Appeal type: Experimental/Investigational
Decision: Upheld

Physician 1: The patient is a 63-year-old female first diagnosed with breast cancer in early 1999. At that time she underwent mastectomy, followed by adjuvant chemotherapy with Adriamycin and Cytoxan, then taxotere. She was then well for five years when she developed recurrence in a supraclavicular node, which was completely resected. No other metastatic disease was found, and she has been receiving further chemotherapy. It is proposed that she be treated with high dose chemotherapy and autologous stem cell rescue. The use of this treatment is the subject of this appeal. For many years, the use of high dose chemotherapy (HDC) with peripheral stem cell transplant (PSCT) has been a very controversial treatment in breast cancer. Primarily, it has been studied in two very separate situations. The first of these is the adjuvant situation, wherein women considered at high risk of relapse (usually meaning they have ten or more positive axillary nodes at the time of diagnosis), are treated with HDC in order to lower their risk of recurrence. Though initial uncontrolled studies looked promising, subsequent randomized studies have not really borne the promise out as yet. Two recent papers were cited in the appeal. A 2003 randomized study by Rodenhius did suggest an improved relapse-free survival in the group treated with HDC, but as yet no real difference in overall survival. A 2003 study by Zander did not show a significant difference in survival in the HDC group. The second group wherein HDC has been used is in women with stage IV or metastatic breast cancer. In general, the data from such studies has looked even less promising than in the high-risk adjuvant group. One could note a recent editorial (see Schmid reference above) for a discussion of this. Indeed, a 2005 study using tandem (two cycles of HDC) therapy in metastatic breast cancer failed to show any benefit from HDC. The patient has had recurrence in the supraclavicular area five years after her original treatment for breast cancer. As such, she appears to fall into the group of women with stage IV or metastatic breast cancer. Though clinical trials do continue in such women, to determine if there is a sub-group wherein HDC might be of advantage, thus far no such group has been identified. Therefore, there is a lack of evidence supporting the proposed therapy in this case. Based upon the information set forth above, I have determined the requested therapy 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 63-year-old female who is requesting approval for high dose chemotherapy and a stem cell transplant (HDC-SCT) to treat a localized recurrence of breast cancer originally diagnosed and treated in 1999. She was diagnosed with post-menopausal, hormone receptor negative, Her-2 neu negative disease, Stage III (4+ lymph nodes), completed aggressive adjuvant therapy after mastectomy, and had surgical extirpation of a documented ipsilateral supraclavicular recurrence five years after the end of therapy. Given the risk for a systemic recurrence, she has been referred for, and offered, HDC-SCT as consolidation for her high-risk disease. The autologous transplant will be part of a clinical trial comparing conditioning/preparative regimens; this trial was not designed to address the controversies that persist surrounding the efficacy of HDC-SCT in the treatment of breast cancer. The referring physician has cited literature, primarily an article from the New England Journal of Medicine, Rodenhuis, et al. 2003, in support of the proposed HDC-SCT. However, in the same issue of this journal, Tallman, et al. reported on a series of patients in which HDC-SCT appeared to be less efficacious than the Rodenhuis report. An editorial that compares and contrasts these reports (see Elfenbein reference above) concludes that neither study is representative of current high-dose chemotherapy. Furthermore, the Zander trial cited by the patients treating provider is not informative or determinative because of the short follow-up time and the patient parameters with regard to definition of high-risk disease. A large cohort of relevant high-risk women were studied in a recently reported in the Journal of National Cancer Institutes with a median follow-up of six years. In each of these studies, there is no definitive evidence in support HDC-SCT providing a survival advantage over other available therapy. In conclusion, HDC-SCT is not considered a standard of care in either community or academic settings for patients with high-risk breast cancer but currently without evidence of active disease. Furthermore, effective standard chemotherapy options remain, should the patients clinical status change. 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 63-year-old woman with a history of breast cancer. She is status post cytotoxic chemotherapy. Her tumors characteristics were ER/PR/Her-2 neu negative (triple negative state). She had approximately a five-year disease free interval when she relapsed with a positive neck node. At her physicians recommendation she is requesting high-dose chemotherapy with autologous stem cell transplant. The Health Plan has denied this request indicating the therapy is experimental and investigational. At the present time there is no evidence that this patient would have an improved survival with high-dose chemotherapy. Several trials in the adjuvant and metastatic setting have not revealed an improved survival rate. In addition, this is not standard treatment. Furthermore, there are several alternative chemotherapeutic options available for this patient. Also, radiation therapy may be an option. 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.