PMID- 37931801
OWN - NLM
STAT- MEDLINE
DCOM- 20240205
LR  - 20240206
IS  - 2666-6367 (Electronic)
IS  - 2666-6367 (Linking)
VI  - 30
IP  - 2
DP  - 2024 Feb
TI  - Long-Term Outcomes of Autologous Stem Cell Transplantation in Patients with Newly 
      Diagnosed POEMS Syndrome.
PG  - 207.e1-207.e7
LID - S2666-6367(23)01646-9 [pii]
LID - 10.1016/j.jtct.2023.11.001 [doi]
AB  - POEMS (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and 
      skin changes) syndrome is a rare form of plasma cell dyscrasia often treated with 
      high-dose chemotherapy and autologous stem cell transplantation (ASCT). ASCT has 
      resulted in satisfactory and sustained therapeutic outcomes. However, a 
      substantial number of patients eventually experience disease progression, 
      requiring second-line treatment. Therefore, it would be of further benefit to 
      identify patients who will acquire the best long-term survival after ASCT. The 
      aim of this study was to fully reveal the outcomes of patients undergoing ASCT in 
      a large series with long-term follow-up. Long-term outcomes of 239 patients with 
      newly diagnosed POEMS syndrome undergoing ASCT at a single center were evaluated 
      retrospectively. Rates of hematologic complete response (CR(H)) and vascular 
      endothelial growth factor (VEGF) complete response (CR(V)) were 57.3% and 68.6%, 
      respectively, with 90.5% of patients achieving an overall clinical response. At a 
      median follow-up of 94 months, the 5-year overall survival (OS) rate was 92.8%, 
      and the 5-year time to next-line treatment (TTNT) rate was 72.2% (median TTNT, 96 
      months). Patients achieving CR(H) (5-year TTNT rate, 82.5% versus 60.7%; P < 
      .0001) or CR(V) (5-year TTNT rate 83.7% versus 54.2%; P < .0001) had better 
      survival outcomes compared to non-CR group patients. Dual hematologic and VEGF 
      complete responses carry further benefit for survival (median TTNT, 129 months 
      versus 68 months; P < .0001). Seven cases of second primary malignancy were 
      recorded, all of which were solid tumors. Front-line ASCT resulted in excellent 
      long-term survival in patients with POEMS syndrome, with the best survival 
      observed in those achieving dual hematologic and VEGF CRs.
CI  - Copyright (c) 2023 The American Society for Transplantation and Cellular Therapy. 
      Published by Elsevier Inc. All rights reserved.
FAU - Li, An-An
AU  - Li AA
AD  - Department of Hematology, Peking Union Medical College Hospital, Chinese Academy 
      of Medical Sciences, Peking Union Medical College, Beijing, China.
FAU - Gao, Xue-Min
AU  - Gao XM
AD  - Department of Hematology, Peking Union Medical College Hospital, Chinese Academy 
      of Medical Sciences, Peking Union Medical College, Beijing, China.
FAU - Zhao, Hao
AU  - Zhao H
AD  - Department of Hematology, Peking Union Medical College Hospital, Chinese Academy 
      of Medical Sciences, Peking Union Medical College, Beijing, China.
FAU - Shen, Kai-Ni
AU  - Shen KN
AD  - Department of Hematology, Peking Union Medical College Hospital, Chinese Academy 
      of Medical Sciences, Peking Union Medical College, Beijing, China.
FAU - Zhang, Lu
AU  - Zhang L
AD  - Department of Hematology, Peking Union Medical College Hospital, Chinese Academy 
      of Medical Sciences, Peking Union Medical College, Beijing, China.
FAU - Cao, Xin-Xin
AU  - Cao XX
AD  - Department of Hematology, Peking Union Medical College Hospital, Chinese Academy 
      of Medical Sciences, Peking Union Medical College, Beijing, China.
FAU - Li, Jian
AU  - Li J
AD  - Department of Hematology, Peking Union Medical College Hospital, Chinese Academy 
      of Medical Sciences, Peking Union Medical College, Beijing, China; State Key 
      Laboratory of Common Mechanism Research for Major Diseases, Beijing, China. 
      Electronic address: lijian@pumch.cn.
LA  - eng
PT  - Journal Article
DEP - 20231104
PL  - United States
TA  - Transplant Cell Ther
JT  - Transplantation and cellular therapy
JID - 101774629
RN  - 0 (Vascular Endothelial Growth Factor A)
SB  - IM
MH  - Humans
MH  - *Hematopoietic Stem Cell Transplantation/methods
MH  - *POEMS Syndrome/therapy/drug therapy
MH  - Vascular Endothelial Growth Factor A/therapeutic use
MH  - Retrospective Studies
MH  - Treatment Outcome
MH  - Transplantation, Autologous/methods
OTO - NOTNLM
OT  - Autologous stem cell transplantation
OT  - Overall survival
OT  - POEMS syndrome
OT  - Time to next-line treatment
EDAT- 2023/11/07 00:42
MHDA- 2024/02/05 06:43
CRDT- 2023/11/06 19:16
PHST- 2023/09/23 00:00 [received]
PHST- 2023/10/30 00:00 [revised]
PHST- 2023/11/01 00:00 [accepted]
PHST- 2024/02/05 06:43 [medline]
PHST- 2023/11/07 00:42 [pubmed]
PHST- 2023/11/06 19:16 [entrez]
AID - S2666-6367(23)01646-9 [pii]
AID - 10.1016/j.jtct.2023.11.001 [doi]
PST - ppublish
SO  - Transplant Cell Ther. 2024 Feb;30(2):207.e1-207.e7. doi: 
      10.1016/j.jtct.2023.11.001. Epub 2023 Nov 4.