Defitelio is a drug specifically used to treat specific diseases and plays an important role in the medical field. The following will introduce the drug properties, therapeutic applications, possible laboratory abnormalities, and prices of Defitelio in detail.

What is Defitelio?

Defibrotide is an original drug produced by Jazz Pharmaceuticals, an Irish biopharmaceutical company. It is a polydisperse mixture of single-stranded oligonucleotides that can stabilize and protect endothelial cells, inhibit thrombosis, and promote fibrinolysis, thereby improving coagulation disorders.

Drug properties

Defibrotide is an injection with a specification of 200mg/2.5mL, which is a transparent light yellow to brown solution. The drug needs to be stored at 20°C-25°C and can be transported at a temperature of 15°C-30°C. The validity period is 40 months.

Indications

Defibrotide sodium is mainly used to treat hepatic veno-occlusive disease (VOD), also known as sinusoidal occlusive syndrome (SOS), in adults and children with renal or pulmonary dysfunction after hematopoietic stem cell transplantation (HSCT). It is also approved for the treatment of patients with severe hepatic VOD.

Defibrotide sodium treats diseases through its multi-target mechanism of action, including inhibiting TNF-α-mediated endothelial cell apoptosis, increasing the expression of tissue plasminogen activator (t-PA) and thrombomodulin, and reducing the expression of von Willebrand factor (vWF) and plasminogen activator inhibitor-1 (PAI-1).

How effective is Defibrotide sodium in treating transplant-associated thrombotic microangiopathy (TA-TMA)?

Transplant-associated thrombotic microangiopathy (TA-TMA) is a serious complication after hematopoietic stem cell transplantation, with an incidence of 0.5% to 76% and a mortality rate of 50% to 90%.

Efficacy studies

Studies have shown that Defibrotide sodium can effectively treat TA-TMA through its anti-thrombotic, thrombolytic, anti-inflammatory and anti-ischemic effects. Low doses (6.25 mg/kg every 12 hours) of Defibrotide sodium have been shown to be safe and effective in the treatment of TA-TMA patients after allogeneic hematopoietic stem cell transplantation. The drug can protect endothelial cells from damage and reduce the expression of endothelial cell proinflammatory factors, thereby reducing the activity of plasma plasminogen activator inhibitor-1 (PAI-1) and increasing the function of tissue plasminogen activator (t-PA) in the body.

Clinical study cases

It has been reported that after treatment with Defibrotide sodium, the patient's platelet count rebounded, hemoglobin levels increased, and fragmented red blood cells in peripheral blood smears decreased, indicating that the condition was relieved.

Defibrotide sodium also has a significant effect in the prevention and treatment of hepatic veno-occlusive disease (VOD) after hematopoietic stem cell transplantation. It can improve the patient's survival rate and liver function, thereby indirectly reducing the risk of TA-TMA.

What laboratory abnormalities may be caused by Defibrotide sodium?

Defibrotide sodium may cause the following laboratory abnormalities when used:

Bleeding tendency and coagulation disorder

Defibrotide sodium may increase the risk of bleeding because it can increase the activity of extracorporeal plasmin. Patients may experience spontaneous bleeding due to coagulation disorders, such as skin bruises, nosebleeds, oral mucosal bleeding, etc. In severe cases, intracranial hemorrhage may occur, which is life-threatening.

Laboratory indicators may show prolonged coagulation time, decreased coagulation factor levels, and prolonged bleeding time.

Anemia and hypofibrinogenemia

Defibrotide sodium may cause anemia. Patients may experience symptoms such as fatigue, dizziness, and palpitations, mainly due to a decrease in the number of red blood cells or a decrease in oxygen-carrying capacity.

Hypofibrinogenemia refers to a decrease in the amount of fibrinogen in the blood, which may increase the risk of bleeding. Laboratory indicators may show a decrease in fibrinogen levels.

Allergic reactions

Although uncommon, Defibrotide sodium may cause allergic reactions, such as urticaria, itching, and shortness of breath. In severe cases, anaphylactic shock may occur.

Laboratory indicators may show eosinophilia, changes in immunoglobulin levels, etc.

Warm reminder: Before using Defibrotide sodium, the doctor will conduct a comprehensive assessment of the patient, including laboratory tests. During the use of Defibrotide sodium, the patient should undergo regular laboratory tests to promptly detect and deal with any possible laboratory abnormalities. The patient should follow the doctor's advice and avoid activities that may increase the risk of bleeding.