Combination of Oral Deferiprone and Subcutaneous Infusion of Desferrioxamine in Chelating Overloaded Iron in Pediatric Patients with Beta-Thalassemia Disease.

Abstract Abstract 4057 Poster Board III-992 Introduction Iron overload is a serious complication found in thalassemia patients requiring regular transfusion. Subcutaneous infusion of desferrioxamine for 8-12 hours/day five days per week creates constraint and incomplete compliance especially pediatric patients. Objective The purpose of this study is to evaluate the effectiveness of daily 50-75 mg/kg of locally-produced oral deferiprone (GPO-L-One from Thai Governmental Pharmaceutical Organization) combined with 40 mg/kg of subcutaneous infusion of desferrioxamine two days per week in pediatric patients with transfusion-dependent thalassemia disease. Methods 49 patients with homozygous beta-thalassemia disease (n=7) and beta-thalassemia/Hb E disease (n=42) with a median age of 10 years were enrolled in the study. Splenectomy was performed in four and eight patients with homozygous beta-thalassemia disease and beta-thalassemia/Hb E disease, respectively. They required regular packed red cell transfusion of 15-20 ml/kg every 3-4 weeks to maintain the pre-transfusion hematocrit at 27%. The median level of ferritin was 2,907 ng/mL (interquartile range 2,419-3,686 ng/mL). The clinical manifestation and complications of iron chelation were closely monitored. Complete blood counts, BUN, creatinine, liver profile, serum ferritin and urine examination were checked monthly. Eight to ten hours of subcutaneous infusion of desferrioxamine was given using an infusion pump. The patients initially received deferiprone at the dose of 50 mg/kg and increased to 75 mg/kg at the fifth month if the declination of serum ferritin was less than 15% of the initial level. Results The patients were divided into three groups according to their serum ferritin levels: group 1, n=9, serum ferritin was more than 4,000 ng/ml; group 2, n=31, serum ferritin between 2,000 and 4,000 ng/ml and group 3, n=9, serum ferritin was less than 2,000 ng/ml. The median required dose of deferiprone in groups 1 and 2 (75 mg/kg) was significantly higher than that of group 3 (50 mg/kg) with a p value of 0.0001. The serum ferritin levels were gradually decreased during one-year studied period as shown in Figure 1. Ten minimal side effects of deferiprone occurred mostly in the first four months of treatment among 8 out of 49 patients including neutropenia less than 1,500/mcl (n=4); platelet counts less than 150,000/mcl (n=2); slightly high serum creatinine (n=2); serum ALT of 292 unit/L (n=1) and proteinuria of 10 mg/m2/h with normalized serum creatinine (n=1). No patient had agranulocytosis. These patients received deferiprone at the dose of 50 mg/kg in 6 cases and 75 mg/kg in 2 cases. All the abnormal laboratory investigations were transient and became normalized within one month. However, deferiprone was discontinued in four patients with ANC of 1,406/mcl, ANC 1,461/mcl, platelet counts of 130,000/mcl and proteinuria. Conclusion The preliminary study of using locally-produced deferiprone combined with two-day infusion of deferrioxamine is tolerable and creates satisfaction to the studied patients. Disclosures: No relevant conflicts of interest to declare.

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Twice Daily Use of Deferasirox Is More Effective in Decreasing Serum Ferritin

Blood ◽

10.1182/blood.v124.21.2675.2675 ◽

2014 ◽

Vol 124 (21) ◽

pp. 2675-2675 ◽

Cited By ~ 1

Author(s):

Fatma Gumruk ◽

Sule Unal ◽

Turan Bayhan ◽

Tuncay Hazirolan ◽

A. Murat Tuncer ◽

...

