Skip to main content

Late Effects after Umbilical Cord Blood Transplantation in Very Young Children after Busulfan-Based, Myeloablative Conditioning.

Publication ,  Journal Article
Allewelt, H; El-Khorazaty, J; Mendizabal, A; Taskindoust, M; Martin, PL; Prasad, V; Page, K; Sanders, J; Kurtzberg, J
Published in: Biol Blood Marrow Transplant
September 2016

Infants and young children who undergo allogeneic cord blood transplantation (CBT) are at increased risk for late effects because of exposure of developing organs to chemotherapy and radiation therapy typically used in transplant conditioning regimens. Busulfan (Bu)-based myeloablative regimens were developed to eliminate radiation exposure in these young children with the hope that late effects would be minimized. We now describe the late effects in 102 consecutive patients surviving a minimum of 5 years (median follow-up, 12.9 years) post-CBT. Patients were conditioned with high-dose chemotherapy using Bu-containing regimens. No patient received total body irradiation. The median age at transplant was 1 year (range, .1 to 2). Diagnoses included inherited metabolic diseases (59.8%), leukemia (17.6%), congenital immune deficiency (20.2%), bone marrow failure/myelodysplastic syndrome (3.9%), and hemoglobinopathy (2%). Among patients surviving 5 years, the overall survival rate at 10 years post-CBT was 93% (95% CI, 84.9 to 96.8). Virtually all patients (98%) experienced at least 1 significant late effect. Most (83.3%) experienced 2 or more late effects, and more than half of the patients (64.7%) experienced 3 or more late effects. The most commonly observed late effects included dental problems (92.2%), short stature (55.9%), cognitive deficits (53.6%), pulmonary dysfunction (18.6%), and abnormal pubertal development (27.9%). This is the first report of late effects of Bu-based conditioning in a cohort of very young patients at the time of transplant. These results will inform clinical care guidelines for long-term follow-up and add to the growing information regarding outcomes of hematopoietic stem cell transplantation.

Duke Scholars

Altmetric Attention Stats
Dimensions Citation Stats

Published In

Biol Blood Marrow Transplant

DOI

EISSN

1523-6536

Publication Date

September 2016

Volume

22

Issue

9

Start / End Page

1627 / 1635

Location

United States

Related Subject Headings

  • Transplantation Conditioning
  • Survival Analysis
  • Myeloablative Agonists
  • Long Term Adverse Effects
  • Infant
  • Immunology
  • Humans
  • Follow-Up Studies
  • Drug-Related Side Effects and Adverse Reactions
  • Cord Blood Stem Cell Transplantation
 

Citation

APA
Chicago
ICMJE
MLA
NLM
Allewelt, H., El-Khorazaty, J., Mendizabal, A., Taskindoust, M., Martin, P. L., Prasad, V., … Kurtzberg, J. (2016). Late Effects after Umbilical Cord Blood Transplantation in Very Young Children after Busulfan-Based, Myeloablative Conditioning. Biol Blood Marrow Transplant, 22(9), 1627–1635. https://doi.org/10.1016/j.bbmt.2016.05.024
Allewelt, Heather, Jill El-Khorazaty, Adam Mendizabal, Mahsa Taskindoust, Paul L. Martin, Vinod Prasad, Kristin Page, Jean Sanders, and Joanne Kurtzberg. “Late Effects after Umbilical Cord Blood Transplantation in Very Young Children after Busulfan-Based, Myeloablative Conditioning.Biol Blood Marrow Transplant 22, no. 9 (September 2016): 1627–35. https://doi.org/10.1016/j.bbmt.2016.05.024.
Allewelt H, El-Khorazaty J, Mendizabal A, Taskindoust M, Martin PL, Prasad V, et al. Late Effects after Umbilical Cord Blood Transplantation in Very Young Children after Busulfan-Based, Myeloablative Conditioning. Biol Blood Marrow Transplant. 2016 Sep;22(9):1627–35.
Allewelt, Heather, et al. “Late Effects after Umbilical Cord Blood Transplantation in Very Young Children after Busulfan-Based, Myeloablative Conditioning.Biol Blood Marrow Transplant, vol. 22, no. 9, Sept. 2016, pp. 1627–35. Pubmed, doi:10.1016/j.bbmt.2016.05.024.
Allewelt H, El-Khorazaty J, Mendizabal A, Taskindoust M, Martin PL, Prasad V, Page K, Sanders J, Kurtzberg J. Late Effects after Umbilical Cord Blood Transplantation in Very Young Children after Busulfan-Based, Myeloablative Conditioning. Biol Blood Marrow Transplant. 2016 Sep;22(9):1627–1635.

Published In

Biol Blood Marrow Transplant

DOI

EISSN

1523-6536

Publication Date

September 2016

Volume

22

Issue

9

Start / End Page

1627 / 1635

Location

United States

Related Subject Headings

  • Transplantation Conditioning
  • Survival Analysis
  • Myeloablative Agonists
  • Long Term Adverse Effects
  • Infant
  • Immunology
  • Humans
  • Follow-Up Studies
  • Drug-Related Side Effects and Adverse Reactions
  • Cord Blood Stem Cell Transplantation