PMID- 33745098
OWN - NLM
STAT- MEDLINE
DCOM- 20220202
LR  - 20220202
IS  - 1573-0646 (Electronic)
IS  - 0167-6997 (Linking)
VI  - 39
IP  - 4
DP  - 2021 Aug
TI  - Significant benefit of everolimus in a patient with urothelial bladder cancer 
      harboring a rare M1043I mutation of PIK3CA.
PG  - 1197-1199
LID - 10.1007/s10637-021-01103-8 [doi]
AB  - Urothelial bladder cancer (UBC) is a common malignancy with considerable 
      mortality worldwide. However, the treatment options of UBC are mainly 
      chemotherapy and immunotherapy, as few targeted agents have demonstrated efficacy 
      against UBC. In recent studies, everolimus has exhibited antitumor activity in 
      patients harboring aberrations in the phosphatidylinositol 3-kinase 
      (PI3K)/protein kinase B (Akt)/ mammalian target of rapamycin (mTOR) pathway in 
      multiple tumor types. Herein, we report the case of a patient with metastatic UBC 
      harboring a rare M1043I mutation of PIK3CA which was detected using DNA-based 
      next-generation sequencing. The patient received everolimus as first-line therapy 
      after palliative transurethral resection. The treatment resulted in complete 
      response within 1 month, and the patient achieved a progression-free survival 
      (PFS) of >6 months according to reports from the last follow-up visit. To our 
      knowledge, this is the first reported case of PIK3CA-mutant UBC for which 
      everolimus therapy demonstrated a significant benefit suggesting that the rare 
      M1043I mutation variant may be a potential biomarker of sensitivity to 
      everolimus. Further insights into its mechanism and clinical studies are needed 
      to clarify the effectiveness of everolimus therapy in patients with PIK3CA M1043I 
      mutation.
CI  - (c) 2021. The Author(s), under exclusive licence to Springer Science+Business 
      Media, LLC, part of Springer Nature.
FAU - Pan, Shouhua
AU  - Pan S
AD  - Department of Urology, Shaoxing People's Hospital, Shaoxing, 312000, China.
FAU - Li, Si
AU  - Li S
AD  - The Medical Department, Jiangsu Simcere Diagnostics Co., Ltd, Nanjing, China.
AD  - Nanjing Simcere Medical Laboratory Science Co., Ltd, Nanjing, China.
AD  - The State Key Laboratory of Translational Medicine and Innovative Drug 
      Development, Jiangsu Simcere Diagnostics Co., Ltd, Nanjing, 210002, China.
FAU - Xiao, Mingzhe
AU  - Xiao M
AD  - The Medical Department, Jiangsu Simcere Diagnostics Co., Ltd, Nanjing, China.
AD  - Nanjing Simcere Medical Laboratory Science Co., Ltd, Nanjing, China.
AD  - The State Key Laboratory of Translational Medicine and Innovative Drug 
      Development, Jiangsu Simcere Diagnostics Co., Ltd, Nanjing, 210002, China.
FAU - Chen, Dongsheng
AU  - Chen D
AD  - The Medical Department, Jiangsu Simcere Diagnostics Co., Ltd, Nanjing, China.
AD  - Nanjing Simcere Medical Laboratory Science Co., Ltd, Nanjing, China.
AD  - The State Key Laboratory of Translational Medicine and Innovative Drug 
      Development, Jiangsu Simcere Diagnostics Co., Ltd, Nanjing, 210002, China.
FAU - Li, Junlong
AU  - Li J
AUID- ORCID: 0000-0002-6272-6582
AD  - Department of Urology, Shaoxing People's Hospital, Shaoxing, 312000, China. 
      junlongli_urology@163.com.
LA  - eng
PT  - Case Reports
PT  - Journal Article
DEP - 20210320
PL  - United States
TA  - Invest New Drugs
JT  - Investigational new drugs
JID - 8309330
RN  - 0 (Antineoplastic Agents)
RN  - 9HW64Q8G6G (Everolimus)
RN  - EC 2.7.1.137 (Class I Phosphatidylinositol 3-Kinases)
RN  - EC 2.7.1.137 (PIK3CA protein, human)
SB  - IM
MH  - Aged, 80 and over
MH  - Antineoplastic Agents/*administration & dosage
MH  - Carcinoma, Transitional Cell/drug therapy/genetics/surgery
MH  - Class I Phosphatidylinositol 3-Kinases/*genetics
MH  - Everolimus/*administration & dosage
MH  - Follow-Up Studies
MH  - High-Throughput Nucleotide Sequencing
MH  - Humans
MH  - Male
MH  - Mutation
MH  - Progression-Free Survival
MH  - Treatment Outcome
MH  - Urinary Bladder Neoplasms/*drug therapy/genetics/surgery
OTO - NOTNLM
OT  - Bladder cancer
OT  - Complete response
OT  - Everolimus
OT  - PIK3CA
OT  - mTOR
EDAT- 2021/03/22 06:00
MHDA- 2022/02/03 06:00
CRDT- 2021/03/21 21:25
PHST- 2021/02/09 00:00 [received]
PHST- 2021/03/12 00:00 [accepted]
PHST- 2021/03/22 06:00 [pubmed]
PHST- 2022/02/03 06:00 [medline]
PHST- 2021/03/21 21:25 [entrez]
AID - 10.1007/s10637-021-01103-8 [pii]
AID - 10.1007/s10637-021-01103-8 [doi]
PST - ppublish
SO  - Invest New Drugs. 2021 Aug;39(4):1197-1199. doi: 10.1007/s10637-021-01103-8. Epub 
      2021 Mar 20.