不看不知道！去纤苷的适应症居然有这么多！

Defitelio was first developed by Italy's Gentium Pharmaceuticals and was approved for marketing in the EU in October 2013. It was approved for marketing in the United States by the U.S. Food and Drug Administration (FDA) on March 30, 2016, under the trade name Defitelio. It is the first drug approved by the US FDA to treat severe hepatic veno-occlusive disease. It is used for adult or pediatric patients with hepatic venous obstruction (VOD) who have undergone blood or bone marrow hematopoietic stem cell transplantation (HSCT) and are accompanied by renal or lung function abnormalities. The drug has also received "orphan drug" designation.

Defibrotide is an adenosine receptor agonist with multiple effects. The adenosine A1/A2 receptors of endothelial cells are involved in the regulation of endothelial cells and the response of endothelial cells to injury. Defibrotide can act on these receptors to produce a variety of downstream effects. Defibrotide can also reduce the expression of cell adhesion molecules on the surface of endothelial cells, thereby reducing the adhesion of leukocytes to vascular endothelial cells and reducing the inflammatory damage of endothelial cells.

The recommended dose of defibrotide for adult and pediatric patients is 6.25 mg/kg given as a 2-hour intravenous infusion every 6 hours. The dose should be based on the patient's baseline weight, which is defined as the patient's weight before the HSCT (hematopoietic stem cell transplant) preparation protocol. Defibrotide was administered for a minimum of 21 days. If signs and symptoms of hepatic veno-occlusive disease have not resolved after 21 days, continue defibrotide until resolution of hepatic veno-occlusive disease or for a maximum of 60 days. Defibrotide must be diluted prior to infusion. Do not coadminister defibrotide with other intravenous medications at the same time and in the same intravenous line.

The most common side effects of defibrotide therapy (incidence ≥10% and independently causal) are hypotension, diarrhea, vomiting, nausea, and epistaxis. The incidence of grade 3-4 reactions related to defibrotide was 7%, mainly hypotension, pulmonary and gastrointestinal bleeding, and abdominal cramps. Only 4% of patients discontinued treatment due to side effects. In other words, the side effects of defibrotide are usually mild, and patients do not need to worry too much.

Note: The above information comes from the Internet and is compiled and edited by Medical Companion Travel (please correct me if there are any errors or omissions). It is only to provide information on the latest drugs on the market in the world and help Chinese patients understand the latest international new drug trends. It is only for internal discussion among medical staff and does not serve as any basis for medication. For specific medication guidelines, please consult the attending physician.