PMID- 37457302
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20230718
IS  - 1758-8340 (Print)
IS  - 1758-8359 (Electronic)
IS  - 1758-8340 (Linking)
VI  - 15
DP  - 2023
TI  - Updated survival outcomes with ivosidenib in patients with previously treated 
      IDH1-mutated intrahepatic-cholangiocarcinoma: an Italian real-world experience.
PG  - 17588359231171574
LID - 10.1177/17588359231171574 [doi]
LID - 17588359231171574
AB  - BACKGROUND: The results of the phase III ClarIDHy trial led to the FDA approval 
      of ivosidenib as a therapeutic option for patients with locally advanced or 
      metastatic cholangiocarcinoma (CCA) harboring isocitrate dehydrogenase 1 (IDH1) 
      mutations. We recently published the first data on the use of ivosidenib in a 
      real-world setting. OBJECTIVE: Here we report the updated survival results of 11 
      patients with locally advanced or metastatic IDH1-mutated CCA who received 
      ivosidenib in clinical practice. PATIENTS AND METHODS: Patients treated with 
      ivosidenib as second- and third-line treatments for advanced CCA have been 
      collected with the aim to evaluate the survival outcomes. A molecular study has 
      been performed by next generation sequencing essay. RESULTS: Overall, 11 patients 
      were included. After a median follow-up of 13.7 months, median progression-free 
      survival from the start of treatment with ivosidenib was 4.4 months (95% CI: 
      2.0-5.8), whereas median overall survival was 15 months (95% CI: 6.6-15.0) 
      regardless of treatment line. Disease control rate was 63%, with two patients 
      achieving a partial response (18%). Eighteen percent of patients experienced at 
      least one treatment-related adverse events (AEs), but no grade ⩾3 was reported. 
      The most frequently observed grade 2 AEs were prolonged QT interval and 
      hypomagnesemia. A molecular profiling was performed on 8 out of 11 patients, 
      highlighting TP53, BAP1, CDKN2A, and CDKN2B as the most common co-altered genes 
      in these patients. CONCLUSION: The present update confirms the results of our 
      previous real-world experience on the use of ivosidenib in IDH1-mutated CCA. 
      Real-world evidence on larger numbers of patients is needed to confirm our 
      findings.
CI  - (c) The Author(s), 2023.
FAU - Rimini, Margherita
AU  - Rimini M
AD  - Vita-Salute University San Raffaele, Milan, Italy.
AD  - Department of Oncology, IRCCS San Raffaele Hospital, via Olgettina N. 60, Milan 
      20132, Italy.
FAU - Burgio, Valentina
AU  - Burgio V
AD  - Department of Oncology, IRCCS San Raffaele Hospital, Milan, Italy.
FAU - Antonuzzo, Lorenzo
AU  - Antonuzzo L
AD  - Clinical Oncology Unit, Careggi University Hospital, Florence, Italy.
AD  - Department of Experimental and Clinical Medicine, University of Florence, 
      Florence, Italy.
FAU - Rimassa, Lorenza
AU  - Rimassa L
AUID- ORCID: 0000-0001-9957-3615
AD  - Department of Biomedical Sciences, Humanitas University, Pieve Emanuele (Milan), 
      Italy.
AD  - Medical Oncology and Hematology Unit, Humanitas Cancer Center, IRCCS Humanitas 
      Research Hospital, Rozzano (Milan), Italy.
FAU - Oneda, Ester
AU  - Oneda E
AD  - Department of Oncology, Poliambulanza Hospital of Brescia, Brescia, Italy.
AD  - Oncology Unit 1, Veneto Institute of Oncology IOV - IRCCS, Padua, Italy.
FAU - Solda, Caterina
AU  - Solda C
AD  - Medical Oncology 1, Veneto Institute of Oncology IOV - IRCCS, Padua, Italy.
FAU - Cito, Pasqua
AU  - Cito P
AD  - Oncologia Medica, Ospedale San Pio Di Castellaneta, Taranto, Italy.
FAU - Nasti, Guglielmo
AU  - Nasti G
AD  - Abdominal Oncology Division, Istituto Nazionale Tumori IRCCS Fondazione Pascale - 
      IRCCS Di Napoli, Naples, Italy.
