Successful allogeneic stem cell transplant for leukocyte adhesion deficiency using an adjusted busulfan-containing regimen.
LAD is a rare and fatal congenital disorder in which there is a defect of the leukocyte adhesion molecule (CD18) on neutrophils. Severe LAD results in frequent and potentially fatal infections. Although allogeneic HSCT is the only curative treatment for severe LAD, strategies for HSCT remain to be established since a high engraftment failure rate and severe persistent infection are still seen. We report a four-month-old boy with LAD who was successfully treated with allogeneic HSCT from an HLA-complete matched sibling following a conditioning regimen of pharmacokinetically adjusted individual busulfan doses.
Yoshimoto
Akira
A
Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan.
Hashii
Yoshiko
Y
Kashiwagi
Hiroko
H
Koizumi
Makoto
M
Tokimasa
Sadao
S
Fujisaki
Hiroyuki
H
Ohta
Hideaki
H
Hanai
Naomi
N
Ozono
Keiichi
K
Hara
Junichi
J
D002066:Busulfan / Q000627:therapeutic use*; D005500:Follow-Up Studies; D006084:Graft Rejection / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D018370:Leukocyte-Adhesion Deficiency Syndrome / Q000601:surgery*; D008297:Male; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous
D007166:Immunosuppressive Agents; D002066:Busulfan