Volume 15, Issue 2 , Pages 133-134, February 2009
Defining Medicare Coverage for MDS Treatment and Research: A Time for Action?
Article Outline
The only therapy with the potential to cure myelodysplastic syndromes (MDS) is allogeneic hematopoietic stem cell transplantation (HCT) from either a related or unrelated donor. Despite recent approvals by the Food and Drug Administration of 3 new agents for the palliative treatment of MDS, the majority of patients will not obtain long-term benefit, and will ultimately die of their disease. Identifying which patients can benefit from HCT and understanding how best to integrate transplantation into their treatment plan is therefore critical in counseling patients with MDS. The issue that now confronts the transplant community and the patients we serve is that we can neither conduct the studies most needed to improve transplant outcomes or even be assured of offering the option of curative therapy to the group of patients who most often are in need.
The heterogeneous nature of MDS, the complexities of the population most at risk for MDS, and the risks associated with HCT frustrate our ability to make clear recommendations to patients in what otherwise would be a discussion of therapy with curative intent versus palliation. The MDS are a diverse collection of disorders in which patients present with sequelae of ineffective hematopoiesis. Although some patients are asymptomatic, most present with signs or symptoms of anemia accompanied by infection or bleeding [1]. MDS becomes more common as we age; although the overall MDS incidence is 3.3 per 100,000, the incidence in patients over 70 is between 15 and 50 per 100,000 [2]. With a median age at presentation of 76 years, comorbid diseases significantly impact treatment options and outcomes. To further complicate management of these patients, current prognostic scoring systems are often poorly predictive for individual patients.
The diversity of MDS, the availability of new nontransplant treatment options, and advances in our ability to treat older patients and those with comorbid conditions renders timely the publication of the evidence-based review of transplantation in MDS in this issue of BBMT [3]. The multidisciplinary panel of experts convened for this review is to be congratulated on a complete evaluation of current evidence and a transparent analysis leading to a series of treatment recommendations in Table 3 of the review. Even with these recommendations, it is clear we need to know more. The panel further identifies 4 priority areas for research as a guide for future studies: “(1) the benefit of using alternative donor sources (eg, cord blood; haploidentical family donors) for patients without matched sibling or unrelated donors; (2) the role and appropriate timing of allogeneic SCT in combination with hypomethylating and immunomodulatory treatment regimens; (3) randomized trials comparing the safety and efficacy of various novel agents for treating MDS; and (4) the influence of the various MDS treatment modalities on patient-reported quality-of-life outcomes.”
Current Medicare policies for reimbursement of HCT for MDS now serve as the major obstacle in addressing these research priorities. Worse still, we cannot offer potentially curative therapy to Medicare beneficiaries or other patients whose health insurance policies follow Medicare guidelines because of practical concerns about reimbursement.
Medicare's current National Coverage Determination (NCD) on HCT does not include allogeneic transplant for MDS. The absence of a national policy allows for coverage at the discretion of local Medicare Contractor Medical Directors. The Medicare contractors have not created explicit local policies, nor are they likely to do so. Additionally, Medicare structure and policies effectively exclude the type of medical appeal process prior to treatment that is routinely available to commercially insured patients. As a result, the procedure is not only too expensive for hospitals to risk nonpayment, but patients and their families become at risk for self-payment if coverage is denied.
For patients whose insurance coverage is governed by Medicare NCDs, HCT can only be assured when and if MDS evolves to acute leukemia. This limitation requires patients to accept increased risks and decreased likelihood of cure to receive definitive treatment. We should consider whether this is an appropriate policy.
The lack of extensive published data for transplantation in patients over the age of 65 years with MDS is, at least in part, a result of these funding policies. Current data in a variety of hematologic diseases using reduced intensity HCT for older patients and those with comorbid conditions validate the safety and effectiveness of the technique 4, 5, 6. These observations and the evidence for transplantation as curative therapy detailed in the adjoining evidence-based review form the foundation for changing the Centers for Medicare and Medicaid Services policy. Building on this foundation, preliminary data from a retrospective study of over 1000 patients with MDS or acute myelogenous leukemia (AML) transplanted between 1995 and 2005 were presented by the Center for International Blood and Marrow Transplant Research at the recent 2008 American Society of Hematology annual meeting [7]. Patients were stratified according to age cohorts for comparison: 40-54, 54-59, 60-64, and ≥65 years. Multivariate analysis revealed no statistically significant impact of age on treatment-related mortality, relapse, or survival. The authors concluded that outcomes for older adults undergoing allogeneic HCT are not significantly different than for younger adults, even after adjusting for multiple risk factors, and age by itself should not be the limiting factor for proceeding to allogeneic HCT in older patients with AML or MDS.
The American Society of Blood and Marrow Transplantation in partnership with the National Marrow Donor Program have committed to address the lack of a NCD for transplantation in MDS. A joint committee of the 2 organizations has already accomplished much background work in preparation for the initiation of a dialogue with the Centers for Medicare and Medicaid Services (CMS). Their efforts have been careful, methodic, and like the current review, evidence-based. This initiative is important to MDS patients in the United States, and both the American Society for Blood and Marrow Transplantation (ASBMT) and the National Marrow Donor Program (NMDP) are to be commended for their leadership.
Research designed to describe optimal treatment strategies for patients with MDS is important, but must not obscure a central point. Therapy with the potential to cure MDS is now available. Reimbursement policies that force patients and their physicians to accept an increased risk of mortality and diminished therapeutic benefit prior to accessing curative treatment are ill-conceived and not acceptable.
Acknowledgments
Financial disclosure: The author has nothing to disclose.
References
- . Myelodysplastic syndromes. Blood. 2008;111:4841–4851
- Epidemiology of myelodysplastic syndromes and chronic myeloproliferative disorders in the United States, 2001-2004. Blood. 2008;112:45–52
- Oliansky DM, Antin JA, Bennett JM, et al. Role of cytotoxic therapy with hematopoietic stem cell transplantation in the therapy of myelodysplastic syndromes: an evidenced-based review. Biol Blood Marrow Transplant.
- . Stem cell transplantation with reduced-intensity conditioning regimens: a review of ten years experience with new transplant concepts and new therapeutic agents. Leukemia. 2006;20:1661–1672
- . Reduced-intensity conditioning and allogeneic hematopoietic stem cell transplantation for acute myeloid leukemia. Expert Rev Anticancer Ther. 2008;8:785–798
- . Viewpoint: what is the role of allogeneic haematopoietic cell transplantation in the era of reduced-intensity conditioning—is there still an upper age limit? A focus on myeloid neoplasia. Leukemia. 2007;21:1357–1362
- Non-myeloablative hematopoietic stem cell transplantation in older patients with AML and MDS: results from the Center for International Blood and Marrow Transplant Research (CIBMTR). Blood. 2008;12:135a;[abstract 346]
Financial disclosure: See Acknowledgments on page 134.
PII: S1083-8791(08)01102-6
doi:10.1016/j.bbmt.2008.12.492
© 2009 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.
Volume 15, Issue 2 , Pages 133-134, February 2009
