Safety of iron chelator in the treatment of beta-thalassemia major: a systematic review with meta-analysis

2014 ◽  
Vol 14 (5) ◽  
pp. 382-385
Author(s):  
Yan XU ◽  
SiHeng ZHANG ◽  
SuJian XIA ◽  
Hong JIANG
Keyword(s):  
Systematic Review ◽  
Meta Analysis ◽  
Thalassemia Major ◽  
Iron Chelator ◽  
Beta Thalassemia ◽  
Beta Thalassemia Major

scholarly journals A Systematic Review and Meta-Analysis on the Epidemiology of Hepatitis B and Hepatitis C Virus among Beta-Thalassemia Major Patients in Pakistan

2021 ◽  
Author(s):  
Usman Waheed ◽  
Noore Saba ◽  
Akhlaaq Wazeer ◽  
Saeed Ahmed
Keyword(s):  
Hepatitis B ◽  
Sample Size ◽  
Meta Analysis ◽  
Thalassemia Major ◽  
World Health ◽  
Baseline Survey ◽  
Beta Thalassemia ◽  
Beta Thalassemia Major ◽  
Open Access Journals ◽  
Pooled Prevalence

AbstractBeta-thalassemia major patients are the leading consumers of blood transfusions in Pakistan and, therefore, have a greater risk of acquiring transfusion-transmitted infections, most notably hepatitis B and C virus (HBV and HCV). The present study includes a comprehensive review on the status of HBV and HCV in beta-thalassemia major patients in Pakistan. For this purpose, we examined original articles assessing the epidemiology of HBV and HCV in transfusion-dependent thalassemia patients.We searched 10 major subscription databases from January through February 2020, that is, Medline, PakMediNet, CINAHL, Scopus, PubMed, Web of Science, Embase, Science Direct, Google Scholar, and Directory of Open Access Journals. The World Health Organization resources were also explored for relevant reports. The search criteria included published articles up to December 31, 2019, with no language restrictions. Articles identified were introduced into the Endnote version X9 software and then screened for relevance and duplication. The results were stated as the pooled prevalence for the overall study and also for region-wise subgroups.A total of 33 studies conducted from 1995 to 2019 were included in the review. All 33 articles yielded information on HCV prevalence, while 19 of them provided information on HBV prevalence. The overall sample size was 8,554 that tested the prevalence of HCV in thalassemia patients. The sample size from the 19 studies that tested the prevalence of HBV was 6,184. The overall pooled prevalence of HBV was computed to be 4.13%, while the pooled prevalence of HCV was 29.79%. The majority of the studies were obtained from the Punjab Province (33.33%), followed by Khyber Pakhtunkhwa Province (24.24%).The total sample size of 33 studies was less than 10% of the total number of estimated thalassemic patients, that is, 100,000. Further studies or a national baseline survey are imperative to confirm the actual frequency of HBV and HCV in thalassemia patients across the country.

scholarly journals Comprehensive Review: Extramedullary Hematopoiesis in Patients with Beta Thalassemia Major (transfusion dependent thalassemia)

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4163-4163
Author(s):  
Eihab A. Subahi ◽  
Fateen Ata ◽  
Hassan Choudry ◽  
Ashraf Tawfiq Soliman ◽  
Vincenzo Desanctis ◽  
...  
Keyword(s):  
Systematic Review ◽  
Retrospective Studies ◽  
Case Reports ◽  
Thalassemia Major ◽  
Extramedullary Hematopoiesis ◽  
Beta Thalassemia ◽  
X Rays ◽  
Descriptive Data ◽  
True Incidence ◽  
Beta Thalassemia Major

