Related Experiment Video
Updated: Jul 14, 2026
In Vivo Inhibition of MicroRNA to Decrease Tumor Growth in Mice
Published on: August 23, 2019
A phase II trial of imatinib therapy for metastatic medullary thyroid carcinoma
J W B de Groot1, B A Zonnenberg, P Quarles van Ufford-Mannesse
1Department of Endocrinology, University Medical Center Groningen, University of Groningen, 9700 AB Groningen, The Netherlands.
Context:
Medullary thyroid carcinoma (MTC) metastasizes early in its clinical course. No effective systemic therapy is available. Generally (somatic or germline), mutations in the rearranged during transfection gene are considered essential in the pathogenesis of MTC.
Objective:
We investigated imatinib, a tyrosine kinase inhibitor, as a potential treatment in patients with disseminated MTC.
Design:
A phase II study was initiated using 600 mg imatinib daily with a possible dose increase to 800 mg in case of progression. Standard Response Evaluation Criteria in Solid Tumors were used using computed tomography or magnetic resonance imaging every 2 months.
Results:
There were 15 patients with disseminated MTC treated for up to 12 months. No objective responses were observed. Four patients had stable disease over 24 months. Three patients stopped treatment due to toxic effects [fatigue (n = 2) and nausea (n = 1)]. In four cases the dose of imatinib was decreased because of toxicity [rash and malaise (n = 2) and laryngeal swelling (n = 2)]. Emergency tracheotomy was performed in two cases due to mucosal swelling of the larynx in patients with recurrent nerve palsy and a narrow vocal cleft. In nine patients with a history of a thyroidectomy, the dose of supplemental thyroid hormone was increased because of serious hypothyroidism.
Conclusions:
Imatinib therapy yielded no objective responses and induced considerable toxicity in patients with MTC. A minority of patients had stable disease. Patients with supplemented hypothyroidism or with recurrent nerve palsy are specifically at risk for serious adverse events and need special attention when treated with imatinib.
Insights
Imatinib treatment showed no objective responses in patients with medullary thyroid carcinoma (MTC). The drug induced significant toxicity, with some patients experiencing stable disease but requiring careful monitoring for adverse events.
Area of Science:
- Oncology
- Pharmacology
Background:
- Medullary thyroid carcinoma (MTC) is characterized by early metastasis and lacks effective systemic therapies.
- Mutations in the rearranged during transfection (RET) gene are crucial in MTC pathogenesis.
- Current treatment options for advanced MTC are limited.
Purpose of the Study:
- To evaluate imatinib, a tyrosine kinase inhibitor, as a treatment for disseminated MTC.
- To assess the efficacy and safety of imatinib in patients with advanced MTC.
Main Methods:
- A Phase II clinical study was conducted.
- 15 patients with disseminated MTC received imatinib (600 mg daily, with a potential increase to 800 mg).
- Tumor response was assessed every 2 months using RECIST criteria via CT or MRI.
Main Results:
- No objective responses were observed in any of the 15 patients.
- Four patients achieved stable disease for over 24 months.
- Significant toxicity was noted, including fatigue, nausea, rash, malaise, and laryngeal swelling, leading to dose reductions or treatment discontinuation in some cases.
Conclusions:
- Imatinib therapy did not demonstrate objective efficacy in patients with disseminated MTC.
- Considerable toxicity was associated with imatinib treatment, impacting patient management.
- Patients with hypothyroidism or recurrent nerve palsy are at higher risk for adverse events and require specialized attention during imatinib therapy.
Related Concept Videos
01:11Clinical Trials: Overview
02:57Targeted Cancer Therapies
There are several types of targeted therapies against specific...
01:16Clinical Trials
There are four phases in a clinical trial. A phase one...