Currently recommended skin scores correlate highly in the assessment of patients with Juvenile Dermatomyositis (JDM).

BACKGROUND: Juvenile Dermatomyositis (JDM) is a rare, chronic, and life-threatening childhood autoimmune disease. Currently, there are recommended, reliable and validated measurement tools for assessment of skin disease activity in JDM including the Disease Activity Score (skinDAS), Cutaneous Assessment Tool (CAT), and the Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI). The Physician's global assessment skin visual analog scale (Skin VAS) is also widely used for skin activity in JDM. For the purpose of comparative international studies, we wanted to compare these tools to the Physician's skin VAS (as a standard) to identify which performs better. OBJECTIVES: We sought to compare the correlations of these scoring tools, and separately assess the responsiveness each tool demonstrates following patient treatment, in order to see if one tool may be preferred. This was determined by assessing how well these tools correlate with each other, and the Physician's skin VAS over time, as well as the responsiveness of each tool after patient treatment. METHODS: Skin scores were recorded at a baseline (first visit after June 1st, 2018) and all follow-up office visits at the Juvenile Dermatomyositis Clinic. Following baseline visits, patients were followed up as clinically indicated. A subset of newly diagnosed patients (inception cohort) was identified. Correlations were assessed at the baseline visit and over time for the whole cohort. The correlations over time were derived using Generalized Estimating Equations (GEEs). Standardized response means with 95% confidence intervals were calculated to test score responsiveness for the nested inception cohort. RESULTS: The skinDAS, CAT and CDASI all correlated highly with each other and with the Physician's skin VAS. The three scoring tools accurately reflected Physician's skin VAS scores over time. In addition, all tools showed moderate to high responsiveness following treatment. CONCLUSION: All studied skin score tools performed well in our study and appear to be useful. Since no tool far outperforms the others, arbitrary consensus will be needed to select a single standard measurement tool for the purposes of efficiency and global comparability.

Assessment of Orofacial Symptoms in Juvenile Idiopathic Arthritis: Validation of a Consensus-Based Short Patient Questionnaire.

OBJECTIVE: To develop, validate, and test the performance of patient-reported outcomes (PROs) in a short patient questionnaire (< 5 minutes in length) in order to assess the multidimensional aspects of orofacial symptoms related to juvenile idiopathic arthritis (JIA) among patients aged ≥ 10 years. METHODS: The study was conducted by an interdisciplinary task force from the Temporomandibular Joint Juvenile Arthritis Working Group. The project consisted of a multiphased approach including the following: (1) conceptual phase with online international survey of 167 healthcare workers, (2) item generation and drafting of preliminary questionnaire(s) (ie, face validity), (3) cognitive script interview and probing (ie, content validity; 16 patients with JIA), (4) assessment of construct validity (ie, convergence and divergence; 53 patients with JIA), (5) test of reliability, and (6) test of clinical performance and psychometric characteristics (95 patients with JIA). RESULTS: In total, 7 PROs were included in the final patient questionnaire: (1) pain frequency, (2) pain intensity, (3) pain location, (4) jaw function, (5) specific questions related to symptoms and dysfunction, (6) changes in face and jaw pain since last visit, and (7) changes in jaw function since last visit. In total, 80% of the patients were able to complete the questionnaire in less than 5 minutes. CONCLUSION: We have developed and validated a short patient questionnaire to assess the multidimensional aspects of JIA-related orofacial symptoms. The PROs included in our questionnaire show acceptable validity and reliability. The questionnaire is applicable to routine monitoring of subjects with JIA, as well as future research studies.

Electronic forms for patient reported outcome measures (PROMs) are an effective, time-efficient, and cost-minimizing alternative to paper forms.

BACKGROUND: Patient reported outcome measures (PROMs) provide valuable insight on patients' well-being and facilitates communication between healthcare providers and their patients. The increased integration of the technology within the healthcare setting presents the opportunity to collect PROMs electronically, rather than on paper. The Childhood Health Assessment Questionnaire (CHAQ) and Quality of My Life (QoML) are common PROMs collected from pediatric rheumatology patients. The objectives of this study are to (a) determine the equivalence of the paper and electronic forms (e-form) of CHAQ and QoML questionnaires; (b) identify potential benefits and barriers associated with using an e-form to capture PROMs; and (c) gather feedback on user experience. METHODS: Participants completed both a paper and an e-form of the questionnaires in a randomized order, following which they completed a feedback survey. Agreement of the scores between the forms were statistically analyzed using the intraclass correlation coefficient (ICC) (95 % Confidence Interval (CI)) and bias was assessed using a Bland-Altman plot. Completion and processing times of the forms were compared using mean and median measures. Quantitative analysis was performed to assess user experience ratings, while comments were qualitatively analyzed to identify important themes. RESULTS: 196 patients participated in this project. Scores on the forms had high ICC agreement > 0.9. New patients took longer than returning patients to complete the forms. Overall, the e-form was completed and processed in a shorter amount of time than the paper form. 83 % of survey respondents indicated that they either preferred the e-form or had no preference. Approximately 10 % of respondents suggested improvements to improve the user interface. CONCLUSIONS: E-forms collect comparable information in an efficient manner to paper forms. Given that patients and caregivers indicated they preferred completing PROMs in this manner, we will implement their suggested changes and incorporate e-forms as standard practice for PROMs collection in our pediatric rheumatology clinic.

Periconceptional folic acid supplementation: a new indication for therapeutic drug monitoring.

The role of periconceptional folic acid supplementation in the prevention of neural tube defects (NTDs) has been well established. Maternal red blood cell (RBC) folate concentration is inversely associated with NTD risk, and concentrations above 906 nmol/L are associated with a low risk of NTDs. Current guidelines call for a minimum of 0.4 mg of folic acid per day for all women who could become pregnant and higher levels of supplementation for women with a family history of NTDs or risk factors associated with NTDs. However, there is variability in supplement adherence and lack of knowledge of conditions that may elevate folate requirements or NTD risk. Therefore, guidance provided to the population as a whole may be inappropriate for individual women. Current data show that a significant proportion of women of childbearing age have RBC folate concentrations below 906 nmol/L, rendering a higher-than-baseline risk for NTDs. Therapeutic drug monitoring (TDM) of RBC folate could be used to identify these women and to help them improve their folate status, thus reducing their risk for having a child with an NTD.This review describes the evolution of the evidence for TDM of RBC folate and preliminary experience with TDM in a population of 12 women who were planning a pregnancy and who were being treated with an atypical antipsychotic.