Autologous stem cell transplantation in immunoglobulin light chain amyloidosis with factor X deficiency. Issue 1 (January 2016)

• Record Type: Journal Article
• Authors: Cordes, Stefan; Gertz, Morie A.; Buadi, Francis K.; Lin, Yi; Lacy, Martha Q.; Kapoor, Prashant; Kumar, Shaji K.; McCurdy, Arleigh; Dispenzieri, Angela; Dingli, David; Hayman, Suzanne R.; Hogan, William J.; Pruthi, Rajiv K.
• Is Part Of: Blood coagulation and fibrinolysis. Volume 27:Issue 1(2016)
• Publication Date: 2016-01
• Subjects: amyloidosis -- autologous transplantation -- factor X deficiency -- haematopoietic stem cell transplantation -- haemorrhagic disorder; Blood -- Coagulation -- Periodicals; Fibrinolysis -- Periodicals; Hemostasis -- Periodicals; Thrombosis -- Periodicals; Blood Coagulation -- Periodicals; Fibrinolysis -- Periodicals; Hemostasis -- Periodicals; Thrombosis -- Periodicals; 612.115
• DOI: 10.1097/MBC.0000000000000367
• Languages: English
• ISSNs: 0957-5235

Abstract

Abstract : Acquired factor X deficiency and associated haemorrhage can be consequences of immunoglobulin light chain amyloidosis. There are limited data on the safety and efficacy of autologous stem cell transplant (ASCT) on factor X deficiency. We retrospectively reviewed immunoglobulin light chain amyloidosis patients with factor X levels below 50%, not on chronic anticoagulation who underwent ASCT at the Mayo Clinic, Rochester, Minnesota, USA, between April 1995 and December 2011. Twenty-seven of 358 patients (7.5%) met study criteria. Median pre-ASCT factor X was 36% (range: 2–49%). The most frequent and severe bleeding complications occurred in patients with factor X levels below 10%. Peri-procedural prophylaxis included activated recombinant factor VII, fresh frozen plasma and platelet transfusions. Steady-state post-ASCT factor X levels were determined in 12 patients. Post-ASCT factor X levels increased in 100% of patients, with median factor X improvement of +32% (range: +8 to +92%). About 46.2% of patients were no longer factor X deficient after ASCT. The degree of improvement in factor X levels was correlated with an improvement in markers of renal involvement by amyloid. Improvement in factor X correlated with an improvement in the degree of total serum protein (ρ = 0.54; P = 0.04) and proteinuria (ρ = −0.54; P = 0.04). Our findings support the decision to offer ASCT to factor X-deficient patients as both appropriate and efficacious. Abstract : Supplemental Digital Content is available in the text