FeedsImmune 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
- For information for patients, see Inherited Immune System Disorders and Inherited Metabolic Storage Disorders and Transplant.
- For additional NMDP outcomes data, see NMDP Transplant Outcomes.
