scholarly journals Causal-Investigative Analysis of the Formation of Anemia in Children

2020 ◽  
Vol 5 (5) ◽  
pp. 271-277
Author(s):  
V. G. Bebeshko ◽  
◽  
K. M. Bruslova ◽  
N. M. Tsvietkova ◽  
L. O. Gonchar ◽  
...  

The purpose of the study was to determine the main causal factors in the formation of anemia in children of Ukraine, depending on the morphometric changes in erythrocytes of blood, indicators of iron metabolism for the formation of a risk group for oncohematological diseases. Material and methods. 770 children were examined: 724 with anemia, 46 with acute lymphoblastic leukemia. We studied the parameters of the erythrocyte lineage of hematopoiesis, morphometric parameters of erythrocytes, indicators of iron metabolism (serum iron, ferritin, transferrin, the transferrin saturation with iron, hematocrit, the content of δ-aminolevulinic acid and coproporphyrin in urine, pituitary thyroid stimulating hormone depending on the type of somatic pathology. Anemic states were distributed taking into account the average erythrocyte volume and the average hemoglobin content in the erythrocyte, and dividing diagnoses microcytic-hypochromic or normocytic-normochromic anemia, respectively. Results and discussion. The study showed that in children with normocytic-normochromic anemia, the number of erythrocytes and hematocrit were lower than in patients with microcytic-hypochromic anemia, while average erythrocyte volume, average hemoglobin content, serum iron, serum ferritin and transferrin saturation with iron were higher. The number of reticulocytes in the peripheral blood in all the examined subjects was standard. That is, the anemic conditions in children differed in ferrokinetic parameters, in particular, with and without iron deficiency. Taking into account the age of the children and the reasons for the development of anemia, a third of the girls of puberty with microcytic-hypochromic anemia had menorrhagias. In children under 6 years of age with normocytic-normochromic anemia, diseases of the gastrointestinal tract were more often registered; in the older 6 years – gastrointestinal diseases, helminthiasis and allergic reactions compared with patients with microcytic-hypochromic anemia. The development of normocytic-normochromic anemia in children and the functioning of the gastrointestinal tract were influenced by drug treatment for chronic pathology in the body. All children with anemia had an irrational diet. Porphyria was diagnosed in 3.8% of children with microcytic-hypochromic anemia. In 12.7% of children with normocytic-normochromic anemia, the serum thyroid stimulating hormone level was at the upper limit of the reference value (mean 3.3±0.6) mU/L), which correlated with a reduced number of erythrocytes in blood (r = -0.65) and increased values of average erythrocyte volume (r = 0.41) and average hemoglobin content (r = 0.35), and indicates changes in the erythrocyte lineage of hematopoiesis associated with the initial manifestations of thyroid hypofunction. An excess of iron was observed in 7.1% of older boys with normocytic-normochromic anemia, which requires additional examination. In patients with acute lymphoblastic leukemia were diagnosed with normocytic-normochromic anemia of varying severity. The serum ferritin level was (272.1±28.4) ng/ml and was significantly higher than in children with normocytic-normochromic anemia. In 12 of 46 patients, transferrin saturation with iron was increased and amounted to (70.2±2.3)%. Moreover, the higher the level of serum iron and serum ferritin, the higher was the transferrin saturation with iron (rs = 0.5; rs = 0.85). An inverse correlation was established between transferrin saturation with iron, patient survival (rs = -0.45) and a higher probability of death (rs = -0.46). Conclusion. Children with normocytic-normochromic anemia require in-depth examination and constitute a risk group for the development of myelodysplastic syndrome and leukemia

2021 ◽  
Vol 15 (8) ◽  
pp. 2013-2016
Author(s):  
Shahid Ishaq ◽  
Muhammad Imran ◽  
Hashim Raza ◽  
Khuram Rashid ◽  
Muhammad Imran Ashraf ◽  
...  

