| dc.description.abstract | Background: We aimed to evaluate hematopoietic stem cell transplantation
(HSCT) related outcomes of patients with severe combined
immunodeficiency (SCID).
Methods: We retrospectively collected data from SCID patients who
were diagnosed, followed up and survived at least 2 years after HSCT.
Results: Forty four SCID patients were included in the study. Median
age of HSCT and follow-up period after HSCT were 7.1 months and
8.7 years, respectively. Human leukocyte antigen (HLA) identical
donors were used in 77.3% (n = 34) of the patients (23 siblings, six
fathers, two mothers, three extended family donors), HLA 1–2 mismatched
family donors in 11.3% (n = 5), and haploidentical family
donors in 11.3% (n = 5). CD3 and CD19 counts were normal in more
than 90% and in 45.4% at last follow-up, respectively. Intravenous
immunoglobulin (IVIG) could be stopped in 72.7% (n = 32) after
HSCT. B+ SCID patients had better CD19 counts than B- (p < .001).
T cell numbers, lymphocyte proliferation, IVIG need, immunoglobulin
levels, antibody responses did not differ among B- and B+ immunophenotypes.
Acute graft-versus-host disease (GVHD) was less in bone
marrow transplanted patients (19.4%) than peripheral stem cell
(58.3%) transplanted ones (p = .024). There was no correlation
between age at transplantation and immune reconstitution. At the
last follow-up, 70.2% and 78.3% of the patients had body weight and
height above 3rd percentile, respectively.
Conclusion: The immune reconstitution and the growth were normal
in the majority of SCID patients after HSCT. It may be rational to use
bone marrow instead of peripheral stem cell, as acute GVHD was less
in bone marrow transplanted patients. | tr_TR |
