High-dose therapy and autologous stem cell transplantation in patients with POEMS syndrome: a retrospective study of the Plasma Cell Disorder sub-committee of the Chronic Malignancy Working Party of the European Society for Blood & Marrow Transplantation

Cook, Gordon; Iacobelli, Simona; van Biezen, Anja; Ziegkos, Dimitris; La Blond, Veronique; Abraham, Julie; McQuaker, Grant; Schoenland, Stefan; Rambaldi, Alessandro; Halaburda, Kazimierz; Rovira, Maria; Sica, Simona; Byrne, Jenny; Garcia Sanz, Ramon; Nagler, Aarnon; van der Donk, Niels W.C.J.; Sinisalo, Marjatta; Cook, Mmark; Kroger, Nicolaus; de Witte, Theo; Morris, Curly; Garderet, Laurent

Abstract

POEMS syndrome is a rare para-neoplastic syndrome secondary to a plasma cell dyscrasia. Effective treatment can control the diseaserelated symptom complex. We describe the clinical outcome of autologous stem cell transplantation for patients with POEMS syndrome, determining the impact of patient- and disease-specific factors on prognosis. One hundred and twenty-seven patients underwent an autologous stem cell transplantation at 1997-2010 with a median age of 50 years (range 26-69 years). Median time from diagnosis to autologous stem cell transplantation was 7.5 months with 32% of patients receiving an autologous stem cell transplantation more than 12 months from diagnosis. Engraftment was seen in 97% patients and engraftment syndrome was documented in 23% of autologous stem cell transplantation recipients. Hematologic response was characterized as complete response in 48.5%, partial response in 20.8%, less than partial response in 30.7%. With a median follow up of 48 months (95%CI: 38.3, 58.6), 90% of patients are alive and 16.5% of patients have progressed. The 1-year non-relapse mortality was 3.3%. The 3-year probabilities of progression-free survival and overall survival are 84% and 94%, respectively, with 5-year probabilities of progression-free survival and overall survival of 74% and 89%. In a cohort of graft recipients, detailed organ-specific symptom response demonstrated clear symptom benefit after autologous stem cell transplantation especially in relation to neurological symptom control. The data analysed in this study demonstrate the clinical utility of autologous stem cell transplantation for patients with POEMS syndrome.

Citation

Cook, G., Iacobelli, S., van Biezen, A., Ziegkos, D., La Blond, V., Abraham, J., McQuaker, G., Schoenland, S., Rambaldi, A., Halaburda, K., Rovira, M., Sica, S., Byrne, J., Garcia Sanz, R., Nagler, A., van der Donk, N. W., Sinisalo, M., Cook, M., Kroger, N., de Witte, T., …Garderet, L. (2017). High-dose therapy and autologous stem cell transplantation in patients with POEMS syndrome: a retrospective study of the Plasma Cell Disorder sub-committee of the Chronic Malignancy Working Party of the European Society for Blood & Marrow Transplantation. Haematologica, 102(1), https://doi.org/10.3324/haematol.2016.148460

• Journal Article Type: Article
• Acceptance Date: Sep 13, 2016
• Online Publication Date: Sep 15, 2016
• Publication Date: Jan 4, 2017
• Deposit Date: Dec 14, 2017
• Publicly Available Date: Dec 14, 2017
• Journal: Haematologica
• Print ISSN: 0390-6078
• Electronic ISSN: 1592-8721
• Publisher: Ferrata Storti Foundation
• Peer Reviewed: Peer Reviewed
• Volume: 102
• Issue: 1
• DOI: https://doi.org/10.3324/haematol.2016.148460
• Public URL: https://nottingham-repository.worktribe.com/output/841912
• Publisher URL: http://www.haematologica.org/content/early/2016/09/09/haematol.2016.148460
• Contract Date: Dec 14, 2017