去纤苷治疗的试验效果怎么样

Trial efficacy data of defibrotide treatment show that defibrotide is a safe and effective drug for the prevention and treatment of hepatic veno-occlusive disease (HVOD) after HSCT (hematopoietic stem cell transplantation). Defibrotide helps restore normal function of the vascular endothelium by preventing platelet aggregation and fibrin formation, thereby improving blood flow. In addition, it can also reduce inflammatory reactions and protect vascular endothelium from damage.

Trial efficacy data of defibrotide treatment

Hepatic veno-occlusive disease (VOD), also known as sinus obstruction syndrome (SOS), is a potentially life-threatening complication of hematopoietic stem cell transplantation (HSCT). Untreated hepatic VOD/SOS is associated with multiorgan failure (MOF) and has a mortality rate of >80%. In a phase 2 study, defibrotide showed good efficacy in the treatment of liver VOD/SOS with MOF.

This Phase 3 study (NCT00358501) investigated the safety and efficacy of defibrotide in patients with established hepatic VOD/SOS and advanced MOF. Thirty-two historical controls were screened from the medical records of 6867 hematopoietic stem cell transplant patients by a blinded independent reviewer, and patients taking 25 mg/kg daily of defibrotide (n = 102) were compared with the 32 historical controls. Baseline characteristics were well balanced between groups. The primary endpoint was survival at day +100 after hematopoietic stem cell transplantation.

Observations

Survival was 38.2% in the defibrotide group and 25% in the control group (estimated difference, 23%; 95.1% confidence interval [CI], 5.2-40.8; P = .0109, propensity-adjusted analysis). The observed complete response (CR) rate at day +100 was 25.5% with defibrotide and 12.5% ​​with control (similar method, difference, 19%; 95.1% confidence interval [CI], 3.5-34.6; P = .0160).

Defibrotide was generally well tolerated and its toxicity was controllable. Associated (AEs) included bleeding or hypotension; the incidence of common bleeding AEs, including alveolar hemorrhage [11.8% and 15.6%] and gastrointestinal bleeding [7.8% and 9.4%], was similar between the defibrotide and control groups, respectively. Defibrotide can significantly improve 100-day survival rate and CR rate.

Defibrinoside usage and dosage

The recommended dose for adult and pediatric patients is 6.25 mg/kg given every 6 hours as a 2-hour intravenous infusion. 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.

Where to buy defibrinoside

As of October 2023, defibrotide has not yet been launched in mainland China, and defibrotide is not yet available in hospital pharmacies in the mainland. Patients in need can purchase it in areas where it is already on the market, or they can obtain it through domestic professional overseas medical service organizations (such as Medical Companion Travel). The medicine can be mailed to their home. It is guaranteed to be genuine and reduces the financial burden. It is affordable and more cost-effective. However, the price is subject to various factors and is not fixed. It is recommended to consult the customer service staff for specific costs and acquisition procedures. Obtaining the medicine is guaranteed.

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References

Richardson PG, Riches ML, Kernan NA, Brochstein JA, Mineishi S, Termuhlen AM, Arai S, Grupp SA, Guinan EC, Martin PL, Steinbach G, Krishnan A, Nemecek ER, Giralt S, Rodriguez T, Duerst R, Doyle J, Antin JH, Smith A, Lehmann L, Champlin R, Gillio A, Bajwa R, D'Agostino RB Sr, Massaro J, Warren D, Miloslavsky M, Hume RL, Iacobelli M, Nejadnik B, Hannah AL, Soiffer RJ. Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure. Blood. 2016 Mar 31;127(13):1656-65. doi: 10.1182/blood-2015-10-676924. Epub 2016 Jan 29. PMID: 26825712; PMCID: PMC4817309.