Results of the Cord Blood Transplantation Study (COBLT): Outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases Journal Article
| Authors: | Martin, P. L.; Carter, S. L.; Kernan, N. A.; Sahdev, I.; Wall, D.; Pietryga, D.; Wagner, J. E.; Kurtzberg, J. |
| Article Title: | Results of the Cord Blood Transplantation Study (COBLT): Outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases |
| Abstract: | The Cord Blood Transplantation Study (COBLT), sponsored by the National Heart, Lung, and Blood Institute, is a phase II multicenter study designed to evaluate the use of cord blood in allogeneic transplantation. In this report, we evaluated the outcomes of cord blood transplantation in 69 patients with lysosomal and peroxisomal storage diseases. Patients with mucopolysaccharidoses I to III, mucolipidoses (ML) II (n = 36), adrenoleukodystrophy (n = 8), metachromatic leukodystrophy (n = 6), Krabbe disease (n = 16), and Tay-Sachs disease (n = 3) were enrolled between August 1999 and June 2004. All patients received the same preparative regimen, graft-versus-host disease (GVHD) prophylaxis, and supportive care. End points included survival, engraftment, GVHD, and toxicity. Sixty-nine patients (64% men; 81% white) with a median age of 1.8 years underwent transplantation with a median cell dose of 8.7 × 10 7/kg. One-year survival was 72% (95% confidence interval, 61%-83%). The cumulative incidence of neutrophil engraftment by day 42 was 78% (95% confidence interval, 67%-87%) at a median of 25 days. Grade II to IV acute GVHD occurred in 36% of patients. Cord blood donors are readily available for rapid transplantation. Cord blood transplantation should be considered as frontline therapy for young patients with lysosomal and peroxisomal storage diseases. © 2006 American Society for Blood and Marrow Transplantation. |
| Keywords: | adolescent; child; preschool child; school child; child, preschool; disease-free survival; antibiotic agent; graft infection; survival rate; retrospective studies; transplantation, homologous; major clinical study; busulfan; allogeneic stem cell transplantation; clinical trial; drug megadose; neutrophil count; phase 2 clinical trial; cyclophosphamide; drug hypersensitivity; survival time; cord blood stem cell transplantation; hla matching; myeloablative conditioning; infant; paracetamol; multicenter study; graft versus host reaction; transplantation conditioning; heparin; methylprednisolone; seizure; mesna; corticosteroid; recombinant granulocyte colony stimulating factor; tacrolimus; graft vs host disease; hemorrhagic cystitis; cyclosporin; phenytoin; thymocyte antibody; lysosome storage disease; diphenhydramine; adrenoleukodystrophy; hurler syndrome; vein occlusion; daclizumab; cord blood transplantation; inborn errors of metabolism; lysosomal storage diseases; peroxisomal storage diseases; disorders of peroxisomal functions; globoid cell leukodystrophy; metachromatic leukodystrophy; mucolipidosis type 2; sanfilippo syndrome; tay sachs disease |
| Journal Title: | Biology of Blood and Marrow Transplantation |
| Volume: | 12 |
| Issue: | 2 |
| ISSN: | 1083-8791 |
| Publisher: | Elsevier Inc. |
| Date Published: | 2006-02-01 |
| Start Page: | 184 |
| End Page: | 194 |
| Language: | English |
| DOI: | 10.1016/j.bbmt.2005.09.016 |
| PUBMED: | 16443516 |
| PROVIDER: | scopus |
| DOI/URL: | |
| Notes: | --- - "Cited By (since 1996): 79" - "Export Date: 4 June 2012" - "CODEN: BBMTF" - "Source: Scopus" |
