scholarly journals BETA THALASSEMIA MAJOR; COMPARISON OF DEFERASIROX VERSUS DESFERRIOXAMINE AS IRON CHELATOR IN MULTITRANSFUSED PATIENTS

2017 ◽  
Vol 24 (02) ◽  
pp. 315-321
Author(s):  
Dr. Asma Mehreen ◽  
Dr. Saeeda Bano ◽  
Dr. Bushra Ujala
Keyword(s):  
Thalassemia Major ◽  
Iron Chelator ◽  
Beta Thalassemia ◽  
Beta Thalassemia Major

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.

scholarly journals Assessment of Subclinical Renal Glomerular and Tubular Dysfunction in Children with Beta Thalassemia Major

Children ◽  
2021 ◽  
Vol 8 (2) ◽  
pp. 100
Author(s):  
Asmaa A. Mahmoud ◽  
Doaa M. Elian ◽  
Nahla MS. Abd El Hady ◽  
Heba M. Abdallah ◽  
Shimaa Abdelsattar ◽  
...  
Keyword(s):  
Cystatin C ◽  
Kidney Injury ◽  
Thalassemia Major ◽  
Beta Thalassemia ◽  
Good Survival ◽  
Control Group ◽  
Healthy Children ◽  
Serum Cystatin C ◽  
Serum Cystatin ◽  
Beta Thalassemia Major

Background: A good survival rate among patients with beta thalassemia major (beta-TM) has led to the appearance of an unrecognized renal disease. Therefore, we aimed to assess the role of serum cystatin-C as a promising marker for the detection of renal glomerular dysfunction and N-acetyl beta-D-glucosaminidase (NAG) and kidney injury molecule 1 (KIM-1) as potential markers for the detection of renal tubular injury in beta-TM children. Methods: This case-control study was implemented on 100 beta-TM children receiving regular blood transfusions and undergoing iron chelation therapy and 100 healthy children as a control group. Detailed histories of complete physical and clinical examinations were recorded. All subjected children underwent blood and urinary investigations. Results: There was a significant increase in serum cystatin-C (p < 0.001) and a significant decrease in eGFR in patients with beta-TM compared with controls (p = 0.01). There was a significant increase in urinary NAG, KIM-1, UNAG/Cr, and UKIM-1/Cr (p < 0.001) among thalassemic children, with a significant positive correlation between serum cystatin-C, NAG and KIM-1 as regards serum ferritin, creatinine, and urea among thalassemic patients. A negative correlation between serum cystatin-C and urinary markers with eGFR was noted. Conclusion: Serum cystatin-C is a good marker for detection of glomerular dysfunction. NAG and KIM-1 may have a predictive role in the detection of kidney injury in beta-TM children.

scholarly journals Left Atrial Strain Identifies Increased Atrial Ectopy in Patients with Beta-Thalassemia Major

Diagnostics ◽  
2020 ◽  
Vol 11 (1) ◽  
pp. 1
Author(s):  
Maria Vlachou ◽  
Vasileios Kamperidis ◽  
Efthymia Vlachaki ◽  
Georgios Tziatzios ◽  
Despoina Pantelidou ◽  
...  
Keyword(s):  
Blood Transfusion ◽  
Left Atrial ◽  
Systolic Pressure ◽  
Thalassemia Major ◽  
Beta Thalassemia ◽  
Tape Recording ◽  
Blood Transfusions ◽  
Systolic Strain ◽  
Peak Systolic Strain ◽  
Beta Thalassemia Major

Patients with beta-thalassemia major (β-ΤΜ) may develop cardiac arrhythmias through a multifactorial mechanism. The current study evaluated the association of cardiac structure and function on echocardiography with atrial ectopic burden on 24-hour tape recording in β-ΤΜ patients. This prospective study included consecutive β-ΤΜ patients. Demographic, laboratory, echocardiographic, cardiac magnetic resonance (CMR) T2* and 24-hour tape recording data were prospectively collected. The patients were classified according to the median value of premature atrial contractions (PACs) on 24-hour tape. In total, 50 β-TM patients (37.6 ± 9.1 years old, 50% male) were divided in 2 groups; PACs ≤ 24/day and > 24/day. Patients with PACs > 24/day were treated with blood transfusion for a longer period of time (39.0 ± 8.6 vs. 32.0 ± 8.9 years, p < 0.007), compared to their counterparts. Older age (OR: 1.121, 95% CI: 1.032–1.217, p = 0.007), longer duration of blood transfusion (OR:1.101, 95% CI:1.019–1.188, p = 0.014), larger LV end-diastolic diameter (OR: 4.522, 95% CI:1.009–20.280, p = 0.049), higher values of LA peak systolic strain (OR: 0.869, 95% CI: 0.783–0.964, p = 0.008), higher MV E/E′ average (OR: 1.407, 95% CI: 1.028–1.926, p = 0.033) and higher right ventricular systolic pressure (OR: 1.147, 95% CI: 1.039–1.266, p = 0.006) were univariably associated with PACs > 24/day. LA peak systolic strain remained significantly associated with PACs > 24/day after adjusting for the duration of blood transfusions or for CMR T2*. The multivariable model including blood transfusion duration and LA peak systolic strain was the most closely associated with PACs > 24/day. Receiver operating characteristic curve analysis identified a left atrial peak systolic strain of 31.5%, as the best cut-off value (83% sensitivity, 68% specificity) for prediction of PACs > 24/day. In β-TM patients, LA peak systolic strain was associated with the atrial arrhythmia burden independently to the duration of blood transfusions and CMR T2*.

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