Keyword(s):

Serum Ferritin ◽

Half Life ◽

Conflicts Of Interest ◽

Maximum Dose ◽

Iron Chelation ◽

Beta Thalassemia ◽

Iron Loading ◽

Once Daily ◽

T2 Mri

Abstract Patients with beta-thalassemia major (BTM) are prone to tissue iron overloading in case that not adequately chelated. Among the iron chelators, development of oral chelators have improved patients’adherence to treatment. Deferasirox is a tridentate iron chelator, used once daily with a half life of 8-16 hours. The negative chelation effect is achieved at doses above 30 mg/kg/day and the currently FDA-approved maximum dose for use in patients is 40 mg/kg/day. However, some of the deferasirox side effects are dose-dependent increasing the occurences of adverse events at high doses. Additionally, although there is data that non-transferrin bound iron is effectively decreased by once daily dosing, the iron chelation effects of the drug may increase since in some of the patients half-life of the drug has been reported to be even less than 7 hours. Herein, we compared the iron chelation effect and tolerability of deferasirox in the same patients who were using deferasirox at a dose of 40 mg/kg/day. The patients with BTM who were under iron chelation with deferasirox at a dose of median 40 mg/kg/day (38-41) once daily for at least 6 months were included (n=10). These patients were receiving deferasirox at a maximum dose in the enrollment to the study related to either serum ferritin levels above 1500 ng/ml or moderate to severe iron loading in cardiac or liver tissues. These patients were put on a twice daily regimen of the same dose and followed up at a median time period of 7 months (4-17). The serum ferritin, ALT, creatinine levels and T2* MRI of heart and liver were obtained at the beginning of the twice daily dosing and by the end of the follow-up time. Patients were given a questionairre to investigate the tolerability and satisfaction of once daily and twice daily use. The median age of the study group was 21 years (3-34), hal were males. The patients’ serum ferritin, cardiac and hepatic iron loading levels were summarized in Table 1. There was a statistically significant decrease in serum ferritin levels with twice daily use of deferasirox compared to once daily use of the same dose. The initial and follow-up ALT and serum creatinine levels did not differ significantly (p>0.05). None of the patient required a dose reduction or cessation of the drug related to a toxicity. The major tolerability concern of the patients in once daily dosing was nausea in 2 of the patients (20%). After twice daily dosing the major concern of the patients was twice daily use of the drug itself in 2 patients (20%). None of the patients reported nausea in twice-dosing. The patients’ satisfaction survey in the end of the study for once or twice daily use was for once daily use revealed preference for twice-use in 5 (50%) of the patients for either no nausea (n=2) with twice-use or better decrease in serum ferritin levels (n=3). Three (30%) preferred once daily use as a better way, related to lesser drug use. Two patients reported that there were no difference in terms of satisfaction. In conclusion, twice daily use of deferasirox at higher doses is much better tolerated and causes a better decline in serum ferritin levels of already high iron burden. Further studies in larger sample groups may be more definitive. Table 1. The initial and follow-up iron overloading evaluations Mean ± SD (Range) p Initial SF (ng/ml) Follow-up SF 2020±983 (1016-4128) 1533±1026 (521-4003) 0.047 Initial cardiac T2* MRI (ms) Follow-up cardiac T2* MRI 25.4±8.6 (14.6-38) 21.5±7.8 (13.8-30) 0.068 Initial liver T2* MRI (ms) Follow-up liver T2* MRI 3.6±1.9 (1.3-8) 6.7±5.1 (2.1-13.6) 0.465 SF: serum ferritin; SD: standard deviation Disclosures No relevant conflicts of interest to declare.

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Real-World Experience of Switching from Deferasirox Dispersible to Film-Coated Tablets: Impact on Adherence to Chelation Therapy, Iron Overload and Renal Function

Blood ◽

10.1182/blood-2018-99-119030 ◽

2018 ◽

Vol 132 (Supplement 1) ◽

pp. 1062-1062

Author(s):

Simon Cheesman ◽

Raakhee Shah ◽

Sara Trompeter ◽

Perla Eleftheriou ◽

Bernadette Hylton ◽

...

