Myelodysplastic Syndromes (MDS) Outcomes

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.

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 myeloablative transplant patients by FAB disease classification 1998-2006. (NMDP data)

Myelodysplastic syndromes: Survival of adult marrow myeloablative transplant patients 1998-2006
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Adult marrow transplants facilitated by the NMDP, 1998-2006 
Disease Stage # of Transplants Kaplan-Meier 5-Year Survival*
RA / RARS 77  37% ± 11%
RAEB / RAEB-T 178  27% ± 7%
* 95% confidence interval

Figure 2.
Myelodysplastic syndromes: Survival of adult PBSC myeloablative transplant patients by FAB disease classification 1998-2006. (NMDP data)

MDS: Survival of adult PBSC transplant patients by FAB disease classification 1998-2006
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Adult PBSC transplants facilitated by the NMDP, 1998-2006 
Disease Stage  # of Transplants Kaplan-Meier 5-Year Survival*
RA / RARS  73 29% ± 15%
RAEB / RAEB-T 127  36% ± 9%
* 95% confidence interval

A large-scale, multi-center phase III clinical trial is underway to determine if graft source (PBSC or marrow) affects transplant outcomes in unrelated donor transplantation for MDS and other hematologic malignancies. The trial is being conducted through the Blood and Marrow Transplant Clinical Trials Network (BMT CTN Study 0201). [1]

As shown in Figure 3, there is a 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 = 0.03). However, a chi-square test, which compares the survival rate of each age group at the five-year time point only, is not significant (chi-square p-value = 0.07).

Figure 3.
Myelodysplastic syndromes: Survival of adult marrow and PBSC transplant patients by age at transplant 1998-2006. (NMDP data)

MDS: Survival of adult marrow and PBSC transplant patients by age 1998-2006
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Adult marrow and PBSC transplants facilitated by the NMDP, 1998-2006 
Age at time of Transplant  # of Transplants Kaplan-Meier 5-Year Survival*
18-54 years  289 29% ± 6%
>55 years 163  19% ± 8%
* 95% confidence interval

Figure 4 shows outcomes for adult MDS patients with RAEB/RAEB-T 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 = 0.11). However, a chi-square test that compares the survival rate of each conditioning regimen at the 5-year time point only is statistically significant (chi-square p-value = 0.003).

Figure 4.
Myelodysplastic syndromes - RAEB/RAEB-T: Survival of adult marrow and PBSC transplant patients by conditioning regimen 1998-2006. (NMDP data)

MDS: Survival of adult marrow and PBSC transplant patients by conditioning regimen 1998-2006
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Adult marrow and PBSC transplants for MDS RAEB/RAEB-T facilitated by the NMDP by conditioning regimen, 1998-2006 
Conditioning Regimen  # of Transplants Kaplan-Meier 5-Year Survival*
Myeloablative  305 30% ± 6%
Non-Myeloablative 147  14% ± 9%
* 95% confidence interval


References

  1. 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



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