Serum Uric Acid As a Predictor Factor of the Response to Deferasirox Therapy for Patients with b-Thalassemia Major

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 5306-5306
Author(s):  
Efthimia Vlachaki ◽  
Vasileios Perifanis ◽  
Antonia Kondou ◽  
Nikolaos Neokleous ◽  
Aikaterini Teli ◽  
...  
Keyword(s):  
Uric Acid ◽  
Serum Uric Acid ◽  
Research Funding ◽  
Iron Chelation ◽  
Thalassemia Major ◽  
Iron Chelator ◽  
Beta Thalassemia ◽  
Positive Correlation ◽  
Significant Difference ◽  
Predictor Factor

Abstract Abstract 5306 Serum uric acid of patients with beta-thalassemia major (b-MA), despite the hyperuricosuria observed in these patients, is usually in the upper normal levels or increased due to excessive catabolism of the red blood cells (ineffective erythropoiesis). Deferasirox a new oral iron chelator with potential nephrotoxicity is recently used as iron overload treatment in patients with b-MA. Aim: Aim of the study is to investigate the effect of deferasirox on uric acid levels of patients with homozygous b-MA. Material-Method: 53 patients were enrolled to the present study with b-MA major, (aged 22.4 ± 14.7 years, range 4–12 years) 36 adults and 17 children, 32 females and 21 male. All the patients were transfusion-dependent with pretransfusional haemoglobin 9gr/dl and treated with iron chelation. The comparison was made between two different time points' measurements of uric acid and ferritin, at the beginning before Deferasirox, and one year later. The blood was taken from the patients early at mornings before the transfusion. Also uric acid was measured in 24 hour urine of patients under deferasirox, or other iron chelation therapy or after weekly discontinuation of deferasirox. Patients taken allopurinol or thiazide or with abnormal kidney function were excluded. Results: There is statistically significant difference (p< 0.001) between the mean annual value of serum uric acid (before 5.2 ± 1.3mg/dl) and ferritin (before 1653,4 ± 1026,3 ng/dl) before and after the start of deferasirox (uric acid after 4.2 ± 1.3 mg/dl and ferritin after 1529,07 ± 1137,44 ng/dl). Also, statistically significant positive correlation between the levels of serum uric acid and ferritin was found during the treatment with deferasirox. However, comparing the uric acid in urine of patients, it was not reported any statistically significant difference between treatment with deferasirox (859,75 ± 122), other iron chelators or without iron chelation for one week (844,32 ± 146). Conclusion: The mechanism of uric acid reduction in patients with b-MA major treated with deferasirox is not clear. However, it does not seem to be associated with excess of excretion by urine. The positive correlation between uric acid level and ferritin, allow us to consider uric acid as a predictor factor of the response to deferasirox therapy. Disclosures: Vlachaki: Novartis Hellas S.A.C.I.: Research Funding. Oikonomou:Novartis Hellas S.A.C.I.: Research Funding.

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

2018 ◽  
Vol 9 (SPL1) ◽  
Author(s):  
Zeina A Munim Al-Thanoon ◽  
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).

scholarly journals Comparison of Compliance of Different Iron Chelators Including Original and Bioequivalents of Deferasirox

Acta Medica ◽  
2020 ◽  
Vol 51 (3) ◽  
pp. 38-43
Author(s):  
Tekin Aksu ◽  
Namık Yaşar Özbek ◽  
Murat Söker ◽  
Çağrı Coşkun ◽  
Zeliha Güzelküçük ◽  
...  
Keyword(s):  
Patient Reported Outcomes ◽  
Iron Chelation ◽  
Treatment Compliance ◽  
Thalassemia Major ◽  
Iron Chelator ◽  
Beta Thalassemia ◽  
Iron Loading ◽  
Patient Reported ◽  
Tablet Formulations ◽  
Oral Chelators

