Sorafenib for the Treatment of Progressive Metastatic Medullary Thyroid Cancer: Efficacy and Safety Analysis

Autor: Gilberto de Castro, Marcos Tavares, Eduardo Furquim Simao, Marcelo V. Negrao, Ana O. Hoff, Luciana A. Castroneves, Alexander A. L. Jorge, Carla Papadia, Marco Aurélio Vamondes Kulcsar, Paulo M. Hoff, Julia Tizue Fukushima, Ricardo Miguel Costa de Freitas, José Viana Lima
Rok vydání: 2016
Předmět:
Adult
Male
Niacinamide
Oncology
Sorafenib
medicine.medical_specialty
Pathology
Time Factors
Cabozantinib
medicine.drug_class
Endocrinology
Diabetes and Metabolism

Antineoplastic Agents
030209 endocrinology & metabolism
Kaplan-Meier Estimate
Vandetanib
Disease-Free Survival
Tyrosine-kinase inhibitor
Thyroid carcinoma
03 medical and health sciences
chemistry.chemical_compound
0302 clinical medicine
Endocrinology
Internal medicine
Carcinoma
medicine
Humans
Longitudinal Studies
Thyroid Neoplasms
Protein Kinase Inhibitors
Aged
Retrospective Studies
business.industry
Phenylurea Compounds
Medullary thyroid cancer
Middle Aged
medicine.disease
digestive system diseases
Carcinoma
Neuroendocrine

Clinical trial
Treatment Outcome
chemistry
030220 oncology & carcinogenesis
Disease Progression
Female
business
medicine.drug
Zdroj: Thyroid. 26:414-419
ISSN: 1557-9077
1050-7256
DOI: 10.1089/thy.2015.0334
Popis: Treatment of advanced medullary thyroid carcinoma (MTC) was recently improved with the approval of vandetanib and cabozantinib. However, there is still a need to explore sequential therapy with more than one tyrosine kinase inhibitor (TKI) and to explore alternative therapies when vandetanib and cabozantinib are not available. This study reports the authors' experience with sorafenib as a treatment for advanced MTC.This is a retrospective longitudinal study of 13 patients with progressive metastatic MTC treated with sorafenib 400 mg twice daily between December 2011 and January 2015. The primary endpoints were to evaluate response and progression-free survival (PFS) in patients treated with sorafenib outside a clinical trial. The secondary endpoint was an assessment of the toxicity profile. One patient was excluded because of a serious allergic skin rash one week after starting sorafenib.The analysis included 12 patients with metastatic MTC (median age 48 years), 10 with sporadic and 2 with hereditary disease. The median duration of treatment was 11 months, and the median follow-up was 15.5 months. At data cutoff, 2/12 (16%) patients were still on treatment for 16 and 34 months. According to Response Evaluation Criteria in Solid Tumors analysis, 10 (83.3%) patients showed stable disease, and two (16.6%) had progression of disease; no partial response was observed. The median PFS was nine months. However, three patients with extensive and rapidly progressive disease died within three months of sorafenib treatment. The median PFS excluding these three patients was 12 months. Adverse events (AE) occurred in nine (75%) patients. The main AEs were skin toxicity, weight loss, and fatigue. Five (41.6%) patients needed dose reduction, and one patient discontinued treatment because of toxicity.Treatment with sorafenib in progressive metastatic MTC is well tolerated and resulted in disease control and durable clinical benefit in 75% of patients. Sorafenib treatment could be considered when vandetanib and cabozantinib are not available or after failing these drugs.
Databáze: OpenAIRE