Mismatched related hematopoietic stem cell transplantation

الملخص EN

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 × 106 / kg recipient weight.

Patients received different conditioning protocols.

All patients received anti-graft versus host disease (GVHD) prophylaxis and all were engrafted.

Mixed chimeras (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 × 106 / 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.