Pediatric Thalassemia Patients Research Articles

Association of serum ferritin trends with liver enzyme patterns in β-thalassemia major: A longitudinal correlational study.

β-Thalassemia major patients require lifelong blood transfusions, leading to iron overload and liver injury. This study examines the longitudinal association between serum ferritin and liver function over 5 years in pediatric patients. This retrospective study included 582 transfusion-dependent thalassemia patients aged 1-18 years. Serum ferritin, aspartate aminotransferase (AST), alanine aminotransferase (ALT), and albumin were measured annually. Correlation and linear regression analyses assessed associations between ferritin trajectories and liver enzymes. Mean ferritin rose from 1820 ± 960 ng/mL at baseline to 4500 ± 1900 ng/mL at year 5, indicating worsening iron overload. AST and ALT levels also steadily climbed over follow-up, whereas albumin declined slightly. Ferritin correlated positively with AST (r = 0.675, P < 0.01) and ALT (r = 0.607, P < 0.01), but not with albumin (r = -0.143, P = 0.153) annually. The regression interaction term showed within-patient ferritin increases over time were independently associated with escalating AST and ALT (P < 0.05), after adjusting for confounders. Rising ferritin levels predict progressive liver injury in regularly transfused pediatric thalassemia patients. Tighter control of iron overload may help preserve hepatic function.

Physical characteristics and factors affecting in pediatric thalassemia

Background: Thalassemia, one of the most common genetic diseases globally, can contribute to physical developmental delays in pediatric patients due to various factors. Chronic anemia, transfusional iron overload and chelation toxicity are key influences on the growth of children with thalassemia. Objective: This study aims to describe the physical development of pediatric thalassemia patients and assess the factors associated with their physical indices. Methods: A cross-sectional study involving 44 pediatric thalassemia patients receiving treatment at the Pediatric Center, Hue Central Hospital, was conducted from February 2023 to December 2023. Results: The study included 44 participants with an average age of 7.5 ± 4.3 years and an equal distribution of males and females. Of these, 64.5% were transfusion-dependent thalassemia patients. Serum ferritin levels exceeded 1000 ng/ml in 43.2% of participants, and the mean hemoglobin concentration was 67.4 ± 16.1 g/L. Of the evaluated children, 31.8% had height-for-age below -2SD, and 43.2% had weight-for-age below -2SD. Statistically significant associations were observed between height-for-age, weight-for-age, and both blood transfusion dependence and serum ferritin concentration (p &lt; 0.05). However, no significant differences were detected between physical indices and disease type or hemoglobin concentration (p &gt; 0.05). Conclusions: This study found that 31.8% of pediatric thalassemia patients experienced stunting, and 43.2% experienced wasting malnutrition. The degree of blood transfusion dependence and serum ferritin concentration were identified as factors associated with a higher prevalence of stunting and wasting malnutrition in children with thalassemia. Key words: Children, thalassemia, physical index, transfusion-dependent thalassemia, non-transfusion-dependent thalassemia.

Pancreatic iron in pediatric transfusion-dependent beta-thalassemia patients: A longitudinal MRI study.

In pediatric transfusion-dependent thalassemia (TDT) patients, we evaluated the prevalence, pattern, and clinical associations of pancreatic siderosis and the changes in pancreatic iron levels and their association with baseline and changes in total body iron balance. We considered 86 pediatric TDT patients consecutively enrolled in the Extension-Myocardial Iron Overload in Thalassemia Network. Iron overload (IO) was quantified by R2* magnetic resonance imaging (MRI). Sixty-three (73%) patients had pancreatic IO (R2*>38Hz). Global pancreas R2* values were significantly correlated with mean serum ferritin levels, MRI liver iron concentration (LIC) values, and global heart R2* values. Global pancreas R2* values were significantly higher in patients with altered versus normal glucose metabolism. Thirty-one patients also performed the follow-up MRI at 18±3months. Higher pancreatic R2* values were detected at the follow-up, but the difference versus the baseline MRI was not significant. The 20% of patients with baseline pancreatic IO showed no pancreatic IO at the follow-up. The 46% of patients without baseline pancreatic IO developed pancreatic siderosis. The changes in global pancreas R2* between the two MRIs were not correlated with baseline serum ferritin levels, baseline, final, and changes in MRI LIC values, or baseline pancreatic iron levels. In children with TDT, pancreatic siderosis is a frequent finding associated with hepatic siderosis and represents a risk factor for myocardial siderosis and alterations of glucose metabolism. Iron removal from the pancreas is exceptionally challenging and independent from hepatic iron status.

