Clinical experience with nimotuzumab in cuban pediatric patients with brain tumors, 2005 to 2007.

Introduction Nimotuzumab, developed in Cuba, is a humanized monoclonal antibody that targets the epidermal growth factor receptor (EGFR). It has been evaluated in malignant brain tumors in adults and children, and shown to be therapeutically safe and effective in terms of increased survival and improved quality of life. Objective Describe nimotuzumab's safety profile and clinical benefits in terms of disease control and survival in pediatric patients with progressive or recurrent primary brain tumors who were included in an expanded access program. Methods An open, prospective clinical study was designed. Between December 2005 and December 2007, 22 patients were included, all of whom had an histological and/or radiological diagnosis of progressive or recurrent primary brain tumor, classified as high-grade malignant glioblastoma (n=6), diffuse brain stem glioma (n=6), ependymoblastoma (n=5), low-grade glioma (n=4), or thalamic tumor (n=1); life expectancy of at least 4 weeks; and a Karnofsky or Lansky Performance Status score of ≥50. Nimotuzumab was administered on a 100 mg weekly intravenous infusion schedule for 6 to 8 weeks, followed by a bi-weekly maintenance phase, as long as there was no deterioration in the patient's functional capacity. Therapeutic protocols were followed for administration as monotherapy or in combination with chemotherapy and/or radiotherapy. All patients received clinical and imaging follow-up. Results Nimotuzumab was well tolerated in all therapeutic modalities, even with prolonged exposure. A minority of patients reported slight or moderate adverse events, such as vomiting, mucositis and chills, as classified by the Common Terminology Criteria for Adverse Events (CTCAE). The disease was controlled in 64% (14/22) of patients; 6-month and 1-year survival rates were 82% and 64%, respectively; average survival was 20.3 months and median survival, 19 months. Recovery of neurological functions and improvement in general status were notable in patients who attained control of the disease. Conclusions As used in this study, nimotuzumab demonstrated a broad safety profile, making it acceptable for chronic use, and implied clinical benefits in terms of increased survival and improved functional status in these patients, compared to findings described in the literature. These results indicate further studies of this product are warranted.