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Objectives: The aim of this study was to determine the anthropometric measurements in transfusion-dependent β-thalassemia children in Pakistan. The secondary aim was to correlate serum ferritin with the physical growth. Methods: We enrolled 367 children (aged 5-17 years) with transfusion-dependent beta-thalassemia major in the study. Anthropometric measurements, serum ferritin levels, and pre-transfusion hemoglobin levels were measured. Serum ferritin was correlated with the height z-score for age. Results: Laboratory evaluation showed that patients had significantly low mean pre-transfusion hemoglobin of 7.66 ± 1.34 g/dl (range 2.5-10.5) and high median (Q3-Q1) serum ferritin of 5012 ng/ml (6829-3532). The median (Q3-Q1) height-for-age z-score of children was low at −2.69 and (−1.46 to −3.80) and 65.4% children had stunted growth (height for age z-score <−2). There was a significant negative correlation between height for age z-score and serum ferritin levels (p < 0.000). Stunting of growth began early during 5-10 years of age but increased markedly with the progress of time. Conclusions: The study showed that children with beta thalassemia major had delayed physical growth possibly secondary to iron overload. Effective and early iron chelation is needed for preventing growth failure in transfusion-dependent beta thalassemia. [ABSTRACT FROM AUTHOR]

Background: Blood transfusion are needed in improving the quality of life of thalassemia major patients. However since it can lead to excess iron, the iron chelation therapy is needed. Deferiprone and deferasirox are the most often used therapy in Indonesia. Objective: The aim of this study was to compare the cost-effectiveness of deferasirox with deferiprone with costeffectiveness analysis (CEA). Methods: Data were taken retrospectively and sampling was done using total sampling based on medical records and hospital information system. Serum ferritin levels of patients consuming deferasirox (n=27) and deferiprone (n=33) were measured to observe the mean changes of serum ferritin levels as effectiveness' parameter. The cost was median of the total direct medical cost, summed from the cost of drugs, medical devices, hospitalization, administration, physician, laboratories and blood bags. Results: Based on the results of this study, the effectiveness of deferasirox (1164 ng/mL) was greater than deferiprone (692 ng/mL). Median total cost of deferasirox was more expensive (Rp 76,610,618.69) than deferiprone (Rp 51,869,965.64). Cost-effectiveness ratio of deferasirox (CER: Rp 65,816.68/effectiveness) was lower than deferiprone (CER: Rp 74,956.60/ effectiveness). None of both medications was dominant and therefore we could not determine which medication was the most cost-effective therapy. Changing of medication from deferiprone to deferasirox requires an extra cost of Rp 52,416.64 per one incremental unit of effectivity. Conclusion: The policy maker in healthcare facility need to consider the budget and whether the incremental cost of deferasirox is proportional to its increased effectiveness. [ABSTRACT FROM AUTHOR]

In iron overload diseases (thalassemia, sickle cell, and myelodysplastic syndrome), iron is deposited in all internal organs, leading to functional abnormalities. Hematopoietic stem cell transplantation (HSCT) is the only treatment offering a potential cure in these diseases. Our aim was to describe the experience in the field and the role of magnetic resonance imaging in the evaluation of iron overload before and after HSCT. Magnetic resonance imaging (MRI), using T2*, is the most commonly used tool to diagnose myocardial-liver iron overload and guide tailored treatment. Currently, HSCT offers complete cure in thalassemia major, after overcoming the immunologic barrier, and should be considered for all patients who have a suitable donor. The overall thalassemia-free survival of low-risk, HLA-matched sibling stem cell transplantation patients is 85-90%, with a 95% overall survival. The problems of rejection and engraftment are improving with the use of adequate immunosuppression. However, a detailed iron assessment of both heart and liver is necessary for pre- and post-transplant evaluation. In iron overload diseases, heart and liver iron evaluation is indispensable not only for the patients' survival, but also for evaluation before and after HSCT. [ABSTRACT FROM AUTHOR]