Effect of serum panel reactive antibodies on allogeneic hematopoietic stem cell transplantation in pediatric thalassemia patients: A single-center experience.

The aim of this study was to assess the impact of serum panel reactive antibodies (PRA) on the outcomes of allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric thalassemia patients. A total of 73 pediatric patients with thalassemia were included in this single-center study. Pre-transplant PRA levels were evaluated, and the patients were divided into two groups: PRA-negative (group 1; n = 44) and PRA-positive (group 2; n = 29). Patient characteristics, including age, gender, donor type, stem cell source, and HLA compatibility, were analyzed. Transplant outcomes, including engraftment, transfusion requirements, and transplant-related complications, were compared between the two groups. Further subgroup analysis was performed based on MFI values. At the time of transplantation, patients in group 1 were younger than those in group 2 (p = .008). The number of fully matched donors within the family (MSD and MFD) was significantly higher in group 1 (p = .049). Additionally, Rh blood group incompatibility was higher in group 2 (p = .03). There was no statistically significant difference in the engraftment days of neutrophils, platelets, and erythrocytes between the two groups. The frequency of poor graft function and graft failure was higher in the group 2, but there was no statistically significant difference. Post-transplant transfusion requirements for platelets and red blood cells were significantly higher in the group 2 (p < .001). Transplant-related complications such as VOD, PRES, and aGvHD were more common in the group 2, but no statistical significance was detected. Serum PRA in pediatric thalassemia patients may impact the outcomes of HSCT. PRA-positive patients had higher rates of blood product transfusion requirements. Although poor graft function, graft failure, and post-transplant complications were more common in the group 2, statistical significance was not observed. Identifying patients with high PRA levels can assist in optimizing transplant strategies and post-transplant care, leading to improved outcomes for the patients.

Characterization of pediatric transfusion-dependent thalassemia patients in a large academic center.

Transfusion-dependent thalassemia patients are at high risk of transfusion-related complications. Yet, there is scanty data on the frequency of transfusion reactions, particularity alloimmunization among pediatric transfusion-dependent thalassemia patients. In addition, there is no consensus on the prophylactic antigen matching for prevention of alloimmunization or the extent of antigen matching for alloimmunized thalassemia patients. We conducted a retrospective study to assess the frequency and specificity of alloimmunization among pediatric transfusion-dependent thalassemia patients receiving ABO, RhD, and K-matched red blood cell units. In addition, we studied the association between patients' characteristics and alloimmunization. The clinical and transfusion records of transfusion-dependent thalassemia patients followed up at our institution between July 2018 and June 2022 were reviewed. Ninety-two transfusion-dependent thalassemia patients having mean age of 13.37 years (SD, 5.56) were included in our study. Eight patients (9%) had developed clinically significant alloantibodies; six patients (6%) developed alloantibody against E antigen while two patients (2%) developed more than one alloantibody. Of alloimmunized patients, five patients had received transfusion outside Canada. Patients' sex, age, having a genotype variant, total number, and duration of transfusion received were not associated with the risk of alloimmunization. The transfusion-recipient's diagnosis of β-thalassemia, having developed autoantibody, and history of receiving transfusion outside Canada were associated with alloimmunization. Blood matching for ABO, RhD, and K antigens resulted in, although not eliminated, lower frequency of alloimmunization than that previously reported among pediatric thalassemia patients. Extending matching to include Rh antigens could further reduce the rate of alloimmunization.

Correlation and Regression Analysis Applied for Types and Number of Blood Transfusions Versus Transfusions Induced ADR Among Pediatric Haemato-Oncology and Thalassemia Patients.

