Rapid Response to Sorafenib in Metastatic Medullary Thyroid Carcinoma.

Summary of "Rapid Response to Sorafenib in Metastatic Medullary Thyroid Carcinoma."

To investigate the efficacy of sorafenib in progressive metastatic Medullary Thyroid Carcinoma (MTC), for which there is currently no effective treatment.
Off-label observational study.
Sorafenib 400 mg twice daily was evaluated. The primary endpoint was the objective Response Evaluation Criteria in Solid Tumours (RECIST) score assessed on day 28 and every 12 weeks thereafter. Additional endpoints were time to response, duration of tumour response, tumour-related symptoms, and changes in tumour markers, calcitonin, and CEA measured initially, at 2 weeks, and then every 4 weeks. Therapy duration was 2 weeks, and 3-12 months.
The 5 patients meeting study criteria received sorafenib 400 mg orally twice a day until disease progression or unacceptable toxicity developed. 2 patients showed a partial response with tumour regression of -46% and -36% after 6 and 9 months, respectively, and 2 patients exhibited tumour regression of -14% and -29%, respectively (stable disease). Ultrasound-documented regression of -37% within 2 weeks occurred in 1 patient. Calcitonin decreased within 2 weeks in all patients by -69, -90, -75, -96, and -39%, respectively. 1 patient died because of progressive ascites from acute renal and hepatocellular failure. 2 patients developed grade 3 hand-foot syndrome within the first month, so that sorafenib was interrupted or reduced; other side effects were rash, fatigue, and hair loss. 3 patients remain on sorafenib, 2 at a reduced dosage (600 mg/d).
These data suggest a possible role for sorafenib in the treatment of progressive metastatic MTC.


Endocrine Practice, Molecular Laboratory, Heidelberg, Germany.

Journal Details

This article was published in the following journal.

Name: Experimental and clinical endocrinology & diabetes : official journal, German Society of Endocrinology [and] German Diabetes Ass
ISSN: 1439-3646


DeepDyve research library

PubMed Articles [18045 Associated PubMed Articles listed on BioPortfolio]

Effect of 3'UTR RET Variants on RET mRNA Secondary Structure and Disease Presentation in Medullary Thyroid Carcinoma.

The RET S836S variant has been associated with early onset and increased risk for metastatic disease in medullary thyroid carcinoma (MTC). However, the mechanism by which this variant modulates MTC pa...

Medullary Thyroid Carcinoma Metastasis to the Breast and Axillary Lymph Nodes.

Breast metastases of medullary thyroid carcinoma (MTC) are extremely rare, and only a few cases have been reported in the literature so far. Here, we report a case of metastatic MTC to the breast and ...

Treatment of advanced thyroid cancer: role of molecularly targeted therapies.

Advanced thyroid cancer is not amenable to therapy with conventional cytotoxic chemotherapy. However, newer advances in the understanding of the molecular pathogenesis of different subtypes of thyroid...

C-Cells and their Associated Lesions and Conditions: A Pathologists Perspective.

This paper updates the histopathology and cytopathology of thyroid tumors and proliferations derived from the para-follicular or C cells. Beginning with an historical over view, including the recognit...

Phase 2 trial of sorafenib in children and young adults with refractory solid tumors: A report from the Children's Oncology Group.

Sorafenib is an oral small molecule inhibitor of multiple kinases controlling tumor growth and angiogenesis. The purpose of the phase 2 study was to determine the response rate of sorafenib and gain f...

Clinical Trials [7258 Associated Clinical Trials listed on BioPortfolio]

Sorafenib Phase II Study for Japanese Anaplastic or Medullary Thyroid Carcinoma Patients

The objectives of this study are to evaluate safety, efficacy and pharmacokinetics of sorafenib for the treatment of Japanese patients with anaplastic thyroid carcinoma (ATC) or locally ad...

Sorafenib as Adjuvant to Radioiodine Therapy in Non-Medullary Thyroid Carcinoma

The purpose of this study is to assess whether therapy with Sorafenib reinduces radioiodine uptake in thyroid carcinoma.

