Instrument for scoring clinical outcome of research for epidermolysis bullosa: a consensus-generated clinical research tool.

Epidermolysis bullosa (EB) is a genetic condition characterized by skin fragility and blistering. There is no instrument available for clinical outcome research measurements. Our aim was to develop a comprehensive instrument that is easy to use in the context of interventional studies. Item collection was accomplished using a two-step Delphi Internet survey process for practitioners and qualitative content analysis of patient and family interviews. Items were reduced based on frequency and importance using a 4-point Likert scale and were subject to consensus (>80% agreement) using the nominal group technique. Pilot data testing was performed in 21 consecutive patients attending an EB clinic. The final score, Instrument for Scoring Clinical Outcome of Research for Epidermolysis Bullosa (iscorEB), is a combined score that contains clinician items grouped in five domains (skin, mucosa, organ involvement, laboratory abnormalities, and complications and procedures; maximum score 114) and patient-derived items (pain, itch, functional limitations, sleep, mood, and effect on daily and leisurely activities; maximum score 120). Pilot testing revealed that combined (see below) and subscores were able to differentiate between EB subtypes and degrees of clinical severity (EB simplex 21.7 ± 16.5, junctional EB 28.0 ± 20.7, dystrophic EB 57.3 ± 24.6, p = 0.007; mild 17.3 ± 9.6, moderate 41.0 ± 19.4, and severe 64.5 ± 22.6, p < 0.001). There was high correlation between clinician and patient subscores (correlation coefficient = 0.79, p < 0.001). iscorEB seems to be a sensitive tool in differentiating between EB types and across the clinical spectrum of severity. Further validation studies are needed.

Infrared thermography to assess proliferation and involution of infantile hemangiomas: a prospective cohort study.

IMPORTANCE: Infantile hemangiomas (IHs) are common benign tumors of infancy that have the potential to interfere with vital organ function and cause permanent disfigurement. Currently, few objective and validated measures exist to assess IHs. OBJECTIVE: To determine the utility of infrared thermography in assessing and monitoring the growth of IHs. DESIGN, SETTING, AND PARTICIPANTS: In a prospective cohort study conducted at an outpatient dermatology clinic of a tertiary care hospital between February 2011 and December 2012, a convenience sample of 42 infants aged 0 to 6 months with an IH were enrolled. The mean age of the study group was 3.7 months, with the majority of IHs being mixed type (57%) affecting the head and neck (81%). Of the infants, 36 (86%) were receiving active treatment during the study period, and patients were followed for a minimum of 3 clinical visits, at least 1 month apart. MAIN OUTCOMES AND MEASURES: Ability of infrared thermography to assess the proliferation and involution of IHs compared with a visual analog scale. Secondary outcomes were reliability, ease of use, and parental acceptance of the instrument. RESULTS: The mean temperature difference at baseline was 1.9°F (95% CI, 1.2°F to 2.7°F), which peaked at 3 months to 2.5°F (95% CI, 0.8°F to 4.2°F), and decreased progressively to 0.2°F (95% CI, -1.1°F to 1.4°F) at 18.5 months (P < .001). This change in temperature was inversely correlated with mean visual analog scale (r = -0.25). Mean temperature differences recorded at baseline and 30 minutes later were not significant (least squares mean baseline temperature, 87.9°F [95% CI, 87.4°F to 88.3°F], vs least squares mean temperature after 30 minutes, 88.1°F [95% CI, 87.7°F to 88.6°F] [P = .14]). Multivariate analysis demonstrated facial location (F(1,365) = 47.63, P < .001), IH type (F(2,365) = 3.26, P = .04), age (F(2,365) = 7.03, P = .001), and surface area at baseline (F(2,365) = 8.18, P < .001) as factors significantly affecting temperature difference over time. Only IH type (Wald χ(22) = 6.79, P = .03) and treatment (Wald χ(21) = 4.29, P = .04) significantly affected time to reach a zero-temperature difference. All caregivers (100%) reported IRT to be easy to implement, quick to perform, and comfortable for their child. CONCLUSIONS AND RELEVANCE: Infrared thermography is a reliable and valid measure of IH growth that is noninvasive, convenient, and well tolerated by infants, making it well suited to daily clinical practice. It has the potential to provide real-time objective results that can be used for routine monitoring and evaluating treatment efficacy.

Propranolol in the management of infantile hemangiomas: clinical response and predictors.

BACKGROUND: Recent data suggest that propranolol is an effective treatment for infantile hemangiomas (IHs). Data on the optimal dose, duration of therapy, and predictors of response are currently lacking. OBJECTIVE: To assess the clinical response to and predictors of propranolol use in the treatment of IH. METHODS: Retrospective cohort study of 44 patients. Two independent assessors evaluated improvement by comparing serial digital photographs using a 100 mm visual analogue scale (VAS), where 5 mm change represented 10% change in the size or appearance of the IH. RESULTS: Propranolol was started at a mean age of 7.8 (SD 8.21) months and was used for 7.3 (SD 4.8) months before weaning. The mean percent improvement compared to baseline (as measured by the VAS) was 78% (SD 23%). Minor adverse events were noted in 32% of patients. The most significant predictor of regrowth after weaning was a IH > 5 cm in size (p  =  .017). CONCLUSIONS: Propranolol is effective in IH, but the side effects and the possibility of regrowth should be considered.

Timolol maleate 0.5% or 0.1% gel-forming solution for infantile hemangiomas: a retrospective, multicenter, cohort study.

Therapeutic options for superficial infantile hemangiomas (IH) are limited. Recently, timolol maleate gel, a topical nonselective beta-blocker, has been reported as a potentially effective treatment for superficial IH. This study is an extension of a previously published pilot study designed to further investigate the efficacy and safety and to identify predictors of good response of topical 0.5% or 0.1% timolol maleate gel-forming solution. This was a retrospective cohort study including patients enrolled from five centers. Patients were included if they were treated with timolol maleate 0.1% or 0.5% gel-forming solution and had photographic documentation of the IH and at least one follow-up visit. Patients with concomitant active treatment using other IH treatments were excluded. The primary endpoint was change in the appearance of IH as evaluated using a visual analog scale (VAS). Data from 73 subjects were available for final analysis. Timolol maleate gel-forming solution 0.5% was used in 85% (62/73) of patients, the remainder being treated with 0.1%. The median age at treatment initiation was 4.27 months (interquartile range [IQR] 2.63-7.21 mos), and patients were treated for a mean of 3.4 ± 2.7 months. All patients except one improved, with a mean improvement of 45 ± 29.5%. Predictors of better response were superficial type of hemangioma (p = 0.01), 0.5% timolol concentration (p = 0.01), and duration of use longer than 3 months (p = 0.04). Sleeping disturbance was noted in one patient. This study further demonstrates the efficacy and tolerability of topical timolol maleate and gradual improvement with longer treatment in patients with superficial IH.