Summary
Background
Epidermolysis bullosa (EB) is a group of rare and currently incurable genetic blistering disorders. As more pathogenic‐driven therapies are being developed, there is an important ...need for EB‐specific validated outcomes measures designed for use in clinical trials.
Objectives
To test the reliability and construct validity of an instrument for scoring clinical outcomes of research for EB (iscorEB), a new combined clinician‐ and patient‐reported outcomes tool.
Methods
We conducted an observational study consisting of independent 1‐day assessments (six assessors) at two academic hospitals. The assessments consisted of iscorEB clinician (iscorEB‐c), Birmingham Epidermolysis Bullosa Severity (BEBS) and global severity assessment for physicians; and iscorEB patient (iscorEB‐p), Quality of Life evaluation in Epidermolysis Bullosa and Children's Dermatology Life Quality Index for patients. Construct validity and intraclass correlation coefficients (ICCs) for interobserver, intraobserver and test–retest reliability were calculated.
Results
Overall, 31 patients with a mean age of 19·5 years (1·8–45·2) were included. Disease severity was mild in 42% of cases, moderate in 29% and severe in 29%. The interobserver ICC was 0·96 for both the clinician‐reported section of iscorEB‐c and BEBS. The ICC for intraobserver reliability was 0·91 and 0·70 for the skin and mucosal domains of iscorEB‐c, respectively. Cronbach's alpha for iscorEB‐c was 0·89. The test–retest reliability of iscorEB‐p was 0·97 and Cronbach's alpha was 0·84. The clinical score differentiated between subjects with mild, moderate and severe disease, and both clinical and patient subscores discriminated between recessive dystrophic EB and other EB subtypes.
Conclusions
iscorEB has robust reliability and construct validity, including strong ability to distinguish EB types and severities. Further studies are planned to test its responsiveness to change.
What's already known about this topic?
Outcome measures that test epidermolysis bullosa (EB) severity are limited.
There is a need for valid tools that assess EB severity in the context of clinical trials.
What does this study add
?
The instrument for scoring clinical outcomes of research for EB (iscorEB) is a new combined clinician‐ and patient‐reported outcomes tool.
The iscorEB has robust reliability and construct validity, including a strong ability to distinguish EB types and severities.
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Tacrolimus ointment is a nonsteroidal topical immunomodulator that was formulated specifically for the treatment of atopic dermatitis. A total of 255 children, 2 to 15 years of age, with moderate to ...severe atopic dermatitis applied 0.1% tacrolimus ointment twice daily for up to 12 months to assess long-term safety and efficacy. Patients on average were treated with tacrolimus ointment for 279 days or 87% of study days. Substantial improvements in the signs and symptoms of atopic dermatitis, percent body surface area affected, and the patient's or parent's assessment of pruritus were observed during the first week of treatment and were maintained throughout the study. Transient skin burning and itching were the most common drug application site adverse events. Occurrence of these symptoms decreased after the first few days of treatment. There was no increased incidence of infections or other significant adverse events. Effectiveness of tacrolimus was maintained with prolonged daily use. Tacrolimus ointment (0.1%) is safe and effective for long-term treatment of atopic dermatitis in children. (J Am Acad Dermatol 2001;44:S58-64.)
Background: An excess of androgen is believed to contribute to development of acne in some patients. Because oral contraceptives (OCs) may reduce the active androgen level, hormonal therapy with OCs ...has been used successfully to treat patients with acne, although this treatment has previously not been studied in placebo-controlled trials. Objective: Our purpose was to evaluate the efficacy of a triphasic, combination OC (ORTHO TRI-CYCLEN Ortho-McNeil Pharmaceutical, Raritan, N.J., norgestimate/ethinyl estradiol) compared with placebo in the treatment of moderate acne vulgaris. Methods: Two hundred fifty-seven healthy female subjects, 15 to 49 years of age with moderate acne vulgaris, were enrolled in a multicenter, randomized, double-blind, placebo-controlled clinical trial. Each month for 6 months, subjects received either 3 consecutive weeks of the OC (i.e., tablets containing a fixed dose of ethinyl estradiol 0.035 mg and increasing doses of norgestimate 0.180 mg, 0.215 mg, 0.250 mg) followed by 7 days of inactive drug or placebo (color-matched tablets). Efficacy was assessed by facial acne lesion counts, an investigator’s global assessment, a subject’s self-assessment, and an analysis of within-cycle variation (cycle 6) in lesion counts. Results: Of the 160 subjects in whom efficacy could be evaluated, the OC group showed a statistically significantly greater improvement than the placebo group for all primary efficacy measures. The mean decrease in inflammatory lesion count from baseline to cycle 6 was 11.8 (62.0%) versus 7.6 (38.6%) ( p = 0.0001), and the mean decrease in total lesion count was 29.1 (53.1%) versus 14.1 (26.8%) ( p = 0.0001) in the OC and placebo groups, respectively. In the investigator’s global assessment, 93.7% of the active treatment group versus 65.4% of the placebo group were rated as improved at the end of the study ( p < 0.001). Six of the seven secondary efficacy measures (total comedones, open comedones, closed comedones, papules, pustules, and the subject’s self-assessment of study treatment) were also significantly more favorable in the OC group compared with the placebo group. Conclusion: An OC containing 0.035 mg of ethinyl estradiol combined with the triphasic regimen of norgestimate is a safe and effective treatment of moderate acne vulgaris in women with no known contraindication to OC therapy. (J Am Acad Dermatol 1997;37:746-54.)
Cutaneous Crohn's disease in children Ploysangam, Tanusin; Heubi, James E.; Eisen, Drore ...
