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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 Jul; 26(7):1342-1349.BB

Abstract

Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life-threatening complication of hematopoietic cell transplantation (HCT) that is traditionally diagnosed using Baltimore or modified Seattle criteria. Whereas the Baltimore criteria require the presence of hyperbilirubinemia (bilirubin ≥2 mg/dL) for a diagnosis of VOD/SOS, the modified Seattle criteria do not. Before approval by the US Food and Drug Administration, defibrotide was available in the United States through an expanded-access study (T-IND). The T-IND protocol initially required post-HCT diagnosis of VOD/SOS by the Baltimore criteria or biopsy but was later amended to include patients diagnosed using the modified Seattle criteria. This post hoc analysis examined the incidence of VOD/SOS with a bilirubin level <2 mg/dL before and after Day 21 post-HCT in T-IND patients enrolled following the amendment allowing for diagnosis by the modified Seattle criteria. Survival of adult and pediatric patients with or without hyperbilirubinemia and with or without multiorgan dysfunction (MOD) was also evaluated. Of 803 post-HCT patients with VOD/SOS enrolled following the protocol amendment, 181 (23%) had a bilirubin level <2 mg/dL and would not have been diagnosed if hyperbilirubinemia was required. The bilirubin level at diagnosis was <2 mg/dL in 165 of 331 patients (50%) diagnosed by the modified Seattle criteria and in 16 of 23 patients (70%) diagnosed by biopsy. VOD/SOS with a bilirubin level <2 mg/dL was more common in pediatric patients (29%), although it also occurred in adult patients (15%). Patients with hyperbilirubinemia had lower Day 100 survival (54% versus 87% in patients with bilirubin <2 mg/dL) and a higher incidence of MOD (41% versus 26% in patients with bilirubin <2 mg/dL). The incidence of treatment-emergent adverse events and serious adverse events was lower in patients with a bilirubin level <2 mg/dL. These results indicate that anicteric VOD/SOS occurs in both adult and pediatric patients post-HCT and can be diagnosed before and after Day 21 in both groups. The worse survival in patients with bilirubin ≥2 mg/dL suggests that requiring hyperbilirubinemia may result in a progressed disease stage associated with worse outcomes. Taken together, these results highlight the importance of awareness and the possibility of VOD/SOS in the absence of elevated bilirubin level.

Authors+Show Affiliations

Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation, University of Regensburg, Regensburg, Germany. Electronic address: selim.corbacioglu@mac.com.Pediatric BMT Service, Memorial Sloan Kettering Cancer Center, New York, New York.Department of Haematological Medicine, King's College Hospital, London, United Kingdom.Jazz Pharmaceuticals, Philadelphia, Pennsylvania.Jazz Pharmaceuticals, Palo Alto, California.Jerome Lipper Multiple Myeloma Center, Dana-Farber Cancer Institute, Harvard Medical School, Boston, Massachusetts.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

32200121

Citation

Corbacioglu, Selim, et al. "Incidence of Anicteric Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome and Outcomes With Defibrotide Following Hematopoietic Cell Transplantation in Adult and Pediatric Patients." Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation, vol. 26, no. 7, 2020, pp. 1342-1349.
Corbacioglu S, Kernan NA, Pagliuca A, et al. 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.
Corbacioglu, S., Kernan, N. A., Pagliuca, A., Ryan, R. J., Tappe, W., & Richardson, P. G. (2020). Incidence of Anicteric Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome and Outcomes with Defibrotide following Hematopoietic Cell Transplantation in Adult and Pediatric Patients. Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation, 26(7), 1342-1349. https://doi.org/10.1016/j.bbmt.2020.03.011
Corbacioglu S, et al. 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. PubMed PMID: 32200121.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Incidence of Anicteric Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome and Outcomes with Defibrotide following Hematopoietic Cell Transplantation in Adult and Pediatric Patients. AU - Corbacioglu,Selim, AU - Kernan,Nancy A, AU - Pagliuca,Antonio, AU - Ryan,Robert J, AU - Tappe,William, AU - Richardson,Paul G, Y1 - 2020/03/19/ PY - 2019/09/26/received PY - 2020/03/10/revised PY - 2020/03/11/accepted PY - 2020/3/23/pubmed PY - 2020/3/23/medline PY - 2020/3/23/entrez KW - Anicteric KW - Baltimore criteria KW - Bilirubin KW - Defibrotide KW - Modified Seattle criteria KW - Veno-occlusive disease/sinusoidal obstruction syndrome SP - 1342 EP - 1349 JF - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JO - Biol. Blood Marrow Transplant. VL - 26 IS - 7 N2 - Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life-threatening complication of hematopoietic cell transplantation (HCT) that is traditionally diagnosed using Baltimore or modified Seattle criteria. Whereas the Baltimore criteria require the presence of hyperbilirubinemia (bilirubin ≥2 mg/dL) for a diagnosis of VOD/SOS, the modified Seattle criteria do not. Before approval by the US Food and Drug Administration, defibrotide was available in the United States through an expanded-access study (T-IND). The T-IND protocol initially required post-HCT diagnosis of VOD/SOS by the Baltimore criteria or biopsy but was later amended to include patients diagnosed using the modified Seattle criteria. This post hoc analysis examined the incidence of VOD/SOS with a bilirubin level <2 mg/dL before and after Day 21 post-HCT in T-IND patients enrolled following the amendment allowing for diagnosis by the modified Seattle criteria. Survival of adult and pediatric patients with or without hyperbilirubinemia and with or without multiorgan dysfunction (MOD) was also evaluated. Of 803 post-HCT patients with VOD/SOS enrolled following the protocol amendment, 181 (23%) had a bilirubin level <2 mg/dL and would not have been diagnosed if hyperbilirubinemia was required. The bilirubin level at diagnosis was <2 mg/dL in 165 of 331 patients (50%) diagnosed by the modified Seattle criteria and in 16 of 23 patients (70%) diagnosed by biopsy. VOD/SOS with a bilirubin level <2 mg/dL was more common in pediatric patients (29%), although it also occurred in adult patients (15%). Patients with hyperbilirubinemia had lower Day 100 survival (54% versus 87% in patients with bilirubin <2 mg/dL) and a higher incidence of MOD (41% versus 26% in patients with bilirubin <2 mg/dL). The incidence of treatment-emergent adverse events and serious adverse events was lower in patients with a bilirubin level <2 mg/dL. These results indicate that anicteric VOD/SOS occurs in both adult and pediatric patients post-HCT and can be diagnosed before and after Day 21 in both groups. The worse survival in patients with bilirubin ≥2 mg/dL suggests that requiring hyperbilirubinemia may result in a progressed disease stage associated with worse outcomes. Taken together, these results highlight the importance of awareness and the possibility of VOD/SOS in the absence of elevated bilirubin level. SN - 1523-6536 UR - https://www.unboundmedicine.com/medline/citation/32200121/Incidence_of_Anicteric_Veno_Occlusive_Disease/Sinusoidal_Obstruction_Syndrome_and_Outcomes_with_Defibrotide_following_Hematopoietic_Cell_Transplantation_in_Adult_and_Pediatric_Patients_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S1083-8791(20)30148-8 DB - PRIME DP - Unbound Medicine ER -
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