Single-institution Experience of Unrelated Cord Blood Transplantation for Primary Immunodeficiency.

[wiskott-aldrich syndrome]

Pediatric patients with primary immunodeficiencies (PID ) constitute life-threatening medical emergencies . In the absence of an HLA-identical hematopoietic stem cell donor , unrelated donor cord blood transplantation (CBT ) is another treatment option . There are little data regarding the outcome of unrelated CBT for PID in Taiwan . We report the results of CBT performed in 8 patients with PID between 2004 and 2013 at Chang Gung Memorial Hospital . The cases included severe combined immunodeficiency (n =4 ), chronic granulomatous disease (n =2 ), Wiskott-Aldrich syndrome (n =1 ), and T -cell immunodeficiency (n =1 ). Median follow-up time was 73 months . Most UCB recipients received a myeloablative conditioning regimen . There were 7 boys and 1 girl with a median age of 2 .5 months at diagnosis (range , antenatal to 17 mo ). Median age at transplant was 5 .5 months (range , 2 to 74 mo ). All but 1 patients engrafted at a median time of 14 days . One developed significant grade III graft-versus-host disease after transplant . Our data show that unrelated CBT in PID is possible . However , no definite conclusions can be drawn from this small number of patients , and more studies are needed to further investigate and confirm these findings .

Diseases
Validation

Diseases presenting "severe combined immunodeficiency" symptom

achondroplasia

alpha-thalassemia

child syndrome

cholangiocarcinoma

junctional epidermolysis bullosa

krabbe disease

omenn syndrome

severe combined immunodeficiency

wiskott-aldrich syndrome

This symptom has already been validated