Veno-Occlusive Disease (VOD) after Hematopoietic Stem Cell Transplant (HSCR): Always a Catastrophic Illness?

• Published in: Blood 2019-11, Vol.134 (Supplement_1), p.5649-5649
• Main Authors: Bernasconi, Paolo, Colombo, Anna Amelia, Caldera, Daniela, Borsani, Oscar
• Format: Article
• Language: English
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood-2019-125608

Introduction Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS), a potential life-threatening complication of conditioning regimens performed for HSCT, is due to an endothelial cell activation/damage and a prothrombotic-hyper-fibrinolytic condition. The clinical diagnosis and grading of VOD/SOS are based on the Seattle, Baltimore and EBMT criteria. Aims The aim of our work was to establish the incidence of VOD/SOS in our patient series, the time of clinical diagnosis, the best treatment options in relation to disease severity, the effectiveness of defibrotide treatment for severe/very severe forms, and the incidence of multi-organ failure (MOF). Methods Patients In the period January 2016-June 2019, 146 allogeneic HSCT were performed at our Institution. Patients' median age was 57 years (range 25-72) and 52% of them were males. The primary disease was AML in 82 (56.1%), MDS in 33 (22.6%), ALL in 16 (10.9%), MM in 5 (3.4%), primary or secondary Myelofibrosis in 10 (6.8%). Performance status was good (Karnofsky score ≥90) in 80% of HSCT. At HSCT, 31 patients (21.2%) had either had a previous hepatic disease or increased levels of alanine transaminases (ALT) and 36 (24.6%) had altered pulmonary functional tests. Thirty-five HSCTs were from HLA identical siblings, 55 from MUD and 35 from haplo-identical family donors. Conditioning regimen were either myeloablative or RIC depending on patients' age and co-morbidities. Stem cell source was BM in 36 (24.6%) patients, peripheral hematopoietic stem cells in 109 (74.6%) and cord blood in one. Standard acute GVHD prophyaxis consisted of Cylosporine A (CsA) and short methotrexate or CsA and micofenolate mofetyl in haplo-transplants. Definition, grading and treatment of VOD Diagnosis and grading of VOD/SOS were based on the Seattle, Baltimore and EBMT criteria. Renal dysfunction was defined as serum creatinine ≥3x baseline value, creatinine clearance or glomerular filtration rate declined to ≤40% of baseline or dialysis dependence due to VOD/SOS. Pulmonary dysfunction was defined as oxygen saturation ≤90% on room air, requirement for supplemental oxygen to maintain oxygen saturation ≥90%, or ventilator dependence not due to infection. Abdomen echo-tomography was performed in all patients to evaluate the presence of ascites. Patients having a pre-transplant liver disease or increased ALT levels received ursodeoxycholic acid (UDCA) as pharmacological prophylaxis Results In our series VOD/SOS occurred in 22/