Details

Autor(en) / Beteiligte

• Brodsky, Andrés Lázaro
• Cordini, Gregorio Raúl
• Qi, Xingshun

Titel

Budd–Chiari Syndrome and Paroxysmal Nocturnal Hemoglobinuria

Ist Teil von

• Budd-Chiari Syndrome, 2019, p.89-105

Ort / Verlag

Singapore: Springer Singapore Pte. Limited

Erscheinungsjahr

2019

Quelle

Alma/SFX Local Collection

Beschreibungen/Notizen

• Budd–Chiari syndrome (BCS) is a frequent thrombotic complication classically found in Western series of patients with paroxysmal nocturnal hemoglobinuria (PNH), with a high morbidity and mortality. Hematopoietic stem cell origin of liver endothelial cells and complement activation by enteric microbiota could explain the high frequency of this unusual thrombosis in PNH patients. However, Asian series of PNH patients show a much lower BCS prevalence, suggesting the existence of other unknown causative factors, genetic or environmental, that could explain this discrepancy. The finding of BCS is an indication to make a peripheral blood flow cytometry study to find PNH in Western patients, but this indication is not so clear in every Asian patient with BCS. Additional clinical findings, including other venous thrombosis, hemolysis, cytopenias, or renal iron overload in magnetic resonance imaging (MRI) studies of abdomen, may be required to increase the probability of PNH as the underlying thrombophilia in Asian cases. With the availability and success of eculizumab as the first complement blocker in PNH with thrombosis, a prompt diagnosis of PNH and immediate start of complement blockade plus anticoagulation are crucial for the prognosis and management of these patients. Preliminary results show that complement blockade markedly improves the results in every step of BCS treatment, preventing the complications of rethrombosis either with medical treatment, or with angioplasty, or with TIPS insertion, or with liver transplantation. Allogeneic bone marrow transplant has been relegated to the rare cases in which a syngeneic donor is available.