Appeal type: Experimental/Investigational
Decision: Upheld

Physician 1:  The patient is a 16-year-old male with pulmonary metastasis and recurrent osteosarcoma. His initial chemotherapy regimen included doxorubicin, high-dose methotrexate and cisplatin. He now has several pulmonary metastases, thought to be non-resectable following surgical exploration. Prior to this, the patient had previous metastatic lung lesions resected and had a local recurrence, for which he underwent a limb amputation. The patient and his family have declined chemotherapy (other than initial chemotherapy for primary disease) with each recurrence. A request has been made for authorization for the patient to undergo cryotherapy for treatment of the pulmonary lesions. The Health Plan has denied this request on the basis that cryotherapy is considered experimental/investigational.The request for cryotherapy is outside of a clinical trial, as the patient does not qualify for the current clinical trial based on his age. Review of the relevant literature pertaining to cryotherapy and lung neoplasms reveals only a small number of citations, many of which involve animal models and bronchogenic tumors, not lung parenchymal. The study in Radiology submitted with this appeal is the largest study. In the absence of good follow-up data and randomized trials, there is insufficient evidence demonstrating the requested procedure is likely to be superior to standard treatment.For the reasons set forth above, I have determined that the requested procedure is not likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. Therefore, the Health Plans denial should be upheld.Physician 2:  The patient is a 16-year-old male with a history of osteogenic sarcoma of the left upper extremity. His initial presentation was in September 2004 with pain in his left arm that was eventually diagnosed as an osteogenic sarcoma of the humerus. He underwent chemotherapy (doxorubicin, methotrexate and cisplatin) and subsequent limb salvage surgery. He was diagnosed with a local recurrence and underwent amputation. He then developed metastatic disease in May 2006 and his first pulmonary nodule was resected. In August 2006, a CT scan of his chest demonstrated bilateral pulmonary metastases. The patient underwent surgical exploration, but was felt to have unresectable disease due to involvement of the right mainstream bronchus.A request was made for the patient to undergo cryotherapy of these pulmonary lesions. The Health Plan has denied this request citing the procedures lack of proven efficacy in this setting.The benefit of cryotherapy for pulmonary metastasis is unknown; the Karamanos group who is involved in a clinical trial has reported short-term outcomes, but no long-term outcomes in terms of disease control. The biology of this patients disease is very foreboding for an aggressive course due to rapid recurrence of bilateral disease within three months of surgical treatment for an isolated metastasis. In this situation, it is unlikely that any further local therapy would be beneficial as subsequent recurrences are extremely probable. Based upon the data currently available, the benefit of the requested procedure has not been established and there are alternative therapies that are likely to have an equivalent or better chance of providing benefit to the patient. Accordingly, I have determined that the requested procedure is not likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. Therefore, the Health Plans denial should be upheld.Physician 3:  The patient is a 16-year-old male who presented at age 15 with a non-metastatic osteosarcoma.  In September 2004 he presented with left upper arm pain and swelling and was found to have a left humerus osteosarcoma. He had no metastases. From September 2004 through December 2004 he received cisplatin, doxorubicin, and high-dose methotrexate. Limb salvage therapy was performed in September 2004.  Pathology revealed 89% necrosis. From January 2005 through May 2005 he completed post-operative chemotherapy. In September 2005 he experienced a peripheral neuropathy of unknown etiology. A left distal humerus recurrence was found in May 2006 with no metastases. The patient then underwent a left arm amputation. Also in May 2006, a surveillance chest CT scan showed a pulmonary nodule. It was resected and revealed osteosarcoma. In August 2006 a surveillance CT scan showed three new lesions (in both lungs, one near the right mainstem bronchus). An attempt was made to excise the mainstem bronchus lesion, but it was found to be not resectable. The patients provider has recommended the patient undergo cryotherapy for treatment of the pulmonary metastases.Cryotherapy is a legitimate treatment modality.  While the benefits of cryotherapy in treatment of pulmonary metastases have not been shown in large population, multi-center, controlled clinical studies, this approach to care has been used extensively for prostate cancer and renal tumors and has been proven safe and effective. Finally, while there are no large studies of cryotherapy in this setting, it should be pointed out that there are no large population, multi-center, controlled clinical studies of chemotherapy in this setting either.  There is a role for local therapy for treating osteosarcoma. The two most important modalities for treating osteosarcoma are chemotherapy and surgery. Chemotherapy is understandably not a good palliative option in this case. The patient has been heavily treated and would require fairly intensive chemotherapy and would experience significant toxicity.  His neuropathy might compound his risk of toxicity. Thus, the more commonly used approaches are not practical options for this patient. In this difficult situation, cryotherapy is a reasonable alternative; it is a minimally invasive alternative to surgery, an approach that could control metastatic disease as surgery would, but without the morbidity and discomfort of bilateral thoracotomies.While the patient is not currently experiencing pain or dyspnea one of the lesions is near the right main stem bronchus. Thus, the patients right lung is threatened by further growth, and in a short time he will probably become more symptomatic. Local therapy is more likely to be effective when lesions are small and patients are well enough to tolerate this therapy. The procedure itself is minimally invasive and therefore, not likely to cause more harm than good.Based upon the information set forth above, I have determined that the requested procedure is likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. Therefore, the Health Plans denial should be overturned.