title: Autologous stem cell transplantation in immunoglobulin light chain amyloidosis with factor X deficiency.
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.
authors.affiliation: aDepartment of Internal Medicine bDivision of Hematology, Mayo Clinic, Rochester, Minnesota, USA.
authors.forename: Stefan
authors.initials: S
authors.lastname: Cordes
authors.forename: Morie A
authors.initials: MA
authors.lastname: Gertz
authors.forename: Francis K
authors.initials: FK
authors.lastname: Buadi
authors.forename: Yi
authors.initials: Y
authors.lastname: Lin
authors.forename: Martha Q
authors.initials: MQ
authors.lastname: Lacy
authors.forename: Prashant
authors.initials: P
authors.lastname: Kapoor
authors.forename: Shaji K
authors.initials: SK
authors.lastname: Kumar
authors.forename: Arleigh
authors.initials: A
authors.lastname: McCurdy
authors.forename: Angela
authors.initials: A
authors.lastname: Dispenzieri
authors.forename: David
authors.initials: D
authors.lastname: Dingli
authors.forename: Suzanne R
authors.initials: SR
authors.lastname: Hayman
authors.forename: William J
authors.initials: WJ
authors.lastname: Hogan
authors.forename: Rajiv K
authors.initials: RK
authors.lastname: Pruthi
mesh_terms: D000686:Amyloidosis / Q000150:complications*; D005171:Factor X Deficiency / Q000628:therapy*; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007147:Immunoglobulin Light Chains / Q000097:blood*; D008297:Male; D012189:Retrospective Studies; D019172:Transplantation Conditioning / Q000379:methods*; D014182:Transplantation, Autologous / Q000379:methods*; D016896:Treatment Outcome
chemical_list: D007147:Immunoglobulin Light Chains