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

Paul L. Martin; Shelly L. Carter; Nancy A. Kernan; Indira Sahdev; Donna Wall; Daniel Pietryga; John E. Wagner; Joanne Kurtzberg

doi:10.1016/j.bbmt.2005.09.016

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

Paul L. Martin, Shelly L. Carter, Nancy A. Kernan, Indira Sahdev, Donna Wall, Daniel Pietryga, John E. Wagner, Joanne Kurtzberg

Research output: Contribution to journal › Article › peer-review

173 Scopus citations

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 × 10⁷/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.

Original language	English (US)
Pages (from-to)	184-194
Number of pages	11
Journal	Biology of Blood and Marrow Transplantation
Volume	12
Issue number	2
DOIs	https://doi.org/10.1016/j.bbmt.2005.09.016
State	Published - Feb 2006

Bibliographical note

Funding Information:
We greatly appreciate the dedication and outstanding care delivered to these patients by their nurses, nurse practitioners, nurse coordinators, social workers, physical, speech and occupational therapists, and other allied health care professionals involved in their care. We are indebted to Angela Norman for assistance in the preparation of the manuscript. This work was supported by a contract from the National Heart, Lung and Blood Institute (grant nos. N01-HB-67138 [P.L.M. and J.K.], N01-HB-67132 [S.L.C., N.A.K., I.S., D.W., and D.P.], and N01-HB 67139 [J.E.W.]).

Keywords

Cord blood transplantation
Inborn errors of metabolism
Lysosomal storage diseases
Peroxisomal storage diseases

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10.1016/j.bbmt.2005.09.016

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Martin, P. L., Carter, S. L., Kernan, N. A., Sahdev, I., Wall, D., Pietryga, D., Wagner, J. E., & Kurtzberg, J. (2006). 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. Biology of Blood and Marrow Transplantation, 12(2), 184-194. https://doi.org/10.1016/j.bbmt.2005.09.016

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. / Martin, Paul L.; Carter, Shelly L.; Kernan, Nancy A. et al.

In: Biology of Blood and Marrow Transplantation, Vol. 12, No. 2, 02.2006, p. 184-194.

Research output: Contribution to journal › Article › peer-review

Martin, PL, Carter, SL, Kernan, NA, Sahdev, I, Wall, D, Pietryga, D, Wagner, JE & Kurtzberg, J 2006, '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', Biology of Blood and Marrow Transplantation, vol. 12, no. 2, pp. 184-194. https://doi.org/10.1016/j.bbmt.2005.09.016

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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 × 107/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.",

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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

Bibliographical note

Keywords

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