title: HLA-haploidentical transplantations for primary immunodeficiencies: a single-center experience.
abstract: SCID is characterized by profound deficiencies of T and B lymphocytes. HSCT is the only curative treatment for children with SCID. The clinical characteristics and outcome of 30 HLA-haploidentical transplantations in 18 patients (15 SCID, two Omenn syndrome, and one MHC Class II deficiency) are reported here. The age of patients at diagnosis ranged from one and half to nine months (median: four months). The median time was one month between the diagnosis and the time of the initial transplantation. Infused CD34+ stem cell dose was ranged between 7 and 94.2 × 10(6) /kg. Nine of 18 patients were found to be positive for CMV antigenemia at diagnosis; therefore, none of them received a conditioning regimen. The most common complication was graft failure (61%), so repeated transplantations (two to four) were performed in seven patients. The mean time of lymphoid engraftment was 17.5 days (median: 16, range: 11-29 days). Ten of 15 SCID (67%) patients survived with a stable complete donor chimerism. However, all three non-SCID patients died. In conclusion, in the absence of a matched family donor, HLA-haploidentical transplantation from parental donors represents a readily available treatment option especially for patients with SCID, offering a high chance of cure.
authors.affiliation: Department of Pediatric Immunology and Allergy, Ankara University School of Medicine, Ankara, Turkey.
authors.forename: Funda Erol
authors.initials: FE
authors.lastname: Cipe
authors.forename: Figen
authors.initials: F
authors.lastname: Dogu
authors.forename: Caner
authors.initials: C
authors.lastname: Aytekin
authors.forename: Mutlu
authors.initials: M
authors.lastname: Yuksek
authors.forename: Tanil
authors.initials: T
authors.lastname: Kendirli
authors.forename: Alisan
authors.initials: A
authors.lastname: Yildiran
authors.forename: Gunseli
authors.initials: G
authors.lastname: Bozdogan
authors.forename: Deniz
authors.initials: D
authors.lastname: Karatas
authors.forename: Ismail
authors.initials: I
authors.lastname: Reisli
authors.forename: Klara
authors.initials: K
authors.lastname: Dalva
authors.forename: Fikret
authors.initials: F
authors.lastname: Arpacı
authors.forename: Aydan
authors.initials: A
authors.lastname: Ikinciogullari
mesh_terms: D046148:Donor Selection; D005260:Female; D005500:Follow-Up Studies; D006084:Graft Rejection / Q000453:epidemiology; D006680:HLA Antigens / Q000276:immunology*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006648:Histocompatibility; D006801:Humans; D007223:Infant; D008297:Male; D010290:Parents; D011183:Postoperative Complications / Q000453:epidemiology; D012086:Reoperation / Q000706:statistics & numerical data; D016511:Severe Combined Immunodeficiency / Q000276:immunology / Q000401:mortality / Q000601:surgery*; D015996:Survival Rate; D016896:Treatment Outcome
chemical_list: D006680:HLA Antigens