Mismatched related hematopoietic stem cell transplantation in primary immunodeficiency.
Hematopoietic stem cell transplantation (HSCT) is the definitive therapy for a variety of primary immunodeficiency syndromes (PIDs). However, no more than 30% of the patients will have a human leukocyte antigen (HLA)-identical sibling. We retrospectively analyzed our results of ten patients with PID; severe combined immunodeficiency (SCID) (n = 7), hyper IgM (HIgM) (n = 1) and combined immunodeficiency (CID) (n = 2), who lacked a fully matched donor and underwent mismatched related HSCT during the period from 2008 to 2010. The median age at the time of transplantation ranged between 3 and 84 months (median 6.5 months). Peripheral blood stem cells (PBSC) were used in all HSCTs. The mean value of the peripheral CD34+ cells infused was 9.19 × 10 (6) /kg recipient weight. Patients received different conditioning protocols. All patients received anti graft versus host disease (GVHD) prophylaxis and all were engrafted. Mixed chimerism (5-55%) was noticed. GVHD was observed in 50% of the patients. Post-transplant follow-up ranged from 3 weeks to 36 months (median 15 months). Five patients are still alive while one patient developed engraftment syndrome followed by graft slippage for which a second transplant with CD34+ stem cells 5.8 × 10 (6) /kg recipient's weight was infused. The others died from sepsis and transplant-related complications. Immune reconstitution was noticed in four patients. In conclusion, HLA-haploidentical stem cell transplantation may be feasible, with appropriate GVHD prophylaxis, for patients with PID who lack a fully matched donor.
Queen Rania Children Hospital, King Hussein Medical Center, Royal Medical Services, Amman, Jordan.
Adel M
AM
Wahadneh
Haifa A
HA
Bin Dahman
Mohammed E
ME
Abu Shukear
Zeyad M
ZM
Habahbeh
Mohammad A
MA
Ajarmeh
Raed M
RM
Zyood
Mueen S
MS
Habashneh
D002648:Child; D002675:Child, Preschool; D005260:Female; D006086:Graft vs Host Disease / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation; D006648:Histocompatibility / Q000276:immunology*; D006801:Humans; D007223:Infant; D008297:Male; D012189:Retrospective Studies; D016511:Severe Combined Immunodeficiency / Q000601:surgery*; D019172:Transplantation Conditioning