Aims Cardiovascular magnetic resonance (CMR) has dramatically changed the clinical practice in thalassemia major (TM), lowering cardiac complications. We prospectively reassessed the predictive value of CMR parameters for heart failure (HF) and arrhythmias in TM. Methods and results We considered 481 white TM patients (29.48 ± 8.93 years, 263 females) enrolled in the Myocardial Iron Overload in Thalassemia (MIOT) network. Myocardial and liver iron overload were measured by T2* multiecho technique. Atrial dimensions and biventricular function were quantified by cine images. Late gadolinium enhancement images were acquired to detect myocardial fibrosis. Mean follow-up was 57.91 ± 18.23 months. After the first CMR scan 69.6% of the patients changed chelation regimen. We recorded 18 episodes of HF. In the multivariate analysis the independent predictive factors were myocardial fibrosis (HR = 10.94, 95% CI = 3.28--36.43, P < 0.0001), homogeneous MIO (compared with no MIO) (HR = 5.56, 95% CI = 1.37--22.51, P = 0.016), ventricular dysfunction (HR = 4.33, 95% CI = 1.39--13.43, P = 0.011). Arrhythmias occurred in 16 patients. Among the CMR parameters only the atrial dilation was identified as univariate prognosticator (HR = 4.26 95% CI=1.54-11.75, P = 0.005). Conclusions CMR guided the change of chelation therapy in nearly 70% of patients, leading to a lower risk of iron-mediated HF and of arrhythmias than previously reported. Homogeneous MIO remained a risk factor for HF but also myocardial fibrosis and ventricular dysfunction identified patients at high risk. Arrhythmias were independent of MIO but increased with atrial dilatation. CMR by a multi-parametric approach dramatically improves cardiac outcomes and provides prognostic information beyond cardiac iron estimation. [ABSTRACT FROM AUTHOR]

Sickle cell disease and homozygous β-thalassaemia are common haemoglobinopathies in Oman, with many implications for local healthcare services. The transfusions of such patients take place in many hospitals throughout the country. Indications for blood transfusions require local recommendations and guidelines to ensure standardised levels of care. This article summarises existing transfusion guidelines for this group of patients and provides recommendations for blood banks and transfusion services in Oman. This information is especially pertinent to medical professionals and policy-makers developing required services for the standardised transfusion support of these patients. [ABSTRACT FROM AUTHOR]

We aimed to evaluate the role of two-dimensional speckle tracking imaging (2DSTI) in detecting early changes of myocardial deformation in patients affected by thalassemia major (TM) and its relation with myocardial iron overload (MIO) detected by T2* cardiovascular magnetic resonance (CMR). We studied 28 TM patients (15 males, 37.4 ± 10 years). All patients underwent CMR and echocardiography in the same day. Segmental and global T2* values were measured. Values of global longitudinal strain (GLS) were derived from the three apical views, while radial and circumferential strain were obtained as average strain from the short axis views at basal, mid and apical level. Six patients (21.4%) showed significant MIO (global heart T2* < 20 ms). GLS showed a significant correlation with T2* values (R = -0.49; P = 0.001) and it was significantly lower in patients with a significant MIO than in those with no significant MIO (-18.3 ± 2 vs. -21.3 ± 2.7, P = 0.02). No significant difference was found for radial and circumferential strain in relation to the severity of MIO. Patients with impaired GLS (<-19.5%) had a significant higher risk of showing significant MIO (Odds-ratio-OR = 17; 95%). GLS is related with global T2* in TM patients. Moreover, GLS can identify TM patients with severe MIO detected by CMR. [ABSTRACT FROM AUTHOR]

Background and Aim: Thalassemia is a chronic disease which is extremely expensive, complex and debilitating. The management skill of thalassemia patients should be enhanced to minimize the risk of disease complications. The main purpose of this study was to develop personal electronic health records for thalassemia major patients. Materials and Methods: This is a developmental applied study which was conducted to develop a personal electronic health record for thalassemia major. First, a questionnaire was prepared to determine the data elements and was filled by Hematology and Oncology professionals in the country (110 persons). Then, based on the results of needs analysis, the system was designed using PHP programming language and MySQL database and was evaluated by 50 thalassemia patients who referred to the Thalassemia Clinic of Bu Ali Sina Hospital of Mazandaran University of Medical of Sciences during the second half of the month of Aban. Finally, a standard questionnaire of usability and user satisfaction assessment was distributed among them. Results: Usability evaluation of the system showed that patients evaluated the system at a good level with a mean rating of 7.91 (out of 9 points). Conclusion: The web-based systems can be used to help thalassemia patients to control injection and reduce the complications of the disease and to promote health. [ABSTRACT FROM AUTHOR]

Copyright of Kirkuk University Journal for Scientific Studies is the property of Republic of Iraq Ministry of Higher Education & Scientific Research (MOHESR) and its content may not be copied or emailed to multiple sites or posted to a listserv without the copyright holder's express written permission. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)

