Table 6.
Experiment studies: decrease ferritin, decrease morbidities.
| Study | Population | Study Design | Complications | ||
|---|---|---|---|---|---|
| Chan JC, 2006 [67] | HbH, ferritin > 900 μg/L Control, HbH, ferritin < 900 μg/L; No chelation except one > 6 m ago |
Age-matched cohort study DFO for 18 months |
17 study cases with 16 control (1 case excluded due to intolerance) | Ferritin < 397 μg/L in two F/U or after 18 months, stop DFO 1. MRI for liver: an improvement in T2-signal intensity ratio 12/16 patients 2. Diastolic dysfunction (E/A ratio) no change throughout the period |
None transfused |
| The OPTIMAL CARE study, 2010 [16] | TI, Hb> 7 g/dL at age 2 6 centers (Lebanon, Italy, Egypt) Transfusion history: 1. Every 1–3 m, regularly transfused 2. Occasionally transfused (severe anemia secondary to infection, surgery, or pregnancy) 3. Non-transfused |
Retrospective cohort | 584 TI, transfusion None (139) Occasional (143) Regular (302) |
Morbidity p value Ferritin ≥ 1000 μg/L iron chelation EMH 0.85 NA PHT NA 0.53 * HF NA 0.45 Thrombosis 1.86* 0.97 Cholelithiasis NA 0.30 * Abnormal liver function 1.74 * NA Leg ulcers 1.29 0.68 DM NA 0.4, Hypothyroidism NA 0.49 Osteoporosis 1.6 0.4 * Hypogonadism 2.63 * 2.51 * |
Iron chelation (+) therapy ≥ 1 year Some morbidities adjusted for age > 35 years old |
| Musallam KM, 2012 [66] | Transfusion-independent β-TI, exclude HCV Patients with at least two elastographies during the study period (Lebanon) |
Retrospective cohort | 42 patients, F/U 4 years, median age 38 years | Transient elastography values for fibrosis 2 patients improve fibrosis stage Significant change in serum ferritin, elastography value |
33.3% iron chelation throughout the study period |
| Krittapoom Akrawinthawong, 2011 [68] | β-TI/HbE age 18–50 years, ferritin > 1000 ng/mL Transfused while Hb< 6 g/dL Exclude kidney, liver, heart disease history, BT > 3500 mL/year and ferritin > 10,000 ng/mL DFP for 1 year |
Prospective cohort | 30 patients unable to use deferoxamine 1 anaphylaxis shock |
↓ Ferritin (p = 0.005) and pulmonary hypertension (p = 0.021) ↓ Oxidative stress markers (p < 0.01) |
|
| Ersi Voskaridou, 2009 [69] | ≤20 RBC unites in their lifetime Liver or cardiac iron overload (serum ferritin ≥ 1000 μg/L, liver MRI T2 <25 ms or cardiac T2 < 28 ms) Exclude pregnant, hepatic failure (transaminase > 500 U/l) or renal failure (eGFR < 60 mL/min) or LVEF< 50% |
11 TI deferasirox for 12 months (one patient Hb 12.3 g/dL) (TI, not separate α- and β- thalassemia) Single-arm prospective |
Liver MRI T2 * improved, GOT, GPT decreased Serum ferritin reduced Mean cardiac T2 and LVEF not change significantly |
* clinical significant; ↓, decrease; EMH, extramedullary hematopoiesis; PHT, pulmonary hypertension; HF, heart failure; DM, diabetes mellitus; DFO, deferoxamine.