| Abstract [eng] |
Sinusoidal obstruction syndrome is a rare life-threatening complication of chemotherapy, mostly associated with hematopoietic stem cell transplantation. Defibrotide is the only drug approved for the treatment of sinusoidal obstruction syndrome, but due to the rarity of the disease and lack of prospective studies, the evidence of its efficacy remains uncertain. Objectives. Systematically assess the effectiveness of the use of defibrotide in children for prophylaxis and treatment of sinusoidal obstruction syndrome in clinical studies, published within the last 10 years. Methods. Literature search in PubMed and Google Scholar was performed between October 15th and December 1st of 2023, according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. We used search keywords: “defibrotide”, “sinusoidal obstruction syndrome, “veno-occlusive disease”, “pediatric”, “children”. Case reports, reviews, meta-analyses, studies published before 2013 and studies with no children were excluded. Results. 10 retrospective and 5 retrospective studies met the inclusion criteria. 7655 patients were included in the studies, with 3121 (40,7%) children. 8 studies evaluated only treatment with defibrotide, 4 studies described only prophylaxis with defibrotide, and 3 studies - both prophylaxis and treatment. 3 studies supported the benefit of sinusoidal obstruction syndrome prophylaxis with defibrotide while 3 studies concluded that the prophylaxis was not useful. All 11 studies that analyzed the efficacy of defibrotide treatment supported the benefit of it. Conclusions. This systematic review concludes that treatment of sinusoidal obstruction syndrome with defibrotide in children is effective. However, there is not enough evidence to support sinusoidal obstruction syndrome prophylaxis with defibrotide. |
