Single-center real-world experience with givosiran for management of acute intermittent porphyria.

Givosiran, an ALAS1 targeted siRNA therapy, decreases the frequency of acute attacks in acute hepatic porphyrias by lowering the neurotoxic heme precursors, aminolevulinic acid and porphobilinogen. Here, we present real-world data on clinical, biochemical, efficacy and safety for fifteen patients with acute intermittent porphyria evaluated at our center. Our experience shows that givosiran reduces the frequency of acute attacks, with eleven out of thirteen patients being attack-free after approximately one year of treatment. Dose reduction was attempted in eight patients due to adverse events, including elevated creatinine, acute pancreatitis, and severe injection site reactions. The most common side effects reported from our cohort were nausea, fatigue, and headaches. In this real-world cohort, fewer hemin-treated events were observed during givosiran treatment. Our experience suggests that personalized dosing can be implemented in selected patients, particularly in those who experience adverse events.