Deferasirox in children with transfusion-dependent thalassemia or sickle cell anemia: A large cohort real-life experience from Turkey (REACH-THEM).

OBJECTIVES: To evaluate the long-term efficacy and safety of deferasirox therapy in a large observational cohort of children with transfusion-dependent thalassemia (TDT) and sickle cell anemia (SCA) in Turkey. METHODS: This was a multicenter, prospective cohort study including TDT and SCA patients aged 2-18 years with iron overload (≥100 mL/kg of pRBC or a serum ferritin [SF] level >1000 µg/L) receiving deferasirox. Patients were followed for up to 3 years according to standard practice. RESULTS: A total of 439 patients were evaluated (415 [94.5%] TDT, 143 [32.6%] between 2 and 6 years). Serum ferritin levels consistently and significantly decreased across 3 years of deferasirox therapy from a median of 1775.5 to 1250.5 µg/L (P < 0.001). Serum ferritin decreases were noted in TDT (1804.9 to 1241 µg/L), SCA (1655.5 to 1260 µg/L), and across age groups of 2-6 years (1971.5 to 1499 µg/L), 7-12 years (1688.5 to 1159.8 µg/L), and 13-18 years (1496.5 to 1107 µg/L). Serum ferritin decreases were also noted for all deferasirox dose groups but only significant in patients with doses ≥30 mg/kg/d (n = 120, -579.6 median reduction, P < 0.001). Only 9 (2%) patients had adverse events suspected to be related to deferasirox. Serum creatinine slightly increased but remained within the normal range. CONCLUSIONS: Deferasirox has long-term efficacy and safety in children with TDT and SCA, although higher doses (≥30 mg/kg/d) may be required to achieve iron balance.

Impaired Hemorheological Parameters and Increased Carotid Intima-Media Thickness in Children with Subclinical Hypothyroidism.

BACKGROUND: Subclinical hypothyroidism (SH) is defined as elevated serum thyroid-stimulating hormone (TSH) concentration associated with normal serum-free thyroxine levels. Effects of hypothyroidism on hemorheology had widely attracted the attention of researchers during the last decade. OBJECTIVE: The purpose of this study is to determine alterations in hemorheological parameters and carotid intima-media thickness (CIMT) in children with SH. METHODS: Fifty-three SH children and 31 healthy controls were enrolled. Erythrocyte deformability and aggregation were determined by an ektacytometer and plasma viscosity (PV) by a cone-plate rotational viscometer. CIMT was evaluated sonographically. RESULTS: Erythrocyte deformability of the SH group measured at 0.53 and 1.69-30 Pa was lower than that of the control group. The erythrocyte aggregation index, aggregation half time and PV were not different between the groups. However, the aggregation amplitude and mean corpuscular hemoglobin concentration were significantly higher in SH compared to the control group. There was a negative correlation between TSH and deformability values measured at 5.33-30.0 Pa. CIMT in patients with SH was significantly higher than in the control group (p = 0.001; SH = 0.48 ± 0.04 mm, control group = 0.43 ± 0.03 mm). CONCLUSION: Impaired hemorheology and increased CIMT are well-known risk factors for developing cardiovascular pathologies. The results of the current study suggest the treatment of children with SH in order to avoid early circulatory problems.

Pentraxin-3 Levels in Beta Thalassemia Major and Minor Patients and Its Relationship With Antioxidant Capacity and Total Oxidant Stress.

Thalassemia major (TM) results in hemolytic anemia, an increase in intestinal iron absorption, and occurrence of iron loading due to erythrocyte transfusion; the disease is characterized by oxidative damage in major organs. Oxidative stress leads to vascular endothelial damage and forms the basis for serious cardiovascular diseases. Pentraxin-3 (PTX-3) is one of the markers of vascular endothelial damage that increases in response to the oxidative stress, which can be used as an early diagnostic marker for inflammation. This study's purpose is to define the relation between PTX-3 and the vascular endothelial damage that increases with oxidative stress in thalassemia patients. Our study included 35 TM patients, 30 ß-thalassemia minor patients, and 30 healthy children. As a result of our study, in TM patients, a positive relation was detected between the PTX-3 levels and the total oxidative stress, triglyceride, and very low-density lipoprotein values, whereas a negative relation was detected with the total antioxidant capacity and high-density lipoprotein values. This result shows that as oxidant stress increases, PTX-3 levels also increase; very low-density lipoprotein and triglyceride contribute to the endothelial damage occurring with oxidative stress. As a result, it was concluded that vascular endothelial damage in thalassemia patients can be evaluated through the serum PTX-3 level.

Investigation of hemorheological parameters at the diagnosis and follow up of children with iron deficiency anemia and mixed anemia.

