Successful treatment of systemic amyloidosis with hepatic involvement and factor X deficiency by high dose melphalan chemotherapy and autologous stem cell reinfusion

European Journal of Haematology

Volumn 72, Issue 3, 2004, Pages 181-185

  Breems, Dimitri A ^a   Sonneveld, Pieter ^a   De Man, Rob A ^a   Leebeek, Frank W G ^a  

^a ERASMUS MEDICAL CENTER   (Netherlands)

Author keywords

AL amyloidosis; Autologous stem cell transplantation; Factor x deficiency; Hepatic amyloidosis; High dose melphalan

Indexed keywords

AMYLOID; BLOOD CLOTTING FACTOR 10; DEXAMETHASONE; DOXORUBICIN; LIVER ENZYME; MELPHALAN; PREDNISONE;

ADULT; AMYLOIDOSIS; ANAMNESIS; ARTICLE; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; BLOOD CLOTTING FACTOR 10 DEFICIENCY; CASE REPORT; CHEMOTHERAPY; CLINICAL EXAMINATION; CLINICAL FEATURE; DIARRHEA; DISEASE COURSE; DISEASE SEVERITY; DRUG MEGADOSE; ENZYME ASSAY; FEVER; FIBRINOLYSIS; HEPATOMEGALY; HUMAN; HUMAN TISSUE; INFUSION; LABORATORY TEST; LEUKOPENIA; LIVER DISEASE; MALE; MUCOSA INFLAMMATION; NEUTROPENIA; PRIORITY JOURNAL; SYSTEMIC DISEASE; TREATMENT OUTCOME;

ALKALINE PHOSPHATASE; AMYLOIDOSIS; ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS; BLOOD COAGULATION DISORDERS; BLOOD COAGULATION TESTS; DEXAMETHASONE; DOXORUBICIN; FACTOR X; FACTOR X DEFICIENCY; GAMMA-GLUTAMYLTRANSFERASE; HEPATOMEGALY; HUMANS; MALE; MELPHALAN; MIDDLE AGED; STEM CELL TRANSPLANTATION; TRANSPLANTATION, AUTOLOGOUS; TREATMENT OUTCOME;

EID: 1342343959     PISSN: 09024441     EISSN: None     Source Type: Journal    
DOI: 10.1046/j.0902-4441.2003.00200.x     Document Type: Article

References (19)

1. FALK RH, COMENZO RL, SKINNER M. The systemic amyloidoses. N Engl J Med 1997;337:898-909.
2. GILLMORE JD, HAWKINS PN, PEPYS MB. Amyloidosis: a review of recent diagnostic and therapeutic developments. Br J Haematol 1997;99:245-256.
3. GERTZ MA, KYLE RA. Hepatic amyloidosis: clinical appraisal in 77 patients. Hepatology 1997;25:118-121.
4. MUMFORD AD, O'DONNELL J, GILLMORE JD, MANNING RA, HAWKINS PN, LAFFAN M. Bleeding symptoms and coagulation abnormalities in 337 patients with AL-amyloidosis. Brit J Haem 2000;110:454-460.
5. LIEBMAN H, CHINOWSKY M, VALDIN J, KENOYER G, FEINSTEIN D. Increased fibrinolysis and amyloidosis. Arch Intern Med 1983;143:678-682.
6. CHANG JC, KANE KK. Pathologic hyperfibrinolysis associated with amyloidosis: clinical response to epsilon amino caproic acid. Am J Clin Pathol 1984;81:382-387.
7. MEYER K, WILLIAMS EC. Fibrinolysis and acquired alpha-2-plasmin inhibitor deficiency in amyloidosis. Am J Med 1985;79:394-396.
8. TAKAHASHI H, KOIKE T, YOSHIDA N, KITAHARA O, HANANO M, SHIBATA A, AOKI N. Excessive fibrinolysis in suspected amyloidosis: demonstration of plasmin-alpha2-plasmin inhibitor complex and von Willebrand factor fragments in plasma. Am J Hematol 1986;23:153-166.
9. SANE DC, PIZZO SV, GREENBERG CS. Elevated urokinase-type plasminogen activator level and bleeding in amyloidosis: case report and literature review. Am J Hematol 1989;31:53-57.
10. LIEBMAN HA, CARFAGNO MK, WEITZ IC, BERARD P, DIIORIO JM, VOSBURGH E, SIMMS RW. Excessive fibrinolysis in amyloidosis associated with elevated plasma single-chain urokinase. Am J Clin Pathol 1992;98:534-541.
11. GLASPY JA. Hemostatic abnormalities in multiple myeloma and related disorders. Hematol Oncol Clin North Am 1992;6:1301-1314.
12. DUNCAN EM, COLE J, CLARKSON AR, LLOYD JV. Poor recovery and short survival of infused factor X in a case of acquired factor X deficiency and amyloidosis. Thromb Haemost 1999;82:1375-1376.
13. BOGGIO L, GREEN D. Recombinant human factor VIIa in the management of amyloid-associated factor X deficiency. Br J Haematol 2001;112:1074-1075.
14. KYLE RA, GERTZ MA. Primary systemic amyloidosis: Clinical and laboratory features in 474 cases. Semin Hematol 1995;32:45-59.
15. SKINNER M, ANDERSON JJ, SIMMS R, FALK R, WANG M, LIBBEY C, JONES LA, COHEN AS. Treatment of 100 patients with primary amyloidosis: A randomized trial of melphalan, prednisone, and colchicine versus cholchicine only. Am J Med 1996;100:290-298.
16. KYLE RA, GERTZ MA, GREIPP PR, WITZIG TE, LUST JA, LACY MQ, THERNEAU TM. A trial of three regimes for primary amyloidosis: Colchicine alone, melphalan and prednisone, and melphalan, prednisone and colchicine. N Engl J Med 1997;336:1202-1207.
17. SANCHORAWALA V, WRIGHT DG, SELDIN DC, DEMBER LM, FINN K, FALK RH, BERK J, QUILLEN K, SKINNER M. An overview of the use of high-dose melphalan with autologous stem cell transplantation for the treatment of AL amyloidosis. Bone Marrow Transplant 2001;28:637-642.
18. CHOUFANI EB, SANCHORAWALA V, ERNST T, QUILLEN K, SKINNER M, WRIGHT DG, SELDIN DC. Acquired factor X deficiency in patients with amyloid light-chain amyloidosis: incidence, bleeding manifestations, and response to high-dose chemotherapy. Blood 2001;97:1885-1887.
19. PEPYS MB, HERBERT J, HUTCHINSON WL, et al. Targeted pharmacological depletion of serum amyloid P component for treatment of human amyloidosis. Nature 2002;417: 254-259.