An analysis of the prevalence of osteoporosis and osteopenia in patients with epidermolysis bullosa: A cross-sectional study.

BACKGROUND: Epidermolysis bullosa (EB) patients have multiple risk factors for osteoporosis. There is limited literature describing the prevalence of bone health in EB, particularly in adults and less severe EB types. OBJECTIVES: To investigate the prevalence of osteopenia or osteoporosis in EB patients from the Australasian Epidermolysis Bullosa Registry (AEBR). METHODS: Of 417 AEBR patients, 72 underwent a dual energy X-ray absorptiometry scan. Bone mineral density (BMD) T and Z-scores, EB Disease Activity and Scarring Index (EBDASI), and Quality of Life in EB (QOLEB) scores were obtained. RESULTS: T-scores of RDEB patients were significantly lower than the diagnostic cut-off value for osteopenia. EBDASI and QOLEB scores were inversely correlated with Z-scores. The prevalence of osteoporosis in adults was 75% in severe EB types (RDEB and JEB). In adults with less severe types (EBS and DDEB), the prevalence of osteopenia was 50% and 33%, respectively. CONCLUSIONS: This is the largest study of osteoporosis in EB to date and the first to include adult patients with EBS. The high prevalence of osteoporosis and osteopenia identified in these patients warrants larger, collaborative international studies. Nevertheless, EB patients with high disease severity and QOL scores, irrespective of type, should receive early osteoporosis screening and prophylaxis.

Evaluation of the toxicity of glucocorticoids in patients with autoimmune blistering disease using the Glucocorticoid Toxicity Index: A cohort study.

BACKGROUND: Glucocorticoids are the mainstay of treatment for autoimmune blistering diseases (AIBDs). The Glucocorticoid Toxicity Index (GTI) is a novel, outcome-based glucocorticoid-induced adverse effects monitoring instrument. OBJECTIVE: To investigate whether the GTI score was able to accurately quantify the glucocorticoid-induced toxicity in patients with AIBDs. METHODS: The prospective cohort study included patients with confirmed diagnoses of AIBDs (group1, currently receiving glucocorticoids; and group 2, had glucocorticoids ceased earlier). Data were collected minimally at baseline (V1) and 3 months (V2). Further data from patients who were able to complete the follow-up visits at 6 months (V3) and 12 months (V4) amid the COVID-19 pandemic were also included. GTI scores were calculated after data collection. RESULTS: Analysis of data from V1 and V2 found a linear correlation between GTI score and prednisone doses (P < .05) and a significant difference in GTI scores between group1 and group 2 (P < .05). Data from V3 and V4 suggested that GTI scores continued to rise progressively alongside increasing cumulative prednisone dose. LIMITATIONS: Small sample size, further exacerbated by the COVID-19 pandemic. Single-center study. CONCLUSION: The GTI sensitively and specifically captured the changes in glucocorticoids toxicity over time among patients with AIBDs. The GTI could be a feasible tool that can be used in future clinical trials as a glucocorticoid-induced toxicity outcome measure.

A comparison study of outcome measures for epidermolysis bullosa: Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) and the Instrument for Scoring Clinical Outcomes of Research for Epidermolysis Bullosa (iscorEB).

BACKGROUND: The success of clinical trials in Epidermolysis Bullosa (EB) is dependent upon the availability of a valid and reliable scoring tool that can accurately assess and monitor disease severity. The Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) and Instrument for Scoring Clinical Outcomes of Research for Epidermolysis Bullosa (iscorEB) were independently developed and validated against the Birmingham Epidermolysis Bullosa Severity Score but have never been directly compared. OBJECTIVE: To compare the reliability, convergent validity, and discriminant validity of the EBDASI and iscorEB scoring tools. METHODS: An observational cohort study was conducted in 15 patients with EB. Each patient was evaluated using the EBDASI and iscorEB-clinician scoring tools by 6 dermatologists with expertise in EB. Quality of life was assessed using the iscorEB-patient and Quality of Life in EB measures. RESULTS: The intraclass correlation coefficients for interrater reliability were 0.942 for the EBDASI and 0.852 for the iscorEB-clinician. The intraclass correlation coefficients for intrarater reliability was 0.99 for both scores. The two tools demonstrated strong convergent validity with each other. CONCLUSION: Both scoring tools demonstrate excellent reliability. The EBDASI appears to better discriminate between EB types and disease severities.