The Importance of New EBMT Criteria on the Diagnosis of Veno-Occlusive Liver Disease in Children
Status PubMed-not-MEDLINE Jazyk angličtina Země Švýcarsko Médium electronic
Typ dokumentu časopisecké články
PubMed
36769475
PubMed Central
PMC9918163
DOI
10.3390/jcm12030826
PII: jcm12030826
Knihovny.cz E-zdroje
- Klíčová slova
- hematopoietic stem cell transplantation, new diagnostic and severity EBMT criteria in childhood, retrospective analysis, veno-occlusive disease of the liver,
- Publikační typ
- časopisecké články MeSH
BACKGROUND: Early recognition and specific therapy facilitate a favorable disease course in hepatic venous-occlusive disease (HVOD) following hematopoietic stem cell transplantation (HCT). Diagnostic and classification criteria, published by the European Society for Blood and Marrow Transplantation (EBMT), better account for clinical differences in disease presentation in pediatric populations. OBJECTIVES: To compare the course of HVOD in children before and after the implementation of new EBMT criteria. MATERIAL AND METHODS: The study retrospectively evaluates 26 HVODs in 179 children treated in a single HCT unit (Slovakia) comparing the period of 2014-2017 using the Baltimore and modified Seattle criteria with the period of 2018-2021, when new EBMT criteria were adopted. RESULTS: No difference in HVOD incidence (11.2% vs. 14.8%, p = 0.46) and in time of diagnosis post-HCT (15.6 days vs. 15.7 days, p = 0.75) was found. With EBMT criteria we observed more frequent anicteric disease at diagnosis (50% vs. 87.5%, p = 0.04), lower serum bilirubin at diagnosis (3.4 mg/dL vs. 1.23 mg/dL, p = 0.045), and non-significant trends of shorter defibrotide treatment (21.7 days vs. 15.6 days, p = 0.73), decreased mortality (30% vs. 6.2%, p = 0.10) and shorter hospitalization (73.1 days vs. 59.6 days, p = 0.54). CONCLUSIONS: Different time periods around the implementation of new criteria are evaluated, underling that pediatric EBMT criteria for post-transplant HVOD diagnosis appear more sensitive.
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Bazarbachi A.H., Al Hamed R., Labopin M., Halaburda K., Labussiere H., Bernasconi P., Schroyens W., Gandemer V., Schaap N.P., Loschi 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;56:917–927. doi: 10.1038/s41409-020-01135-3. PubMed DOI
Bonifazi F., Barbato F., Ravaioli F., Sessa M., Defrancesco I., Arpinati M., Cavo M., Colecchia A. Diagnosis and Treatment of VOD/SOS After Allogeneic Hematopoietic Stem Cell Transplantation. Front. Immunol. 2020;11:489. doi: 10.3389/fimmu.2020.00489. PubMed DOI PMC
Cairo M.S., Cooke K.R., Lazarus H.M., Chao N. Modified diagnostic criteria, grading classification and newly elucidated pathophysiology of hepatic SOS/VOD after haematopoietic cell transplantation. Br. J. Haematol. 2020;190:822–836. doi: 10.1111/bjh.16557. PubMed DOI PMC
Carreras E. How I manage sinusoidal obstruction syndrome after haematopoietic cell transplantation. Br. J. Haematol. 2015;168:481–491. doi: 10.1111/bjh.13215. PubMed DOI
Kernan N.A., Grupp S., Smith A.R., Arai S., Triplett B., Antin J.H., Lehmann L., Shore T., Ho V.T., Bunin N., et al. Final results from a defibrotide treatment-IND study for patients with hepatic veno-occlusive disease/sinusoidal obstruction syndrome. Br. J. Haematol. 2018;181:816–827. doi: 10.1111/bjh.15267. PubMed DOI PMC
Richardson P., Aggarwal S., Topaloglu O., Villa K.F., Corbacioglu S. Systematic review of defibrotide studies in the treatment of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) Bone Marrow Transplant. 2019;54:1951–1962. doi: 10.1038/s41409-019-0474-8. PubMed DOI PMC
