Outcomes after Unrelated Umbilical Cord Blood Transplantation for Children with Osteopetrosis

Robert Chiesa, Annalisa Ruggeri, Annalisa Paviglianiti, Marco Zecca, Marta Gónzalez-Vicent, Victoria Bordon, Jerry Stein, Sarah Lawson, Sophie Dupont, Edoardo Lanino, Manuel Abecasis, Amal Al-Seraihy, Chantal Kenzey, Marc Bierings, Franco Locatelli, Eliane Gluckman, Ansgar Schulz, Andrew Gennery, Kristin Page, Joanne KurtzbergVanderson Rocha

Risultato della ricerca: Contributo in rivistaArticolo in rivista


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.
Lingua originaleEnglish
pagine (da-a)1997-2002
Numero di pagine6
RivistaBiology of Blood and Marrow Transplantation
Stato di pubblicazionePubblicato - 2016


  • Osteopetrosis
  • Stem cell transplantation
  • Umbilical cord blood transplantation


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