Patient characteristics
Table 1 Patient and transplant characteristics describes details of 183
children undergoing first allogeneic HCT for NMD in Florida during
2010-2019, who had data on GVHD reported to CIBMTR. The most common
diagnoses included sickle cell disease (24.0%), severe aplastic anemia
(21.9%), primary immunodeficiencies including severe combined
immunodeficiency (24.1%), thalassemia (11.5%) and bone-marrow failure
syndromes (8.2%). Median age of children was 7 years (range 0-21) with
even distribution among <3 years, 3-9 years, and ≥10 years of
age groups. Graft types included bone marrow (BM), umbilical cord blood
(UCB), and peripheral blood (PB) used in 69.9%, 18.6%, and 9.8% of
patients, respectively. Donors were related in 53.6% of cases and
unrelated in the rest. Fully HLA-matched donors were used in 65.6% of
HCT, mismatched in 31.1%, and for 3.3% of patients the degree of match
was unknown. Conditioning regimens were myeloablative in 55.2% of
patients, and 4.4% of patients did not receive any conditioning, while
all other patients received non-myeloablative or reduced intensity
regimens. Low dose (200cGy) total body irradiation (TBI) was used in
7.6% of HCT recipients. Anti-thymocyte globulin was used in
conditioning of 55.2% of patients and alemtuzumab in 34.4%. Rituximab
was used as part of GVHD prophylaxis by one center for a majority of
their transplant recipients, and thus 23.0% of patients received a
single dose of rituximab during conditioning.