Abstract:
:To date, there is a lack of long-term safety and efficacy data for iron chelation therapy in transfusion-dependent patients with sickle cell disease (SCD). To evaluate the long-term safety and efficacy of deferasirox (a once-daily oral iron chelator), patients with SCD completing a 1-year, Phase II, randomized, deferoxamine (DFO)-controlled study entered a 4-year extension, continuing to receive deferasirox, or switching from DFO to deferasirox. Average actual deferasirox dose was 19·4 ± 6·3 mg/kg per d. Of 185 patients who received at least one deferasirox dose, 33·5% completed the 5-year study. The most common reasons for discontinuation were withdrawal of consent (23·8%), lost to follow-up (9·2%) and adverse events (AEs) (7·6%). Investigator-assessed drug-related AEs were predominantly gastrointestinal [including nausea (14·6%), diarrhoea (10·8%)], mild-to-moderate and transient in nature. Creatinine clearance remained within the normal range throughout the study. Despite conservative initial dosing, serum ferritin levels in patients with ≥ 4 years deferasirox exposure significantly decreased by -591 μg/l (95% confidence intervals, -1411, -280 μg/l; P = 0·027; n = 67). Long-term deferasirox treatment for up to 5 years had a clinically acceptable safety profile, including maintenance of normal renal function, in patients with SCD. Iron burden was substantially reduced with appropriate dosing in patients treated for at least 4 years.
journal_name
Br J Haematoljournal_title
British journal of haematologyauthors
Vichinsky E,Bernaudin F,Forni GL,Gardner R,Hassell K,Heeney MM,Inusa B,Kutlar A,Lane P,Mathias L,Porter J,Tebbi C,Wilson F,Griffel L,Deng W,Giannone V,Coates Tdoi
10.1111/j.1365-2141.2011.08720.xsubject
Has Abstractpub_date
2011-08-01 00:00:00pages
387-97issue
3eissn
0007-1048issn
1365-2141journal_volume
154pub_type
杂志文章,多中心研究,随机对照试验abstract::The spleen acts as a blood filter and lymphopoietic organ. Asplenic and hyposplenic individuals are more susceptible to serious infections caused by encapsulated bacteria but they can be protected by antibiotic prophylaxis and immunizations. Recent progress in vaccinology means prophylaxis is now successful in the vas...
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