Abstract
Background Medullary thyroid cancer (MTC) is a rare disease, the prognosis of advanced and
metastatic disease is poor and few therapeutic options are available in this setting.
Based on the results of phase II and III studies with sorafenib in differentiated
thyroid cancer and the lack of availability of registered tyrosine kinase inhibitors,
vandetabin and cabozantinib in Hungary, we designed a uncontrolled, prospective efficacy
and safety study of patients with metastatic MTC treated with first-line sorafenib
in five Hungarian oncology centers.
Methods Ten consecutive patients with progressive or symptomatic metastatic MTC were included
and started sorafenib 400 mg twice a day between June 2012 and March 2016. The primary
end point was median progression-free survival (mPFS). Secondary endpoints included
disease control rate, biochemical response, symptomatic response and toxicity.
Results Four patients achieved partial remission (40%) according to RECIST 1.1 evaluation.
Five patients had stable disease beyond 12 months (50%) and one patient had progressive
disease (10%). Median PFS was 19.1 months. The disease control rate was 90%. Association
between radiologic response and biochemical or symptomatic response was inconsistent.
Most common side effects were Grade 1-2 fatigue (60%), palmar-plantar erythrodysesthesia,
rash/dermatitis 50-50%, alopecia 40%.
Conclusions In our prospective case series in patients with MTC first-line sorafenib showed
at least similar efficacy as in other small phase II trials and case reports. Based
on comparable efficacy with registered tyrosine kinase inhibitors and it’s manageable
toxicity profile, we believe that sorafenib has role in the sequential treatment of
MTC.
Key words
sorafenib - medullary thyroid cancer - first-line - progression free survival - safety
