Main Article Content
Amyloidosis, VWS, Bortezomib
Acquired von Willebrand syndrome (AVWS) is a rare hemorrhagic disorder that occurs in patients with no prior personal or family history of bleeding. Here, we describe a case of AVWS occurring after autologous stem cell transplantation (ASCT). Interestingly, AVWS developed after bortezomib-based induction and conditioning regimens. Recent evidence suggests that the proximity of the bortezomib therapy to the collection of stem cells with consequent depletion of regulatory T cells after the conditioning regimen could explain some of the unusual autoimmune complications reported in patients receiving bortezomib prior to ASCT. In addition, this patient developed a secondary MGUS post-ASCT, which may have also contributed to the AVWS. To the best of our knowledge, this is the first case of post-ASCT AVWS reported. Prospective data is needed to better elucidate the mechanisms by which these unusual complications occur in patients receiving bortezomib prior to ASCT.
1. Rosenzweig M, Giralt S, Landau H. Light-chain amyloidosis: SCT, novel agents and beyond. Bone Marrow Transplant. 2013;48:1022-1027.
2. Jimenez-Zepeda VH, Franke N, Reece DE, et al. Autologous stem cell transplant is an effective therapy for carefully selected patients with AL amyloidosis: experience of a single institution. Br J Haematol. 2014;164:722-728.
3. Roy V. Autologous stem cell transplant for Al amyloidosis. Bone Marrow Res. 2012;2012:238961.
4. Comenzo RL. Amyloidosis. Curr Treat Options Oncol. 2006;7:225-236.
5. Dispenzieri A, Buadi F, Kumar SK, et al. Treatment of Immunoglobulin Light Chain Amyloidosis: Mayo Stratification of Myeloma and Risk-Adapted Therapy (mSMART) Consensus Statement. Mayo Clin Proc. 2015;90:1054-1081.
6. Sanchorawala V, Brauneis D, Shelton AC, et al. Induction Therapy with Bortezomib Followed by Bortezomib-High Dose Melphalan and Stem Cell Transplantation for Light Chain Amyloidosis: Results of a Prospective Clinical Trial. Biol Blood Marrow Transplant. 2015;21:1445-1451.
7. Voisin S, Hamidou M, Lefrancois A, Sigaud M, Mahe B, Trossaert M. Acquired von Willebrand syndrome associated with monoclonal gammopathy: a single-center study of 36 patients. Medicine (Baltimore). 2011;90:404-411.
8. Federici AB RJ, Bucciarelli P, Budde U, van Genderen PJ, Mohri H, Meyer D, Rodeghiero F, Sadler JE. Acquired von Willebrand syndrome: data from an international registry. Thromb Haemost. 2000;84:345-349.
9. Franchini M, Lippi G. Acquired von Willebrand syndrome: an update. Am J Hematol. 2007;82:368-375.
10. Lee A, Sinclair G, Valentine K, James P, Poon MC. Acquired von Willebrand syndrome: von Willebrand factor propeptide to von Willebrand factor antigen ratio predicts remission status. Blood. 2014;124:e1-3.
11. Scott JPV, E. A.; Schroeder, T.; Foster, P. A.; Gill, J. C.; Montgomery, R. R. he Von Willebrand factor propolypeptide, Von Willebrand antigen II , distinguishes acquired Von Willebrand syndrome due to decreased synthesis of Von Willebrand factor from AvWS due to increased clearance of vWF. Blood. 1995;86:(Suppl 1).
12. Federici AB, Stabile F, Castaman G, Canciani MT, Mannucci PM. Treatment of acquired von Willebrand syndrome in patients with monoclonal gammopathy of uncertain significance: comparison of three different therapeutic approaches. Blood. 1998;92:2707-2711.
13. Kos CA, Ward JE, Malek K, et al. Association of acquired von Willebrand syndrome with AL amyloidosis. Am J Hematol. 2007;82:363-367.