In:
American Journal of Gastroenterology, Ovid Technologies (Wolters Kluwer Health), Vol. 114, No. 1 ( 2019-10), p. S578-S578
Abstract:
Acute Hepatic Porphyria (AHP) is a family of rare genetic diseases due to enzyme deficiencies in heme biosynthesis in the liver that can lead to neurovisceral attacks and chronic symptoms. (1, 2) AHP is also associated with multiple long-term complications, including neuropathy (3,4), hypertension (5,6), chronic kidney disease (7), and hepatocellular carcinoma (8,9). METHODS: ENVISION (NCT03338816) was a Phase 3 global, multicenter, randomized, double-blind, placebo-controlled trial with an open label extension to evaluate the efficacy and safety of subcutaneous givosiran in AHP. Eligible patients had an AHP diagnosis and ≥2 attacks within the prior 6 months. At baseline detailed medical history and laboratory assessments were obtained. RESULTS: Ninety-four AHP patients enrolled, median (range) age 37.5 (19-65) years, 89% female, and median (range) of 6.5 (0.1–43) years since diagnosis. Patients had a median of 4 composite attacks in the preceding 6 months and 40% were on hemin prophylaxis prior to study. Half the patients experienced daily chronic symptoms and 29% used opioids daily/most days between attacks. Comorbidities reported on medical history included neuropathy (38%), central venous access complications (71%), iron overload (33%), liver disease (28%), and chronic kidney disease (18%). At baseline 17% of patients had liver transaminases 〉 ULN and 34% had an eGFR 〈 60 mL/min/1.73m2. CONCLUSION: The baseline disease characteristics of the Phase 3 study population are consistent with findings reported in the AHP literature, underscoring the high rate of liver disease and central venous access complications in these patients, and highlighting the multi-systemic nature of AHP and the medical challenge it presents for diagnosis and treatment.
Type of Medium:
Online Resource
ISSN:
0002-9270
,
1572-0241
DOI:
10.14309/01.ajg.0000593520.44985.d3
Language:
English
Publisher:
Ovid Technologies (Wolters Kluwer Health)
Publication Date:
2019
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