Upfront Radiotherapy May Benefit Medulloblastoma Patients With Li-Fraumeni Syndrome
Patients with sonic hedgehog medulloblastoma and Li-Fraumeni syndrome have a “dismal” prognosis, according to researchers.
Patients with sonic hedgehog medulloblastoma and Li-Fraumeni syndrome have a “dismal” prognosis, according to researchers.
The overall response rate after 2 treatment cycles was 78%.
RNA sequencing confirmed the alterations most frequently described in low-grade glioma and revealed rare gene fusions.
Of the patients who completed follow-up, 60% had a targeted therapy recommendation.
The median duration of response was not reached at a median follow-up of 26 months.
Of the 65 patients who achieved a complete remission with chemotherapy, 63 remained in remission after whole ventricular radiotherapy and at the final follow-up.
The treatment proved noninferior to cisplatin-based radiochemotherapy.
There was no significant difference in rates of secondary neoplasms at 10 years.
Metastatic relapse was associated with elevated tumor markers, but it was not associated with focal or whole ventricular radiotherapy.
Hispanic and non-Hispanic patients had a similar risk of death.