Aim: To determine correlation of iron profile in children with different stages of chronic kidney disease (CKD) presenting to tertiary care hospital. Methodology: A total of 81 children with chronic kidney disease stage having glomerular filtration rate (GFR) less than 90 (ml/min/m2) aged 1 – 14 years of either sex were included. Three ml serum sample was taken in vial by hospital duty doctor for serum ferritin level, serum iron, transferrin saturation and total iron binding capacity. The sample was sent to hospital laboratory for reporting. Iron profiling was done evaluating hemoglobin (g/dl), serum iron (ug/dl), serum ferritin (ng/ml), transferrin saturation (%) and total iron binding capacity (ug/dl) while iron load was defined as serum ferritin levels above 300 ng/ml. Correlation of iron profile with different stages of CKD was determined applying one-way analysis of variance (ANOVA). Results: In a total 81 children, 46 (56.8%) were boys while overall mean age was 7.79±2.30 years. Mean duration on hemodialysis was 11.52 ± 9.97 months. Iron overload was observed in 26 (32.1%) children. Significant association of age above 7 years (p=0.031) and residential status as rural (p=0.017) was noted with iron overload whereas iron overload was increasing with increase in stages of CKD (p=0.002). Hemoglobin levels decreased significantly with increase in stages of CKD (p<0.001). Serum iron levels increased significantly with increase in the CKD stages (p=0.039). Serum ferritin levels were increasing significantly with the increase in CKD stages (p=0.031). Transferrin saturation also increased significant with increase in CKD stages (p=0.027). Conclusion: High frequency of iron overload was noted in children with CKD on maintenance hemodialysis and there was linear relationship with stages of CKD and iron overload. Significant correlation of hemoglobin, serum iron, serum ferritin and transferrin saturation was observed with different stages of CKD. Keywords: Iron overload, maintenance hemodialysis, ferritin level.


1981 ◽  
Vol 27 (2) ◽  
pp. 276-279 ◽  
Author(s):  
F Peter ◽  
S Wang

Abstract Ferritin values for 250 selected sera were compared with values for iron, total iron-binding capacity (TIBC), and transferrin saturation, to assess the potential of the ferritin assay for the detection of latent iron deficiency. The specimens were grouped (50 in each group) according to their values for iron and TIBC. In Group 1 (low iron, high TIBC) the saturation and ferritin values both indicated iron deficiency in all but one. In the 100 specimens of Groups 2 (normal iron, high TIBC) and 4 (normal iron, high normal TIBC), the saturation values revealed 16 iron-deficient cases, the ferritin test 55. For Groups 3 (low iron, normal TIBC) and 5 (low iron, low TIBC), the ferritin test revealed fewer cases of iron deficiency than did the saturation values (37 cases vs 51 cases, in the 100 specimens). Evidently the ferritin test detects iron deficiency in many cases for whom the serum iron and TIBC tests are not positively indicative. The correlation of serum ferritin with iron, TIBC, and transferrin saturation in the five groups was good only in the case of specimens for which the TIBC was normal; if it was abnormal the correlation was very poor.


Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 3540-3540
Author(s):  
Carole Beaumont ◽  
Jean Delaunay ◽  
Gilles Hetet ◽  
Mariane de Montalembert ◽  
Bernard Grandchamp ◽  
...  

Abstract DMT1 is a divalent metal transporter with 12 transmembrane domains. It is expressed at the apical membrane of duodenal enterocytes, where it mediates pH-dependent uptake of Fe2+. In erythroid cells, it is found in the endosomal membrane where it transfers iron internalized through the transferrin-transferrin receptor pathway from the endosome to the cytosol. The same homozygous G&gt;A substitution resulting in the G185A replacement is responsible for a severe hypochromic microcytic anemia in both the mk mouse and in the Belgrade rat. In humans, a homozygous G&gt;C mutation has been described in a Czech patient, affecting the last nucleotide of exon 12. This mutation leads to the G399A replacement, without affecting the transport function of the protein. However, this mutation also induces a preferential in-frame skipping of exon 12, albeit not in all tissues. Accordingly, the patient has impaired iron acquisition in erythrocytes while duodenal iron absorption is increased leading to progressive iron overload. Here, we report a female patient born in 1996, with low birth weight and hypochromic microcytic anemia (Hb = 7.5 g/dl; MCV = 53 fL). She was transfused at day 0 and put on oral iron treatment. She was then lost to follow-up for five years. At the age of five, more extensive explorations showed a persistent microcytic anemia. The bone marrow displayed normal cellularity, 30% of nucleated cells were erythroid precursors with a moderate maturation defect, acidophilic forms being under-represented as compared to more immature forms. Soluble transferrin receptors were increased (8.3 mg/L; N = 0.83–1.76). Following oral iron therapy, serum ferritin levels remained low (15–25 μg/L; N = 14–197) despite an increase in transferrin saturation from 68 to 95 %. This high transferrin saturation resulted from the combination of reduced transferrin levels (1.64 g/L, N = 2.2–4.0) and increased serum iron levels (35 μmol/L; N = 11–24). On the other hand, hemoglobin raised from 7 to 9 g/dL only through increased number of RBC (5 to 5.7 T/L), since MCV and MCHC remained unchanged. We sequenced the entire transferrin receptor cDNA in this patient and found no mutation. We then sequenced the exons and the intron-exon boundaries of the DMT1 gene and found two heterozygous mutations. One mutation was a deletion of a GTG codon in exon 5, leading to the V114 in-frame deletion, in transmembrane domain 2. The other mutation is a G&gt;T substitution in exon 8 leading to the G212V replacement in transmembrane domain 5. Both parents were asymptomatic, the father being heterozygous for the delV114 mutation and the mother heterozygous for the G212V mutation. This is the second patient described with a neonatal hypochromic microcytic anemia due to DMT1 mutations. Our data suggest that the two combined DMT1 mutations are responsible for the defect in iron utilization by erythroid cells, resulting in persistant microcytosis and impaired red cell maturation. The effect of the mutations on intestinal iron absorption is more difficult to evaluate since iron therapy allowed serum iron and transferrin saturation to increase but serum ferritin remained low and hemoglobin did not reach normal values.


Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 5382-5382
Author(s):  
Rodolfo D Cancado ◽  
Paulo CJL Santos ◽  
Samuel Rostelato ◽  
Cristiane T Terada ◽  
Iris Gonzales ◽  
...  

Abstract Hereditary hemochromatosis (HH) is a disorder characterized by increased intestinal iron absorption, which leads to a progressive accumulation of iron in the body. This iron overload has been associated with mutations in HFE gene (C282Y, H63D and S65C) and other genes. The objectives of this study were to assess the frequencies of functional mutations in HFE and TFR2 genes and to investigate their relationship with the iron status in a sample of blood donors. Blood donors (n=542) were recruited at the Hemocenter of the Santa Casa Hospital, Sao Paulo, Brazil. The genotypes for HFE (C282Y, H63D and S65C) TFR2 (Y250X and Q690P) gene mutations were evaluated by PCR-RFLP. The concentrations of serum iron and total iron-binding capacity (TIBC) were measured by automation system Advia®(Bayer Diagnostics) and serum ferritin by Axsym System®(Abbott Laboratories). The frequencies of HFE 282Y, HFE 63D and HFE 65C alleles were 2.1, 13.6 and 0.6%, respectively. The frequency C282Y allele (2.1%) in Brazilian blood donors is lower than that observed in blood donors from Northern Europe (5.1 to 8.2%, P&lt;0.05). The TFR2 250X and TFR2 690P alleles were not found in these subjects. The iron status was similar between HFE genotypes in women. However, men carrying HFE 282CY genotype had higher serum ferritin and lower TIBC concentrations when compared to the HFE 282CC genotype carriers. HFE 282CY genotype was also associated with higher transferrin saturation in men who donated blood at the first time. Moreover, male donors with HFE 63DD plus 63HD genotypes had higher serum iron and transferrin saturation when compared to those with HFE 63HH genotype. A relationship between HFE CY/HH/SS haplotype and lower TIBC concentrations was also found in men. The HFE 282Y and HFE 65C alleles were rare while the HFE 63D was frequent in blood donors. The mutations in TFR2 gene were not found in this study. The HFE 282Y and HFE 63D alleles were associated with alterations on iron status only in male blood donors.


Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 5145-5145
Author(s):  
Heinz Ludwig ◽  
Georg Endler ◽  
Brigitte Klement ◽  
Wolfgang Hüubl ◽  
Tim Cushway

