Summary of patient characteristics, n = 52
Age at investigation | |
Median, y (range) | 18 (2-65) |
Sex, n (%) | |
Female | 32 (62) |
Male | 20 (38) |
Ethnicity, n (%) | |
British Indian | 1 (2) |
British Pakistani | 4 (8) |
Druze | 1 (2) |
East African | 3 (6) |
Fijian Indian | 1 (2) |
Finnish | 23 (44) |
North African | 1 (2) |
Puerto Rican | 1 (2) |
Swedish | 2 (4) |
White (not specified) | 15 (28) |
Initial condition, n (%) | |
Sibling with biallelic ERCC6L2 mutation without diagnosis of BMF, MDS, or AML | 5 (10) |
BMF | 32 (62) |
MDS | 9 (17) |
MDS/AML∗ | 2 (4) |
AML | 3 (6) |
Other HM (ALL) | 1 (2) |
ERCC6L2 genotype, n (%) | |
Homozygous | 39 (75) |
Compound heterozygous | 13 (25) |
Individuals by ERCC6L2 mutation type, n (%) | |
Biallelic frameshift | 29 (55) |
Biallelic nonsense | 5 (10) |
Biallelic splicing | 1 (2) |
Biallelic exon deletion | 4 (8) |
Biallelic missense | 4 (8) |
Mixed | 9 (17) |
Age at investigation | |
Median, y (range) | 18 (2-65) |
Sex, n (%) | |
Female | 32 (62) |
Male | 20 (38) |
Ethnicity, n (%) | |
British Indian | 1 (2) |
British Pakistani | 4 (8) |
Druze | 1 (2) |
East African | 3 (6) |
Fijian Indian | 1 (2) |
Finnish | 23 (44) |
North African | 1 (2) |
Puerto Rican | 1 (2) |
Swedish | 2 (4) |
White (not specified) | 15 (28) |
Initial condition, n (%) | |
Sibling with biallelic ERCC6L2 mutation without diagnosis of BMF, MDS, or AML | 5 (10) |
BMF | 32 (62) |
MDS | 9 (17) |
MDS/AML∗ | 2 (4) |
AML | 3 (6) |
Other HM (ALL) | 1 (2) |
ERCC6L2 genotype, n (%) | |
Homozygous | 39 (75) |
Compound heterozygous | 13 (25) |
Individuals by ERCC6L2 mutation type, n (%) | |
Biallelic frameshift | 29 (55) |
Biallelic nonsense | 5 (10) |
Biallelic splicing | 1 (2) |
Biallelic exon deletion | 4 (8) |
Biallelic missense | 4 (8) |
Mixed | 9 (17) |
Per the ELN2022 classification.21