• Ming-Jen Lee, Hung-Chou Kuo, Lin-Na Chou, Marianne T Sweetser, and Jiaan-Der Wang.
• Department of Neurology and Medical Genetics, National Taiwan University Hospital, Taipei City, Taiwan. Electronic address: mjlee@ntu.edu.tw.
• J Formos Med Assoc. 2024 Jun 1; 123 (6): 679686679-686.

Background/PurposeAcute hepatic porphyrias (AHP) are rare genetic disorders associated with acute neurovisceral attacks and chronic symptoms. This analysis was conducted to examine the long-term efficacy and safety of givosiran in Taiwanese participants in the ENVISION study (NCT03338816).MethodsPatients (age ≥12 years) with AHP and recurrent attacks were randomized to receive givosiran 2.5 mg/kg or placebo for 6 months during the double-blind period. Patients then switched from placebo to givosiran (placebo crossover group) or continued taking givosiran (continuous givosiran group) during a 30-month open-label extension period. The total study duration was 36 months. An analysis was conducted that included patients enrolled in Taiwan (N = 7).ResultsDuring the double-blind period and open-label extension period, the median annualized attack rates were 0.0 and 0.0, respectively, in the continuous givosiran group (n = 5) and 15.1 and 4.6, respectively, in the placebo crossover group (n = 2; 70 % decrease). Median annualized days of hemin use in the double-blind period and open-label extension period were 0.0 and 0.0, respectively, in the continuous givosiran group, and 23.8 and 5.0, respectively, in the placebo crossover group (79 % decrease). EQ-5D VAS scores remained relatively stable in both groups, and PPEQ responses indicated improved functioning and satisfaction in both groups. Delta-aminolevulinic acid and porphobilinogen levels remained low with long-term givosiran treatment. Serious adverse events were reported by 3 patients (43 %).ConclusionLong-term efficacy and safety results in the Taiwan cohort are consistent with those in the global cohort.Copyright © 2023 Formosan Medical Association. Published by Elsevier B.V. All rights reserved.

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