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Defibrotide for Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease Prophylaxis in High-Risk Adult Patients: A Single-Center Experience Study.
Biol Blood Marrow Transplant. 2018 07; 24(7):1471-1475.BB

Abstract

Sinusoidal obstruction syndrome (SOS), also known as hepatic veno-occlusive disease (VOD), is a serious complication after hematopoietic stem cell transplantation (HSCT). SOS/VOD usually occurs within 3 weeks of HSCT, but the 2016 European Society for Blood and Marrow Transplantation diagnosis criteria have been revised to include late forms. Prophylactic use of defibrotide is recommended in the pediatric setting, but its value remains uncertain in the adult population. We report here a single-center series of 63 adult patients considered at high risk for SOS/VOD who received defibrotide prophylaxis in combination with ursodeoxycholic acid between May 2012 and August 2016. The median duration of defibrotide therapy was 23 days. Bleeding occurred in 14 patients (21.5%). Defibrotide prophylaxis was discontinued in 7 patients (10.8%): 4 cases (6.3%) due to bleeding and 3 cases (4.6%) because of the need for antithrombotic therapy. Overall, SOS/VOD occurred in 4 cases (6.3%) within 21 days after HSCT (days 13 and 14) in 2 cases and late-onset SOS/VOD (days 57 and 58) in the other 2 cases. SOS/VOD was moderate in 1 case, very severe in 3 cases, with 2 deaths related to SOS/VOD. Cumulative incidence of grades II to IV acute graft-versus-host disease and transplant-associated thrombotic microangiopathy were 22.2% and 3.2%, respectively. With a median follow-up of 31 months (range, 10.7 to 60.3), the rates of 2-year overall survival, progression-free survival, incidence of relapse, and nonrelapse mortality were 56.5%, 49%, 28.7%, and 22.3%, respectively. In our experience defibrotide prophylaxis is associated with a low incidence of SOS/VOD after allogeneic HSCT in a high-risk adult population with an acceptable safety profile.

Authors+Show Affiliations

Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France.Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France.Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France; Université Pierre et Marie Curie (UPMC), Sorbonne Universités, Paris, France.Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France.Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France.Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France; Université Pierre et Marie Curie (UPMC), Sorbonne Universités, Paris, France; INSERM, UMRs 938, Paris, France.Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France; Université Pierre et Marie Curie (UPMC), Sorbonne Universités, Paris, France; INSERM, UMRs 938, Paris, France.Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France; Université Pierre et Marie Curie (UPMC), Sorbonne Universités, Paris, France.Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France.Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France.Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France.Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France.Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France.Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France.Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France; Université Pierre et Marie Curie (UPMC), Sorbonne Universités, Paris, France.UPAC & C (Unité de préparation des anticancéreux et contrôle), Saint Antoine Hospital, AP-HP, Paris, France.UPAC & C (Unité de préparation des anticancéreux et contrôle), Saint Antoine Hospital, AP-HP, Paris, France.Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France; Université Pierre et Marie Curie (UPMC), Sorbonne Universités, Paris, France.Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France; Université Pierre et Marie Curie (UPMC), Sorbonne Universités, Paris, France; INSERM, UMRs 938, Paris, France.Hematology and Cellular Therapy Service, Saint Antoine Hospital, AP-HP, Paris, France; Université Pierre et Marie Curie (UPMC), Sorbonne Universités, Paris, France; INSERM, UMRs 938, Paris, France. Electronic address: mohamad.mohty@inserm.fr.

