Zmeny v diagnostike a liečbe venookluzívnej choroby pečene u detí

Czech version

Authors: M. Füssiová ¹; P. Švec ¹; J. Horáková ¹; A. Kolenova ¹; P. Sedláček ²; P. Rohoň ³; I. Boďová ¹; J. Adamčáková ¹; T. Sýkora ¹; V. Dobšinská ¹; M. Pozdechová ¹; D. Dóczyová ¹; S. Vargová ¹
Authors‘ workplace: Transplantačná jednotka kostnej drene, Klinika detskej hematológie a onkológie, NÚDCH, Bratislava, Slovensko ¹; Klinika dětské hematologie a onkologie 2. LF UK a FN Motol, Praha, ČR ²; Klinika hematológie a transfuziológie LF UK a UN, Bratislava, Slovensko ³
Published in: Transfuze Hematol. dnes,29, 2023, No. 2, p. 124-131.
Category: Review/Educational Papers
doi: https://doi.org/10.48095/cctahd2023prolekare.cz3

Overview

Sinusoidal obstruction syndrome of the liver or hepatic veno-occlusive disease (HVOD) is a serious and potentially life-threatening complication of hematopoietic stem cell transplantation. Expert analyses show differences in HVOD between adult and paediatric patients (incidence, genetic predisposition, prevention, risk factors, clinical course, treatment, survival and mortality). The new criteria for the diagnosis and classification of the disease recommended by the European Society for Blood and Marrow Transplantation (EBMT) are a key factor in the management of HVOD in childhood. These international recommendations promote early diagnosis and early defibrotide treatment of the clinical complication.

Keywords:

hematopoietic stem cell transplantation – veno-occlusive disease of the liver – new paediatric EBMT criteria for diagnostics and classification – defibrotide

Sources

1. 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 Transplant. 2018; 53 (2): 138–145.

2. Bonifazi F, Barbato F, Ravaioli F, et al. Diagnosis and treatment of VOD/SOS after allogeneic hematopoietic stem cell transplantation. Front Immunol. 2020; 11: 489.

3. Lee AC, Aung L. Treatment of hepatic veno-occlusive disease in children with N-acetylcysteine. Pediatr Blood Cancer. 2019; 66 (2): e27518.

4. Mahadeo KM, Bajwa R, Abdel-Azim H, et al. Diagnosis, grading, and treatment recommendations for children, adolescents, and young adults with sinusoidal obstructive syndrome: an international expert position statement. Lancet Haematol. 2020; 7 (1): e61–e72.

5. Corbacioglu S, Jabbour EJ, Mohty M. Risk factors for development of and progression of hepatic veno-occlusive disease/sinusoidal obstruction syndrome. Biol Blood Marrow Transplant. 2019; 25 (7): 1271–1280.

6. Richardson P, Aggarwal S, Topaloglu O, Villa KF, Corbacioglu S. Systematic review of defibrotide studies in the treatment of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS). Bone Marrow Transplant. 2019; 54 (12): 1951–1962.

7. Mahadeo K, Bajwa R. Hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation. J Pediatr Intensive Care. 2014; 3 (3): 183–193.

8. Richardson PG, Carreras E, Iacobelli M, Nejadnik B. The use of defibrotide in blood and marrow transplantation. Blood Adv. 2018; 2 (12): 1495–1509.

9. Ravaioli F, Colecchia A, Alemanni LV, et al. Role of imaging techniques in liver veno-occlusive disease diagnosis: recent advances and literature review. Expert Rev Gastroenterol Hepatol. 2019; 13 (5): 463–484.

10. Corbacioglu S, Topaloglu O, Aggarwal S. A systematic review and meta-analysis of studies of defibrotide prophylaxis for veno-occlusive disease/sinusoidal obstruction syndrome. Clin Drug Investig. 2022; 42 (6): 465–476.

11. Szmit Z, Gorczyńska E, Mielcarek-Siedziuk M, Ussowicz M, Owoc-Lempach J, Kałwak K. Veno-occlusive disease in children and adolescents after hematopoietic stem cell transplantation: Did the Modified Seattle Criteria fit the characteristics of pediatric population? Adv Clin Exp Med. 2020; 29 (3): 339–344.

12. Szmit Z, Gorczynska E, Król A, et al. Introduction of new pediatric EBMT criteria for VOD diagnosis: is it time-saving or money-wasting? : prospective evaluation of pediatric EBMT criteria for VOD. Bone Marrow Transplant. 2020; 55 (11): 2138–2146.

13. Kernan NA, Grupp S, Smith AR, et al. Final results from a defibrotide treatment-IND study for patients with hepatic veno-occlusive disease/sinusoidal obstruction syndrome. Br J Haematol. 2018; 181 (6): 816–827.

