Reversal of Complications Following Intensive Combined Chelation in β-Thalassemia Major Patients

• Published in: Blood 2007-11, Vol.110 (11), p.LB4-LB4
• Main Authors: Farmaki, Kallistheni, Tzoumari, Ioanna, Pappa, Christina, Tompoukis, Christos, Papagianni, Aggeliki, Aggelopoulos, Nikos, Mitas, Demetrios, Berdoukas, Vasilios
• Format: Article
• Language: English
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood.V110.11.LB4.lb4

Life-threatening sequelae in β-thalassemia major patients (TM), result from transfusion iron overload (Fe load). Combined chelation with desferrioxamine (DFO) and deferiprone (DFP) is well tolerated and produces a synergistic effect superior to either drug alone. Combination can place all TMp in negative net iron balance and effect significant reduction in Fe load. 50 TM (24 males, 26 females) aged 8–48 yrs, were switched from monotherapy with DFO, to an individually tailored regimen (DFO 40–60 mg/kg/day- DFP 75–100 mg/kg/day) for 5–6yrs. Fe load was evaluated by mean ferritin levels (MEIA) and non-invasive heart &hepatic iron quantification, by annual Signa-MRI 1.5 Tesla, multi-echo T2 sequences. The heart was evaluated by Echo Doppler. Endocrine function was assessed by:- thyroid: FT4, FT3 &TSH in TRH-test, - Gonads: estradiol, progesterone, testosterone, Free-testosterone levels &LHRH stimulation- Glucose tolerance: OGGT with glucose &insulin measurements at each time and area under the curve (AUC). Insulin sensitivity and beta-cell function were assessed by indices of homeostasis model assessment (ISIHOMA &SCHOMA). Subsequently, all patients survived, even though with DFO alone in the previous decade, mortality ranged from 13.3–14.3%. In patients who accepted the treatment well, a trend analysis (PROC MIXED in SAS), revealed a negative trend of ferritin over time (p