Efficacy of Deferasirox as an  Oral Iron Chelator in Paediatric  Thalassaemia Patients

Jaiswal, Shikha; Hishikar, Rajesh; Khandwal, Onkar; Agarwal, Manju; Joshi, Usha; Halwai, Ajay; Maheshwari, Basant; Sheohare, Raka

doi:10.7860/jcdr/2017/22650.9395

Cited by 7 publications

(10 citation statements)

References 16 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…5. Chelation Therapy in studied patients: Deferasirox alone in a dose of 20-30 mg/kg/day once daily before meals [17], or Desferrioxamine alone 20-40 mg/kg/day for 6 days /week but in patients with persistently high serum ferritin levels above 3000 ng/ml, Deferasirox is combined with Desferrioxamine in a dose of 20-40 mg/kg/day for 10 days per month either by subcutaneous infusion using infusion pump in 8-12 hours/day or by continuous intravenous infusion for 8-10 hours/day (combined chelation therapy [18].…”

Section: Radiologicalmentioning

confidence: 99%

Magnetic Resonance Imaging Versus Serum Iron Status as Diagnostic Tools for Pituitary Iron Overload in Children with Beta Thalassemia

Morad

El-Shanshory

Hablas

et al. 2021

JAMMR

View full text Add to dashboard Cite

Background: Thalassemia is a common genetic disorder associated with endocrine disorders. Iron deposition may start in the anterior pituitary gland, but clinical signs are usually not evident until puberty. Aim of the study was to evaluate pituitary iron overload in children with β thalassemia using MRI T2* and correlate MRI T2* and biochemical markers of iron overload with pituitary hormones. Patients and Methods: This study was carried out on 30 children with β-thalassemia major (19 females and 11 males ) with their age ranging from 10 - 18 years and mean age value of 12.8 ± 2.4 and 30 healthy children of matched age and sex as controls in the period from September 2018 to September 2019. For all patients the following were done: complete clinical evaluation including anthropometric data and Tanner staging, laboratory investigations including serum iron status, thyroid function, basal and growth hormone provocation test by clonidine, Follicle stimulating hormone (FSH) , Luteinizing hormone (LH) , and pituitary MRI T2*. Results: Weight, Z score of weight, height, Z score of height and Body mass index (BMI) were found significantly lower in patients compared with controls. Patients had delayed puberty compared with controls. Pituitary MRI T2* was found significantly lower in patients compared with controls (P=0.001). FSH, LH, and provocative growth hormone levels were found significantly lower in patients compared to controls (p <0.001). Serum ferritin & iron were found significantly higher in patients than controls (P<0.001). Significant negative correlations were found between serum ferritin and Weight, Height, Z score of Weight, Z score of Height, BMI and pituitary hormones. Significant negative correlations were found between Pituitary MRI T2* and serum ferritin. Significant positive correlations were found between Pituitary MRI T2* and Weight, Height, Z score of Weight, Z score of Height and pituitary hormons. The pituitary T2* carried the sensitivity of 80% ,100% and 100% and specificity of 70% ,83.3% and 100% for predicting GH hormone abnormality, LH and FSH respecetively. Conclusions: There were a significant positive correlations between pituitary MRI T2*and anthropometric measurement and pituitary hormones of studied patients. There was a significant negative correlation between Pituitary T2* and serum ferritin. Pituitary iron overload can be detected by MRI T2* which is a diagnostic tool in detecting pituitary iron overload with subsequent effect on hormonal secretion especially growth hormone. Recommendations: Children with thalassemia should undergo meticulous follow up regarding regular blood transfusion, regular iron chelators in a proper manner and doses and routine use of MRI T2* in the evaluation of pituitary iron overload before irreversible damage occur in the pituitary gland.

show abstract

Section: Radiologicalmentioning

confidence: 99%

Magnetic Resonance Imaging Versus Serum Iron Status as Diagnostic Tools for Pituitary Iron Overload in Children with Beta Thalassemia

Morad

El-Shanshory

Hablas

et al. 2021

JAMMR

View full text Add to dashboard Cite

show abstract

“…[ 7 ] The iron overload resulting from the timely blood transfusions is the culprit of the disease process in thalassemia patients that can lead to organ injury and collapse. [ 8 ] In thalassemia patients, treatment with iron-chelating agents have demonstrated significant decline in organ damage due to iron overload and enhance survival rate. [ 9 ] Of late, there are three iron-chelating agents available on the market for the management of iron burdens such as deferoxamine, deferasirox, and deferiprone.…”