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;29:339–344. doi: 10.17219/acem/115070. PubMed DOI
Szmit Z., Gorczynska E., Król A., Ussowicz M., Mielcarek-Siedziuk M., Olejnik I., Panasiuk A., Kałwak K. 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;55:2138–2146. doi: 10.1038/s41409-020-0918-1. PubMed DOI
Dhir A., Wadhwa A., Haines H., Chewning J., Murthy S., Kim J., Beatty L., Mixon E., Fowler K.B., Gage J., et al. Retrospective Application of Sinusoidal Obstruction Syndrome/Veno-occlusive Disease Diagnostic Criteria in a Pediatric Hematopoietic Stem Cell Transplant Cohort. J. Pediatr. Hematol. Oncol. 2022;44:E343–E348. doi: 10.1097/MPH.0000000000002267. PubMed DOI
Yoon J.H., Choi C.W., Won J.H. Hepatic sinusoidal obstruction syndrome/ veno-occlusive disease after hematopoietic cell transplantation: Historical and current consider-ations in Korea. Korean J. Intern. Med. 2021;36:1261–1280. doi: 10.3904/kjim.2021.082. PubMed DOI PMC
Mahadeo K.M., Bajwa R., Abdel-Azim H., Lehmann L.E., Duncan C., Zantek N., Vittorio J., Angelo J., McArthur J., Schadler K., 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:e61–e72. doi: 10.1016/S2352-3026(19)30201-7. PubMed DOI
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;17:1713–1720. doi: 10.1016/j.bbmt.2011.06.006. PubMed DOI
Corbacioglu S., Jabbour E.J., Mohty M. Risk Factors for Development of and Progression of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome. Biol. Blood Marrow Transplant. 2019;25:1271–1280. doi: 10.1016/j.bbmt.2019.02.018. PubMed DOI
Richardson P.G., Carreras E., Iacobelli M., Nejadnik B. The use of defibrotide in blood and marrow transplantation. Blood Adv. 2018;2:1495–1509. doi: 10.1182/bloodadvances.2017008375. PubMed DOI PMC
Mohty M., Malard F., Abecassis M., Aerts E., Alaskar A.S., Aljurf M., Arat M., Bader P., Baron F., Bazarbachi A., 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;50:781–789. doi: 10.1038/bmt.2015.52. PubMed DOI PMC
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;15:130–139. PubMed
Corbacioglu S., Carreras E., Ansari M., Balduzzi A., Cesaro S., Dalle J.H., Dignan F., Gibson B., Guengoer T., Gruhn B., 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;53:138–145. doi: 10.1038/bmt.2017.161. PubMed DOI PMC
Corbacioglu S., Richardson P.G. Defibrotide for children and adults with hepatic veno-occlusive disease post hematopoietic cell transplantation. Expert. Rev. Gastroenterol. Hepatol. 2017;11:885–898. doi: 10.1080/17474124.2017.1370372. PubMed DOI
Corbacioglu S., Kernan N.A., Pagliuca A., Ryan R.J., Tappe W., Richardson P.G. 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;26:1342–1349. doi: 10.1016/j.bbmt.2020.03.011. PubMed DOI PMC
Jones R.J., Lee K.S., Beschorner W.E., Vogel V.G., Grochow L.B., Braine H.G., Vogelsang G.B., Sensenbrenner L.L., Santos G.W., Saral R. Venoocclusive disease of the liver following bone marrow transplantation. Transplantation. 1987;44:778–783. doi: 10.1097/00007890-198712000-00011. PubMed DOI
Ragoonanan D., Khazal S.J., Wang J., Payne A., Kohorst M., Harden A., Tewari P., Petropoulos D., Shoberu B., Kebriaei P., et al. Improved detection of sinusoidal obstructive syndrome using pediatric-AYA diagnostic criteria and severity grading. Bone Marrow Transplant. 2021;56:175–184. doi: 10.1038/s41409-020-00998-w. PubMed DOI
Corbacioglu S. The coming of age of the pediatric EBMT criteria. Bone Marrow Transplant. 2021;56:767–768. doi: 10.1038/s41409-020-01141-5. PubMed DOI PMC