Introduction: Blood transfusion is a remedial intercession and a fundamental fragment of Current medical services framework. As indicated by the WHO the bonding is an action of moving blood or blood-items taken from a giver into the vasculature and in this manner the flowing blood of the recipient, done by embeddings an IV Needle/Catheter in the patient and followed by use of blood or the blood-items. In any case, the dangers of non-irresistible difficulties have gotten more obvious. These nonirresistible complexities called as adverse transfusion reactions (ATRs) can either be intense in nature or follow a postponed course. Transfusion reaction is any unanticipated impact that happens in a patient during or subsequent to accepting blood and the blood-items. These can be agreed as intense transfusion responses, happening inside 24 hours of transfusion and deferred bonding responses happens inside the space of days or long periods of bonding. Intense and moderate responses can be additionally classified as insusceptible interceded and non-immune-intervened. Methodology: The prospective and observational study was carried at Department of Pediatric Oncology & Thalassemia unit for a period of 6 months. The population required for the study includes patients undergoing blood transfusions. Results: In study data out of 83 units, 61 units of PCV was transfused (73.49%), 6 units of Platelet was transfused (7.22%), 14 units of Whole Blood was transfused (16.86%), 1 units of Fresh Frozen Plasma was transfused (1.20%), and 1 units of RBC was transfused (1.20%). The chance of an ADR occurrence with every transfusion is 0.4. The P value is less than .05 so it is statically significant. It is positively correlated, having a high impact on the number of transfusion and rate of incidence of ADR. Conclusion: While a Blood transfusion is given for unavoidable situation the important factors to be kept under consideration are ADR following transfusion which can be the common complication observed. The study also concludes that the rate of incidence of ADR is increased significantly as the number of transfusion increases.

TO STUDY EFFECTIVENESS OF DEFERASIROX, AN ORAL IRON CHELATOR IN BLOOD TRANSFUSION DEPENDENT PEDIATRIC THALASSEMIA PATIENTS

Background: Inblood transfusion related thalassemia patients, high iron state is observed because of regular blood transfusion. Our body has limited mechanism to excrete iron, excess of iron gets deposited in various organs causing their dysfunction. To prevent the complication associated with this iron overload use of iron chelators becomes important in management of such patients. Aims /Objectives: 1.To determine effectiveness of deferasirox, an oral iron chelator to decrease iron overload in reference to serum ferritin level in blood transfusion dependent pediatric thalassemia patients. 2.To determine effectiveness of deferasirox in reducing iron deposition induced organ damage. Methods: This is a hospital based retrospective observationalstudy conducted on 80 blood transfusion dependent thalassemia patients admitted in tertiary care hospital. Data was collected in pre-designed case record sheets after taking consent from parents. Results:in the present study the mean age was 7.12 years for females and 10.93 years for males. The mean of pre and post blood transfusion hemoglobin was significantly higher (7.07 and 10.76 respectively). The mean blood transfusion days are 26.33. Mean annual blood requirement is 198.99. Mean drop rate is 1.02. On comparing mean ferritin levels, there was a significant difference between baseline (711.81), start of deferasirox treatment (1271.71) and the end of 2nd dose increment of deferasirox (2076.11). The mean ferritin decreased significantly after starting deferasirox (from 1360.03 to 1271.71) by the end of1stdose increment (from 1832.41 to 1696.75) by the end of 2nd dose increment (from 2097.32 to 2076.11) , but when we compare mean ferritin after end of 2nddose increment of deferasirox (2076.11) with6 month back mean ferritin (2506) or current mean ferritin (2514.90) there is increase in ferritin.Mean blood transfusions at start of deferasirox is (11.025), at 1st dose increment is (26.13), at 2nd dose increment is (42.12).There were no organ complications seen with the present population at the end of the study Conclusion: from the results of our study, we state that using deferasirox as oral iron chelator in blood transfusion dependent pediatric thalassemia patients helps in reducing iron overload till we reach maximum dose of oral deferasirox but beyond that there is significant increase in serum ferritin level,thereby necessitating use of another iron chelator along with oral deferasirox(either deferoxamine, deferiprone or some new iron chelator),. Also in this study it is observed that there is no iron deposition related significant organ damage.

HUBUNGAN TINGKAT KEPATUHAN KONSUMSI OBAT KELASI BESI DENGAN KADAR FERRITIN PADA PASIEN THALASSEMIA ANAK DI RSUD DR. MOEWARDI