Background The life of patients with thalassemia major depends on blood transfusions, while repeated blood transfusions may cause adverse effects such as iron deposition in various organs, includ- ing heart and lungs, which eventually increases the pulmonary arterial pressure. Objective This study was proposed to know the occurrence of pulmonary hypertension in patients with thalassemia major, mea- sured by echocardiography in the Thalassemia Clinic, Department of Child Health, Medical School, Padjadjaran University/Hasan Sadikin Hospital, Bandung. Methods A descriptive cross-sectional study was carried out on 30 patients with thalassemia major, aged 10-14 year-old who received repeated blood transfusions. The study was conducted from April to May 2002. Subjects were examined right after a blood transfusion completed and the pulmonary arterial pres- sure was assessed using Doppler–echocardiography and 2-D echocardiography. Results Twenty two out of 30 subjects showed pulmonary hyper- tension, with pulmonary arterial pressure ranged between 32.3 to 46.2 mmHg. According to the age group, pulmonary hypertension was found in 12 out of 17 subjects aged 10-12 years old and 10 out of 13 subjects aged 13-14 years old. Conclusion The occurrence of pulmonary hypertension in patients with thalassemia major at Hasan Sadikin Hospital was 22/30 and seemed to increase with the age of the patients

Thalassemia is associated with several challenging comorbidities. Here we report a 20 year old thalassemic who presented with cauda equina syndrome due to paraspinal extra medullary hematopoiesis (EMH) and was treated with hydroxyurea, repeated blood transfusions, and radiotherapy. Thus compressive myelopathy due to EMH was successfully managed conservatively.

Abstract Spinal cord compression due to extramedullary hematopoiesis (EMH) is a rare complication of thalassemia and generally presents as paraparesis with sensory impairment. Complete paraplegia is extremely rare in EMH due to thalassemia although it is known to occur in polycythemia vera and sickle cell anemia. Cases presenting with paraparesis have been treated with either surgery or radiotherapy with equal frequency and efficacy. Almost all reported cases with paraplegia have been treated with surgery with or without radiation therapy. As paraplegia secondary to EMH is rare, there is no consensus on treatment. We hereby report a case of thalassemia with paraplegia treated successfully with surgery. Treatment options for cord compression include primarily blood transfusion, surgery, and radiation therapy. Because of the extreme rarity of this condition, direct comparisons between various treatment modalities are not possible. The bias toward surgery is due to its immediate decompressing effect. EMH in thalassemia has also been treated with transfusion therapy with the rationale that correction of anemia would downregulate erythropoietin and lead to reversal of EMH. However, improvement with blood transfusion alone is usually incomplete and slow. Our patient did not improve after adequate blood transfusion and hence underwent surgical decompression after which he showed rapid and complete neurologic recovery. Therefore, we conclude that surgical decompression of EMH tissue has to be considered early in patients presenting with paraplegia if symptom shows no improvement after adequate blood transfusion.

INTRODUCTION Thalassemia is a heterogeneous group of gene disorders caused byan inherited mutation or deletion of genes at chromosome 16 & 11 resulting in decreased synthesis of adult hemoglobin. Its incidence is higher in people/children having a positive family history of Thalassemia Among its various types Beta Thalassemia major is the one which requires regular blood transfusions. OBJECTIVES The main objective of the study was to determine the frequency of different types of complications in patients suffering from B-Thalassemia. METHODOLOGY The study was conducted in two private originations in Peshawar i.e. Fatimid Foundation and Hamza Foundation.150 patients) were studied, the study was Cross-Sectional, descriptive type and sampling type was Non-Probability, convenience type sampling. All necessary data were obtained by using semi structured questionnaire, having both closed and open ended questions. Pilot Study was conducted on 15 patients (10%of target questionnaires). After completing the pilot study necessary changes were made in questionnaire and a well-informed, well-designed and organized questionnaire was finalized. An informed consent was taken from respondents and the responses were obtained each from the parents & from children themselves (in case of older children). Data was collected and results were presented in the form of tables and charts. Manual analysis of the data was carried out. RESULTS According to the study, among 150 patients, Splenectomy was done in about 86% patients. Consanguinity among the parents of these patients was about 76%, while awareness only 72% of patients. About 64% patients were having psychological problem. Among neurological problems 57% of patients had headache. Among complications, majority of patients had palpitations about 54% joint pains about 32% & history of bones fractures about 21%. Socioeconomic condition of majority of them was poor about 52%. All of them were taking primary treatment i.e. Blood transfusion &Chelating Therapy to prevent iron overload. CONCLUSSIONS Thalassemia major is a life threatening disease causing morbidity and mortality among those who are not treated properly. The incidence of the disease is high among those with a family history of Thalassemia.

