[Successful treatment of agammaglobulinemia by HLA-mismatched unrelated cord blood stem cell transplantation--the first case report].
OBJECTIVE
To evaluate cord blood stem cell transplantation (CBT) in the treatment of X-linked agammaglobulinemia, and observe the courses of the hematopoietic and immune reconstitution.


METHODS
A 14-year-old male patient with agammaglobulinemia received CBT from a 1/6 HLA-mismatched unrelated cord blood. The conditioning regimen was Bu/Cy/anti-CD3 antibody. CsA was given together with MMF and MTX for prophylaxis of GVHD. The patient received 0.42 x 10(8) nucleated cells/kg, containing 0.35 x 10(6) CD34(+) cells/kg.


RESULTS
The recipient showed hematopoietic reconstitution on day 30 post-transplantation when ANC was 0.5 x 10(9)/L and BPC 20 x 10(9)/L. Sex chromosome analysis showed engraftment (donor 46, XX/recipient 46, XY = 4:1) on day 45. The recipient's blood group changed from AB to O, IgG from 1.1 g/L to 3.5 g/L, sex chromosome from 46, XY to full 46, XX, and mature B cells in peripheral blood from 0 to 5% on day 100, indicating immune reconstitution. At the last follow-up of 360 days, the patient without acute or chronic GVHD showed normal hemogram and myelogram, IgG 13.5 g/L and 10% mature B cells in peripheral blood, indicating the hematopoiesis and immune persistent reconstitution. No acute or chronic GVHD was developed.


CONCLUSIONS
This is the first case report of successful treatment of X-linked agammaglobulinemia by HLA-mismatched unrelated CBT.
Wan
Ding-ming
DM
Department of Hematology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou 450052, China.
Liu
Chang-feng
CF
Wang
Gui-ju
GJ
Sun
Hui
H
Sun
Ling
L
Jiang
Zhong-xing
ZX
Guo
Wan-li
WL
Qin
Ling
L
Liu
Shao-jun
SJ
D000293:Adolescent; D000361:Agammaglobulinemia / Q000601:surgery*; D036101:Cord Blood Stem Cell Transplantation / Q000379:methods*; D006086:Graft vs Host Disease / Q000517:prevention & control; D006680:HLA Antigens / Q000276:immunology; D006801:Humans; D008297:Male; D019172:Transplantation Conditioning; D016896:Treatment Outcome
D006680:HLA Antigens