Peripheral blood stem cell transplantation from HLA-mismatched unrelated donor or haploidentical donor for the treatment of X-linked agammaglobulinemia.
- Author:
Ling NIE
1
;
Tao SU
;
Kai-Tai YANG
;
Liang ZHAO
;
Jian HU
;
Shuang-Hui YANG
;
Ya-Jing XU
;
Bin FU
Author Information
1. Department of Hematology, Xiangya Hospital, Central South University, Changsha 410008, China. fu.bin@csu.edu.cn.
- Publication Type:Case Reports
- MeSH:
Agammaglobulinemia;
therapy;
Genetic Diseases, X-Linked;
therapy;
Graft vs Host Disease;
HLA Antigens;
Hematopoietic Stem Cell Transplantation;
Humans;
Peripheral Blood Stem Cell Transplantation;
Treatment Outcome;
Unrelated Donors;
Young Adult
- From:
Chinese Journal of Contemporary Pediatrics
2020;22(8):821-827
- CountryChina
- Language:Chinese
-
Abstract:
Allogeneic stem cell transplantation (allo-SCT) is currently the only curative option for patients with X-linked agammaglobulinemia (XLA). In this study, patient 1 aged 4 years who underwent allogeneic peripheral blood stem cell transplantation (allo-PBSCT) from HLA-mismatched unrelated donor; patient 2 aged 24 years (childhood onset) with primary cutaneous acral CD8 T cell lymphoma who underwent allo-PBSCT from haploidentical relative donor. Both were treated by reduced toxicity myeloablative conditioning with post-transplantation cyclophosphamide (PTCy), anti-thymocyte globulin (ATG), methotrexate (MTX) and cyclosporine (CsA) for graft-versus-host-disease (GVHD) prophylaxis. In patient 1, neutrophil and platelet engraftment were observed on day 11 post-transplantation; the donor chimerism dropped on day 90 post-transplantation, and recovered on day 150 with donor lymphocyte infusion (DLI). In patient 2, neutrophil and platelet engraftment were observed on days 20 and 87 post-transplantation respectively, with complete donor chimerism on day 30 post-transplantation. The serum levels of IgG, IgM and IgA and the percentage of CD19 B cells in peripheral blood of patients 1 and 2 returned to normal within 2 months and more than 1 year after transplantation respectively. There was no evidence of acute GVHD for the two patients. Patient 1 developed a limited type of skin chronic GVHD after DLI, which disappeared after anti-GVHD treatment. This is the first report of successful treatment for two XLA patients using PTCy with allo-PBSCT from HLA-mismatched unrelated donor or haploidentical donor, combining with improved conditioning, which expands the pool of eligible donors for patients with XLA.
