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

被引：42

作者：

Faraci, Maura ^{[1
]}

Bertaina, Alice ^{[2
]}

Luksch, Roberto ^{[3
]}

Calore, Elisabetta ^{[4
]}

Lanino, Edoardo ^{[1
]}

Saglio, Francesco ^{[5
]}

Prete, Arcangelo ^{[6
]}

Menconi, Mariacristina ^{[7
]}

De Simone, Giusy ^{[8
]}

Tintori, Veronica ^{[9
]}

Cesaro, Simone ^{[10
]}

Santarone, Stella ^{[11
]}

Orofino, Maria Grazia ^{[12
]}

Locatelli, Franco ^{[2
]}

Zecca, Marco ^{[13
]}

机构：

[1] Ist Giannina Gaslini, IRCCS, Dept Hematol Oncol, Hematopoiet Stem Cell Unit, Genoa, Italy

[2] Osped Bambino Gesit, IRCCS, Dept Pediat Hematol & Oncol, Rome, Italy

[3] Fdn IRCCS Ist Nazl Tumori, Dept Pediat Oncol, Milan, Italy

[4] Azienda Osped Univ Padova, Dept Womens & Childrens Hlth, Pediat Hematol Oncol Unit, Padua, Italy

[5] Regina Margherita Childrens Hosp, Pediat Oncohematol Stem Cell Transplantat & Cellu, Turin, Italy

[6] UO Pediat S Orsola Malpighi Univ Bologna, Oncol Hematol & Hematopoiet Stem Cell Transplant, Bologna, Italy

[7] Univ Pisa, Pediat Clin, Haematopoiet Stem Cell Transplantat Unit, Pisa, Italy

[8] Santobono Pausilipon Hosp, Dept Hematooncol, BMT Unit, Naples, Italy

[9] Meyer Childrens Hosp, Dept Pediat Oncol, Transplantat Unit, Florence, Italy

[10] Azienda Osped Univ Integrata, Pediat Hematol Oncol, Verona, Italy

[11] Osped Civile, Dipartimento Ematol Med Trasfus & Biotecnol, Pescara, Italy

[12] Hosp Binaghi & Microcitem, Bone Marrow Transplant Ctr, Cagliari, Italy

[13] IRCCS Policlin San Matteo, Pediat Hematol Oncol, Pavia, Italy

来源：

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION | 2019年 / 25卷 / 02期

关键词：

Sinusoidal obstruction syndrome; Children; Diagnostic criteria; Severity grading; HEPATIC VENOOCCLUSIVE DISEASE; BONE-MARROW-TRANSPLANTATION; RISK-FACTORS; DEFIBROTIDE; DIAGNOSIS; ADULTS; BLOOD; PROPHYLAXIS;

D O I：

10.1016/j.bbmt.2018.09.027

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Sinusoidal obstruction syndrome (SOS), also known as veno-occlusive disease (VOD), is a potentially life-threatening complication that may develop after hematopoietic stem cell transplantation (HSCT). The aims of this retrospective multicenter study were to evaluate the incidence of SOS/VOD in a large cohort of children transplanted in centers across Italy by applying the new European Society for Blood and Marrow Transplantation (EBMT) criteria and to analyze the risk factors underlying this complication. We retrospectively reviewed data of pediatric HSCTs performed in 13 AIEOP (Associazione Italiana di Ematologia e Oncologia Pediatrica)-affiliated centers between January 2000 and April 2016. The new pediatric EBMT criteria were retrospectively applied for diagnoses of SOS/VOD and severity grading. Among 5072 transplants considered at risk for SOS/VOD during the study period, 103 children (2%) developed SOS/VOD, and the grade was severe or very severe in all patients. The median time of SOS/VOD occurrence was 17 days after HSCT (range, 1 to 104). Sixty-nine patients (67%) were treated with defibrotide for a median time of 16 days (range, 4 to 104). In multivariable analysis age < 2 years, use of busulfan during the conditioning regimen, female gender, and hemophagocytic lymphohistiocytosis were risk factors statistically associated with the development of SOS/VOD. The overall mortality directly related to SOS/VOD was 15.5%. Overall survival at 1 year was worse in patients with SOS/VOD (P = .0033), and this difference disappeared 5 years after HSCT. Nonrelapse mortality was significantly higher 1 and 5 years after transplantation in patients who developed SOS/VOD (P < .001). Based on the application of new EBMT criteria, the overall incidence of SOS/VOD recorded in this large Italian pediatric retrospective study was 2%. Nonrelapse mortality was significantly higher in patients who developed SOS/VOD. Identifying the risk factors associated with SOS/VOD can lead to more effective early treatment strategies of this potentially fatal HSCT complication in childhood. (C) 2018 Published by Elsevier Inc. on behalf of American Society for Blood and Marrow Transplantation.

引用

页码：313 / 320

页数：8

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