Treatment of congenital Langerhans cell histiocytosis with cobimetinib.

We report a case of congenital multisystem Langerhans cell histiocytosis with cutaneous and hematopoietic involvement. After the failure of first-line (vinblastine and prednisolone) and second-line (vincristine and cytarabine) therapies, treatment with cobimetinib, a mitogen-activated protein kinase (MEK) inhibitor, led to the remission of disease and a sustained response after 11 months of ongoing treatment. Protein kinase inhibitors targeting BRAF or MEK could represent a promising future therapeutic option, also in children with LCH.

Lymphoplasmacytic plaque in children: Case report and literature review.

We report a case of benign lymphoplasmacytic plaque (LPP) in a child. These asymptomatic erythematous papulonodular lesions are an emerging clinicopathological entity. Herein, we describe a previously unreported site for LPP lesions, namely, the volar wrist and the distal ipsilateral palm.

Belgian atopic dermatitis guidelines.

Atopic dermatitis (AD) is one of the most common, bothersome and difficult to treat skin disorders. Recent introduction of new systemic treatments has revolutionized the management of AD. The goal of this guideline is to provide evidence-based recommendations for the management of patients suffering from atopic dermatitis that easily can be implemented in clinical practice. These recommendations were developed by 11 Belgian AD experts. Comments of all experts on the proposed statements were gathered, followed by an online voting session. The most relevant strategies for the management and treatment of AD in the context of the Belgian health care landscape are discussed. General measures, patient education and adequate topical treatment remain the cornerstones of AD management. For moderate to severe AD, the introduction of biologics and JAK inhibitors show unprecedented efficacy, although currently access is limited to a subgroup of patients meeting the reimbursement criteria.

Real-Life Effectiveness of Apremilast for the Treatment of Psoriasis in Belgium: Results From the Observational OTELO Study.

INTRODUCTION: Apremilast is approved for the treatment of psoriasis and psoriatic arthritis. However, data on the efficacy and safety of apremilast in clinical practice are limited. We assessed the real-world use and effectiveness of apremilast in patients with moderate to severe plaque psoriasis visiting dermatologist practices in Belgium, from the perspectives of the patient and the physician. METHODS: This prospective observational study enrolled adults aged 18 years or more initiating apremilast between 6 April 2017 and 30 June 2018, per Belgian reimbursement criteria. Primary outcome was the Patient Benefit Index for Skin Diseases (PBI-S). Secondary outcomes included the Patient Global Assessment (PtGA), Dermatology Life Quality Index (DLQI), Psoriasis Area and Severity Index (PASI), and body surface area (BSA). Patients were followed up for up to 18 months. RESULTS: Overall, 122 enrolled patients received at least one dose of apremilast, of which 89 received treatment for more than 150 days and were included in the reference population. Treatment goals most frequently identified (at least 70% of patients) as "very important" in the PBI-S were related to physical impairments. After 6 months of apremilast treatment, 61-78% of patients reported they had achieved these goals; only 12.5% assessed their disease as severe (PtGA, 53.6% at apremilast initiation) and over half reported a DLQI score of 5 or less, indicating improved quality of life. As assessed by the physician, 68.4% and 35.1% of patients achieved at least a 50% and 75% reduction in PASI, respectively, at month 6. Apremilast was well tolerated with no new safety signals identified. CONCLUSIONS: Our real-world data indicate that apremilast fulfils the expectations of Belgian patients with moderate to severe psoriasis, and from the perspectives of both the patient and physician, apremilast has a positive impact on their disease. TRIAL REGISTRATION: ClinicalTrials.gov Identifier NCT03097003.

Disease burden and treatment history among adults with atopic dermatitis receiving systemic therapy: baseline characteristics of participants on the EUROSTAD prospective observational study.

BACKGROUND: Insights into the real-world treatment paradigm and long-term burden of atopic dermatitis (AD) are needed to inform clinical and health policy decisions. METHODS: The prospective, observational EUROSTAD study enrolled adults with moderate-to-severe AD starting or switching systemic therapy (51 sites in 10 European countries). We report the baseline characteristics, treatment patterns, and outcomes of these patients using descriptive statistics. RESULTS: A 12-month enrollment period of EUROSTAD was completed and 308 patients were enrolled: average age 37 years, AD duration 25 years, 43% were female. Most patients reported use of systemic therapy (93%) and ≥1 atopic comorbidity (82%). Mean [standard deviation] disease severity/burden measures were high: Investigator's Global Assessment (3.1 [0.8]), Eczema Area and Severity Index (16.2 [10.9]), Peak Pruritus Numerical Rating Scale (5.5 [2.5]), sleep impairment Visual Analog Scale (49.8 [31.6]) scores, and time lost from work (4.1 [13.7] days/year) or usual activities (16.8 [38.7] days/year). Most patients showed borderline or clinical levels of anxiety (59%) and/or depression (63%) using the Hospital Anxiety and Depression Scale. CONCLUSIONS: Adults with moderate-to-severe AD starting/switching systemic treatment enrolled in EUROSTAD have a high burden of longstanding disease despite continuous use of topical drugs, emollients, and systemic therapies.