Skip to main page content
Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Multicenter Study
. 2017 Jan;102(1):160-167.
doi: 10.3324/haematol.2016.148460. Epub 2016 Sep 15.

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

Affiliations
Free PMC article
Multicenter Study

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 et al. Haematologica. .
Free PMC article

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.

Figures

Figure 1.
Figure 1.
The clinical presentation of engraftment syndrome (ESy) in POEMS syndrome patients undergoing autologous stem cell transplantation (ASCT) (n=29).
Figure 2.
Figure 2.
Response to autologous stem cell transplantation (ASCT) in patients with POEMS syndrome. (A) Cumulative incidence of complete hematologic response (CHR) post ASCT. (B) Individual symptomatic domain score modification pre- and post-ASCT, where the Y-axis represents the absolute number of patients (n). Comparative analysis was performed using the Stuart-Maxwell test to determine statistically significant differences on comparative groups of outcomes. (C) Domain-specific response grading post-ASCT, where the Y-axis represents the absolute number of patients (n). CR: complete response; “Keeps CR”: no symptoms pre- and remain asymptomatic post ASCT; “No change”: mild, moderate and severe before ASCT remain in a similar category post ASCT.
Figure 3.
Figure 3.
(A) Cumulative rate of disease progression and (B) progression-free survival (PFS) of patients with POEMS syndrome undergoing an autologous stem cell transplantation (ASCT). (C) PFS in sub-group assessed for organ response. Shaded area represents 95% confidence intervals.
Figure 4.
Figure 4.
Cumulative non-relapse mortality of patients with POEMS syndrome undergoing an autologous stem cell transplantation (ASCT).
Figure 5.
Figure 5.
Overall survival of patients with POEMS syndrome undergoing an autologous stem cell transplantation (ASCT).

Similar articles

See all similar articles

Cited by 6 articles

See all "Cited by" articles

Publication types

MeSH terms

Feedback