JAMA dermatology最新文献

Importance: Although treatment for chronic urticaria (CU) has improved over the past decades, evidence regarding costs and net benefits associated with these treatment strategies have yet to be comprehensively characterized and synthesized.

Objective: To summarize the cost and cost-effectiveness of CU management strategies.

Evidence review: An extensive systematic literature search of 6 databases (MEDLINE, Embase, PubMed Cochrane, Scopus, and CINAHL) and gray literature sources, without language restriction, was conducted and updated to March 23, 2024. Articles that performed cost analysis or full economic evaluation among patients with CU were included. Two reviewers independently extracted data, such as annual costs of health care services or incremental cost-effectiveness ratio (ICER) per quality-adjusted life-year (QALY). All monetary values were converted and inflated to 2023 US dollars. Evidence-based synthesis for health benefit was judged using the Evidence Rating Matrix by the Institute for Clinical and Economic Review.

Findings: Seventeen unique studies (11 cost analysis studies and 6 full economic evaluations) were included. With the wide variation in health care resources, services that included biologic omalizumab utilization had higher annual health care cost estimations for CU management than services that did not include omalizumab prescription (median [IQR] cost, $6933 [$5988-$8717] vs $5621 [$2488-$8754]). The biologic omalizumab, 300 mg, for H1 antihistamine-refractory chronic spontaneous urticaria (CSU) (3 studies) was found to have a median (IQR) ICER of $89 005 ($36 058-$145 694) per QALY (evidence rating as incremental or better; moderate certainty with substantial net health benefit). Routine laboratory testing among patients with CSU with otherwise normal histories and physical examination findings (1 study) had ICERs ranging from $1 427 928 to $1 950 524 per QALY (evidence rating as comparable or inferior; moderate certainty that the net health benefit is inferior).

Conclusions and relevance: With limited evidence of cost-effectiveness, biologic omalizumab, 300 mg, for H1 antihistamine-refractory CSU was found to be cost-effective in US health care services at the willingness to pay threshold of $150 000 per QALY. Meanwhile, routine laboratory testing among patients with CSU without compelling indication was not cost-effective. Future studies in more diverse CU populations and resource settings are needed to fill evidence gaps.

Importance: There is a burgeoning interest in therapeutic development for cutaneous neurofibromas (cNFs), a major cause of morbidity in persons with neurofibromatosis type 1 (NF1). To determine meaningful clinical trial outcomes, deeper understanding is needed regarding how cNFs are associated with quality of life (QoL). However, this understanding has been hampered by challenges in recruiting participants with this rare genetic disease.

Objective: To develop a large, crowdsourced validated registry of persons with NF1 and determine the association of specific cNF features with QoL, pain, and itch.

Design, setting, and participants: From May 2021 to December 2023, a decentralized platform was developed and recruited persons 40 years or older with NF1 and at least 1 cNF from 49 states and 12 countries, who provided clinical survey data, detailed photographs, and genetic sequencing data. Photographs from 583 participants were scored on 12 features of cNFs, including general severity, number, size, facial severity, color, and subtypes.

Exposure: cNF features derived from participant-supplied photographs.

Main outcomes and measures: Total Skindex scores and subdomain scores (symptoms, emotion, function, pain, and itch).

Results: Of 583 participants, 384 (65.9%) were female, and the mean (range) age was 51.7 (40.0-83.0) years. Female sex, general severity, number, size, and facial severity of cNFs were negatively associated with QoL, as demonstrated by increased total Skindex scores. QoL had the largest association with the number of cNFs and presence of facial cNFs. Increasing number of cNFs was associated with worse QoL, and even individuals with a low cNF burden (<10 total cNFs) experienced a decrease in QoL.

Conclusions and relevance: The results of this study suggest that reducing cNF number, particularly on the face, may be associated with improved QoL in individuals with NF1. In addition, early intervention before the development of numerous tumors may lead to the highest benefit in QoL. These data potentially provide insight into which individuals and cNF tumors may benefit most from therapy and highlights the utility of a completely decentralized, photograph-validated and age-controlled study for rare genetic disease. This cohort will allow analysis of disease and tumor heterogeneity after full phenotypic expression is achieved in NF1 and potentially serves as an example in its design for other rare diseases that struggle from poor recruitment.

Importance: Although tobacco smoking is established as a risk factor for hidradenitis suppurativa (HS), studies on the effects of smoking cessation on HS are limited, and evidence is lacking.

Objective: To examine the association between changes in smoking status and the development of HS.

Design, setting, and participants: This population-based cohort study enrolled participants from the Korean National Health Insurance Service database who had undergone 2 consecutive biennial health examinations (2004-2005 and 2006-2007) as the primary cohort. Within the primary cohort, the secondary cohort comprised individuals who underwent all biennial health examinations throughout the follow-up period and maintained the same smoking status from 2006 to 2007 to the end of the follow-up period. Data were analyzed from July to December 2023.

Exposures: Changes in smoking habit status.

Main outcomes and measures: Risk of HS development. The HS risk according to change in smoking status between the 2 consecutive health examinations was estimated using a Cox proportional hazards model.

Results: Of the 6 230 189 participants enrolled, the mean (SD) age was 47.2 (13.5) years, and 55.6% were male. During 84 457 025 person-years of follow-up, 3761 HS events occurred. In the primary cohort, compared to those who consistently reported active smoking at both checkups (ie, sustained smokers), lower HS risk was seen among those who were confirmed to smoke initially but quit by the second checkup (ie, smoking quitters) (adjusted hazard ratio [AHR], 0.68; 95% CI, 0.56-0.83), those who maintained cessation status throughout (AHR, 0.67; 95% CI, 0.57-0.77), and those who reported never smoking at either checkup (ie, never smokers) (AHR, 0.57; 95% CI, 0.52-0.63). Those who initially quit smoking but resumed by the second checkup and those who had no previous smoking history but started at the second checkup (ie, new smokers) exhibited similar HS risk as sustained smokers. The secondary cohort results aligned with those of the primary cohort, showing a more pronounced risk reduction with smoking cessation (AHR, 0.57; 95% CI, 0.39-0.83). Considering time-smoking interaction, the cumulative incidence and the risk of HS in smoking quitters were similar to those in sustained smokers in the early stages of observation. However, 3 to 4 years after smoking cessation, the rate decelerated, resembling that of never smokers, and there was a statistically significant decrease in the risk that persisted (between 3 and 6 years from the index date: AHR, 0.58; 95% CI, 0.36-0.92; and ≥12 years from the index date: AHR, 0.70; 95% CI, 0.50-0.97). New smokers initially paralleled never smokers but accelerated after 2 to 3 years, reaching sustained smokers' levels.

Conclusions and relevance: In this cohort study, quitting smoking and sustaini