Учёные записки Санкт-Петербургского государственного медицинского университета им. Акад. И.П. Павлова (Jun 2021)

Risk factors analysis and defibrotide efficacy in the treatment of children and adults with sinusoidal obstructive syndrome/veno-occlusive liver disease after allogeneic hematopoietic stem cell transplantation

  • M. M. Kanunnikov,
  • J. Z. Rakhmanova,
  • M. V. Barabanshikova,
  • N. V. Levkovsky,
  • A. I. Wafina,
  • O. V. Paina,
  • T. A. Bykova,
  • Е. V. Semenova,
  • О. V. Goloshapov,
  • E. V. Morozova,
  • I. S. Mоiseev,
  • L. S. Zubarovskaya

DOI
https://doi.org/10.24884/1607-4181-2020-27-4-62-70
Journal volume & issue
Vol. 27, no. 4
pp. 62 – 70

Abstract

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Introduction. Sinusoidal obstructive syndrome (SOS)/veno-occlusive liver disease (VOD) is one of the most dangerous complication of allogeneic hematopoietic stem cell transplantation (alloHSCT).The objective of our study was to analyze risk factors associated with SOS/VOD in children and adults after alloHSCT.Methods and materials. The study included 76 patients who were diagnosed with the development of SOS/VOD after alloHSCT performed in Raisa Gorbacheva Memorial Research Institute for Pediatric Oncology, Hematology and Transplantation between 2001 and 2019. 25 patients (32.9 %) were younger than 18 years, 51 patients (68.1 %) — 18 years and older. Among them, 17 patients were treated with defibrotide at a dose of 25 mg/kg per day, median therapy duration — 21 day.Results. One-year overall survival (OS) was significantly higher when partial and complete response to the therapy was achieved — 45 %, than when there was no response — 0 % in the general group (p = 0.001). According to the results of multivariate analysis of unrelated alloHSCT HR 2.040 (95 %CI 1.112-3.744, р = 0.021), acute GVHD HR 0.496 (95 % CI 0.272-0.903, р = 0.022), moderate/severe SOS/VOD HR 2.423 (95 % CI 1.298-4.524, p = 0.005) statistically significantly influenced the 1-year OS. Defibrotide and accompanying therapy did not significantly influence the 1-year OS in children and adults (n=76) - 53 % and 54 % (p=0.86), respectively. In a multivariate analysis. unrelated alloHSCT HR 8.172 (95 %CI 2.176-30.696, р=0.002) and moderate and severe SOS/VOD HR 9.077 (95 % CI 2.425-33.978, р=0.001) significantly influenced the 1-year OS in the pediatric group.Conclusion. The understanding of risk factors of adverse prognosis in patients SOS/VOD facilitates selection of patients who will benefit the most from therapy with defibrotide. Early administration of defibrotide in the course of VOD/SOS is crucial to achieve response.

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