Abstract Abstract Introduction: Thalassemia is one of the most common hemoglobinopathies, with around 5% of world's population expected to have some degree and type of thalassemia. Beta thalassemia (BT) occurs due to a deficient production of the beta-globin chain of hemoglobin. BT is classified into minor, intermedia, and major, defined by their clinical and laboratory findings. Extramedullary hematopoiesis is one of the complications of BT, primarily seen in minor/intermedia subtypes. EMH is the production of blood cells outside the marrow as a compensatory response to longstanding hypoxia. Due to chronic transfusions, it is not expected in patients with beta thalassemia major (BTM). However, there are multiple case reports of EMH in BTM. The incidence of EMH in BTM is thought to be <1%. However, given the increasing cases, it seems that the true incidence is much higher than expected. This systematic review aims to pool the available data and provide cumulative evidence on the occurrence of EMH in BTM patients. Methods: This is a systematic review of case reports, series, and retrospective studies that presented data on the occurrence of EMH in BTM patients. Data were recorded and analyzed in Microsoft Excel 2016 and SPSS 26. The protocol has been registered in PROSPERO: CRD42021242943. Results: Data from 253 cases of EMH in BTM patients were extracted from 2 retrospective studies and 71 case reports (some of the descriptive data from case reports and most of the descriptive data from retrospective studies was missing). The mean age at presentation was 35.3 +/- 0.5 years. Males constituted 57.7% of the total population. Mean hemoglobin at presentation with EMH was 8.2 +/- 2.1 mg/dL. Lower limb weakness was the most common presenting feature (N=23). Other features included localized pain, urinary incontinence, and an incidental finding of EMH. Magnetic resonance imaging (MRI) was the most widely used diagnostic modality (N=226). However, computed tomography scans, positron emission tomography scans, tissue biopsy, myelograms, and X-rays were also used to diagnose EMH. Various treatments were reported either alone or in combination. Overall, blood transfusion was the commonest reported treatment (N=30), followed by radiotherapy (N=20), surgery (N=15), hydroxyurea (N=12), steroids (N=6), and exchange transfusion (N=2). An outcome was reported in 20% of patients, all recovered, with no reported mortality. Conclusion: EMH is considered a rare phenomenon in BTM patients. However, there are many cases reported in which EMH occurred in BTM despite chronic blood transfusions. EMH can occur in any organ system and can present with a varied clinical feature. MRI can effectively diagnose EMH, and conservative management is reported to have similar results compared to invasive treatments. More extensive studies, focusing especially on outcomes, are required to enhance the guidelines on managing EMH in patients with BTM. Figure 1 Figure 1. Disclosures No relevant conflicts of interest to declare.

Deferasirox-induced liver injury and Fanconi syndrome in a beta-thalassemia major male

2020 ◽  
Vol 13 (7) ◽  
pp. e234542 ◽  
Author(s):  
Jacqueline Fraser ◽  
Rowena Brook ◽  
Tony He ◽  
Diana Lewis
Keyword(s):  
Thalassemia Major ◽  
Iron Chelator ◽  
Clinical Findings ◽  
Beta Thalassemia ◽  
Beta Thalassemia Major ◽  
First Case ◽  
Dispersible Tablets ◽  
Clinical Awareness ◽  
Supportive Management ◽  
Acute Cholestasis

A 33-year-old male presenting with subacute abdominal pain was found to have hyperbilirubinaemia, hypokalaemia and hyponatraemia. This was in the setting of transitioning between deferasirox iron chelator formulations, from dispersible tablets to film-coated tablets for ongoing treatment of chronic iron overload secondary to transfusion requirement for beta-thalassemia major. A liver biopsy demonstrated acute cholestasis with patchy confluent hepatocellular necrosis and mild to moderate microvesicular steatosis. Based on the histological, biochemical and clinical findings, the diagnosis of hepatotoxicity and Fanconi-like syndrome was made. The patient improved clinically and biochemically with cessation of the deferasirox film-coated tablets and supportive management. To our knowledge, this is the first case report of hepatotoxicity and Fanconi-like syndrome occurring due to deferasirox film-coated tablets with previous tolerance of dispersible deferasirox tablets. It is important to raise clinical awareness of this potentially severe complication.

Single-Centre, North American Experience with Compassionate Use of Deferiprone in Patients with Beta-Thalassemia Major,

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 3185-3185 ◽  
Author(s):  
Filip Miscevic ◽  
Kevin H.M. Kuo ◽  
Richard Ward
Keyword(s):  
North America ◽  
Thalassemia Major ◽  
Iron Chelator ◽  
Adherence Rate ◽  
Beta Thalassemia ◽  
Compassionate Use ◽  
Single Centre ◽  
Beta Thalassemia Major ◽  
Cardiac Siderosis ◽  
Oral Iron Chelator

