Appeal type: Medical necessity
Decision: Overturned in Part

This is a patient who was prescribed IVIG as maintenance therapy for myasthenia gravis. The appeal stated the following: ""This patient has failed multiple other treatments but has responded to the requested treatment. The plan states the treatment is not FDA-approved, and suggested using any of three other treatments (steroids, chemotherapy) that are, significantly, also not Food and Drug Administration(FDA)-approved. In fact, the only FDA-improved RX for myasthenia gravis is pyridostigmine, which the patient failed; it is standard of care to use non-FDA-approved RX in all cases of generalized myasthenia gravis." It was noted that "the patient's formal commercial insurance paid for IVIG for years." The health plan's determination is overturned in part. IVIG (Gamunex-C) is not medically necessary as maintenance therapy for myasthenia gravis. IVIG is well established as a medication to be used in the setting of myasthenia gravis exacerbations, flares or myasthenic crises. There is no evidence in the medical literature that it is effective as maintenance therapy for this disorder. There are evidence-based treatments, including steroids, oral immunosuppressants (methotrexate, azathioprine), eculizumab (Soliris, FDA-approved for acetylcholine receptor antibody positive myasthenia gravis) and rituximab. There is no rationale why the patient cannot use an evidence-based FDA-approved treatment such as eculizumab or an evidence-based non-FDA approved treatment such as rituximab, a steroid or an oral immunosuppressant for maintenance therapy.
The patient had been symptomatic for myasthenia gravis, had a thymectomy, and had been treated with intravenous immunoglobulin (IVIG) at which time she was started on rituximab. She was on prednisone because of worsening. Azathioprine resulted in severe nausea, mycophenolate resulted in recurrent herpetic outbreaks that were continuing. Mycophenolate was not tolerated. Rituximab caused severe exhaustion for two weeks after treatment but was continuing.  There is no documentation of a failure of rituximab. She received IVIG. She was otherwise treated with Gamunex-C infusions on a monthly basis as detailed in office notes  Her antibody status was documented as seronegative disease.
She was noted to be off of IVIG while she changed insurances and was more symptomatic.
Based on the provided information, the patient has been treated with steroids. She has failed three oral immunosuppressants (azathioprine, methotrexate, mycophenolate). She is currently being treated with rituximab with a recent worsening of symptoms. She is not a candidate for eculizumab as she is not acetylcholine receptor antibody positive. Given the worsening of symptoms, and the ongoing treatment with rituximab, and given the lack of known efficacy for IVIG as maintenance therapy (and its known efficacy in treating flares), a one-time treatment of IVIG (2 grams per square meter [gm/sq m] over five days) is indicated to treat the current flare. Again, there is no evidence that maintenance IVIG is effective in treating myasthenia gravis based on current peer-reviewed medical literature and its use as maintenance therapy beyond a single course is not medically necessary. The prior determination is partially upheld, allowing for one additional treatment to treat the current flare.