Pembrolizumab in Practice: Assessing Safety and Effectiveness Through a Retrospective Frame

Shalini Thomas1, Princy Palatty1, Laxmi Govindraj1

  • 1Department of Pharmacology, Amrita Institute of Medical Science and Research Center, Amrita Vishwa Vidhyapeetham, Ernakulam, IND.

Cureus
|February 20, 2026
PubMed

Insights

Pembrolizumab shows significant effectiveness in treating various cancers, with a 14.5% objective response rate and 61.3% disease control rate. Most adverse drug reactions were mild to moderate, indicating a favorable safety profile for this immunotherapy.

Area of Science:

  • Oncology
  • Immunotherapy
  • Pharmacovigilance

Background:

  • Immune evasion is a key mechanism in tumor progression.
  • Programmed cell death protein 1 (PD-1) inhibitors, like pembrolizumab, modulate T-cell responses to enhance anti-tumor immunity.
  • Pembrolizumab has shown efficacy in metastatic non-small-cell lung cancer and other malignancies.

Purpose of the Study:

  • To evaluate the clinical effectiveness of pembrolizumab across diverse cancer types over a five-year period.
  • To analyze the pattern and characteristics of adverse drug reactions (ADRs) associated with pembrolizumab treatment.

Main Methods:

  • Retrospective analysis of 111 patients receiving at least one dose of pembrolizumab between August 2019 and July 2024.
  • Data collection included patient demographics, pembrolizumab therapy details, documented adverse events, and treatment response using RECIST criteria.
  • Statistical analysis was performed to assess treatment effectiveness and identify factors associated with toxicity.

Main Results:

  • Pembrolizumab demonstrated an objective response rate (ORR) of 14.5% and a disease control rate (DCR) of 61.3% across 22 cancer types.
  • Breast cancer patients exhibited favorable outcomes, with 33.3% achieving complete/partial response and 55.6% stable disease.
  • Adverse events occurred in 80.2% of patients, primarily mild to moderate (Grade 1-2); Grade 3 thrombocytopenia was observed in five patients and successfully managed. Concomitant lenvatinib increased toxicity, particularly thyroid dysfunction (p=0.041). Patients experiencing ADRs showed better clinical outcomes.

Conclusions:

  • Pembrolizumab is an effective treatment option for various cancers, offering a meaningful ORR and DCR with a generally manageable safety profile.
  • Immune-related adverse events are common but typically mild to moderate; careful monitoring is essential, especially when combined with lenvatinib.
  • The occurrence of adverse reactions may correlate with improved clinical outcomes, highlighting the need for personalized treatment strategies and vigilant patient management.

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