OBJECTIVE: We aimed to investigate the effects of iron deficiency anemia (IDA) and vitamin B12 deficiency coexisting with IDA which is called as mixed anemia (MA) on hemorheological parameters, to compare them with each other and healthy controls, and to assess the changes in hemorheological parameters after treatment. MATERIALS AND METHODS: 32 IDA patients (mean age:6.3 ± 5.3 years), 30 MA patients (mean age:7.2 ± 5.4 years), and 31 healthy controls (mean age:7.1 ± 5.2 years) were enrolled. Erythrocyte deformability and aggregation were determined by an ectacytometer, plasma and whole blood viscosities by a cone-plate rotational viscometer. Differences between IDA and MA, and healthy controls were compared. Hemorheological parameters were repeated in the patient groups after treatment and compared with the initial results. RESULTS: In both of the patient groups, erythrocyte deformability, whole blood and plasma viscosities were found to be significantly decreased before treatment, compared with the controls. After treatment these parameters were found to be increased significantly. There were no significant differences in these parameters between the IDA and MA group. Additionally, no statistically significant alteration was found in erythrocyte aggregation measurements of both patient groups. CONCLUSION: This study indicates that IDA and MA have similar effects on hemorheological parameters. When vitamin B12 deficiency accompanies IDA which is called as MA, no further alterations occur in hemorheological parameters. The adequate treatment of these anemias not only corrects the hematological parameters, but also by helping to normalize the hemorheological parameters, may contribute to the regulation of microvascular perfusion.

Investigation of hemorheological parameters at the diagnosis and the follow-up of nutritional vitamin B12 deficient children.

We aimed to investigate the effects of vitamin B12 deficiency on hemorheological parameters, and the changes in these parameters following vitamin B12 treatment. 33 patients (mean-age:7 ± 5.7 years) diagnosed as nutritional vitamin B12 deficiency, and 31 age and sex matched controls (mean-age:7.1 ± 5.2 years) were enrolled. Erythrocyte deformability and aggregation were determined by an ectacytometer, plasma and whole blood viscosities by a cone-plate rotational viscometer. The differences between patients and controls were compared. Hemorheological parameters were repeated in the patient group following vitamin B12 treatment, and the results were compared with the initial results. In vitamin B12 deficiency, erythrocyte deformability and whole blood viscosity were found to be significantly decreased, eythrocyte aggregation was found to be significantly increased compared with the controls. Plasma viscosity was found to be decreased in deficiency but this decrease was not statistically significant. In patient group, erythrocyte deformability, whole blood and plasma viscosities were found to be significantly increased and erythrocyte aggregation was significantly decreased, after treatment. This study indicates that vitamin B12 deficiency has important effects on hemorheological parameters and adequate treatment of deficiency not only corrects the hematological parameters, but also by helping to normalize the hemorheological parameters, may contribute to the regulation of microvascular perfusion.

Prevalence and Risk Factors of Anemia among Adolescents in Denizli, Turkey.

OBJECTIVE: The aim of this study was to evaluate the prevalence and risk factors of anemia among adolescents in Denizli where Mediterranean cuisine (fresh fruit and vegetables) is adopted. METHODS: We accepted hemoglobin values below 12 g/dl for girls and 13 g/dl for boys as the criteria of anemia. We recorded a detailed history including nutritional habits, consumption of animal source foods especially red meat, consumption of fresh fruit and vegetables, presence of parasitic infestation, psychosocial status, school success and any materials consumed except ordinary food. FINDINGS: We investigated the prevalence of anemia in 1120 children (672 girls and 448 boys), aged 12 to 16 years. We found that the overall prevalence of anemia was 5.6%. 8.3% of the girls and 1.6% of the boys were anemic. We diagnosed iron deficiency anemia in 37(59%) anemic patients and combined iron deficiency and vitamin B12 deficiency anemia in 26 (41%) anemic patients. None of the patients had folic acid deficiency. CONCLUSION: Our results suggest that the socioeconomic status of the family, traditional eating habits of the region, the fear of gaining weight and irregular eating habits are of great importance in the development of adolescent anemia in Denizli.

Presacral and intrathoracic extramedullary hematopoiesis: a case report.

Extramedullary hematopoiesis (EMH) refers to the location of hematopoietic elements in locations other than the bone marrow and peripheral blood. It may be seen as a compensatory condition in many hematological conditions including thalassemia, or it may occur as an incidental finding. Intrathoracic EMH commonly develops in the posteroinferior mediastinum. Presacral EMH is an extremely rare condition, and there are a limited number of case reports published in the literature. We are reporting a case of EMH that involves the posterior mediastinum and presacral region on computed tomography and magnetic resonance imaging in a patient with thalassemia intermedia.

Conjunctival melanoma in a six-year-old female.

Conjunctival melanoma (CM) is a highly malignant tumor that derives from melanocytes and is rarely seen in children. This report describes a 6-year-old female diagnosed with CM.