Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Pediatric Pilocytic Astrocytoma.
Treatment: Mekinist.
The insurer denied Mekinist. 
The health plan's determination is overturned.

The patient has a history of hypothalamic optic pathway pilocytic astrocytoma. The patient was diagnosed at almost 2 years ago. The patient's tumor was resected, and the patient was started on Carboplatin and vincristine. The patient then had hydrocephalus and required another surgical resection that was complicated by further visual loss and pan hypopituitarism. The patient was restarted on carboplatin and vincristine but had intermittent Syndrome of Inappropriate Antidiuretic Hormone (Secretion) (SIADH), seizures, and progressively worsening vision. The patient was then treated with weekly vinblastine treatments for over a year. Later, the presented with fever and seizure-like activity and a magnetic resonance imaging (MRI) scan showed concern for recurrence of disease. The patient then had worsening of her hydrocephalus with need for second ventriculoperitoneal (VP) shunt placement.
The requested health service / treatment of Mekinist is medically necessary for this patient.
This patient has a history of a progressive, recurrent hypothalamic pilocytic astrocytoma for which Mekinist is being recommended as a targeted therapy based on genetic changes noted within the patient's specific tumor. The use of Mekinist is clinically appropriate based on the current peer-reviewed medical literature. As a young child with a progressive pilocytic astrocytoma, treatment options are limited, and the patient has already failed the more standard forms of chemotherapy. There have been several reports of the response of Mekinist in the treatment of low-grade gliomas. As per Miller et al, two patients with inoperable chemotherapy-resistant optic pathway tumors showed a response to Mekinist. Mekinist has been found to have benefit in the treatment of BRAF mutated gliomas, such as the one that this patient has. As per a study by Sent et al, for 18 patients with low-grade gliomas and aberrant signaling in the MAPK(mitogen activated protein kinase) pathway, 6 had partial response, 2 had minor response and 10 had stable disease, showing that all patients had at least some response to the therapy. Kondyll et al demonstrated response in another 6 patients.
This patient has limited treatment options at this time. The patient is not a candidate for further surgery, has failed chemotherapy and is not a candidate for radiation therapy at this time. Mekinist has a strong possibility to be effective in preventing further tumor growth in this patient and potentially prolonging her life. Truong et al discussed the potential benefit in targeted therapy for MAPK alterations in pediatric gliomas, showing the biology of gliomas, the oncogenic mutations in the MAPL pathway (which Mekinist targets) and the notable pre-clinical data and clinical trials for this particular targeted therapy. Arnold et al were also able to show a response for pediatric glioma patients receiving Mekinist in combination with an MTORC1/2 inhibitor.
This treatment is in accordance with generally accepted standards of medical practice and is clinically appropriate in terms of type, frequency, extent, site and duration and considered effective for the patient's medical condition. It is provided for the direct care and treatment of the patient's tumor and is not for the convenience of anyone. There are no alternative treatment options for the patient at this time, so this is not more costly than an alternative drug or service.
Therefore, based on the clinical information provided for review, and the current peer-reviewed medical literature, and standards of care in the field, the request for Mekinist therapy for this patient is medically necessary at this time.