Defitelio is an important therapeutic drug that has shown significant clinical value in specific medical fields. Its unique pharmacological mechanism and clear indications make it a key choice for the treatment of certain serious diseases.
Indications of Defitelio
Defitelio is mainly used to treat hepatic veno-occlusive disease (VOD), also known as sinusoidal venous occlusive syndrome (SOS), after hematopoietic stem cell transplantation (HSCT). This disease causes liver dysfunction due to obstruction of small veins in the liver, which can be life-threatening in severe cases. Defitelio provides patients with an effective treatment option through a multi-target mechanism of action.
Core treatment needs for hepatic veno-occlusive disease
VOD is common in hematopoietic stem cell transplant patients undergoing high-intensity chemotherapy or radiotherapy, and its pathological characteristics are endothelial damage and fibrin deposition of small veins in the liver. Clinical manifestations include jaundice, ascites and hepatomegaly, which may lead to multiple organ failure if not intervened in time. Defitelio significantly improves the survival rate of patients by stabilizing endothelial cell function, inhibiting thrombosis and promoting fibrinolysis.
Clinical validation and efficacy advantages
Multiple clinical trials have shown that the survival rate of the Defibrotide treatment group is significantly higher than that of the historical control group. In a key study, the 100-day survival rate of VOD patients treated with Defibrotide can reach 38%-45%, while traditional supportive treatment is only 25%-30%. It is well tolerated and has controllable adverse reactions, further consolidating its first-line position in VOD treatment.
Based on existing evidence, Defibrotide has become the only specific drug approved by European and American authorities for VOD after HSCT, filling the treatment gap in this field.
Applicable population of Defibrotide
The scope of application of Defibrotide covers adults and children, but the specific application needs to be combined with the individual characteristics of the patient.
Dosage consistency between adult and pediatric patients
The recommended dose of Defibrotide is 6.25 mg/kg every 6 hours, which is suitable for adult and pediatric patients. The dose calculation is based on the patient's baseline weight before hematopoietic stem cell transplantation preparation to avoid medication deviations due to weight fluctuations during treatment. The pharmacokinetic characteristics of the pediatric population are similar to those of adults, and clinical trial data support its safety and efficacy in pediatric patients.
Treatment conditions and contraindication management
The use of Defibrotide requires specific conditions: the patient must be hemodynamically stable and have no severe bleeding tendency. Anticoagulants or fibrinolytic drugs must be discontinued before treatment to avoid the risk of bleeding. Defibrotide is prohibited for patients with active bleeding or who need polyvasopressors to maintain blood pressure. Strict indication screening is the core prerequisite for ensuring the treatment effect.
Through standardized medication processes, Defibrotide can provide clear survival benefits for eligible patients while minimizing treatment risks.
Special population medication for Defibrotide
The use of Defibrotide in pregnant women, lactating women and elderly patients must follow specific guidelines.
Risk control for pregnant and lactating women
Animal experiments have shown that Defibrotide may affect fetal development, and pregnant women should only consider using it when the potential benefits outweigh the risks. Lactating women need to suspend breastfeeding to avoid the risk of bleeding in infants caused by the drug being transmitted through breast milk. During treatment, the health status of mothers and infants needs to be closely monitored and the treatment plan should be adjusted in a timely manner.
Individualized medication strategy for elderly patients
There is currently insufficient data to support the difference in efficacy between elderly patients and younger people, but attention should be paid to their liver and kidney function and concomitant medication. Elderly people often have multi-system diseases. When using Defibrotide, coagulation function monitoring should be strengthened, and the duration of treatment should be adjusted according to clinical response. Individualized evaluation is the key to ensuring safe medication for elderly patients.
For medication management of special populations, precise plans should be formulated based on the specific conditions of the patients to minimize risks and maximize efficacy.
