Publications by authors named "Iyar Mazar"
Article Synopsis
- The Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) is a 10-item tool designed to evaluate the severity of symptoms in patients with mitochondrial disease, assessing its psychometric properties based on data from a clinical trial.
- Participants in the trial, mostly women with an average age of 45, filled out the PMMSA daily for about 14 weeks while testing elamipretide in a randomized, placebo-controlled study.
- Results showed that while the PMMSA items did not form a single construct, four related to tiredness were grouped as a "general fatigue" score, which proved to be stable and correlated well with other health measures, with meaningful reductions being linked to symptom improvement.
vFor youth with life-limiting chronic illnesses, transitioning to adulthood in line with age-norms may be difficult due to symptom severity and shortened survival. This study explores whether individuals with Barth syndrome (BTHS), a condition uniquely characterized by extreme prognostic uncertainty, experience similar or different challenges compared to youth with other conditions. During focus groups with adults with BTHS ( = 12) and caregivers ( = 13), participants reported that the ability to independently manage one's health condition, the social/emotional impacts of BTHS, and the ability to set goals in the context of future uncertainty challenge their transition to adulthood.
View Article and Find Full Text PDFBackground: Advanced systemic mastocytosis (AdvSM), indolent systemic mastocytosis (ISM), and smoldering systemic mastocytosis (SSM) are rare diseases characterized by neoplastic mast cell infiltration of more than one organ. A content-valid patient-reported outcome (PRO) questionnaire that assesses relevant signs and symptoms that are important and understandable to individuals with a condition is critical for assessing new treatment benefit as well as supporting product labeling claims. Notably, no such PRO questionnaire has been developed in accordance with regulatory and scientific guidelines for use in AdvSM, ISM, and SSM patient populations.
View Article and Find Full Text PDFBackground: Barth Syndrome (BTHS) is a rare genetic disorder that presents as a complex of debilitating symptoms and reduced life expectancy. Well-developed, BTHS-specific assessments measuring primary signs and symptoms of BTHS are not currently available, making it difficult to evaluate treatment effects in BTHS clinical studies. The objective of this research was to develop symptom-focused patient-reported outcome (PRO) measures for use in clinical studies with adolescents and adults with BTHS.
View Article and Find Full Text PDFObjectives: Primary mitochondrial myopathy (PMM) is a genetic condition characterized by life-limiting symptoms such as muscle weakness, fatigue, and pain. Because these symptoms are best reported by individuals with PMM, the objective of this qualitative research study was to develop a PMM-specific patient-reported outcome (PRO) questionnaire.
Method: Individuals with PMM were interviewed, identifying the most salient symptoms of PMM and assessing the resulting questionnaire's relevance and comprehensibility.
Background: Barth syndrome (BTHS, OMIM 302060) is a rare, life-threatening, x-linked genetic disorder that occurs almost exclusively in males and is characterized by cardiomyopathy, neutropenia, skeletal muscle myopathy primarily affecting larger muscles, and shorter stature in youth. A greater number of individuals with BTHS are now surviving into adulthood due to advancements in diagnosis and disease management. Given these improvements in life expectancy, understanding the disease experience over time has become increasingly important to individuals with the condition, treatment developers, and regulatory agencies.
View Article and Find Full Text PDFBackground: Rheumatoid arthritis (RA), a chronic, progressive inflammatory, autoimmune disease, can substantially reduce health-related quality of life (HRQoL) and lead to severe disability and early mortality. Patient-reported outcome (PRO) instruments are used to assess the patient experience of RA symptoms and impacts, and can capture RA treatment effects. To address limitations in existing PRO instruments, this research aimed to establish the content validity of a new instrument, the Rheumatoid Arthritis Symptom Questionnaire (RASQ), to assess the signs and symptoms of RA.
View Article and Find Full Text PDFBackground: Despite an increased use of patient-reported outcomes (PROs) in oncology clinical trials, integrating the patient perspective into drug approval decisions and documentation has been challenging.
Objectives: To review important regulatory and measurement terminology, and to provide oncology outcomes researchers and those involved with building oncology programs with tools to plan PRO data collection, particularly in relation to drug efficacy claims for drug labeling in the United States.
Discussion: When contemplating a PRO measurement strategy for oncology clinical trials, outcomes researchers are challenged in several ways.
Objective: The emergence of various modes of administration for cancer treatment, including oral administration, brings into focus the importance of patient preference for administration. The purpose of this research was to evaluate the administration preferences of cancer patients, specifically between oral and intravenous (IV) treatment, as well as the factors contributing to preference.
Methods: A literature search was conducted in OvidSP to identify research in which the preferences of cancer patients for oral or IV treatment have been evaluated.
Introduction: Patient-reported outcome (PRO) measures serve to capture vital patient information not otherwise obtained by primary study endpoints. This paper examines how PROs are utilized as endpoints in industry-sponsored metastatic breast cancer clinical trials.
Methods: A search was conducted in the clinicaltrials.
Article Synopsis
- Patient-reported outcome (PRO) instruments measure the signs, symptoms, and effects of treatments for diseases like advanced or metastatic breast cancer, and this research looks into their use in trials of 16 approved therapies.
- A comprehensive literature search resulted in 1,727 publications, but only 25 met the specific criteria concerning the use of PROs, identifying 11 different PRO instruments, with the EORTC QLQ-C30 being the most frequently used.
- While PROs are often included in clinical trials, many articles lack detailed information on their administration and reporting, suggesting that although useful, PROs are rarely used to directly support regulatory label claims.
Article Synopsis
- The paper discusses the importance of patient-reported outcome (PRO) data in oncology clinical trials and how it can be better utilized to showcase treatment benefits from the patients' viewpoints.
- It suggests methods for pharmaceutical sponsors to enhance the value of PRO data for key stakeholders such as payers, healthcare providers (HCPs), and patient advocacy groups, ultimately aiming for more patient-centered care.
- Real-world examples illustrate how PRO data can influence reimbursement decisions, improve communication with advocacy groups, and aid HCPs in making better treatment choices, emphasizing the need for integrating this data into health care practices.
Patient-reported outcomes for US oncology labeling: review and discussion of score interpretation and analysis methods.
Expert Rev Pharmacoecon Outcomes Res
July 2016
Article Synopsis
- The paper addresses challenges in interpreting patient reported outcome (PRO) scores, especially in the context of regulatory approval for oncology treatments.
- It emphasizes that score interpretation is not standardized and varies based on study design and sample characteristics.
- Researchers have multiple methods available for analyzing PRO scores, which should align with their understanding of the patient population and the specific goals of their study.