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Myelodysplastic Syndromes (MDS)

Approximately 21,000 people are diagnosed with Myelodysplastic Syndromes (MDS) in the United States each year, with increasing incidence with age. [1] With the development of reduced-intensity conditioning regimens making transplant more feasible for older patients, more MDS patients are now eligible for allogeneic hematopoietic cell transplantation (HCT), the only potentially curative treatment option. [2]

Figure 1 shows that annual transplants for MDS have steadily increased, making MDS the second most common indication for unrelated donor transplants facilitated by the National Marrow Donor Program^® (NMDP)/Be The Match^®.

Figure 1. Unrelated HCT by Patient Diagnosis, Malignant Diseases

Unrelated HCT by Patient Diagnosis, Malignant Diseases

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A 2010 decision by the Centers for Medicare and Medicaid Services (CMS) to cover transplants for MDS when performed as part of a clinical study approved by Medicare allows physicians to offer transplant as an option for older patients. Physicians can enroll Medicare-eligible patients with MDS in a CMS-approved study conducted by CIBMTR^® (Center for International Blood and Marrow Transplant Research^®), our research program.

Preliminary results from the CIBMTR clinical trial showed that in patients who are eligible for allogeneic HCT, there was no difference in 100-day mortality or overall survival at 2 years for patients age 55-64 years compared to patients age 65 years and older. [3]

Advances

Clinical advances that have improved the outcomes of allogeneic transplants in patients with MDS include:

Improvements in post-transplant clinical care
The use of hypomethylating agents to induce a remission or stabilize the disease, allowing a bridge to transplant for many patients [4,5]
Research demonstrating that physical function and organ comorbidities, not age alone, should be the drivers for transplant eligibility [6,7]
A 2012 update to the International Prognostic Scoring System (IPSS) to better risk stratify patients with MDS [8]

Outcomes

Improved Survival

Transplant outcomes have steadily improved over time (see Figures 2 and 3).

Outcomes for allogeneic transplantation in adults with MDS are shown below. View additional MDS slides showing demographic data and transplant trends.

Data in this section have been prepared by CIBMTR, our research program.

Figure 2. MDS Survival Over Time, Unrelated HCT

MDS Survival Over Time, Unrelated HCT

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Figure 3. MDS Adult Unrelated HCT Improved Survival Over Time

MDS Adult Unrelated HCT Improved Survival Over Time

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Figure 4. MDS Survival, Unrelated Marrow HCT, by Disease Status

MDS Survival, Unrelated Marrow HCT, by Disease Status

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Figure 5. MDS Survival, Unrelated PBSC HCT, by Disease Status

MDS Survival, Unrelated PBSC HCT, by Disease Status

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Figure 6. MDS Survival, Unrelated HCT, by Age

MDS Survival, Unrelated HCT, by Age

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Figure 7. MDS Survival, by Preparative Regimen, Adults ≥55 Years

MDS Survival, by Preparative Regimen, Adults ≥55 Years

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Figure 8. MDS Survival, HLA-Matched Sibling Donor HCT, by Disease Status

MDS Survival, HLA-Matched Sibling Donor HCT, by Disease Status

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Referral Timing Guidelines

These guidelines highlight disease categories that include patients at risk for disease progression and who should be referred for a consultation for autologous or allogeneic transplantation [9].

High-resolution HLA typing is recommended at diagnosis for all patients

Transplant Consultation Guidelines: Myelodysplastic Syndromes – Adult

Any intermediate or high IPSS or IPSS-R score
Any MDS with poor prognostic features including:

Treatment-related MDS
Refractory cytopenias
Adverse cytogenetics and molecular features
Transfusion dependence
Failure of hypomethylating agents or chemotherapy
Moderate to severe marrow fibrosis

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View complete Referral Timing Guidelines

Transplant Consultation Guidelines: Myelodysplastic Syndromes – Pediatric

Pediatric Myelodysplastic Syndromes

At diagnosis for all subtypes

Juvenile Myelomonocytic Leukemia (JMML)

At diagnosis