Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Esophageal Cancer
Treatment: Radiological Services - Proton Beam Therapy, Pre-service
The insurer denied the radiological services - proton beam therapy, pre-service.
The denial is overturned. 

The patient is a male with a diagnosis of stage T2N3 (stage 2, cancer has spread to at least 10 axillary lymph nodes) lower esophageal cancer. Neoadjuvant chemotherapy and radiation were recommended, and proton therapy was requested.

Yes, the proton beam therapy is medically necessary.

There are several trials showing that proton therapy is safe and effective for the treatment of esophageal cancer. A Japanese study evaluated 48 patients with esophageal carcinoma treated with proton beam radiation therapy. The authors concluded that "The results suggest that proton beam therapy is an effective treatment for patients with locally confined esophageal cancer. Additional studies are required to determine the optimal total dose, fractionation schedule, and best combinations of protons and conventional X-rays with or without chemotherapy."

Another study performed dosimetric comparisons of 10 esophageal patients with 3D (three-dimensional) vs. IMRT (intensity-modulated radiation therapy) vs. proton therapy. The authors concluded that "In patients receiving radiation therapy for esophageal cancer, proton plans are technically feasible while achieving adequate coverage with lower doses delivered to the lungs and cardiac structures. This may result in decreased cardiopulmonary toxicity and less morbidity to esophageal cancer patients."

A more recent study from MD Anderson Cancer Center compared proton therapy and photon IMRT for patients with esophageal cancer. 343 esophageal cancer patients who received definitive chemoradiotherapy with either PBT (proton beam therapy) or IMRT were retrospectively analyzed. The authors concluded that "Compared with IMRT [intensity-modulated radiation therapy], PBT [proton beam therapy] might be associated with improved OS [overall survival], PFS [progression-free survival], and LRFFS [loco-regional failure-free survival], especially in EC [esophageal cancer] patients with locally advanced disease."

A recent randomized phase IIB trial by Lin, et al randomly assigned patients to PBT or IMRT (50.4 Gy [gray]), stratified for histology, resectability, induction chemotherapy, and stage for the treatment of locally advanced esophageal cancer. The posterior mean total toxicity burden (TTB) was 2.3 times higher for IMRT than PBT. The mean post-operative complications (POC) score was 7.6 times higher for IMRT versus PBT. The authors concluded that for locally advanced esophageal cancer, PBT reduced the risk and severity of AEs (adverse events) compared with IMRT while maintaining similar PFS.

The patient in this case is a male with a diagnosis of loco-regionally advanced GE (gastroesophageal) junction carcinoma. Neoadjuvant chemotherapy and radiation were requested, and proton beam radiation therapy was requested. In this case, a proton/IMRT comparison plan was done. The heart V30 (30 gray) was 41% (exceeded tolerance) and 16.1% with proton plan. The mean heart dose was 25.76 Gy with photon (exceed tolerance) and 10.83 Gy with proton therapy. The proton plan also significantly reduced dose to the lung, small bowel and liver. Given that there are multiple trials, including a randomized trial showing that proton therapy is clinically superior compared to photon therapy, and that the dosimetric comparison for this patient also showed dosimetric advantage of proton therapy, the requested proton therapy is considered medically necessary.

Studies (including a randomized trial) have shown that proton therapy is clinically superior compared to photon therapy for the treatment of esophageal cancer. In this case, a comparison plan was done. Because of the extensive nodal involvement and near proximity to the heart, the photon plan showed that the heart dose would exceed tolerance, which may result in significant risk of heart injury. The proton plan was able to reduce the doses to the heart and lung to acceptable levels. Per NCCN (National Comprehensive Cancer Network) Guideline, proton therapy should be used if other techniques such as photon 3D or IMRT cannot adequately spare the surrounding critical structures. Proton therapy, in this case, would be a medically appropriate treatment option.