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Outcomes after Unrelated Umbilical Cord Blood Transplantation for Children with Osteopetrosis.

Authors :
Chiesa R
Ruggeri A
Paviglianiti A
Zecca M
Gónzalez-Vicent M
Bordon V
Stein J
Lawson S
Dupont S
Lanino E
Abecasis M
Al-Seraihy A
Kenzey C
Bierings M
Locatelli F
Gluckman E
Schulz A
Gennery A
Page K
Kurtzberg J
Rocha V
Source :
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation [Biol Blood Marrow Transplant] 2016 Nov; Vol. 22 (11), pp. 1997-2002. Date of Electronic Publication: 2016 Jul 25.
Publication Year :
2016

Abstract

Hematopoietic stem cell transplantation (HSCT) is the only curative treatment for most children with osteopetrosis (OP). Timing of HSCT is critical; therefore, umbilical cord blood transplantation (UCBT) is an attractive option. We analyzed outcomes after UCBT in 51 OP children. Median age at UCBT was 6 months. Seventy-seven percent of the cord blood grafts had 0 or 1 HLA disparity with the recipient. Conditioning regimen was myeloablative (mostly busulfan-based in 84% and treosulfan-based in 10%). Antithymocyte globulin was given to 90% of patients. Median number of total nucleated and CD34 <superscript>+</superscript> cells infused was 14 × 10 <superscript>7</superscript> /kg and 3.4 × 10 <superscript>5</superscript> /kg, respectively. Median follow-up for survivors was 74 months. Cumulative incidence (CI) of neutrophil recovery was 67% with a median time to recovery of 23 days; 33% of patients had graft failure, 81% of engrafted patients had full donor engraftment, and 19% had mixed donor chimerism. Day 100 CI of acute graft-versus-host disease (grades II to IV) was 31% and 6-year CI of chronic graft-versus-host disease was 21%. Mechanical ventilation was required in 28%, and veno-occlusive disease was diagnosed in 16% of cases. Six-year overall survival rate was 46%. Comparative studies with other alternative donors should be performed to evaluate whether UCBT remains a valid alternative for children with OP without an HLA-matched donor.<br /> (Copyright © 2016 The American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.)

Details

Language :
English
ISSN :
1523-6536
Volume :
22
Issue :
11
Database :
MEDLINE
Journal :
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Publication Type :
Academic Journal
Accession number :
27470286
Full Text :
https://doi.org/10.1016/j.bbmt.2016.07.015