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
Gordon Cook,
Simona Iacobelli,
Anja van Biezen,
Dimitris Ziagkos,
Veronique LeBlond,
Julie Abraham,
Grant McQuaker,
Stefan Schoenland,
Alessandro Rambaldi,
Kazimierz Halaburda,
Maria Rovira,
Simona Sica,
Jenny Byrne,
Ramon Garcia Sanz,
Arnon Nagler,
Niels W.C.J. van de Donk,
Marjatta Sinisalo,
Mark Cook,
Nicolaus Kröger,
Theo De Witte,
Curly Morris,
Laurant Garderet
Affiliations
Gordon Cook
St James’s Institute of Oncology, Leeds, UK
Simona Iacobelli
Centro Interdipartimentale di Biostatistica e Bioinformatica, Università Tor Vergata, Rome, Italy
Anja van Biezen
EBMT Data Office Leiden, LUMC, the Netherlands
Dimitris Ziagkos
Department of Medical Statistics and Bioinformatics, Leiden University Medical Centre, Leiden, the Netherlands
Veronique LeBlond
Groupe Hospitalier Pitié-Salpetriere, Paris, France
Julie Abraham
CHRU Limoges, France
Grant McQuaker
Beatson, West of Scotland Cancer Centre, Glasgow, UK
Stefan Schoenland
University of Heidelberg, Germany
Alessandro Rambaldi
Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy
Kazimierz Halaburda
Institute of Hematology and Transfusion Medicine, Warsaw, Poland
Maria Rovira
Hospital Clinic, Barcelona, Spain
Simona Sica
Università Cattolica S. Cuore, Rome, Italy
Jenny Byrne
Nottingham University Hospitals Trust, Nottingham, UK
Ramon Garcia Sanz
Universitario de Clinico, Salamanca, Spain
Arnon Nagler
Chaim Sheba Medical Center, Tel-Hashomer, Israel
Niels W.C.J. van de Donk
VU University Medical Center, Amsterdam, the Netherlands
Marjatta Sinisalo
Department of Medicine, Tampere University Hospital, Tampere, Finland
Mark Cook
Queen Elizabeth Hospital, Birmingham, UK
Nicolaus Kröger
University Hospital Hamburg, Germany
Theo De Witte
Radboud university-Nijmegen medical centre, Nijmegen, the Netherlands
Curly Morris
Queens University, Belfast, Northern Ireland, UK and
POEMS syndrome is a rare para-neoplastic syndrome secondary to a plasma cell dyscrasia. Effective treatment can control the disease-related 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 between 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 repsonse 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 analyzed in this study demonstrate the clinical utility of autologous stem cell transplantation for patients with POEMS syndrome.
