Scholarlink

<p>We report a pooled analysis of real-world data of adult patients from two European registries of defibrotide use (DEFIFrance and EBMT Post-Authorization Safety Study [EBMT PASS]) to provide insights into the duration of defibrotide therapy to achieve resolution of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) symptoms by severity. Overall, 275 defibrotide-treated adults with VOD/SOS post-hematopoietic cell transplantation (HCT) were included (severe VOD/SOS, <i>n</i> = 215; nonsevere VOD/SOS, <i>n</i> = 60). Median time from the start of defibrotide treatment to VOD/SOS resolution was 18.5 and 16.5 days in patients with severe and nonsevere VOD/SOS, respectively. Among patients with VOD/SOS resolution, symptoms resolved after &gt;21 days in 31% (<i>n</i> = 40/128) and 32% (<i>n</i> = 14/44) with severe and nonsevere VOD/SOS, respectively. In all patients with VOD/SOS resolution independent of severity (<i>n</i> = 174), the Kaplan-Meier–estimated survival at Day 100 post-HCT was 82% (95% confidence interval [CI]: 75%, 87%); this was 78% (95% CI: 70%, 84%) and 91% (95% CI: 78%, 96%) in patients with severe and nonsevere VOD/SOS, respectively. Serious treatment-emergent adverse events of interest occurred in 31% of patients with VOD/SOS resolution. This pooled analysis supports the utility of defibrotide treatment for VOD/SOS and highlights that treatment may often need to be continued beyond 21 days to achieve resolution.</p>

错误：搜索内容不能为空，请输入英文关键词
错误：关键词超出字数限制，请精简
高级检索

Time to resolution of veno-occlusive disease/sinusoidal obstruction syndrome post-HCT in defibrotide-treated adults from two European registries

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2026年6月20日

查看全文
  • Mohamad Mohty,
  • Régis Peffault de Latour,
  • Myriam Labopin,
  • Vian Amber,
  • Amélie Petitprez,
  • Deborah Gutierrez,
  • Nalina Dronamraju,
  • Hélène Labussière-Wallet,
  • Didier Blaise

摘要

We report a pooled analysis of real-world data of adult patients from two European registries of defibrotide use (DEFIFrance and EBMT Post-Authorization Safety Study [EBMT PASS]) to provide insights into the duration of defibrotide therapy to achieve resolution of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) symptoms by severity. Overall, 275 defibrotide-treated adults with VOD/SOS post-hematopoietic cell transplantation (HCT) were included (severe VOD/SOS, n = 215; nonsevere VOD/SOS, n = 60). Median time from the start of defibrotide treatment to VOD/SOS resolution was 18.5 and 16.5 days in patients with severe and nonsevere VOD/SOS, respectively. Among patients with VOD/SOS resolution, symptoms resolved after >21 days in 31% (n = 40/128) and 32% (n = 14/44) with severe and nonsevere VOD/SOS, respectively. In all patients with VOD/SOS resolution independent of severity (n = 174), the Kaplan-Meier–estimated survival at Day 100 post-HCT was 82% (95% confidence interval [CI]: 75%, 87%); this was 78% (95% CI: 70%, 84%) and 91% (95% CI: 78%, 96%) in patients with severe and nonsevere VOD/SOS, respectively. Serious treatment-emergent adverse events of interest occurred in 31% of patients with VOD/SOS resolution. This pooled analysis supports the utility of defibrotide treatment for VOD/SOS and highlights that treatment may often need to be continued beyond 21 days to achieve resolution.