Publications by authors named "Malgorzata Ciepielewska"
To estimate time-to-progression milestones in people with amyotrophic lateral sclerosis (PALS) treated versus not treated with intravenous (IV) edaravone (Radicava IV, Mitsubishi Tanabe Pharma America [MTPA], hereafter "IV edaravone") in a real-world setting. IV edaravone is US FDA approved for the treatment of ALS and was shown in clinical trials to slow the rate of physical functional decline. This retrospective observational analysis included PALS continuously enrolled in Optum's Clinformatics Data Mart between 8 August 2017 and 31 December 2021.
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- Erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) are rare genetic disorders that lack comprehensive management data, prompting a study of their characteristics and treatment in real-world U.S. settings.
- The study reviewed medical records of 299 EPP and 91 XLP patients, revealing a mean diagnosis delay of 2.9 years and highlighting common pre-diagnostic tests and lifestyle recommendations.
- Findings indicated a significant number of healthcare visits post-diagnosis and identified unmet needs, such as the need for quicker diagnoses, effective symptom relief, and better prevention of phototoxic reactions.
Background: We aimed to evaluate overall survival in US patients with amyotrophic lateral sclerosis (ALS) treated with intravenous (IV) edaravone compared with those not treated with IV edaravone in a real-world setting.
Methods: This exploratory retrospective comparative effectiveness observational analysis included patients with ALS who were enrolled in an administrative claims database from 8 August 2017 to 31 March 2020. Propensity score matching identified IV edaravone-treated patients (cases) and non-edaravone-treated patients (controls) matched for covariates: age, race, geographic region, sex, pre-index disease duration, insurance, history of cardiovascular disease, riluzole prescription, gastrostomy tube placement, artificial nutrition, noninvasive ventilation, and all-cause hospitalisation.
Background: Drug formulation and route of administration can have an impact on not only patients' quality of life and disease outcomes but also costs of care. It is essential for decision makers to use appropriate economic modeling methods to guide drug coverage policies and to support patients' decision-making.
Purpose: To illustrate key cost considerations for decision makers in economic evaluation of innovative oral formulations as alternatives to intravenous medication.