Purpose ― We performed a meta-analysis of all eligible case-control studies published to date, to assess the association of salivary ferritin and iron with hematological disorders. Methods ― We conducted Medline/PubMed and Scopus searches for papers published from January 1, 1980, through January 1, 2013. Data were merged the summary mean difference were estimated using either a random-effects model or a fixed-effects model. Results ― There were 3 studies including 196 cases and 70 controls. There was a statistically significant difference in the salivary ferritin and iron between hematological diseases and control groups, and the summary estimate of mean difference was 1.37 (95%CI: 0.01–2.74) and 2.23 (0.92–3.54) in salivary ferritin and iron, respectively. The stratification showed the same statistically significant differences in the salivary ferritin between Iron deficiency anemia (IDA) with thalassemia intermedia (TI), and IDA with thalassemia major (TM) groups. Besides, the stratification showed that there were statistically significant differences in the salivary iron between IDA with TM groups, while no statistically significant difference was observed between IDA with TI. Conclusion ― Saliva ferritin iron and ferritin increase significantly in patients with thalassemia compared to IDA. Salivary ferritin and iron can be used as a diagnostic marker and new discriminating indices routinely to differentiate IDA from thalassemia, especially thalassemia trait; furthermore, their easy applicability, safe, relatively simple, and noninvasive procedure may be an important advantage compared to blood sample collection. Consequently, it could be promising to develop a simplified testing and differentiating procedure, which could reach many clinical decision-makers as a diagnostic potential.

Background and Objective: The people with Thalassemia, because of frequent blood transfusion and sever complication ,require constant medical supervision, and their psychological problems are paramount. This study aimed to determine Self-Concept and related factors in children and adolescents with Thalassemia major aged 7-18 in Golestan province ,2012. Material and Methods: This cross-sectional study was conducted on 155, using census sampling, children and adolescents with Thalassemia major refereeing to therapeutic and health centers for blood transfusion. The instruments were Piers-Harris self-concept questionnaire and demographic data . The analysis was performed by SPSS software using ANOV and t-test. Results: The mean of self-concept was low, and there was significant relationship between self-concept and variables such as age, educational status, dropout , order of birth, the number of children in family and parents’ education level. But no significant difference was observed between self-concept and the factors such as sport, the frequency of transfusion, economic status. Conclusion: The results revealed that the self-concept of the thalassaemic patients is low. Some non-adjustable factors exacerbate this condition, but encouraging them to continue their education, informing their parents more about their children’s disease can be helpful to improve their self-concept.

Thalassemia major is a genetic blood disorder that is detected by the symptoms of chronic and severe anemia, failure to thrive, an enlarged liver and spleen, bone deformities particularlya deformed face and bulging forehead. Due to changes in physical appearance, the disease can influence on other aspects of the patient's life, so the disease could strongly impact on the mental health of these patients and their families. Previous studies show that 80% of patients with thalassemia major have at least one psychiatric disorder. The aim of this paper is to review the mental health status of patients with Beta-thalassemia major in Iran.

AIM: To investigate ophthalmologic findings in children with thalassemia major (TM) and compare the findings with healthy controls. METHODS: In a cross-sectional study, 43 children with thalassemia major from pediatric hematology outpatient clinics from two university hospitals and age/sex matched 47 healthy children were included in the study. After a complete ophthalmic examination, tear function tests including the Schirmer test, fluorescein tear break-up time (BUT), ultrasound pachymetry, and axial length measurement were performed. Obtained data was recorded for statistical analysis and the values of right eyes were compared between groups. RESULTS: The mean best corrected visual acuity was 1.34±0.75 in TM and 1.08±0.28 in controls. It was found lower than 0.1 logMAR unit in 10 (23.2%) children with TM and 2 (4.2%) in controls, and the difference was statistically significant (P＜0.05). The mean central corneal thickness was 540±26.95 in children with TM and 536.98±20.45µm in controls (P＞0.05). The mean axial length was 22.53±0.50 in TM and 22.57±0.43mm in the control group. The mean Schirmer test score was 19.94±6.91 in TM and 24.22±3.95mm in the control group (P＜0.01). The mean BUT score was 9.62±1.28 in TM and 9.73±0.6s in the control group (P＞0.05). CONCLUSION: In TM, while corneal thickness, axial length, and BUT are close to controls, the Schirmer scores are less than normal. The study revealed that TM may be affected by the tear function and visual acuity.

Background: Diabetes mellitus is a major complication of iron overload in patients with beta thalassemia major. Design: This is a descriptive study conducted in a Tertiary Care Teaching Hospital to analyze beta cell function and insulin resistance, and their relation to iron overload status in beta thalassemia major. Fasting glucose, two-hour post load glucose, fasting insulin, alanine amino transaminase (ALT), and ferritin were used as outcome measures. The homeostatic model assessment (HOMA model) was used to calculate the beta cell function and insulin resistance index. Results: Of the 30 cases, 20% had impaired fasting glucose, 3.3% had impaired glucose tolerance, and none had diabetes. Fasting glucose was not significant between the cases and controls (P = 0.113). Fasting insulin (P = 0.001), ferritin (P = 0.001), and ALT (P = 0.001) levels were significantly high in the cases. Insulin resistance index was significantly higher in the cases (P = 0.001) as also the beta cell function (P = 0.001). With increase in age and the number of units transfused there is a decline in beta cell function, fasting insulin, and insulin resistance after attaining the maximum level. This suggests that initial insulin resistance is followed by insulin depletion due to loss of beta cell function, leading to diabetes mellitus. Conclusion: Impaired glucose tolerance (IGT) and insulin resistance precede the onset of insulin-dependent diabetes and adequate chelation therapy is essential for delaying the onset or for prevention of diabetes.