Section: Significance Of Iron Chelation Therapymentioning

confidence: 99%

“…[ 6 12 ] These patients require safe and effective iron chelation therapy as our body has a restricted physiologic mechanism to eliminate iron. [ 8 ] Primarily, the iron chelators bind to labile iron molecules with low molecular weight, so at any time only a minor portion of human body iron is accessible for chelation. [ 2 ] Therefore, the treatment with an iron-chelating agent is maximum effective if its activity exists throughout the day.…”

Section: Significance Of Iron Chelation Therapymentioning

confidence: 99%

See 1 more Smart Citation

Effectiveness and tolerability of twice daily dosing of deferasirox in unresponsive and intolerant transfusion-dependent beta-thalassemia patients: A narrative review

et al. 2020

View full text Add to dashboard Cite

Chronic iron overload in beta-thalassemia patients after continuous blood transfusions has caused notable morbidity and mortality in these patients. The once-a-day oral iron chelator, deferasirox has established efficacy and bearable safety in adults and pediatric thalassemia patients. It is now extensively used for the management of transfusional hemosiderosis. However, a number of studies have revealed a few patients continued to be none respondent or intolerant toward the once-a-day regimen of deferasirox even after the administration of maximum dose recommended by the World Health Organization. In the literature, there were three studies showing the boon of twice in a day dosing of deferasirox among transfusional-dependent beta thalassemia patients. Therefore, a nonsystematic review was conducted on above three studies to ascertain the enhanced effectiveness and tolerability of twice per day regimen of deferasirox with the same total dose as that of once daily regimen of deferasirox in unresponsive or intolerant transfusion-dependent beta-thalassemia (TDT) patients. All the above studies concluded that the twice per day regimen of deferasirox was more efficacious and tolerable among TDT patients when compared to the once-a-day regimen with the same total daily dose. Although there was a significant good results from these studies, there is a need to conduct either muticenter study or randomized control study in a larger number of patients for the better confirmation of the results as all the above studies were conducted in the small number of TDT patients.

show abstract

“… 43 JaiSwal et al later reported a significant mean reduction in SF of 1207.11 ng/ml (32.38% decrease) after 12 months therapy with DFX at a mean dose of 38 mg/kg/d in 45 heavily transfused thalassemia patients. 44 A prospective observational study including 176 patients with TDT (total 267), reported long-term results in pediatric patients treated with DFX and documented a decrease in median SF of 575 ng/ml after five years of DFX therapy with a mean dose of 25.8 mg/kg/d. 45 A Cochrane review reported by Bollig et al indicated similar efficacy of DFX as compared to DFO (depending on the ratio of DFX to DFO dose, generally showing similar results at a mean ratio of 1 mg of DFX to 1.8 mg of DFO).…”

Section: Evidence In Tdtmentioning

confidence: 99%

Deferasirox: Over a Decade of Experience in Thalassemia

Moukalled

Bou‐Fakhredin

Taher

2018

Mediterr J Hematol Infect Dis

View full text Add to dashboard Cite

Thalassemia incorporates a broad clinical spectrum characterized by decreased or absent production of normal hemoglobin leading to decreased red blood cell survival and ineffective erythropoiesis. Chronic iron overload remains an inevitable complication resulting from regular blood transfusions (transfusion-dependent) and/or increased iron absorption (mainly non-transfusion-dependent thalassemia), requiring adequate treatment to prevent the significant associated morbidity and mortality. Iron chelation therapy has become a cornerstone in the management of thalassemia patients, leading to improvements in their outcome and quality of life. Deferasirox (DFX), an oral iron chelating agent, is approved for use in transfusion dependent and non-transfusion-dependent thalassemia and has shown excellent efficacy in this setting. We herein present an updated review of the role of deferasirox in thalassemia, exploring over a decade of experience, which has documented its effectiveness and convenience; in addition to its manageable safety profile.

show abstract

Efficacy of Deferasirox as an Oral Iron Chelator in Paediatric Thalassaemia Patients

Cited by 7 publications

References 16 publications

Magnetic Resonance Imaging Versus Serum Iron Status as Diagnostic Tools for Pituitary Iron Overload in Children with Beta Thalassemia

Magnetic Resonance Imaging Versus Serum Iron Status as Diagnostic Tools for Pituitary Iron Overload in Children with Beta Thalassemia

Effectiveness and tolerability of twice daily dosing of deferasirox in unresponsive and intolerant transfusion-dependent beta-thalassemia patients: A narrative review

Deferasirox: Over a Decade of Experience in Thalassemia

Contact Info

Product

Resources

About