Appeal type: Medical necessity
Decision: Overturned

Diagnosis:  Epithelial fibrosarcoma
Issue under review:  Proton beam therapy

Determination: The proton beam therapy is medically necessary.

Proton beam radiation therapy is a type of very precise external radiation treatment in which protons are used to treat cancer and other conditions.  Compared to photon, a proton beam that is delivered with sufficient energy has a low "entrance dose."  A proton beam also has a high-dose "Bragg peak" region, which is designed to cover the entire tumor, and there is no "exit dose" beyond the tumor involved in proton radiation therapy.  Because of this unique property, proton beam therapy is highly successful in minimizing damage to normal tissue and surrounding organs, resulting in fewer side effects.  It is an FDA-approved procedure and it is not subject to IRB (institutional review board) review.

Fibrosarcoma is a relatively rare disease, and clinical data with proton therapy is limited.  However, there are studies that show that proton therapy is superior in sparing the surrounding tissue compared to photon based techniques.  A 2012 study compared 3D, IMRT (intensity-modulated radiation therapy) and proton therapy techniques for retroperitoneal sarcomas.  The bowel and kidney dose was significantly reduced with proton therapy compared to photon therapy and the authors concluded, "Based on Quantitative Analysis of Normal Tissue Effects in Clinic benchmarks, the dosimetric advantage of proton therapy may be less gastrointestinal and genitourinary toxicity." 

Another 2017 study evaluated patient with recurrent pancreatic cancer following definitive radiation that received reirradiation using proton therapy.  There was a rate of 13% acute  grade 3 toxicities attributed to proton therapy.  The median overall survival (OS) was 16.7 months and OS at 1 year was 67%.  The authors concluded that "PRT was well tolerated, resulted in prolonged clinical outcomes compared to historical controls, and should be considered as a treatment option with concurrent chemotherapy in selected patients with locally-recurrent pancreatic cancer."

Another 2017 review article by Frisch also concluded that "Proton therapy is already an internal part in the multidisciplinary management of childhood sarcomas in contiguity to sensitive structures, especially at the base of skull, spine and pelvis.  It offers special advantages for tumours requiring high-precision radiotherapy, particularly when total resection seems not feasible."  

This patient is a young female with a diagnosis of recurrent and oligometastatic epithelial fibrosarcoma s/p prior chest wall resections and radiation to the chest wall and thoracic spine.  The patient subsequently presented with oligometastatic sacral lesion.  Local radiation therapy was recommended, and proton beam radiation therapy was requested.  In this case, the patient is a very young female who wishes strongly to preserve her ovarian functions.  A proton therapy/photon comparison plan was done, and the dose to the ovaries was significantly higher with photon compared to proton therapy (which was 0 cGy).  Since the ovaries are very radiosensitive, proton therapy can help with ovarian preservation.  In addition, proton therapy may also reduce the risk of secondary malignancy.  

The health plan has previously denied coverage for proton beam therapy, as they do not consider it to be medically necessary for cancer that has metastasized to the bone. However, as stated above, the patient wishes to preserve her ovarian functions.  As such, due to the location of the lesion, the radiosensitivity of the ovaries, and the results of the results of the proton/photon comparison plan, proton beam therapy is the better option for this particular patient.

Proton beam radiation therapy, therefore, is not unproven and is a medically necessary treatment option.