Appeal type: Experimental/Investigational
Decision: Upheld

Physician 1The patient is a 57-year-old female presenting with stage IV metastatic carcinoma of the breast. Her evident metastases were numerous axillary lymph nodes and multiple hepatic metastases. She is apparently HER-2/neu positive. FISH status is not included in the information submitted. The patient has achieved an apparent complete remission with Taxotere and Herceptin. She is in good clinical condition and is a candidate for further treatment. It has been recommended that the patient receive high dose chemotherapy (HDC) and autologous stem cell rescue, outside of a clinical trial. The Health Plan had denied authorization and coverage for the recommended therapy on the basis it is considered experimental for patients with metastatic breast cancer.High dose chemotherapy with autologous stem cell rescue for breast cancer as an adjuvant therapy or for metastatic disease has remained an attractive but unproven superior modality to conventional treatment. Patients with metastatic disease in complete remission (i.e., low tumor burden) appear to benefit more than those who are not in this state. However, even in this group of patients, high lymph node positive numbers or ratio, and HER-2/neu positive status remain adverse prognostic features. This patient has both of these features. As such, the requested therapy is not likely to be of greater benefit to this patient than conventional therapy.Based upon the information set forth above, I have determined that the requested therapy is not likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. The Health Plans denial should be upheld.Physician 2The patient is a 57-year-old patient who presented in November 2003 with an axillary mass. She was found to have right breast cancer with 14/14 nodes being positive. In addition, PET scan revealed evidence of metastatic disease in her liver. She was treated with Herceptin and Taxotere, and experienced a complete remission after ten cycles of therapy. It is now being proposed that she receive high dose chemotherapy (HDC) and autologous stem cell rescue, outside of a clinical trial.Overall, in randomized studies high dose chemotherapy and autologous stem cell transplant have not shown a survival advantage over conventional therapy in patients with metastatic breast cancer. The remaining issue of some continued controversy is whether there is a subgroup within the entire group of patients with metastatic disease, who would benefit from HDC. Particularly, this has focused on the small percentage of patients who enter a complete remission after initial therapy. Presumably such patients have had their tumor load reduced to microscopic levels and might theoretically be cured by further aggressive treatment. However, it remains unclear as to whether such a subgroup exists who would indeed benefit from HDC. These patients rightfully should be treated on a randomized clinical trial until the data develops that HDC is superior therapy. Accordingly, the use of HDC in a patient such as this patient is of unproven benefit. Therefore, I have determined that the requested therapy is not likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. The Health Plans denial should be upheld.Physician 3The patient is a 57-year-old female with stage IV breast cancer who had a complete radiographic remission following an initial course of Taxotere and Herceptin immuno-chemotherapy. Her physicians have recommended a course of high dose chemotherapy (HDC) with stem cell support (autologous stem cell transplantation) for consolidation. There are no clinical trials available locally. The Health Plan has denied coverage for the requested therapy on the basis it is considered experimental for treatment of the patients medical condition.The role of HDC and autologous stem cell transplantation has been the subject of many studies over the last decade without a consensus in the academic oncologic community or the literature. According to National Comprehensive Cancer Network guidelines HDC with stem cell transplantation is of unproven efficacy for patients with breast cancer. Although there is a cogent argument put forward by the patients physicians regarding her particular care, there is no data to support the use, off protocol, of this modality in this setting. Despite the potential inconvenience, participation in a clinical trial for patients with this clinical situation is imperative in order to understand the current and future role of this therapeutic approach. Off protocol use is not supported by evidence of benefit over further salvage, standard dose chemotherapeutic regimens. Therefore, I have determined that the requested therapy is not likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. The Health Plans denial should be upheld.