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To assess the psychometric properties of the disease-specific Hypoparathyroidism Symptom Diary (HypoPT-SD) patient-reported outcome (PRO) tool, which consists of a 7-item symptom subscale, a 4-item impact subscale, a single item for anxiety, and a single item for sadness or depression, using data from the BALANCE randomized, placebo-controlled Phase 3b/4 study (NCT03324880). Eligible patients had symptomatic hypoparathyroidism (HypoPT) at baseline and were aged 18-85 years (inclusive). Patients received recombinant human parathyroid hormone (1-84) or placebo. The HypoPT-SD was filled in daily; data recorded at baseline and Weeks 4, 12, and 26 (end of treatment [EOT]) were included in this analysis. Inter-item and item-total correlations were used to assess HypoPT-SD structure; Cronbach's coefficient α was used to analyze the internal consistency and reliability, and intraclass correlations were used to measure test-retest reliability. Construct validity was determined using correlational analyses between HypoPT-SD scores and scores from other conceptually similar PRO tools. Ability to detect change was assessed and thresholds for meaningful within-patient change were established. The psychometric analysis population (N=93) was predominantly female (88.2%) and white (96.8%), with a mean age of 48.5 years. Inter-item correlations ranged from 0.35 to 0.85 at baseline and from 0.49 to 0.93 at EOT. Item-total correlations ranged from 0.57 to 0.83 at baseline and from 0.69 to 0.88 at EOT. Cronbach's α values at baseline were 0.90 (symptom subscale) and 0.88 (impact subscale). Intraclass correlation coefficients for both subscales in stable patients exceeded 0.70. Significant cross-sectional correlations were observed with most of the conceptually linked PRO tools analyzed, and HypoPT-SD scores were responsive to change. Potential changes of 1.5 (symptom subscale) and 0.8 (impact subscale) were determined as meaningful change thresholds for within-patient improvements. The HypoPT-SD is a reliable measure of key symptoms and impacts of HypoPT.

Background Patient-reported outcome measures that facilitate self-report by children are needed to reduce the bias of proxy report. We previously developed an electronic Pediatric Asthma Symptom Diary (ePASD) to assess the severity of daily asthma symptoms and proximal impacts in children aged 6–11 years with mild to severe asthma. The ePASD, administered via a digital application with visuals, sounds, and text, is uniquely designed to minimize the importance of reading skills on children’s ability to self-report accurately. Here, we describe the ePASD’s psychometric properties. Methods Ninety-one children aged 6–11 years with mild to severe asthma and their caregivers participated in 2 study visits, which consisted of training on the provisioned device and completing asthma-specific clinical outcome assessment (COA) questionnaires. The children self-completed the ePASD at home twice daily for 8 consecutive days. The scoring of the ePASD was guided by factor analyses, inter-item correlations, and internal consistencies. Reliability, discriminating ability, construct validity, and responsiveness were evaluated for ePASD items and candidate scores. Results All COAs included in the study—the ePASD, Asthma Control Questionnaire (ACQ), Childhood Asthma Control Test, Pediatric Asthma Quality of Life Questionnaire–Standardized (PAQLQ[S]), and global ratings—demonstrated that the children exhibited few asthma-related symptoms and impacts at all timepoints, and consequently, showed little change over time. Internal consistencies (all Cronbach’s alphas ≥ 0.52) and test-retest reliabilities (all intraclass correlation coefficients ≥ 0.60) were largely satisfactory. Patterns of convergent and divergent correlations supported the construct validity of ePASD scores. The ePASD symptom scores correlated moderately to strongly with PAQLQ(S) Symptom scores (all correlations ≥ − 0.46) and with ACQ scores (all correlations ≥ 0.42), as predicted. Evidence of the discriminating ability of ePASD items and composite scores was demonstrated by known-groups analyses. Conclusions The ePASD is a reliable and valid measure of asthma symptoms and proximal impacts in children aged 6–11 years with mild, moderate, or severe asthma. These results lay the psychometric groundwork for use of the ePASD in future clinical trials for the management of pediatric asthma. An ongoing pediatric asthma treatment trial is anticipated to provide evidence of the ePASD’s responsiveness to change.

Background The Severity of Alopecia Tool (SALT) is a clinician‐reported outcome measure of scalp hair loss in alopecia areata (AA). Objectives To characterise the magnitudes of change in SALT scores corresponding to meaningful treatment benefits from the patient's perspective. Methods Anchor‐based methods for the estimation of meaningful within‐patient change thresholds were applied to pooled data from a randomised, double‐blind trial of ritlecitinib. Anchors included a patient‐reported measure of change in AA severity, the Patient Global Impression of Change (PGI‐C) and three items comprising the Patient Satisfaction with Hair Growth (P‐Sat) questionnaire. After reviewing Pearson correlations between change‐from‐baseline SALT scores and each anchor to confirm adequate association, potential thresholds were computed as mean change‐from‐baseline SALT scores among patients who reported moderate improvement on the PGI‐C and/or moderate satisfaction on each of three P‐Sat items at week 24. Results Six hundred and fifty participants (86% adults, 14% adolescents) had mean (standard deviation) SALT scores of 90.6 (14.3) at baseline, suggesting a sample with primarily severe AA. Correlations between SALT change‐from‐baseline scores and the patient‐reported items supported their use as anchors. Estimates based on patients reporting moderate improvement in AA (n = 102) on the PGI‐C and those reporting moderate satisfaction on the P‐Sat item related to the amount of hair growth at week 24 (n = 122) were −42.2 (26.1) and −43.1 (26.8), respectively. Supportive estimates based on the remaining P‐Sat items were similar in magnitude. Conclusions Among patients with severe AA, SALT change‐from‐baseline scores of 42 or 43 represent meaningful improvements. While the achievement of low SALT scores of ≤10–≤20 have been used to characterise efficacy in clinical trials, the amount of change required to meet this endpoint far exceeds the estimates in this study. The treatment goals of individual patients must be considered when evaluating benefit in both clinical trials and clinical practice. Applying anchor‐based methods, we used pooled data from a randomised, double‐blind trial of ritlecitinib to estimate meaningful within‐patient change thresholds for the Severity of Alopecia Tool (SALT), a clinician‐reported measure of scalp hair loss in alopecia areata (AA). Based on patients reporting moderate improvement in AA on the Patient Global Impression of Change and reporting moderate satisfaction on the Patient Satisfaction with Hair Growth at Week 24, estimates of meaningful SALT change from baseline scores were 42 and 43, respectively.