Efficacy and Safety of Pembrolizumab in Patients Enrolled in KEYNOTE-030 in the United States

An Expanded Access Program

Tara C. Gangadhar, Wen Jen Hwu, Michael A. Postow, Omid Hamid, Adil Daud, Roxana S Dronca, Richard W Joseph, Steven J. O'Day, F. S. Hodi, Anna C. Pavlick, Harriet Kluger, Romina P. Oxborough, Aiming Yang, Mihaela Gazdoiu, Debra A. Kush, Scot Ebbinghaus, April K.S. Salama

Research output: Contribution to journalArticle

6 Citations (Scopus)

Abstract

KEYNOTE-030 (ClinicalTrials.gov ID, NCT02083484) was a global expanded access program that allowed access to pembrolizumab, an antiprogrammed death 1 antibody, for patients with advanced melanoma before its regulatory approval. Patients with unresectable stage III/IV melanoma that progressed after standard-of-care therapy, including ipilimumab and, if BRAF V600 mutant, a BRAF inhibitor, were eligible to receive pembrolizumab 2 mg/kg every 3 weeks. Response was assessed by immune-related response criteria by investigator review. Adverse events (AEs) were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events, version 4.0. In the United States, 979 patients enrolled between April and September 2014. Of the 947 evaluable patients, 621 (65.6%) remained on treatment and transitioned to receive commercial pembrolizumab following approval by the Food and Drug Administration, whereas 326 (34.4%) discontinued, most commonly for disease progression (39.6%) or death (26.4%). Objective response rate was 14.5% (95% confidence interval, 12.2%-16.8%) in the treated population (n=947) and 22.1% (95% confidence interval, 18.8%-25.5%) in patients who had ≥1 response assessment reported (n=619). Twelve patients achieved complete response. One hundred eighty-one (19.1%) patients experienced ≥1 treatment-related AE, most commonly general disorders (8.0%), skin/subcutaneous tissue disorders (7.3%), and gastrointestinal disorders (6.4%); 29 (3.1%) patients experienced ≥1 grade 3/4 treatment-related AE. Immune-mediated AEs were also reported. There were no treatment-related deaths. The safety and efficacy observed in this expanded access program were consistent with those previously reported for similar populations and support the use of pembrolizumab for patients with advanced melanoma.

Original languageEnglish (US)
Pages (from-to)334-340
Number of pages7
JournalJournal of Immunotherapy
Volume40
Issue number9
DOIs
StatePublished - 2017

Fingerprint

Safety
Melanoma
Confidence Intervals
Therapeutics
pembrolizumab
National Cancer Institute (U.S.)
Subcutaneous Tissue
United States Food and Drug Administration
Standard of Care
Terminology
Population
Disease Progression
Research Personnel
Skin
Antibodies

Keywords

  • expanded access program
  • immunotherapy
  • melanoma
  • PD-1
  • pembrolizumab

ASJC Scopus subject areas

  • Immunology and Allergy
  • Immunology
  • Pharmacology
  • Cancer Research

Cite this

Efficacy and Safety of Pembrolizumab in Patients Enrolled in KEYNOTE-030 in the United States : An Expanded Access Program. / Gangadhar, Tara C.; Hwu, Wen Jen; Postow, Michael A.; Hamid, Omid; Daud, Adil; Dronca, Roxana S; Joseph, Richard W; O'Day, Steven J.; Hodi, F. S.; Pavlick, Anna C.; Kluger, Harriet; Oxborough, Romina P.; Yang, Aiming; Gazdoiu, Mihaela; Kush, Debra A.; Ebbinghaus, Scot; Salama, April K.S.

In: Journal of Immunotherapy, Vol. 40, No. 9, 2017, p. 334-340.