Objective: The current iron chelation therapy regimens include deferoxamine (DFO), deferiprone (DFP), and deferasirox (DFX) in transfusion-dependent patients. Compliance with iron-chelating therapy is one of the significant determinants of mortality and morbidities related to iron loading in chronically transfused patients. This survey aims to compare the compliance and the satisfaction of DFO, DFP, and DFX formulations in patients who have iron-overloading in three hematology centers from Turkey. Moreover, bioequivalent (generic) formulations of dispersible DFX tablet compared with the original formulation in terms of taste and treatment compliance. Patients and Methods: A written questionnaire with a list of pre-set questions was applied to measure patient-reported outcomes to a total of 85 patients, where 77 had beta-thalassemia major, 7 had beta-thalassemia intermedia, and 1 had sickle cell anemia diagnoses. Results: The patients’ median age at enrollment was 15 years (range 7 – 42). The compliance was below 50% in 8 (18.6%), 4 (16%), and 5 (6.7%) in patients receiving DFO, DFP, and DFX, respectively. Additionally the compliance was below 80% in 16 (37.2%), 9 (36%), and 17 (22.6%) in patients receiving DFO, DFP, and DFX, respectively. It was found that 39 (47%) patients had compliance problems due to the dispersible DFX tablet formulations’ taste, except combination therapies. There was no difference between the currently used oral chelators in terms of taste and treatment compliance. Conclusion: This study draws attention to compliance problems in patients with iron-loading anemias, partly due to the unpleasant taste of DFX. Improving patient satisfaction and compliance with iron-chelator therapy may reduce complications of iron overload.

Iron Chelation Therapy and Mobilization of Hematopoietic Peripheral Progenitors in Patients with β-Thalassemia Major

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 5178-5178
Author(s):  
Gian Luca Forni ◽  
Marina Podestà ◽  
Marco Musso ◽  
Giovanna Piaggio ◽  
Khaled M Musallam ◽  
...  
Keyword(s):  
Myelodysplastic Syndromes ◽  
Research Funding ◽  
Chelation Therapy ◽  
Iron Status ◽  
Iron Chelation ◽  
Thalassemia Major ◽  
Iron Chelator ◽  
Iron Chelation Therapy ◽  
Colony Forming Unit

Abstract Abstract 5178 Several reports established an association between iron chelation therapy with deferasirox and hematopoietic improvement in patients with myelodysplastic syndromes. Data in β-thalassemia major (TM) patients is absent. We evaluated levels (frequency and aboslute number) of several hematopoietic peripheral progenitors (HPP: Colony Forming Unit-Granulocyte/Macrophage [CFU-GM], Erythroid Burst-Forming Unit [BFU-E], Colony Forming Unit-Granulocyte, Erythrocyte, Macrophage, Megakaryocyte [CFU-GEMM], and Long Term Culture-Initiating Cells [LTC-IC]) in 26 TM patients (median age 28. 5 years, 42. 3% males) and 12 age-matched controls. All TM patients had to be using the same iron chelator for at least 6 months with >80% compliance. The levels of all HPP were significantly higher in TM patients than controls, and varied between splenectomized and nonsplenectomized patients (lower CFU-GM and BFU-E, and higher LTC-IC in splenectomized patients). Patients using deferasirox (n=9) showed significantly higher levels of BFU-E compared with both deferoxamine (n=10) and deferiprone (n=7) treated patients (p<0. 01). After adjusting for age, sex, splenectomy status, serum ferritin changes, the association between higher BFU-E levels and deferasirox compared with deferoxamine or deferiprone therapy remained statistically significant (R2=0. 698, p=0. 006). Mobilization of BFU-E in TM patients receiving regular iron chelation therapy depends on the type of chelator used, independent of iron status. Disclosures: Forni: Novartis Pharmaceuticals: Research Funding; Ferrokin: Research Funding. Musallam:Novartis Pharmaceuticals: Honoraria.

scholarly journals Systemic Heme and Iron Overload Results in Depletion of Serum Hemopexin, Haptoglobin and Transferrin and Correlates with Markers of Endothelial Activation and Lipid Oxidation in Beta Thalassemia Major and Intermedia

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 2469-2469
Author(s):  
Francesca Vinchi ◽  
Gregory M Vercellotti ◽  
John D. Belcher ◽  
Eitan Fibach ◽  
Hala Zreid ◽  
...  
Keyword(s):  
Oxidative Stress ◽  
Iron Overload ◽  
Research Funding ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Thalassemia Major ◽  
Endothelial Activation ◽  
Beta Thalassemia ◽  
Iron Chelation Therapy ◽  
Cell Disease