Abstract Abstract 5145 Introduction and aims: Iron deficiency as a major component in the pathogenesis of anemia in cancer is not acknowledged by most oncologists, possibly except when arising from GI blood loss. Iron deficiency is associated with clinical symptoms such as cognitive impairment, fatigue, and reduced exercise performance. New iron formulations are available that allow rapid iron supplementation with single infusions. This treatment could ameliorate symptoms of iron deficiency and correct anemia. Here, we studied iron parameters and their correlation with erythropoiesis and inflammatory markers in a large unselected cohort of patients with cancer. In addition, we investigated the suitability of serum ferritin and transferrin saturation (TSAT) as parameter for assessment of the iron status. Patients and methods: Data from 1627 patients (median age: 66.4 years, range: 20–97 years) presenting sequentially at the Center for Oncology and Hematology, Wilhelminenspital, Vienna between October 01, 2009 and January 26, 2010, have retrospectively been analyzed. Patients were at different stages of their disease or may not have had an established diagnosis at the time of testing. In patients with multiple testing during this period only the first sample taken was included. TSAT (n=1516), serum ferritin (n=887), serum iron, CRP, and complete blood count, were determined by using standard techniques. Commonly used definitions for absolute iron deficiency (AID), [TSAT <20% and serum ferritin <30ng/ml, in case serum ferritin was not available TSAT <10%] and for functional iron deficiency (FID), [TSAT <20% and serum ferritin ≥30ng/ml, in case serum ferritin was not available TSAT between 10 and 20%] have been applied. Fisher's exact test was used for comparison of frequencies and Pearson's product moment correlation coefficient for evaluation of correlation. Results: Table 1 shows the distribution of TSAT and serum ferritin categories in 1627 patients with cancer. AID was found in 116 patients (7.7%) of the 1516 patients for whom TSAT was available. Eighty-three (72%) of the AID patients presented with anemia (defined by hemoglobin <12g/dl). AID was most common in patients with colorectal and pancreatic cancer (12% and 11%, respectively), and not present in patients with testicular and prostate cancer (p=0.013). FID was diagnosed in 530 patients (35%) and 222 (42%) of them were found to be also anemic. Multivariate analysis revealed a statistically significant correlation between TSAT and serum ferritin (R=0.286, p<0.001), serum iron (R=0.874, p<0.001), hemoglobin (R=0.201, p<0.001) and CRP (R=-0.205, p<0.001) (figure 1). Serum ferritin, in contrast, did not correlate with serum iron (R=0.051, p=0.132), but correlated with hemoglobin (R=-0.259, p<0.001), TSAT (R=0.286, p<0.001), and CRP (R=0.396, p<0.001). Conclusion: AID (7.7%) and even more so FID (35%) are frequent co-morbidities in patients with various types of cancer. Seventy-two percent of patients with AID and 42% with FID presented with overt anemia. TSAT correlated closely with serum iron and hemoglobin levels and seems to be the preferred parameter for assessment of iron status in patients with chronic diseases often complicated by increased inflammation. Serum ferritin was found to be an inadequate parameter for assessment and monitoring of iron status. As iron deficiency has been linked with various symptoms, the question arises whether iron supplementation would benefit patients with FID without overt anemia. Future studies should evaluate the role of novel intravenous iron preparations in ameliorating the symptoms of iron deficiency with or without anemia. Disclosures: Klement: Vifor Pharma Ltd: Employment. Cushway:Vifor Pharma Ltd.: Employment.


Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 3354-3354
Author(s):  
Nicola J Svenson ◽  
Russell Patmore ◽  
Heidi J Cox ◽  
James R Bailey ◽  
Stephen Holding