Keyword(s):

Renal Function ◽

Serum Creatinine ◽

Iron Overload ◽

Serum Ferritin ◽

Patient Adherence ◽

Beta Thalassemia ◽

Daily Dose ◽

Baseline Values ◽

The Mean ◽

The Impact

Abstract Background Chronic iron overload is an important complication of long-term blood transfusions for severe beta-thalassemias, sickle cell disease and other blood disorders. Iron chelation therapy (ICT) is required to bind and excrete excess iron, which would otherwise accumulate and lead to organ damage or failure. Deferasirox is a once-daily, orally administered ICT approved for the treatment of chronic iron overload due to frequent blood transfusions in patients with beta-thalassemia major and other anaemias. A film-coated tablet (FCT) formulation was launched in the UK in 2016 and replaced the dispersible tablet (DT) formulation. In the context of a randomised clinical trial, the FCT formulation showed greater adherence and patient satisfaction, better palatability and fewer tolerability concerns than the DT. Furthermore, treatment compliance by pill count was higher with FCT (92.9%) than with DT (85.3%) (Taher et al, 2017). Little information exists however about compliance, efficacy and tolerability outside of a clinical trial setting. Objectives We wished to assess in a 'real world' situation, the effects of switching the deferasirox formulation from DT to FCT on patient adherence to ICT, iron overload and renal function. Methods Patients receiving ICT with deferasirox who were switched from the DT to FCT formulations were followed over a 12-month period and results audited using hospital dispensing and biochemistry records. The date of the first FCT prescription was defined as baseline. The initial daily dose used for switching from DT to FCT was as per manufacturer's recommendations: 70% of the DT daily dose. The impact on iron overload was assessed by comparing serum ferritin levels at 3, 6, 9 and 12 months post-switch with baseline values. The impact on renal function was assessed by comparing serum creatinine levels at 3, 6, 9 and 12 months post-switch with baseline values as well as the number of serum creatinine increases of 30% or greater above baseline. The changes in serum ferritin and creatinine were subsequently analysed by paired t-test. The Proportion of Days Covered (PDC) was calculated as a measure of patient adherence to ICT in the 12 months before and after switching formulations. Results 74 patients switched from deferasirox DT to FCT with the following diagnoses: beta-thalassemia (n = 45), sickle-cell disease (9), thalassemia-intermedia (6), HbE-thalassemia (5), other transfusion-dependent disorders (9). The median age was 36 (range: 1-78yo), mean baseline serum ferritin was 2767µg/L (range: 412-8742), mean baseline creatinine was 64.5 umol/L (range: 17-140) and the median prescribed daily dose of DT was 1250mg (range: 62.5 - 3500). The mean PDC in the 12 months prior to switching formulations was 0.80 (range: 0.31-1.00). This increased to 0.91 (range: 0.21-1.00) in the 12 months following the switch to FCT. The median prescribed daily dose of FCT was 900mg (range: 90 - 2520) The mean changes in ferritin and creatinine at 3, 6, 9 and 12 months post-switch are shown in the table. 6 out of 74 patients (8%) had a creatinine increase of >30% from baseline whilst receiving the FCT, occurring after an average of 120 days (range: 30-260). All 6 patients were managed by dose adjustment of FCT and creatinine returned to the normal range in 5 out of 6 cases. Conclusions The switch from deferasirox DT to FCT resulted in improved patient adherence to chelation, a reduction in mean serum ferritin and a modest rise in mean serum creatinine. Some patients showed a reversible rise in creatinine from baseline. The median daily dose of FCT prescribed was 72% of the DT formulation, approximately equivalent according to the known bioavailability of the different preparations and suggesting that improvements in serum ferritin were due to the more consistent daily administration of the FCT rather than an increased daily deferasirox dose. We suggest that when the fall in ferritin is abrupt and/or to levels <1000µg/L, serum creatinine should be followed particularly carefully to avoid over-exposure to deferasirox from the FCT. We further speculate that patients who may have over-reported adherence to the DT prior to switching may be most susceptible to this effect. Reference Taher, A. T., et al. (2017). "New film-coated tablet formulation of deferasirox is well tolerated in patients with thalassemia or lower-risk MDS: Results of the randomized, phase II ECLIPSE study." American journal of hematology 92(5): 420-428. Table Table. Disclosures Garbowski: Vifor: Consultancy. Porter:Novartis: Consultancy; Cerus: Honoraria; Agios: Honoraria.