Research output: Contribution to journalArticle

Gangadhar, TC, Hwu, WJ, Postow, MA, Hamid, O, Daud, A, Dronca, RS, Joseph, RW, O'Day, SJ, Hodi, FS, Pavlick, AC, Kluger, H, Oxborough, RP, Yang, A, Gazdoiu, M, Kush, DA, Ebbinghaus, S & Salama, AKS 2017, 'Efficacy and Safety of Pembrolizumab in Patients Enrolled in KEYNOTE-030 in the United States: An Expanded Access Program', Journal of Immunotherapy, vol. 40, no. 9, pp. 334-340. https://doi.org/10.1097/CJI.0000000000000186
Gangadhar, Tara C. ; Hwu, Wen Jen ; Postow, Michael A. ; Hamid, Omid ; Daud, Adil ; Dronca, Roxana S ; Joseph, Richard W ; O'Day, Steven J. ; Hodi, F. S. ; Pavlick, Anna C. ; Kluger, Harriet ; Oxborough, Romina P. ; Yang, Aiming ; Gazdoiu, Mihaela ; Kush, Debra A. ; Ebbinghaus, Scot ; Salama, April K.S. / Efficacy and Safety of Pembrolizumab in Patients Enrolled in KEYNOTE-030 in the United States : An Expanded Access Program. In: Journal of Immunotherapy. 2017 ; Vol. 40, No. 9. pp. 334-340.
@article{da9a1cf599d64d37a6efe0edb3a15068,
title = "Efficacy and Safety of Pembrolizumab in Patients Enrolled in KEYNOTE-030 in the United States: An Expanded Access Program",
abstract = "KEYNOTE-030 (ClinicalTrials.gov ID, NCT02083484) was a global expanded access program that allowed access to pembrolizumab, an antiprogrammed death 1 antibody, for patients with advanced melanoma before its regulatory approval. Patients with unresectable stage III/IV melanoma that progressed after standard-of-care therapy, including ipilimumab and, if BRAF V600 mutant, a BRAF inhibitor, were eligible to receive pembrolizumab 2 mg/kg every 3 weeks. Response was assessed by immune-related response criteria by investigator review. Adverse events (AEs) were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events, version 4.0. In the United States, 979 patients enrolled between April and September 2014. Of the 947 evaluable patients, 621 (65.6{\%}) remained on treatment and transitioned to receive commercial pembrolizumab following approval by the Food and Drug Administration, whereas 326 (34.4{\%}) discontinued, most commonly for disease progression (39.6{\%}) or death (26.4{\%}). Objective response rate was 14.5{\%} (95{\%} confidence interval, 12.2{\%}-16.8{\%}) in the treated population (n=947) and 22.1{\%} (95{\%} confidence interval, 18.8{\%}-25.5{\%}) in patients who had ≥1 response assessment reported (n=619). Twelve patients achieved complete response. One hundred eighty-one (19.1{\%}) patients experienced ≥1 treatment-related AE, most commonly general disorders (8.0{\%}), skin/subcutaneous tissue disorders (7.3{\%}), and gastrointestinal disorders (6.4{\%}); 29 (3.1{\%}) patients experienced ≥1 grade 3/4 treatment-related AE. Immune-mediated AEs were also reported. There were no treatment-related deaths. The safety and efficacy observed in this expanded access program were consistent with those previously reported for similar populations and support the use of pembrolizumab for patients with advanced melanoma.",
keywords = "expanded access program, immunotherapy, melanoma, PD-1, pembrolizumab",
author = "Gangadhar, {Tara C.} and Hwu, {Wen Jen} and Postow, {Michael A.} and Omid Hamid and Adil Daud and Dronca, {Roxana S} and Joseph, {Richard W} and O'Day, {Steven J.} and Hodi, {F. S.} and Pavlick, {Anna C.} and Harriet Kluger and Oxborough, {Romina P.} and Aiming Yang and Mihaela Gazdoiu and Kush, {Debra A.} and Scot Ebbinghaus and Salama, {April K.S.}",
year = "2017",
doi = "10.1097/CJI.0000000000000186",
language = "English (US)",
volume = "40",
pages = "334--340",
journal = "Journal of Immunotherapy",
issn = "1053-8550",
publisher = "Lippincott Williams and Wilkins",
number = "9",

}

TY - JOUR

T1 - Efficacy and Safety of Pembrolizumab in Patients Enrolled in KEYNOTE-030 in the United States

T2 - An Expanded Access Program

AU - Gangadhar, Tara C.

AU - Hwu, Wen Jen

AU - Postow, Michael A.

AU - Hamid, Omid

AU - Daud, Adil

AU - Dronca, Roxana S

AU - Joseph, Richard W

AU - O'Day, Steven J.

