Retrospective data collection of psoriasis treatment with fumaric acid esters in children and adolescents in Germany (KIDS FUTURE study)

Abstract

SummaryBackground: Given that there is no standard systemic treatment for children and adolescents with plaque psoriasis, this non-interventional, multicenter, retrospective study collected data on the efficacy and safety of long-term treatment with fumaric acid esters (FAEs) in this particular patient group. Patients and methods: In patients younger than 18 years of age at the start of FAE treatment, data on efficacy and safety was retrospectively collected for at least 36 months. Results: Data from 127 patients (aged 6-17 years) was collected for treatment durations of up to 60 months. Physician's Global Assessment, Psoriasis Area and Severity Index, and Body Surface Area showed marked improvement in the first six months. After 36 months, these parameters had, on average, improved by up to two-thirds of baseline values. Thirty-seven patients experienced at least one adverse event (AE), which was FAE-related in 36 individuals. Three AEs (proteinuria (one case), flushing (two cases)) persisted during the observation period while on treatment. Fifteen AEs led to the discontinuation of therapy; nearly all of these cases were related to gastrointestinal disorders. Conclusions: The KIDS FUTURE study -for the first time -included a larger population of children and adolescents with psoriasis who were treated with FAEs. The data obtained suggests that long-term FAE therapy in this patient group may be effective and safe. The results are currently being verified in an ongoing clinical study.

Long‐term real‐life safety profile and effectiveness of fumaric acid esters in psoriasis patients: a single‐centre, retrospective, observational study

“…A previously published retrospective study (‘FUTURE’ study) of 984 psoriasis patients with a mean duration of 44 months of continuous FAE monotherapy reported greater improvement, as assessed with the dynamic PGA: 67% of patients were documented as improved or clear after 0.5 years, 78% after 2 years and 82% after 3 years of FAE therapy. However, the following parameters differed, preventing direct comparison: (i) different PGA scores were used and (ii) patients who stopped FAE monotherapy within the first 2 years because of lack of efficacy or insufficient tolerability were not recorded in the FUTURE study . In our study, all patients were included in data analysis, irrespective of treatment discontinuation.…”

“…We acknowledge several potential limitations of this observational study. First, the study's retrospective design may have resulted in recall bias . Incomplete data recording in the hospital digital database and patients lost to follow‐up resulted in missing data, particularly on continuation of FAE therapy regimens.…”

Clinical use of dimethyl fumarate in moderate‐to‐severe plaque‐type psoriasis: a European expert consensus

“…Although the use of DMF in the paediatric population is off‐label, there are numerous case reports, case series and unpublished clinical experience to suggest that FAEs may be an effective treatment option for children and adolescents if a systemic therapy is indicated . A German multicentre, retrospective study on the efficacy and safety of long‐term use of FAEs in children and adolescents showed that FAEs were often used successfully in clinical practice as off‐label therapy . Trials of DMF in paediatric populations and for more moderate disease are ideally needed.…”

S2k‐Leitlinie zur Therapie der Psoriasis bei Kindern und Jugendlichen – Kurzfassung Teil 2

“…Zunächst war ein Fertigarzneimittel mit einem Wirkstoffgemisch aus Dimethylfumarat und Salzen von Ethylhydrogenfumarat (Fumaderm ® ) in Deutschland verfügbar, seit 2017 existiert zudem eine Zulassung der europäischen Arzneimittelagentur (EMA) für ein Dimethylfumarat‐Monopräparat (Skilarence ® ). Über die Anwendung von Fumaderm ® bei Kindern und Jugendlichen mit Psoriasis vulgaris wurden mehrere Fallberichte, Fallserien und retrospektive Datenerhebungen publiziert . Auch für die Anwendung eines Dimethylfumarat‐Monopräparates existiert eine retrospektive Datenerhebung, allerdings in der Indikation multiple Sklerose .…”