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

29477779

Citation

Picod, Adrien, et al. "Defibrotide for Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease Prophylaxis in High-Risk Adult Patients: a Single-Center Experience Study." Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation, vol. 24, no. 7, 2018, pp. 1471-1475.
Picod A, Bonnin A, Battipaglia G, et al. Defibrotide for Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease Prophylaxis in High-Risk Adult Patients: A Single-Center Experience Study. Biol Blood Marrow Transplant. 2018;24(7):1471-1475.
Picod, A., Bonnin, A., Battipaglia, G., Giannotti, F., Ruggeri, A., Brissot, E., Malard, F., Médiavilla, C., Belhocine, R., Vekhoff, A., Gueye, M. S., Lapusan, S., Adaeva, R., Isnard, F., Legrand, O., Baylatry, M. T., Joly, A. C., Labopin, M., Duléry, R., & Mohty, M. (2018). Defibrotide for Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease Prophylaxis in High-Risk Adult Patients: A Single-Center Experience Study. Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation, 24(7), 1471-1475. https://doi.org/10.1016/j.bbmt.2018.02.015
Picod A, et al. Defibrotide for Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease Prophylaxis in High-Risk Adult Patients: a Single-Center Experience Study. Biol Blood Marrow Transplant. 2018;24(7):1471-1475. PubMed PMID: 29477779.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Defibrotide for Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease Prophylaxis in High-Risk Adult Patients: A Single-Center Experience Study. AU - Picod,Adrien, AU - Bonnin,Agnès, AU - Battipaglia,Giorgia, AU - Giannotti,Federica, AU - Ruggeri,Annalisa, AU - Brissot,Eolia, AU - Malard,Florent, AU - Médiavilla,Clémence, AU - Belhocine,Ramdane, AU - Vekhoff,Anne, AU - Gueye,Mor Sény, AU - Lapusan,Simona, AU - Adaeva,Rosa, AU - Isnard,Françoise, AU - Legrand,Ollivier, AU - Baylatry,Minh-Tam, AU - Joly,Anne-Christine, AU - Labopin,Myriam, AU - Duléry,Rémy, AU - Mohty,Mohamad, Y1 - 2018/03/01/ PY - 2017/12/18/received PY - 2018/02/14/accepted PY - 2018/2/27/pubmed PY - 2019/6/14/medline PY - 2018/2/26/entrez KW - Defibrotide KW - Endothelial cell activation syndrome KW - Hematopoietic stem cell transplantation KW - Hepatic veno-occlusive disease KW - Sinusoidal obstruction syndrome SP - 1471 EP - 1475 JF - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation JO - Biol. Blood Marrow Transplant. VL - 24 IS - 7 N2 - Sinusoidal obstruction syndrome (SOS), also known as hepatic veno-occlusive disease (VOD), is a serious complication after hematopoietic stem cell transplantation (HSCT). SOS/VOD usually occurs within 3 weeks of HSCT, but the 2016 European Society for Blood and Marrow Transplantation diagnosis criteria have been revised to include late forms. Prophylactic use of defibrotide is recommended in the pediatric setting, but its value remains uncertain in the adult population. We report here a single-center series of 63 adult patients considered at high risk for SOS/VOD who received defibrotide prophylaxis in combination with ursodeoxycholic acid between May 2012 and August 2016. The median duration of defibrotide therapy was 23 days. Bleeding occurred in 14 patients (21.5%). Defibrotide prophylaxis was discontinued in 7 patients (10.8%): 4 cases (6.3%) due to bleeding and 3 cases (4.6%) because of the need for antithrombotic therapy. Overall, SOS/VOD occurred in 4 cases (6.3%) within 21 days after HSCT (days 13 and 14) in 2 cases and late-onset SOS/VOD (days 57 and 58) in the other 2 cases. SOS/VOD was moderate in 1 case, very severe in 3 cases, with 2 deaths related to SOS/VOD. Cumulative incidence of grades II to IV acute graft-versus-host disease and transplant-associated thrombotic microangiopathy were 22.2% and 3.2%, respectively. With a median follow-up of 31 months (range, 10.7 to 60.3), the rates of 2-year overall survival, progression-free survival, incidence of relapse, and nonrelapse mortality were 56.5%, 49%, 28.7%, and 22.3%, respectively. In our experience defibrotide prophylaxis is associated with a low incidence of SOS/VOD after allogeneic HSCT in a high-risk adult population with an acceptable safety profile. SN - 1523-6536 UR - https://www.unboundmedicine.com/medline/citation/29477779/Defibrotide_for_Sinusoidal_Obstruction_Syndrome/Veno_Occlusive_Disease_Prophylaxis_in_High_Risk_Adult_Patients:_A_Single_Center_Experience_Study_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S1083-8791(18)30101-0 DB - PRIME DP - Unbound Medicine ER -