Sorafenib Tosylate in Treating Patients With Metastatic, Locally Advanced, or Recurrent Medullary Thyroid Cancer

This phase II trial studies how well sorafenib tosylate works in treating patients with medullary thyroid cancer that has spread to other parts of the body (metastatic), spread to the tiss...

Thyroid Cancer and Sunitinib

Due to arguments showing that angiogenesis could be involved in progression of metastatic thyroid carcinoma and to objective response during previous studies with sunitinib (an angiogenic ...

Study of Anlotinib in Patients With Medullary Thyroid Carcinoma(ALTER01031)

To compare the effects and safety of Anlotinib with placebo in patients with Medullary Thyroid Carcinoma.

Medical and Biotech [MESH] Definitions

A carcinoma composed mainly of epithelial elements with little or no stroma. Medullary carcinomas of the breast constitute 5%-7% of all mammary carcinomas; medullary carcinomas of the thyroid comprise 3%-10% of all thyroid malignancies. (From Dorland, 27th ed; DeVita Jr et al., Cancer: Principles & Practice of Oncology, 3d ed, p1141; Segen, Dictionary of Modern Medicine, 1992)

Similar to MEN2A, it is also caused by mutations of the MEN2 gene, also known as the RET proto-oncogene. Its clinical symptoms include medullary carcinoma (CARCINOMA, MEDULLARY) of THYROID GLAND and PHEOCHROMOCYTOMA of ADRENAL MEDULLA (50%). Unlike MEN2a, MEN2b does not involve PARATHYROID NEOPLASMS. It can be distinguished from MEN2A by its neural abnormalities such as mucosal NEUROMAS on EYELIDS; LIP; and TONGUE, and ganglioneuromatosis of GASTROINTESTINAL TRACT leading to MEGACOLON. It is an autosomal dominant inherited disease.

A form of multiple endocrine neoplasia characterized by the presence of medullary carcinoma (CARCINOMA, MEDULLARY) of the THYROID GLAND, and usually with the co-occurrence of PHEOCHROMOCYTOMA, producing CALCITONIN and ADRENALINE, respectively. Less frequently, it can occur with hyperplasia or adenoma of the PARATHYROID GLANDS. This disease is due to gain-of-function mutations of the MEN2 gene on CHROMOSOME 10 (Locus: 10q11.2), also known as the RET proto-oncogene that encodes a RECEPTOR PROTEIN-TYROSINE KINASE. It is an autosomal dominant inherited disease.

A group of carcinomas which share a characteristic morphology, often being composed of clusters and trabecular sheets of round "blue cells", granular chromatin, and an attenuated rim of poorly demarcated cytoplasm. Neuroendocrine tumors include carcinoids, small ("oat") cell carcinomas, medullary carcinoma of the thyroid, Merkel cell tumor, cutaneous neuroendocrine carcinoma, pancreatic islet cell tumors, and pheochromocytoma. Neurosecretory granules are found within the tumor cells. (Segen, Dictionary of Modern Medicine, 1992)

Mucocellular carcinoma of the ovary, usually metastatic from the gastrointestinal tract, characterized by areas of mucoid degeneration and the presence of signet-ring-like cells. It accounts for 30%-40% of metastatic cancers to the ovaries and possibly 1%-2% of all malignant ovarian tumors. The lesions may not be discovered until the primary disease is advanced, and most patients die of their disease within a year. In some cases, a primary tumor is not found. (From Dorland, 27th ed; Holland et al., Cancer Medicine, 3d ed, p1685)

Quick Search

DeepDyve research library

Relevant Topics

Thyroid Disorders
The thyroid is a butterfly-shaped gland in the neck, just above thecollarbone and is an endocrine gland that make hormones. These Thyroid hormones control the rate of many activities in the body, including how fast the body burns calories and how fast th...

Radiology is the branch of medicine that studies imaging of the body; X-ray (basic, angiography, barium swallows), ultrasound, MRI, CT and PET. These imaging techniques can be used to diagnose, but also to treat a range of conditions, by allowing visuali...

Searches Linking to this Article