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An epidemiological study on the clinico-hematological profile of pediatric patients with congenital hemolytic anemia

International Journal of Contemporary Pediatrics ◽

10.18203/2349-3291.ijcp20170021 ◽

2017 ◽

Vol 4 (2) ◽

pp. 374

Author(s):

Santosh Kumar ◽

Deepa Singh ◽

Abhay Garg

Keyword(s):

Hemolytic Anemia ◽

Pediatric Patients ◽

Public Health Problem ◽

Beta Thalassemia ◽

Total Serum ◽

Major Public Health Problem ◽

Inherited Disorders ◽

Hb E ◽

The Mean ◽

Congenital Hemolytic Anemia

Background: Among the inherited disorders of blood, haemoglobinopathy and thalassaemia constitute a major bulk of congenital hemolytic anemia in India.Methods: The present cross sectional study was conducted on pediatric patients aged 0-15 years admitted in pediatric ward of Department of Pediatrics, MGM Medical College and LSK Hospital, Kishanganj, Bihar, India between December 2015 to November, 2016. The data on socio-demographic profile, relevant clinical history and examination and hematological parameters were assessed. Results: Out of 211 patients evaluated, most common cause of congenital hemolytic anemia was Hb E Beta thalassemia (39.8%), followed by beta thalassemia (27.9%), beta thalassemia trait (14.2%), Hb E disease (11.3 %) and Hb E trait (6.6%). There was male preponderance (male 63%, female 37 %). The mean hemoglobin was found to be lowest in patients of β thalassemia (5.1 gm/gl) and HbE β thalassemia (5.8 gm/dl). The mean total serum bilirubin was found to be highest among β Thalassemia patients (3.0 mg/dl). Hepatomegaly was the most common clinical finding among the study population (57.8%), followed by splenomegaly (54.9%) and hemolytic facies and jaundice (both 53%).Conclusions: The incidence of HbE beta thalassemia is relatively high in comparison to other varieties of thalassemias and is a major public health problem in this area of the country.

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A Study of Phenotypic Heterogeneity in Hb E-Beta Thalassemia and Its Correlation with Hb E Level

Blood ◽

10.1182/blood.v124.21.1365.1365 ◽

2014 ◽

Vol 124 (21) ◽

pp. 1365-1365 ◽

Cited By ~ 1

Author(s):

Maitryee Bhattyacharyya ◽

Basab Bagchi ◽

Meet Kumar

Keyword(s):

Disease Severity ◽

Conflicts Of Interest ◽

Age Of Onset ◽

Clinical Phenotype ◽

Phenotypic Heterogeneity ◽

Clinical Severity ◽

Beta Thalassemia ◽

Disease Modifying Drugs ◽

Hb E ◽

Size Growth

Abstract INTRODUCTION: Hb E-beta thalssemia has variable clinical phenotype, varying from NTDT to severe transfusion dependent thalassemia. Factors influencing the severity include HbF level, type of beta mutation, co-inheritance of alpha mutation (including triplication and quadruplication), and various polymorphisms. We evaluate the possible correlation between HbE levels and clinical severity in patients in Hb E-beta thalassemia. AIMS AND OBJECTIVES: To study the phenotypic heterogeneity of Hb E-beta thalassemia patients with respect to Hb E levels. MATERIALS AND METHODS: Patients >18years were enrolled and evaluated for clinical phenotype as per Mahidol score (age of onset, age of first transfusion, requirement for transfusion, spleen size, growth retardation and steady-state hemoglobin), and classified into mild, moderate and severe. Further biochemical data was evaluated including serum ferritin. The clinical phenotype severity was correlated with HbE and HbF levels. Patients on disease modifying drugs (eg hydroxyurea) were excluded. RESULTS: A total of 120 patients of Hb E-beta thalassemia were enrolled with M:E = 1.1:1. 65.8% patients were 18-30 years age range, while 5.8% were above 50years of age. As per Mohidol score, 39.2% (47) patients were mild, 45.8% (55) were moderate and 15% (18) were severe phenotype. Observations amongst patients of different clinical severity are as shown in Table 1. Abstract 1365. Table 1.CharacterMildModerateSevereRangeP valueNumber475518Baseline Hb (g/dl)7.9+-0.96.4+-1.16.2+-0.73.8-10.30.000Age at presentation (years)18.3+-8.7613.1+-8.03.16+-2.081-440.000Age at 1st transfusion (years)22.1+-11.415.1+-8.763.9+-1.91-600.000Freq of transfusion (annual)2.5+-4.512.2+-7.619.3+-11.40-480.000Spleen size (cms)5.6+-2.47.8+-2.910.4+-3.40-150.000Serum ferritin (ng/ml)792.5+-534.61206.5+-523.91570.5+-581.264.2-2924.20.000 Hb E levels of the patients ranged from 28% to 83.5%, and HbF levels ranged from 3.1% to 49.4%. On statistical analysis, difference in HbE levels was significant between severe vs mild and intermediate groups (p=0.0005 and 0.012 respectively), but not amongst mild vs intermediate groups (p=0.21). Also, difference in HbF levels was significant between mild vs moderate and severe groups (p=0.007 and 0.004 respectively), but not between moderate vs severe groups (p=0.26). Regression analysis showed HbF and HbE to be independent parameters of severity and both have negative correlation with disease severity. CONCLUSION: HbE is an independent parameter of severity and correlated negatively with disease severity in HbE-beta thalassemia. Disclosures No relevant conflicts of interest to declare.

