The treatment of diencephalic syndrome is directed toward the specific symptoms that are apparent in each individual. Treatment may require the coordinated efforts of a team of specialists. Pediatricians, surgeons, neurologists, oncologists, radiation oncologists, and other healthcare professionals may need to systematically and comprehensively plan an affect child’s treatment.
Specific therapeutic procedures and interventions may vary, depending upon numerous factors, such as disease stage; tumor size and specific location; specific tumor type; the presence or absence of certain symptoms; an individual’s age and general health; and/or other elements. Decisions concerning the use of particular drug regimens and/or other treatments should be made by physicians and other members of the health care team in careful consultation with the patient based upon the specifics of his or her case; a thorough discussion of the potential benefits and risks, including possible side effects and long-term effects; patient preference; and other appropriate factors. Psychosocial support for the entire family is essential as well.
There is no agreed upon consensus for the best treatment for individuals with diencephalic syndrome and there are no standardized treatment protocols or guidelines just for tumor causing the diencephalic syndromes, however, protocols and often clinical trials are available for specific tumor types that cause the syndrome. Various treatments have been reported in the medical literature as part of single patient reports, small series of patients or more extensive, tumor-type based studies. Treatment trials would be very helpful to determine the long-term safety and effectiveness of specific medications and treatments for individuals with diencephalic syndrome.
Surgery, radiation, and chemotherapy alone or in various combinations have been used to treat this diencephalic syndrome. In some cases, physicians may recommend surgical excision and removal of as much as the tumor as possible (resection). However, because of the area of the brain that is usually affected, surgical removal of the entire tumor is often not possible. Additionally, surgery, even to remove only a portion of the tumor, carries risks due to the tumor’s location deep within the brain. However, biopsy is usually indicated to not only determine the histological subtype of the tumor, but its molecular subtype; this is especially useful in low-grade gliomas where molecular findings can guide therapy.
Radiation therapy can be used to directly destroy cancer cells or to destroy cancer cells left over after surgery. However, the potential for serious short and long-term side effects exists. Radiation therapy is especially avoided in children less than 5 years of age because of the potential for serious side effects.
Chemotherapy, the use of one or more anti-cancer drugs, has also been used to treat individuals with diencephalic syndrome, particularly those with low grade gliomas. Chemotherapy may be used instead of radiation in very young children to avoid damage to the developing brain. Chemotherapy may also be administered after radiation in an attempt to destroy any cells that remain or may be given during the course of radiation treatment. The type of chemotherapeutic drug therapy used is determined by a neuro-oncologist who examines the grade of tumor, previous treatment, and current health status of the affected individual. Chemotherapeutic drugs that have been used for diencephalic syndrome include carboplatin, carboplatin-vincristine, carboplatin-vincristine-temador, low dose cisplatin-etoposide, and other drug regimens. Recently, molecularly targeted therapies (biologic therapy) have become available for treatment of low-grade pediatric gliomas. Bevacizumab, which targets vascular endothelial growth factor, has been successfully used for some patients with diencephalic gliomas. Also, agents interfering with RAS-MAPK signaling hold great promise for treatment of diencephalic tumors and prospective clinical trials are underway or soon to open for newly-diagnosed patients. These trials require molecular characterization of the tumor for entry.