Scenesse improves life quality in EPP adults: Real-world study
Study, largest of its kind, measures short- and long-term treatment
Short- and long-term treatment with Scenesse (afamelanotide) improves life quality in adults with erythropoietic protoporphyria (EPP), according to the largest real-world study of its kind.
The therapy’s safety profile among those over 70 was consistent with that of the overall patient population, with nausea, cold-like symptoms, headache, and fatigue being among the most common side effects reported, researchers said.
“It can be concluded that [Scenesse] significantly increases the [quality of life] of patients in comparison with no treatment,” they wrote.
The study, “German Cohort Observational Study to Investigate the Short- and Long-Term Safety and Clinical Effectiveness of Afamelanotide 16 mg (SCENESSE) in Patients With Erythropoietic Protoporphyria (EPP),” was published in Photodermatology, Photoimmunology & Photomedicine. The study was funded by Clinuvel, the company marketing Scenesse.
EPP is a genetic disease marked by skin photosensitivity, meaning that light exposure can cause tingling, itching, burning, redness, swelling, and intense pain in the skin. Such symptoms typically arise in infancy, but can also manifest in adolescence or adulthood.
Reducing painful reactions to sunlight
Inserted as an implant under the skin, Scenesse is approved in the U.S., Europe, and Australia as a skin-protective treatment for adults with EPP. It’s designed to reduce light sensitivity by increasing the production of eumelanin, a brown pigment that protects the skin from the sun’s ultraviolet rays, so it’s expected to reduce painful reactions to sunlight and ease EPP symptoms.
As part of the treatment’s marketing approval in Europe, a disease registry and a post-authorization safety study, or PASS (EUPAS13004), were established to assess the long-term safety and efficacy of Scenesse in real-world settings.
Researchers in Germany reported PASS data from four German EPP centers collected from 2016 to 2021. The disease registry consisted of 200 EPP patients treated with Scenesse, 79.5% of whom had never received the therapy before PASS.
A mean of 11.2 doses of Scenesse were administered per patient, with the number of doses ranging from one to 27. The maximum number of doses in any given year was six, and the minimum was zero. Implant usage was consistent over time, with some patients requesting additional treatments over the winter months.
Scenesse’s effectiveness was evaluated by its impact on quality of life, as measured by the EPP-specific quality of life questionnaire (EPP-QoL), in which higher total scores indicate a better quality of life.
During each two-month period, patients who had assessments were identified and their total EPP-QoL scores included in the analysis for that period. As of June 2021, 91% of the patients were still receiving treatment within the registry.
In all but three two-month periods from January 2017 to June 2021, EPP-QoL scores significantly increased, indicating a marked improvement in life quality in EPP patients treated with Scenesse.
The team noted that although changes in EPP-QoL scores were significant, the smallest improvements occurred in May and June, when patients usually experience more EPP symptoms due to increasing daylight intensity. The greatest improvements were seen in November and December. Patients reported a significant increase of at least 55% in EPP-QoL scores during these most challenging months, demonstrating a positive impact, the researchers said.
About half of the patients (54%) reported one or more treatment-emergent adverse events (TEAEs) related to treatment. The most common TEAEs were vitamin D deficiency (29%), nausea (18.5%), cold-like symptoms (18%), headache (16%), and fatigue (10.5%). Four patients (2%) reported serious TEAEs related to treatment. Vitamin D deficiency was a consequence of lack of sunlight exposure and not considered to be treatment-related.
With short-term treatment, defined as receiving five or fewer implants, less than half (45.5%) of the patients reported a TEAE related to treatment. Nausea (14.5%) and headache (13.5%) were the most common TEAEs in these patients.
For long-term usage, defined as receiving 15 or more implants, about one in 10 patients (11.3%) reported having one or more TEAEs related to treatment. The most common TEAEs observed during long-term treatment included vitamin D deficiency (13.2%) and cold-like symptoms (7.5%).
The safety profile of the seven patients older than 70 was consistent with that of the overall patient population. A total of 17 TEAEs were reported in this subgroup (71.4%), with one patient (14.3%) experiencing vomiting related to treatment. Three of these patients reported serious TEAEs, none related to treatment.
“Data from the largest EPP Disease Registry in the world and largest patient cohort ever reported has … shown that [Scenesse] provides a significantly higher [quality of life], with overall health benefits for patients over the long term,” the researchers wrote.
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