Appeal type: Experimental/Investigational
Decision: Upheld

Physician 1The patient is a 55-year-old man with a history of renal cell carcinoma who underwent nephrectomy in March 2002. Prior to that time he had been diagnosed with acute myelogenous leukemia (AML), which went into remission in 2001 with chemotherapy. He has not had a relapse of his acute leukemia. In May 2003, the patient was found to have multiple lung metastases from his renal cell carcinoma. As of June 2004 these appeared to have increased in size.The patient has a fully HLA-matched sibling who has already donated allogeneic stem cells. It has been proposed that the patient be treated with non-myeloablative allogeneic stem cell transplant. The goals of this therapy would be to control his metastatic renal cell carcinoma, and presumably to provide further treatment of his AML. The Health Plan has denied authorization for the requested therapy on the basis that it is considered investigational.The submitted literature includes a variety of small studies utilizing the requested approach in metastatic renal cell carcinoma, which is relatively unresponsive to conventional therapy. Many institutions have embarked on clinical trials of non-myeloablative allogeneic stem cell transplantation in metastatic renal cell carcinoma, particularly after the report of some success in 19 patients in the report by Childs. However, as yet there are no controlled comparisons of non-myeloablative allogeneic stem cell therapy to more conventional therapies in the literature.Accordingly, this treatment concept remains unproven for metastatic renal cell carcinoma, with no firm evidence that it is more likely to be beneficial than other treatments, e.g., IL-2 and/or interferon. 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 2The patient is a 55-year-old male with simultaneous diagnoses of acute myelogenous leukemia (AML) and metastatic renal cell carcinoma. The patient has experienced transient partial regression of renal cell carcinoma lesions following nephrectomy. He has not had a trial of biotherapy such as IL-2 or interferon. The patients provider has recommended non-myeloablative allogeneic stem cell transplantation. The patient has a histocompatible sibling. The stem cell product has been cryopreserved. The Health Plan has denied authorization for the requested therapy on the basis that it is considered investigational.The patient is technically eligible for the study used at Scripps because its eligibility criteria are extremely liberal. However, all of the data for allogeneic stem cell transplantation in renal cancer are for cytokine-therapy failures. In contrast with some of the data submitted, most investigators consider high-dose IL-2 to have an overall response rate of 15-20% and complete remission rate of 5-7% rather than the 2% cited from a single source (Negrier). On the contrary, the data for allogeneic transplants in renal cancer has not held up in studies outside of the National Institutes of Health. Despite the large number of centers offering a protocol for these patients, enthusiasm for its use has markedly diminished. The total number of such transplants over the past two years is probably no greater than one to two per center. Furthermore, the patient has no clear indication for allogeneic transplant as consolidation for his AML since it has been in remission for over two years. 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 3The patient is a 55-year-old male with a history of acute myelogenous leukemia (AML) diagnosed in approximately September 2001. Treatment was appropriate and resulted in a complete remission, which has lasted three years to date. He had a simultaneous right hypernephroma ultimately treated empirically. The hypernephroma has recurred diffusely in his lungs and is progressive and symptomatic. He is otherwise in good health. It has been recommended the patient undergo non-myeloablative allogeneic stem cell transplantation. The Health Plan has denied the request on the basis that the requested therapy is considered investigational.The treatment available for recurrent hypernephroma is largely palliative and ineffective (except for surgical extirpation of single metastases). While progestational agents such as interferon and IL-2 produce a minority of responses, significant impact is anecdotal. Chemotherapy is even less effective and rarely used. Avastin has recently shown some efficacy, though only in a distinct minority of patients. Allogeneic transplantation has recently been shown to have significant activity, albeit in a small series of patients. Nonetheless, the complete remission rate appears substantially more than high-dose IL-2. This patient is an excellent candidate for such treatment. The major rationale for this is his metastatic hypernephroma since the patient has experienced a three-year remission from AML. The requested therapy is likely to be of greater benefit than any conventional therapies. Therefore, I have determined that the requested therapy is likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. The Health Plans denial should be overturned.