Abstract Beta thalassemia is an inherited hemoglobinopathy due to reduced synthesis of Beta globin chains and, consequently, of hemoglobin A (a2b2). The clinical manifestations are mainly the result of chronic anemia and iron overload. The latter is due to increased iron absorption, induced by accelerated but ineffective erythropoiesis, and recurrent red blood cell transfusions. Alfa-chains and iron excess promote oxidative damage of red blood cell membrane, resulting in macrophage sequestration and extravascular hemolysis, and to a lower extent, in intravascular hemolysis, with consequent release of hemoglobin (Hb), heme and iron. Increasing evidence suggests that free heme exerts vasculotoxic, pro-inflammatory and procoagulant effects due to its ability to trigger endothelial and immune cells activation. In addition, a role for heme and iron has been postulated in the pathogenesis of other vascular diseases, including atherosclerosis. In mouse models of Beta thalassemia and sickle cell disease, circulating heme levels are elevated and correlate with the exhaustion of systemic scavengers for hemoglobin and heme, haptoglobin and hemopexin, respectively, as well as with severe endothelial dysfunction and inflammation. Hemopexin-based therapies significantly improve endothelial damage, vascular oxidative stress and inflammation in these mice (Vinchi et al., Circulation 2013, Blood 2016; Vercellotti GM. et al., Mol Med 2016). Whereas more data are reported on sickle patients in this regard, few data are available in patients with Beta thalassemia. In the present study, we examined serum samples from a cohort of 60 patients with Beta thalassemia major (age 11.5 ± 6.8, 44% males-56% females, Hb 7.69 ± 1.22 mg/dl, transfused every 3-4 weeks) and 7 patients with Beta thalassemia intermedia (age 14 ± 12 , 70% males-30% females, Hb 8.4 ± 0.74 mg/dl, transfused every 4-5 weeks). 10% of the patients received inconsistent iron chelation therapy. Serum from 10 healthy subjects (age 22.7±15.3, 50% males-50% females, Hb 13.12±1.15 mg/dl) served as control. Both groups of patients show high systemic heme and iron levels, which associate with a severe drop in serum haptoglobin, hemopexin and transferrin. Consistently, transferrin saturation (12.4±2 vs 79.6±24 %) and serum ferritin (55.14 ±0.23 vs 4919.2 ±2657.4 ng/ml) are elevated. Interestingly, these patients present with high systemic levels of the soluble adhesion molecules sVCAM-1 and sICAM-1, markers of enhanced endothelial activation. In addition, they show increased levels of serum malondialdehyde, a well-known marker of lipid peroxidation and oxidative stress, and high levels of circulating oxidized low density lipoproteins (oxLDL). All parameters significantly correlate with increased systemic heme and iron indices as well as decreased haptoglobin, hemopexin and transferrin levels. In conclusion, Beta thalassemia patients show a strong correlation between systemic heme and iron overload, depletion of the respective scavengers, and markers of oxidative stress and endothelial dysfunction, thus confirming studies in animal models. These results emphasize the involvement of serum hemoglobin, heme and iron in the pathophysiology of Beta thalassemia, including vascular dysfunction, and the key protective role of their carriers. These findings are relevant for disorders hallmarked by vasculopathy, such as sickle cell disease and Beta thalassemia, as well as cardiovascular diseases, such as atherosclerosis. Our data support the potential therapeutic benefit of the administration of hemoglobin/heme scavengers along with efficient iron chelation therapy to counteract heme- and iron-driven toxicity. (The last three authors equally contributed to the work) ****P<0.0001 Disclosures Vercellotti: CSL-Behring: Research Funding; Imara: Research Funding. Belcher:Cydan/Imara: Research Funding; CSL-Behring: Research Funding.

scholarly journals Investigation of JAK2V617F Mutation Prevalence in Patients with Beta Thalassemia Major

