A Nonrandomized Controlled Trial

[Posted 17/Jan/2023]

AUDIENCE: Dermatology

KEY FINDINGS: These findings suggest that apremilast was a safe and efficacious add-on treatment in recalcitrant dermatomyositis, with an overall response rate of 87.5% and associations with downregulation of multiple inflammatory pathways.

BACKGROUND: Cutaneous disease in dermatomyositis has no standardized treatment approach and so presents a challenging task for patients and clinicians. Purpose of this trial was to study the efficacy and safety of apremilast as an add-on therapy in patients with recalcitrant cutaneous dermatomyositis.

DETAILS: This phase 2a, open-label, single-arm nonrandomized controlled trial was conducted at a single center from June 2018 to June 2021. Participants were 8 patients with recalcitrant cutaneous dermatomyositis, defined by a cutaneous disease activity severity index (CDASI) score greater than 5 despite treatment with steroids, steroid-sparing agents, or both. Data were analyzed from June 2018 to June 2021. The primary outcome was the overall response rate (ORR) at 3 months. Key secondary outcomes were the safety and toxicity of apremilast and the durability of response at 6 months. The CDASI, muscle score, dermatology life quality index (DLQI), and depression assessments were performed at baseline and regularly until month 7. Skin biopsies were performed at baseline and 3 months after apremilast (defined as 3 months into active apremilast therapy) and tested for gene expression profiling and immunohistochemical stains. Adverse events were assessed using the Common Terminology Criteria for Adverse Events version 5.0. Among 8 patients with recalcitrant cutaneous dermatomyositis (all women; mean [SD] age, 54 [15.9] years), a response was found at 3 months after apremilast among 7 patients (ORR, 87.5%). The mean (SD) decrease in CDASI was 12.9 (6.3) points at 3 months (P .001). Apremilast was well tolerated, with no grade 3 or higher adverse events. Sequencing of RNA was performed on skin biopsies taken from 7 patients at baseline and at 3 months after therapy. Appropriate negative (ie, no primary antibody) and positive (ie, tonsil and spleen) controls were stained in parallel with each set of slides studied. Of 39,076 expressed genes, there were 195 whose expression changed 2-fold or more at P < .01 (123 downregulated and 72 upregulated genes). Gene set enrichment analysis identified 13 pathways in which apremilast was associated with downregulated expression, notably signal transducers and activators of transcription 1 (STAT1), STAT3, interleukin 4 (IL-4), IL-6, IL-12, IL-23, interferon γ (IFNy), and tumor necrosis factor α (TNFα) pathways. In immunohistochemical staining, there was a mean (SD) decrease in phosphorylation levels STAT1 (22.3% [28.3%] positive cells) and STAT3 (13.4% [11.6%] positive cells) at the protein level, a downstream signaling pathway for the downregulated cytokines.

Copyright © American Medical Association. All Rights Reserved.

Source: Bitar, C., Ninh, T., Brag, K., et al. (2022). Apremilast in Recalcitrant Cutaneous Dermatomyositis: A Nonrandomized Controlled Trial. JAMA Dermatol. 2022;158(12): 1357-1366. Published: December, 2022. DOI: 0.1001/jamadermatol.2022.3917.

A Case Series

[Posted 13/Jan/2026]

AUDIENCE: General Surgery, Dermmatology, Internal Medicine

KEY FINDINGS: While hypopigmentation is rare with PSL treatment, it can occur even with conservative low-fluence settings and adequate intervals between sessions. One possible mechanism is thermal beam- stacking, where slow hand movement during treatment may lead to repeated pulses on the same area. This can result in localised thermal accumulation, potentially causing subcellular disruption of melanosomes without overt melanocyte loss. These findings suggest the need for clinician vigilance in monitoring for hypopigmentation, as re-pigmentation may not be achievable. Larger, controlled studies are needed to clarify risk factors and guide safer practice.

BACKGROUND: Picosecond lasers (PSL) are increasingly used for treating melasma, with fewer adverse effects reported compared to Q-switched lasers (QSL). However, the incidence of hypopigmentation following PSL treatment remains unexplored in detail. This case series aims to explore outcomes of hypopigmentation following PSL therapy in patients with melasma, and explore potential contributing factors.

DETAILS: A retrospective chart review identified four patients with hypopigmentation following PSL treatment for melasma, including one referral from another clinic. Across the cohort, 796 patients underwent 3096 sessions between 2021 and 2025. Treatments used 755 and/or 1064-nm wavelengths at low fluences, with intervals of 4–12 weeks. Three in-clinic patients (0.38%, 95% CI 0.13%–1.10%) and one referral developed hypopigmentation. Changes occurred across both wavelengths and beam profiles. Histology demonstrated preserved melanocyte density with reduced melanin pigment and melanosome content. None of the cases showed meaningful re-pigmentation at 6-month follow-up. No cases of post-inflammatory hyperpigmentation (PIH) were observed.

