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Sunitinib for refractory malignant pheochromocytoma: two case reports
Junya Hata1, Nobuhiro Haga, Kei Ishibashi
1Department of Urology, Fukushima Medical University School of Medicine, 1 Hikarigaoka, Fukushima, 960-1295, Japan, akju826@fmu.ac.jp.
Abstract:
Sunitinib, a molecular-targeted therapy, is a potential new treatment strategy for malignant pheochromocytoma. However, because of the rarity of malignant pheochromocytoma and the consequent limited number of patients available for clinical study, there is no good evidence of the efficacy of sunitinib for malignant pheochromocytoma. The present report describes our experience with sunitinib for refractory malignant pheochromocytoma. Two patients were treated with sunitinib at a standard dose (50 mg daily; 4 weeks on, 2 weeks off) after cyclophosphamide/vinblastine/dacarbazine chemotherapy, because vascular endothelial growth factor (VEGF)-positive cells were partly observed by immunohistochemical staining. Both patients were assessed as having stable disease according to the Response Evaluation Criteria in Solid Tumors 1.1. The duration of overall survival from the time sunitinib was initiated was 13 and 8 months, respectively, and the progression-free survival was 5 and 4 months, respectively. Adverse events were evaluated according to the Common Terminology Criteria for adverse events of the US Department of Health and Human Services version 4.0. One patient experienced hypothyroidism (Grade 2) and thrombocytopenia (Grade 2). The other patient experienced anorexia (Grade 3) and general malaise (Grade 3). In conclusion, sunitinib was effective in the treatment of malignant pheochromocytoma when VEGF-positive cells were observed in the tumor specimens.
Insights
Sunitinib shows promise in treating malignant pheochromocytoma, particularly when tumors express vascular endothelial growth factor (VEGF). This targeted therapy demonstrated stable disease in two patients, offering a potential new option for this rare cancer.
Area of Science:
- Oncology
- Pharmacology
Background:
- Malignant pheochromocytoma is a rare cancer with limited treatment options.
- Molecular-targeted therapies like sunitinib are being investigated for their potential efficacy.
Observation:
- Two patients with refractory malignant pheochromocytoma received sunitinib after conventional chemotherapy.
- Tumor specimens showed partial expression of vascular endothelial growth factor (VEGF)-positive cells, indicating potential for targeted therapy.
Findings:
- Both patients achieved stable disease according to Response Evaluation Criteria in Solid Tumors 1.1.
- Overall survival was 13 and 8 months, with progression-free survival of 5 and 4 months, respectively.
- Adverse events included hypothyroidism, thrombocytopenia, anorexia, and general malaise, manageable with dose adjustments.
Implications:
- Sunitinib may be an effective treatment for malignant pheochromocytoma, especially in cases with VEGF-positive tumors.
- Further clinical studies are warranted to confirm sunitinib's efficacy and safety in a larger patient cohort.
- Targeted therapy based on immunohistochemical markers like VEGF could improve outcomes for rare endocrine tumors.
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