Abstract Abstract 3185 Introduction: Deferiprone (DFP) is a thrice daily, oral iron chelator with cardiac-specific properties. It has been demonstrated to improve LV EF% and reduce cardiac related morbidity and mortality in patients with Beta Thalassemia Major (TM). It was approved as second line therapy by European regulatory agencies in 1999, but despite being developed by a Toronto-based pharmaceutical company, has yet to be approved by Health Canada or the FDA. Since July 2004 it has been available via a compassionate use program (CUP). The Red Blood Cell Disorders Program (RBCDP, Toronto General Hospital, Canada) is Canada's largest adult Hemoglobinopathy comprehensive care centre. In mid 2009, the RBCDP took a programmatic decision to systematically enrol those patients with significant cardiac siderosis, and hence at risk of cardiac events and death, in the CUP. Patients intolerant of or having a suboptimal responsive to Deferoxamine or/and Deferasirox were also considered for DFP therapy. Here we report on a single centre experience with this oral chelator within a North American healthcare system. Methods: Approval was received from the Research Ethics Board and data was collected retrospectively from the RBCDP database, electronic patient records, and CUP monitoring records. Patients complied with weekly CBC monitoring through the use of community based laboratories with electronic transmission of results in a timely manner. All patients who were prescribed DFP for at least 12 months between January 2009 and January 2011 were included in the analysis. Efficacy data and adverse events are reported here. Results: Thirty-four patients were identified (19 females and 15 males; 29 beta-Thalassemia Major, 4 Hemoglobin E/b-Thalassemia compound heterozygote, 1 homozygous sickle cell disease) for a total of 28.72 patient-years observed. 2 patients were excluded from analysis due to incomplete data. Nineteen patients (58%) were splenectomised. Mean age at the start of DFP therapy was 31.07 years (SEM +/− 5.18 years). 16 patients received DFP in combination with another chelator. 22/34 were prescribed DFP for severe cardiac siderosis. Sixteen (48%) of the patients were prescribed at 75 mg/kg/day and 17 (52%) at 100 mg/kg/day. Mean physician-assessed adherence rate was 68% (SEM +/− 12%), with 64% of patients having >90% adherence rate. Serial T2* and ejection fraction (EF) measurements from cardiac MRI were available in 22 patients, with a mean change in T2* of +2.6 ms/year (SEM +/− 0.5 ms/year) and EF +1.5%/year (SEM +/− 0.3%/year). There was a significant change in T2* measurements after an average of 425 days (SEM +/− 91 days) of DFP therapy (P=0.0006). No significant change was observed in LIC (−2.84 mg/g dry weight; P = 0.067) or ferritin values (542.6 umol/L; P=0.327). Neutropenia (ANC < 1.5) was observed in 4 patients totalling 11 instances but no agranulocytosis was observed. Four patients had an asymptomatic transient increase in alanine transferase (> 5x upper limit of normal). Four patients had arthralgias, which resolved with dose reduction. Discussion: We believe that the RBCDP currently has the largest active population of patients receiving chelation with DFP in North America. Initial data presented here demonstrates the drug to be effective in controlling cardiac iron overload; is well tolerated; and reveals no new adverse effects. Our experience mirrors that of Thalassemia centres outside of North America over the past 10 years. Longer follow up of this cohort is ongoing. Disclosures: Off Label Use: Deferiprone is an unlicensed medication in USA and Canada, and is available through compassionate use. It is an oral iron chelator. Kuo:Novartis Canada: Research Funding.

The Global survival rate of patients with beta thalassemia major: a systematic review and meta-analysis

2021 ◽  
Vol 7 (2) ◽  
Author(s):  
Maryam Soltani ◽  
Soheil Hassanipour ◽  
Yousef Veisani ◽  
Mitra Darbandi ◽  
Shahab Rezaiean
Keyword(s):  
Survival Rate ◽  
Meta Analysis ◽  
Random Effect ◽  
Random Effect Model ◽  
Survival Rates ◽  
Thalassemia Major ◽  
Beta Thalassemia ◽  
Policy Makers ◽  
Beta Thalassemia Major ◽  
Effect Model

Objectives: Thalassemia is a public health challenge in the countries entitled belt thalassemia but there is no pooled estimated of the survival rate on thalassemia major patients. The aim of this meta-analysis was to evaluate the pooled 10-, 15-, 20-, and 30-year survival rates of the patients with beta thalassemia major around the world. Methods: A comprehensive literature search of five international databases including Medline/PubMed, Scopus, Embase, Web of knowledge and ProQuest was conducted to identify studies reporting survival rate of beta thalassemia major until March 2020. Results: From 714 retrieved studies, seven studies with 8777 subjects included in the meta-analysis. Base on random effect model, the 10-, 15-, 20-, and 30-year survival rates were 98.39, 95.07, 90.41, and 82.93 percent, respectively. Conclusions: This meta-analysis provided acceptable results for estimating survival rate of beta-thalassemia compared to other studies. Hence, these results can be effectively used to develop and implement prevention and treatment interventions for policy makers.

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