Into the Light: Afamelanotide and the Treatment of Erythropoietic Protoporphyria in the United States.

BACKGROUND: Erythropoietic protoporphyria (EPP) is a rare disease that causes disabling cutaneous photosensitivity with pain and burning sensations. In 2019, afamelanotide, an α-melanocyte-stimulating hormone analogue, was approved in the United States for treatment of EPP. In this study, patients receiving afamelanotide filled out questionnaires assessing the benefit of treatment. Outcomes measured included: return to normal activities, experience of phototoxic reactions, effect on patient confidence, and more. Patients ranked their experience on a descriptive scale ranging from "very much" to "never". RESULTS: Prior to treatment, 75% of patients indicated that EPP affected their lives "very much" or "a lot". This number fell to 11% after the 1st implant and to 0% after each subsequent implant. The number of patients that willingly ventured outside increased with each subsequent implant. CONCLUSION: The results of this study clearly show that afamelanotide treatment can dramatically and positively impact the lives of EPP patients.  Citation: Resnik SR, Targett D, Resnik BI. Into the light: afamelanotide and the treatment of erythropoietic protoporphyria in the United States. J Drugs Dermatol. 2023;22(9):941-949. doi:10.36849/JDD.7126R1.

Outcome Measures for the Evaluation of Treatment Response in Hidradenitis Suppurativa for Clinical Practice: A HiSTORIC Consensus Statement.

Importance: Although several clinician- and patient-reported outcome measures have been developed for trials in hidradenitis suppurativa (HS), there is currently no consensus on which measures are best suited for use in clinical practice. Identifying validated and feasible measures applicable to the practice setting has the potential to optimize treatment strategies and generate generalizable evidence that may inform treatment guidelines. Objective: To establish consensus on a core set of clinician- and patient-reported outcome measures recommended for use in clinical practice and to establish the appropriate interval within which these measures should be applied. Evidence Review: Clinician- and patient-reported HS measures and studies describing their psychometric properties were identified through literature reviews. Identified measures comprised an item reduction survey and subsequent electronic Delphi (e-Delphi) consensus rounds. In each consensus round, a summary of outcome measure components and scoring methods was provided to participants. Experts were provided with feasibility characteristics of clinician measures to aid selection. Consensus was achieved if at least 67% of respondents agreed with use of a measure in clinical practice. Findings: Among HS experts, response rates for item reduction, e-Delphi round 1, and e-Delphi round 2 surveys were 76.4% (42 of 55), 90.5% (38 of 42), and 92.9% (39 of 42), respectively; among patient research partners (PRPs), response rates were 70.8% (17 of 24), 100% (17 of 17), and 82.4% (14 of 17), respectively. The majority of experts across rounds were practicing dermatologists with 18 to 19 years of clinical experience. In the final e-Delphi round, most PRPs were female (12 [85.7%] vs 2 males [11.8%]) and aged 30 to 49 years. In the final e-Delphi round, HS experts and PRPs agreed with the use of the HS Investigator Global Assessment (28 [71.8%]) and HS Quality of Life score (13 [92.9%]), respectively. The most expert-preferred assessment interval in which to apply these measures was 3 months (27 [69.2%]). Conclusions and Relevance: An international group of HS experts and PRPs achieved consensus on a core set of HS measures suitable for use in clinical practice. Consistent use of these measures may lead to more accurate assessments of HS disease activity and life outcomes, facilitating shared treatment decision-making in the practice setting.

A case of Stevens-Johnson syndrome treated with oral cyclosporine.

Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN) can occur at any age and are commonly caused by adverse drug events. Rapid diagnosis of SJS/TEN is imperative, followed by immediate cessation of offending agent and induction of appropriate treatment. Cyclosporine, a calcineurin inhibitor, has been reported to have a promising therapeutic effect in SJS/TEN patients with few side effects. Diagnosis of SJS/TEN in children is especially challenging as many of the symptoms mimic that of an upper respiratory infection, or other viral entities such as cocksackie A, roseola, or herpes simplex virus. We recommend initiating cyclosporine modified treatment, especially in children, upon any suspicion of SJS/TEN in a patient in order to halt the disease progression as early as possible.

An unusual case of pyogenic granuloma-like Kaposi sarcoma.

Kaposi sarcoma (KS) is not typically included in the differential diagnosis of lesions with clinical characteristics of pyogenic granuloma. However, cases of pyogenic granuloma-like Kaposi sarcoma have been reported in the literature. This variant is extremely rare and possesses clinical and histological findings consistent with both conditions. We report an elderly, immunocompetent man with pyogenic granuloma-like Kaposi sarcoma, which was clinically consistent with a pyogenic granuloma and possessed histological findings consistent with Kaposi sarcoma and pyogenic granuloma.

Ethical coding.

It is ethical, legal, and proper for a dermatologist to maximize income through proper coding of patient encounters and procedures. The overzealous physician can misinterpret reimbursement requirements or receive bad advice from other physicians and cross the line from aggressive coding to coding fraud. Several of the more common problem areas are discussed.