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

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Breems DA, Sonneveld P, de Man RA, Leebeek FWG. Successful treatment of systemic amyloidosis with hepatic involvement and factor X deficiency by high dose melphalan chemotherapy and autologous stem cell reinfusion.

Eur J Haematol 2004: 72: 181–185. © Blackwell Munksgaard 2004. Abstract:

Systemic amyloidosis with hepatic involvement is a rare disorder, which is characterized by the deposits of amyloid fibrils in the liver. The prognosis is poor and the median survival is 13 months. Bleeding problems resulting from coagulopathy frequently complicates systemic amyloidosis. We present two patients with a severe factor X deficiency and hepatomegaly as the presenting abnormalities of systemic amyloidosis. One of the patients was treated with high dose melphalan chemotherapy and autologous stem cell reinfusion, resulting in a normalization of the liver enzyme tests and the factor X level. The diagnosis and treatment of systemic amyloidosis with hepatic involvement and the management of the multifactorial coagulopathy in these cases is discussed.
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Keywords: AL amyloidosis; autologous stem cell transplantation; factor x deficiency; hepatic amyloidosis; high dose melphalan

Document Type: Research Article

Affiliations: 1: Hematology 2: Gastroenterology, Erasmus Medical Center, Rotterdam, The Netherlands

Publication date: March 1, 2004

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