![[object Object],[object Object],[object Object],Thérapeutique des hyperchylomicronémies par déficit en LPL Perspectives de thérapie génique Conflict of interest. EB has received fees for presentation and participation to advisory boards from Astra Zeneca, MSD, AMT, GSK, Genzyme, Sanofi Aventis, Novo Nordisk, Lilly, Pfizer, Takeda.](https://image.slidesharecdn.com/bruckert27-05-11-hyperchylomicronemies-110711073551-phpapp02/85/12-eme-journee-Hyperchylomicronemie-nouvelles-perspectives-de-therapie-genique-1-320.jpg)
![Capillary endothelium Muscle Lipoprotein Lipase (LPL): Mechanism of action ,[object Object],[object Object],[object Object],Heparan Sulphate/ GPIHBP1 LPL ApoCII](data:image/gif;base64,R0lGODlhAQABAIAAAAAAAP///yH5BAEAAAAALAAAAAABAAEAAAIBRAA7)
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- 4. Pancreatitis over 350 times more common in LPLD than in healthy controls Gaudet et al Atherosclerosis Suppl 2010 Severe HyperTG (> 10.0 mmol/L) n = 354 Moderate HyperTG (5.0 - 9.0 mmol/L) n = 487 All n = 354 LPLD only n = 28 15.8 ± 16.3 (0.008) 55.8 ± 56.9 (< 0.001) 361.4 ± 401.2 (< 0.001) Odd Ratio ± SE (p-value)
- 5. Postprandial Chylomicronemia Causes Pancreatitis in LPL Deficiency fat intake high levels of newly-formed chylomicrons in circulation post meal lodging of aggregates in capillaries - reduced blood flow - oedema - increased pressure acute pancreatitis
- 7. Glybera Administration Injections ultrasound or electro-physiologically guided Spinal anesthesia
- 10. TG in CT-AMT-011-01 over time
- 11. Yet, Persistent LPL expression after TG reversal Injected muscle Non-injected muscle 3x10 11 gc/kg (subj007) 3x10 11 gc/kg (subj008) (32 weeks after administration) (26 weeks after administration) CT AMT-010 Immunohistochemistry using specific anti-LPL antibody (5D2)
Notes de l'éditeur
- AMT-011 study
- The observed lack of effect within twoyears prior to therapy can be attributed to small numbers not burn out.