Publikationsdatum:
2006-11-16
Beschreibung:
Introduction: The safety and tolerability profile of the oral iron chelator deferasirox has been established in five comprehensive trials (with a 1-year core phase) in adults and children with a range of transfusion-dependent anemias. Following completion of the core phase, pts entered an extension phase that will last for 4 years. This analysis presents cumulative long-term safety data during deferasirox treatment. Methods: Safety was assessed monthly, primarily by evaluating the incidence and type of adverse events (AEs) and measuring laboratory parameters. Results: A total of 1033 pts have received deferasirox: 703 initially received it during the core phases (deferasirox cohort) and 330, who initially received deferoxamine (DFO), crossed over to deferasirox in the extension phases (crossover cohort); 433 (41.9%) were pediatric pts aged 2-4% overall) drug-related AEs during deferasirox treatment (median 1.5–2.5 years; n=1033) AE Frequency, n (% of all pts) Severity, n (% of all pts) Mild Moderate Severe Nausea 99 (9.6) 85 (8.2) 12 (1.2) 2 (0.2) Diarrhea 91 (8.8) 71 (6.9) 18 (1.7) 2 (0.2) Abd pain 52 (5.0) 37 (3.6) 11 (1.1) 4 (0.4) Vomiting 49 (4.7) 40 (3.9) 8 (0.8) 1 (0.1) Rash 49 (4.7) 29 (2.8) 16 (1.5) 4 (0.4) Upper abd pain 44 (4.3) 34 (3.3) 9 (0.9) 1 (0.1) During the extension phases there were no changes in markers of liver or renal function that were consistently or significantly different from the core study. Ten pts (1.0%) had urinary total protein/creatinine ratio 〉1.0 at 2 consecutive visits. There have been no cases of progressive increases in serum creatinine, indicative of renal insufficiency or failure. Physical and sexual development proceeded normally in all pediatric pts. Conclusions: The safety and tolerability of deferasirox in pts receiving up to 2.5 years of treatment was similar to that observed during the 1-year core trials, with most AEs being mild, transient and easily manageable. These data demonstrate that long-term treatment with deferasirox is generally well tolerated in adults and children with a variety of transfusion-dependent anemias.
Print ISSN:
0006-4971
Digitale ISSN:
1528-0020
Thema:
Biologie
,
Medizin
Permalink