Immune Deficiencies and Metabolic Disorders Outcomes
The survival graphs below illustrate outcomes of unrelated donor hematopoietic cell transplants (bone marrow, PBSC, or cord blood transplants — BMT) facilitated by the National Marrow Donor Program (NMDP) for patients with inherited immune deficiencies and metabolic disorders.
Figure 1 shows survival curves for selected immune deficiencies.
Immune deficiencies: Survival of pediatric marrow transplant patients 1998-2006. (NMDP data)
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| Pediatric marrow transplants facilitated by the NMDP, 1998-2006 |
| Disease |
# of Transplants |
Kaplan-Meier 5-Year Survival* |
| Wiskott-Aldrich Syndrome |
48 |
80% ± 12% |
| Severe Combined Immunodeficiency (SCID) |
35 |
75% ± 15% |
* 95% confidence interval
Figure 2 shows survival curves for selected inherited metabolic disorders.
Inherited metabolic disorders: Survival of pediatric marrow myeloablative transplant patients 1998-2006. (NMDP data)
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| Myeloablative marrow transplants facilitated by the NMDP, 1998-2006 |
| Disease |
# of Transplants |
Kaplan-Meier 5-Year Survival* |
| Hurler syndrome |
30 |
61% ± 18% |
| Adrenoleukodystrophy (ALD) / Metachromatic Leukodystrophy (MLD) |
29 |
50% ± 20% |
* 95% confidence interval
|
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