Copyright © Wiley Periodicals LLC. All rights reserved

Source: Hang, X. and Lim, D. S. Hypopigmentation Following Picosecond Laser Treatment for Melasma: A Case Series. Lasers Surg. Med.. 2025; Published: December, 2025. DOI: 10.1002/lsm.70077.

[Posted 7/Jan/2026]

AUDIENCE: Dermatology, Family Medicine

KEY FINDINGS: Both N-NAIL and NAPSI provide accurate cutoff values in a psoriasis population. Therefore, these scoring tools may not only be used to assess severity but also in clinical trials for the inclusion of NP patients in a psoriasis population to create homogeneity between studies. Authors prefer using the N-NAIL, with a cutoff value of 2, because it showed better accuracy compared to the NAPSI.

BACKGROUND: It is challenging to distinguish nail psoriasis (NP) from nonspecific nail changes, contributing to heterogeneity in clinical trials. Existing scoring tools for NP are currently used to assess severity after diagnosis is established. The aim of this study is to evaluate the diagnostic performance of two of these severity scoring tools.

DETAILS: A cohort study was conducted with psoriasis patients and matched controls. Fingernails were scored using the Nail Psoriasis Severity Index (NAPSI) and the Nijmegen-Nail Psoriasis Activity Index Tool (N-NAIL). To determine their diagnostic properties, cutoff values were established. Receiver operating characteristic (ROC) curves were constructed, and sensitivity and specificity were calculated for various cutoff points. The best cutoff value was chosen based on the Youden Index and clinical reasoning. In total, 104 psoriasis patients were included, of which 68 were clinically diagnosed with NP. For the N-NAIL, a cutoff value of 2 showed the best accuracy in the psoriasis population (sensitivity = 83.8% and specificity = 83.3%) and the general population (sensitivity = 83.8% and specificity = 67.3%). For the NAPSI, a cutoff value of 7 showed the best accuracy in the psoriasis population (sensitivity = 80.9% and specificity = 69.4%), while a cutoff value of 10 was optimal in the general population (sensitivity = 72.1% and specificity = 70.2%).

Copyright © John Wiley & Sons Ltd. All rights reserved.

Source: Rikken, E. C. C., van Hal, T. W., van den Reek, J. M. P. A., et al. What Is the Diagnostic Capacity of Existing Severity Scoring Tools for Nail Psoriasis? International Journal of Dermatology. 2026; 65(1): 86-92. Published: January, 2026. DOI: 10.1111/ijd.70024.

A Prospective Interventional Study

[Posted 29/Sep/2025]

AUDIENCE: General Surgery, Internal Medicine, Hematology

KEY FINDINGS: Curettage remains optimal for thick, hyperkeratotic SKs, enabling histopathological confirmation. The 532 nm laser, preferred subjectively for convenience, may suit small, non-hyperkeratotic lesions but warrants further validation. Propane-butane cryotherapy offers a cost-effective alternative. The discordance between patient preferences (prioritizing convenience) and clinical efficacy underscores the need for personalized treatment strategies balancing outcomes, tolerability, and cosmetic expectations.

BACKGROUND: Seborrheic keratosis (SK) is the most prevalent benign skin tumor associated with aging, posing esthetic concerns and potential discomfort. The rising demand for cosmetic interventions prompts the exploration of effective removal methods. This study compares three treatment modalities: curettage, 532 nm laser, and propane-butane cryotherapy.

DETAILS: A prospective interventional clinical study was conducted with 30 subjects, treating 123 SK using curettage, 532 nm laser, and propane-butane cryotherapy. Randomized allocation and evaluations at 0, 4, 8, and 12 weeks were employed. Objective measures included clearance rates (assessed by a dermatologist) and blinded cosmetic ratings by 125 observers. Subjective outcomes encompassed patient-reported pain (VAS), side effects, satisfaction, and preferences. Curettage achieved the highest clearance rate (87.5% vs. laser: 55%, cryotherapy: 50%; p < 0.01). However, patients perceived complete healing most frequently with laser (90% vs. curettage: 87%, cryotherapy: 53%). Despite lower efficacy, 67% favored laser for future treatments, valuing minimal bleeding and no dressings. Observers rated curettage's cosmetic outcomes superior (50% vs. laser: 22.5%). Laser caused the fewest side effects (mean 0.93/lesion) but highest pain (VAS: 4.62 vs. cryotherapy: 3.85). Cryotherapy showed efficacy comparable to laser (50% vs. 55%) but more adverse events.

Copyright © Wiley Periodicals LLC. All rights reserved

Source: Timmermann, V., Krengel, S., Mrowka, P,, et al. (2024). Practical Approaches for Seborrheic Keratosis Treatment: Curettage Versus 532-nm Lithium Borate Laser Versus Cryotherapy: A Prospective Interventional Study. Lasers in Surgery and Medicine. 2025; 57(7): 581-589. Published: September, 2025. DOI: 10.1002/lsm.70042.

