Clinico-Hematological Profile and Management of Children With Non-Transfusion Dependent Thalassemia (NTDT) at a Pediatric Center in Northern India

Objective: To study the clinico-hematological profile, complications, and management of children with non-transfusion dependent thalassemia (NTDT) in northern India. Method: We retrieved and analyzed the data of 69 children with NTDT diagnosed between January, 2006 to December, 2018, aged under 18 years from our unit’s records. Result: The participants mean (SD) age was 4.4 (3.1) years, and they presented with anemia (29%), jaundice (13%), hemolytic facies (13%), splenomegaly (87%), thromboembolism (2.9%) and pathological short stature (28.5%). The most common cause of NTDT was ?-thalassemia (45%), followed by either compound-heterozygous or homozygous for E?-thalassemia mutation. The most frequent single genotype observed was compound heterozygous for IVS1-5 (G>C) and codon 26 (G>A). The mean (SD) follow-up duration was 3.5 (2.4) years. On follow-up, 27 children (%) remained transfusion free, and 30 (%) needed occasional transfusions. 63% of patients initially presenting with pathological short stature showed improvement in growth. Amongst children older than 10 years (n=20), subclinical hypothyroidism was detected in 6 children and impaired glucose tolerance test in 1 child. Conclusion: Eß-thalassemia was the commonest cause of NTDT in this population.

Cell-Based Gene Therapy for ?-Thalassemia

The United States Food and Drug Administration (FDA) approved betibeglogene autotemcel (beti-cel), the first cell-based gene therapy for adult and pediatric patients with ?-thalassemia in August, 2022. This update details this and other novel therapies that have emerged in the treatment of ?-thalassemia, apart from transfusion and iron chelation, with particular focus on newly approved gene therapy

Cardiac iron overload evaluation in thalassaemic patients using T2* magnetic resonance imaging following chelation therapy: a multicentre cross-sectional study

Abstract Introduction Magnetic resonance imaging (MRI) T2* technique is used to assess iron overload in the heart, liver and pancreas of thalassaemic patients. Optimal iron chelation and expected tissue iron response rates remain under investigation. The objective of this study was to analyse serum ferritin and the iron concentration in the heart, liver and pancreas measured by MRI T2*/R2* during regular chelation therapy in a real-world cohort of patients with thalassemia. Methods We evaluated thalassaemic patients ≥ 7 years old undergoing chelation/transfusion therapy by MRI and assessed serum ferritin at baseline and follow-up from 2004-2011. Results We evaluated 136 patients, 92% major thalassaemic, with a median age of 18 years, and median baseline ferritin 2.033ng/ml (range: 59-14,123). Iron overload distribution was: liver (99%), pancreas (74%) and heart (36%). After a median of 1.2 years of follow-up, the iron overload in the myocardium reduced from 2,63 Fe mg/g to 2,05 (p 0.003). The optimal R2* pancreas cut-off was 148 Hertz, achieving 78% sensitivity and 73% specificity. However, when combining the R2* pancreas cut off ≤ 50 Hertz and a ferritin ≤ 1222 ng/ml, we could reach a negative predictive value (NPV) of 98% for cardiac siderosis. Only 28% were undergoing combined chelation at baseline assessment, which increased up to 50% on follow up evaluation. Conclusions Chelation therapy significantly reduced cardiac siderosis in thalassaemic patients. In patients with moderate/severe liver iron concentration undergoing chelation therapy, ferritin levels and myocardium iron improved earlier than the liver siderosis.

Research progress of small molecule fluorescent probes for ferrous ion and heme / 药学学报

Small molecule fluorescent probes have gained widespread attention for their advantages of high selectivity, sensitivity, and easy to operate, and have played a critical role in the detection of various species. They have also demonstrated great potential in the field of biomedical research. Iron, as the most abundant transition metal in the human body, plays a vital role in many physiological functions. Due to the influence of the reductive microenvironment of cell, ferrous ion (Fe2+) is the main component of labile iron in living cells. Heme, consisting of Fe2+ and protoporphyrin IX, is one of the main signaling molecules that wrap biological iron in the human body, and also participates in many physiological and pathological processes. Therefore, the development of small molecule fluorescent probes for detecting Fe2+ and heme as effective monitoring tools will help to further understand their pathological and physiological functions, with potential applications in other fields. This review summarizes the research progress of small molecule fluorescent probes for Fe2+ and heme detection in recent years, and provides insights into future directions for their development.

