Advances in Clinical and Experimental Medicine

Title abbreviation: Adv Clin Exp Med
5-Year IF – 2.0, IF – 1.9, JCI (2024) – 0.43
Scopus CiteScore – 4.3
Q1 in SJR 2024, SJR score – 0.598, H-index: 49 (SJR)
IC – 171.00; MNiSW – 70 pts
Initial editorial assessment and first decision within 24 h

ISSN 1899–5276 (print), ISSN 2451-2680 (online)
Periodicity – monthly

Download original text (EN)

Advances in Clinical and Experimental Medicine

2020, vol. 29, nr 3, March, p. 339–344

doi: 10.17219/acem/115070

Publication type: original article

Language: English

License: Creative Commons Attribution 3.0 Unported (CC BY 3.0)

Download citation:

  • BIBTEX (JabRef, Mendeley)
  • RIS (Papers, Reference Manager, RefWorks, Zotero)

Veno-occlusive disease in children and adolescents after hematopoietic stem cell transplantation: Did the Modified Seattle Criteria fit the characteristics of pediatric population?

Zofia Szmit1,A,B,C,D,E,F, Ewa Gorczyńska1,A,C,D,E,F, Monika Mielcarek-Siedziuk1,A,E,F, Marek Ussowicz1,A,E,F, Joanna Owoc-Lempach1,A,B,E,F, Krzysztof Kałwak1,A,C,D,E,F

1 Department of Pediatric Hematology/Oncology and Bone Marrow Transplantation, Wroclaw Medical University Supraregional Center of Pediatric Oncology “Cape of Hope”, Poland

Abstract

Background. Hepatic veno-occlusive disease (VOD) is a life-threatening complication following hematopoietic stem cell transplantation (HSCT) and associated with a high mortality rate. Therefore, accurate and immediate diagnosis is crucial for implementing appropriate treatment.
Objectives. In our single-center retrospective study, we assessed the accuracy of the Modified Seattle Criteria in children and adolescents undergoing HSCT, and compared them to the diagnostic criteria recently established by the European Society for Blood and Marrow Transplantation (EBMT).
Material and Methods. Retrospective analysis of medical records of 951 HSCT procedures performed in 850 children and young adults in the years 2001–2015 in the Department of Pediatric Hematology, Oncology and Bone Marrow Transplantation of Wroclaw Medical University Supraregional Center of Pediatric Oncology “Cape of Hope” in Wrocław, Poland.
Results. Among the 850 children, 48 were diagnosed with VOD according to the Modified Seattle Criteria (5.05%). Thirteen patients (27%) developed VOD later than within 20 days after transplantation, as required in the diagnostic criteria. Five of the 6 patients who died from VOD were diagnosed with late-onset VOD. Using the categories of symptoms described in the Modified Seattle Criteria, hepatomegaly and weight gain were the most common symptoms in the analyzed cohort (81.25% and 68.75%). Fourteen patients (29%) never demonstrated elevated plasma bilirubin level (>2 mg/dL), as suggested in the Modified Seattle Criteria. Twenty-nine patients (64%) had increased platelet consumption requiring daily transfusions. Only 5 patients with decreased plasma antithrombin III (ATIII) activity level (<80%) on the day of HSCT developed VOD despite supplementation of ATIII.
Conclusion. The Modified Seattle Criteria seemed to not meet the special needs of the pediatric population. The new diagnostic criteria proposed by the EBMT appear to be more adequately tailored to the pediatric population and may significantly change the conception of VOD in the future. The surprisingly low incidence of VOD in our cohort may suggest a beneficial role of monitoring and early supplementation of ATIII.

Key words

hematopoietic stem cell transplantation, pediatric, veno-occlusive disease

References (33)