Thalassemia is a hereditary hemolytic disease caused by mutations in the hemoglobin synthesis chain. Transfusion-dependent thalassemia patients require iron chelation therapy to prevent complications of iron overload. Adherence with regular iron chelation consumption is important for good survival, reducing morbidity and mortality in thalassemia patients. This research is an analysis of an observational study with a cross-sectional approach. This research was conducted at RSUD Dr. Moewardi. The subjects of this study were pediatric thalassemia patients who were taking deferiprone iron chelating drugs. The sample was taken using purposive sampling method, by analysing medical record data and the MMAS-8 questionnaire. Data were examined using the chi-square test with the SPSS application. Results: There were 56 subjects to be analyzed consisting of 23 males and 33 females. Based on the chi-square test, there was a significant relationship between consumption of iron chelating drugs and ferritin levels with p = 0.026. In addition, another study was conducted using the chi-square test regarding nutritional status and duration of thalassemia illness on ferritin levels. The results obtained were p value = 0.977 for the relationship between nutritional status and p value = 0.977 for the long duration of thalassemia illness. It can be concluded that both of them have no significant effect on ferritin levels. Adherence with taking iron chelating drugs has a significant relationship to ferritin levels in thalassemia patients.

Predictors of Non-adherence to iron chelation therapy in pediatric thalassemia patients

Objective: This study was conducted to identify predictors of non-adherence to iron chelation therapy among children suffering from β-thalassemia major across different treatment regimens.Materials and Methods: It was a cross-sectional study carried out from 1st January 2019 to 30th June 2019. The study was conducted at the Pakistan Institute of Thalassemia, Islamabad. Children between the ages of 2 -16 years suffering from β-Thalassemia major and taking iron chelation therapy were included in the study. Chelation adherence for this analysis was defined as the percent of doses taken in the last 12 weeks out of those prescribed. Guardians of patients were interviewed using a questionnaire and medical records were checked. Data were analysed using SPSS 20.0. Multivariate analysis was conducted to identify the predictors for non-adherence to chelation therapy. The significant value was set at ≤ 0.05.Results: Mean age of the patients in our study was 8.90± 3.74 years. There were 33 males and 64 females. Most of the patients n=87 (89.7%) were taking oral iron chelation therapy. The mean score for chelation adherence was 67.12%. Among the multiple demographic, medical-related, and patient-related factors analysed, travel time from the patient’s residence to the treatment centre and the number of transfusions per year were found to be significant predictors (p-value ≤0.05) of non-adherence to iron chelation therapy. Conclusion: Overall, the study provides strong evidence that healthcare-related factors play a major role in patients’ adherence to treatment. A systemic approach should be taken to ensure patient adherence during the management of paediatric thalassemic patients.

Identification of osteopenia and osteoporotic changes in pediatric thalassemia patients at thalassemia center, Misan city, Iraq; An intervention study

Background: Thalassemia syndrome is a heterogeneous group of hereditary disorders affecting the synthesis of globin chains of adult hemoglobin's, leading to ineffective erythropoiesis, hemolysis, and moderate to severe anemia. To identify the incidence of osteopenia and osteoporosis and it is relation to some of the probable risk factors of decreasing BMD among thalassemia patients attended Misan thalassemia center in Misan city in Iraq.&#x0D; Methods: A randomized cross-sectional study, applied through a period from September 2020 to September 2021, on 50 thalassemia patients attended thalassemia Center Amarah, Misan, Iraq. 24 were males and 26 were females, we divided them in to two groups according to age (10-14) years, (&gt;14-18) years. They were scanned for bone mineral density (BMD) at lumbar spine.&#x0D; Results: 90% of patients had reduction in BMD patients mostly among THM patients. 40% had positive family history of thalassemia, Vitamin D3 level were low in (80%) of patients &amp; DEXA studies were mostly between [(-1) -(-2.5)] before treatment that slightly improved after therapy in ranges of osteopenia but not in osteoporosis which increase severity as well.&#x0D; Conclusions: Most thalassemia patient had severe anemia, corrected by frequent blood transfusion are complicated by increasing serum ferritin level and decreasing level of vitamin D3 in association with reduction in bone density mass in ranges of osteopenia and osteoporosis which correlates positively with increasing ages, early age at diagnosis and female gender this altered bone mass density require family awareness for better nutrition and optimum medical therapy and compliance to improve outcomes. &#x0D;

Mobilization of backup autologous peripheral stem cells in pediatric thalassemia patients: A single center experience

Mobilization of backup autologous peripheral stem cells in pediatric thalassemia patients: A single center experience

Efficacy and Safety of Iron Chelation Therapy After Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric Thalassemia Patients: A Retrospective Observational Study.