A rare case of bronze diabetes in a patient with thalassemia

Secondary haemochromatosis (also known as bronze diabetes) is a perilous medical condition that can occur as a complication of frequent blood transfusions. Thalassemia major which occurs due to a decrease in the beta globulin chain can lead to severe anemia, extramedullary hematopoiesis and splenomegaly. Becauseof this, the affected patients requiredcontinuous blood transfusions throughout their life and as a consequence, it may lead to iron overload. A 26-year-old male presented with a complaint of darkening skin, joint pain and fever. He was a known case of thalassemia major and was undergoing blood transfusions three times a week. Further laboratory findings revealed decreased hemoglobin, abnormal liver function tests and increased blood glucose levels. The patient was managed with IV insulin and chelation therapy. The patient responded to treatment and was better on subsequent follow-up. The diagnostic and therapeutic challenges along with the epidemiological dataemphasize the need of raising the awareness of physicians to this devastating condition.

Naringin is a promising natural compound for therapy of iron-overload disorders

Abstract Naringin has been shown to exhibit satisfying iron chelation capacity. Considering the side effects of routinely-used iron chelator (desferrioxamine, DFO), we decided to evaluate the iron chelation potency of naringin to discover whether or not it can be a promising natural substitute for treatment of excessive iron-related diseases. 35 mice were classified into five groups of 7 and subjected to iron dextran administration to induce the iron-overload condition. Iron-overloaded mice were then treated with normal saline (as control), naringin or DFO Morphology changes, and iron deposition in liver tissues were studied using H&E and Perl's staining. The results revealed that naringin is more potent than DFO in removing excessive iron ions deposited in liver tissues, indicating that naringin is a promising natural compound for therapy of iron overload disorders

Influencing factors of iron metabolism assessment in patients with myelodysplastic syndrome: A retrospective study / 中华血液学杂志

Objective: To analyze the influencing factors of iron metabolism assessment in patients with myelodysplastic syndrome. Methods: MRI and/or DECT were used to detect liver and cardiac iron content in 181 patients with MDS, among whom, 41 received regular iron chelation therapy during two examinations. The adjusted ferritin (ASF) , erythropoietin (EPO) , cardiac function, liver transaminase, hepatitis antibody, and peripheral blood T cell polarization were detected and the results of myelofibrosis, splenomegaly, and cyclosporine were collected and comparative analyzed in patients. Results: We observed a positive correlation between liver iron concentration and ASF both in the MRI group and DECT groups (r=0.512 and 0.606, respectively, P<0.001) , only a weak correlation between the heart iron concentration and ASF in the MRI group (r=0.303, P<0.001) , and no significant correlation between cardiac iron concentration and ASF in the DECT group (r=0.231, P=0.053) . Moreover, transfusion dependence in liver and cardiac [MRI group was significantly associated with the concentration of iron in: LIC: (28.370±10.706) mg/g vs (7.593±3.508) mg/g, t=24.30, P<0.001; MIC: 1.81 vs 0.95, z=2.625, P<0.05; DECT group: liver VIC: (4.269±1.258) g/L vs (1.078±0.383) g/L, t=23.14, P<0.001: cardiac VIC: 1.69 vs 0.68, z=3.142, P<0.05]. The concentration of EPO in the severe iron overload group was significantly higher than that in the mild to moderate iron overload group and normal group (P<0.001) . Compared to the low-risk MDS group, the liver iron concentration in patients with MDS with cyclic sideroblasts (MDS-RS) was significantly elevated [DECT group: 3.80 (1.97, 5.51) g/L vs 1.66 (0.67, 2.94) g/L, P=0.004; MRI group: 13.7 (8.1,29.1) mg/g vs 11.6 (7.1,21.1) mg/g, P=0.032]. Factors including age, bone marrow fibrosis, splenomegaly, T cell polarization, use of cyclosporine A, liver aminotransferase, and hepatitis antibody positive had no obvious effect on iron metabolism. Conclusion: There was a positive correlation between liver iron concentration and ASF in patients with MDS, whereas there was no significant correlation between cardiac iron concentration and ASF. Iron metabolism was affected by transfusion dependence, EPO concentration, and RS.

