Comparative effectiveness of human hematin and heme arginate in the management of porphyria attacks: an observational study across three hospitals in Colombia.

Abstract

Background: Porphyria is an orphan disease classified as a genetic disorder caused by a partial or high-grade deficiency of enzymes involved in the synthesis of heme, an essential component of hemoglobin. This deficiency results in the accumulation of porphyrins (ALAS1 and PBG), intermediates in the heme metabolic pathway. This accumulation triggers porphyria attacks. In Colombia the Heme Arginate and Human Hematin are the therapeutics alternatives for the management of porphyria Attacks.

Objective: To evaluate the comparative effectiveness of heme arginate versus human hemin for treating porphyria attacks in hospitalized patients across three institutions in Medellin, Colombia.

Methods: An observational and analytical study was conducted to compare the outcomes of treatment with human hematin versus heme arginate in clinical episodes of patients diagnosed with porphyria between 2015-2021.

Results: In episodes treated with heme arginate (ArgH), 75% achieved pain control or reduction, 41.6% showed a reduction in opioid dosage, and 88.8% achieved resolution of the Porphyria attack. For episodes treated with human hematin (HH), 85.3% achieved pain control or reduction, 53.6% showed a reduction in opioid dosage, and 90.2% achieved resolution of the attack. When evaluating the effectiveness of both treatments, no statistically significant differences were observed across the three predefined effectiveness outcomes of the study.

Conclusions: This study provides a comparative evaluation of heme arginate (ArgH) and human hematin (HH) in the management of Porphyria attacks, demonstrating that both treatments are similarly effective in achieving pain control, reducing opioid use, and resolving clinical attacks.