Background: The development of anti-red blood cell antibodies (both allo-and autoantibodies) remains a major problem in thalassemia major patients. We studied the frequency of red blood cell (RBC) alloimmunization and autoimmunization among thalassemia patients who received regular transfusions at our center and analyzed the factors, which may be responsible for development of these antibodies. Materials and Methods: The study was carried out on 319 multiply transfused patients with β-thalassemia major registered with thalassemia clinic at our institute. Clinical and transfusion records of all the patients were examined for age of patients, age at initiation of transfusion therapy, total number of blood units transfused, transfusion interval, status of splenectomy or other interventions. Alloantibody screening and identification was done using three cell and 11 cell panel (Diapanel, Bio-rad, Switzerland) respectively. To detect autoantibodies, autocontrol was carried out using polyspecific coombs (IgG + C3d) gel cards. Results: Eighteen patients out of total 319 patients (5.64%) developed alloantibodies and 90 (28.2%) developed autoantibodies. Nine out of 18 patients with alloantibodies also had autoantibodies. Age at first transfusion was significantly higher in alloimmunized than non-immunized patients (P = 0.042). Out of 23 alloantibodies, 52.17% belonged to Rh blood group system (Anti-E = 17%, Anti D = 13%, Anti-C = 13%, Anti-C w = 9%), 35% belonged to Kell blood group system, 9% of Kidd and 4% of Xg blood group system. Conclusion: Alloimmunization was detected in 5.64% of multitransfused thalassemia patients. Rh and Kell blood group system antibodies accounted for more than 80% of alloantibodies. This study re-emphasizes the need for RBC antigen typing before first transfusion and issue of antigen matched blood (at least for Rh and Kell antigen). Early institution of transfusion therapy after diagnosis is another means of decreasing alloimmunization.

Aim: Endocrinopathies are common in patients with thalassemia major (TM) and affect their life quality. Our aim was to identify the frequency of growth retardation and endocrine complications in these patients. Materials and Methods: Sixty-two patients aged 3-18 years with TM were evaluated retrospectively for height, weight, body mass index (BMI), and pubertal stage. Blood tests for endocrine function, and oral glucose tolerance test (OGTT) results were recorded. Results: The mean age of 62 subjects (33 females/29 males) was 10.4±3.9 years. The frequency of ≤-2 standard deviation scores was 37.1% for height, 33.9% for weight and 11.3% for BMI. Short stature, being underweight, and low BMI were significantly more prevalent in children over 7 years old (p<0.001). Delayed puberty/hypogonadism was present in 37% of 19 adolescents. Thirteen percent of the subjects had vitamin D deficiency (<10 ng/mL), hyperparathyroidism was observed in 29% of the subjects, while subclinical hypothyroidism (thyroid-stimulating hormone 5-10 IU/mL) was determined in 3 (5.5%) of the 55 subjects. In OGTT, impaired fasting glucose was seen in 7 subjects (14.5%), impaired glucose tolerance in 3 (6.3%), diabetes mellitus in 1 (2.1%), and hypoglycemia at 120-min was observed in 5 subjects (10.4%). Overall, 67.7% of the 62 subjects had height standard deviation score ≤-2 and/or at least one endocrinopathy. Conclusion: Growth retardation and endocrine problems are still a serious problem in TM patients, and develop particularly in those older than 7 years. Additionally, attention must be paid to hypoglycemia in these patients as well as diabetes. [ABSTRACT FROM AUTHOR]

