Outcomes after Unrelated Umbilical Cord Blood Transplantation for Children with Osteopetrosis

Robert Chiesa, Annalisa Ruggeri, A. L. 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, E. Gluckman, Ansgar Schulz, Andrew Gennery, Kristin Page, J. KurtzbergVanderson Rocha

Research output: Contribution to journalArticle

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+ 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.

Original languageEnglish
Pages (from-to)1997-2002
Number of pages6
JournalBiology of Blood and Marrow Transplantation
Volume22
Issue number11
DOIs
Publication statusPublished - Nov 1 2016

Keywords

  • Osteopetrosis
  • Stem cell transplantation
  • Umbilical cord blood transplantation

ASJC Scopus subject areas

  • Hematology
  • Transplantation

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    Chiesa, R., Ruggeri, A., Paviglianiti, A. L., 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., ... Rocha, V. (2016). Outcomes after Unrelated Umbilical Cord Blood Transplantation for Children with Osteopetrosis. Biology of Blood and Marrow Transplantation, 22(11), 1997-2002. https://doi.org/10.1016/j.bbmt.2016.07.015