ReviewIntractable & rare diseases research2020
Therapeutic strategies for acute intermittent porphyria.
Review in Intractable & rare diseases research, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
13 citing papers in PubMed, 31 citations in OpenAlex.
- Acute intermittent porphyria presenting as posterior reversible encephalopathy syndrome: a case report.Journal of medical case reports · 2026Article
- Porphyria presenting as posterior reversible encephalopathy syndrome.Acta neurologica Belgica · 2025Article
- An Adolescent Female With Disordered Eating and Cannabis Use Found to Have Acute Intermittent Porphyria.Case reports in psychiatry · 2025Article
- German Real-World Experience of Patients with Diverse Features of Acute Intermittent Porphyria Treated with Givosiran.Journal of clinical medicine · 2024Article
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- [Abdominal pain with neuropsychiatric symptoms and ventilatory failure as a presentation of acute porphyria].Revista medica del Instituto Mexicano del Seguro Social · 2023Article
- Review
- Idiosyncratic drug-induced liver injury caused by givosiran in a patient with acute intermittent porphyria.Molecular genetics and metabolism reports · 2023Article
- Elagolix is porphyrogenic and may induce porphyric attacks in patients with the acute hepatic porphyrias.Molecular genetics and metabolism reports · 2022Article
- Mechanistic modelling of enzyme-restoration effects of new recombinant liver-targeted proteins in acute intermittent porphyria.British journal of pharmacology · 2022Article
- Acute Hepatic Porphyria: Pathophysiological Basis of Neuromuscular Manifestations.Frontiers in neuroscience · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Acute intermittent porphyria (AIP) is an autosomal dominant disease caused by mutations in porphobilinogen deaminase (PBGD), the third enzyme of the heme synthesis pathway. Symptoms of AIP usually manifest as intermittent acute attacks with occasional neuropsychiatric crises. The management of AIP includes treatment of acute attacks, prevention of attacks, long-term monitoring and treatment of chronic complications. Intravenous injection of heme is the most effective method of treating acute attacks. Carbohydrate loading is used when heme is unavailable or in the event of mild attacks. Symptomatic treatment is also needed during attacks. Prevention of attacks includes eliminating precipitating factors, heme prophylaxis and liver transplantation. New treatment options include givosiran (siRNA) to down-regulate ALA synthase-1 (ALAS1) and the messenger RNA of PBGD (PBGD mRNA) delivered to the liver cells of patients with AIP. Long-term monitoring of chronic complications includes regular liver-kidney function and hepatocellular carcinoma (HCC) screening.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.