Objective: To compare the mean serum ferritin levels in thalassaemia major patients after giving deferasirox and deferoxamine. This is a randomised control trial conducted at the Department of Paediatrics, Civil Hospital, Karachi from 29th January 2014 to 28th July 2014. Methods: A total of 160 patients of either gender, with age between 1 to 14 years, who received blood transfusion at least once a month for one year and had serum ferritin >1000 mcg/L were included. Each enrolled patient was randomly allocated to group-A (deferoxamine) or group-B (deferasirox). P-value post-treatment iron profile was done in both groups to assess the iron status in the body. Descriptive statistics were applied to calculate mean and standard deviation for the quantitative variables. Frequencies and percentages were calculated for the qualitative variables. Independent sample t-test was applied to compare mean change in serum ferritin level in both groups. Effect modifiers were controlled by stratification. Paired t-test was also applied post stratification and p-value 0.05 was considered as significant. Results: Overall there were 96 male and 64 female patients. The overall mean age of study subjects was 7.54 ± 4.21 years. In the deferasirox group, mean age was 6.35 ± 4.11 years, mean weight was 18.01 ± 6.74 kg, mean height was 102.04 ± 19.48 cm, and mean duration of transfusion was 7.48 ± 3.99 months/year. In the deferoxamine group, mean age was 8.74 ± 3.97 years, mean weight was 20.44 ± 6.77 kg, mean height was 102.19 ± 20.85 cm, and mean duration of transfusion was 8.14 ± 3.55 months/year. In the deferasirox group, before treatment mean serum ferritin level was 1385.73 ± 117.01 mcg/L. After treatment mean serum ferritin level was reduced to 1047.59 ± 117.08 mcg/L. In the deferoxamine group, before treatment mean serum ferritin level was 1362.58 ± 134.42 mcg/L. After treatment mean serum ferritin level was reduced to 1124.36 ± 134.52 mcg/L. Post-treatment the serum ferritin level between two groups was significantly different with p<0.01. The mean difference in serum ferritin level in pre- and post-treatment among two groups was highly significant with p<0.01. Conclusion: Deferasirox is an effective, safe and tolerable chelation therapy for the treatment of thalassaemia major with iron overload due to its ability to provide constant chelation coverage and potential to improve compliance. [ABSTRACT FROM AUTHOR]

Background and Purpose: There is no Persian scale for assessing the quality of life of patients with thalassemia major. Thus, this study was conducted to translate and validate the 36-item thalassemia-specific Transfusion-Dependent Quality of Life (TranQoL) questionnaire. Methods: The questionnaire was first translated into Persian and then was backward-translated into English. Afterward, panels of experts commented on the Persian translation, and then its face validity was confirmed and content validity index (CVI) and content validity ratio (CVR) were calculated. The factorial structure and the reliability of the translated questionnaire were assessed using exploratory factor analysis (EFA) and internal consistency method, respectively. Results: The CVI and CVR of the questionnaire were .95 and .84, respectively. Five items were deleted during EFA, and 9 factors were extracted from the 31 remaining items. The reliability of the questionnaire was confirmed through a Cronbach's alpha coefficient of .9. Conclusion: The Persian translation of the TranQoL questionnaire is highly reliable and valid, and thus, it can be used as a specific measure for assessing the quality of life among patients with thalassemia. [ABSTRACT FROM AUTHOR]

A substantial proportion of patients with transfusion-dependent beta-thalassemia major suffer from chronic liver disease. Iron overload resulting from repeated transfusions and HCV infection has been implicated in the development of liver fibrosis. Hepatic siderosis and fibrosis were assessed in 99 transfusion-dependent thalassemia (TDT) patients using transient elastography (TE) and liver iron concentration (LIC) assessed by T2*MRI at baseline and after 4 years. Data were analyzed retrospectively. At baseline, the overall mean liver stiffness measurement (LSM) was 7.4 ± 3.2 kPa and the mean LIC was 4.81 ± 3.82 mg/g dw (n = 99). Data available at 4 ± 1.5 years showed a significant reduction in LSM (6.6 ± 3.2 kPa, p 0.017) and hepatic siderosis measured by LIC (3.65 ± 3.45 mg/g dw, p 0.001). This result was confirmed when considering patients with iron overload at the time of the first measurement (n = 41) and subjects treated with a stable dose of deferasirox over the entire period of observation (n = 39). A reduction of LSM, yet not statistically significant, was achieved in patients on combined deferoxamine + deferiprone, while the group on deferoxamine (n = 11) remained stable over time. HCV-RNA positivity was found in 33 patients at T0, 20 of which were treated during the observation period. Patients who underwent anti-HCV therapy showed a more evident reduction in LSM (9 ± 3 vs 7 ± 3.1 kPa, p 0.016). Adequate chelation therapy is mandatory in order to prevent liver disease progression in TDT. Patients could benefit from regular non-invasive assessment of liver fibrosis by TE to indirectly monitor treatment adequacy and therapeutic compliance. [ABSTRACT FROM AUTHOR]

Copyright of Journal of Taibah University Medical Sciences is the property of Elsevier B.V. and its content may not be copied or emailed to multiple sites or posted to a listserv without the copyright holder's express written permission. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)

