Adjunctive use of Polypodium leucotomos extract in patients with erythropoietic protoporphyria: An exploratory study.
Adjunctive use of Polypodium leucotomos extract may improve quality of life in some patients with erythropoietic protoporphyria who have incomplete symptom control on afamelanotide, but further studies are necessary to validate these results.
Where it sits
this study against the rest of the afamelanotide corpusSummary and findings
This study evaluated the safety and efficacy of Polypodium leucotomos extract (PLE) as an adjunctive therapy in eight adults with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) who had ongoing symptoms despite regular afamelanotide implants. Participants received 480 mg of oral PLE daily for 4 months. Statistically significant improvements in quality of life (QoL) were observed at Day 60 but not at Day 120.
Abstract
Erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) cause severe photosensitivity, resulting in significant quality of life (QoL) impairment. This study aims to evaluate the safety and efficacy of Polypodium leucotomos extract (PLE) as an adjunctive therapy in patients with persistent symptoms despite standard dosing of afamelanotide. In this prospective single-center cohort study, eight adults with confirmed EPP or XLP and ongoing symptoms despite regular afamelanotide implants every 2 months were enrolled. Participants received 480 mg oral PLE daily for 4 months. QoL and symptom severity were measured using questionnaires at baseline, Day 60, and Day 120. Six participants completed the study. Statistically significant improvements in QoL were observed on Day 60 (p = 0.014), but not at Day 120 (p = 0.152). Half of participants reported reduced reaction severity. No adverse events occurred. Adjunctive PLE improved short-term QoL in participants with incomplete symptom control on afamelanotide alone and was well tolerated. Larger studies are warranted.
Background
This paper addresses the need for effective adjunctive therapies in patients with erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), conditions known for causing severe photosensitivity and significant impairment in quality of life (QoL). Previous treatments, including afamelanotide, may not fully alleviate symptoms for all patients. The exploration of Polypodium leucotomos extract (PLE) as an adjunctive therapy is significant given the limited options available for these conditions.
Methods
This was a prospective single-center cohort study involving eight adults with confirmed EPP or XLP who continued to experience symptoms despite regular afamelanotide implants administered every 2 months. Participants received 480 mg of oral PLE daily for a duration of 4 months. QoL and symptom severity were assessed using questionnaires at baseline, Day 60, and Day 120.
Results
At Day 60, a statistically significant improvement in QoL was observed with a p-value of 0.014. However, at Day 120, the improvement was not statistically significant, with a p-value of 0.152. Half of the participants reported a reduction in reaction severity. Six out of eight participants completed the study without any adverse events.
Interpretation
The findings suggest that while PLE may provide short-term improvements in QoL for patients with EPP or XLP who do not achieve complete symptom control with afamelanotide, the clinical significance of these improvements is uncertain given the lack of sustained effects at Day 120. The small sample size and single-center design limit the reliability of these results, and further research is needed to confirm these findings in larger populations.
Key findings
- 480 mg oral PLE daily for 4 months.
- Statistically significant improvement in QoL on Day 60, p=0.014.
- No statistically significant improvement in QoL at Day 120, p=0.152.
- Half of participants reported reduced reaction severity.
- Six participants completed the study.
Limitations
- small sample size of eight participants
- single-center study limits generalizability
- short follow-up duration of 4 months
- statistically significant improvement not sustained at Day 120