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Current and Future Treatments for Malignant Pheochromocytoma and Sympathetic Paraganglioma

  • Sarcomas (SR Patel, Section Editor)
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Abstract

Pheochromocytomas (PHs) and sympathetic paragangliomas (SPGs) are rare neuroendocrine tumors. Approximately 17 % of these tumors are malignant, but because no molecular or histologic markers for malignancy exist, patients are often diagnosed with malignant PHs or SPGs after unresectable disease has formed. Patients with progressive metastatic tumors and overwhelming symptoms are currently treated with systemic chemotherapy and radiopharmaceutical agents such as metaiodobenzylguanidine. These therapies lead to partial radiographic response, disease stabilization, and symptomatic improvement in approximately 40 % of patients, and systemic chemotherapy is associated with a modest improvement in overall survival duration. However, over the past decade, substantial progress has been made in clinical, biochemical, and radiographic diagnosis of PHs and SPGs. Approximately 50 % of patients with malignant PHs and SPGs have been found to carry hereditary germline mutations in the succinate dehydrogenase subunit B gene (SDHB), and anti-angiogenic agents such as sunitinib have been found to potentially play a role in the treatment of malignant disease, especially in patients with SDHB mutations. In some patients, treatment with sunitinib has been associated with partial radiographic response, disease stabilization, decreased fluorodeoxyglucose uptake on positron emission tomography, and improved blood pressure control. These findings have led to the development of prospective clinical trials of new targeted therapies for metastatic disease. Here, we provide an updated review of the clinical and genetic predictors of malignant disease, radiographic diagnosis of malignant disease, and information from the most relevant studies of systemic therapies, as well as proposed treatment guidelines for patients with metastatic or potentially malignant PHs and SPGs.

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Acknowledgments

This work has been supported in through MD Anderson’s Cancer Center support Grant CA016672 and by the generous support of Mr. Clarence Cazalot and Mrs. Margaret Cazalot, Mr. William Granek and Mrs. Marle Granek, and the courage and support of Ms. Natalie Papadam and the Team NAT. We thank Erica Goodoff, Department of Scientific Publications at MD Anderson, for assistance with editing the manuscript.

Conflict of Interest

Camilo Jimenez declares no conflict of interest.

Eric Rohren declares no conflict of interest.

Mouhammed Amir Habra declares no conflict of interest.

Thereasa Rich declares no conflict of interest.

Paola Jimenez declares no conflict of interest.

Montserrat Ayala-Ramirez declares no conflict of interest.

Eric Baudin declares no conflict of interest.

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Authors and Affiliations

  1. Department of Endocrine Neoplasia and Hormonal Disorders, Unit 1461, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Blvd, Houston, TX, 77030, USA

    Camilo Jimenez, Mouhammed Amir Habra & Montserrat Ayala-Ramirez

  2. Department of Nuclear Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX, USA

    Eric Rohren

  3. Department of Surgical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA

    Thereasa Rich

  4. Hemato-Oncologos Asociados, Bogota, Colombia

    Paola Jimenez

  5. Département de Médecine Nucléaire et de Cancérologie Endocrinienne, Institut Gustave Roussy 39 Bis, Rue Camille Desmoulins, 94800, Villejuif, France

    Eric Baudin

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  1. Camilo Jimenez
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  3. Mouhammed Amir Habra
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Correspondence to Camilo Jimenez.

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Jimenez, C., Rohren, E., Habra, M.A. et al. Current and Future Treatments for Malignant Pheochromocytoma and Sympathetic Paraganglioma. Curr Oncol Rep 15, 356–371 (2013). https://doi.org/10.1007/s11912-013-0320-x

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