PMID- 35315510
OWN - NLM
STAT- MEDLINE
DCOM- 20220510
LR  - 20220707
IS  - 1097-0142 (Electronic)
IS  - 0008-543X (Linking)
VI  - 128
IP  - 11
DP  - 2022 Jun 1
TI  - A phase 2 study of sorafenib combined with conventional therapies in refractory 
      central nervous system leukemia.
PG  - 2138-2147
LID - 10.1002/cncr.34182 [doi]
AB  - BACKGROUND: Patients with refractory central nervous system leukemia (CNSL) have 
      a dismal prognosis and lack effective therapy. Case reports have shown that 
      sorafenib is effective against brain metastases, including leukemia. METHODS: To 
      explore the efficacy of sorafenib combined with conventional therapies for 
      refractory CNSL, a phase 2 study was conducted. The primary end point was the 
      complete remission rate (CRR) within 8 weeks of treatment. Secondary end points 
      included the overall response rate (ORR), event-free survival (EFS), overall 
      survival (OS), and adverse events (AEs). RESULTS: Twenty-six patients with 
      refractory CNSL were enrolled; they included 17 with isolated CNSL, 7 with 
      hematological relapse, and 2 with another extramedullary relapse. After 8 weeks 
      of treatment, 21 patients achieved complete remission, 2 achieved partial 
      remission, and 3 achieved no remission for a CRR of 80.8% (95% CI, 62.1%-91.5%) 
      and an ORR of 88.5% (95% CI, 71.0%-96.0%). Twenty patients survived, and 6 died. 
      The 2-year EFS and OS rates were 75.0% (95% CI, 54.5%-88.3%) and 76.9% (95% CI, 
      54.2%-90.4%), respectively. Six patients experienced grade 3 or 4 
      treatment-related AEs, including moderate chronic graft-vs-host disease (n = 3), 
      grade 3 or 4 acute graft-vs-host disease (n = 2), and grade 3 skin rash (n = 1). 
      No treatment-related deaths occurred during the therapy of refractory CNSL. 
      CONCLUSIONS: Sorafenib combined with conventional therapies is effective and safe 
      for refractory CNSL. LAY SUMMARY: Sorafenib combined with conventional therapies 
      is effective and safe for refractory central nervous system leukemia.
CI  - (c) 2022 American Cancer Society.
FAU - Chen, Xiaoxia
AU  - Chen X
AD  - Department of Hematology, Nanfang Hospital, Southern Medical University, 
      Guangzhou, China.
FAU - Huang, Junwei
AU  - Huang J
AD  - Department of Hematology, Nanfang Hospital, Southern Medical University, 
      Guangzhou, China.
FAU - Xu, Na
AU  - Xu N
AD  - Department of Hematology, Nanfang Hospital, Southern Medical University, 
      Guangzhou, China.
FAU - Fan, Zhiping
AU  - Fan Z
AD  - Department of Hematology, Nanfang Hospital, Southern Medical University, 
      Guangzhou, China.
FAU - Nie, Danian
AU  - Nie D
AD  - Department of Hematology, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, 
      Guangzhou, China.
FAU - Huang, Fen
AU  - Huang F
AD  - Department of Hematology, Nanfang Hospital, Southern Medical University, 
      Guangzhou, China.
FAU - Sun, Qixin
AU  - Sun Q
AD  - Department of Geriatrics, Hematology and Oncology Ward, Guangzhou First People's 
      Hospital, School of Medicine, South China University of Technology, Guangzhou, 
      China.
FAU - Zhang, Xinyou
AU  - Zhang X
AD  - Department of Hematology, Shenzhen People's Hospital, Shenzhen, China.
FAU - Liang, Xinquan
AU  - Liang X
AD  - Department of Hematology, First People's Hospital of Chenzhou, Chenzhou, China.
FAU - Shi, Pengcheng
AU  - Shi P
AD  - Department of Hematology, Nanfang Hospital, Southern Medical University, 
      Guangzhou, China.
FAU - Wang, Zhixiang
AU  - Wang Z
AD  - Department of Hematology, Nanfang Hospital, Southern Medical University, 
      Guangzhou, China.
FAU - Liu, Hui
AU  - Liu H
AD  - Department of Hematology, Nanfang Hospital, Southern Medical University, 
      Guangzhou, China.
FAU - Xu, Jun
AU  - Xu J
AD  - Department of Hematology, Nanfang Hospital, Southern Medical University, 
      Guangzhou, China.
FAU - Dai, Min
AU  - Dai M
AD  - Department of Hematology, Nanfang Hospital, Southern Medical University, 
      Guangzhou, China.
FAU - Yu, Guopan
AU  - Yu G
AUID- ORCID: 0000-0002-5732-0318
AD  - Department of Hematology, Nanfang Hospital, Southern Medical University, 
      Guangzhou, China.
FAU - Zhang, Yu
AU  - Zhang Y
AD  - Department of Hematology, Nanfang Hospital, Southern Medical University, 
      Guangzhou, China.
FAU - Sun, Jing
AU  - Sun J
AD  - Department of Hematology, Nanfang Hospital, Southern Medical University, 
      Guangzhou, China.
FAU - Liu, Qifa
AU  - Liu Q
AUID- ORCID: 0000-0003-4015-3952
AD  - Department of Hematology, Nanfang Hospital, Southern Medical University, 
      Guangzhou, China.
FAU - Xuan, Li
AU  - Xuan L
AUID- ORCID: 0000-0002-6176-7411
AD  - Department of Hematology, Nanfang Hospital, Southern Medical University, 
      Guangzhou, China.
LA  - eng
PT  - Clinical Trial, Phase II
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20220322
PL  - United States
TA  - Cancer
JT  - Cancer
JID - 0374236
RN  - 9ZOQ3TZI87 (Sorafenib)
SB  - IM
MH  - Central Nervous System
MH  - *Central Nervous System Neoplasms/drug therapy
MH  - *Graft vs Host Disease
MH  - Humans
MH  - *Leukemia
MH  - Recurrence
MH  - Retrospective Studies
MH  - Sorafenib
OTO - NOTNLM
OT  - acute leukemia (AL)
OT  - combination strategy
OT  - conventional central nervous system (CNS)-directed therapy
OT  - refractory central nervous system leukemia (CNSL)
OT  - sorafenib
EDAT- 2022/03/23 06:00
MHDA- 2022/05/11 06:00
CRDT- 2022/03/22 08:59
PHST- 2022/01/28 00:00 [revised]
PHST- 2021/11/10 00:00 [received]
PHST- 2022/02/18 00:00 [accepted]
PHST- 2022/03/23 06:00 [pubmed]
PHST- 2022/05/11 06:00 [medline]
PHST- 2022/03/22 08:59 [entrez]
AID - 10.1002/cncr.34182 [doi]
PST - ppublish
SO  - Cancer. 2022 Jun 1;128(11):2138-2147. doi: 10.1002/cncr.34182. Epub 2022 Mar 22.