2019 ◽  
Author(s):  
Zari Tahannejad Asadi ◽  
Reza Yarahmadi ◽  
Najmaldin Saki ◽  
Mohammad Taha Jalali ◽  
Ali Amin Asnafi ◽  
...  
Keyword(s):  
Genetic Disorder ◽  
Thalassemia Major ◽  
Janus Kinase ◽  
Jak2v617f Mutation ◽  
Beta Thalassemia ◽  
Common Mutation ◽  
Signal Transducers ◽  
Significant Difference ◽  
Polymerase Chain ◽  
Low Prevalence

AbstractBackgroundBeta (β)–thalassemia major is a genetic disorder with anemia and an increased level of erythropoietin by Janus kinase/signal transducers and activators of transcription (JAK/STAT) signaling pathway. JAK plays an important role in cell signaling, and the common mutation in the JAK2 gene in myeloid disorders is called JAK2V617F.MethodsA total of 75 patients with beta (β)-thalassemia major patients, including 34 males (45%) and 41 females (55%), were enrolled in this study. The presence of the JAK2V617F mutation was assessed using the amplification-refractory mutation–polymerase chain reaction (ARMS-PCR) technique.ResultsAmong the 75 patients, 14 patients (19%) tested positive and 61 patients (81%) tested negative for JAK2V617F mutation. We observed no statistically significant difference in sex, age, genotype, and JAK2V617F mutation among patients (P> .05). However, a significant difference between blood-transfusion frequency and JAK2V617F mutation was observed (P <.05).ConclusionDue to the low prevalence of JAK2V617F mutation in thalassemia, using a larger population of the patients to investigate this mutation in ineffective erythropoiesis can be useful.

scholarly journals Serum uric acid levels in essential hypertension and its correlation with the severity of hypertension

2020 ◽  
Vol 7 (11) ◽  
pp. 1738
Author(s):  
Waseem Ramzan Dar ◽  
Sunil Kumar Gupta ◽  
Afzal Ahmad
Keyword(s):  
Uric Acid ◽  
Essential Hypertension ◽  
Serum Uric Acid ◽  
Serum Uric Acid Level ◽  
Laboratory Data ◽  
Social Software ◽  
National Committee ◽  
Hypertensive Patients ◽  
Co Morbidity ◽  
Significant Difference

Background: Serum uric acid has been closely linked and considered as an independent risk factor for development of hypertension. This study was carried out to assess the serum uric acid levels in essential hypertension and its correlation with the severity and known duration of hypertension.Methods: The present observational case control study was conducted between November 2019 to February 2020 on total 100 out patients (30-65 years) of which 50 known cases of essential hypertension irrespective of treatment status and were graded into different stages of hypertension as per Joint National Committee VII (JNC VII) guidelines. Rest 50 patients who were age- sex matched and without any co-morbidity were included as control. Relevant clinical and laboratory data were recorded using proforma. Statistical analysis was done using Statistical package for social software (SPSS) software.Results: Mean systolic/diastolic blood pressure (BP) was found significantly higher in cases 168/102 mmHg compare to control 114/74 mmHg. Among cases maximum patients were found in stage 2 hypertension which has significant difference in mean systolic/diastolic BP (186/112 mmHg, p<0.001) compare with stage 1 (146/94 mmHg) and isolated hypertensive patients (148/85 mmHg). Serum uric acid levels were significantly higher in stage 2 than other two hypertensive stages among cases and also compare to controls. Patients with >5 years of hypertension also has significant high serum uric acid level than <5 years of hypertension.Conclusions: Serum uric acid can be used probably as an early biochemical marker to determine the severity of hypertension as stage 2 hypertensive had more elevation in serum uric acid levels as compared to other hypertensive patients.

scholarly journals Relationship between Vitamin B9 (Folic Acid), Vitamin B12 (Cobalamin), and Peripheral Neuropathy in Children with Beta-Thalassemia Major

2021 ◽  
Vol 9 (2) ◽  
Author(s):  
Uni Gamayani ◽  
Titin Junaidi ◽  
Nushrotul Lailiyya ◽  
Nur Suryawan ◽  
Nanan Sekarwana
Keyword(s):  
Folic Acid ◽  
Peripheral Neuropathy ◽  
Vitamin B12 ◽  
Thalassemia Major ◽  
Beta Thalassemia ◽  
Control Group ◽  
Case Group ◽  
Beta Thalassemia Major ◽  
Vitamin B9 ◽  
Significant Difference