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Transfusional Iron Overload in Sickle Cell Patients:Outcomes of Chelation Therapy

Blood ◽

10.1182/blood-2021-151743 ◽

2021 ◽

Vol 138 (Supplement 1) ◽

pp. 4177-4177

Author(s):

Daniel Adamkiewicz ◽

Abhishek A. Mangaonkar ◽

James Son ◽

Hongyan Xu ◽

Leigh Wells ◽

...

Keyword(s):

Sickle Cell ◽

Serum Ferritin ◽

Chelation Therapy ◽

Conflicts Of Interest ◽

Chelating Agent ◽

Regulatory Peptides ◽

Beta Thalassemia ◽

Glycoprotein Hormone ◽

Liver Iron ◽

Number Of Patients

Abstract Iron (Fe) overload is not rare among sickle cell disease (SCD) patients. It results from either chronic transfusions for primary or secondary stroke prevention, or more commonly sporadic, mostly unnecessary transfusions and are associated with significant morbidity, secondary to hepatic, cardiac and renal Fe deposition. Previous studies at our Center showed that 12% of the adult SCD population had Fe overload, and vast majority of these (80%) resulted from episodic transfusions (Son et al, 2013). Subsequent studies showed that the Fe regulatory peptide, hepcidin was appropriately upregulated in SCD subjects with Fe overload, and the plasma levels of the glycoprotein hormone erythroferrone (ERFE) was not as high as that found in patients with transfusion dependent beta thalassemia (TDT) due to the absence of significant ineffective erythropoiesis in SCD (Thawer et al, 2017, Mangaonkar et al, Brit. J. Haematol, 2020). We report on the utilization and outcomes in Fe overloaded SCD patients who were prescribed the oral chelating agent deferasirox. 22 patients were prescribed deferasirox; median age was 38, 12 female 10 male. 21 had Hb SS, and 1 had s-b 0 thalassemia. Deferasirox dose ranged from 720-2500 mg/day (12 to 28 mg/Kg/day). Nonadherence was ascertained by patients' own admission. Figures 1-3 show the pre and post ferritin levels in subjects who were on deferasirox, and in controls; patients who took deferasirox had a more pronounced decrease in their ferritin (p=.004 vs p=.74). Although hepatic MRI for liver iron content (LIC) was not available on all patients, in those who underwent MRI there was a correlation between LIC and serum ferritin obtained at close temporal proximity (Fig. 4). Most common side effects of deferasirox were gastrointestinal (abdominal pain, nausea, vomiting, diarrhea), which were seen less commonly with the newer oral formulation (Jadenu). Several conclusions can be drawn from our observation on relatively small number of patients: 1) Deferasirox is effective in decreasing Fe overload as shown by serum ferritin levels 2) Second generation of oral deferasirox is better tolerated, and therefore is associated with improved adherence, 3) Documentation of a decrease in LIC with chelation will be important for the reversal of Fe induced organ damage, and 4) Parallel studies of the levels of Fe regulatory peptides hepcidin and erythroferrone (ERFE) will clarify the effect of chelation therapy on biomarkers of Fe metabolism. Figure 1 Figure 1. Disclosures No relevant conflicts of interest to declare.

