Treatment of a patient with classical paroxysmal nocturnal hemoglobinuria and Budd-Chiari syndrome, with complement inhibitor eculizumab: Case Report

Hippokratia

Volumn 17, Issue 1, 2013, Pages 81-84

  Mandala, Eudokia ^a   Lafaras, C ^b   Goulis, I ^a   Tsioni, K ^a   Georgopoulou, V ^c   Ilonidis, G ^a  

^a ARISTOTLE UNIVERSITY MEDICAL SCHOOL   (Greece)

^b THEAGENIO CANCER HOSPITAL   (Greece)

^c Aristotle University of Thessaloniki   (Greece)

Author keywords

Budd Chiari syndrome; Eculizumab; Paroxysmal nocturnal haemoglobinuria (PNH)

Indexed keywords

ACENOCOUMAROL; CORTICOSTEROID; ECULIZUMAB; HEMOGLOBIN; LACTATE DEHYDROGENASE; LOW MOLECULAR WEIGHT HEPARIN;

ABDOMINAL PAIN; ADULT; ARTICLE; ASCITES; BLOOD TRANSFUSION; BONE MARROW BIOPSY; BUDD CHIARI SYNDROME; CASE REPORT; CLINICAL EXAMINATION; COMPUTER ASSISTED TOMOGRAPHY; DISEASE SEVERITY; DOPPLER ECHOGRAPHY; FATIGUE; FLOW CYTOMETRY; HEMOLYTIC ANEMIA; HEPATOSPLENOMEGALY; HOSPITAL ADMISSION; HUMAN; LIVER SCINTISCANNING; LOW BACK PAIN; MALE; NUCLEAR MAGNETIC RESONANCE IMAGING; PARACENTESIS; PAROXYSMAL NOCTURNAL HEMOGLOBINURIA; QUALITY OF LIFE; THROMBOSIS; TRANSJUGULAR INTRAHEPATIC PORTOSYSTEMIC SHUNT; TREATMENT DURATION; TREATMENT OUTCOME;

EID: 84879698392     PISSN: 11084189     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (23)

1. Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1995; 333: 1253-1258.
2. Parker C, Omine M, Richards S, Nishimura J, Bessler M, Ware R, et al; International PNH Interest Group. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood. 2005; 106: 3699-3709.
3. Risitano AM. Paroxysmal nocturnal hemoglobinuria and other complement-mediated hematological disorders. Immunobiology. 2012; 217: 1080-1087.
4. de Latour RP, Mary JY, Salanoubat C, Terriou L, Etienne G, Mohty M, et al; French Society of Hematology; French Association of Young Hematologists. Paroxysmal nocturnal hemoglobinuria: natural history of disease subcategories. Blood. 2008; 112: 3099-3106.
5. Valla DC. The diagnosis and management of the Budd-Chiari syndrome: consensus and controversies. Hepatology. 2003; 38: 793-803.
6. Ganguli SC, Ramzan NN, McKusick MA, Andrews JC, Phyliky RL, Kamath PS. Budd-Chiari syndrome in patients with hematological disease: a therapeutic challenge. Hepatology. 1998; 27: 1157-1161.
7. Menon KV, Shah V, Kamath PS. The Budd-Chiari syndrome. N Engl J Med. 2004; 350: 578-585.
8. Qi X, He C, Han G, Yin Z, Wu F, Zhang Q, et al. Prevalence of paroxysmal nocturnal hemoglobinuria in Chinese patients with Budd-Chiari syndrome or portal vein thrombosis. J Gastroenterol Hepatol. 2013; 28: 148-152.
9. Hillmen P, Hall C, Marsh JC, Elebute M, Bombara MP, Petro BE, et al. Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2004; 350: 552-559.
10. Hillmen P, Young NS, Schubert J, Brodsky RA, Socié G, Muus P, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006; 355: 1233-1243.
11. Hillmen P, Muus P, Dührsen U, Risitano AM, Schubert J, Luzzatto L, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007; 110: 4123-4128.
12. Richards SJ, Rawstron AC, Hillmen P. Application of flow cytometry to the diagnosis of paroxysmal nocturnal hemoglobinuria. Cytometry. 2000; 42: 223-233.
13. Perelló A, García-Pagán JC, Gilabert R, Suárez Y, Moitinho E, Cervantes F, et al. TIPS is a useful long-term derivative therapy for patients with Budd-Chiari syndrome uncontrolled by medical therapy. Hepatology. 2002; 35: 132-139.
14. Socié G, Mary JY, de Gramont A, Rio B, Leporrier M, Rose C, et al. Paroxysmal nocturnal haemoglobinuria: long-term follow-up and prognostic factors. French Society of Haematology. Lancet. 1996; 348: 573-577.
15. Hill A, Ridley SH, Esser D, Oldroyd RG, Cullen MJ, Kareclas P, et al. Protection of erythrocytes from human complement-mediated lysis by membrane-targeted recombinant soluble CD59: a new approach to PNH therapy. Blood. 2006; 107: 2131-2137.
16. Rother RP, Bell L, Hillmen P, Gladwin MT. The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin: a novel mechanism of human disease. JAMA. 2005; 293: 1653-1662.
17. Maroney SA, Cunningham AC, Ferrel J, Hu R, Haberichter S, Mansbach CM, et al. A GPI-anchored co-receptor for tissue factor pathway inhibitor controls its intracellular trafficking and cell surface expression. J Thromb Haemost. 2006; 4: 1114-1124.
18. Nafa K, Bessler M, Mason P, Vulliamy T, Hillmen P, Castro-Malaspina H, et al. Factor V Leiden mutation investigated by amplification created restriction enzyme site (ACRES) in PNH patients with and without thrombosis. Haematologica. 1996; 81: 540-542.
19. Brodsky RA, Young, NS, Antonioli E., Risitano AM, Schrezenmeier H, Schubert J, et al. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood. 2008; 111: 1840-1847.
20. Kuo GP, Brodsky RA, Kim HS. Catheter-directed thrombolysis and thrombectomy for the Budd-Chiari syndrome in paroxysmal nocturnal hemoglobinuria in three patients. J Vasc Interv Radiol. 2006; 17: 383-387.
21. Brodsky R.A. How I treat paroxysmal nocturnal hemoglobinuria. Blood. 2009; 113: 6522-6527.
22. Hillmen P. Update in paroxysmal nocturnal hemoglobinuria. Clin Adv Hematol Oncol. 2012; 10: 391-393.
23. Emadi A, Brodsky RA. Successful discontinuation of anticoagulation following eculizumab administration in paroxysmal nocturnal hemoglobinuria. Am J Hematol. 2009; 84: 699-701.