Baseline urinary ALA and PBG as criteria for starting pharmacologic prophylactic treatment in acute intermittent porphyria treated with givosiran.

ABSTRACT:

Introduction

For patients with acute intermittent porphyria (AIP), a true attack could be difficult to distinguish from chronic abdominal pain. This study focused on treatment responses from two patients with confirmed elevated biochemical data (delta-aminolevulinic acid (ALA), porphobilinogen (PBG)) and clinical evidence for acute attacks before starting givosiran.

Methods

Data from patients who participated in the phase III givosiran trial in Taiwan between May 2018 and May 2021 were reviewed. The pre-trial and post-trial biochemical data (urinary ALA/PBG), annualized attack rate (AAR), for two participants were obtained from our hospital record.

Results

Two patients had detailed records of biochemical evidence of acute attacks pre-trial (ALA:11.66-79.8 mg/24-h urin

SUBMITTER: Kuo HC 

PROVIDER: S-EPMC11667067 | biostudies-literature | 2024 Dec

REPOSITORIES: biostudies-literature