Posterior reversible encephalopathy syndrome (PRES) is a serious adverse event associated with calcineurin inhibitors used for graft-versus-host disease (GVHD) prophylaxis. We compared the incidence of PRES in children with thalassemia (n = 222, 1.4 to 17.8 years old) versus sickle cell disease (SCD; n = 59, 2 to 17 years old) who underwent hematopoietic cell transplantation from HLA-matched siblings or alternative donors and analyzed the risk factors for PRES. Overall, 31 children developed calcineurin inhibitor-related PRES (11%), including 30 patients with seizures and 1 patient without seizures. PRES incidence was significantly higher in SCD patients (22%; 95% confidence interval [CI], 10% to 32%) than in thalassemia patients (8%; 95% CI, 5% to 12%; P  = .002). In multivariate analysis, factors associated with PRES were hypertension (hazard ratio [HR], 5.87; 95% CI, 2.57 to 13.43; P  = .0001), SCD (HR, 2.49; 95% CI, 1.25 to 4.99; P  = .009), and acute GVHD (HR 2.27; 95% CI, 1.06 to 4.85; P   =  .031). In the entire cohort overall survival (OS) was significantly higher in patients without versus with PRES (90% versus 77%; P  = .02). In a subgroup analysis that including matched sibling transplants, OS and disease-free survival (DFS) were similar in thalassemia patients without PRES (92% and 88%, respectively) and with PRES (82% and 73%, respectively), whereas SCD patients with PRES had significantly lower OS (67%) and DFS (67%) than patients without PRES (94% and 94%, respectively; P  = .008). Thus, SCD patients had a significantly higher incidence of PRES than thalassemia patients, and hypertension and GVHD were the 2 main risk factors for PRES in patients with hemoglobinopathies. Although PRES did not significantly influence survival in patients with thalassemia, patients with SCD had significantly lower survival after PRES. [ABSTRACT FROM AUTHOR]

Objectives: Recent advancements have promoted the use of T2* magnetic resonance imaging (MRI) in the non-invasive detection of iron overload in various organs for thalassemia major patients. This study aims to determine the iron load in the heart and liver of patients with thalassemia major using T2* MRI and to evaluate its correlation with serum ferritin level and iron chelation therapy. Methods:This cross-sectional study included 162 subjects diagnosed with thalassemia major, who were classified into acceptable, mild, moderate, or severe cardiac and hepatic iron overload following their T2* MRI results, respectively, and these were correlated to their serum ferritin levels and iron chelation therapy. Results:The study found that 85.2% of the subjects had normal cardiac iron stores. In contrast, 70.4% of the subjects had severe liver iron overload. A significant but weak correlation (r = −0.28) was found between cardiac T2* MRI and serum ferritin, and a slightly more significant correlation (r = 0.37) was found between liver iron concentration (LIC) and serum ferritin. Discussion:The findings of this study are consistent with several other studies, which show that patients generally manifest with liver iron overload prior to cardiac iron overload. Moreover, iron accumulation demonstrated by T2* MRI results also show a significant correlation to serum ferritin levels. Conclusion:This is the first study of its kind conducted in Indonesia, which supports the fact that T2* MRI is undoubtedly valuable in the early detection of cardiac and hepatic iron overload in thalassemia major patients. [ABSTRACT FROM PUBLISHER]

Objective: Iron accumulation in the endocrine glands has been implicated in the aetiopathogenesis of decreased reproductive capacity in patients with beta-thalassemia major (β-TM). The aim of the current study was to investigate the serum concentration of anti-Müllerian hormone (AMH), a marker of ovarian reserve, in women with transfusion-dependent β-TM.Study Design: In this case-control study, we recruited 43 women with transfusion-dependent TM and 44 age-matched healthy controls. Hormonal and haematological parameters, serum level of AMH, antral follicle count, and ovarian volume were assessed.Results: Twenty-two of the 43 women were hypogonadotropic, 8 with primary amenorrhea and 14 with secondary amenorrhea. FSH, LH, estradiol, prolactin, and AMH levels; antral follicle count; and ovarian volume were significantly lower in women with TM compared with the control group (p<0.05 for all).Conclusion: AMH level and other ovarian reserve markers are significantly diminished in women with transfusion-dependent TM compared to age-matched controls. Our findings support a deleterious effect of iron overload on ovarian tissue. [ABSTRACT FROM AUTHOR]

Thalassemia is a common genetic disorder. It has been estimated that in India nearly 5 crore people are thalassemia carriers. They are asymptomatic and are detected on blood tests. These people are at same risk of developing iron deficiency anemia as general population and need iron therapy in the presence of iron deficiency anemia. Nearly 12,000 children with thalassemia major (Homozygous state) are born every year. These children often present with significant anemia along with hepatosplenomegaly during infancy and require early diagnosis and institution of therapy with repeated blood transfusions and chelation therapy. Adequate dose of chelation therapy is essential to maintain serum ferritin around 1000 ng/ml. With present protocol of management, thalassemic children have near normal life. Bone marrow transplantation offers cure for these children; results of bone marrow transplantation are best when performed below 7 y of age. [ABSTRACT FROM AUTHOR]

