Heme arginate improves acute liver failure secondary to erythropoietic protoporphyria: a case report.

INTRODUCTION

Erythropoietic protoporphyria (EPP) is a rare inherited disorder with limited therapeutic options. For EPP patients with advanced cholestasis or liver failure, liver transplantation has long been regarded as the definitive therapy to address the hepatic crisis but does not offer a cure. Previous studies have reported that intravenous hemin/hematin may benefit some EPP patients, while the efficacy of intravenous heme arginate in treating EPP remains unreported.

CASE PRESENTATION: We report a 24-year-old male with EPP and severe cholestasis. From 2017 to 2024, the patient was hospitalized eight times for recurrent episodes of jaundice and abdominal pain. These symptoms were effectively alleviated through a comprehensive treatment including artificial liver support (PE + DPMAS), hepatoprotective therapy, and high-glucose infusion. During his latest hospitalization in 2025, artificial liver support failed to improve the liver dysfunction, leading to progressive deterioration to liver failure. Notably, after two doses of intravenous heme arginate, the total bilirubin level gradually declined with significant improvement of ascites, coagulation dysfunction and neuropsychiatric symptoms. The patient was discharged with hepatoprotective therapy and light avoidance with no recurrence at 6-month follow-up.

CONCLUSION

This case suggests that intravenous heme arginate may provide clinical benefit in patients with EPP-associated liver failure and contributes to the limited literature supporting heme-based therapy in treating EPP hepatic crisis.