Studies on the increased body iron load in patients with thalassemia major have thoroughly demonstrated the problems caused by iron overload. In patients who undergo hematopoietic stem cell transplantation (HSCT) as curative therapy, iron overload continues long after transplantation. There are few pediatric studies on chelation therapy in the posttransplant period. In this study, we present the outcomes of our patients who received posttransplant oral chelation therapy. This retrospective observational study evaluated the outcomes of pediatric patients with thalassemia major who used oral chelation therapy after allogeneic HSCT at the Akdeniz University Pediatric Bone Marrow Unit between January 2008 and October 2019. Deferasirox therapy was initiated in 58 pediatric patients who underwent HSCT for thalassemia. Pretreatment mean serum ferritin was 2166±1038 ng/mL. Treatment was initiated at a mean of 12±6.7 months after transplantation and continued for a mean of 15.7±11.5 months. At treatment discontinuation, the mean serum ferritin was 693±405 ng/mL and the mean reduction was -1472.75±1121.09 ng/mL (P<0.001 vs. posttreatment). Serum ferritin was below 500 ng/mL in 52% of the patients at treatment discontinuation. Manageable side effects such as nausea, vomiting, liver enzyme elevation, and proteinuria were observed in 17% of the patients, while one patient developed ototoxicity. Deferasirox therapy effectively reduces iron overload in the posttransplant period. Studies evaluating the effects of early treatment on the graft may help to establish guidelines for posttransplant chelation therapy. Clear guidelines are needed regarding when to initiate and discontinue treatment.

“Efficacy and Safety of Deferasirox in Pediatric Thalassemia Patients: Experience from a Tertiary Care Children Hospital of Bangladesh”

Background: Thalassemia is an inherited hemoglobin disorder; mostly require life-long blood transfusions, leading to chronic iron overload which causes growth failure, delayed sexual development in adolescents and vital organ dysfunctions. So, children with thalassemia need lifelong iron chelation therapy. Hence, this study conducted with the aim of to evaluate efficacy and safety of Deferasirox in pediatric thalassemia patients.&#x0D; Materials and methods: This is an observational, prospective, single-centered hospital-based study conducted in a tertiary care teaching hospital from June 2018 to December 2019. Total sixty children (age 2-18 years) with beta thalassemia major and hemoglobin E beta thalassemia with iron overload were enrolled to commence deferasirox. Efficacy and safety we observed by measuring serum ferritin three monthly and SGPT, SGOT &amp; serum creatinine monthly.&#x0D; Results: The serum ferritin level of 72% patients reduced significantly after 12 months in comparison to baseline level. There was no serious adverse effect except mild abdominal pain, nausea &amp; vomiting and transaminitis.&#x0D; Conclusion: Deferasirox is efficacious in reducing iron overload of the body when administer at optimum dose over at least one year and presenting a safe as well as convenient alternative for most of the transfusion dependent pediatric thalassemia patients.

Cardiac MRI for Iron Overload in pediatric thalassemia patients- Right Age to Start in a Resource Constrained Environment.

The aim of this article is to assess the prevalence and severity of myocardial iron overload in thalassemia patients who present for the first time for cardiac MRI and to define the right age to start screening, in a resource-constrained environment. All MRI scans done at our institute for myocardial iron overload assessment in thalassemia patients, between 2015 and 2018 were analysed. Patients up to the age of 30years were included. There were a total of 600 patients, (Age group between 2 and 30years). All these scans were retrospectively analysed and severity of myocardial iron overload was categorized as normal, mild, moderate and severe based on the bright blood T2* equivalent values at 1.5T. Overall prevalence of myocardial iron overload was 32.3%, while the prevalence of myocardial iron overload in patients in the age group 0-10 was 10.2%.There were 40 patients in the age group 0-6years, of whom, only 2 had myocardial iron overload. In patients less than or equal to 6years of age, the number of patients with iron overload was very small and this may be used to decide the optimal age for scanning.

