Appeal type: Experimental/Investigational
Decision: Upheld Decision of Health Plan

Physician 1: The patient was diagnosed with chronic lymphocytic leukemia (CLL) in 2000. He was initially treated with chemotherapy and immunotherapy. A bone marrow biopsy in June 2005 revealed relapse of the CLL as well as myelodysplasia. It was recommended that he undergo bone marrow transplantation but no HLA-identical match, either related or unrelated could be found. The patient then went to Israel and received a haploidentical graft from his daughter. The patient died in October 2005.At issue is whether haploidentical stem cell transplantation was likely to be more beneficial for the patient than any available standard therapy.The standard transplant for myelodysplasia is an allogeneic transplant with HLA-identical stem cells, either from a relative or an unrelated donor. Unfortunately, no such match can be found in many patients, as was the case here. In an effort to deal with this problem, a variety of investigational approaches have arisen including the use of haploidentical transplants. This consists of the transplantation of stem cells from mismatched donors, i.e., donors whose cells are not immunologically identical to those of the recipient.The very serious problem with mismatched donors is the potential for graft versus host disease (GVH), which can be extremely debilitating and life threatening. A variety of complex manipulations of these mismatched cells are being looked at in an effort to allow for their use as donor cells without the problem of GVH. One major drawback of this approach is that in many studies the elimination of the GVH has also interfered with the anti-leukemic impact of the treatment.Though there have been anecdotal reports of success with this approach, there is no convincing data that the use of haploidentical stem cell transplant is a viable and successful therapy in the management of leukemia and/or myelodysplasia. Therefore, I have determined that the procedure at issue was 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 had aggressive chronic lymphocytic leukemia (CLL) and myelodysplasia. He became pancytopenic. He was referred to a center in Israel for a haploidentical stem cell transplant in August 2005. Unfortunately, the patient subsequently developed neurological complications and multi-organ failure. He died in October 2005. The Health Plan has denied coverage for haploidentical stem cell transplantation on the basis that it is considered experimental/investigational.Review of the relevant scientific literature reveals insufficient data supporting the safety and efficacy of haploidentical transplants in patients with end-stage CLL and myelodysplasia. In this case, the patient was gravely ill and it does not appear he was treated on a protocol with supporting background literature, a specific study question, and clear eligibility criteria. As such, there was inadequate evidence supporting the use of haploidentical stem cell transplantation in this clinical setting. Therefore, I have determined that the procedure at issue was 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 3: The patients family has requested reimbursement for haploidentical stem cell transplantation performed for treatment of chronic lymphocytic leukemia (CLL) and myelodysplasia. The patient was diagnosed with CLL in 2000, treated extensively and then experienced a relapse in June 2005. The patients provider indicated that he proposed a transplant procedure but since his medical center did not have relevant transplant experience, he recommended a provider in Israel.  Successful engraftment occurred, but the patient died from a complication in October 2005.At issue in this case is whether the procedure at issue was likely to be more beneficial for treatment of the patients medical condition than any available standard therapy.  Review of the relevant scientific data reveals there is no treatment alternative for myelodysplasia that offers a chance for significant amelioration of disease process, cure or long-term survival other than allogeneic stem cell transplant. Allogeneic transplantation has been in continuous development since the 1960s. Progress has extended the age of the recipient significantly and matched unrelated donors and haploidentical donors are used in a number of clinical transplant centers.With regard to the patients CLL, the remaining treatments that were available to him left essentially no chance for long-term amelioration, and use of these alternatives would have been severely compromised by the presence of myelodysplasia. The regimen for stem cell transplant offers the most effective therapy for CLL, and requires hematopoietic stem cell rescue.  In addition, the use of allogeneic donor stem cells offers the useful advantage of immune therapy to target the leukemia by graft versus leukemia effects, which have been described and taken advantage of since the 1970s.The patient had two unusual conditions, which when combined are lethal with non-transplant based therapy. Given the absence of alternative therapies, the procedure at issue was likely to be more beneficial for treatment of the patients medical condition than any remaining treatment options. Accordingly, the Health Plans denial should be overturned.