Effect of Aloe-emodin on Aβ42 Aggregation and Cytotoxicity in Presence of Al3+ / 中国实验方剂学杂志

ObjectiveTo study the inhibitory effect of aloe-emodin （AE） on aluminum ion （Al3+）-induced β-amyloid protein 42 （Aβ42） aggregation and its depolymerization on formed Aβ42-Al3+ aggregates in vitro， and to investigate the effect of AE on the cytotoxicity of Aβ42 aggregation in the presence of Al3+. MethodThe Aβ42 group， Aβ42+Al3+ group， Aβ42+AE group， Aβ42+Al3++AE group and the depolymerization test group were set up in the experiment. The aggregation fibrosis process， aggregation morphology， aggregation size and cytotoxicity of Aβ42 in each group were detected by thioflavin T （ThT） fluorescence assay， transmission electron microscopy （TEM）， dynamic light scattering （DLS） experiment and thiazolyl blue （MTT） cytotoxicity assay. ResultCompared with the Aβ42 group， Al3+ could promote Aβ42 aggregation， increase the fluorescence intensity of ThT by 124.48%， induce the aggregation of Aβ42 to form fiber bundles with larger particle size， and significantly reduce the cell viability of human neuroblastoma SH-SY5Y cells （P<0.01）， thus reducing the cell survival rate to 51.05%. AE not only inhibited Aβ42 aggregation， but also inhibited Al3+-induced Aβ42 aggregation in a concentration-dependent manner. Compared with the Aβ42+Al3+ group， high concentration of AE could reduce the ThT fluorescence intensity to 41.66%， and change the polypeptide aggregation pathway to form amorphous aggregates with small particle size. Besides， it significantly inhibited the cytotoxicity of Aβ42 induced by Al3+ （P<0.01）， and restored the cell survival rate to 84.87%. Further depolymerization was conducted， AE could depolymerize Aβ42-Al3+ aggregates to make the formed aggregates disappear and form some small-particle short fibers and amorphous structure aggregates with low toxicity. ConclusionAE can inhibit Aβ42 aggregation and cytotoxicity in the presence of Al3+， depolymerize the formed Aβ42-Al3+ aggregates and alleviate the cytotoxicity， thus laying the foundation for exploring the mechanism of AE in the treatment of Alzheimer's disease.

Effect of Aloe-emodin on Aβ42 Aggregation and Cytotoxicity in Presence of Al3+ / 中国实验方剂学杂志

ObjectiveTo study the inhibitory effect of aloe-emodin （AE） on aluminum ion （Al3+）-induced β-amyloid protein 42 （Aβ42） aggregation and its depolymerization on formed Aβ42-Al3+ aggregates in vitro， and to investigate the effect of AE on the cytotoxicity of Aβ42 aggregation in the presence of Al3+. MethodThe Aβ42 group， Aβ42+Al3+ group， Aβ42+AE group， Aβ42+Al3++AE group and the depolymerization test group were set up in the experiment. The aggregation fibrosis process， aggregation morphology， aggregation size and cytotoxicity of Aβ42 in each group were detected by thioflavin T （ThT） fluorescence assay， transmission electron microscopy （TEM）， dynamic light scattering （DLS） experiment and thiazolyl blue （MTT） cytotoxicity assay. ResultCompared with the Aβ42 group， Al3+ could promote Aβ42 aggregation， increase the fluorescence intensity of ThT by 124.48%， induce the aggregation of Aβ42 to form fiber bundles with larger particle size， and significantly reduce the cell viability of human neuroblastoma SH-SY5Y cells （P<0.01）， thus reducing the cell survival rate to 51.05%. AE not only inhibited Aβ42 aggregation， but also inhibited Al3+-induced Aβ42 aggregation in a concentration-dependent manner. Compared with the Aβ42+Al3+ group， high concentration of AE could reduce the ThT fluorescence intensity to 41.66%， and change the polypeptide aggregation pathway to form amorphous aggregates with small particle size. Besides， it significantly inhibited the cytotoxicity of Aβ42 induced by Al3+ （P<0.01）， and restored the cell survival rate to 84.87%. Further depolymerization was conducted， AE could depolymerize Aβ42-Al3+ aggregates to make the formed aggregates disappear and form some small-particle short fibers and amorphous structure aggregates with low toxicity. ConclusionAE can inhibit Aβ42 aggregation and cytotoxicity in the presence of Al3+， depolymerize the formed Aβ42-Al3+ aggregates and alleviate the cytotoxicity， thus laying the foundation for exploring the mechanism of AE in the treatment of Alzheimer's disease.

