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
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 afte...
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Academician I.P. Pavlov First St. Petersburg State Medical University
2021-06-01
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| Series: | Учёные записки Санкт-Петербургского государственного медицинского университета им. Акад. И.П. Павлова |
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| Online Access: | https://www.sci-notes.ru/jour/article/view/773 |
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| author | 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 |
| author_facet | 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 |
| author_sort | M. M. Kanunnikov |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-862fc09720c946678588c33ff6d4150f |
| institution | Kabale University |
| issn | 1607-4181 2541-8807 |
| language | Russian |
| publishDate | 2021-06-01 |
| publisher | Academician I.P. Pavlov First St. Petersburg State Medical University |
| record_format | Article |
| series | Учёные записки Санкт-Петербургского государственного медицинского университета им. Акад. И.П. Павлова |
| spelling | doaj-art-862fc09720c946678588c33ff6d4150f2025-08-20T03:35:27ZrusAcademician I.P. Pavlov First St. Petersburg State Medical UniversityУчёные записки Санкт-Петербургского государственного медицинского университета им. Акад. И.П. Павлова1607-41812541-88072021-06-01274627010.24884/1607-4181-2020-27-4-62-70535Risk 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 transplantationM. M. Kanunnikov0J. Z. Rakhmanova1M. V. Barabanshikova2N. V. Levkovsky3A. I. Wafina4O. V. Paina5T. A. Bykova6Е. V. Semenova7О. V. Goloshapov8E. V. Morozova9I. S. Mоiseev10L. S. Zubarovskaya11Pavlov UniversityPavlov UniversityPavlov UniversityPavlov UniversityPavlov UniversityPavlov UniversityPavlov UniversityPavlov UniversityPavlov UniversityPavlov UniversityPavlov UniversityPavlov UniversityIntroduction. 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.https://www.sci-notes.ru/jour/article/view/773sinusoidal obstructive syndrome/veno-occlusive liver diseaseallogeneic hematopoietic stem cell transplantationdefibrotide |
| spellingShingle | 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 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 Учёные записки Санкт-Петербургского государственного медицинского университета им. Акад. И.П. Павлова sinusoidal obstructive syndrome/veno-occlusive liver disease allogeneic hematopoietic stem cell transplantation defibrotide |
| title | 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 |
| title_full | 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 |
| title_fullStr | 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 |
| title_full_unstemmed | 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 |
| title_short | 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 |
| title_sort | 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 |
| topic | sinusoidal obstructive syndrome/veno-occlusive liver disease allogeneic hematopoietic stem cell transplantation defibrotide |
| url | https://www.sci-notes.ru/jour/article/view/773 |
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