FAU - Lavacchi, Daniele
AU  - Lavacchi D
AD  - Clinical Oncology Unit, Careggi University Hospital, Florence, Italy.
FAU - Zanuso, Valentina
AU  - Zanuso V
AD  - Department of Biomedical Sciences, Humanitas University, Pieve Emanuele (Milan), 
      Italy.
AD  - Medical Oncology and Hematology Unit, Humanitas Cancer Center, IRCCS Humanitas 
      Research Hospital, Rozzano (Milan), Italy.
FAU - Rizzato, Mario Domenico
AU  - Rizzato MD
AD  - Medical Oncology 1, Veneto Institute of Oncology IOV - IRCCS, Padua, Italy.
AD  - Department of Surgical, Oncological and Gastroenterological Sciences, University 
      of Padua, Padua, Italy.
FAU - Zaniboni, Alberto
AU  - Zaniboni A
AD  - Department of Oncology, Poliambulanza Hospital of Brescia, Brescia, Italy.
AD  - Oncology Unit 1, Veneto Institute of Oncology IOV - IRCCS, Padua, Italy.
FAU - Ottaiano, Alessandro
AU  - Ottaiano A
AD  - Abdominal Oncology Division, Istituto Nazionale Tumori IRCCS Fondazione Pascale - 
      IRCCS Di Napoli, Naples, Italy.
FAU - Persano, Mara
AU  - Persano M
AD  - Department of Oncology, University Hospital of Cagliari, Cagliari, Italy.
FAU - Cornara, Noemi
AU  - Cornara N
AUID- ORCID: 0000-0001-6007-5520
AD  - Department of Oncology, IRCCS San Raffaele Hospital, Milan, Italy.
FAU - Scartozzi, Mario
AU  - Scartozzi M
AD  - Department of Oncology, University Hospital of Cagliari, Cagliari, Italy.
FAU - Cascinu, Stefano
AU  - Cascinu S
AD  - Vita-Salute University San Raffaele, Milan, Italy.
FAU - Casadei-Gardini, Andrea
AU  - Casadei-Gardini A
AD  - Vita-Salute University San Raffaele, Milan, Italy.
LA  - eng
PT  - Journal Article
DEP - 20230712
PL  - England
TA  - Ther Adv Med Oncol
JT  - Therapeutic advances in medical oncology
JID - 101510808
PMC - PMC10345913
OTO - NOTNLM
OT  - IDH1 mutation
OT  - cholangiocarcinoma
OT  - ivosidenib
OT  - next generation sequencing
OT  - target therapy
COIS- LR has received consulting fees from Amgen, ArQule, AstraZeneca, Basilea, Bayer, 
      BMS, Celgene, Eisai, Exelixis, Genenta, Hengrui, Incyte, Ipsen, IQVIA, Lilly, 
      MSD, Nerviano Medical Sciences, Roche, Sanofi, Servier, Taiho Oncology, 
      Zymeworks; lecture fees from AbbVie, Amgen, Bayer, Eisai, Gilead, Incyte, Ipsen, 
      Lilly, Merck Serono, Roche, Sanofi; travel expenses from AstraZeneca; and 
      institutional research funding from Agios, ARMO BioSciences, AstraZeneca, 
      BeiGene, Eisai, Exelixis, Fibrogen, Incyte, Ipsen, Lilly, MSD, Nerviano Medical 
      Sciences, Roche, Zymeworks. CS has received consulting fees from Roche, 
      AstraZeneca, Eisai. The other coauthors declare to have no conflict of interest.
EDAT- 2023/07/17 06:42
MHDA- 2023/07/17 06:43
PMCR- 2023/07/12
CRDT- 2023/07/17 04:36
PHST- 2023/01/17 00:00 [received]
PHST- 2023/04/05 00:00 [accepted]
PHST- 2023/07/17 06:43 [medline]
PHST- 2023/07/17 06:42 [pubmed]
PHST- 2023/07/17 04:36 [entrez]
PHST- 2023/07/12 00:00 [pmc-release]
AID - 10.1177_17588359231171574 [pii]
AID - 10.1177/17588359231171574 [doi]
PST - epublish
SO  - Ther Adv Med Oncol. 2023 Jul 12;15:17588359231171574. doi: 
      10.1177/17588359231171574. eCollection 2023.