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Outcomes after Unrelated Umbilical Cord Blood Transplantation for Children with Osteopetrosis. Biol Blood Marrow Transplant 2016 Nov;22(11):1997-2002



Pubmed ID




Scopus ID

2-s2.0-84992374778 (requires institutional sign-in at Scopus site)   17 Citations


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+ cells infused was 14 × 107/kg and 3.4 × 105/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.

Author List

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, Eurocord, Inborn Errors Working Party, Cell Therapy, Immunobiology Working Party of the European Group for Blood and Marrow Transplantation


Kristin Page MD, MHS, MEd Associate Professor in the Pediatrics department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Child, Preschool
Cord Blood Stem Cell Transplantation
Graft Survival
Graft vs Host Disease
Infant, Newborn
Recovery of Function
Survival Analysis
Transplantation Conditioning
Treatment Outcome
Unrelated Donors