TY - JOUR
T1 - Efficacy and Long-Term Outcomes of Autologous Stem Cell Transplantation in POEMS Syndrome
T2 - A Nationwide Survey in Japan
AU - Kawajiri-Manako, Chika
AU - Sakaida, Emiko
AU - Ohwada, Chikako
AU - Miyamoto, Toshihiro
AU - Azuma, Taichi
AU - Taguchi, Jun
AU - Mori, Takehiko
AU - Hasegawa, Yuichi
AU - Kondo, Tadakazu
AU - Yujiri, Toshiaki
AU - Yoshimitsu, Makoto
AU - Imada, Kazunori
AU - Kurahashi, Shingo
AU - Kahata, Kaoru
AU - Ichinohe, Tatsuo
AU - Hirokawa, Makoto
AU - Atsuta, Yoshiko
AU - Nakaseko, Chiaki
N1 - Funding Information:
Financial disclosure : This study was supported in part by the Grant-in-Aid for Scientific Research (C) of the Japan Society for the Promotion of Science KAKENHI [15K09494].
Publisher Copyright:
© 2018 The American Society for Blood and Marrow Transplantation
PY - 2018/6
Y1 - 2018/6
N2 - POEMS syndrome is a rare plasma cell dyscrasia presenting with polyneuropathy, λ-type M protein, vascular endothelial growth factor elevation, and systemic manifestations. The standard treatment has not been established, but autologous stem cell transplantation (ASCT) has exhibited effectiveness in this syndrome. However, the efficacy and long-term outcomes of ASCT have not been systematically studied. To clarify the efficacy and long-term outcomes of ASCT-treated patients in Japan, we performed a multicenter retrospective study assessing the clinical course of patients registered to the Japan Society for Hematopoietic Cell Transplantation Transplant Registry Unified Management Program (TRUMP) database. Between January 2000 and December 2011, 95 patients (58 men) were registered to the TRUMP database with a median age of 53 years (range, 28 to 72). The conditioning regimen was melphalan in 93 of 94 patients (99%), and 69 patients (74.2%) received a melphalan dose ≥ 200 mg/m 2 . The median CD34 cell dose was 2.47 × 10 6 /kg (range,.31 to 20). After ASCT, patient performance status was dramatically improved (Eastern Cooperative Oncology Group performance status 0 to 1: 20.0% versus 71.6%, P <.0001). Over a median follow-up of 46.6 months 10 patients died, and 5-year overall survival was 88.8% (n = 95). Progression-free survival at 3 years was 78.3% (n = 70; median follow-up, 54.4 months). These data support the promising role of ASCT in patients with POEMS syndrome for both prolonging survival and improving quality of life. However, disease recurrence remains a major issue for long-term survivors.
AB - POEMS syndrome is a rare plasma cell dyscrasia presenting with polyneuropathy, λ-type M protein, vascular endothelial growth factor elevation, and systemic manifestations. The standard treatment has not been established, but autologous stem cell transplantation (ASCT) has exhibited effectiveness in this syndrome. However, the efficacy and long-term outcomes of ASCT have not been systematically studied. To clarify the efficacy and long-term outcomes of ASCT-treated patients in Japan, we performed a multicenter retrospective study assessing the clinical course of patients registered to the Japan Society for Hematopoietic Cell Transplantation Transplant Registry Unified Management Program (TRUMP) database. Between January 2000 and December 2011, 95 patients (58 men) were registered to the TRUMP database with a median age of 53 years (range, 28 to 72). The conditioning regimen was melphalan in 93 of 94 patients (99%), and 69 patients (74.2%) received a melphalan dose ≥ 200 mg/m 2 . The median CD34 cell dose was 2.47 × 10 6 /kg (range,.31 to 20). After ASCT, patient performance status was dramatically improved (Eastern Cooperative Oncology Group performance status 0 to 1: 20.0% versus 71.6%, P <.0001). Over a median follow-up of 46.6 months 10 patients died, and 5-year overall survival was 88.8% (n = 95). Progression-free survival at 3 years was 78.3% (n = 70; median follow-up, 54.4 months). These data support the promising role of ASCT in patients with POEMS syndrome for both prolonging survival and improving quality of life. However, disease recurrence remains a major issue for long-term survivors.
KW - Autologous stem cell transplantation (ASCT)
KW - POEMS syndrome
KW - Vascular endothelial growth factor (VEGF)
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U2 - 10.1016/j.bbmt.2018.01.026
DO - 10.1016/j.bbmt.2018.01.026
M3 - Article
C2 - 29409882
AN - SCOPUS:85043458147
SN - 1083-8791
VL - 24
SP - 1180
EP - 1186
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
IS - 6
ER -