See the DrugPatentWatch profile for Defitelio
Defitelio, a prescription medication, is indicated for the treatment of severe veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), in pediatric patients who have undergone hematopoietic stem cell transplantation (HSCT) [1][2]. It is also approved for the treatment of VOD/SOS in adult and pediatric patients who have not undergone HSCT [1][2].
How does Defitelio work?
Defitelio's mechanism of action involves increasing the levels of certain enzymes, tissue plasminogen activator (t-PA) and prostacyclin, while decreasing plasminogen activator inhibitor-1 (PAI-1) [1]. This results in an antithrombotic and profibrinolytic effect, which helps to restore blood flow in the constricted hepatic veins [1]. By reducing the severity of VOD/SOS, Defitelio aims to improve survival rates in patients undergoing HSCT [3].
What are the benefits of Defitelio?
The primary benefit of Defitelio is its ability to treat severe VOD/SOS, a potentially life-threatening complication following HSCT [1][2][3]. Clinical studies have demonstrated that Defitelio can improve overall survival in these patients [3].
What are the potential side effects of Defitelio?
Common side effects of Defitelio can include hypotension, diarrhea, vomiting, nausea, and febrile neutropenia [1]. Less common but serious side effects may involve hypertension, hypersensitivity reactions, and issues related to the infusion itself [1].
How is Defitelio administered?
Defitelio is administered intravenously (IV) over a period of two hours [1]. Dosing is based on the patient's body weight [1].
What is the availability and cost of Defitelio?
Defitelio is available by prescription [2]. Information regarding the specific cost of Defitelio is not publicly detailed, but as a specialized medication for a severe condition, it is generally considered to be expensive [4]. Patient assistance programs may be available to help offset costs [2].
What is VOD/SOS and why is it serious?
Veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), is a serious condition that can occur after stem cell transplantation [3]. It involves damage to the small blood vessels in the liver, leading to blockages that impede blood flow [3]. This blockage can cause liver damage, enlargement, and abdominal pain, and in severe cases, it can be fatal [3].