The survival graphs below illustrate outcomes of unrelated donor hematopoietic cell transplants (bone marrow, PBSC, or cord blood transplants — BMT) coordinated by the National Marrow Donor Program® (NMDP) for adult patients with MDS.
These survival curves do not take into account the International Prognostic Scoring System that correlates blast percentage, cytogenetics and number of lineages affected by cytopenia at diagnosis with outcome.
Figure 1.
Myelodysplastic syndromes: Survival of adult marrow transplant patients by WHO disease classification 2000-2009. (NMDP data)
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Figure 2.
Myelodysplastic syndromes: Survival of adult PBSC transplant patients by WHO disease classification 2000-2009. (NMDP data)
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Both PBSC and cord blood are being studied under FDA investigational new drug (IND) protocols. Researchers continue to investigate rates of graft-versus-host disease (GVHD) and other characteristics of both stem cell sources.
In one such study, the NMDP and the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) conducted a large-scale, multi-center phase III clinical trial to determine if graft source (PBSC or marrow) affects transplant outcomes in unrelated donor transplantation for MDS and other hematologic malignancies. [1]
The study found that PBSC and bone marrow transplant recipients had comparable two-year survival: 51% vs. 46%, respectively (p=0.29). However, chronic GVHD developed more often and was more severe in patients who received PBSC compared with those receiving marrow. [2]
As shown in Figure 3, there is no significant difference in five-year survival for adult patients (≥18 years of age) undergoing marrow or PBSC transplantation for myelodysplastic syndromes (MDS) by age at time of transplant (18-54 yrs. vs. ≥55 yrs.) as measured by the log-rank test, which compares the whole curve of each age group (log-rank p-value = 0.168).
Figure 3.
Myelodysplastic syndromes: Survival of adult marrow and PBSC transplant patients by age at transplant 2000-2009. (NMDP data)
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Figure 4 shows outcomes for adult MDS patients undergoing either myeloablative or non-myeloablative marrow or PBSC transplantation. There is no statistically significant difference in five-year survival between these two patient groups as measured by a log-rank test (log-rank p-value = 0.707).
Figure 4.
Myelodysplastic syndromes: Survival of adult marrow and PBSC transplant patients by conditioning regimen 2000-2009. (NMDP data)
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References
- BMT CTN Protocol 0201 - A Phase III randomized multicenter trial comparing G-CSF mobilized peripheral blood stem cell with marrow transplantation from HLA compatible unrelated donors.
https://web.emmes.com/study/bmt/protocol/0201__protocol/
0201__protocol.html
- Anasetti C, Logan BR, Lee SJ, et al. Peripheral-blood stem cells versus bone marrow from unrelated donors. N Engl J Med. 2012; 367(16): 1487-1496.
http://www.nejm.org/doi/full/10.1056/NEJMoa1203517