Deferasirox observational studies

Observational studies The usefulness and clinical indications of iron chelation therapy in patients with myelodysplastic ndromes are still under investigation [6, 7 ]. There are at least two possible mechanisms for deferasirox-induced improvement of erythropoiesis in myelodysplasia. First, iron overload can cause the formation of reactive oxygen species, which may worsen inefficient erythropoiesis, while deferasirox reduces oxidative stress parameters in iron-overloaded patients with myelodysplasia. Secondly, deferasirox is a more potent B cell NF-kB inhibitor than other chelators. 

Observational studies In a retrospective study of dose escalation of deferasirox to above 30 mg/kg/day in 264 heavily iron-loaded patients, gastrointestinal hemorrhage and lenticular opacities developed as suspected adverse reactions in one patient each, but no details were given [16 ]. There was no worsening of renal or hepatic function. 

Side effects were also noted in observational studies of diseases ofher than thalassaemias. A phase II clinical trial studying iatrogenic iron overload in patients (n = 10) undergoing induction/consolidation chemotherapy for increased serum ferritin due to acute myeloid leukaemia was halted due to poor tolerance of deferasirox (20mg/kg/day) due primarily to GI effects [19 ]. Patients had received the therapies for a median duration of 72 days (range 19-130 days). The authors concluded that the use of deferasirox was poorly tolerated. 

Observational studies of deferoxamine in comparison with deferasirox or deferiprone are discussed xmder those sections. 

Aydinok Y, Unal S, Oymak Y, Verqin C, Turker ZD, YUdiz D, et al. Observational study comparing long-term safety and efficacy of deferasirox with desferrioxamine therapy in chelation-naive children with transfusional iron overload. Eur J Haematol May 2012 88(5) 431-8. 

Skin In a prospective study in 78 patients with P-thalassemia aged 10 months to 37 years, skin disorders of any kind were observed in 65 (83%) pruritus and xerosis were the most common (Table 1) [3 ]. Adverse events can occur simultaneously xerosis, for example, was often associated with pruritus. Systemic medication was common in these patients 40 received deferoxamine by slow infusion over 8-12 hours, 5-7 days a week 25 received deferi-prone and 10 received deferasirox. Subcutaneous infusion of deferoxamine is a frequent cause of local reactions, reflected in this series by the occurrence of skin erythema or irritation in 10 patients. Xerosis was less common in those who received deferasirox than in those who received deferoxamine or deferiprone. Xerosis can occur because of iron storage and also in zinc deficiency. The high spontaneous prevalence of a variety of skin disorders in patients with p-thalassemia, as noted in this study, is of interest in the context of attributing skin events to the use of chelating agents. 

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