{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,1]],"date-time":"2026-05-01T13:41:59Z","timestamp":1777642919461,"version":"3.51.4"},"reference-count":27,"publisher":"Wiley","issue":"10","license":[{"start":{"date-parts":[[2024,7,17]],"date-time":"2024-07-17T00:00:00Z","timestamp":1721174400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Liver International"],"published-print":{"date-parts":[[2024,10]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background and Aims<\/jats:title><jats:p>Acute porphyria is a chronic recurrent disease with late diagnosis, heterogeneous clinical presentations and potentially devastating complications. The study aimed at providing real\u2010world evidence on the natural course of acute porphyria, patient characteristics, disease burden, and healthcare utilization before diagnosis.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This observational study used anonymized claims data covering 8\u2009365\u2009867 persons from German statutory health insurance, spanning 6\u2009years (2015\u20132020). Patients with at least one diagnosis of acute porphyria during the index period (2019\u20132020) were classified into three groups by attack frequency. These findings were compared with two age\u2010 and sex\u2010adjusted reference groups: the general population and fibromyalgia patients. Prevalence over the index period was calculated for all porphyria patients and those with active acute porphyria.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>We revealed a prevalence of 79.8 per 1\u2009000\u2009000 for acute porphyria, with 12.9 per 1\u2009000\u2009000 being active cases. Acute porphyria patients, particularly with frequent attacks, demonstrated a higher comorbidity burden compared to the general population. Within the year before the recorded diagnosis, patients with acute porphyria required a median of 23.0 physician visits, significantly higher than the general population's 16.0. Additionally, 33.8% were hospitalized at least once during this period, a notably higher proportion than the general population (19.3%).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>This study's findings, collected before the introduction of givosiran, as the first approved preventive therapy for acute porphyria in Europe, highlight the need for healthcare strategies and policies tailored to the complex needs of acute porphyria patients. The significant healthcare demands, heightened comorbidity burden, and increased healthcare system utilization emphasize the urgency of developing a comprehensive support infrastructure for these patients. Also, these acute porphyria real\u2010world findings provide additional insights on disease characteristics in Germany.<\/jats:p><\/jats:sec>","DOI":"10.1111\/liv.16037","type":"journal-article","created":{"date-parts":[[2024,7,17]],"date-time":"2024-07-17T10:48:55Z","timestamp":1721213335000},"page":"2639-2650","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["High disease burden and healthcare resource usage in patients with acute porphyria\u2014A population\u2010based analysis"],"prefix":"10.1111","volume":"44","author":[{"given":"Ulrich","family":"St\u00f6lzel","sequence":"first","affiliation":[{"name":"Department of Internal Medicine II Porphyria Centre, Klinikum Chemnitz  Chemnitz Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3679-7907","authenticated-orcid":false,"given":"Bjoern","family":"Ambrosius","sequence":"additional","affiliation":[{"name":"Alnylam Germany GmbH  Munich Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sarah","family":"Brun","sequence":"additional","affiliation":[{"name":"Alnylam Germany GmbH  Munich Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6206-0226","authenticated-orcid":false,"given":"Frank","family":"Tacke","sequence":"additional","affiliation":[{"name":"Department of Hepatology and Gastroenterology Charit\u00e9 \u2013 Universit\u00e4tsmedizin Berlin, Campus Virchow\u2010Klinikum (CVK) and Campus Charit\u00e9 Mitte (CCM)  Berlin Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2024,7,17]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.3389\/fnins.2021.715523"},{"key":"e_1_2_13_3_1","doi-asserted-by":"publisher","DOI":"10.1002\/jimd.12612"},{"key":"e_1_2_13_4_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00277-019-03831-7"},{"key":"e_1_2_13_5_1","doi-asserted-by":"publisher","DOI":"10.7326\/0003-4819-142-6-200503150-00010"},{"key":"e_1_2_13_6_1","doi-asserted-by":"publisher","DOI":"10.3390\/metabo4040977"},{"key":"e_1_2_13_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.amjmed.2014.06.036"},{"key":"e_1_2_13_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgmr.2022.100842"},{"key":"e_1_2_13_9_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12325-022-02172-8"},{"key":"e_1_2_13_10_1","doi-asserted-by":"publisher","DOI":"10.1002\/hep.30936"},{"issue":"10","key":"e_1_2_13_11_1","first-page":"986","article-title":"Hypertension and renal impairment as complications of acute porphyria","volume":"7","author":"Church SE","year":"1992","journal-title":"Nephrol Dial Transplant"},{"key":"e_1_2_13_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0016-5085(83)80081-X"},{"key":"e_1_2_13_13_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-012-9576-9"},{"key":"e_1_2_13_14_1","doi-asserted-by":"publisher","DOI":"10.1007\/978-94-007-7596-1"},{"key":"e_1_2_13_15_1","doi-asserted-by":"publisher","DOI":"10.3389\/fpubh.2021.675523"},{"key":"e_1_2_13_16_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-012-9544-4"},{"key":"e_1_2_13_17_1","unstructured":"European Medicines Agency (EMA).Public summary of opinion on orphan designation: Synthetic double\u2010stranded siRNA oligonucleotide directed against delta aminoevulinic acid synthase 1 mRNA covalently linked to a ligand containing three Nacetylgalactosamine residues for the treatment of acute hepatic prophyria (EU\/3\/16\/1731).2016."},{"key":"e_1_2_13_18_1","doi-asserted-by":"publisher","DOI":"10.1007\/8904_2015_406"},{"key":"e_1_2_13_19_1","unstructured":"Alnylam Germany GmbH.Dossier zur Nutzenbewertung gem\u00e4\u00df \u00a7 35a SGB V. 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