High disease burden and healthcare resource usage in patients with acute porphyria—A population-based analysis
Publication Details
Liver International
October 2024
Author(s)
Ulrich Stölzel1, Bjoern Ambrosius2, Sarah Brun2, Frank Tacke3
Affiliations
1Department of Internal Medicine II, Porphyria Centre, Klinikum Chemnitz, Chemnitz, Germany; 2Alnylam Germany GmbH, Munich, Germany; 3Department of Hepatology and Gastroenterology, Charité - Universitätsmedizin Berlin, Campus Virchow-Klinikum (CVK) and Campus Charité Mitte (CCM), Berlin, Germany
Abstract
Background and Aims:
Acute porphyria is a chronic recurrent disease with late diagnosis, heterogeneous clinical presentations and potentially devastating complications. The study aimed at providing real-world evidence on the natural course of acute porphyria, patient characteristics, disease burden, and healthcare utilization before diagnosis.
Methods:
This observational study used anonymized claims data covering 8 365 867 persons from German statutory health insurance, spanning 6 years (2015–2020). Patients with at least one diagnosis of acute porphyria during the index period (2019–2020) were classified into three groups by attack frequency. These findings were compared with two age- and sex-adjusted 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.
Results:
We revealed a prevalence of 79.8 per 1 000 000 for acute porphyria, with 12.9 per 1 000 000 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%).
Conclusions:
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-world findings provide additional insights on disease characteristics in Germany.
PMID
39016183
DOI
10.1111/liv.16037
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