A Novel Tool for Measuring Quality of Life in Pediatric Localized Scleroderma (SURe)

University of Pittsburgh researchers have developed "The Localized Scleroderma Quality of Life Instrument," a patient-reported outcome (PRO) specifically designed to measure the quality of life for pediatric patients (ages 8-20) with localized scleroderma. This instrument captures four theoretical domains relevant to these patients' experiences and has undergone rigorous testing, including focus groups, cognitive testing, and multi-center psychometric evaluation. The tool offers a unique and validated method to assess quality of life in this rare disease population and is currently the only such instrument available. 

Description

The Localized Scleroderma Quality of Life Instrument (LoSQI) is a PRO tool explicitly developed for pediatric localized scleroderma patients. This instrument measures four critical domains of quality of life: Uncomfortable Skin Sensations, Body Image and Social Support, Physical Functioning and Musculoskeletal Sequelae, and Medication Side Effects. The development process followed "best practices" in patient-centered outcome creation, including formative research, item generation, survey development, and comprehensive psychometric evaluation across multiple centers. The LoSQI is now undergoing adaptation for adult use to further expand its applicability.

Applications

Patient-reported outcome tool
Clinical trials and research on pediatric localized scleroderma
Quality of life assessment in rare diseases
Adaptation for adult localized scleroderma research

Advantages

The Localized Scleroderma Quality of Life Instrument (LoSQI) is the only validated PRO tool specifically developed for pediatric localized scleroderma. It provides both qualitative and quantitative evidence for assessing quality of life in this unique patient population. Developed following best practices for patient-centered outcomes, the LoSQI ensures high relevance and applicability in clinical and research settings. The ongoing adaptation for adult use further broadens its potential impact and utility in the field of rare disease research.

Invention Readiness

The LoSQI has reached the prototype stage following a multi-phase development process, including formative research with focus groups involving children, adolescents, and parents, and cognitive testing to refine survey items. A multi-centered psychometric evaluation was conducted to establish the reliability and validity of the instrument. The current instrument has been validated through data collection across three different clinical sites, enhancing the sample size due to the rare nature of the disease. Further adaptation and validation of the instrument for adult use are underway, involving a comprehensive psychometric assessment to ensure its reliability and applicability in broader patient populations.

IP Status

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