Dit valt enorm tegen; Glybera is een hele dure zeer nare behandeling en in het gunstigste geval halveren de ziekenhuisdagen (143 naar 75 en van 116 naar 107).
Ik denk niet dat Glybera vergoed gaat worden door de overheid.
Dit middel zal geen succes worden en goedkeuring in de USA kan men wel vergeten.
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Specifically, the data show that these patients have:
a range of approximately 40-50% lower post-treatment documented pancreatitis events and abdominal pain events consistent with pancreatitis;
no severe pancreatitis up to six years post-treatment; and
an approximately 50% lower post-treatment hospitalization rate and number of days spent in the hospital for documented pancreatitis, including only one ICU stay that occurred following treatment.
Glybera was generally well tolerated, with most adverse events such as myalgia, muscular stiffness, and burning sensation from injection site reactions occurring around the time of administration. No long-term safety concerns have been identified.
"It is encouraging to see that Glybera has been associated with reduced incidence of severe pancreatitis in a significant portion of treated patients and with less time spent in intensive care units," said Daniel Gaudet, MD, Ph.D., Associate Professor of Medicine, Université de Montréal and primary investigator. “Glybera is the first approved gene therapy in the western world and the way that this technology will be transferred to healthcare systems might influence the future of gene therapy for other diseases and have an important impact on the future of LPLD management.”
The aim of the analysis was to measure incidence and severity of acute pancreatitis episodes and utilization of health resources (hospitalizations, ICU stays, days-in-hospital) over an extended period up to six years post-treatment. An independent adjudication board of three experts blinded to patient identification and pre- or post-treatment period information performed a retrospective review of individual hospital records and categorized each abdominal pain event that lead to hospitalization before and after treatment with Glybera. This six-year analysis represents an extension of the three-year retrospective review that formed a part of the data package upon which the European Commission approved Glybera in October 2012 under exceptional circumstances for the treatment of a subset of patients with LPLD. The data confirm the trend exhibited in the first case note review.
Glybera is indicated for the treatment of adult patients diagnosed with familial LPLD confirmed by genetic testing and suffering from severe or multiple pancreatitis attacks despite dietary fat restrictions. LPLD results in hyper-chylomicronemia, or dramatic and potentially life-threatening increases in the level of large fat-carrying particles, called chylomicrons, in the blood after eating. In many cases, LPLD and the associated elevated levels of chylomicrons can cause acute and potentially life-threatening inflammation of the pancreas, known as pancreatitis, thus leading to frequent hospitalizations. Recurrent pancreatitis can lead to chronic abdominal pain, pancreatic insufficiency, which is an inability to properly digest food due to a lack of digestive enzymes made by the pancreas, and diabetes. There is no other approved treatment for LPLD.