Beta Thalassemia Major with Diabetes Mellitus: A Case Report

Thalassemia occurs due to defects in normal hemoglobin production. Globally it is most common inherited anaemia. Diabetes is a complication of b-thalassemia major. We report a case of Diabetes mellitus in a known case of beta thalassemia major. Patient had undergone splenectomy 1 year back. Patient is taking chelating agent Defasirox 1000mg orally once a day in the morning. Family history reveals, born through third degree consanguineous marriage. The patient was then subjected for laboratory examination reveals BSLwas 490, urine ketone 2+, urine sugar 3+, ABG was normal, HbA1c was 13 & 3 month old report of serum ferritin 1200 ng/dl. TFTand GH studies normal. Multidisciplinary management was instituted. Blood sugar level got controlled over subcutaneous insulin. Patient may have landed in Diabetic ketoacidosis but was promptly diagnosed & treated. This case is presented for its rarity. Due to increase in life expectancy of patient with thalassemia major, patient will expose more years of hyperglycemia and diabetes. Sustaining metabolic control and controlling cardiovascular risk factors helps to prevent future complications.

Neuroprotective effects of kukoamine A on 6-OHDA-induced Parkinson's model through apoptosis and iron accumulation inhibition / 中草药·英文版

Objective: Parkinson's disease (PD) is characterized by the loss of dopaminergic neurons in substantia nigra (SN). Our previous study demonstrated kukoamine A (KuA) to exhibit strong neuroprotective effects through antioxidative stress, and autophagy in MPTP/MPP

Effects of lead exposure and chelation therapy in different developmental stages on mouse short-term and long-term cognitive functions / 环境与职业医学