Embaby M.M., Rangarajan H.G., Abu-Arja R., Auletta J.J., Stanek J., Pai V., Nicol K.K., Bajwa R.S. Refractory Thrombocytopenia Is a Valid Early Diagnostic Criteria for Hepatic Veno-Occlusive Disease in Children. Biol. Blood Marrow Transplant. 2020;26:546–552. doi: 10.1016/j.bbmt.2019.11.012. PubMed DOI
McDonald G.B., Hinds M.S., Fisher L.D., Schoch H.G., Wolford J.L., Banaji M., Hardin B.J., Shulman H.M., Clift R.A. Veno-occlusive disease of the liver and multiorgan failure after bone marrow transplantation: A cohort study of 355 patients. Ann. Intern. Med. 1993;118:255–267. doi: 10.7326/0003-4819-118-4-199302150-00003. PubMed DOI
Deleve L.D., Wang X., Tsai J., Kanel G., Strasberg S., Tokes Z.A. Sinusoidal obstruction syndrome (veno-occlusive disease) in the rat is prevented by matrix metalloproteinase inhibition. Gastroenterology. 2003;125:882–890. doi: 10.1016/S0016-5085(03)01056-4. PubMed DOI
Ravaioli F., Colecchia A., Alemanni L.V., Vestito A., Dajti E., Marasco G., Sessa M., Pession A., Bonifazi F., Festi D. Role of imaging techniques in liver veno-occlusive disease diagnosis: Recent advances and literature review. Expert. Rev. Gastroenterol. Hepatol. 2019;13:463–484. doi: 10.1080/17474124.2019.1588111. PubMed DOI
Faraci M., Bertaina A., Luksch R., Calore E., Lanino E., Saglio F., Prete A., Menconi M., De Simone G., Tintori V., 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;25:313–320. doi: 10.1016/j.bbmt.2018.09.027. PubMed DOI
Nauffal M., Kim H.T., Richardson P.G., Soiffer R.J., Antin J.H., Cutler C., Nikiforow S., Gooptu M., Koreth J., Romee R., et al. Defibrotide: Real-world management of veno-occlusive disease/sinusoidal obstructive syndrome after stem cell transplant. Blood Adv. 2022;6:181–188. doi: 10.1182/bloodadvances.2021005410. PubMed DOI PMC
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;42:465–476. doi: 10.1007/s40261-022-01140-y. PubMed DOI PMC
Kammersgaard M.B., 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;54:1406–1418. doi: 10.1038/s41409-018-0426-8. PubMed DOI PMC
Hildebrandt G.C., Chao N. Endothelial cell function and endothelial-related disorders following haematopoietic cell transplantation. Br. J. Haematol. 2020;190:508–519. doi: 10.1111/bjh.16621. PubMed DOI PMC
Mahadeo K., Bajwa R. Hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation. J. Pediatr. Intensive Care. 2014;3:183–193. doi: 10.3233/PIC-14102. PubMed DOI PMC
Pagliuca S., Michonneau D., Sicre de Fontbrune F., Sutra del Galy A., Xhaard A., Robin M., Peffault de Latour R., Socie G. Allogeneic reactivity-mediated endothelial cell complications after HSCT: A plea for consensual definitions. Blood Adv. 2019;3:2424–2435. doi: 10.1182/bloodadvances.2019000143. PubMed DOI PMC
Ruutu T., Eriksson B., Remes K., Juvonen E., Volin L., Remberger M., Parkkali T., Hägglund H., Ringdén O. Ursodeoxycholic acid for the prevention of hepatic complications in allogeneic stem cell transplantation. Blood. 2002;100:1977–1983. doi: 10.1182/blood-2001-12-0159. PubMed DOI
Bohte A.E., Dierselhuis M.P., van Noesel M.M., Lequin M.H. Imaging features of hepatic sinusoidal obstruction syndrome or veno-occlusive disease in children. Pediatr. Radiol. 2022;52:122–133. doi: 10.1007/s00247-021-05174-w. PubMed DOI PMC
Schechter T., Perez-Albuerne E., Lin T.F., Irwin M.S., Essa M., Desai A.V., Frangoul H., Yanik G., Dupuis L.L., Jacobsohn D., et al. Veno-occlusive disease after high-dose busulfan-melphalan in neuroblastoma. Bone Marrow Transplant. 2020;55:531–537. doi: 10.1038/s41409-018-0298-y. PubMed DOI
