ORIGINAL ARTICLE Open-label, single-arm phase II study of pemetrexed in the treatment of patients with recurrent or metastatic nasopharyngeal carcinoma who have had prior platinum-based chemotherapy Yang Zhang • Liping Zhao • Peiyu Huang • Jingxun Wu • Fenghua Wang • Yan Huang • Li Zhang Received: 27 May 2012 / Accepted: 1 August 2012 / Published online: 19 August 2012 Ó Springer-Verlag 2012 Abstract Background This study was designed to evaluate the anti- tumor activity and toxicity of single agent pemetrexed in the treatment of patients with recurrent or metastatic nasopharyngeal carcinoma (NPC) who had been treated with platinum-based chemotherapy. Patients and methods This is an open-label, single-arm phase II trial. All patients were treated with pemetrexed. Pemetrexed was given at the dosage of 500 mg/m 2 on day 1, with folic acid and vitamin B12 supplementation, each cycle repeated every 3 weeks. Treatment continued until patient had disease progression or unacceptable toxicities. Results Thirty-five patients were enrolled in this trial. Thirty patients of total were suitable for tumor response assessment. One (2.9 %) patient achieved partial response (PR); 14 (40.0 %) patients, stable disease (SD); and 15 (42.9 %), progressed disease (PD). The progress-free sur- vival (PFS) was 1.5 months (range, 1.5–14.4 months) and median overall survival (OS) was 13.3 months (95 % CI 8.2–18.4 months). Treatment was well tolerated in most patients. Only one patient (2.9 %) experienced grade 4 toxicity of thrombocytopenia and neutropenia. The most seen toxicities were neutropenia (37.1 %), aminotransfer- ase increase (31.4 %), and rash (22.9 %). Conclusions Although pemetrexed induced a response in a patient, it has limited single-agent activity in advanced NPC patients pretreated with platinum-based chemotherapy. Keywords Pemetrexed Á Nasopharyngeal carcinoma Á Metastatic Á Chemotherapy Introduction Nasopharyngeal carcinoma (NPC) is common in Southern China, with an annual incidence of 15–50 per 100,000 [1]. NPC is a highly radiosensitive tumor, and radiotherapy is the standard treatment for this disease. Patients with loco- regionally advanced NPC are reported to have a 5-year survival rate of only 40 % despite treatment with standard radiotherapy (RT). Majority of this group of these patients are died of distant recurrences [2]. The role of chemotherapy in the management of locally recurrent or metastatic disease is regarded as palliative treatment. Platinum-based combination regimens are the standard chemotherapy for metastatic NPC Yang Zhang and Liping Zhao contributed equally to this work. Y. Zhang Á L. Zhao Á P. Huang Á F. Wang Á Y. Huang Á L. Zhang State Key Laboratory of Oncology in South China, Guangzhou, Guangdong, People’s Republic of China Y. Zhang Clinical Trials Center, Cancer Center, Sun Yet-Sen University, Guangzhou, Guangdong, People’s Republic of China L. Zhao Á J. Wu Á F. Wang Á Y. Huang Á L. Zhang (&) Department of Medical Oncology, Cancer Center, Sun Yet-Sen University, 651# Dongfeng Road East, Guangzhou 510060, Guangdong, People’s Republic of China e-mail: zhangli6@mail.sysu.edu.cn P. Huang Department of Nasopharyngeal Carcinoma, Cancer Center, Sun Yet-Sen University, Guangzhou, Guangdong, People’s Republic of China Present Address: J. Wu Department of Medical Oncology, The First Affiliated Hospital of Xiamen University, Xiamen, Fujian, People’s Republic of China 123 Cancer Chemother Pharmacol (2012) 70:611–615 DOI 10.1007/s00280-012-1950-7