Unrelated umbilical cord blood transplantation in children with immune deficiency: results of a multicenter study.
Bone Marrow Transplant
; 45(2): 283-8, 2010 Feb.
Article
em En
| MEDLINE
| ID: mdl-19543326
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 x 10(7)/kg (range 1.0-31.2) and a median CD34 of 2.7 x 10(5)/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.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Transplante de Células-Tronco de Sangue do Cordão Umbilical
/
Síndromes de Imunodeficiência
Tipo de estudo:
Clinical_trials
Limite:
Child
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Child, preschool
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Female
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Humans
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Infant
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Male
Idioma:
En
Revista:
Bone Marrow Transplant
Assunto da revista:
TRANSPLANTE
Ano de publicação:
2010
Tipo de documento:
Article
País de afiliação:
Estados Unidos