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Background: The Localized Scleroderma Quality of Life Instrument (LoSQI) is a disease‐specific patient‐reported outcome (PRO) measure designed for children and adolescents with localized scleroderma (LS; morphea). This tool was developed using rigorous PRO methods and previously cognitively tested in a sample of paediatric patients with LS. Objective: The purpose of this study was to evaluate the psychometric properties of the LoSQI in a clinical setting. Methods: Cross‐sectional data from four specialized clinics in the US and Canada were included in the analysis. Evaluation included reliability of scores, internal structure of the survey, evidence of convergent and divergent validity, and test–retest reliability. Results: One hundred and ten patients with LS (age: 8–20 years) completed the LoSQI. Both exploratory and confirmatory factor analysis supported the use of two sub‐scores: Pain and Physical Functioning, and Body Image and Social Support. Correlations with other PRO measures were consistent with pre‐specified hypotheses. Limitations: This study did not evaluate longitudinal validity or responsiveness of scores. Conclusion: Results from a representative sample of children and adolescents with LS continue to support the validity of the LoSQI when used in a clinical setting. Future work to evaluate the responsiveness is ongoing. [ABSTRACT FROM AUTHOR]

Summary: Background: Paediatric localized scleroderma (LS) can negatively impact health‐related quality of life (HRQoL) by causing skin fibrosis, abnormal limb development, disfigurement, and side‐effects from immunosuppressive treatment. Studies to date have rarely included qualitative data gathered directly from paediatric patients with LS. Objectives: To assess the impact of LS on HRQoL among affected youth and their caregivers using qualitative description. Methods: Youth with all subtypes of LS and their caregivers were purposively sampled to participate in age‐appropriate focus groups (younger children, early adolescents, adolescents). Each group started with a drawing exercise followed by in‐depth discussion of topics including skin symptoms (e.g. itch, pain, tightness), functional impairment, physical appearance, family and peer relationships, and treatment burden. Focus groups were transcribed verbatim and co‐coded, with adjudication of differentially applied codes. The study findings were triangulated via comparison with adult reports and published literature. Results: Eleven youth aged 9–16 years and 16 caregivers participated in three focus groups each. Major identified areas of impact included uncomfortable skin symptoms, physical functioning limitations, extracutaneous manifestations, body image, bullying and teasing, unwanted questioning from others, and treatment side‐effects and burden. Conclusions: This is the first qualitative study of HRQoL in LS to include all major LS subtypes. We identified domains of HRQoL impacted by LS, some of which replicate earlier findings and some of which were novel. As impact also changed with developmental stage, our findings support the need for ongoing, formal evaluation of HRQoL in children and adolescents with LS. What is already known about this topic?Paediatric localized scleroderma (LS) negatively impacts health‐related quality of life (HRQoL) via skin fibrosis, musculoskeletal and other extracutaneous manifestations from the disease process, and side‐effects of systemic immunosuppression.The full impact of LS and its treatment on HRQoL is incompletely understood, with only one published qualitative study of youth with LS, which was limited to facial involvement.There are no qualitative studies of HRQoL in other LS subtypes to date. What does this study add?This is the first qualitative evaluation of HRQoL in youth with LS inclusive of all disease subtypes.Our study confirms that LS affects HRQoL across multiple distinct domains, including uncomfortable skin sensations, impacts on body image, bullying and teasing from peers, unwanted intrusive questioning, physical limitations, extracutaneous manifestations and high treatment burden.These results indicate the need for ongoing clinical assessment of paediatric patients in these domains. What are the clinical implications of the work?These results support the need to care for patients with LS holistically by synthesizing cutaneous, musculoskeletal and extracutaneous disease assessments with multidimensional evaluation of psychosocial impact and adverse effects of treatments.The development of an LS‐specific HRQoL measure would advance such efforts. [ABSTRACT FROM AUTHOR]

Summary: Localized scleroderma, also called morphoea, is a rare condition affecting about three in 100,000 people. It typically causes changes to the skin and underlying tissue, sometimes as deep as the muscle or bone. The authors, from the U.S.A., wanted to develop a survey that would let children and adolescents tell researchers and doctors about how localized scleroderma affects their daily life. The authors first identified a number of important ways localized scleroderma can affect children and adolescents. They then created survey questions asking about these problems. Next, the authors had children and adolescents with localized scleroderma fill out the survey to see if they could read and understand the questions and instructions. The authors also asked children if there were any questions missing from the survey. The authors found that overall the survey performed well. Children and adolescents aged 8‐18 years could read the questions and understand the survey. Children and adolescents also suggested that three questions be added to the survey to make it more complete. The final version of the survey included questions about how a child's skin feels (e.g. if it hurts or itches), limitations in their daily activities, how they feel about their body, their relationships with friends and strangers, and finally how their medications make them feel. This survey, The Localized Scleroderma Quality of Life Instrument, is the first of its kind. Doctors and researchers can use it to better understand what life is like for children and adolescents with localized scleroderma. This is a summary of the study: A novel patient‐reported outcome for paediatric localized scleroderma: a qualitative assessment of content validity Linked Article: Zigler et al. Br J Dermatol 2020; 182:625–635 [ABSTRACT FROM AUTHOR]