TABLE 1. Participant clinical characteristics
Variables
All participants N = 37 Male N = 15 Female N = 22
P-values
Age, years 14.6 ± 3.2 13.5 ± 3.4 15.3 ± 2.8 0.09
Bone age, years 14.3 ± 3.4 12.7 ± 3.9 15.4 ± 2.5 0.013
Tanner stage Median 1, n (%) 2-3, n (%) 4-5, n (%) 4 (2-5) 5 (14%) 10 (27%) 22 (59%) 2 (1-4) 5 (33%) 5 (33%) 5 (33%) 4 (4-5) 1 (5%) 4 (18%) 17 (77%)
0.004
Height, cm 147.8 ± 12.3 144.5 ± 16.1 150 ± 8.6 0.28
Height z-score -1.2 ± 1.2 -1.5 ± 1.2 -1.0 ± 1.1 0.21
BMI, kg/m2 19 (17-22) 18 (17-19) 21 (19-23) 0.003
BMI z-score 0 (-0.5-0.8) -0.3 (-0.4-0.2) 0.4 (-0.6-0.9) 0.22
α-thalassemia subtypes Hb H-CS, n (%) Hb H-Paksé, n (%) 32 (86%) 5 (14%) 13 (87%) 2 (13%) 19 (86%) 3 (14%)
0.98
Prednisolone therapy, n (%) 6 (16%) 2 (13%) 4 (18%) 0.69
Age at first blood transfusion 4.0 (1.0-8.0) 3.0 (1.0-6.0) 5.5 (1.7-8.8) 0.09
Average pre-transfusion Hb, (g/dL) 8.8 ± 1.0 9.4 ± 1.0 8.4 ± 0.7 0.002
Blood transfusion interval, weeks 4 (4-6) 4 (4-6) 4 (4-5) 0.59
Transfused blood volume, mL/kg/year 103 ± 43 108 ± 51 110 ± 22 0.46
Average serum ferritin, ng/mL 958 ± 513 949 ± 494 964 ± 537 0.98
Liver iron concentration (mg/g dry weight) 6.0 (3.6-8.3) 5.3 (2.3-6.3) 6.3 (4.9-9.8) 0.18
Iron chelation therapy None, n (%) Deferasirox, n (%) Deferiprone, n (%) Desferoxamine, n (%) 11 (30%) 8 (22%) 17 (46%) 1 (3%) 5 (33%) 5 (33%) 5 (33%) 0 6 (27%) 3 (14%) 12 (55%) 1 (5%)
0.23
Endocrine complication Short stature Hypothyroidism Delayed puberty 8 (22%) 3 (8%) 6 (16%) 5 (33%) 0 3 (20%) 3 (14%) 3 (14%) 3 (14%) 0.23 0.19 0.67