Appeal type: Experimental/Investigational
Decision: Upheld

Physician 1: The patient is a 45-year-old woman diagnosed with breast cancer in March 2005. At that time she was found to have a 7.1 cm invasive ductal carcinoma, and she was begun on aggressive chemotherapy with TAC. This ended in June 2005, at which time she had a mastectomy. At that surgery, it was determined that she had had an excellent response to the chemotherapy, with minimal cancer in the breast. Six nodes, however, were found to be positive. The patient had two further cycles of chemotherapy since the surgery, and it is now proposed that she be treated with high dose chemotherapy (HDC) with autologous stem cell transplant. Authorization and coverage for the proposed treatment has been denied by the Health Plan as investigational.The use of HDC in the various stages of breast cancer has been a very controversial topic for many years. It is clear that it is not beneficial in stage IV disease. The question is whether it might be beneficial in women with earlier stage disease, where they are known to have a high risk of recurrence. Specifically, this has usually been defined as women whose cancers had ten or more positive nodes in the axilla.There have recently been reported a number of randomized studies, comparing the results of HDC with standard chemotherapy. Some of these studies have suggested no benefit; one has suggested a benefit in relapse-free survival (but not overall survival) and, furthermore, this may be in women with HER2 negative tumors, such as this patients tumor. The data is clearly very controversial, at best. To further confound the data is the fact that on the HDC trials the chemotherapy was not as effective as modern era therapy, such as what this patient received. Though we only know the patients nodal count (six positive) after chemotherapy, she does not definitively fall into a category of ten or more positive nodes that most of the studies required.The overall consensus in oncology is predominantly that HDC with stem cell transplant is an unproven therapy. 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 2: The patient is 45-year-old female who discovered a mass in her left breast in March 2005. A core biopsy demonstrated moderately differentiated invasive ductal carcinoma, which was estrogen and progesterone receptor positive and did not overexpress HER-2/neu. An MRI scan revealed a 7.1 cm mass and ductal involvement extending to the nipple. Neoadjuvant chemotherapy was recommended with the TAC regimen for four to six cycles, which was followed by a left modified radical mastectomy. Surgical pathology revealed 6 out of 15 axillary lymph nodes were positive for carcinoma.  The patients provider has recommended high-dose chemotherapy and autologous stem cell transplant for the patient. The Health Plan has denied authorization for the proposed therapy citing a lack of established efficacy.Though the submitted materials include several articles indicating a benefit in terms of relapse free survival or event-free survival, there is no data showing overall survival in HER-2/neu individuals with high-dose chemotherapy. The submitted article by Rodehius, et al. indicates overall survival in HER-2/neu individuals, however, this is a subgroup with unplanned analysis. Thus, at this time there is insufficient evidence demonstrating the proposed therapy has clinical value in this setting. 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 3: The patient is a 45-year-old female who has breast cancer. She underwent a core biopsy, which showed moderately differentiated invasive ductal carcinoma, which was estrogen and progesterone receptor positive and did not overexpress HER-2/neu. An MRI scan revealed a mass measuring 7.1 cm and ductal involvement to the nipple. Chemotherapy was given with four to six cycles followed by left modified radical mastectomy. Surgical pathology revealed 6 out of 15 positive axillary lymph nodes. It has been recommended the patient undergo high-dose chemotherapy (HDC) and autologous stem cell transplant. This therapy has been denied by the Health Plan as investigational therapy.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. There is no data to support the use, off protocol, of this modality in this setting. 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.