Long-term retrospective analysis of gene therapy with Alipogene Tiparvovec and its effect on lipoprotein lipase deficiency-induced pancreatitis
Open accessAbstract
Alipogene tiparvovec (Glybera) is a gene therapy product approved in Europe under the "exceptional circumstances" pathway as a treatment for lipoprotein lipase deficiency (LPLD), a rare genetic disease resulting in chylomicronemia and a concomitantly increased risk of acute and recurrent pancreatitis, with potentially lethal outcome. This retrospective study analyzed the frequency and severity of pancreatitis in 19 patients with LPLD up to 6 years after a single treatment with alipogene tiparvovec. An independent adjudication board of three pancreas experts, blinded to patient identification and to pre- or post-gene therapy period, performed a retrospective review of data extracted from the patients' medical records and categorized LPLD-related acute abdominal pain events requiring hospital visits and/or hospitalizations based on the adapted 2012 Atlanta diagnostic criteria for pancreatitis. Both entire disease time period data and data from an equal time period before and after gene therapy were analyzed. Events with available medical record information meeting the Atlanta diagnostic criteria were categorized as definite pancreatitis; events treated as pancreatitis but with variable levels of laboratory and imaging data were categorized as probable pancreatitis or acute abdominal pain events. A reduction of approximately 50% was observed in all three categories of the adjudicated post-gene therapy events. Notably, no severe pancreatitis and only one intensive care unit admission was observed in the post-alipogene tiparvovec period. However, important inter- and intraindividual variations in the pre- and post-gene therapy incidence of events were observed. There was no relationship between the posttreatment incidence of events and the number of LPL gene copies injected, the administration of immunosuppressive regimen or the percent triglyceride decrease achieved at 12 weeks (primary end point in the prospective clinical studies). Although a causal relationship cannot be established and despite the limited number of individuals evaluated, results from this long-term analysis suggest that alipogene tiparvovec was associated with a lower frequency and severity of pancreatitis events, and a consequent overall reduction in health care resource use up to 6 years posttreatment.
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The first approved gene therapy in Europe, alipogene tiparvovec (Glybera), incorporated a 12-week immunosuppression regimen with ciclosporin (3mg/kg/day) and MMF (2g/day).
“The first approved gene therapy in Europe, alipogene tiparvovec (Glybera), incorporated 12-week immunosuppression regimen with ciclosporin (3mg/kg/day) and MMF (2g/day) (32).”
From Immunomodulation in Administration of RAAV: Preclinical and Clinical Adjuvant Pharmacotherapies (2021) · cites this paper as [32]
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Self-citation
- Authors
- Gaudet, D., Stroes, E. S., Méthot, J., Brisson, D., Tremblay, K., Bernelot Moens, S. J., Iotti, G., Rastelletti, I., Ardigo, D., Corzo, D., Meyer, C., Andersen, M., Ruszniewski, P., Deakin, M., & Bruno, M. J.
- Affiliations
- Université de Montréal (CA)
- Amsterdam UMC Location University of Amsterdam (NL)
- Academic Medical Center, Amsterdam, The Netherlands
- Chiesi (Italy) (IT)
- Chiesi Farmaceutici, Parma, Italy
- UniQure (Netherlands) (NL)
- Hôpital Beaujon (FR)
- University Hospitals of North Midlands NHS Trust (GB)
- Erasmus MC (NL)
- ORCID
- Venue
- Human Gene Therapy
- Publisher
- SAGE Publications
- Published
- 2016-11
- Volume
- 27
- Issue
- 11
- Pages
- 916-925
- ISSN
- 1043-0342 (ISSN-L)
- 1557-7422
- OpenAlex
- W2512734535
- Cited by
- 100
- References
- 38
- Citations by year
- 2026: 4 · 2025: 6 · 2024: 12 · 2023: 10 · 2022: 7 · 2021: 8 · 2020: 11 · 2019: 11 · 2018: 12 · 2017: 18 · 2016: 1
- FWCI
- 11.55
- Type
- journal article
- Language
- en
- Open access
- green
- pure.amsterdamumc.nl
- Locations
- Human Gene Therapy (publishedVersion)
- PubMed (publishedVersion)
- EUR Research Repository (Erasmus University Rotterdam) (publishedVersion)
- Pure Amsterdam UMC (submittedVersion, other-oa)
- Data Archiving and Networked Services (DANS) (submittedVersion)
- EUR Research Repository (Erasmus University Rotterdam) (submittedVersion)
- Corpus Université Laval (Université Laval) (submittedVersion)
- Topics
- Health Sciences › Medicine › Cardiology and Cardiovascular Medicine › Lipid metabolism and disorders
- Health Sciences › Medicine › Surgery › Pancreatitis Pathology and Treatment
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Keywords
- lipoprotein lipase deficiency, pancreatitis, gene therapy, chylomicronemia, long-term safety, health care utilization, triglyceride lowering, immunosuppression, rare genetic disorders
- MeSH terms
- Dependovirus, Adult, Europe, Female, Genetic Vectors, Humans, Lipoprotein Lipase, Hyperlipoproteinemia Type I, Male, Middle Aged, Pancreatitis, Retrospective Studies, Time Factors, Genetic Therapy, Young Adult
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