TY - JOUR
T1 - Cemiplimab in locally advanced or metastatic cutaneous squamous cell carcinoma
T2 - prospective real-world data from the DRUG Access Protocol
AU - Verkerk, Karlijn
AU - Geurts, Birgit S.
AU - Zeverijn, Laurien J.
AU - van der Noort, Vincent
AU - Verheul, Henk M.W.
AU - Haanen, John B.A.G.
AU - van der Veldt, Astrid A.M.
AU - Eskens, Ferry A.L.M.
AU - Aarts, Maureen J.B.
AU - van Herpen, Carla M.L.
AU - Jalving, Mathilde
AU - Gietema, Jourik A.
AU - Devriese, Lot A.
AU - Labots, Mariette
AU - Barjesteh van Waalwijk van Doorn-Khosrovani, Sahar
AU - Smit, Egbert F.
AU - Bloemendal, Haiko J.
N1 - Funding Information:
The DRUG Access Protocol is supported by all participating pharmaceutical companies: Bayer, Janssen, Lilly, Merck, Novartis, Roche, and Sanofi.The DRUG Access Protocol team thanks Sanofi for providing cemiplimab and their financial support; the health insurance companies for their collaboration with the DRUG Access Protocol team and for providing access through the personalized reimbursement scheme; I. Versteegden, J. Westra, and J.J.M. Schoep for their facilitating services; all participating hospitals for supporting and facilitating the conduct of the DRUG Access Protocol and all patients and their families for their participation.
Publisher Copyright:
© 2024 The Author(s)
PY - 2024/4/1
Y1 - 2024/4/1
N2 - Background: The DRUG Access Protocol provides patients with cancer access to registered anti-cancer drugs that are awaiting reimbursement in the Netherlands and simultaneously collects prospective real-world data (RWD). Here, we present RWD from PD-1 blocker cemiplimab in patients with locally advanced or metastatic cutaneous squamous cell carcinoma (laCSCC; mCSCC). Methods: Patients with laCSCC or mCSCC received cemiplimab 350 mg fixed dose every three weeks. Primary endpoints were objective clinical benefit rate (CBR), defined as objective response (OR) or stable disease (SD) at 16 weeks, physician-assessed CBR, defined as clinician's documentation of improved disease or SD based on evaluation of all available clinical parameters at 16 weeks, objective response rate (ORR), and safety, defined as grade = 3 treatment related adverse events (TRAEs) occurring up to 30 days after last drug administration. Secondary endpoints included duration of response (DoR), progression-free survival (PFS), and overall survival (OS). Findings: Between February 2021 and December 2022, 151 patients started treatment. Objective and physician-assessed CBR were 54.3% (95% CI, 46.0–62.4) and 59.6% (95% CI, 51.3–67.5), respectively. ORR was 35.1% (95% CI, 27.5–43.3). After a median follow-up of 15.2 months, median DoR was not reached. Median PFS and OS were 12.2 (95% CI, 7.0-not reached) and 24.2 months (95% CI, 18.8-not reached), respectively. Sixty-eight TRAEs occurred in 29.8% of patients. Most commonly reported TRAE was a kidney transplant rejection (9.5%). Interpretation: Cemiplimab proved highly effective and safe in this real-world cohort of patients with laCSCC or mCSCC, confirming its therapeutic value in the treatment of advanced CSCC in daily clinical practice. Funding: The DRUG Access Protocol is supported by all participating pharmaceutical companies: Bayer, Janssen, Lilly, Merck, Novartis, Roche, and Sanofi.
AB - Background: The DRUG Access Protocol provides patients with cancer access to registered anti-cancer drugs that are awaiting reimbursement in the Netherlands and simultaneously collects prospective real-world data (RWD). Here, we present RWD from PD-1 blocker cemiplimab in patients with locally advanced or metastatic cutaneous squamous cell carcinoma (laCSCC; mCSCC). Methods: Patients with laCSCC or mCSCC received cemiplimab 350 mg fixed dose every three weeks. Primary endpoints were objective clinical benefit rate (CBR), defined as objective response (OR) or stable disease (SD) at 16 weeks, physician-assessed CBR, defined as clinician's documentation of improved disease or SD based on evaluation of all available clinical parameters at 16 weeks, objective response rate (ORR), and safety, defined as grade = 3 treatment related adverse events (TRAEs) occurring up to 30 days after last drug administration. Secondary endpoints included duration of response (DoR), progression-free survival (PFS), and overall survival (OS). Findings: Between February 2021 and December 2022, 151 patients started treatment. Objective and physician-assessed CBR were 54.3% (95% CI, 46.0–62.4) and 59.6% (95% CI, 51.3–67.5), respectively. ORR was 35.1% (95% CI, 27.5–43.3). After a median follow-up of 15.2 months, median DoR was not reached. Median PFS and OS were 12.2 (95% CI, 7.0-not reached) and 24.2 months (95% CI, 18.8-not reached), respectively. Sixty-eight TRAEs occurred in 29.8% of patients. Most commonly reported TRAE was a kidney transplant rejection (9.5%). Interpretation: Cemiplimab proved highly effective and safe in this real-world cohort of patients with laCSCC or mCSCC, confirming its therapeutic value in the treatment of advanced CSCC in daily clinical practice. Funding: The DRUG Access Protocol is supported by all participating pharmaceutical companies: Bayer, Janssen, Lilly, Merck, Novartis, Roche, and Sanofi.
KW - Cemiplimab
KW - Cutaneous squamous cell carcinoma
KW - Immune checkpoint blockade
KW - Real-world data
U2 - 10.1016/j.lanepe.2024.100875
DO - 10.1016/j.lanepe.2024.100875
M3 - Article
SN - 2666-7762
VL - 39
JO - The Lancet Regional Health – Europe
JF - The Lancet Regional Health – Europe
M1 - 100875
ER -