Appeal type: Experimental/Investigational
Decision: Overturned

Physician 1The patient is a 56-year-old man who was diagnosed with myelodysplastic syndrome (MDS) in 1992. His specific form of MDS is refractory sideroblastic anemia. Over the years he has been on a variety of treatments, including Neupogen to stimulate his white blood cell count, and Epogen, to increase his hemoglobin. It appears that the patients hemoglobin has not been responding well to his current treatment and he has been on thalidomide for an unknown period of time. He requests continuation of thalidomide therapy. The Health Plan has denied the request on the basis that thalidomide is considered experimental for treatment of the patients medical condition.MDS encompasses a wide range of subtypes, but all are characterized by a bone marrow that is not functioning normally, such that one or the other of the formed elements of the blood is deficient. Very often such patients become transfusion dependent, as current treatments are often not very effective.Thalidomide, which is useful in certain malignancies, is under study in the treatment of MDS. Several abstracts related to such application of the drug are included in the submitted documentation. Also included is a review of thalidomides use in many conditions, including MDS. In particular this review documented the current knowledge about thalidomide in MDS, demonstrating in many studies (albeit in small numbers of patients) that indeed thalidomide can be useful in terms of increasing or maintaining hemoglobin levels. There are reports of patients no longer being transfusion dependent upon treatment with thalidomide. Thus, thalidomide is becoming an accepted and potentially valuable therapy and is likely to be of real clinical benefit for this patient. 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.Physician 2The patient is a 56-year-old male with longstanding refractory anemia, due to myelodysplasia syndrome (MDS) with excess ringed sideroblasts, despite appropriate erythropoietin therapy. He has slowly developed worsening anemia which is transfusion supported. He is in otherwise good health. The patient has requested authorization for thalidomide therapy. The Health Plan has denied the request indicating that thalidomide is considered experimental for treatment of myelodysplasia.Thalidomide, among its multiple therapeutic uses, has been shown to successfully stabilize and reverse anemia in a portion of patients with myelodysplasia caused anemia. Myelodysplasia has multiple subtypes clearly reflecting different diseases and likely etiologies. The particular subtype the patient has tends to be more responsive than others (i.e., those associated with significant neutropenia and thrombocytopenia) to thalidomide; this is well documented in the literature. Furthermore, the degree of responsiveness to thalidomide in such MDS associated anemias is statistically greater than that seen in glioblastoma multiforme and renal cell carcinoma, two conditions for which treatment with thalidomide has already been approved. 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. Therefore, the Health Plans denial should be overturned.Physician 3The patient is a 56-year-old man with an excellent performance status and a diagnosis of myelodysplastic syndrome (MDS) with refractory anemia. The patient has been receiving growth factors with marginal response. He has requested authorization for treatment with thalidomide in addition to his growth factors. The Health Plan has denied this request citing the experimental status of thalidomide therapy in this clinical setting.There is literature that appears to show some benefit from thalidomide therapy in refractory anemias associated with MDS. However, the use of thalidomide in the setting of MDS has not been validated by multi-institutional, prospective, clinical trials to date. The standard of care for a patient who is younger than 60 years of age with refractory disease and good performance status would be a chemotherapy-based regimen, including strong consideration of dose-intensive therapy. This standard of care is consistent in all databases, including National Comprehensive Cancer Network guidelines. Although the convenience and relatively safe profile of thalidomide is attractive, there is not sufficient evidence that standard approaches have been ineffective.  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.