Efficacy of deferasirox in children with β‐thalassemia: Single‐center 3 year experience

Ayçicek, Ali; Köç, Ahmet; Abuhandan, Mahmut

doi:10.1111/ped.12323

Cited by 9 publications

(6 citation statements)

References 20 publications

(63 reference statements)

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“…We identified four RCTs, two NRCTs, five NCTs and two ROS reporting DFP‐related AEs in 667 patients using DFP doses of 50–75 mg/kg/d . Occurrence of mild‐to‐moderate neutropenia was reported in 425 patients with a pooled incidence of 7.1% (95% CI 2.3–13.7).…”

Section: Resultsmentioning

confidence: 99%

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Safety profiles of iron chelators in young patients with haemoglobinopathies

Botzenhardt

Chan

et al. 2017

European J of Haematology

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Background: This review describes the safety of deferoxamine (DFO), deferiprone (DFP), deferasirox (DFX) and combined therapy in young patients less than 25 yr of age with haemoglobinopathies. Methods: Searches in electronic literature databases were performed. Studies reporting adverse events associated with iron chelation therapy were included. Study and reporting quality was assessed using AHRQ Risk of Bias Assessment Tool and McMaster Quality Assessment Scale of Harms. Prospective clinical studies were pooled in a random-effects meta-analysis of proportions. Results: Safety data of 2040 patients from 34 studies were included. Ninety-two case reports of 246 patients were identified. DFX (937 patients) and DFP (667 patients) possess the largest published safety evidence. Fewer studies on combination regimens are available. Increased transaminases were seen in all regimens (3.9-31.3%) and gastrointestinal disorders with DFP and DFX (3.7-18.4% and 5.8-18.8%, respectively). Therapy discontinuations due to adverse events were low (0-4.1%). Reporting quality was selective and poor in most of the studies. Conclusion: Iron chelation therapy is generally safe in young patients, and published data correspond to summary of product characteristics. Each iron chelation regimen has its specific safety risks. DFO seems not to be associated with serious adverse effects in recommended doses. In DFP and DFX, rare, but serious, adverse reactions can occur. Data on combined therapy are scarce, but it seems equally safe compared to monotherapy.

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Section: Resultsmentioning

confidence: 99%

“…One RCT, one NRCT, 10 NCTs and three ROS were identified from the literature counting 37 patients (37,50,52,(58)(59)(60)(61)(62)(63)(64)(65)(66)(67)(68)(69)(70). Investigated DFX doses ranged from 10 to 40 mg/ kg/d.…”

Section: Deferasirox (Dfx) Monotherapymentioning

confidence: 99%

Safety profiles of iron chelators in young patients with haemoglobinopathies

Botzenhardt

Chan

et al. 2017

European J of Haematology

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“…23 Similar findings were also observed in other independent real-world evidence studies that enrolled patients 2-17 years of age with a serum ferritin level higher than 1000 ng/mL, to achieve treatment targets of 500−1000 ng/mL using DT doses of 10 to ≥30 mg/kg/day. [24][25][26] The ENTRUST long-term (5-year) observational study was the first to specifically assess the safety of deferasirox DT in young children receipt of more than 20 pRBC units and serum ferritin level higher than 1000 ng/mL, and showed comparable compliance, safety, and efficacy of the deferasirox granules formulation and deferasirox DT. 30 Collectively, data from deferasirox in various formulations supported its safety and efficacy in children as young as 2 years of age, while eligibility criteria in most trials required abnormal LIC (≥2-3 mg/g) and/or serum ferritin levels higher than 500-1000 ng/mL (Figure 1).…”

Section: Deferasiroxmentioning

confidence: 99%

Iron chelation therapy for children with transfusion‐dependent β‐thalassemia: How young is too young?

Forni,

Kattamis,

Kuo

et al. 2024

Pediatric Blood & Cancer

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In this review, we provide a summary of evidence on iron overload in young children with transfusion‐dependent β‐thalassemia (TDT) and explore the ideal timing for intervention. Key data from clinical trials and observational studies of the three available iron chelators deferoxamine, deferiprone, and deferasirox are also evaluated for inclusion of subsets of young children, especially those less than 6 years of age. Evidence on the efficacy and safety of iron chelation therapy for children ≥2 years of age with transfusional iron overload is widely available. New data exploring the risks and benefits of early‐start iron chelation in younger patients with minimal iron overload are also emerging.

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“… 5 It has been in use in Turkey since 2007 and is used as the first-choice iron chelator in our center. 7 The film-coated tablet (FCT) form of DFX was approved by the FDA in 2015 and promises a more comfortable use than the original form. The DT formulation of DFX needs to be taken on an empty stomach with water, apple juice, or orange juice to obtain optimal bioavailability.…”

Section: Introductionmentioning

confidence: 99%

Switching from Deferasirox Dispersible Tablets to Deferasirox Film-Coated Tablets: Is There an Effect on Ferritin Levels in Children and Adolescents with Transfusion-Dependent Anemia?

Arslantaş,

Ayçiçek,

Tekgündüz

et al. 2024

Turkish Archives of Pediatrics

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Objective: To investigate the effect of switching from deferasirox dispersible tablet (DT) to deferasirox film-coated tablet (FCT) on serum ferritin (SF) levels in transfusion-dependent patients. Materials and Methods: Patients who received regular erythrocyte transfusion and whose treatment was switched from DT to FCT were included in the study. FCT start date was taken as the index date. Patients were followed over 2 equal and long periods, both before and after index date. Results: Thirty-two patients were included, and the comparison periods ranged from 4 to 12 months. The SF values increased from a median of 1723 ng/mL (range 717-5369 ng/mL) to 1.853 ng/mL (range 924-5478 ng/mL) after switching from DT to FCT ( P = .036). While there was a significant increase in median SF after switching in Turkish patients (1467 ng/mL to 1778 ng/mL, P = .010) and patients ≥12 years (1598-1848 ng/mL, P = .009), there was an insignificant ( P = .859) decrease in SF in immigrant children. Considering only the post-switch period, there was a non-significant increase in median SF in the entire cohort, while SF decreased significantly in immigrant children ( P = .026). No serious side effects were observed in any patient that would cause discontinuation of treatment. Conclusion : Overall, higher SF value was observed with FCT compared to DT in short term. There were different results between patient groups. Our results suggest that FCT is more effective than DT in patients with high basal ferritin and who are actually incompatible with treatment and should be preferred first in these patients.

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Efficacy of deferasirox in children with β‐thalassemia: Single‐center 3 year experience

Abstract: Deferasirox is safe and well tolerated; doses 21-24 mg/kg per day were not able to maintain stable iron balance, but ≥ 30 mg/kg per day was able to reduce iron in regularly transfused pediatric patients.

Cited by 9 publications

References 20 publications

Safety profiles of iron chelators in young patients with haemoglobinopathies

Safety profiles of iron chelators in young patients with haemoglobinopathies

Iron chelation therapy for children with transfusion‐dependent β‐thalassemia: How young is too young?

Switching from Deferasirox Dispersible Tablets to Deferasirox Film-Coated Tablets: Is There an Effect on Ferritin Levels in Children and Adolescents with Transfusion-Dependent Anemia?

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