Results From the BE BRIGHT Open-Label Extension Trial

[Posted 10/Sep/2025]

AUDIENCE: Dermatology, Family Medicine

KEY FINDINGS: Almost two-thirds of bimekizumab-treated patients achieved and maintained complete skin clearance through 4 years, making bimekizumab an effective, rapid, and durable long-term treatment option.

BACKGROUND: Patients with moderate to severe psoriasis experience significant burden on quality of life. Long-term management with latest-generation biologics can facilitate sustained complete skin clearance and improved patient well-being. Aim of this study is to report 4-year end-of-study bimekizumab efficacy and safety in patients with moderate to severe psoriasis.

DETAILS: Data were pooled from 3 phase 3 trials (BE VIVID, BE READY, and BE SURE) and their open-label extension (OLE; BE BRIGHT). Efficacy is reported for patients who received bimekizumab continuously from baseline into the OLE. Safety is reported for patients who received >=1 bimekizumab dose. Seven hundred seventy-one patients received bimekizumab from baseline into the OLE. A high proportion achieved complete skin clearance (100% improvement from baseline in Psoriasis Area and Severity Index) at Week 52 (76.2%) and maintained this to Week 196 (64.7%). The rate of treatment-emergent adverse events over 4 years was 169.8/100 patient-years (N = 1495) and did not increase with longer exposure. The most common treatment-emergent adverse events were nasopharyngitis, oral candidiasis, and upper respiratory tract infection, consistent with bimekizumab's known safety profile.

Copyright © Elsevier Ltd. All rights reserved.

Source: Blauvelt, A., Langley, R. G., Lebwohl, M., et al. (2024). Bimekizumab Durability of Efficacy Through 196 Weeks and Safety Through 4 Years in Patients With Moderate to Severe Plaque Psoriasis: Results From the BE BRIGHT Open-Label Extension Trial. Journal of the American Academy of Dermatology. 2025; 93: 644-653. Published: September, 2025. DOI: 10.1016/j.jaad.2025.04.038.

[Posted 28/Aug/2025]

AUDIENCE: Neurology, Pediatric, Neurosurgery

KEY FINDINGS: Infants up to 6 weeks of age with genetically diagnosed SMA who were treated with risdiplam before the development of clinical signs or symptoms appeared to have better functional and survival outcomes at 12 and 24 months than untreated infants in natural history studies. Larger, controlled studies with longer follow-up are needed to further understand the relative efficacy and safety of presymptomatic treatment of SMA with risdiplam.

BACKGROUND: Risdiplam, an oral pre–messenger RNA splicing modifier, is an efficacious treatment for persons with symptomatic spinal muscular atrophy (SMA). The safety and efficacy of risdiplam in presymptomatic disease are unclear.

DETAILS: Authors conducted an open-label study of daily oral risdiplam (with the dose adjusted to 0.2 mg per kilogram of body weight) in infants 1 day (birth) to 42 days of age with genetically diagnosed SMA but without strongly suggestive clinical signs or symptoms. The primary outcome, assessed in infants with two SMN2 copies and a baseline ulnar compound muscle action potential (CMAP) amplitude of at least 1.5 mV, was the ability to sit without support at month 12. Natural history studies have shown that the majority of infants with two SMN2 copies who are untreated would have a severe SMA phenotype (type 1), would never sit independently, would receive permanent ventilation and feeding support, or would die by 13 months of age. Secondary outcomes that were assessed over a period of 24 months included survival, ventilatory support, motor milestones, the development of clinically manifested SMA, feeding, and growth. A total of 26 infants with two, three, or four or more copies of SMN2 were enrolled. After 12 months of treatment, 21 infants (81%) could sit unsupported for 30 seconds, 14 (54%) could stand alone, and 11 (42%) could walk alone. A total of 4 of 5 infants (80%; 95% confidence interval, 28 to 100) with two SMN2 copies and a baseline ulnar CMAP amplitude of at least 1.5 mV were able to sit without support for at least 5 seconds. Three infants were withdrawn from the study by a parent or caregiver after the month 12 visit. Of 23 infants who completed 24 months of treatment, all were alive without the use of permanent ventilation or feeding support. Over a period of 24 months, nine treatment-related adverse events were reported in 7 infants; none of these events were serious.

Copyright © Massachusetts Medical Society. All rights reserved.

Source: Finkel, R. S., Servais, L., Vlodavets, D., et al. (2024). Risdiplam in Presymptomatic Spinal Muscular Atrophy. N Engl J Med. 2025; 393(7): 671-682. Published: August 13, 2025. DOI: 10.1056/NEJMoa2410120.