Paraganglioma and pheochromocytoma are very rare and, among the many difficulties faced by patients, there is that of having few options for treatment. A study makes for an important step forward
An important news for patients with pheochromocytoma paraganglioma, a very rare type of neuroendocrine tumor: a drug, already available in Italy and used to treat various forms of cancer, significantly prolongs the life span of patients before the disease progresses. This is demonstrated by the results of an international trial coordinated by researchers from the Gustave Roussy Institute in Paris and presented during the annual congress of the European Society of Medical Oncology (Esmo), which have achieved the greatest evidence available so far on this group of patients.
The experimentation
The phase two FIRSTMAPPP study enrolled 78 patients with progressing paraganglioma or pheochromocytoma from 15 European hospital centers over eight years. Participants, on average 53 years old, received the drug sunitinib (a tyrosine kinase inhibitor) or a placebo and the results indicate that at 12 months after treatment, 39.5% of patients treated with the drug had not had any progress of the cancer, versus 18.9% in the placebo group. Sunitinib the first drug to demonstrate statistically significant advantage in patients with pheochromocytoma or paraganglioma – comments Nicola Fazio, director of the division of gastrointestinal medical oncology and neuroendocrine tumors of the European Institute of Oncology -. To date for patients with these neoplasms there is no standard definable therapy: In advanced disease, chemotherapy with alkylating agents is mostly proposed. I believe the important results of this trial will change our clinical practice in this area.
What are neuroendocrine tumors
Neuroendocrine tumors (Neuroendocrine tumours, NETs in English) are a heterogeneous group of neoplasms deriving precisely from neuroendocrine cells, scattered practically everywhere in the human body: they are, in fact, specialized cells that function as part of both the nervous and endocrine systems, in the production of hormones. Neuroendocrine tumors are rare as they have an incidence of less than six new cases per 100 thousand people per year – explains Fazio -: according to the latest from the Airtum Cancer Registries, in Italy, as a whole, they affect about 3 thousand people every year, but if we consider that there are various types of neuroendocrine tumors one can imagine the rarity of the individual subtypes. The most frequent are those of the gastroenteropancreatic tract (about two thirds of cases), followed by broncho-pulmonary ones (20-30%) or other body regions (10%) such as skin, thyroid, parathyroid and adrenal glands. recognize them. In view of the rarity and biological and clinical heterogeneity of neuroendocrine tumors, the treatment of affected patients should be managed. in a specialist and multidisciplinary field. In short, since these are infrequent and little known pathologies, it is difficult to recognize them and treat them correctly, without wasting precious time for the sick.
One case per million people
Pheochromocytoma and paraganglioma are very rare neuroendocrine tumors: in fact they have an incidence of less than one new case per year per million people. Pheochromocytoma is a tumor of the adrenal gland, while paragangliomas arise in the paragangliomas, present from the skull base to the pelvic floor – says the expert -. They can produce hormonal substances, which in some cases give rise to very severe syndromes, characterized by increased blood pressure, sweating, palpitations, and more. In some cases, then, they arise in the context of an inherited genetic syndrome. Treatment depends on the stage and degree of aggressiveness of the tumor: in localized forms it is evaluated surgical excision, while in the advanced ones you go from chemotherapy, to radioreceptor therapy with Lutetium, to therapy with metaiodobebzilguanidine (MIBG), to locoregional treatments of interventional radiology. However, since there is no validated and considered standard therapy, it is preferable, if any, to evaluate patient inclusion in experimental clinical trials.
Specialized centers and a team of different experts
Although neuroendocrine tumors are malignant tumors, in many cases they have a more indolent course than non-neuroendocrine tumors of the same organs and this allows many patients with advanced disease to to be able to live with the disease and therapies for a long time. It is very difficult to give univocal information for all patients – concludes Fazio -, but one thing is true regardless of the type of NET in question: it is essential to contact a highly specialized center, with specific experience in the field and equipped with a multi-specialist group dedicated to the pathology, in so that you can properly customize the diagnostic-therapeutic approach. To set up an adequate treatment it is necessary to be sure of the diagnosis and to better characterize the disease. The specific and multi-specialized competence specific to the sector is therefore crucial.
October 12, 2021 (change October 12, 2021 | 18:07)
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