Efficacy and Long-Term Outcomes of Autologous Stem Cell Transplantation in POEMS Syndrome: A Nationwide Survey in Japan

Chika Kawajiri-Manako, Emiko Sakaida, Chikako Ohwada, Toshihiro Miyamoto, Taichi Azuma, Jun Taguchi, Takehiko Mori, Yuichi Hasegawa, Tadakazu Kondo, Toshiaki Yujiri, Makoto Yoshimitsu, Kazunori Imada, Shingo Kurahashi, Kaoru Kahata, Tatsuo Ichinohe, Makoto Hirokawa, Yoshiko Atsuta, Chiaki Nakaseko

Research output: Contribution to journalArticle

5 Citations (Scopus)

Abstract

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/m2. The median CD34 cell dose was 2.47 × 106/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.

Original languageEnglish
JournalBiology of Blood and Marrow Transplantation
DOIs
Publication statusAccepted/In press - 2018 Jan 1

Fingerprint

POEMS Syndrome
Stem Cell Transplantation
Japan
Melphalan
Registries
Databases
Transplants
Paraproteinemias
Survival
Surveys and Questionnaires
Polyneuropathies
Cell Transplantation
Vascular Endothelial Growth Factor A
Multicenter Studies
Disease-Free Survival
Survivors
Retrospective Studies
Quality of Life
Recurrence

Keywords

  • Autologous stem cell transplantation (ASCT)
  • POEMS syndrome
  • Vascular endothelial growth factor (VEGF)

ASJC Scopus subject areas

  • Hematology
  • Transplantation

Cite this

Efficacy and Long-Term Outcomes of Autologous Stem Cell Transplantation in POEMS Syndrome : A Nationwide Survey in Japan. / Kawajiri-Manako, Chika; Sakaida, Emiko; Ohwada, Chikako; Miyamoto, Toshihiro; Azuma, Taichi; Taguchi, Jun; Mori, Takehiko; Hasegawa, Yuichi; Kondo, Tadakazu; Yujiri, Toshiaki; Yoshimitsu, Makoto; Imada, Kazunori; Kurahashi, Shingo; Kahata, Kaoru; Ichinohe, Tatsuo; Hirokawa, Makoto; Atsuta, Yoshiko; Nakaseko, Chiaki.

In: Biology of Blood and Marrow Transplantation, 01.01.2018.

Research output: Contribution to journalArticle

Kawajiri-Manako, C, Sakaida, E, Ohwada, C, Miyamoto, T, Azuma, T, Taguchi, J, Mori, T, Hasegawa, Y, Kondo, T, Yujiri, T, Yoshimitsu, M, Imada, K, Kurahashi, S, Kahata, K, Ichinohe, T, Hirokawa, M, Atsuta, Y & Nakaseko, C 2018, 'Efficacy and Long-Term Outcomes of Autologous Stem Cell Transplantation in POEMS Syndrome: A Nationwide Survey in Japan', Biology of Blood and Marrow Transplantation. https://doi.org/10.1016/j.bbmt.2018.01.026
Kawajiri-Manako, Chika ; Sakaida, Emiko ; Ohwada, Chikako ; Miyamoto, Toshihiro ; Azuma, Taichi ; Taguchi, Jun ; Mori, Takehiko ; Hasegawa, Yuichi ; Kondo, Tadakazu ; Yujiri, Toshiaki ; Yoshimitsu, Makoto ; Imada, Kazunori ; Kurahashi, Shingo ; Kahata, Kaoru ; Ichinohe, Tatsuo ; Hirokawa, Makoto ; Atsuta, Yoshiko ; Nakaseko, Chiaki. / Efficacy and Long-Term Outcomes of Autologous Stem Cell Transplantation in POEMS Syndrome : A Nationwide Survey in Japan. In: Biology of Blood and Marrow Transplantation. 2018.
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abstract = "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/m2. The median CD34 cell dose was 2.47 × 106/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.",
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AU - Miyamoto, Toshihiro

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AU - Mori, Takehiko

AU - Hasegawa, Yuichi

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AU - Yujiri, Toshiaki

AU - Yoshimitsu, Makoto

AU - Imada, Kazunori

AU - Kurahashi, Shingo

AU - Kahata, Kaoru

AU - Ichinohe, Tatsuo

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AU - Nakaseko, Chiaki

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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/m2. The median CD34 cell dose was 2.47 × 106/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.

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