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Pharmacometabolomics for Predicting Variable Busulfan Exposure in Pediatric HSCT Patients

Blood ◽

10.1182/blood.v128.22.2218.2218 ◽

2016 ◽

Vol 128 (22) ◽

pp. 2218-2218

Author(s):

Bora Kim ◽

Kyung Taek Hong ◽

Ji Won Lee ◽

Kyung-Sang Yu ◽

In-Jin Jang ◽

...

Keyword(s):

Liver Function ◽

Serum Ferritin ◽

Serum Ferritin Level ◽

Pediatric Patients ◽

Conflicts Of Interest ◽

Ferritin Level ◽

Elevated Serum ◽

Hematopoietic Stem ◽

Potential Biomarker ◽

Mitochondrial Fatty Acid

Abstract Optimal dosing for busulfan is important for minimization of systemic toxicity from overexposure and graft failure or relapse from underexposure. Herein, we investigated potential markers for predicting individual variation in the pharmacokinetics of busulfan, and suggested possible mechanism for inter-individual variability by using pharmacometabolomics. Fifty-nine pediatric patients undergoing busulfan-based conditioning chemotherapy for hematopoietic stem cell transplantation were divided into three groups according to the area under the concentration-time curve (AUC) of busulfan; low-, medium-, and high-AUC group. Nontargeted metabolic profiling of baseline urine samples showed that deferoxamine metabolites were abundant, while 2 acylcarnitines and phenylacetylglutamine were significantly lower in high-AUC group, compared with low-AUC group. Higher level of deferoxamine, an iron-chelating agent for the patients with a high serum ferritin level during the conditioning chemotherapy, suggested pharmacokinetic interaction between serum ferritin level and busulfan exposure. Retrospective analysis of the correlation between serum ferritin level and busulfan AUC showed positive correlation in 130 pediatric patients. The optimal busulfan dose to meet the target AUC of 18,750 μg*h/L/day was calculated to be 119.7 ± 30.1 mg/m2 in patients with ferritin < 1,000 ng/mL and 106.1 ± 29.3 mg/m2in patients with ferritin ≥ 1,000 ng/mL (P=0.021). Mechanismly, previous studies have indicated that ferritin, acylcarnitine and phenylacetylglutamine are closely associated with liver function. Increased serum ferritin is thought to be responsible for an increased production of oxygen free radicals and activation of GSH turnover, which means reduction of GSH levels in both plasma and erythrocytes and this depletion seems to be related to the decrease of busulfan metabolism. Down regulation of acylcarnitines is associated with deregulation of mitochondrial fatty acid β-oxidation by hepatic injury. PAGN, a marker of waste nitrogen scavenger, was down-regulated which indicates ammonia-related metabolism could also be involved in busulfan exposure. Hyperammonemia, a clinical condition of elevated ammonia levels, is mainly lead by liver cell damage. Taken together, our findings demonstrate that elevated serum ferritin levels are a potential biomarker correlated with busulfan exposure and provide some evidence that busulfan metabolism seems to decrease in the patients with reduced liver function. Disclosures No relevant conflicts of interest to declare.

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Clinical-Reported Outcomes Of Deferoxamine Versus Deferasirox In The Treatment Of Homozygous BetaThalassemia

International Journal of Research in Pharmaceutical Sciences ◽

10.26452/ijrps.v9ispl1.1382 ◽

2018 ◽

Vol 9 (SPL1) ◽

Author(s):

Zeina A Munim Al-Thanoon ◽

Mustafa Basil ◽

Nasih A Al-Kazzaz

Keyword(s):

