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

@entity1 @entity137 @entity353 @entity2857 @entity1295 @entity14820 @entity259 @entity4733 @entity56 @entity74 @entity4149
BACKGROUND: Treatment of advanced @entity259 ( @entity259 ) was recently improved with the approval of @entity4733 and @entity14820 . However, there is still a need to explore sequential therapy with more than one @entity4149 kinase inhibitor (TKI) and to explore alternative therapies when @entity4733 and @entity14820 are not available. This study reports the authors' experience with @entity2857 as a treatment for advanced @entity259 . METHODS: This is a retrospective longitudinal study of 13 @entity1 with progressive metastatic @entity259 treated with @entity2857 400   mg twice daily between December 2011 and January 2015. The primary endpoints were to evaluate response and progression-free survival (PFS) in @entity1 treated with @entity2857 outside a clinical trial. The secondary endpoint was an assessment of the @entity137 profile. One @entity1 was excluded because of a serious @entity56 rash one week after starting @entity2857 . RESULTS: The analysis included 12 @entity1 with metastatic @entity259 (median age 48 years), 10 with sporadic and 2 with @entity74 . The median duration of treatment was 11 months, and the median follow-up was 15.5 months. At data cutoff, 2/12 (16%) @entity1 were still on treatment for 16 and 34 months. According to Response Evaluation Criteria in Solid Tumors analysis, 10 (83.3%) @entity1 showed stable disease, and two (16.6%) had progression of disease; no partial response was observed. The median PFS was nine months. However, three @entity1 with extensive and rapidly progressive disease died within three months of @entity2857 treatment. The median PFS excluding these three @entity1 was 12 months. Adverse events (AE) occurred in nine (75%) @entity1 . The main AEs were @entity137 , @entity353 , and @entity1295 . Five (41.6%) @entity1 needed dose reduction, and one @entity1 discontinued treatment because of @entity137 . CONCLUSIONS: Treatment with @entity2857 in progressive metastatic @entity259 is well tolerated and resulted in disease control and durable clinical benefit in 75% of @entity1 . @entity2857 treatment could be considered when @entity4733 and @entity14820 are not available or after failing these drugs.
