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Exp Clin Endocrinol Diabetes 2019; 127(04): 240-246
DOI: 10.1055/s-0044-100378
Article
© Georg Thieme Verlag KG Stuttgart · New York

First Line Sorafenib Treatment for Metastatic Medullary Thyroid Cancer: Efficacy and Safety Analysis

Judit Kocsis
1   Institute of Oncology, Faculty of Medicine, University of Debrecen, Debrecen, Hungary
,
Éva Szekanecz
1   Institute of Oncology, Faculty of Medicine, University of Debrecen, Debrecen, Hungary
,
Ali Bassam
2   Pándy Kálmán Békés County Hospital, Department of Oncology and Radiotherapy, Gyula, Hungary
,
Andrea Uhlyarik
3   Military Hospital Budapest, Department of Oncology
,
Zsuzsanna Pápai
3   Military Hospital Budapest, Department of Oncology
,
Gábor Rubovszky
4   Department of Chemotherapy B and Clinical Pharmacology, National Institute of Oncology, Budapest Hungary
,
Emese Mezősi
5   Ist Department of Internal Medicine, University of Pécs, Pécs, Hungary
,
Károly Rucz
5   Ist Department of Internal Medicine, University of Pécs, Pécs, Hungary
,
Ildikó Garai
6   Scanomed Kft, Hungary
,
Endre Nagy
7   Division of Endocrinology, Department of Medicine, Faculty of Medicine, University of Debrecen, Debrecen, Hungary
,
Iván Uray
1   Institute of Oncology, Faculty of Medicine, University of Debrecen, Debrecen, Hungary
,
Zsolt Horváth
4   Department of Chemotherapy B and Clinical Pharmacology, National Institute of Oncology, Budapest Hungary
› Author Affiliations
Further Information

Publication History

received 22 November 2017
first decision 28 December 2017

accepted 05 January 2018

Publication Date:
05 March 2018 (online)

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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
 

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