Background/Hypothesis: Creeping monotony in the treatment provides minimal motivation for children with beta-thalassemia major, leading to noncompliance for adequate blood transfusions and iron chelation therapy. This study was envisaged to formulate a clinical compliance score and correlate the score with their linear growth velocity and weight gain. Materials and Methods: A prospective longitudinal study among 30 thalassemia children aged between 1 and 18 years was conducted in the Department of Pediatrics, Tata Main Hospital (TMH), Jamshedpur, from July 2012 to January 2014. Relevant clinical information and investigations were recorded using a predesigned pro forma. Compliance score was calculated using pre-, post-transfusion hemoglobin and serum ferritin levels. The height velocity and weight gain were calculated. The same patients were re-assessed in January 2015. Results and Discussion: Mean age of children was 10.18 ± 4.98 years. Forty percent were females. Using the scoring table, 33% were in good category of compliance while 4% had poor compliance. The mean height velocity was significantly higher in good compliance category as compared to average or poor score category in 2014 (P = 0.017). Good compliance score children continued to maintain higher height velocity during the 1-year follow-up (P = 0.02). Average weight gain was higher in good compliance category. Conclusion: This paper brings out a simple, regularly monitored scoring system to promote growth in thalassemia children through improved compliance to treatment, which incentivized them to maintain or improve their scores for better height velocity and weight gain. No such compliance score has been tabulated for thalassemia patients as of date. [ABSTRACT FROM AUTHOR]

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Thalassemia is a congenital hemolytic disease which is treated by repeated blood transfusion. Chronic iron overload is currently considered to be the primary cause of mortality in β-thalassemia, mainly due to the induction of left-sided cardiac failure. Iron overload results from a number of mechanisms associated with the disease itself. In addition to chronic iron overload thalassemic patients are more prone for procoagulant status which in turn lead to clinical thrombotic events. The hypercoagulable state in thalassemia is due to multiple elements, a combination of which is often the drive behind a clinical thromboembolic events. PAI-1 study was done in thalassemia major patients receiving multiple blood transfusion as a marker for procoagulant status. Total of 30 thalassemic patients on repeated blood transfusion was included in the study and total of 30 healthy age and sex matched controls were included in the study. It was also found that there was significant differences between cases and controls. The mean level of PAI 1 in controls was 3047 ± 414 pg/ml, the value in cases was 3683 ± 358 pg/ml. The level was significantly increased ( p < 0.05) in the cases compared to controls. PAI-1 levels were also compared with the total number of blood transfusion which correlates well. [ABSTRACT FROM AUTHOR]

Thalassemia major is a worldwide healthcare problem requiring high cost medical treatment. Patients usually die of treatment associated complications in the second decade. However, improvements in treatment and meticulous follow-up strategies for treatment associated complications have led to an increase in expected life span of the patients. Regular transfusions cause progressive iron deposition in critical organs such as heart, liver and lungs. Iron deposition in the lungs results in respiratory dysfunction and impairment in quality of life of patients with thallassemia. Although iron deposition in respiratory system has been a known reality for many years in thallassemia, the effect of this deposition on respiratory functions has been an ongoing debate. [ABSTRACT FROM AUTHOR]

Objective: Diabetes mellitus is a common endocrinopathy in patients with β-thalassaemia major (β-TM), which is high prevalent in southern China. This study aimed to determine the cause and prevalence of glycaemic disorders in Chinese children with β-TM.Methods: In this prospective study, fasting glucose and insulin (FINS) levels were assessed in 267 β-TM and 80 non-TM control children. Homeostatic model assessment (HOMA) and the quantitative insulin sensitivity check index (QUICKI) were evaluated. Iron overload was assessed by serum ferritin (SF), total units of blood transfused and cardiac T2*.Results: β-TM had higher FPG (P < 0.001), FINS (P < 0.001) and HOMA-IR (P < 0.05), but lower QUICKI (P < 0.01) than those of controls. The impaired fasting glucose (IFG) was present in 30% of children, whereas 2% had diabetes. The prevalence of IFG in β-TM group was higher in children aged >10 years (OR 6.5; 95% CI 3.7-11.4; P < 0.001), SF of >2500 μg/l (OR 4.8; 95% CI 2.1-11.1; P < 0.01), serum ALT levels of >50 IU/l (OR 2.1; 95% CI 1.2-3.7; P < 0.05) and cardiac T2* of <20 ms (OR 3.3; 95% CI 1.7-6.6; P < 0. 01). The children on deferiprone (DFP) had a reduced incidence of glycaemic aberrations than those on other chelating agents (OR 0.4; 95% CI 0.23-0.8; P < 0.05).Conclusions: Our data suggest that IFG occurred in 30% of β TM children, perhaps due to insulin resistance secondary to iron overload. Deferiprone-containing chelating agent may have a protective effect. [ABSTRACT FROM AUTHOR]