June 13, 2012Current Opinion in Lipidology
Gene therapy for lipoprotein lipase deficiency
Why the study?
Does AAV1-LPLS447X gene therapy reduce pancreatitis incidence and improve clinical signs in patients with lipoprotein lipase deficiency?
Population
Patients with lipoprotein lipase deficiency
Design
Review
Follow-up
up to 2 years
Authors
Discussion
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Member takes
Implication
May support AAV1-LPLS447X evaluation in LPL deficiency; leaves open need for randomized confirmation of benefit.
Structured PICO
Does AAV1-LPLS447X gene therapy reduce pancreatitis incidence and improve clinical signs in patients with lipoprotein lipase deficiency?
P
Population
Patients with lipoprotein lipase deficiency (a rare inherited disease characterized by severe hypertriglyceridaemia, chylomicronaemia and risk of recurrent pancreatitis or other complications)
I
Intervention
AAV1-LPLS447X gene therapy (alipogene tiparvovec) via intramuscular administration
O
Outcome
Overall pancreatitis incidence and signs of clinical improvement
AAV1-LPLS447X gene therapy appears to offer a positive benefit-risk ratio, particularly for patients at highest risk of complications from lipoprotein lipase deficiency.
Cite This Study
Gaudet et al. (2012) studied this question.