Final results from a defibrotide treatment-IND study for patients with hepatic veno-occlusive disease/sinusoidal obstruction syndrome.
Final results from a defibrotide treatment-IND study for patients with hepatic veno-occlusive disease/sinusoidal obstruction syndrome.
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DOI：
10.1111/bjh.15267
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发表时间：
2018-06
影响因子：
6.5
通讯作者：
Richardson P
中科院分区：
文献类型：
--
作者：
Kernan NA;Grupp S;Smith AR;Arai S;Triplett B;Antin JH;Lehmann L;Shore T;Ho VT;Bunin N;Iacobelli M;Liang W;Hume R;Tappe W;Soiffer R;Richardson P
肝静脉闭塞性疾病/静脉窦阻塞综合征（VOD/SOS）是造血干细胞移植（HSCT）调理和化疗的潜在危及生命的并发症。在美国，去纤肽被批准用于治疗HSCT后伴有肺部或肾脏功能障碍（即多器官功能障碍（MOD））的肝脏VOD/SOS，在欧盟，用于治疗年龄大于1个月的HSCT后患者的严重VOD/SOS。Defibrotide作为一种试验性药物可通过扩展准入治疗项目（T‐IND; NCT00628498）获得。在完成的T - IND中，Kaplan-Meier估计1000例HSCT后记录的去纤维肽治疗患者的Day +100生存率为58.9%[95%置信区间（CI）， 55.7 - 60.9%]。根据年龄和MOD状态分析100天生存率，并进行事后分析，根据移植类型、VOD/SOS发病时间（≤21天或>21天）和VOD/SOS诊断后开始去纤肽治疗时间确定100天生存率。儿童患者+100天生存率为67.9% (95% CI, 63.8 - 71.6%)，成人患者为47.1% （95% CI, 43.2 - 51.8%）。所有无MOD患者亚组的Day +100生存率均高于有MOD患者；更早的去纤维肽起始也与更高的Day +100生存率相关。在完成的T - IND研究中，去纤维肽的安全性与之前的报道相似。
Hepatic veno‐occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life‐threatening complication of haematopoietic stem cell transplant (HSCT) conditioning and chemotherapy. Defibrotide is approved for treatment of hepatic VOD/SOS with pulmonary or renal dysfunction [i.e., multi‐organ dysfunction (MOD)] after HSCT in the United States and severe VOD/SOS after HSCT in patients aged older than 1 month in the European Union. Defibrotide was available as an investigational drug by an expanded‐access treatment programme (T‐IND; NCT00628498). In the completed T‐IND, the Kaplan–Meier estimated Day +100 survival for 1000 patients with documented defibrotide treatment after HSCT was 58·9% [95% confidence interval (CI), 55·7–61·9%]. Day +100 survival was also analysed by age and MOD status, and post hoc analyses were performed to determine Day +100 survival by transplant type, timing of VOD/SOS onset (≤21 or >21 days) and timing of defibrotide treatment initiation after VOD/SOS diagnosis. Day +100 survival in paediatric patients was 67·9% (95% CI, 63·8–71·6%) and 47·1% (95% CI, 42·3–51·8%) in adults. All patient subgroups without MOD had higher Day +100 survival than those with MOD; earlier defibrotide initiation was also associated with higher Day +100 survival. The safety profile of defibrotide in the completed T‐IND study was similar to previous reports.
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影响因子：
4.8
作者：
Mohty M;Malard F;Abecassis M;Aerts E;Alaskar AS;Aljurf M;Arat M;Bader P;Baron F;Bazarbachi A;Blaise D;Ciceri F;Corbacioglu S;Dalle JH;Dignan F;Fukuda T;Huynh A;Masszi T;Michallet M;Nagler A;NiChonghaile M;Okamoto S;Pagliuca A;Peters C;Petersen FB;Richardson PG;Ruutu T;Savani BN;Wallhult E;Yakoub-Agha I;Duarte RF;Carreras E
通讯作者：
Carreras E
影响因子：
6.5
作者：
Richardson, Paul G.;Smith, Angela R.;Soiffer, Robert J.
通讯作者：
Soiffer, Robert J.
影响因子：
6.5
作者：
Dignan, Fiona L.;Wynn, Robert F.;Potter, Michael N.
通讯作者：
Potter, Michael N.
影响因子：
4.3
作者：
Richardson, Paul G.;Smith, Angela R.;Soiffer, Robert J.
通讯作者：
Soiffer, Robert J.
影响因子：
4.8
作者：
Corbacioglu S;Carreras E;Ansari M;Balduzzi A;Cesaro S;Dalle JH;Dignan F;Gibson B;Guengoer T;Gruhn B;Lankester A;Locatelli F;Pagliuca A;Peters C;Richardson PG;Schulz AS;Sedlacek P;Stein J;Sykora KW;Toporski J;Trigoso E;Vetteranta K;Wachowiak J;Wallhult E;Wynn R;Yaniv I;Yesilipek A;Mohty M;Bader P
通讯作者：
Bader P