AU - Hodi, F. S.

AU - Pavlick, Anna C.

AU - Kluger, Harriet

AU - Oxborough, Romina P.

AU - Yang, Aiming

AU - Gazdoiu, Mihaela

AU - Kush, Debra A.

AU - Ebbinghaus, Scot

AU - Salama, April K.S.

PY - 2017

Y1 - 2017

N2 - KEYNOTE-030 (ClinicalTrials.gov ID, NCT02083484) was a global expanded access program that allowed access to pembrolizumab, an antiprogrammed death 1 antibody, for patients with advanced melanoma before its regulatory approval. Patients with unresectable stage III/IV melanoma that progressed after standard-of-care therapy, including ipilimumab and, if BRAF V600 mutant, a BRAF inhibitor, were eligible to receive pembrolizumab 2 mg/kg every 3 weeks. Response was assessed by immune-related response criteria by investigator review. Adverse events (AEs) were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events, version 4.0. In the United States, 979 patients enrolled between April and September 2014. Of the 947 evaluable patients, 621 (65.6%) remained on treatment and transitioned to receive commercial pembrolizumab following approval by the Food and Drug Administration, whereas 326 (34.4%) discontinued, most commonly for disease progression (39.6%) or death (26.4%). Objective response rate was 14.5% (95% confidence interval, 12.2%-16.8%) in the treated population (n=947) and 22.1% (95% confidence interval, 18.8%-25.5%) in patients who had ≥1 response assessment reported (n=619). Twelve patients achieved complete response. One hundred eighty-one (19.1%) patients experienced ≥1 treatment-related AE, most commonly general disorders (8.0%), skin/subcutaneous tissue disorders (7.3%), and gastrointestinal disorders (6.4%); 29 (3.1%) patients experienced ≥1 grade 3/4 treatment-related AE. Immune-mediated AEs were also reported. There were no treatment-related deaths. The safety and efficacy observed in this expanded access program were consistent with those previously reported for similar populations and support the use of pembrolizumab for patients with advanced melanoma.

AB - KEYNOTE-030 (ClinicalTrials.gov ID, NCT02083484) was a global expanded access program that allowed access to pembrolizumab, an antiprogrammed death 1 antibody, for patients with advanced melanoma before its regulatory approval. Patients with unresectable stage III/IV melanoma that progressed after standard-of-care therapy, including ipilimumab and, if BRAF V600 mutant, a BRAF inhibitor, were eligible to receive pembrolizumab 2 mg/kg every 3 weeks. Response was assessed by immune-related response criteria by investigator review. Adverse events (AEs) were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events, version 4.0. In the United States, 979 patients enrolled between April and September 2014. Of the 947 evaluable patients, 621 (65.6%) remained on treatment and transitioned to receive commercial pembrolizumab following approval by the Food and Drug Administration, whereas 326 (34.4%) discontinued, most commonly for disease progression (39.6%) or death (26.4%). Objective response rate was 14.5% (95% confidence interval, 12.2%-16.8%) in the treated population (n=947) and 22.1% (95% confidence interval, 18.8%-25.5%) in patients who had ≥1 response assessment reported (n=619). Twelve patients achieved complete response. One hundred eighty-one (19.1%) patients experienced ≥1 treatment-related AE, most commonly general disorders (8.0%), skin/subcutaneous tissue disorders (7.3%), and gastrointestinal disorders (6.4%); 29 (3.1%) patients experienced ≥1 grade 3/4 treatment-related AE. Immune-mediated AEs were also reported. There were no treatment-related deaths. The safety and efficacy observed in this expanded access program were consistent with those previously reported for similar populations and support the use of pembrolizumab for patients with advanced melanoma.

KW - expanded access program

KW - immunotherapy

KW - melanoma

KW - PD-1

KW - pembrolizumab

UR - http://www.scopus.com/inward/record.url?scp=85032871868&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85032871868&partnerID=8YFLogxK

U2 - 10.1097/CJI.0000000000000186

DO - 10.1097/CJI.0000000000000186

M3 - Article

VL - 40

SP - 334

EP - 340

JO - Journal of Immunotherapy

JF - Journal of Immunotherapy

SN - 1053-8550

IS - 9

ER -