Appeal type: Experimental/Investigational
Decision: Overturned

Diagnosis: Desmoid Tumors. 
Treatment: Percutaneous Cryoablation. 

The health plan denied the requested cryoablation. 
The health plan's determination is overturned. 

The patient is a male with a history of familial adenomatosis polyposis (FAP) and multiple bilateral anterior abdominal walls and a retroperitoneal desmoid tumors. He had multiple imaging studies (information provided). The abdominal wall lesions have been slightly enlarging in size and documented to be painful. The largest documented lesion measured 2.7x5.1x3 cm. The lesions have been previously treated with intralesional steroid injections with symptoms relieved.  No records were forwarded if imaging studies were done between the steroid injection period and cryoablation. According to records, the patient is also being treated with Sorafenib for desmoid tumors though the duration is unknown. 

He underwent computed tomography (CT) and ultrasound (US) guided cryoablation of 4 anterior abdominal wall lesions. Follow up scans have shown stabilization of the tumor sizes. Of note, the retroperitoneal lesion was not treated with cryoablation and according to a urology note, this tumor has resolved. Cryosurgical tumor ablation has not been medically proven to be effective and therefore is considered investigational as a treatment method for desmoid tumors. 

The health plan's determination is overturned, in whole. 

Yes. Desmoid tumors are uncommon tumors and are considered to be benign tumors with a highly variable clinical course. Their behavior can range from being indolent, rapidly growing, spontaneous regression, symptomatic or asymptomatic. Because the rate of spontaneous regression or stable disease ranges from 5-50%, the 2019 The Desmoid Tumor Working Group consensus recommends active observation as the first line approach for 1-2 years. The patient's tumor was observed for >1 year and was found to be enlarging and symptomatic. 

The traditional gold standard for abdominal wall desmoid tumors is surgical resection with wide margins. In this incident, the number of lesions would make surgical resection difficult and possibly disfiguring. Of note, the patient is already on a tyrosine kinase inhibitor with progression and symptomatic disease. Compared to other treatment options, nontoxic and cytotoxic systemic therapy, tyrosine kinase systemic therapy, and radiation therapy, early studies of cryoablation showed comparable treatment response, recurrence rates, and symptoms relieved. To date, no single or combination therapy has been shown to be superior to any other. Cryoablation is a reasonable alternative to other available therapies for this patient. -Surgery recurrence rate: 25-35%, -Radiation therapy. Recurrence rate 20%. Response rate 68%, Control rate 96%, -Tamoxifen: Response rate 31%, Control rate 67%, -Chemotherapy : Response rate 53%, Control rate 80%, -Cryoablation: (Response rate 40%. Recurrence rate 4 -17%, Control rate 42-95.7% Symptomatic improvement 89%)

The determination that cryoablation therapy is likely to be more beneficial than any standard treatment or treatments for the insured's life threatening or disabling condition or disease is based on its theoretically low side-effect profile. Radiation therapy can result in radiation dermatitis, unintentional radiation of surrounding healthy tissue, and limited future ability to use this modality again in the same area. Cryoablation probes can be better controlled to avoid unintentional targets and can be performed in the same area multiple times. According to studies, complication rate (neuroplexia) ranges from 0-11%. Cryoablation also does not have any systemic treatment toxicities seen in systemic therapies. 

Without a specific superior treatment modality, Cryoablation short term results being comparable to existing treatments, and its low side-effect profile, it is likely to be more beneficial than any standard treatment available if treatment is indicated.