Appeal type: Experimental/Investigational
Decision: Overturned

Physician 1:  The patient is a 44-year-old male originally diagnosed with Stage IVB Hodgkins Disease in 1975 treated with C-MOP chemotherapy and a single fraction of thoracic radiation. He next presented in June 2004 with an abnormal chest X-ray showing a spiculated 1.4cm nodule in the right upper lobe. Ultimately he underwent resection of an invasive non-small cell lung cancer and per the record, he had a 1.7cm lung tumor resected with pleural invasion but no nodal involvement He received no adjuvant therapy for what appears to have been a Stage I lung cancer. Within the same timeframe he was noted to have a progressive thrombocytopenia and was subsequently diagnosed with myelodysplastic syndrome (MDS). Treatment with darbapoietin was recommended, and given his lung cancer in June 2004, aggressive therapy such as transplant was deferred.In the interval from September 2005 through May 2006 the patient underwent CT of the chest, abdomen, and pelvis and PET scan with no evidence of metastatic disease. In June 2006 he was noted to have worsening platelet and white cell counts, though it appears his anemia was stable on darbapoietin. Repeat imaging took place in August 2006 in consideration of possible transplant. The patient received approval for HLA typing and Unrelated Donor Search in July 2006 which did occur, and potential donors were identified. The patient then requested authorization for matched-unrelated-donor (MUD) allogenic transplant. The Health Plan denied his request citing the investigational nature of the therapy in this clinical context.This case appears to present an issue of timing and  consideration of whether the risk of recurrence of the patients lung cancer is low enough and his chances of surviving his lung cancer high enough to balance the curative intent and the significant risks associated with high-dose chemotherapy and MUD transplant.  In this case, the patient is not so symptomatic that he is requiring red cell transfusion or having recurrent infections. He has not been tried on FDA approved therapy beyond Aranesp; other therapies are available which have the capacity to delay disease progression though would not cure it. Finally, the fact the patient has had a recent diagnosis of lung cancer is of significant concern, especially in light of recent CT findings which revealed a 6mm nodule in the anterior left upper lobe. Although not compared with prior scans, it is sufficient to warrant short term surveillance and recurrence of his lung cancer has not yet been definitively ruled-out. Given his relatively good health and manageable disease, transplant is not appropriate at this juncture as it would expose him to significant toxicity and considerable risk of infection, GVHD, or even mortality.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 2:  The patient is a 44-year-old male diagnosed with stage IVB Hodgkins Disease in 1975, and treated with radiation therapy and chemotherapy. He was well until he was found to have an abnormal chest x-ray in 2004 which led to the resection of a 1.7cm adenocarcinoma from the right upper lobe of the lung in June 2004. At that time a bone marrow biopsy revealed myelodysplastic syndrome (MDS). By May 2005 the patient  had refractory cytopenia, though with a minimal percentage of blasts. More recently, the patient has had severe cytopenia of two cell lines, though as yet he has not required red blood cell transfusion. The patient has been seen by several physicians and it has been recommended that he undergo allogenic stem cell transplant. An unrelated marrow donor match has been identified and the patient submitted a request for transplant authorization to his Health Plan. The therapy is deemed investigational in this setting and authorization was denied by the Health Plan.This patients survival from his MDS is not likely to extend beyond three to five years.  Nonetheless, the patients history of lung cancer, resected two years ago, certainly complicates the clinical decision making process.  Though only two years have gone by since the surgery, the majority of recurrences destined to occur with this cancer would have already happened. Thus even at two years, the patients chance for cure is considerably higher than 65%. Based on these factors, allogenic transplant is likely to be of clinical benefit and is not an investigational therapy. However, it is also noted that the patient has a new 6mm nodule in the upper lobe of his left lung. If this turned out to be a recurrence of his lung cancer, then it would be inappropriate to consider transplant for his MDS. If this turns out to be a benign process, then the patient should be authorized to proceed with MUD allogenic transplant. 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 once cancer recurrence in the left lung has been ruled out. Therefore, the Health Plans denial should be conditionally overturned.Physician 3:  The patient is a 44-year-old male with high-risk treatment-related myelodysplastic syndrome (MDS), most likely from Hodgkins disease as a youth. He has also been diagnosed and treated for early stage non-small cell lung cancer with surgical ablation occurring approximately two years ago. The patient is now thrombocytopenic and neutropenic and has requested authorization for allogenic transplant to treat his MDS. The Health Plan has classified this therapy as experimental/investigational and denied the requested authorization.In the absence of a history of lung cancer, allogenic transplant is the standard of care for the treatment of MDS. It is also known that statistically, most clinically aggressive cancers tend to recur within the first one to two years following definitive therapy.These concerns must be balanced against the present threat of progressive MDS in a relatively young man with a good performance status and a qualified unrelated donor match. He has already waited a year for transplant and passed a metastatic work-up just a few months ago. In the absence of aggressive therapy with the MUD transplant, the patient faces grave risks of bleeding and infection from bone marrow failure and a very high risk of transformation to a poor-prognosis acute leukemia. In this reviewers opinion, the medical necessity of therapy for the present threat outweighs the theoretical threat of recurrent lung cancer. 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.