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Disease-Specific Indications and Outcomes

  • AML - Adult
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Research Spotlight
  • Younger matched unrelated donors provide outcome benefits over older matched siblings for MDS patients undergoing allogeneic transplant

    March 2022

  • Review: Scientific advances in myelodysplastic syndromes

    March 2019

  • Adding genomic data to new prognostic model improves predictors of post-HCT outcomes in patients with MDS

    December 2018

  • Transplant Indications and Outcomes
  • Disease-Specific Indications and Outcomes
  • MDS
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  • Case Study: Management of a Patient with Intermediate-Risk MDS
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Patient Resources

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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

Download slide "Unrelated HCT by Patient Diagnosis, Malignant Diseases"

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

Download slide "MDS Survival Over Time, Unrelated HCT"

Figure 3. MDS Adult Unrelated HCT Improved Survival Over Time

MDS Adult Unrelated HCT Improved Survival Over Time

Download slide "MDS Adult Unrelated HCT Improved Survival Over Time"

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

MDS Survival, Unrelated Marrow HCT, by Disease Status

Download slide "MDS Survival, Unrelated Marrow HCT, by Disease Status"

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

MDS Survival, Unrelated PBSC HCT, by Disease Status

Download slide "MDS Survival, Unrelated PBSC HCT, by Disease Status"

Figure 6. MDS Survival, Unrelated HCT, by Age

MDS Survival, Unrelated HCT, by Age

Download slide "MDS Survival, Unrelated HCT, by Age"

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

MDS Survival, by Preparative Regimen, Adults ≥55 Years

Download slide "MDS Survival, by Preparative Regimen, Adults ≥55 Years"

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

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

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

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].

Transplant Consultation Guidelines: Myelodysplastic Syndromes – Adult

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

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

Download as a slide (PPT)

Transplant Consultation Guidelines: Myelodysplastic Syndromes – Pediatric

Pediatric Myelodysplastic Syndromes

  • At diagnosis for all subtypes

Download as a slide (PPT)

Juvenile Myelomonocytic Leukemia (JMML)

  • At diagnosis

Download as a slide (PPT)
View complete Referral Timing Guidelines

References

  1. SEER Cancer Statistics Review 1975-2012: MDS. Website accessed 8 March, 2018. Access
  2. Karanes C, Nelson GO, Chitphakdithai P, et al. Twenty years of unrelated donor hematopoietic cell transplantation for adult recipients facilitated by the National Marrow Donor Program. Biol Blood Marrow Transplant. 2008; 14(9, Suppl.): 8-15. Access
  3. Atallah E, Horowitz MM, Logan B, et al. Outcome of patients 65 years and older with myelodysplastic syndrome (MDS) receiving allogeneic hematopoietic stem cell transplantation compared to patients 55-64 years of age (abstract). Blood. 2015; 126(23): 193. Access
  4. Field T, Perkins J, Huang Y, et al. 5-Azacitidine for myelodysplasia before allogeneic hematopoietic cell transplantation. Bone Marrow Transplant. 2010; 45(2): 255-260. Access
  5. Damaj G, Duhamel A, Robin M, et al. Impact of Azacitidine before allogeneic stem-cell transplantation for myelodysplastic syndromes: A study by the Société Française de Greffe de Moelle et de Thérapie-Cellulaire and the Groupe-Francophone des Myélodysplasies. J Clin Oncol. 2012; 30(36): 4533-4540. Access
  6. Platzbecker U, Schetelig J, Finke J, et al. Allogeneic hematopoietic cell transplantation in patients aged 60-70 years with de novo high-risk myelodysplastic syndrome or secondary acute myelogenous leukemia: comparison with patients lacking donors who received azacitidine. Biol Blood Marrow Transplant. 2012; 18(9): 1415-1421. Access
  7. McClune BL, Weisdorf DJ, Pedersen TL, et al. Effect of age on outcome of reduced-intensity hematopoietic cell transplantation for older patients with acute myeloid leukemia in first complete remission or with myelodysplastic syndrome. J Clin Oncol. 2010; 28(11): 1878-1887. Access
  8. Greenberg PL, Tuechler H, Schanz J, et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012; 120(12): 2454-2465. Access
  9. NMDP/Be The Match and ASBMT Recommended Timing for Transplant Consultation. Download (PDF)
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Slides may be downloaded and used without permission for one-time presentation or personal use.  For publication, use in print or web, or repeated presentations, please submit a Copyright Request Form.

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