• Print
  • Bookmark and Share
  • RSS Feeds Feeds

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.

Figure 1.
Immune deficiencies: Survival of pediatric marrow transplant patients 1998-2006. (NMDP data)

Immune deficiencies: Survival of pediatric marrow transplant patients 1998-2006
View larger version    Download slide (PPT)

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.

Figure 2.
Inherited metabolic disorders: Survival of pediatric marrow myeloablative transplant patients 1998-2006. (NMDP data)

Inherited metabolic disorders: survival of pediatric marrow transplant patients 1998-2006

View larger version   Download slide (PPT)

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


Subscribe to E-News
Quick Reference: Referral Guidelines and Post-Transplant Guidelines