Long-Term Control of Hypercortisolism by Vandetanib in a Case of Medullary Thyroid Carcinoma with a Somatic RET Mutation

Paepegaey, Anne-Cécile; Cochand‐Priollet, Béatrix; Louiset, Estelle; Sarfati, P O; Alifano, Marco; Burnichon, Nelly; Bienvenu-Perrard, M.; Lahlou, Najiba; Bricaire, Léopoldine; Groussin, Lionel

doi:10.1089/thy.2016.0334

Cited by 19 publications

(12 citation statements)

References 9 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…On the other hand, during the vandetanib treatment, a mild ACTH response to desmopressin was reported, suggesting a TKI anti-secretory action. No signs of recurrence of hypercortisolism were reported after 3 years and 8 months from the beginning of the therapy, indicating the long-term effectiveness of vandetanib in maintaining the control of hypercortisolism in MTC-related CS 54…”

Section: Clinical Trials With Vandetanib In Mtcmentioning

confidence: 78%

See 1 more Smart Citation

Evaluating vandetanib in the treatment of medullary thyroid cancer: patient-reported outcomes

Fallahi¹,

Ferrari²,

Elia³

et al. 2019

CMAR

View full text Add to dashboard Cite

Medullary thyroid cancers (MTCs) are neuroendocrine tumors, which secrete calcitonin and carcinoembryonic antigen, both of which can serve as tumor markers. Extensive and accurate surgical resection is the primary treatment for MTC, whereas the use of external beam radiotherapy is limited. Moreover, since MTC is derived from thyroid parafollicular cells or C cells, it is not responsive to either radioiodine or thyroid-stimulating hormone suppression, and therefore, they cannot be considered as treatment strategies. Traditional therapies for advanced or metastatic progressive medullary thyroid cancer (pMTC) are poorly effective. Among the new approaches tested in clinical trials, targeted chemotherapies with tyrosine kinase inhibitors (TKIs) are now available and they represent effective interventions for progressive disease, with additional investigational options emerging. This paper reviews the efficacy and safety of vandetanib in patients with a pMTC, as it has been shown to improve progression-free survival (30.5 vs 19.3 months in controls). Vandetanib is approved by the FDA and EMA for symptomatic or progressive MTC in patients with unresectable locally advanced or metastatic disease in adults, adolescents, and children older than 5 years. The most common adverse events in vandetanib-treated patients are diarrhea, rash, folliculitis, nausea, QTc prolongation, hypertension, and fatigue. More data are required to deepen our knowledge on molecular biology of tumor and host defense, with the aim to achieve better prognosis and higher quality of life for affected patients.

show abstract

Section: Clinical Trials With Vandetanib In Mtcmentioning

confidence: 78%

“…Recently it has been described the case of a 58-year-old man with MTC and CS, resistant to various combined anti-cortisolic drugs 54. After the beginning of the treatment with vandetanib, a reversal of the hypercortisolism was shown, with no changes in tumor size.…”

Section: Clinical Trials With Vandetanib In Mtcmentioning

confidence: 98%

Evaluating vandetanib in the treatment of medullary thyroid cancer: patient-reported outcomes

Fallahi¹,

Ferrari²,

Elia³

et al. 2019

CMAR

View full text Add to dashboard Cite

show abstract

“…At variance with other TKIs, vandetanib treatment has also been tested in children, and it has been demonstrated to be safe and effective in controlling childhood MTC [79] . Moreover, evidence show the usefulness of vandetanib in reverting ectopic ACTH secretion and the consequent paraneoplastic Cushing's syndrome which could develop in patients with very advanced disease [80][81][82][83] .…”

Section: Vandetanibmentioning

confidence: 99%

Systemic treatment of advanced, metastatic, medullary thyroid carcinoma

Matrone¹,

Gambale²,

Prete³

et al. 2021

JCMT

View full text Add to dashboard Cite

Medullary thyroid carcinoma (MTC) is a rare endocrine tumor, which arises from thyroid parafollicular C cells. Through its ability to metastasize by blood and lymphatic vessels, it can show a more aggressive clinical behavior than differentiated thyroid cancers. Mutation of RET gene is the main molecular alteration involved in MTC origin. In the case of germline RET mutation, MTC can be inherited in an autosomal dominant way and show three different phenotypes: familial medullary thyroid carcinoma and multiple endocrine neoplasia types IIA and IIB. In addition, in sporadic cases, somatic RET mutation remains the key molecular alteration in most of cases. Total thyroidectomy with prophylactic or therapeutic central compartment lymph nodes dissection is the surgical treatment of choice. Further surgical treatments and local therapies should be used in the case of single or few local or distant metastasis. However, in cases with large metastatic spread of the disease, particularly in those with significant tumor progression, additional systemic treatments are needed. In this review, we discuss the key points of systemic treatment in advanced, metastatic MTC. We provide an update on the main aspects (from biological rationale to clinical experience) of each treatment, focusing our attention on the drugs used in clinical practice in the last years. Finally, we give insights about the emerging treatments from highly selective RET inhibitors to new radionuclide therapy.

show abstract

“…Surgical intervention with bilateral adrenalectomy is another option available to some patients. However systemic therapy with tyrosine kinase inhibitors offers a further management strategy for disease control of metastatic and is now considered a first line therapy for ECS in the setting of unresectable disease or progressive, metastatic disease [8, 9].…”

Section: Introductionmentioning

confidence: 99%

Sporadic Medullary Thyroid Carcinoma with Paraneoplastic Cushing Syndrome

Pivovarova

Patrick

Reddy

2019

Case Reports in Endocrinology

View full text Add to dashboard Cite

Medullary thyroid cancer (MTC) is a rare form of neoplasm affecting the thyroid gland. This neuroendocrine tumor is capable of releasing active substances causing systemic manifestation in the form of flushing, diarrhea, and uncommonly, Ectopic Cushing's syndrome (ECS). MTC can be hereditary as a part of multiple endocrine neoplasm type 2 syndrome (MEN2) or arise sporadically. We report a case of a 74-year-old female presenting with chronic diarrhea, in whom diagnosis of sporadic MTC was delayed due to previous history of gastrointestinal (GI) disturbances. The patient developed liver metastases yielding ACTH dependent Cushing's Syndrome leading to abnormal clinical presentation and laboratory values driven by elevated cortisol level. Metastatic MTC should be considered in patients presenting with chronic diarrhea and weakness unexplained by other GI related causes.

show abstract

Long-Term Control of Hypercortisolism by Vandetanib in a Case of Medullary Thyroid Carcinoma with a Somatic RET Mutation

Abstract: Three years and eight months after vandetanib initiation, there was no sign of recurrence of hypercortisolism. This case illustrates the long-term effectiveness of vandetanib in maintaining the control of hypercortisolism in MTC-related CS.

Cited by 19 publications

References 9 publications

<p>Evaluating vandetanib in the treatment of medullary thyroid cancer: patient-reported outcomes</p>

<p>Evaluating vandetanib in the treatment of medullary thyroid cancer: patient-reported outcomes</p>

Systemic treatment of advanced, metastatic, medullary thyroid carcinoma

Sporadic Medullary Thyroid Carcinoma with Paraneoplastic Cushing Syndrome

Contact Info

Product

Resources

About