Lankester A.C., Albert M.H., Booth C., Gennery A.R., Güngör T., Hönig M., Morris E.C., Moshous D., Neven B., Schulz A., et al. EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity. Bone Marrow Transplant. 2021;56:2052–2062. doi: 10.1038/s41409-021-01378-8. PubMed DOI PMC
Ansari M., Théoret Y., Rezgui M.A., Peters C., Mezziani S., Desjean C., Vachon M.F., Champagne M.A., Duval M., Krajinovic M., et al. Association between busulfan exposure and outcome in children receiving intravenous busulfan before hematopoietic stem cell transplantation. Drug Monit. 2014;36:93–99. doi: 10.1097/FTD.0b013e3182a04fc7. PubMed DOI
Yakushijin K., Atsuta Y., Doki N., Yokota A., Kanamori H., Miyamoto T., Ohwada C., Miyamura K., Nawa Y., Kurokawa M., et al. Sinusoidal obstruction syndrome after allogeneic hematopoietic stem cell transplantation: Incidence, risk factors and outcomes. Bone Marrow Transplant. 2016;51:403–409. doi: 10.1038/bmt.2015.283. PubMed DOI
Stanworth S.J., Navarrete C., Estcourt L., Marsh J. Platelet refractoriness—Practical approaches and ongoing dilemmas in patient management. Br. J. Haematol. 2015;171:297–305. doi: 10.1111/bjh.13597. PubMed DOI
Kleinman S., Silvergleid A., Tirnauer J.S. Refractoriness to Platelet Transfusion Therapy—UpToDate. 2021. [(accessed on 31 December 2021)]. Available online: https://www.uptodate.com/contents/refractoriness-to-platelet-transfusion-therapy.
Rebulla P. Formulae for the definition of refractoriness to platelet transfusion. Transfus. Med. 1993;3:91–92. doi: 10.1111/j.1365-3148.1993.tb00108.x. PubMed DOI
Lassau N., Leclère J., Auperin A., Bourhis J.H., Hartmann O., Valteau-Couanet D., Benhamou E., Bosq J., Ibrahim A., Girinski T., 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;204:545–552. doi: 10.1148/radiology.204.2.9240551. PubMed DOI
Nishida M., Kahata K., Hayase E., Shigematsu A., Sato M., KudoNishida M., Kahata K., Hayase E., Shigematsu A., Sato M., et al. Novel Ultrasonographic Scoring System of Sinusoidal Obstruction Syndrome after Hematopoietic Stem Cell Transplantation. Biol. Blood Marrow Transplant. 2018;24:1896–1900. doi: 10.1016/j.bbmt.2018.05.025. PubMed DOI
Özkan S.G., Pata C., Şekuri A., Çınar Y., Özkan H.A. 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;61:103370. doi: 10.1016/j.transci.2022.103370. PubMed DOI
Chan S.S., Colecchia A., Duarte R.F., Bonifazi F., Ravaioli F., Bourhis J.H. Imaging in Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome. Biol. Blood Marrow Transplant. 2020;26:1770–1779. doi: 10.1016/j.bbmt.2020.06.016. PubMed DOI
Strouse C., Zhang Y., Zhang M.J., DiGilio A., Pasquini M., Horowitz M.M. Risk Score for Development of Veno-Occlusive Disease After Allogeneic Hematopoietic Cell Transplant. Biol. Blood Marrow Transplant. 2018;24:2072. doi: 10.1016/j.bbmt.2018.06.013. PubMed DOI PMC
Jiang S., Penack O., Terzer T., Schult D., Majer-Lauterbach J., Radujkovic A., Blau I.W., Bullinger L., Muller-Tidow C., Dreger P., et al. Predicting sinusoidal obstruction syndrome after allogeneic stem cell transplantation with the EASIX biomarker panel. Haematologica. 2021;106:446–453. doi: 10.3324/haematol.2019.238790. PubMed DOI PMC
Carreras E., Dufour C., Mohty M., Kröger N. Hematopoietic Stem Cell Transplantation and Cellular Therapies The EBMT Handbook. Springer; Berlin/Heidelberg, Germany: 2019. pp. 373–702. PubMed
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. doi: 10.3389/fimmu.2021.641427. PubMed DOI PMC
Luft T., Dreger P., Radujkovic A. Endothelial cell dysfunction: A key determinant for the outcome of allogeneic stem cell transplantation. Bone Marrow Transplant. 2021;56:2326–2335. doi: 10.1038/s41409-021-01390-y. PubMed DOI PMC