14. Cairo MS, Cooke KR, Lazarus HM, Chao N. Modified diagnostic criteria, grading classification and newly elucidated pathophysiology of hepatic SOS/VOD after haematopoietic cell transplantation. Br J Haematol. 2020; 190 (6): 822–836.

15. Bazarbachi AH, al Hamed R, Labopin M, et al. Underdiagnosed veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) as a major cause of multi-organ failure in acute leukemia transplant patients: an analysis from the EBMT Acute Leukemia Working Party. Bone Marrow Transplant. 2021; 56 (4): 917–927.

16. Corbacioglu S, Kernan NA, Pagliuca A, Ryan RJ, Tappe W, Richardson PG. Incidence of anicteric veno-occlusive disease/sinusoidal obstruction syndrome and outcomes with defibrotide following hematopoietic cell transplantation in adult and pediatric patients. Biol Blood Marrow Transplant. 2020; 26 (7): 1342–1349.

17. 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.

18. Deleve LD, Wang X, Tsai J, Kanel G, Strasberg S, Tokes ZA. Sinusoidal obstruction syndrome (veno-occlusive disease) in the rat is prevented by matrix metalloproteinase inhibition. Gastroenterology. 2003; 125 (3): 882–890.

19. Carreras E, Dufour C, Mohty M, Kröger N. Hematopoietic stem cell transplantation and cellular therapies. In: The EBMT Handbook. 2019; 373–377.

20. Carreras E. How I manage sinusoidal obstruction syndrome after haematopoietic cell transplantation. Br J Haematol. 2015; 168: 481–491.

21. Faraci M, Bertaina A, Luksch R, et al. Sinusoidal obstruction syndrome/veno-occlusive disease after autologous or allogeneic hematopoietic stem cell transplantation in children: a retrospective study of the Italian Hematology-Oncology Association-Hematopoietic Stem Cell Transplantation Group. Biol Blood Marrow Transplant. 2019; 25 (2): 313–320.

22. Schechter T, Perez-Albuerne E, Lin TF, et al. Veno-occlusive disease after high-dose busulfan-melphalan in neuroblastoma. Bone Marrow Transplant. 2020; 55 (3): 531–537.

23. Tewari P, Wallis W. Manifestations and management of veno-occlusive disease/sinusoidal obstruction syndrome in the era of contemporary therapies – hematology & oncology. Clin Adv Hematol Oncol. 2017; 15 (2): 130–139.

24. Yakushijin K, Atsuta Y, Doki N, et al. Sinusoidal obstruction syndrome after allogeneic hematopoietic stem cell transplantation: Incidence, risk factors and outcomes. Bone Marrow Transplant. 2016; 51 (3): 403–409.

25. Corbacioglu S, Chao NJ, Newburger P, Rosmarin AG. Hepatic sinusoidal obstruction syndrome (veno-occlusive disease) in children. UpToDate 2022. Dostupné na: https: //www.uptodate.com/contents/hepatic-sinusoidal-obstruction- syndrome-veno-occlusive-disease-in-children? search=VOD%20in%20children&source=search _result&selectedTitle=1~91&usage_type=default&display_rank=1

26. Strouse C, Zhang Y, Zhang MJ, et al. Risk score for development of veno-occlusive disease after allogeneic hematopoietic cell transplant. Biol Blood Marrow Transplant. 2018; 24 (10): 2072.

27. Jiang S, Penack O, Terzer T, et al. Predicting sinusoidal obstruction syndrome after allogeneic stem cell transplantation with the EASIX biomarker panel. Haematologica. 2021; 106 (2): 446–453.

28. Hildebrandt GC, Chao N. Endothelial cell function and endothelial-related disorders following haematopoietic cell transplantation. Br J Haematol. 2020; 190 (4): 508–519.

29. Lia G, Giaccone L, Leone S, Bruno B. Biomarkers for early complications of endothelial origin after allogeneic hematopoietic stem cell transplantation: do they have a potential clinical role? Front Immunol. 2021; 12: 641427.

30. Luft T, Dreger P, Radujkovic A. Endothelial cell dysfunction: a key determinant for the outcome of allogeneic stem cell transplantation. Bone Marrow Transplant. 2021; 56 (10): 2326–2335.

31. Akil A, Zhang Q, Mumaw CL, et al. Biomarkers for diagnosis and prognosis of sinusoidal obstruction syndrome after hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2015; 21 (10): 1739–1745.

32. Corbacioglu S, Richardson PG. Defibrotide for children and adults with hepatic veno-occlusive disease post hematopoietic cell transplantation. Expert Rev Gastroenterol Hepatol. 2017; 11 (10): 885–898.

33. Stanworth SJ, Navarrete C, Estcourt L, Marsh J. Platelet refractoriness – practical approaches and ongoing dilemmas in patient management. Br J Haematol. 2015; 171 (3): 297–305.

