Scenesse Increases Sunlight Tolerance in Adults With EPP, Real-world Data Show
Treatment with Scenesse increased damage-free light exposure from a median of 10 minutes to three hours.
The study, “Increased phototoxic burn tolerance time and quality of life in patients with erythropoietic protoporphyria treated with afamelanotide – a three years observational study,” was published in the Orphanet Journal of Rare Diseases.
Scenesse, by Clinuvel, is a skin implant that works as an antioxidant and protector against the effects of sunlight, significantly extending the time patients can be outdoors without phototoxic reactions — damage to the skin induced by sunlight exposure.
The approved dose of Scenesse is 16 mg (in one implant), given under the skin once every two months, during the spring and summer months. Its benefits are expected to last approximately two months.
Scenesse’s effectiveness was shown in two Phase 3 clinical trials with EPP patients. The results showed that the implant significantly extended the time patients were exposed to direct sunlight with no pain compared with the placebo group.
Now, researchers reported real-world data of Scenesse treatment in a Swiss group of 39 adults with EPP. They conducted an analysis of clinical data collected between 2016 and 2018.
The implant’s effectiveness was measured with a new parameter, named phototoxic burn tolerance time (PBTT), defined as the maximum time patients are able to spend in sunlight without experiencing damage to the skin. The team then calculated a phototoxic burn protection factor (PBPF) for each patient by dividing PBTT under treatment by PBTT before treatment.
“PBTT is easily assessed in real-life situations and seems to reflect the reported real-world experience of patients more adequately than any previously applied measurement method,” the researchers wrote.
Additional parameters included the severity of phototoxic reactions, assessed by the 11-point Likert-type visual analogue scale, in which a score of zero meant no pain and a score of 10 meant the worst possible pain. Quality of life also was assessed, using an EPP-specific questionnaire.
A total of 33 patients began treatment before 2016, with a median duration of 3.5 years. Due to reimbursement issues, 20 patients (51%) interrupted their treatment — over a median of 413 days.
Before treatment with Scenesse, these individuals were only able to endure sunlight without damage to the skin for a median of 10 minutes (PBTT range from 2-to-120 minutes). Under treatment, PBTT significantly increased to a median of 180 minutes, or three hours (range 15-to-420 minutes).
All patients under treatment had their sunlight tolerance significantly increased. The extent of the PBTT improvement was independent of PBTT before treatment. Median PBPF was 15.
Treatment efficacy, as assessed by PBTT increases, lowered when the interval between Scenesse doses exceeded the recommendation of 60 days.
Maximum pain severity had a median score of 10 before treatment (data of 36 patients), decreasing significantly to a median of six under Scenesse (data from 31 patients). All participants experienced pain reduction.
Quality of life increased from a median of 49.1% before treatment to 81.4% after Scenesse. Treatment interruption did not preclude improvements in quality of life under treatment.
In turn, treatment adherence during the period of analysis was 97.4%. The two patients who stopped treatment did so because of an intended pregnancy — a compelling reason for treatment interruption — and of an adverse event not related to Scenesse.
Overall, these individuals received between one and six doses of therapy per year, depending on their medical needs and reimbursement decisions.
“Our real-life data suggest that, so far, the effectiveness of afamelanotide [Scenesse] may have been underestimated and may go beyond the reported benefit in the RCTs [randomized, controlled clinical trials],” the scientists wrote.
“In this regard, we suggest PBTT and PBPF as a reasonable, clinically meaningful and easily applicable parameter to evaluate the real-world effectiveness of any treatment in EPP,” they added.