  1. McDonald GB, Hinds MS, Fisher LD, et al. Veno-occlusive disease of the liver and multiorgan failure after bone marrow transplantation: A cohort study of 355 patients. Ann Intern Med. 1993;118(4):255–267.
  2. Tasu JP, Rocher L, Péletier G, et al. Hepatic venous pressure gradients measured by duplex ultrasound. Clin Radiol. 2002;57(8):746–752. doi:10.1053/crad.2002.0951
  3. Brown P. Doppler sonography: A noninvasive method for evaluation of hepatic venocclusive disease. AJR Am J Roentgenol. 1990;154(4):712–724.
  4. Carreras E, Díaz-Beyá M, Rosiñol L, Martínez C, Fernández-Avilés F, Rovira M. The incidence of veno-occlusive disease following allogeneic hematopoietic stem cell transplantation has diminished and the outcome improved over the last decade. Biol Blood Marrow ­Transplant. 2011;17(11):1713–1720. doi:10.1016/j.bbmt.2011.06.006
  5. Bearman SI, Anderson GL, Mori M, Hinds MS, Shulman HM, McDonald GB. Venoocclusive disease of the liver: Development of a model for predicting fatal outcome after marrow transplantation. J Clin Oncol. 1993;11(9):1729–1736.
  6. Jones RJ, Lee KS, Beschorner WE et al. Venoocclusive disease of the liver following bone marrow transplantation. Transplantation. 1987;44(6):778–783.
  7. Coppell J, Richardson PG, Soiffer R, et al. Hepatic veno-occlusive disease following stem cell transplantation: Incidence, clinical course, and outcome. Biol Blood Marrow Transplant. 2010;16(2):157–168. doi:10.1016/j.bbmt.2009.08.024
  8. Shulman HM, Hinterberger W. Hepatic veno-occlusive disease: Liver toxicity syndrome after bone marrow transplantation. Bone ­Marrow Transplant. 1992;10(3):197–214.
  9. Corbacioglu S, Carreras E, Ansari M, et al. Diagnosis and severity criteria for sinusoidal obstruction syndrome/veno-occlusive disease in pediatric patients: A new classification from the European Society for Blood and Marrow Transplantation. Bone Marrow Tranplant. 2017;53(2):138–145. doi:10.1038/bmt.2017.161
  10. Mohty M, Malard F, Abecassis M, et al. Revised diagnosis and severity criteria for sinusoidal obstruction syndrome/veno-occlusive disease in adult patients: A new classification from the European Society for Blood and Marrow Transplantation. Bone Marrow Transplant. 2016;51(7):906–912. doi:10.1038/bmt.2016.130
  11. Mohty M, Malard F, Abecassis M, et al. Sinusoidal obstruction syndrome/veno-occlusive disease: Current situation and perspectives. A position statement from the European Society for Blood and Marrow Transplantation (EBMT). Bone Marrow Transplant. 2015;50(6):781–789. doi:10.1038/bmt.2015.52
  12. Hasegawa S, Horibe K, Kawabe T, et al. Veno-occlusive disease of the liver after allogeneic bone marrow transplantation in children with hematologic malignancies: Incidence, onset time and risk factors. Bone Marrow Transplant. 1998;22(12):1191–1197.
  13. Maximova N, Ferrara G, Minute M, et al. Experience from a single paediatric transplant centre with identification of some protective and risk factors concerning the development of hepatic veno-occlusive disease in children after allogeneic hematopoietic stem cell transplant. Int J Hematol. 2014;99(6):766–772. doi:10.1007/s12185-014-1578-y
  14. Cheuk DKL, Wang P, Lee TL, et al. Risk factors and mortality predictors of hepatic veno-occlusive disease after pediatric hematopoietic stem cell transplantation. Bone Marrow Transplant. 2007;40(10):935–944. doi:10.1038/sj.bmt.1705835
  15. Lee SH, Yoo KH, Sung KW, et al. Hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation: Incidence, risk factors, and outcome. Bone Marrow Transplant. 2010;45(8):1287–1293. doi:10.1038/bmt.2009.349
  16. Corbacioglu S, Greil J, Peters C, et al. Defibrotide in the treatment of children with veno-occlusive disease (VOD): A retrospective multicentre study demonstrates therapeutic efficacy upon early intervention. Bone Marrow Transplant. 2004;33(2):189–195. doi:10.1038/sj.bmt.1704494
  17. Corbacioglu S, Carreras E, Mohty M, et al. Defibrotide for the treatment of hepatic veno-occlusive disease: Final results from the International Compassionate Use Program. Biol Blood Marrow Transplant. 2016;22(10):1874–1882. doi:10.1016/j.bbmt.2016.07.001