Abstract Introduction Iron deficiency anaemia (IDA) and anaemia of chronic inflammation (AI) are the most prevalent causes of iron related anaemia in subjects with gastrointestinal disorders contributing significantly to morbidity and mortality. Diagnosis of IDA and AI is not always straight forward and currently a combination of several serum parameters (ferritin, transferrin, transferrin saturation, iron and C-reactive protein) is required. Subjects with a mixed aetiology can be difficult to interpret using traditional serum parameters, particularly in the presence of an inflammatory process. Hepcidin (a 25 amino-acid peptide hormone) in conjunction with reticulocyte haemoglobin equivalent (RetHe) has the potential to differentiate IDA from AI and in cases of mixed aetiology replacing the traditional laboratory parameters (serum iron, CRP, transferrin saturation and ferritin). Aim The aim of the study was to evaluate the performance of a commercially available ELISA assay and investigate whether hepcidin and RetHe can differentiate AI from mixed aetiology. Method The study investigated 77 patients with gastrointestinal disorders associated with anaemia in a secondary care setting using a traditional pathway of 6 tests (figure 1): Complete Blood Count (CBC), Reticulocytes, serum ferritin, CRP, transferrin, serum Iron. Hepcidin concentration was measured using a commercially available ELISA method (DRG Diagnostic GmbH, Marburg, Germany), CBC and RetHe using a Sysmex XE-2100 CBC analyser, iron parameters and CRP using Beckman Coulter platforms. Results Hepcidin correlated well with ferritin R2 = 0.79, p<0.0001. The results were compared to traditional parameters with Receiver Operator Curves (ROC) used to determine diagnostic cut off concentrations (table 1). Table 1. Sensitivity and specificity of serum ferritin and serum hepcidin used to determine diagnostic cut off values. Selected cut off values IDA AI Serum ferritin 30.0µg/L Sensitivity 83% Specificity 64% Sensitivity 55% Specificity 75% Serum hepcidin 8ng/mL Sensitivity 73% Specificity 72% Sensitivity 70% Specificity 67% Serum hepcidin 40ng/mL Sensitivity 98% Specificity 32% Sensitivity 25% Specificity 91% Ferritin was unable to distinguish IDA from AI in mixed aetiology situations. This gives rise to a new proposed 2 step pathway (figure 2) using 3 tests: CBC, RetHe and hepcidin differentiating IDA from AI in mixed aetiology cases indicating the cause of the anaemia. The RetHe value can then be used to predict the response to oral iron. Conclusion Serum hepcidin may not yet replace serum ferritin as the preferred iron status marker, but in conjunction with RetHe it may distinguish mixed aetiology subjects. This offers the potential development of a clearer clinical pathway for investigation of difficult subjects, including reduction in the number of tests required during anaemia investigations and shorter diagnosis times. The advantage of hepcidin together with RetHe over traditional iron parameters is both as a real time marker of iron status and an indication of likelihood of response to iron therapy. The patient would benefit from a shorter recovery time, unnecessary testing, reduction in ineffective treatment and overall reduction in costs. Figure 1. Current diagnostic testing pathway using 6 independent tests with serum ferritin used as the primary indicator of iron stores. Figure 1. Current diagnostic testing pathway using 6 independent tests with serum ferritin used as the primary indicator of iron stores. Figure 2. Suggestion of a new 2 step diagnostic testing pathway with serum hepcidin as the primary indicator and reticulocyte haemoglobin equivalent as the predictor of iron deficiency and response to oral iron. Figure 2. Suggestion of a new 2 step diagnostic testing pathway with serum hepcidin as the primary indicator and reticulocyte haemoglobin equivalent as the predictor of iron deficiency and response to oral iron. Disclosures Patmore: Janssen: Honoraria; Gilead: Honoraria.


Author(s):  
Prabhavathi V. ◽  
Prasad D. K. V. ◽  
Sravani K.

Background: Thyroid dysfunction is a common disorder in pregnancy along with anemia. But no study has evaluated the association between them. To estimate the prevalence of thyroid dysfunction and its association with anemia types in pregnant women during 1st trimester.Methods: Three hundred and eighty pregnant women with <12 weeks of gestational age were selected for the study with no history of thyroid dysfunction and anemia. All the pregnant women were classified into A, euthyroid and B, thyroid dysfunction groups. The B group was again subdivided into hypothyroid, subclinical hypothyroid (SCH), hyperthyroid according to nature of dysfunction. 5 ml of blood sample was collected from all subjects to analyse thyroid hormones and erythrocyte indices.Results: Out of 380 subjects, euthyroid was found to be 77.9%, and rest 22.1% were with thyroid dysfunction. Out of 84 thyroid dysfunction, hypothyroid was found to be 7.9%, SCH 13.9% and hyperthyroid was 0.3%. Out of 296 euthyroid women, anemia was identified in 97 pregnant women (32.8%) whereas in thyroid dysfunction women it was 43 women out of 84 (51.2%) which is a statistically significant. Significantly higher frequency of microcytic hypochromic anemia and normocytic normochromic anemia types were also found in thyroid dysfunction groups compared to euthyroid group (p<0.05). However, no significance between the thyroid dysfunction groups, Statistically significant difference was observed in the Hb concentration, RBC count, MCV, MCH and PCV between euthyroid and different thyroid dysfunction conditions (p<0.05). A statistically significant positive correlation was found between fT4 and erythrocyte indices.Conclusions: As fT4 and TSH correlated with erythrocyte indices, it is advisable to screen for thyroid dysfunction and vice versa so as to prevent the complications associated with anemia and thyroid dysfunction.


2015 ◽  
Vol 55 (1) ◽  
pp. 44
Author(s):  
Desmansyah Desmansyah ◽  
Rini Purnamasari ◽  
Theodorus Theodorus ◽  
Sulaiman Waiman