Serum ferritin levels and endocrine disorders in children with thalassemia major

Background Endocrine disorders in thalassemia major children patients occur due to iron overload and hemosiderosis in endocrine organs. Early detection is needed to prevent complications and improve the quality of life. An association between serum ferritin and endocrine disorders in thalassemia patients has been inconclusive to date.&#x0D; Objective To analyze for possible associations between serum ferritin and endocrine disorders (short stature, delayed puberty, delayed bone age, hypothyroidism, impaired glucose tolerance, and diabetes mellitus) and the incidence of each disorder in thalassemia major.&#x0D; Methods There were 115 thalassemia major patients aged 10-18 years involved in our cross-sectional study from June 2019 - June 2020 in the Pediatrics Department, DR. M Hosein Hospital, Palembang, South Sumatera. Anthropometry and socioeconomic status informations were collected from physical examination and interview. Ferritin, FT4, TSH, Hb and glucose levels measured by using standard methods for each item in the laboratory, mean while the skeletal age assessment was determined by using FELS method. &#x0D; Results This study included 83 (72.2%) girls and 32 (27.8%) boys. There were 89 (77.4%) subjects with short stature, 74 (64.4%) with delayed bone age, 30 (26.1%) with impaired glucose tolerance, 25 (21.7%) with delayed puberty, 4 (3.5%) with diabetes mellitus (DM), and none with hypothyroidism. Bivariate and multivariate analyses revealed no associations between serum ferritin and short stature, delayed bone age, impaired glucose tolerance, delayed puberty, and DM.&#x0D; Conclusion There is a high prevalence of endocrine disorders in pediatric thalassemia patients, especially short stature and delayed bone age. However, there are no associations between serum ferritin and endocrine disorders in these patients.

High Prevalence of Antiphospholipid Antibodies in Children with Non-Transfusion Dependent Thalassemia and Possible Correlations with Microparticles.

Antiphospholipid antibodies (APAs) are developed antibodies to the phospholipid surfaces or to the proteins binding to phospholipids. Increased PS-expressed red blood cells (RBC) may be a risk factor in APAs development. There were only a few studies reported on APAs in thalassemia, and their associations to PS-expressing microparticles (MPs), remain unknown. The presence of antiphospholipid antibodies (APAs) in pediatric thalassemia patients and their associations with phosphatidylserine (PS)- expressing microparticles (MPs), a previously unexplored relationship, were the aims of this report. We identified that positive APA rates were highest in non-transfusion-dependent thalassemia (NTDT) subjects, followed by transfusion-dependent thalassemia (TDT) subjects and controls (29.5%, 18.2% and 17.9%, respectively). Subgroup analysis demonstrated that positive anti- ?2 glycoprotein I (a?2-GPI)- IgG rates in NTDT subjects were significantly greater than in controls and that the percentage of MPs was also significantly elevated in thalassemia subjects. More specifically, there were significant correlations found between a?2GPI-IgG and anticardiolipin (aCL)-IgM APAs, and MPs.

Prediction, management, and prognosis of mixed chimerism after hematopoietic stem cell transplantation in transfusion-dependent pediatric thalassemia patients.

Early-onset mixed chimerism (MC) with a high proportion of residual host cells is considered a signal of graft rejection in patients undergoing allogenic hematopoietic stem cell transplantation for transfusion-dependent thalassemia. In order to prevent graft rejection and minimize the risk of treatment-related graft-versus-host disease (GVHD), we established a hierarchical management system based on chimerism analysis. This retrospective study provides a comprehensive review of the characteristics, interventions, and outcomes of the 38 patients who developed MC after transplantation among the 144 pediatric thalassemia patients between July 2007 and January 2019 at our center. A sibling donor, a blood type-matched donor, conditioning regimens without fludarabine, and transplants containing <10×108 total nucleated cells/kg were identified to be associated with the development of MC. Among the 38 patients developing MC, only four patients rejected the grafts. The response rate to donor lymphocyte infusion (DLI, only for patients receiving sibling donor transplantation) and cytokine immunomodulation without DLI was 70.6% and 42.9%, respectively. Patients that developed GVHD after DLI or cytokine therapy had a more significant increase in donor cell chimerism (16%, range 0%-35%) than those without (8.5%, range -21% to 40%, P=.049). However, even when treatment-related GVHD was included, patients with MC had a lower cumulative incidence of total acute GVHD than patients with complete donor chimerism (29.2% vs 48.0%, P=.030). Interventions based on chimerism analysis were effective in preventing graft rejection and did not increase treatment-related GVHD in thalassemia patients with MC.

Health-Related Quality of Life in Thai Children with Thalassemia as Evaluated by PedsQL and EQ-5D-Y: A Single-Center Experience.