34. Rebulla P. Formulae for the definition of refractoriness to platelet transfusion. Transfus Med. 1993; 3 (1): 91–92.

35. Kleinman S, Silvergleid A, Tirnauer J S. Refractoriness to platelet transfusion therapy. UpToDate 2021. Dostupné na: https: //www.uptodate.com/contents/refractoriness-to-platelet-transfusion-therapy

36. Chan SS, Colecchia A, Duarte RF, Bonifazi F, Ravaioli F, Bourhis JH. Imaging in hepatic veno-occlusive disease/sinusoidal obstruction syndrome. Biol Blood Marrow Transplant. 2020; 26 (10): 1770–1779.

37. Lassau N, Leclère J, Auperin A, et al. Hepatic veno-occlusive disease after myeloablative treatment and bone marrow transplantation: value of gray-scale and Doppler US in 100 patients. Radiology. 1997; 204 (2): 545–552.

38. Berzigotti A. Non-invasive evaluation of portal hypertension using ultrasound elastography. J Hepatol. 2017; 67 (2): 399–411.

39. Özkan SG, Pata C, Şekuri A, Çınar Y, Özkan HA. Transient elastography of liver: Could it be a guide for diagnosis and management strategy in hepatic veno-occlusive disease (sinusoidal obstruction syndrome) ? Transfus Apher Sci. 2022; 61 (1): 103370.

40. Kammersgaard MB, Kielsen K, Heilmann C, Ifversen M, Müller K. Assessment of the proposed EBMT pediatric criteria for diagnosis and severity grading of sinusoidal obstruction syndrome. Bone Marrow Transplant. 2019; 54 (9): 1406–1418.

41. Taketomo C K, Hurlburt Hodding J, Kraus DM. Pediatric & neonatal dosage handbook with international trade names index. 25. vyd. United States: Wolters Kluwer; 2018; 583–585.

42. Chao N. How I treat sinusoidal obstruction syndrome. Blood. 2014; 123 (26): 4023–4026.

43. 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.

44. NHS England. Clinical Commissioning Policy (Revised) Use of defibrotide in severe veno-occlusive disease following stem cell transplant (all ages). Commissioning position. 2021. Dostupné na: https: //www.england.nhs.uk/publication/defibrotide-for- severe-veno-occlusive-disease-following-stem-cell-transplant/

45. Defibrotide side effects: common, severe, long term-Drugs.com. 2021. Dostupné na: https: //www.drugs.com/sfx/defibrotide-side-effects.html

46. Karagun BS, Akbas T, Erbey F, Sasmaz İ, Antmen B. The prophylaxis of hepatic veno-occlusive disease/sinusoidal obstruction syndrome with defibrotide after hematopoietic stem cell transplantation in children: single center experience. J Pediatr Hematol Oncol. 2022; 44 (1): e35–e39.

47. Miśkiewicz-Bujna J, Miśkiewicz-Migoń I, Rosa M, Liszka K, Kałwak K. Defibrotide in preventing veno-occlusive disease after hematopoietic stem cell transplantation in children with osteopetrosis. Acta Haematol Pol. 2021; 52 (2): 140–141.

48. Lankester AC, Albert MH, Booth C, et al. EBMT/ESID Inborn Errors Working Party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity. Bone Marrow Transplant. 2021; 56 (9): 2052–2062.

49. Road O. Defitelio (defibrotid): Nepoužívajte na profylaxiu venookluzívneho ochorenia (veno-occlusive disease, VOD) po transplantácii hematopoetických kmeňových buniek (hematopoietic stem-cell transplantation, HSCT). North Yorkshire UK, 2022. Dostupné na: https: //www.sukl.sk/buxus/docs/Bezpecnost_liekov/OznamyDrzitelov/DHPC_Defitelio_14_06_2022.pdf

50. Study comparing efficacy and safety of defibrotide vs best supportive care in the prevention of hepatic veno-occlusive disease in adult and pediatric patients. 2022. Dostupné na: https: //beta.clinicaltrials.gov/study/NCT02851407?tab=results

51. Grupp SA, Corbacioglu S, Kang HJ, Teshima T, Khaw SL, et. al. Defibrotide plus best standard of care compared with best standard of care alone for the prevention of sinusoidal obstruction syndrome (HARMONY): a randomised, multicentre, phase 3 trial. Lancet Haematol. 2023; 10 (5): e333–e345.

52. Myers KC, Lawrence J, Marsh RA, Davies SM, Jodele S. High-dose methylprednisolone for veno-occlusive disease of the liver in pediatric hematopoietic stem cell transplantation recipients. Biol Blood Marrow Transplant. 2013; 19 (3): 500–503.

53. Gloude NJ, Jodele S, Teusink-Cross A, et al. Combination of high-dose methylprednisolone and defibrotide for veno-occlusive disease in pediatric hematopoietic stem cell transplant recipients. Biol Blood Marrow Transplant. 2018; 24 (1): 91–95.

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Article was published in

Transfusion and Haematology Today

2023 Issue 2