Pembrolizumab May Lead to Favorable Long-Term Outcomes in Head and Neck Cancer

Share this content:
The anti-programmed death 1 (PD-1) antibody pembrolizumab was determined to be an effective second-line option for patients with head and neck cancers.
The anti-programmed death 1 (PD-1) antibody pembrolizumab was determined to be an effective second-line option for patients with head and neck cancers.

Pembrolizumab leads to durable anticancer activity, high survival rates, and a tolerable safety profile among heavily pretreated patients with advanced head and neck squamous cell carcinoma (HNSCC), according to a study published in the British Journal of Cancer.1

The standard first-line treatment for patients with HNSCC is chemotherapy (eg, cetuximab, platinum, plus fluorouracil), but therapeutic options for patients who fail initial therapy have been limited. Immunotherapies, such as the anti-programmed death 1 (PD-1) antibody pembrolizumab, are an effective second-line option for this patient population.

Continue Reading Below

For the KEYNOTE-012 phase 1b study ( Identifier: NCT01848834), researchers enrolled 192 patients with recurrent/metastatic HNSCC into 2 cohorts; patients assigned to the initial cohort received pembrolizumab 10 mg/kg and patients in the expansion cohort received pembrolizumab 200 mg. Tumor response was assessed every 8 weeks, and adverse events (AE) were monitored throughout and for 30 days following treatment.

The long-term analysis represented pooled data from both cohorts collected more than 18 months after the last patient began treatment. At the time of analysis (April 26, 2016), the median follow-up was 9 months, and 88% of patients (n = 168) had discontinued treatment; the most common reasons for stopping treatment included disease progression (n = 124) or AEs (n = 23). Nine percent of patients (n = 18) were still receiving therapy at the conclusion of the study, and 3% (6) had received at least 2 years of pembrolizumab at that point.

The overall response rate was 18% (95% CI, 13-24). The median duration of response was not evaluable, and 85% of treatment responses lasted for at least 6 months. The overall survival rate after 1 year was 38%.

Any grade treatment-related AEs were observed in 64% of patients (n = 123). Grade 3 to 4 AEs occurred in 13% of patients (n = 24) and included AST/ALT elevations, hypothyroidism, fatigue, and appetite decrease; no treatment-related AEs were found to be causal for any reported deaths.

The authors concluded that the “data demonstrate that a subset of patients will receive long-term benefit from treatment with pembrolizumab, a paradigm rarely observed with existing cytotoxic or targeted therapies for recurrent or metastatic HNSCC. Combination studies of pembrolizumab with additional immune-targeting agents and with radiation therapy are under way.”


  1. Mehra R, Seiwert TY, Gupta S, et al. Efficacy and safety of pembrolizumab in recurrent/metastatic head and neck squamous cell carcinoma: pooled analyses after long-term follow-up in KEYNOTE-012 [published online June 29, 2018]. Br J Cancer. doi: 10.1038/s41416-018-0131-9

Related Resources

You must be a registered member of Cancer Therapy Advisor to post a comment.

Sign Up for Free e-newsletters

Regimen and Drug Listings


Bone Cancer Regimens Drugs
Brain Cancer Regimens Drugs
Breast Cancer Regimens Drugs
Endocrine Cancer Regimens Drugs
Gastrointestinal Cancer Regimens Drugs
Gynecologic Cancer Regimens Drugs
Head and Neck Cancer Regimens Drugs
Hematologic Cancer Regimens Drugs
Lung Cancer Regimens Drugs
Other Cancers Regimens
Prostate Cancer Regimens Drugs
Rare Cancers Regimens
Renal Cell Carcinoma Regimens Drugs
Skin Cancer Regimens Drugs
Urologic Cancers Regimens Drugs