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Cord Blood Stem Cells

Unrelated umbilical cord blood transplantation in children with immune deficiency: results of a multicenter study

Bone Marrow Transplantation volume 45, pages 283–288 (2010)Cite this article

Abstract

In the absence of a related donor, unrelated cord blood transplant (CBT) may be a potential option for patients with a primary immune deficiency (PID). Most published experience consists of single-center data using multiple preparative regimens and GVHD prophylaxis. We report the results of a multicenter prospective trial of unrelated CBT for PID. A total of 24 children with PID, with a median age of 1 year (range: 0.23–7.81 years) and a median weight of 10.5 kg (range: 4–24.4 kg) received unrelated CBT between 1999 and 2003. All patients received a fully ablative conditioning regimen with identical GVHD prophylaxis and supportive care. Most patients (79%) received a 1 or 2 HLA Ag-mismatched cord unit with a median nucleated cell infused of 9.3 × 107/kg (range: 1.0–31.2) and a median CD34 of 2.7 × 105/kg 2.9 (range: 0.6–84.5). The cumulative incidence of neutrophil engraftment by day 42 was 58% (95% CI: 38–79%) at a median of 19 days. Cumulative incidence estimates of grade III–IV acute GVHD at day 100 and chronic GVHD at 1 year were 29% (95% CI: 10–48%) and 24% (95% CI: 3–44%), respectively. The probability of survival at 180 days and 1 year was 66.7% (95% CI: 44.3–81.7%) and 62.5% (95% CI: 40.3–78.4%), respectively. Unrelated CBT should be considered in children with PID.

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Acknowledgements

The Cord Blood Transplantation Study (COBLT) was conducted and supported by NHLBI in collaboration with the COBLT Study Investigators. This paper was prepared using a limited access dataset obtained from NHLBI and does not necessarily reflect the opinions or views of COBLT or NHLBI. This study was supported in part by Vanderbilt CTSA Grant UL1 RR024975 from NCRR/NIH.

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Authors and Affiliations

  1. Pediatric Stem Cell Transplant Program, Vanderbilt University, Nashville, TN, USA

    H Frangoul, B Manes, C Calder & J Domm

  2. Department of Biostatistics, Vanderbilt University, Nashville, TN, USA

    L Wang & F E Harrell Jr

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  1. H Frangoul
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  2. L Wang
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  3. F E Harrell Jr
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Correspondence to H Frangoul.

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Frangoul, H., Wang, L., Harrell, F. et al. Unrelated umbilical cord blood transplantation in children with immune deficiency: results of a multicenter study. Bone Marrow Transplant 45, 283–288 (2010). https://doi.org/10.1038/bmt.2009.137

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