Background Lead (Pb) exposure impairs cognitive functions of children. Whether Pb exposure in different developmental stages induces long-term cognitive impairment, and whether chelation therapy could mitigate the cognitive impairment is rarely reported. Objective This experiment is designed to investigate effects of Pb exposure and chelation therapy during different developmental stages (breastfeeding, weaning, and early puberty periods) on mouse short-term and long-term cognitive functions. Methods C57BL/6 male mice in breastfeeding period, weaning period, and early puberty period (postnatal day 2, 21, and 41; PND 2, PND 21, and PND 41, n=30, respectively) were randomly divided into control, Pb exposure, and Pb+dimercaptosuccinic acid (DMSA) treatment groups (n=10 in each group). The control groups received standard food and deionized water. The Pb exposure mice received standard food and free drinking water containing Pb acetate (0.1% for dams, and 0.05% for pups). After receiving Pb acetate for 19 d, the Pb+DMSA treatment groups were given 1 mmol·kg−1·d−1 DMSA for 6 d with gastric infusion. Whole blood Pb levels were measured after DMSA treatment on experimental day 25. The effects on short-term cognitive function were tested in the Morris Water Maze task by the analyses of escape latency on PND 75−79, as well as target quadrant time and times of platform-crossing on PND 80. Hippocampal long-term potentiation of field excitatory postsynaptic potential (fEPSP) of mice on PND 365 was induced to demonstrate the effects on long-term cognitive function. Results The blood Pb levels among the Pb, Pb+DMSA, and control groups were statistically different for each developmental stage (Fbreastfeeding period=43.47, Fweaning period=228.6, Fearly period of puberty=274.2, all P<0.001). Compared to the counterpart control groups, blood Pb levels of the pb exposure groups (386.4, 265.0, and 178.1 μg·L−1 in breastfeeding period, weaning period, and early puberty period, respectively) were significantly higher for all stages. After the chelation therapy, the blood Pb significantly decreased for all stages (28.68, 47.29, and 20.93 μg·L−1 in the three periods, respectively, all P<0.001) and the Pb levels of the mice exposed in the breastfeeding period decreased most (by 92.58%, 82.15%, and 88.25% in the three periods, respectively, P<0.01). In the water maze task, the mice exposed to Pb in the breastfeeding period had a gentler decrease in escape latency (from 54.20 s on day 1 to 30.54 s on day 5, by 43.65 % decrease) than the control group (from 32.44 s on day 1 to 15.20 s on day 5, by 53.14 % decrease) (P<0.01) and a significant decrease in target quadrant time (P<0.05). After the chelation therapy, the escape latency of the DMSA-treated mice in the breastfeeding period (from 40.94 s on day 1 to 20.87 s on day 5, by 48.99 % decrease) was steeper than that of the Pb-exposed mice (P<0.05). The differences in the escape latency, target quadrant time, and times of platform-crossing were not significant between the Pb-exposed mice and the control mice in the weaning period and early period of puberty (all P>0.05). After the chelation therapy, such differences were also not significant compared with before therapy. Due to the small sample size, data were merged for different developmental stages in the long-term potentiation test. The amplitudes of fEPSP induced in the control, Pb-exposed, and DMSA treatment groups were significantly different (Fgroups=212.2, Ftime=11.36. P<0.001). The average fEPSP amplitude induced in the last 10 min recorded in the hippocampal slices in the Pb exposure group was significantly lower than that in the control group (P<0.05). After the DMSA treatment, no significant differences were observed in the fEPSP amplitudes between the Pb exposure group and the DMSA treatment group (P>0.05). When observing the fEPSP data by developmental stages, the fEPSP amplitude in the breastfeeding Pb-exposure group was 27.2% lower than that of the breastfeeding control group, while such changes were not obvious in the weaning period or in the early period of puberty. The fEPSP amplitude in breastfeeding DMSA treatment group was 44.3% higher than that of the breastfeeding Pb exposure group, while such changes were not observed in the weaning period or in early period of puberty. Conclusion Pb exposure during different developmental stages, especially in breastfeeding period, could affect short-term and long-term cognitive functions of mice. The harmful effects may be partially reversed by DMSA chelation therapy, especially being treated in breastfeeding period.

Chelation activity and protective effect of Caulerpa lentillifera aqueous extract against lead acetate-induced toxicity in Sprague Dawley rats

Lead is a multisystemic toxicant when it accumulates and is hazardous to health. This study was conducted to determinethe effect of Caulerpa lentillifera aqueous extract on lead accumulation in the internal organs and liver functions oflead-intoxicated rats. Sprague Dawley rats (n = 24) were divided into four groups (n = 6). Group I was healthy ratstreated with saline (2 ml/kg bwt) daily for 28 days. Group II was healthy rats treated with C. lentillifera aqueousextract (500 mg/kg bwt) daily for 28 days. Group III was intoxicated with lead acetate (20 mg/kg bwt) daily for 4 daysand then treated with saline (2 ml/kg bwt) for another 24 days. Group IV was intoxicated with lead acetate (20 mg/kgbwt) daily for 4 days and then treated with C. lentillifera aqueous extract (500 mg/kg bwt) for another 24 days. Leadaccumulation in the internal organs was measured by the atomic absorption spectrophotometer and the liver functionmarkers were determined using assay kits. Lead intoxication significantly (p < 0.05) increased lead accumulation inthe rats’ internal organs and affected their liver functions. C. lentillifera aqueous extract supplementation to leadintoxicated rats significantly (p < 0.05) decreased lead accumulation in the rats’ internal organs and protected theirliver functions. This study concludes that C. lentillifera aqueous extract possesses the capability of a chelating agentand attenuates the effect of lead on lead-intoxicated rats

