This trial is evaluating whether Axitinib will improve 1 primary outcome and 1 secondary outcome in patients with Pheochromocytoma. Measurement will happen over the course of Up to 16 weeks.
This trial requires 25 total participants across 2 different treatment groups
This trial involves 2 different treatments. Axitinib is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.
Symptoms occur in nearly all cases of pheochromocytoma, and may include orthostatic syncope, headache, palpitation and sweating. Symptoms of adrenal hormone secretion may present similarly. When there is an abnormal adrenergic response to exercise, it may be difficult to distinguish pheochromocytoma from catecholamine secreting tumors such as sympathetic adenoma. Many different symptoms may occur in pheochromocytoma.
Treatment of symptomatic and incidentally detected tumors is more frequently resection, adjuvant radiotherapy, transcatheter arterial chemoembolization, or embolization. These techniques may be used alone or in combination with surgery. Other treatment options exist, but the optimal therapeutic modality needs to be determined using randomized controlled trials.
The cause of pheochromocytoma remains unknown; however, it is generally diagnosed by clinical examination, the discovery of catecholamines from a biopsy or the use of tests such as CT scans or 123I-MIBG (123I metaiodobenzylguanidine) scintigraphy. If pheochromocytoma is associated with signs and symptoms of high blood pressure (hypertension), further testing can be performed to exclude the presence of a tumor.
As with other endocrine disorders, many patients have a long and healthy life after surgery and medical or physical therapy. Although surgery should always be recommended, the possibility of cure should be evaluated individually and thoroughly before it is performed.
The frequency of pheochromocytoma in the general population is low. The presence of multiple endocrine neoplasia type 2 has the highest frequency and accounts for the majority of cases.
Pheochromocytoma is an uncommon catecholamine-producing tumor originating in the paraganglia of the sympathetic nervous system. It is diagnosed by measurement of plasma or urinary catecholamine levels, and surgical removal is curative for the majority of patients. Pheochromocytoma commonly presents with a history of hypertension and/or paroxysmal pain at sites such as the neck, abdomen, and extremities. The treatment is surgical resection of the tumor, removal of catecholamine-producing sympathetic nervous system tissue, and medications to control blood pressure and prevent and treat postoperative hypertension.
Very common (>10%) side effects include diarrhea, fatigue, nausea, and vomiting. Other common effects include hyperglycemia, headache, and asthenia. Rare (<5%) side effects include hypovolemia, liver inflammation, and gastrointestinal perforations. Symptoms such as fatigue, nausea, dizziness, and constipation are often mild.
This questionnaire survey was a unique contribution to the evaluation of the need for pheochromocytoma clinical trials. As a result of this survey, certain groups need to be considered for clinical trials regarding the effects of pheochromocytoma. Physicians should consider administering pheochromocytomas as soon as the cancer is diagnosed and/or the condition is progressing. Further research of pheochromocytoma is a great area of interest for the future.
Axitinib in combination with capecitabine and oxaliplatin achieves a significantly longer OS than capecitabine and oxaliplatin. The PFS in the first line chemotherapy group was 4.8 months and 7.3 months in the second line therapy group.
A recent meta-analysis shows significant, yet limited, survival benefit from axitinib in combination with chemotherapy for mRCC. [About half of patients with mRCC treated with axitinib in combination with cisplatin or another chemotherapy respond, compared with about 30% of mRCC treated with the same chemotherapy alone.] There is high toxicity, however, including a significant rate of side effects such as diarrhea, nausea, fatigue, and vomiting. Although a trial would be prohibitively expensive, axitinib, if used in combination with chemotherapy, merits further investigation.
We found no statistically significant differences in the incidence of development of pheochromocytoma (11%) and hypertension (15%) between males and females of the study group (p=0.5, 0.8). The mean age at onset of hypertension was 41 +/- 15.5 years, similar in males (38.8 years) and females (40.6 years). This implies that there is no gender difference in the incidence of the disease. Further studies will be necessary to support these findings.
Axitinib improved only a few domains of the PFS, indicating a small clinical effect, but it did not improve quality of life overall or on specific HRQoL scales.