Serum Ferritin ◽

Iron Chelation ◽

Iron Chelator ◽

Beta Thalassemia ◽

Control Group ◽

Current Standard ◽

Cross Sectional ◽

Significant Difference ◽

Multiple Blood ◽

Group 2

Iron chelation therapy with deferoxamine (DFO),the current standard for the treatment of iron overload in patients with betathalassemia,requires regular subcutaneous or intravenous infusions. This can lead to reduced quality of life and poor adherence,resulting in increased morbidity and mortality in iron-overloaded patients with beta-thalassemia. Deferasirox (DFX) is an orally administered iron chelator that has been approved for use in many countries. The requirement of an effective,well tolerated iron chelator with a less demanding mode of administration has led to the development of deferasirox. The present study was aimed to compare the satisfaction and compliance with deferoxamine versus deferasirox (Exjade®),a novel oral iron chelator in patients with transfusion - dependent beta- thalassemia. A cross-sectional,single-center investigation study was carried out in the Thalassemia Center of Ibn-Atheer Teaching Hospital in Nineveh province,Iraq. One hundred and eight thalassemic patients aged between 2- 20 years old having received multiple blood transfusions and a serum ferritin greater than 1500 ng/ml. Patients were randomised into two groups. Group 1 received deferoxamine at a dose of 20-50mg/kg/day and group 2 received deferasirox at the dose of 10-30 mg/kg/day. Another 56 apparently healthy volunteers were used as a control group. The assessment of chelation was done during the period between November 2013 and February 2014 by measurement of serum ferritin. Satisfaction and compliance was assessed by using a special questionnaire prepared by the researcher. Out of the 108 thalassemic patients enrolled there was no discontinuation in treatment with the two drugs under study. The serum ferritin did not change significantly in any of the chelation groups. In comparison with the patients who were treated with DFO,those receiving DFX reported a significantly higher rate of compliance and satisfaction (P < 0.05). However,no significant difference was observed between the two groups regarding their satisfaction (P > 0.05).Compliance with deferasirox (50 %) was more than that with deferoxamine (20 %). Satisfaction with deferoxamine was significantly lower than deferasirox (p= 0.00).

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Haematological Disease Pattern at Specialized HOPD - A study in Bangabandhu Sheikh Mujib Medical University

Haematology Journal of Bangladesh ◽

10.37545/haematoljbd201817 ◽

2018 ◽

Vol 2 (02) ◽

pp. 39-41

Author(s):

Md. Rafiquzzaman Khan ◽

Arifur Rahman ◽

Khaza Amirul Islam ◽

AQM Ashraful Haque ◽

Masuda Begum

Keyword(s):

Iron Deficiency ◽

Chronic Myeloid Leukaemia ◽

Myeloid Leukaemia ◽

Iron Deficiency Anaemia ◽

Beta Thalassemia ◽

Female Ratio ◽

Deficiency Anaemia ◽

Beta Thalassaemia ◽

Hb E ◽

Medical University

The aim of this retrospective observational study was to observe the pattern and frequency of haematological disorders among the patients attending in the specialized Haematology outpatient Department (HOPD) in Bangabandhu Sheikh Mujib Medical University. Consecutive 201 patients over the period of one year were enrolled. Their age ranged from 01 to 72 years with a mean age of 36.76 years. Most of the patients (34.3%) were in between the ages of 31 to 45 years followed by 16 to 30 years (27.9%). Male to female ratio was 0.65. Iron deficiency anaemia is the most common (24.9%) followed by chronic myeloid leukaemia (11.9%), Hb E beta thalassaemia (9.5%), idiopathic thrombocytopenic purpura (9.5%), beta thalassaemia trait (7.0%), Hb E trait (5.5 %), aplastic anaemia (5.0%), multiple myeloma (3.5%), acute lymphoblastic leukaemia (3.0%). Acute myeloid leukaemia, autoimmune haemolytic anaemia, chronic lymphocytic leukaemia, anaemia of chronic disease, non-Hodgkin lymphoma, polycythaemia, beta thalassemia major and alpha thalassemia was 2.5%, 2.5%, 2.0%, 1.5%, 1.5%, 1.5%, 1.0% and 1.0%, respectively. In the present study, we observed that iron deficiency anaemia the most common non-malignant disease and chronic myeloid leukaemia is the common haematological malignancy.

