Table 5.
Study | Study Subject | N, Study Design | Morbidity Ferritin Level (Patient Number)/Patient Number (%) | Note: |
---|---|---|---|---|
THALASSA trial, 2012 [52] THALASSA trial, 1 year extension 2013 [53] Ali T Taher et al., 2014 [54]; Part of THALASSA trial |
NTDT ≥ 10 years and LIC ≥ 5 mg Fe/g dw (MRI) and Ferritin > 300 ng/mL in 2 consecutive values ≥ 14 days; 1 year result No transfusion within 6 months or chelation therapy within 1 month Exclude HBV, HCV carrier NTDT age ≥ 10 years LIC ≥ 5 mg Fe/g dw and Ferritin > 300 μg/mL Deferasirox-naïve, no other chelation therapy within 1 month of study entry Deferasirox vs. placebo 1 year and then 1 year extension placebo switched to deferasirox |
166 patients Phase 2, prospective, randomized, double-blind, placebo-controlled trial 130 patients 130 patients |
Start LIC ≥ 5 mg Fe/g dw and Ferritin > 300 ng/mL Suspended Ferritin < 100 ng/mL or LIC < 3 mg Fe/g dw ↓ LIC significantly, ↓ serum ferritin level significantly ↓ LIC significantly, ↓ serum ferritin level significantly Trend decreasing in mean ALT Trend increase serum creatinine and decrease creatinine clearance; increase urinary protein/creatinine ratio initially and then stable Ferritin 800 ≥ μg/L predict LIC ≥ 5 mg Interruption (ferritin <300 μg/L) Dose escalation (ferritin > 2000 μg/L) |
|
Pootrakul P et al., 2003 [55] | Deferiprone 1 year in Thailand Transfused while Hb < 5 g/dL |
7 β-TI/HbE, 2 β-TI |
Significant fall in liver iron, serum ferritin, red cell membrane iron and serum non-transferrin bound iron | 3 transfused 6 not transfused |
Vassilis Ladis et al., 2010 [56] | Ferritin 500 ≥ µg/L and LIC ≥ 2 mg Fe/g dw (MRI) NTDT, some <20 pRBC Exclude renal, liver function impairment or a life expectancy <2 years |
11 TI patients Deferasirox discontinue ferritin < 250 µg/L and LIC < 2 mg Fe/g dw |
1 patient excluded for 6 months of iron chelation therapy 1 patient 12 months LIC decrease, p = 0.044 LVEF no change |