Total patients | 27 |
Sex (%) | Â |
 Male | 18 (66.7) |
 Female | 9 (33.3) |
Age at transplant (median) | 12 months (range: 4–107 months) |
Gene (%) | Â |
 TCIRG1 compound heterozygous mutation | 18 (66.7) |
 CLCN7 compound heterozygous mutation | 2 (7.4) |
 CLCN7 heterozygous mutation | 4 (14.8) |
Visual impairment | 26/27 |
Hearing impairment | 5/27 |
Splenomegaly | 23/27 |
Abnormal haemogram | 27/27 |