Background Iron deficiency is considered to be a major public health problem around the world due to its high prevalence as well as its effect on growth, development, and infection-resistance in children. In malaria-endemic areas, malaria infection is thought to contribute to the occurrence of iron deficiency, by means of hepcidin and hemolysis mechanisms. Objective To assess the prevalence of asymptomatic vivax malaria, compare hemoglobin levels and iron status parameters between vivax malaria-infected and uninfected children, assess the prevalence of iron deficiency, and evaluate a possible correlation between vivax malaria infection and iron deficiency. Methods This cross-sectional study was conducted from February to April 2013 at Sanana City of Sula Islands District, North Maluku. Six parameters were evaluated in 5-11-year-old children: malaria parasite infection, hemoglobin level, serum iron concentration, total iron-binding capacity (TIBC), serum transferrin saturation, and serum ferritin concentration. Results Among 296 children aged 5-11 years, 75 (25.3%) were infected with Plasmodium vivax. In infected children, hemoglobin, serum iron, transferrin saturation, TIBC and serum ferritin were significantly lower than in non-infected children (P<0.01). Using a serum ferritin cut-off of <15 μg/dL, 142 (48.0%) of the children were found to be iron deficient. There was a strong correlation between vivax malaria infection and iron deficiency (OR 3.573; 95%CI 2.03-6.29). ConclusionThe prevalence of asymptomatic vivax malaria infection was 25.3%. The hemoglobin level and iron status parameters in vivax malaria-infected subjects were significantly lower than in uninfected children. The prevalence of iron deficiency was 48.0% for all study subjects. Malaria vivax infection was correlated with iron deficiency in 5-11-year-old children at Sanana City.


Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 4846-4846 ◽  
Author(s):  
Peter L. Greenberg ◽  
Charles A. Schiffer ◽  
Charles Asa Koller ◽  
Barinder Kang ◽  
Jodie Decker ◽  
...  

Abstract Introduction: Approximately 60% of patients with myelodysplastic syndromes (MDS) require ongoing red blood cell transfusions, which can lead to significant iron overload and associated morbidities. Historically, many of these patients have not received iron chelation therapy due to burdensome administration of deferoxamine. Deferasirox (Exjade®, ICL670) is a once-daily, oral iron chelator recently approved for the treatment of chronic iron overload due to blood transfusions. This ongoing study is designed to evaluate the efficacy and safety of deferasirox in Low/Int-1-risk MDS patients. In addition, this is the first prospective, multicenter trial to evaluate liver iron concentration (LIC) using the MRI R2 parameter in this population. Methods: This ongoing study will enroll 30 patients at three US centers. Deferasirox will be administered at 20–30 mg/kg/day for 12 months. Iron burden is being monitored by monthly serum ferritin evaluations, and LIC by MRI R2 at baseline, 6 and 12 months. Serum iron, transferrin, transferrin saturation, labile plasma iron (LPI), and urinary hepcidin are being assessed throughout the study. In addition, serum creatinine, calculated creatinine clearance, echocardiograms and hematological status are being monitored. In this report, we are presenting the baseline data for the currently enrolled patients. Results: As of May 2006, 14 patients (9 male, 5 female; aged 55–81 years) were enrolled. All patients were Caucasian with equal distribution of Low- and Int-1-risk MDS. The mean interval from MDS diagnosis to screening was 4 years, ranging from &lt;1 to 12 years. The table summarizes baseline iron parameters in these patients: Parameter n Mean ± SD Median Range Normal range n/a, not applicable LIC, mg Fe/g dw 14 21.8 ± 11.0 23.5 3.8–40.5 &lt;1.3 Serum ferritin,μg/L 14 4645 ± 3804 3534.5 1433–15380 20–360 Serum iron, μg/dL 14 205.9 ± 26.5 200 165.9–252.0 50–160 Transferrin, mg/dL 14 143 ± 19 142.5 106–172 200–400 Transferrin saturation, % 14 113.8 ± 8.5 114 95–124 15–50 LPI, μmol/L 14 0.7 ± 0.7 0.6 0–1.9 0 Num. of lifetime transfusions 14 106.3 ± 115.5 47.5 30–352 n/a Renal function: Calculated creatinine clearance at baseline was normal (&gt;80 mL/min) in 46% of patients, mildly impaired (50–80 mL/min) in 46% and moderately impaired (30–50 mL/min) in 8% of patients. Hematological parameters: neutropenia (&lt;1800/μL): 1 patient; thrombocytopenia (&lt;100,000/μL): 3 patients; neutropenia and thrombocytopenia: 1 patient. Concurrent therapies: Revlimid: 2 patients; and hydroxyurea: 1 patient. Conclusions: Baseline iron burden in these patients demonstrates a high degree of iron overload, as measured by LIC via MRI, as well as serum ferritin, serum iron and transferrin saturation. Based on NCCN guidelines for the management of iron overload, the degree of iron overload observed meets criteria for treatment. This ongoing study is assessing the safety and efficacy of deferasirox in this population.


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