Association of vitamin D deficiency and bone mass with liver and heart iron overload in transfusion dependent thalassemia

Background: Transfusion dependent thalassemia patients are reported to have Vitamin D insufficiency/deficiency in many countries. Vitamin D hydroxylation occurs in the liver; whether liver iron overload interferes with this step has not been addressed till date. This study helps to establish an association between liver iron concentration (LIC) and heart iron concentration (MIC) with vitamin D levels and Bone Mass Density in these patients.Methods: A cross sectional study was done by including transfusion dependent Thalassemia patients (TM) if they had an assessment of Liver and cardiac iron done by T2*MRI and bone mineral density by DEXA. Clinical data regarding age, gender, type of iron chelation therapy and laboratory data of S. ferritin and Vitamin D was collected. Data was assessed using appropriate statistical methods.Results: Among 40 TM patients were taken and mean age was 17.6 years. Vitamin D deficiency was identified in 26(65%). 20 out of them had an LIC>7mg/g DW and 6 had MIC>1.65mg/g DW. There was a significant association between LIC>7mg/g and vitamin D level<20 ng/ml and a significant inverse correlation between LIC and vitamin D, suggesting that liver iron overload may indeed affect vitamin D metabolism. Osteopenia was present in 32.5% and osteoporosis was present in 27.5 % of all TM patients. Reduced Bone Mass Density was also found to be linked with iron over load.Conclusions: Regular monitoring of vitamin D levels and supplementation is required in patients with severe liver and heart iron load. More studies are needed to confirm these results.

Hearing Loss in Thalassemic Children on Chelation Therapy

Background: Thalassemia is a common genetic hematological disorder worldwide. It is also common in North India includingJammu region. These patients need lifelong repeated blood transfusions and iron chelation therapy for their survival. Chelationtherapy is known to be associated with various complications including sensorineural hearing loss (SNHL). Till now, no dataare available regarding SNHL in pediatric thalassemia major patients in Jammu region. Hence, we planned a study to assessthe prevalence of hearing loss in children with thalassemia major in the age group of 10–20 years.Methods: All the children with beta-thalassemia major in the age group of 10–20 years registered with Thalassemia Day CareCenter, Department of Pediatrics, SMGS Hospital, Government Medical College, Jammu, were enrolled in this cross-sectionalstudy. Hearing was assessed by pure tone audiometry. Clinical and demographic data of these patients were recorded onpretested pro forma and analyzed.Results: A total number of 34 children with thalassemia in the age group of 10–20 years were enrolled in this study whichcomprised 18 males and 16 females. Out of these 34 patients, 5 (14.7%) were found to have SNHL and 1 (2.9%) had conductivehearing loss. Four of the five patients in SNHL group had low- as well as high-frequency mild hearing loss (25–40 db) whileone patient had high-frequency mild hearing loss at 4000 HZ. Four out of these five patients had unilateral hearing loss on theleft side while one had bilateral SNHL. Two out of five patients in the SNHL group were taking chelation therapy in the form ofcombination of deferiprone and deferasirox at the dose of 75–100 mg/kg/day and 30–40 mg/kg/day, respectively, for more than5 years. The other three patients were taking only deferasirox at the dose of 30–40 mg/kg/day for more than 5 years.Conclusions: Regular blood transfusions and chelation therapy are essential for long-term survival of thalassemia majorpatients but are also associated with complications like SNHL.