Journal of the American Academy of Dermatology,
05/1997, Letnik:
36, Številka:
5
Journal Article
Recenzirano
Although cutaneous Crohn's disease is well recognized in adults, in children it is extremely rare.
Our purpose was to describe five children with cutaneous Crohn's disease and to review the ...literature.
The medical records of five children with cutaneous Crohn's disease were retrospectively reviewed for clinical features and laboratory data. An extensive review of the literature was conducted.
Five children, one boy and four girls, 6 to 12 years of age at onset, had cutaneous manifestations of Crohn's disease. Three had genital swelling, and the other 2 had buttock abscesses. Most were seen before the diagnosis of gastrointestinal Crohn's disease was made. There have been 80 cases of cutaneous Crohn's disease described, including our series. Only 14 were in children. Two thirds of children with cutaneous Crohn's disease had genital involvement compared with about half of the adult cases. Sixteen of the 80 patients had cutaneous lesions without preceding gastrointestinal Crohn's disease. Of these, approximately 70% had genital lesions.
Although Crohn's disease is common in children, cutaneous manifestations are rarely a presenting sign. However, when cutaneous Crohn's disease is present in children, it commonly precedes the gastrointestinal disease.
Objectives: The objectives of this study were to determine which factors in early pubertal girls might be predictive of later, severe facial acne.
Study design: The study was a 5-year longitudinal ...cohort study, with yearly visits from 1987 through 1991, in a volunteer sample of 439 black and 432 white fourth- and fifth-grade girls with consent from their legal guardians. The subjects were recruited from public and parochial schools in Cincinnati, Ohio. The degree of facial acne was classified annually as mild, moderate, or severe. Blood samples were obtained at the first, third, and fifth years of the study. Using the acne status during the fifth year of the study as the outcome variable, we determined the contributions from the prior acne status and the serum levels of dehydroepiandrosterone sulfate (DHEAS), testosterone, free testosterone (FT), estradiol (E
2 ), progesterone, and testosterone-estrogen binding globulin (TEBG) and compared the results at various ages and at times before and after menarche.
Results: No racial differences in acne or hormone levels were found. There was a progressive increase in the number of acne lesions with age and maturation. The girls exhibited many more comedonal than inflammatory acne lesions, regardless of age. The girls in whom severe acne developed by the fifth year of the study had significantly more comedones and inflammatory lesions than girls with mild or moderate acne, as early as age 10 years, approximately 2
½ years before menarche, a time when their degree of acne was mild. Girls with mild comedonal acne had significantly later onset of menarche (12.5 compared with 12.2 years) than girls with severe comedonal acne. Girls in whom severe comedonal acne developed had significantly higher levels of serum DHEAS and, in a longitudinal analysis, somewhat higher levels of testosterone and FT in comparison with girls who had mild or moderate comedonal acne. Serum E
2 , testosterone/E
2 , progesterone, and TEBG values were no different in girls with severe compared with mild or moderate comedonal acne.
Conclusions: The early development of comedonal acne may be one of the best predictors of later, more severe disease. The adrenal hormone DHEAS appears to play an important role in the initiation of acne. DHEAS, testosterone, and FT are associated with the perpetuation of severe comedonal acne. Early recognition of young girls at risk of having severe comedonal acne may enable the clinician to intervene and thus prevent unwanted sequelae.
J Pediatr 1997;130:30-9
Guidelines of care for acne vulgaris management Strauss, John S., MD; Krowchuk, Daniel P., MD; Leyden, James J., MD ...
Journal of the American Academy of Dermatology,
04/2007, Letnik:
56, Številka:
4
Journal Article
Recenzirano
Disclaimer Adherence to these guidelines will not ensure successful treatment in every situation. Furthermore, these guidelines should not be deemed inclusive of all proper methods of care or ...exclusive of other methods of care reasonably directed to obtaining the same results. The ultimate judgment regarding the propriety of any specific therapy must be made by the physician and the patient in light of all the circumstances presented by the individual patient.
Although under-five (U5) mortality in Uganda has dropped over the past two decades, rates in urban slum neighborhoods remain high. As part of a broader verbal and social autopsy study of U5 deaths, ...this study explored the perspectives of volunteer community health workers, called Village Health Teams (VHTs), on why children under five in Kampala's informal settlements are still dying despite living in close proximity to nearby health facilities.
This exploratory, qualitative study took place between January and March 2020 in the Rubaga division of Kampala, Uganda. VHTs from the slums of Kawaala and Nankulabye parishes, both located near a large government health center, were interviewed by a trained local interviewer to determine their perceptions of barriers to care-seeking and attribution for U5 childhood deaths. All interviews were audiotaped, transcribed into English, imported into NVivo V 12.0 and thematically analyzed using the Attride-Stirling framework.
20 VHTs were interviewed, yielding two global themes, the first focusing on VHTs perceptions of their role in the community to promote positive health outcomes, and the second focusing on VHTs' perceptions of how prompt care-seeking is disincentivized. Within the latter theme, three inter-related sub-themes emerged: disincentives for care-seeking at the health system level, which can drive negative beliefs held by families about the health system, and in turn, drive incentives for alternative health behaviors, which manifest as "incentivized delays" to care-seeking.
This study illustrates VHT perspectives on the complex interactions between health system disincentives and the attitudes and behaviors of families with a sick child, as well as the reinforcing nature of these factors. Findings suggest a need for multi-pronged approaches that sensitize community members, engage community and health system leadership, and hold providers accountable for providing high-quality care. VHTs have enormous potential to foster improvement if given adequate resources, training, and support.
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