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4CPS-227 Real world evidence of the use of defibrotide for prophylaxis of veno-occlusive disease after post-haematopoietic stem-cell transplantation in children
  1. M Bettio1,
  2. D Mengato1,
  3. A Francavilla2,
  4. F Venturini1
  1. 1Padova University Hospital, Pharmacy Unit, Padova, Italy
  2. 2University of Padova, Unit of Biostatistics- Epidemiology- And Public Health- Department of Cardiac- Thoracic- And Vascular Sciences, Padova, Italy


Background and Importance Hepatic veno-occlusive disease (VOD) is a life-threatening condition caused by the obstruction of liver sinusoids.

Since 2014, in Italy the standard of care for the management of VOD is represented by defibrotide. Recent evidence suggested that defibrotide could help preventing the onset of hepatic VOD when allogeneic haematopoietic stem cell transplantation is needed. On June 2022, however, a ‘direct health professional communication’ issued by the European Medicines Agency (EMA) invoked not to use defibrotide anymore for VOD prophylaxis due to lack of effectiveness.

Aim and Objectives The aim of this work is to explore the difference in the incidence of VODs at 30 days in 2 groups of children, with and without prophylaxis therapy with defibrotide before undergoing haematopoietic stem cell transplantation.

Material and Methods A single-centre, retrospective study was conducted at a University Hospital. All data were collected from electronic health records. These data were cross-checked with data from an integrated analytics application (Qlikview®, QlikTech International AB, King of Prussia, USA).

All paediatric patients (age <18 years) undergoing haematopoietic stem cell transplantation for onco-haematological diseases and considered at high-risk for developing VOD were enrolled. We observed an initial group, called the ‘intervention’ group, consisting of patients who had received the drug, compared with a ‘historical’ control group of patients with similar baseline characteristics but who did not have access to defibrotide.

Results Between 2020 and 2022, data were collected from 27 patients. The baseline characteristics of the two group were similar regarding of age (9 years old for both groups), gender and onco-haematological disease, all showing no statistically significant differences. In terms of outcome, we witnessed only one episode of VOD, in the treatment group (1 of 11 patients, 9%), at 30 days after transplantation. No episodes were documented in the controls.

Conclusion and Relevance According to the recent statement made by EMA, our data – although not definitive – show that proportion of VOD in children undergoing blood stem transplantation in patients who received a prophylaxis treatment with defibrotide was comparable with the one in children where no prophylaxis strategy has been adopted.

Conflict of Interest No conflict of interest

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