  18. Dignan F, Gujral D, Ethell M, et al. Prophylactic defibrotide in allogeneic stem cell transplantation: Minimal morbidity and zero mortality from veno-occlusive disease. Bone Marrow Transplant. 2007;40(1):79–82. doi:10.1038/sj.bmt.1705696
  19. Richardson PG, Ho VT, Giralt S, et al. Safety and efficacy of defibrotide for the treatment of severe hepatic veno-occlusive disease. Ther Adv Hematol. 2012;3(4):253–265. doi:10.1177/2040620712441943
  20. Richardson PG, Riches ML, Kernan NA, et al. Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure. Blood. 2016;127(13):1656–1666. doi:10.1182/blood-2015-10-676924
  21. Forrest DL, Thompson K, Dorcas VG, Couban SH, Pierce R. Low molecular weight heparin for the prevention of hepatic veno-occlusive disease (VOD) after hematopoietic stem cell transplantation: A prospective phase II study. Bone Marrow Transplant. 2003;31(12):1143–1149. doi:10.1038/sj.bmt.1704087
  22. Imran H, Tleyjeh IM, Zirakzadeh A, Rodriguez V, Khan SP. Use of prophylactic anticoagulation and the risk of hepatic veno-occlusive disease in patients undergoing hematopoietic stem cell transplantation: A systematic review and meta-analysis. Bone Marrow Transplant. 2006;37(12):677–686. doi:10.1038/sj.bmt.1705297
  23. Park SH, Lee MH, Lee H, et al. A randomized trial of heparin plus ursodiol vs heparin alone to prevent hepatic veno-occlusive disease after hematopoietic stem cell transplantation. Bone Marrow Transplant. 2002;29(2):137–143. doi:10.1038/sj.bmt.1703342
  24. Haussmann U, Fischer J, Eber S, Scherer F, Seger R, Gungor T. Hepatic veno-occlusive disease in pediatric stem cell transplantation: Impact of pre-emptive antithrombin III replacement and combined antithrombin III/defibrotide therapy. Haematologica. 2006;(6):795–800. doi:10.3324/haematol.13619
  25. Peres E, Kintzel P, Dansey R, et al. Early intervention with antithrombin III therapy to prevent progression of hepatic venoocclusive disease. Blood Coagul Fibrinolysis. 2008;19(3):203–207. doi:10.1097/MBC.0b013e3282f2b5d9.
  26. Myers KC, Dandoy C, El-Bietar J, Davies SM, Jodele S. Veno-occlusive disease of the liver in the absence of elevation in bilirubin in pediatric patients after hematopoietic stem cell transplantation. Biol Blood Marrow Transpl. 2015;21(2):379–381. doi:10.1016/j.bbmt.2014.09.026
  27. Naples JC, Skeens MA, Auletta J, et al. Anicteric veno-occlusive disease after hematopoietic stem cell transplantation in children. Bone Marrow Transplant. 2016;51(1):135–137. doi:10.1038/bmt.2015.208
  28. Reiss U, Cowan M, McMillan A, Horn B. Hepatic venoocclusive disease in blood and bone marrow transplantation in children and young adults: Cohort of 241 patients. J Pediatr Hematol Oncol. 2002;24(9):746–750.
  29. Barker CC, Butzner JD, Anderson RA, Brant R, Sauve RS. Incidence, survival and risk factors for the development of veno-occlusive disease in pediatric hematopoietic stem cell transplant recipients. Bone Marrow Transplant. 2003;32(1):79–87. doi:10.1038/sj.bmt.1704069
  30. Jevtic D, Zecevic Z, Veljkovic D, Dopsaj V, Radojicic Z, Elezovic I. Veno-occlusive disease in pediatric patients after hematopoietic stem cell transplantation: Relevance of activated coagulation and fibrinolysis markers and natural anticoagulants. J Pediatr Hematol Oncol. 2011;33(3):227–234. doi:10.1097/MPH.0b013e31820539fd
  31. Cacchione A, LeMaitre A, Couanet DV, et al. Risk factors for hepatic veno-occlusive disease: A retrospective unicentric study in 116 children autografted after a high-dose BU-thiotepa regimen. Bone Marrow Transplant. 2008;42(7):449–454. doi:10.1038/bmt.2008.186
  32. Horn B, Reiss U, Matthay K, McMillan A, Cowan M. Veno-occlusive disease of the liver in children with solid tumors undergoing autologous hematopoietic progenitor cell transplantation: A high incidence in patients with neuroblastoma. Bone Marrow Transplant. 2002;29(5):409–415. doi:10.1038/sj.bmt.1703393
  33. Toh HC, McAfee SL, Sackstein R, Cox BF, Colby C, Spitzer TR. Late onset veno-occlusive disease following high-dose chemotherapy and stem cell transplantation. Bone Marrow Transplant. 1999;24(8):891–895.
© Wroclaw Medical University