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The Relationship between Serum Ferritin Levels and 5-Year All-Cause Mortality in Hemodialysis Patients

Blood Purification ◽

10.1159/000515639 ◽

2021 ◽

pp. 1-7

Author(s):

Emre Erdem ◽

Ahmet Karatas ◽

Tevfik Ecder

Keyword(s):

Serum Ferritin ◽

Hemodialysis Patients ◽

Rank Test ◽

Significant Difference ◽

All Cause Mortality ◽

Group 3 ◽

Group 2 ◽

The Relationship ◽

Group 1

Introduction: The effect of high serum ferritin levels on long-term mortality in hemodialysis patients is unknown. The relationship between serum ferritin levels and 5-year all-cause mortality in hemodialysis patients was investigated in this study. Methods: A total of 173 prevalent hemodialysis patients were included in this study. The patients were followed for up to 5 years and divided into 3 groups according to time-averaged serum ferritin levels (group 1: serum ferritin <800 ng/mL, group 2: serum ferritin 800–1,500 ng/mL, and group 3: serum ferritin >1,500 ng/mL). Along with the serum ferritin levels, other clinical and laboratory variables that may affect mortality were also included in the Cox proportional-hazards regression analysis. Results: Eighty-one (47%) patients died during the 5-year follow-up period. The median follow-up time was 38 (17.5–60) months. The 5-year survival rates of groups 1, 2, and 3 were 44, 64, and 27%, respectively. In group 3, the survival was lower than in groups 1 and 2 (log-rank test, p = 0.002). In group 1, the mortality was significantly lower than in group 3 (HR [95% CI]: 0.16 [0.05–0.49]; p = 0.001). In group 2, the mortality was also lower than in group 3 (HR [95% CI]: 0.32 [0.12–0.88]; p = 0.026). No significant difference in mortality between groups 1 and 2 was found (HR [95% CI]: 0.49 [0.23–1.04]; p = 0.063). Conclusion: Time-averaged serum ferritin levels >1,500 ng/mL in hemodialysis patients are associated with an increased 5-year all-cause mortality risk.

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Liver Enzymes in Children with beta-Thalassemia Major: Correlation with Iron Overload and Viral Hepatitis

Open Access Macedonian Journal of Medical Sciences ◽

10.3889/oamjms.2015.059 ◽

2015 ◽

Vol 3 (2) ◽

pp. 287-292 ◽

Cited By ~ 7

Author(s):

Khaled M. Salama ◽

Ola M. Ibrahim ◽

Ahmed M. Kaddah ◽

Samia Boseila ◽

Leila Abu Ismail ◽

...

Keyword(s):

Blood Transfusion ◽

Iron Overload ◽

Viral Hepatitis ◽

Serum Ferritin ◽

Liver Enzymes ◽

Transferrin Saturation ◽

Thalassemia Major ◽

Beta Thalassemia ◽

Elevated Liver Enzymes ◽

Hcv Antibody

BACKGROUND: Beta Thalassemia is the most common chronic hemolytic anemia in Egypt (85.1%) with an estimated carrier rate of 9-10.2%. Injury to the liver, whether acute or chronic, eventually results in an increase in serum concentrations of Alanine transaminase (ALT) and Aspartate transaminase (AST).AIM: Evaluating the potentiating effect of iron overload & viral hepatitis infection on the liver enzymes.PATIENTS AND METHODS: Eighty (80) thalassemia major patients were studied with respect to liver enzymes, ferritin, transferrin saturation, HBsAg, anti-HCV antibody and HCV-PCR for anti-HCV positive patients.RESULTS: Fifty % of the patients were anti-HCV positive and 55% of them were HCV-PCR positive. Patients with elevated ALT and AST levels had significantly higher mean serum ferritin than those with normal levels. Anti-HCV positive patients had higher mean serum ferritin, serum ALT, AST and GGT levels and higher age and duration of blood transfusion than the negative group. HCV-PCR positive patients had higher mean serum ferritin and serum ALT and also higher age and duration of blood transfusion than the negative group.CONCLUSION: Iron overload is a main leading cause of elevated liver enzymes, and presence of HCV infection is significantly related to the increased iron overload.

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