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

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- an...

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Main Authors: 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
Format: Article
Published: Ferrata Storti Foundation 2017
Online Access:https://eprints.nottingham.ac.uk/48729/
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author 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
author_facet 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
author_sort Cook, Gordon
building Nottingham Research Data Repository
collection Online Access
description 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.
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spelling nottingham-487292020-05-04T18:31:45Z https://eprints.nottingham.ac.uk/48729/ 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 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. Ferrata Storti Foundation 2017-01-04 Article PeerReviewed 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 and Garderet, Laurent (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). pp. 160-167. ISSN 0390-6078 http://www.haematologica.org/content/early/2016/09/09/haematol.2016.148460 doi:10.3324/haematol.2016.148460 doi:10.3324/haematol.2016.148460
spellingShingle 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
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
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
title_full 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
title_fullStr 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
title_full_unstemmed 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
title_short 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
title_sort 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
url https://eprints.nottingham.ac.uk/48729/
https://eprints.nottingham.ac.uk/48729/
https://eprints.nottingham.ac.uk/48729/