Efficacy and Adverse Effects of Oral Chelating Therapy (Deferasirox) in Multi-Transfused Beta-Thalassemia Major Patients

Ejaz, Muzamil Shabana

doi:10.12669/pjms.313.6972

Cited by 17 publications

(20 citation statements)

References 22 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…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%

See 1 more Smart Citation

Safety profiles of iron chelators in young patients with haemoglobinopathies

Botzenhardt

Chan

et al. 2017

European J of Haematology

View full text Add to dashboard Cite

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.

show abstract

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

View full text Add to dashboard Cite

show abstract

“…Those 2 agents are preferred in some patients since they have oral forms. 17,18 Ocular and auditory side effects of DFS and DFP are rare. 6,19 Some patients included in our study were administered DFS and DFP due to intolerance to subcutaneous injections.…”

Section: Discussionmentioning

confidence: 99%

Olfactory Dysfunction in β Thalassemia Major Patients Treated With Iron-Chelating Agents

et al. 2019

View full text Add to dashboard Cite

Ocular and ophthalmological adverse effects may be seen in β-thalassemia major (BTM) patients treated with regular blood transfusions and iron-chelating agents. We hypothesized that olfactory dysfunction may be present in this population. In this study, we aimed to investigate olfactory dysfunction in patients with BTM and determine the etiological factors. A total of 43 patients with BTM were included in the study. Forty-three patients without any nasal complaints, history of facial trauma, or nasal surgery were included as the controls. All participants had nasal endoscopy. The iron-chelating agents used, their duration of use, as well as hemoglobin and ferritin levels of the BTM patients were recorded. Sniffin’ Sticks test (SST) was used to assess olfactory functions, and BTM and control groups were compared for the results. The correlations of SST scores with the other study parameters were analyzed. Eight (18.6%) of 43 patients in the BTM group had hyposmia while none of the patients in the control group had hyposmia ( P < .001). Older age, low-hemoglobin level, and longer use of deferoxamine were found to be correlated with olfactory dysfunction. Olfactory dysfunction may be seen in BTM patients treated with iron-chelating agents. The results of this study suggest that screening for olfactory function may be needed in routine follow-up of BTM patients.

show abstract

“…Selain itu, dijelaskan bahwa awalan dalam menggunakan terapi kelasi besi dan kemampuan absorbsi obat tiap pasien menjadi masalah tersendiri terhadap respon yang terjadi pada pasien. 13 Solanki dkk 14 melakukan penelitian terhadap 50 anak yang menderita talasemia β dengan usia 3-14 tahun. Hasil penelitian mendapatkan 32 (64%) anak mengalami eutiroid, 17 (34%) hipotiroid subklinis, dan 1 (2%) hipotiroid dan tidak didapatkan hipotirod sekunder.…”

Section: Pembahasanunclassified

Perbedaan Kadar Thyroid Stimulating Hormone dan Free Thyroxine pada Pasien Talasemia Β-Mayor dengan Kelasi Besi Deferasirox dan Deferiprone

Krisbiyantoro

Salimo²,

Moelyo³

2018

View full text Add to dashboard Cite

Universitas Sebelas Maret/RS dr. Moewardi Surakarta Latar belakang. Talasemia β Mayor merupakan kelainan herediter yang disebabkan gangguan produksi rantai globin. Transfusi rutin menyebabkan kelebihan besi yang tertimbun dalam jaringan sehingga menyebabkan kardiomiopati, gangguan liver, dan komplikasi endokrin. Tiroid merupakan kelenjar endokrin yang berperan penting bagi anak. Pengendapan besi di kelenjar tiroid dapat menyebabkan gangguan fungsi tiroid. Pemberian kelasi besi deferiprone dan deferasirox dan pengaruhnya pada kadar TSH dan FT4 perlu dievaluasi lebih lanjut. Tujuan. Menganalisis perbedaan kadar TSH dan FT4 pada pasien anak dengan talasemia β mayor menggunakan kelasi besi deferiprone dan deferasirox Metode. Penelitian analitik dengan rancangan potong lintang (cross sectional) terhadap 43 pasien talasemia β mayor anak berusia 9-18 tahun pada bulan April sampai Juni 2017. Pemilihan subjek dilakukan secara consecutive sampling. Data di analisis dengan SPSS 20 mengunakan uji t independen dan uji man whitney. Hasil. Rerata usia pasien 12,5+3,12 tahun. Rerata kadar TSH kelompok deferiprone dan deferasirox adalah 3.051,78 IU/ml dan 2.351,29 IU/ml. Sedangkan rerata kadar FT4 untuk kelompok deferiprone dan deferasirox 15.424,12 mmol/l dan 15.822,75 IU/ ml. Tidak terdapat perbedaan kadar TSH dan FT4 pada kelompok yang mendapatkan deferasirox dan deferiprone berturut-turut nilai TSH (p=0,148; p>0,05) dan FT4 (p=0,836; p>0,05). Kesimpulan. Tidak terdapat perbedaan kadar TSH dan FT4 pada pasien talasemia beta mayor yang mendapatkan kelasi deferasirox maupun deferiprone. Sari Pediatri 2017;19(4):209-13Kata kunci: TSH, FT4, deferiprone, deferasirox The Differences of Thyroid Stimulating Hormone dan Free Thyroxin levels in Major Beta Thalassemia with Deferasirox and Deferiprone Chelation TherapyAries Krisbiyantoro, Harsono Salimo,* Annang Giri Moelyo* Background. Major ß Thalassemia represents a group of recessively inherited hemoglobin disorder, which is characterized by reduced synthesis of globins chains. Frequent blood transfusions can lead to iron overload. Its can induced cardiomyopathy, liver disease, and endocrine complications. The thyroid is the important glands in children. Iron overload can cause thyroid disarrangement. Deferiprone and deferasirox treatment are needed to evaluate about the effect in thyroid gland. Objective. To know the differences in TSH and FT4 level in mayor thalassemia patient with deferiprone and deferasirox chelation therapy Methods. This study was a cross-sectional study. It was performed in March to Juni 2017. Forty three patients, age between 9 years to 18 years old as subject to this study. This study examine the TSH and FT4 levels in deferiprone and deferasirox groups which met the inclusion and exclusion criteria. Differences in TSH and FT4 levels were compared between the two groups then analyzed with the Mann -Whitney test . Results. The mean age of the subject was 12,5 +3.12 years old. The mean of TSH level in deferiprone and deferasirox group was 3.051,78 IU/ml and 2.35...

show abstract

Efficacy and Adverse Effects of Oral Chelating Therapy (Deferasirox) in Multi-Transfused Beta-Thalassemia Major Patients

Cited by 17 publications

References 22 publications

Safety profiles of iron chelators in young patients with haemoglobinopathies

Safety profiles of iron chelators in young patients with haemoglobinopathies

Olfactory Dysfunction in β Thalassemia Major Patients Treated With Iron-Chelating Agents

Perbedaan Kadar Thyroid Stimulating Hormone dan Free Thyroxine pada Pasien Talasemia Β-Mayor dengan Kelasi Besi Deferasirox dan Deferiprone

Contact Info

Product

Resources

About