Vitamin B9 (folic acid) and B12 (cobalamin) are essential vitamins that play roles in the process of hematopoiesis and maintaining the function of peripheral nerves. Therefore, these deficiencies may create a risk for peripheral neuropathy in beta-thalassemia major patients. The purpose of this study is to determine the relationship between vitamin B9 level, vitamin B12 level, and peripheral neuropathy in beta-thalassemia major children. It was an observational analytical study with a case-control design has been conducted at Dr. Hasan Sadikin General Hospital Bandung, Indonesia, in May–July 2019. There were 47 beta-thalassemia major children with peripheral neuropathy (case) and 41 healthy children (control). All subjects completed a general demographic questionnaire, underwent neurological examination, and were tested for vitamin B9 and B12 serum levels. Data were then analyzed using the unpaired t test to compare the vitamin levels between both groups and Spearman’s rank correlation test to investigate the correlation between vitamin levels and the number of affected nerves in the case group. Comparison of folic acid levels in the case group (21.52±6.22 ng/mL) and the control group (23.81±7.51 ng/mL) showed no significant difference (p=0.19). In contrast, cobalamin in the case group (288.57±168.61 ng/mL) and the control group (385.95±197.48 ng/mL) showed a significant difference (p=0.01). In addition, there was a moderate correlation (p=0.004, r=0.41) between folic acid level and the number of motoric nerves affected in the case group. In conclusion, cobalamin level correlates with peripheral neuropathy in beta-thalassemia major patients, and folic acid level correlates with the number of affected nerves, especially motoric nerves. HUBUNGAN ANTARA VITAMIN B9 (ASAM FOLAT), VITAMIN B12 (KOBALAMIN), DAN NEUROPATI PERIFER PADA ANAK DENGAN TALASEMIA BETA MAYORVitamin B9 (asam folat) dan B12 (kobalamin) merupakan vitamin esensial yang berperan dalam proses hematopoiesis dan menjaga fungsi saraf tepi. Defisiensi vitamin ini dapat menimbulkan risiko neuropati perifer pada pasien talasemia beta mayor. Tujuan penelitian ini mengetahui hubungan antara kadar vitamin B9, vitamin B12, dan neuropati perifer pada anak talasemia beta mayor. Metode penelitian ini adalah analitik observasional dengan rancangan studi kasus kontrol yang dilakukan di RSUP Dr. Hasan Sadikin Bandung, Indonesia pada Mei–Juli 2019. Terdapat 47 anak talasemia beta mayor dengan neuropati perifer (kelompok kasus) dan 41 anak sehat (kelompok kontrol). Seluruh subjek penelitian mengisi kuesioner demografi umum, menjalani pemeriksaan fisis neurologis, serta dilakukan tes kadar vitamin B9 dan B12 serum. Uji t test tidak berpasangan digunakan untuk membandingkan kadar vitamin pada kedua kelompok dan uji korelasi Spearman untuk membandingkan kadar kedua vitamin tersebut dengan jumlah saraf yang terkena pada kelompok kasus. Perbandingan kadar asam folat kelompok kasus (21,52±6,22 ng/mL) dan kelompok kontrol (23,81±7,51 ng/mL) menunjukkan perbedaan yang tidak bermakna (p=0,19), sedangkan perbandingan kadar kobalamin kelompok kasus (288,57±168,61 ng/mL) dan kelompok kontrol (385,95±197,48 ng/mL) menunjukkan perbedaan yang bermakna (p=0,01). Selain itu, terdapat korelasi sedang (p=0,004; r=0,41) antara kadar asam folat dam jumlah saraf motorik yang terkena pada kelompok kasus. Kesimpulan, kadar kobalamin berhubungan dengan neuropati perifer pada penderita talasemia beta mayor dan kadar asam folat berhubungan dengan jumlah saraf yang terkena, terutama saraf motorik.

scholarly journals Elevated serum uric acid and triglycerides level in the patients with Type II Diabetes Mellitus- a Nepalese case control study

