Appeal type: Experimental/Investigational
Decision: Overturned

Physician 1:  The patient is a 39-year-old female with primary autonomic insufficiency which is manifest by disabling orthostatic hypotension and bilateral tonic pupils. There is reported confirmation of the diagnosis with a positive ganglionic acetylcholine receptor antibody test. The patient has failed standard therapy including midodrine and Florinef and is requesting authorization for a treatment protocol which involves plasmapheresis, prednisone and Imuran. The Health Plan considers the proposed therapy experimental/ investigational and denied the request for authorization and coverage.This patient has a subacute, chronic autonomic insufficiency and has undergone an extensive work-up; no other treatable disorder has been identified. She has been diagnosed with primary autoimmune autonomic failure and there is no known standard therapy for this disorder. There is a single case reported in the New England Journal of Medicine (2005) of a patient with positive ganglionic acetylcholine receptor antibodies who was treated with plasmapheresis and had a very positive response. The records submitted did not include the test results for the ganglionic acetylcholine receptor antibody which were conducted by an out-of-state expert in the field. If the presence of these antibodies cannot be confirmed, then there is insufficient evidence to support treatment with plasmapheresis. However, if the patient has indeed tested positive for the ganglionic acetylcholine receptor antibody, then the patient should be authorized to proceed with the plasmapheresis/prednisone/Imuran therapy. As such, 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 pending confirmatory testing. Therefore, the Health Plans denial should be overturned.Physician 2:  The patient is a 39-year-old female with severe orthostasis and tonic pupils who has been found to have an autoimmune autonomic neuropathy. Her provider indicates a consulting expert has confirmed the presence of ganglionic acetylcholine receptor antibodies. Treatment with plasmapheresis, prednisone and Imuran has been proposed and a request for authorization was submitted to the Health Plan. The Health Plan denied the request based on a determination that the therapy is experimental/investigational for the treatment of the patients autonomic neuropathy.This patient has a rare autoimmune neuropathy (autonomic) and standard approaches to treatment have been exhausted. The literature contains case reports describing the success of similar multimodal treatment with plasmapheresis, steroids and Imuran. Because symptomatic therapy has not provided relief, the proposed therapy makes good scientific sense as it removes the harmful antibodies and has been anecdotally successful. 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.Physician 3:  The patient is a 39-year-old female who has been diagnosed as having a rare neuropathy with predominantly autonomic features. An extensive workup indicates a probable autoimmune basis for her condition. The symptoms are disabling and she has severe orthostasis despite appropriate maximal standard medical therapy. The patients gammopathy is without evidence of amyloidosis or neoplasm. The patients provider has requested authorization for treatment with plasmapheresis, prednisone and Imuran. The Health Plan denied authorization citing the investigational nature of the therapy in this clinical context.The patient has a rare form of autoimmune paraprotein-associated neuropathy with autonomic impairment. All standard therapies have failed. The literature supports a benefit from plasmapheresis for other severe forms of polyneuropathy with an autoimmune basis and gammopathy. The Health Plan in fact considers plasmapheresis for some variations of autoimmune paraproteinemia polyneuropathy to be medically necessary. The patients condition is rare enough that controlled trials of treatment with plasmapheresis have not been conducted, and probably will not be. Given the failure of standard therapies and evidence that other forms of immune-mediated neuropathy have been effectively treated with plasmapheresis, the proposed treatment of this patients autoimmune autonomic neuropathy is medically appropriate.Based upon the information set forth above, 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.