title: 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.
abstract: 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.
authors.affiliation: St James's Institute of Oncology, Leeds, UK g.cook@leeds.ac.uk.
authors.forename: Gordon
authors.initials: G
authors.lastname: Cook
authors.affiliation: Centro Interdipartimentale di Biostatistica e Bioinformatica, Università Tor Vergata, Rome, Italy.
authors.forename: Simona
authors.initials: S
authors.lastname: Iacobelli
authors.affiliation: EBMT Data Office Leiden, LUMC, the Netherlands.
authors.forename: Anja
authors.initials: A
authors.lastname: van Biezen
authors.affiliation: Department of Medical Statistics and Bioinformatics, Leiden University Medical Centre, Leiden, the Netherlands.
authors.forename: Dimitris
authors.initials: D
authors.lastname: Ziagkos
authors.affiliation: Groupe Hospitalier Pitié-Salpetriere, Paris, France.
authors.forename: Veronique
authors.initials: V
authors.lastname: LeBlond
authors.affiliation: CHRU Limoges, France.
authors.forename: Julie
authors.initials: J
authors.lastname: Abraham
authors.affiliation: Beatson, West of Scotland Cancer Centre, Glasgow, UK.
authors.forename: Grant
authors.initials: G
authors.lastname: McQuaker
authors.affiliation: University of Heidelberg, Germany.
authors.forename: Stefan
authors.initials: S
authors.lastname: Schoenland
authors.affiliation: Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy.
authors.forename: Alessandro
authors.initials: A
authors.lastname: Rambaldi
authors.affiliation: Institute of Hematology and Transfusion Medicine, Warsaw, Poland.
authors.forename: Kazimierz
authors.initials: K
authors.lastname: Halaburda
authors.affiliation: Hospital Clinic, Barcelona, Spain.
authors.forename: Maria
authors.initials: M
authors.lastname: Rovira
authors.affiliation: Università Cattolica S. Cuore, Rome, Italy.
authors.forename: Simona
authors.initials: S
authors.lastname: Sica
authors.affiliation: Nottingham University Hospitals Trust, Nottingham, UK.
authors.forename: Jenny
authors.initials: J
authors.lastname: Byrne
authors.affiliation: Universitario de Clinico, Salamanca, Spain.
authors.forename: Ramon Garcia
authors.initials: RG
authors.lastname: Sanz
authors.affiliation: Chaim Sheba Medical Center, Tel-Hashomer, Israel.
authors.forename: Arnon
authors.initials: A
authors.lastname: Nagler
authors.affiliation: VU University Medical Center, Amsterdam, the Netherlands.
authors.forename: Niels W C J
authors.initials: NW
authors.lastname: van de Donk
authors.affiliation: Department of Medicine, Tampere University Hospital, Tampere, Finland.
authors.forename: Marjatta
authors.initials: M
authors.lastname: Sinisalo
authors.affiliation: Queen Elizabeth Hospital, Birmingham, UK.
authors.forename: Mark
authors.initials: M
authors.lastname: Cook
authors.affiliation: University Hospital Hamburg, Germany.
authors.forename: Nicolaus
authors.initials: N
authors.lastname: Kröger
authors.affiliation: Radboud university-Nijmegen medical centre, Nijmegen, the Netherlands.
authors.forename: Theo
authors.initials: T
authors.lastname: De Witte
authors.affiliation: Queens University, Belfast, Northern Ireland, UK and.
authors.forename: Curly
authors.initials: C
authors.lastname: Morris
authors.affiliation: Hôpital Saint Antoine, Paris, France.
authors.forename: Laurant
authors.initials: L
authors.lastname: Garderet
mesh_terms: D000328:Adult; D000368:Aged; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D018450:Disease Progression; D005260:Female; D006084:Graft Rejection; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods; D006801:Humans; D008297:Male; D008875:Middle Aged; D016878:POEMS Syndrome / Q000175:diagnosis / Q000401:mortality / Q000628:therapy*; D012189:Retrospective Studies; D016019:Survival Analysis; D061665:Time-to-Treatment; D019172:Transplantation Conditioning; D014182:Transplantation, Autologous; D016896:Treatment Outcome