative effectiveness of oral prostacyclin pathway drugs on hospitalization in patients with pulmonary hypertension in the United States: a retrospective database analysis.

McConnell John W JW Tsang Yuen Y Pruett Janis J Iii William Drake WD

Pulmonary circulation 20201001 4

Two oral medications targeting the prostacyclin pathway are available to treat pulmonary arterial hypertension in the United States: oral treprostinil and selexipag. We compared real-world hospitalization in patients receiving these medications. A retrospective administrative claims study was conducted using the Optum® Clinformatics® Data Mart database. Patients with pulmonary hypertension were identified using diagnostic codes. Cohort inclusion required age ≥ 18 years, first oral treprostinil o ...[more]

PMID: 33240480

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Project description:IntroductionSpinal muscular atrophy (SMA) is a rare, genetic neuromuscular disorder caused by deletion/mutation of the survival motor neuron 1 gene, characterized by progressive loss of motor neurons, resulting in increasing muscular weakness, deteriorating motor function, and, in its most severe form, death before 2 years. Nusinersen, an antisense oligonucleotide that increases expression of the functional SMN protein, was approved for SMA by US and European regulatory agencies in 2016 and 2017, respectively. The indicated regimen requires intrathecal injections every 4 months, following the first four injections during the loading phase. Adherence is integral to treatment success. Adherence to nusinersen may pose particular challenges as most patients with SMA are young children who require complex multidisciplinary care (including ongoing intrathecal treatment administration and potential specialized anesthetic and surgical procedures) at specialized centers. However, real-world data on adherence to nusinersen are limited.MethodsWe conducted a retrospective claims database analysis from December 23, 2016, to November 20, 2019, to study nusinersen adherence and discontinuation/persistence in US patients with SMA types 1-3 who completed the loading phase, and to determine the impact of non-adherence or treatment discontinuation on SMA-related comorbidities, health care resource utilization (HCRU), and costs.ResultsWe identified 23 patients with SMA type 1, 41 patients with SMA type 2, and 260 patients with SMA type 3 who had completed the loading phase. Deviations from the indicated nusinersen treatment schedule were frequent in real-world usage, with most patients receiving ≥1 dose outside the scheduled interval. Across SMA types, non-adherent patients were more likely to have had SMA-related comorbidities (e.g., feeding difficulties, dyspnea and respiratory anomalies, and muscle weakness) and greater HCRU. Persistence rates 12 months after treatment initiation for patients with SMA types 1, 2, and 3 were 55.2%, 42.4%, and 54.6%, respectively. Patients who discontinued nusinersen and those who did not had generally similar comorbidity profiles. Discontinuation was associated with greater health care costs across SMA types.ConclusionOur analysis of claims data indicated that discontinuation and non-adherence to nusinersen treatment were prevalent, and associated with greater frequency of comorbidities, greater HCRU, and increased costs for patients.

Dataset Information

Comparative effectiveness of oral prostacyclin pathway drugs on hospitalization in patients with pulmonary hypertension in the United States: a retrospective database analysis.

Publications

Comparative effectiveness of oral prostacyclin pathway drugs on hospitalization in patients with pulmonary hypertension in the United States: a retrospective database analysis.

Similar Datasets

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