scholarly journals Efficacy and Tolerability of Pharmacotherapy Options for the Treatment of Medullary Thyroid Cancer

2012 ◽  
Vol 6 ◽  
pp. CMO.S8305 ◽  
Author(s):  
Hari A. Deshpande ◽  
Khushboo Sheth ◽  
Julie A. Sosa ◽  
Sanziana A. Roman

Metastatic and unresectable medullary thyroid carcinoma (MTC) is often difficult to treat as it is relatively unresponsive to radiation and conventional chemotherapy. This emphasizes the importance of the development of targeted therapies for advanced MTC. Vandetanib was approved by the US Food and Drug Administration for the treatment of symptomatic or progressive MTC in patients with advanced disease in April 2011. This therapy proved to be a breakthrough in the management of MTC. We review the efficacy and safety of this novel treatment and other treatments that are being evaluated in this disease.


2018 ◽  
Vol 29 ◽  
pp. viii476
Author(s):  
J. Hernando Cubero ◽  
E. Grande ◽  
P. Jimenez Fonseca ◽  
C. Villabona ◽  
J.M. Trigo Perez ◽  
...  


2018 ◽  
Vol 127 (04) ◽  
pp. 240-246 ◽  
Author(s):  
Judit Kocsis ◽  
Éva Szekanecz ◽  
Ali Bassam ◽  
Andrea Uhlyarik ◽  
Zsuzsanna Pápai ◽  
...  

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.



2015 ◽  
Vol 81 (1) ◽  
pp. 1-8 ◽  
Author(s):  
Faris K. Azar ◽  
Stephanie L. Lee ◽  
Jennifer E. Rosen

Medullary thyroid carcinoma is uncommon but accounts for approximately 4 to 5 per cent of all thyroid cancers. Although most cases are sporadic, close to one-fourth of cases result from germline mutations in the RET proto-oncogene. These mutations are clinically important because they predict the earliest age of medullary thyroid cancer diagnosis and clinical aggressiveness, which guide individualized management. This review covers the presentation, diagnosis, workup, current management, and future directions of the management of medullary thyroid carcinoma. Today's chance for cure depends on early and appropriate surgical resection. Further investigation of the cellular signaling pathways shown to be essential for the growth and spread of medullary thyroid carcinoma remains an active field with hope for providing targeted systemic therapy for patients with progressive disease.



2017 ◽  
Vol 8 (5) ◽  
pp. 5-11
Author(s):  
Zoya S. Matveeva ◽  
Anatoliy F. Romanchishen ◽  
Alexandr V. Gostimsky ◽  
Kristina V. Vabalayte

The article presents results of diagnostics, surgical treatment and follow-up of patients with rare hereditary-conditioned forms of thyroid cancer – medullary thyroid carcinoma in content of multiple endocrine neoplasia syndrome. Particular attention is paid to the examination and tactics of treatment of children and adolescents with family genetically confirmed Sipple syndrome. The disease is diagnosed in 4 families. Syndrome of multiple endocrine neoplasia 2a type we found in 7 (0.024%) of 29,325 children and adult patients. All the children were from families in which one of the blood relatives suffered medullary thyroid cancer. The family nature of the disease was confirmed by molecular genetic studies that revealed mutations in C634 (T1900C) in the 11 exon of the RET gene. Only in 3 out of 7 cases thyroidectomy was prophylactic. Four children were fond foci of medullary carcinoma in the removed thyroid gland. In total, 22 operations were performed for the members of 4 families suffering from the family syndrome MEN-2a. The article shows that if a patient is diagnosed Sippl's syndrome, all his blood relatives need to be checked for the mutation of the RET gene to identify familial medullary thyroid cancer, adenomas of parathyroid glands and pheochromocytomas. Early removal of the thyroid gland (in children under the age of 5 years) prevents medullary cancer, and timely diagnosis and adequate surgical removal of neoplasms of parathyroid glands and adrenal glands ensure recovery of the patients. Closest relatives should be checked for the level of calcium and calcitonin, catecholamines, vanillylmandelic acid and metanephrine, ACTH, cancer-embryonic antigen.





Thyroid ◽  
2014 ◽  
Vol 24 (9) ◽  
pp. 1329-1340 ◽  
Author(s):  
Mario E. Lacouture ◽  
Kathryn Ciccolini ◽  
Richard T. Kloos ◽  
Mark Agulnik


2003 ◽  
Vol 49 (5) ◽  
pp. 45-47
Author(s):  
A. A. Ilyin ◽  
P. O. Rumyantsev ◽  
P. A. Isayev ◽  
V. S. Medvedev ◽  
B. M. Vtyurin ◽  
...  

This article focuses on sporadic and familial variants of medullary thyroid cancer.



2020 ◽  
Vol 2020 ◽  
pp. 1-4
Author(s):  
Richard J. Robbins ◽  
Jessica S. Thomas ◽  
Patricia Mejia Osuna ◽  
Jawairia Shakil

We report the case of a woman with a sporadic medullary thyroid carcinoma. Genomic analysis found that her tumor did not contain any common RET mutations but did harbor a BRAF V600E mutation. Only one other well-confirmed example of the BRAF V600E mutation has been reported in an MTC patient. We conclude that this common BRAF mutation may independently drive neoplastic transformation of human parafollicular C cells.



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