This very rare and challenging benign cortical brain tumour typically involves a single site in the posterior fossa of the brain and, if surgically removed, carries a very poor prognosis with over 50% of children dying in the year after diagnosis. In this situation, chemotherapy followed by radiation is often recommended.
Many cases of ependymomas are occurring in children and teens, especially in the first two decades of life. This information would help the surgeon and the child and adolescent patient and their parents discuss treatment options. Because fewer than 10% of all cases of ependymomas are diagnosed before 15 years of age, ependymoma awareness campaigns need to extend beyond children of all ages to younger teens.
There does not currently seem to be any significant difference between the age of onset of ependymomas and their tumor location. However, the occurrence of these ependymomas in adults raises the possibility of this tumor's having a genetic cause - which is not well understood.
The usual causes of ependymoma include genetic disorders, prenatal exposure to tobacco smoke and prenatal and early-childhood environmental factors. The high rate of recurrence observed previously in this study suggests that more aggressive surgical excision may be advisable in patients with this condition.
There is a high incidence of relapse after total surgical resection. The use of radiotherapy or chemotherapy must be carefully considered, depending on disease risk and patient comorbidities as well as disease aggressiveness. The role of high-dose radiation therapy, in particular radiation therapy with proton beams, should be explored further.
Patients who have not undergone initial resection of ependymoma have a very high risk of recurrence or of the development of second tumors, and their prognoses are generally poor.
When people are asked if they have experienced any symptoms, some say yes. Pain or stiffness in either or both legs is another symptom of ependymoma. Children more often have tumour in one or more regions of the body than adults. Increased intracranial pressure and ocular signs, such as decreased vision and eye pain, are symptoms of tumour in the optic nerve.
Familial ependymomas occur rarely, with familial cases representing 1.0% of this group. Of the 17 familial cases studied in this series, 14 tumors occurred in 1 family member. Familial cases are most likely caused by somatic mutations or by germ line mutations. Further study is needed to determine the underlying causes of familial cases of ependymomas.
Brigatinib exert anti-proliferative effects in vitro and in vivo by inhibiting PI3K/AKT, MAPK/ERK, and STAT3 signaling and by blocking the production of angiogenic proteins from ECs.
In patients with a surgically cured ependymoma, postoperative radiotherapy and prolonged follow up should be recommended if the relapse occurs, especially with tumor grade IV.
brigatinib was the first drug approved by regulatory agencies for adults with relapsed or refractory Hodgkin's lymphoma or relapsing indolent lymphoma. However, clinical trials involving brigatinib that included post-hoc analysis of survival data are no longer being published in peer-reviewed journals. Although the efficacy and safety of brigatinib has been demonstrated in patients with relapsed or refractory Hodgkin's lymphoma and indolent lymphoma, patients who have received brigatinib have discontinued treatment citing safety concerns, exacerbation of CLL, and possible worsening of myelofibrosis. [Find more clinical trials at clinicalTrials.gov (e.g.
Brigatinib has shown significant single agent activity in phase I trials in patients whose tumors carried EGFR exon 19 deletions. It has demonstrated clear dose-dependent efficacy at this dose in patients with tumors carrying T790M mutations. Results from a recent clinical trial support continued investigations of brigatinib in combination with other agents.