Effectiveness and safety of immune checkpoint inhibitors: A retrospective study in Taiwan

Abstract

BackgroundSince 2012, several immune checkpoint inhibitors have been approved by the Taiwan FDA for various types of cancer treatment. However, none of them are covered by Taiwan National Health Insurance due to the fact that they are expensive, and there is a lack of clinical evidence as to their effectiveness.ObjectivesThis study was aimed toward an exploration of clinical experiences with use of immune checkpoint inhibitors, including indications, prescription types, drug effectiveness, adverse drug event types, and incidence, all of which shall serve as references for future clinical drug use.MethodsThis is a retrospective study focusing on three immune checkpoint inhibitors (ipilimumab, nivolumab, and pembrolizumab), which are available for cancer treatment in Taiwan. We collected data from medical records for the period from January 1st, 2015 to January 12th, 2017 at National Cheng Kung University Hospital (NCKUH), a medical center in southern Taiwan, and recorded these cases until May 31st, 2017. Overall survival (OS) and progression-free survival (PFS) were estimated using the Kaplan-Meier method, and adverse drug reaction odds ratios were analyzed using a chi-square analysis.ResultsThe 50 patients under consideration in this study had used any one of the immune checkpoint inhibitors in NCKUH. Non-small cell lung cancer (n = 24, 48%) accounted for the highest percentage, followed by hepatocellular carcinoma (n = 4, 8%). The median OS was not reached, and the PFS for all immunotherapies was 4.9 months. The median OS period and PFS for non-small cell lung cancer (NSCLC) patients were 13 and 4.9 months, respectively, which were similar to those in many clinical trials. For NSCLC patients, the OS and PFS were only 0.63 and 1.37 months for squamous cell type NSCLC, and for patients who were PD-L1 negative, the OS and PFS were only 11.53 and 2.6 months, respectively. The most common adverse events in this study included fatigue (42%), rashes (22%), nausea (20%), and fever (20%), while one patient developed severe deep venous thrombosis and tissue inflammation, which was not confirmed in previous clinical trials.ConclusionsThe histological subtype, the intensity of the PD-L1 expression, and the timing of treatment affected the NSCLC therapeutic results. It is recommended that clinical tests be conducted in order to enhance therapeutic effectiveness. It is expected that more testing, observation-based studies, and research results will validate their efficacy and the tolerance levels of patients.

Estimation of the Percentage of US Patients With Cancer Who Are Eligible for and Respond to Checkpoint Inhibitor Immunotherapy Drugs

“…Off-label use of checkpoint inhibitor drugs can be notable, with estimates of between 18% and 30%, depending on the drug and study. 22,23 Popular articles have described the use of “desperation oncology,” in which immunotherapy is used to treat a variety of malignant neoplasms in patients nearing death. 24,25 Estimating off-label use is difficult, as there is no reliable registry showing how many patients are exposed to therapy and what percentage respond.…”

Bone Marrow Findings of Immune-Mediated Pure Red Cell Aplasia Following Anti-Programmed Cell Death Receptor-1 Therapy: A Report of Two Cases and Review of Literature

“…Common side effects include fatigue, pruritus, diarrhea, pyrexia, cough, dyspnea, musculoskeletal pain, constipation and nausea. Immune-mediated adverse events such as pneumonitis, colitis, hepatitis and thyroid disorder have been reported [10,11]. With the broad use of anti-PD-1 drugs in clinical practice, rarer side effects are emerging.…”

Adverse Events of Oncologic Immunotherapy and Their Management

“…[475658596061] For example, in a retrospective study of 50 patients with nonsmall cell lung cancer, who were treated with an immune checkpoint inhibitor, the most frequent irAEs were fatigue (42%), rash (22%), nausea (20%), and fever (20%). [62] Similarly, a retrospective analysis to assess the safety profile of nivolumab in 576 patients with advanced melanoma found that 71% of patients experienced irAEs, with the most common irAEs being fatigue (25%), pruritus (17%), diarrhea (13%), and rash (13%). [63]…”