Real-World Data on Ivosidenib in Patients with Previously Treated Isocitrate Dehydrogenase 1-Mutated Intrahepatic Cholangiocarcinomas : An Early Exploratory Analysis
© 2022. The Author(s), under exclusive licence to Springer Nature Switzerland AG..
BACKGROUND: The results of the phase III ClarIDHy trial have led to US FDA approval of ivosidenib as a therapeutic option for patients with locally advanced or metastatic cholangiocarcinoma (CCA) harboring isocitrate dehydrogenase 1 (IDH1) mutations.
OBJECTIVE: In this study, we report the first real-world experience including eight patients with previously treated locally advanced or metastatic IDH1-mutated CCA treated with ivosidenib.
PATIENTS AND METHODS: Patients treated with ivosidenib as second and third line for advanced CCA were collected with the aim of evaluating the survival outcomes. A molecular study has been performed by next-generation sequencing assay.
RESULTS: After a median follow up of 9.4 months, median progression-free survival (PFS) from the start of treatment with ivosidenib was 4.4 months (95% confidence interval [CI] 3.3-5.8), whereas median overall survival (OS) was not reached. The disease control rate was 62.5%, with two patients achieving a partial response (25%); 12.5% of patients experienced a treatment-related adverse event (AE), but no grade 3 or higher AEs were reported. The observed grade 2 AEs were prolonged QT interval and hypomagnesemia (25% of the sample). Molecular profiling was performed on six of eight patients, highlighting TP53, BAP1, CDKN2A and CDKN2B as the most common co-altered genes in these patients.
CONCLUSION: Efficacy outcomes were consistent with those reported in the ClarIDHy trial. Real-world experiences on larger samples are needed in order to confirm our results.
| Medienart: |
E-Artikel |
|---|
| Erscheinungsjahr: |
2022 |
|---|---|
| Erschienen: |
2022 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:17 |
|---|---|
| Enthalten in: |
Targeted oncology - 17(2022), 5 vom: 17. Sept., Seite 591-596 |
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
Rimini, Margherita [VerfasserIn] |
|---|
| Links: |
|---|
| Themen: |
EC 1.1.1.41 |
|---|
| Anmerkungen: |
Date Completed 28.09.2022 Date Revised 13.10.2022 published: Print-Electronic Citation Status MEDLINE |
|---|
| doi: |
10.1007/s11523-022-00917-7 |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
NLM346316847 |
|---|
| LEADER | 01000naa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM346316847 | ||
| 003 | DE-627 | ||
| 005 | 20231226031138.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 231226s2022 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1007/s11523-022-00917-7 |2 doi | |
| 028 | 5 | 2 | |a pubmed24n1154.xml |
| 035 | |a (DE-627)NLM346316847 | ||
| 035 | |a (NLM)36114954 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a Rimini, Margherita |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Real-World Data on Ivosidenib in Patients with Previously Treated Isocitrate Dehydrogenase 1-Mutated Intrahepatic Cholangiocarcinomas |b An Early Exploratory Analysis |
| 264 | 1 | |c 2022 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ƒaComputermedien |b c |2 rdamedia | ||
| 338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Date Completed 28.09.2022 | ||
| 500 | |a Date Revised 13.10.2022 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a © 2022. The Author(s), under exclusive licence to Springer Nature Switzerland AG. | ||
| 520 | |a BACKGROUND: The results of the phase III ClarIDHy trial have led to US FDA approval of ivosidenib as a therapeutic option for patients with locally advanced or metastatic cholangiocarcinoma (CCA) harboring isocitrate dehydrogenase 1 (IDH1) mutations | ||
| 520 | |a OBJECTIVE: In this study, we report the first real-world experience including eight patients with previously treated locally advanced or metastatic IDH1-mutated CCA treated with ivosidenib | ||
| 520 | |a PATIENTS AND METHODS: Patients treated with ivosidenib as second and third line for advanced CCA were collected with the aim of evaluating the survival outcomes. A molecular study has been performed by next-generation sequencing assay | ||
| 520 | |a RESULTS: After a median follow up of 9.4 months, median progression-free survival (PFS) from the start of treatment with ivosidenib was 4.4 months (95% confidence interval [CI] 3.3-5.8), whereas median overall survival (OS) was not reached. The disease control rate was 62.5%, with two patients achieving a partial response (25%); 12.5% of patients experienced a treatment-related adverse event (AE), but no grade 3 or higher AEs were reported. The observed grade 2 AEs were prolonged QT interval and hypomagnesemia (25% of the sample). Molecular profiling was performed on six of eight patients, highlighting TP53, BAP1, CDKN2A and CDKN2B as the most common co-altered genes in these patients | ||
| 520 | |a CONCLUSION: Efficacy outcomes were consistent with those reported in the ClarIDHy trial. Real-world experiences on larger samples are needed in order to confirm our results | ||
| 650 | 4 | |a Journal Article | |
| 650 | 7 | |a Pyridines |2 NLM | |
| 650 | 7 | |a Isocitrate Dehydrogenase |2 NLM | |
| 650 | 7 | |a EC 1.1.1.41 |2 NLM | |
| 650 | 7 | |a ivosidenib |2 NLM | |
| 650 | 7 | |a Q2PCN8MAM6 |2 NLM | |
| 650 | 7 | |a Glycine |2 NLM | |
| 650 | 7 | |a TE7660XO1C |2 NLM | |
| 700 | 1 | |a Burgio, Valentina |e verfasserin |4 aut | |
| 700 | 1 | |a Antonuzzo, Lorenzo |e verfasserin |4 aut | |
| 700 | 1 | |a Rimassa, Lorenza |e verfasserin |4 aut | |
| 700 | 1 | |a Oneda, Ester |e verfasserin |4 aut | |
| 700 | 1 | |a Lavacchi, Daniele |e verfasserin |4 aut | |
| 700 | 1 | |a Personeni, Nicola |e verfasserin |4 aut | |
| 700 | 1 | |a Ratti, Francesca |e verfasserin |4 aut | |
| 700 | 1 | |a Pedica, Federica |e verfasserin |4 aut | |
| 700 | 1 | |a Della Corte, Angelo |e verfasserin |4 aut | |
| 700 | 1 | |a Persano, Mara |e verfasserin |4 aut | |
| 700 | 1 | |a De Cobelli, Francesco |e verfasserin |4 aut | |
| 700 | 1 | |a Aldrighetti, Luca |e verfasserin |4 aut | |
| 700 | 1 | |a Scartozzi, Mario |e verfasserin |4 aut | |
| 700 | 1 | |a Cascinu, Stefano |e verfasserin |4 aut | |
| 700 | 1 | |a Casadei-Gardini, Andrea |e verfasserin |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t Targeted oncology |d 2009 |g 17(2022), 5 vom: 17. Sept., Seite 591-596 |w (DE-627)NLM187616825 |x 1776-260X |7 nnns |
| 773 | 1 | 8 | |g volume:17 |g year:2022 |g number:5 |g day:17 |g month:09 |g pages:591-596 |
| 856 | 4 | 0 | |u http://dx.doi.org/10.1007/s11523-022-00917-7 |3 Volltext |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |d 17 |j 2022 |e 5 |b 17 |c 09 |h 591-596 | ||