2017 ◽  
Vol 2 (2) ◽  
pp. 26-34
Author(s):  
Hridaya Parajuli ◽  
Jyotsna Shakya ◽  
Bashu Dev Pardhe ◽  
Puspa Raj Khanal ◽  
Narayan Prasad Parajuli ◽  
...  
Keyword(s):  
Diabetes Mellitus ◽  
Type 2 Diabetes ◽  
Uric Acid ◽  
Serum Uric Acid ◽  
Type Ii Diabetes ◽  
Type Ii Diabetes Mellitus ◽  
Diabetic Patients ◽  
Type Ii ◽  
Significant Difference

Background: Hyperuricemia is associated with type 2 diabetes, which is a metabolic disorder of multiple etiologies resulting from defects in insulin action. The present study wascarried out to look for any association between uric acid and Type II Diabetes Mellitus and also status of triacylglycerol level among those patients.Methods: The blood samples were collected 100 diabetic and 100 non-diabetic individuals in the department of biochemistry and then analyzed for estimation of blood glucose, Uric Acid and Triacylglycerol level.Results: The average level of serum uric acid in diabetic patients was higher (5.706±1.617) in comparison to non diabetic subjects (4.322±0.784) with statistically significant difference (p≤0.05). For female the result indicate there was a positive correlation between (FBS and triglycerides) and (triglycerides and uric acids) which was statistically significant (r =-0.465, n = 41, p = 0.002) and(r =-0.370, n = 41, p = 0.017) respectively.Conclusions: This study documents that hyperuricemia is associated with type 2 diabetes mellitus. Furthermore, the serum triacylglycerol and serum uric acid is also found to be associated risk factors for diabetic complications. Hence, timely diagnosis and management of diabetes is vital to control the complications related to diabetes.Ann. Clin. Chem. Lab. Med. 2016:2(1); 26-34

Fibroblast Growth Factor 23 (FGF23) and Klotho Protein in Beta-Thalassemia

2020 ◽  
Vol 52 (03) ◽  
pp. 194-201
Author(s):  
Dimitrios Stefanopoulos ◽  
Narjes Nasiri-Ansari ◽  
Ismene Dontas ◽  
Andromachi Vryonidou ◽  
Antonis Galanos ◽  
...  
Keyword(s):  
Growth Factor ◽  
Fibroblast Growth Factor ◽  
Fibroblast Growth Factor 23 ◽  
Iron Chelation ◽  
Thalassemia Major ◽  
The Body ◽  
Beta Thalassemia ◽  
Fibroblast Growth ◽  
Control Subjects ◽  
Number Of Patients

AbstractDerangements in phosphate and calcium homeostasis are common in patients with beta-thalassemia. Fibroblast growth factor 23 (FGF23) is among the main hormones regulating phosphate levels, while several studies underline an interplay between iron (Fe) and FGF23. Herein, we investigated, for the first time, the serum intact molecule (iFGF23) and the carboxyl-terminal fragment (C-FGF23) and Klotho levels simultaneously in patients with beta-thalassemia major receiving iron chelation regimens in comparison to healthy control subjects. We also correlated them with the body iron burden. The observational case-control study included 81 subjects (40 thalassemic patients and 41 healthy controls). Serum iFGF23, C-FGF23 and Κlotho were measured by ELISA. Parathormone, 25-hydroxycholecalciferol, calcium, and phosphorus were measured in blood and/or urine. The degree of hemosiderosis was evaluated by assessing the serum ferritin levels and performing T2* MRI measurements. Serum C-FGF23 levels were significantly lower in patients compared to control subjects (p=0.04), while iFGF23 and Klotho levels did not differ. Serum C-FGF23 levels were negatively correlated with ferritin (r=–0,421, p=0.018), whereas there were no significant correlations of each of the three factors with the iron chelation therapy. Decreased serum C-FGF23 levels were found in βTh patients which may be attributed to inhibition of proteolytic cleavage of iFGF23. Further studies in a greater number of patients will shed more light on the disturbances of the iFGF23, Klotho and C-FGF23 in thalassemia and their possible role in bone disease of such patients.

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