Phase II Trial of Trifluridine/Tipiracil Plus Irinotecan in Patients with Advanced, Refractory Biliary Tract Carcinoma.


Journal

The oncologist
ISSN: 1549-490X
Titre abrégé: Oncologist
Pays: England
ID NLM: 9607837

Informations de publication

Date de publication:
03 10 2023
Historique:
received: 21 12 2022
accepted: 23 04 2023
medline: 4 10 2023
pubmed: 20 6 2023
entrez: 20 6 2023
Statut: ppublish

Résumé

We sought to determine the safety and efficacy of trifluridine/tipiracil in combination with irinotecan in a phase II trial setting for refractory, advanced unresectable biliary tract carcinoma (BTC). A total of 28 patients (27 were evaluable) with advanced BTCs who progressed on at least one prior systemic therapy were enrolled and were treated with trifluridine/tipiracil 25 mg/m2 (days 1-5 of 14-day cycle) and irinotecan 180 mg/m2 (day 1 of the 14-day cycle). The primary endpoint for the study was 16-week progression-free survival (PFS16) rate. Overall survival (OS), progression-free survival (PFS), objective response rate (ORR), disease control rate (DCR), and safety were pre-specified secondary endpoints. Of 27 patients, PFS16 rate was 37% (10/27; 95% CI: 19%-58%), thereby meeting the criteria for success for the primary endpoint. The median PFS and OS of the entire cohort were 3.9 months (95% CI: 2.5-7.4) and 9.1 months (95% CI: 8.0-14.3), respectively. In the patients evaluable for tumor response (n = 20), the ORR and DCR were 10% and 50%, respectively. Twenty patients (74.1%) had at least one grade 3 or worse adverse event (AE), and 4 patients (14.8%) had grade 4 AEs. A total of 37% (n = 10/27) and 51.9% (n = 14/27) experienced dose reductions in trifluridine/tipiracil and irinotecan, respectively. Delay in therapy was noted in 56% of the patients while 1 patient discontinued the therapy, primarily due to hematologic AEs. The combination of trifluridine/tipiracil plus irinotecan is a potential treatment option for patients with advanced, refractory BTCs with good functional status and no targetable mutations. A larger randomized trial is needed to confirm these results. (ClinicalTrials.gov Identifier: NCT04072445).

Sections du résumé

BACKGROUND
We sought to determine the safety and efficacy of trifluridine/tipiracil in combination with irinotecan in a phase II trial setting for refractory, advanced unresectable biliary tract carcinoma (BTC).
METHODS
A total of 28 patients (27 were evaluable) with advanced BTCs who progressed on at least one prior systemic therapy were enrolled and were treated with trifluridine/tipiracil 25 mg/m2 (days 1-5 of 14-day cycle) and irinotecan 180 mg/m2 (day 1 of the 14-day cycle). The primary endpoint for the study was 16-week progression-free survival (PFS16) rate. Overall survival (OS), progression-free survival (PFS), objective response rate (ORR), disease control rate (DCR), and safety were pre-specified secondary endpoints.
RESULTS
Of 27 patients, PFS16 rate was 37% (10/27; 95% CI: 19%-58%), thereby meeting the criteria for success for the primary endpoint. The median PFS and OS of the entire cohort were 3.9 months (95% CI: 2.5-7.4) and 9.1 months (95% CI: 8.0-14.3), respectively. In the patients evaluable for tumor response (n = 20), the ORR and DCR were 10% and 50%, respectively. Twenty patients (74.1%) had at least one grade 3 or worse adverse event (AE), and 4 patients (14.8%) had grade 4 AEs. A total of 37% (n = 10/27) and 51.9% (n = 14/27) experienced dose reductions in trifluridine/tipiracil and irinotecan, respectively. Delay in therapy was noted in 56% of the patients while 1 patient discontinued the therapy, primarily due to hematologic AEs.
CONCLUSION
The combination of trifluridine/tipiracil plus irinotecan is a potential treatment option for patients with advanced, refractory BTCs with good functional status and no targetable mutations. A larger randomized trial is needed to confirm these results. (ClinicalTrials.gov Identifier: NCT04072445).

Identifiants

pubmed: 37339254
pii: 7204081
doi: 10.1093/oncolo/oyad144
pmc: PMC10546810
doi:

Substances chimiques

Irinotecan 7673326042
tipiracil NGO10K751P
Trifluridine RMW9V5RW38

Banques de données

ClinicalTrials.gov
['NCT04072445']

Types de publication

Clinical Trial, Phase II Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

917-e966

Informations de copyright

© The Author(s) 2023. Published by Oxford University Press.

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Auteurs

Sri Harsha Tella (SH)

Department of Oncology, Mayo Clinic, Rochester, MN, USA.

Nathan Foster (N)

Department of Biostatistics, Mayo Clinic, Rochester, MN, USA.

Shi Qian (S)

Department of Biostatistics, Mayo Clinic, Rochester, MN, USA.

Tran Nguyen (T)

Department of Oncology, Mayo Clinic, Rochester, MN, USA.

Mitesh J Borad (MJ)

Department of Oncology, Mayo Clinic, Scottsdale, AZ, USA.

Robert R McWilliams (RR)

Department of Oncology, Mayo Clinic, Rochester, MN, USA.

Steven R Alberts (SR)

Department of Oncology, Mayo Clinic, Rochester, MN, USA.

Wen Wee Ma (W)

Department of Oncology, Mayo Clinic, Rochester, MN, USA.

Sakti Chakrabarti (S)

Department of Oncology, University Hospitals Seidman Cancer Center, Case Western Reserve University, Cleveland, OH, USA.

Briant Fruth (B)

Department of Biostatistics, Mayo Clinic, Rochester, MN, USA.

Jaclynn Wessling (J)

Department of Biostatistics, Mayo Clinic, Rochester, MN, USA.

Mindy Hartgers (M)

Department of Oncology, Mayo Clinic, Rochester, MN, USA.

Leslie Washburn (L)

Department of Oncology, Mayo Clinic, Rochester, MN, USA.

Martin E Fernandez-Zapico (ME)

Department of Oncology, Mayo Clinic, Rochester, MN, USA.

Tara L Hogenson (TL)

Department of Oncology, Mayo Clinic, Rochester, MN, USA.

Henry Pitot (H)

Department of Oncology, Mayo Clinic, Rochester, MN, USA.

Zhaohui Jin (Z)

Department of Oncology, Mayo Clinic, Rochester, MN, USA.

Amit Mahipal (A)

Department of Oncology, Mayo Clinic, Rochester, MN, USA.
Department of Oncology, University Hospitals Seidman Cancer Center, Case Western Reserve University, Cleveland, OH, USA.

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