BackgroundThalassemia remains a challenging chronic disease in Thailand, but national prenatal screening, along with better treatment and management, may have improved health-related quality of life (HRQoL) for pediatric patients. We aimed to measure the HRQoL of transfusion-dependent (TDT) and non-transfusion dependent (NTDT) of these pediatric patients at our institute.MethodsWe included all patients 2 – 18 years old, with TDT and NTDT, using the Pediatric Quality of Life Inventory 4.0 Generic Core Scales (PedsQL) and the EuroQol Group's Five Dimensions for Youth (EQ-5D-Y) instruments. Patients and caregivers responded as appropriate for age.ResultsMean PedsQL total summary scores (TSS) (SD) of child self-reports and parent proxy-reports were 81.00 (10.94) and 78.84 (16.72) from 150 participants. Mean EQ-5D-Y VAS (SD) for children was 89.27 (11.56) and 86.72 (10.62) for parent proxies. The most problematic EQ-5D-Y dimension was “having pain or discomfort”. These scores had significant correlations between the child and parental proxy perspectives, as well as between the PedsQL and EQ-5D-Y. An age of 8 – 12 years and oral chelation therapy predicted lower self-reported PedsQL TSS. Parental proxy-report predictors for reduced PedsQL TSS and EQ-5D-Y VAS were primary school education for children, parental proxy secondary school education, Universal Coverage insurance, and TDT.ConclusionHRQoL scores of our pediatric thalassemia patients had improved from the previous decade, and these findings may represent our better standard of care. Some sociodemographic and clinical characteristics may present negative impacts on HRQoL. More exploration is needed to understand predictors and further improve HRQoL, especially for TDT patients.

Effectiveness and tolerability of twice daily dosing of deferasirox in unresponsive and intolerant transfusion-dependent beta-thalassemia patients: A narrative review

Chronic iron overload in beta-thalassemia patients after continuous blood transfusions has caused notable morbidity and mortality in these patients. The once-a-day oral iron chelator, deferasirox has established efficacy and bearable safety in adults and pediatric thalassemia patients. It is now extensively used for the management of transfusional hemosiderosis. However, a number of studies have revealed a few patients continued to be none respondent or intolerant toward the once-a-day regimen of deferasirox even after the administration of maximum dose recommended by the World Health Organization. In the literature, there were three studies showing the boon of twice in a day dosing of deferasirox among transfusional-dependent beta thalassemia patients. Therefore, a nonsystematic review was conducted on above three studies to ascertain the enhanced effectiveness and tolerability of twice per day regimen of deferasirox with the same total dose as that of once daily regimen of deferasirox in unresponsive or intolerant transfusion-dependent beta-thalassemia (TDT) patients. All the above studies concluded that the twice per day regimen of deferasirox was more efficacious and tolerable among TDT patients when compared to the once-a-day regimen with the same total daily dose. Although there was a significant good results from these studies, there is a need to conduct either muticenter study or randomized control study in a larger number of patients for the better confirmation of the results as all the above studies were conducted in the small number of TDT patients.

Effectiveness of Deferasirox in Pediatric Thalassemia Patients: Experience from a Tertiary Care Hospital of Odisha.

BACKGROUND AND OBJECTIVES:Patients with beta-thalassemia require lifelong blood transfusions, leading to chronic iron overload, which can lead to growth retardation, as well as hinder sexual development during the adolescent period and dysfunction of organs such as heart, pancreas, and endocrine glands. These patients are in need of lifelong transfusion therapy and hence lifelong iron chelation therapy as well. Hence, this study was aimed to assess the effectiveness of deferasirox for iron chelation in pediatric thalassemia cases in a tertiary care hospital of Eastern India.SUBJECTS AND METHODS:This prospective, observational, hospital-based study was conducted from June 2015 to December 2016. Two hundred and fifty patients were assessed for eligibility, of which 174 were included. Effectiveness of deferasirox was observed by measuring serum ferritin levels which were monitored at the end of every 3 months till 1 year. We also evaluated the compliance with deferasirox therapy in the same study cohort.RESULTS:The serum ferritin level reduced significantly at the end of 12 months in comparison to baseline (P = 0.04). There was a mean absolute decrease in serum ferritin only in the dose range of 21–30 mg/kg/day. Approximately 90% of the patients had 100% compliance with deferasirox therapy.CONCLUSIONS:Deferasirox is an effective iron chelator when started at an optimum time and with optimum dose. At least 1 year of deferasirox therapy is needed for a significant lowering of serum ferritin levels of pediatric thalassemia patients on multiple blood transfusions.