Diving deep into chelation therapy for coronary artery disease: a review

Chelation therapy is still a mainstay therapy when it comes to dealing with heavy metal intoxication. The ability of various chelators to bind metal and other chemical molecules led to the idea whether chelation therapy can be used as an alternative therapy to enchain calcium element that is known to be present in the atherosclerotic plaque. Various studies have been conducted, one of which is a large trial to assess chelation therapy study to show in case ethylenediaminetetraacetic acid (EDTA) chelator can be proven by evidence-based in managing coronary heart disease. Despite the favorable results that were found in many literature studies, the results of the study are still under debate in various aspects. To address this issue, we conducted a review article to discuss comprehensively the general description of EDTA as chelation therapy, various mechanisms that can explain the use of EDTA in the management of coronary heart disease, the pharmacokinetic aspects of EDTA chelation therapy, as well as describing various existing studies with a good level of evidence to review the effectiveness of these therapies against coronary artery disease.

Knowledge, attitude and practice in parents of chronically transfused thalassemic patients regarding thalassemia in thalassemia day care unit in government medical college, Amritsar, Punjab, India

Background: Thalassemia is a chronic debilitating disease that affects nearly 200 million people worldwide. A caregiver who has good knowledge regarding the disease can not only provide a better quality of care to his/her ward but also may spread knowledge in the society in which he/she lives which helps immensely in raising community awareness related to the disease.Methods: 50 caregivers (either mother or father) of chronically transfused thalassemic children were questioned regarding their knowledge, attitude and practice towards this disease, using a self-constructed questionnaire.Results: It was found that despite adequate knowledge regarding every aspect of this disease, there is a lack of attitude of practice towards prevention of this disease in their subsequent child or in their near and dear ones.Conclusions: There is a need to promote an attitude of practice in prevention of the birth of thalassemic children as mere knowledge regarding the disease is not enough in the present scenario where the disease burden is increasing as is the financial and emotional burden on the families. Role of Government sponsorship, a social worker and a child psychologist in the Thalassemia day care unit cannot be minimized.

Impacts of transfusion in β-thalassemia major patients receiving multiple blood transfusions

Background: Beta-thalassemia major is one of major public health problems in India. Thalassemia major is a transfusion-dependent severe anemia and these children experience various problems if the transfusion is inadequate but at the same time repeated blood transfusions are associated with hazards like iron overload.Methods: This study was conducted at thalassemia ward of S.M.S Hospital and J.K.LON paediatrics Hospital, Jaipur from April 2012 to Nov. 2013. 145 thalassemia patients on transfusion therapy attending outdoor or being admitted were assessed after obtaining due permission from the authorities and consent from the guardian or parents of patients.Results: Present study comprised 145 beta-thalassemia major patients with 51 (35%) females and 94 (65%) males in which youngest patient is 3 years old and the oldest 33 years. 104 (72%) were Hindus and 41 (28%) were Muslims. Consanguinity was found in a significant proportion 16% (23) of the parents of the patients. 137 (94%) patients had serum ferritin ³1000ng/ml. Out 145 thalassemic patients, 108 (74.5%) have total serum bilirubin >1(mg/dl), 103 (71%) have SGPT level >35IU, 103 (71%) have SGOT level >40IU), 38 (26.2%) have serum alkaline phosphatase level >390 IU, 35 (24.1%) have serum creatinine level >1.6mg/dl, 42 (29%) have serum urea level >45mg/dl, 28 (19.3%) have serum uric acid level >6mg/dl.Conclusions: It is suggested to revise and devise suitable transfusion regime so that a balance between adequate transfusion and minimum side effects of multiple transfusions is maintained. Systemic effects of multiple transfusions should be rigorously and meticulously studied.

Evaluation Of Free Radical Scavenging Ability And Antiradical Activities Of Ximenia Caffra Fruit Extracts At Different Ripening Stages

Objective: To evaluate the free radical scavenging ability and antiradical activities of Ximenia caffra fruit extracts in their different ripening stages. Methods: Using standard procedures, Ximenia caffra fruit extracts were determined for ferrous ions chelating ability, nitric oxide and hydroxyl radical scavenging ability. Antiradical activities were assessed using 1,1-diphenyl-2-picrylhydrazyl (DPPH) in which the fruit extracts were evaluated for the number of antiradical units (AU515), the number of antiradical units per 1 mg of extracts (EAU515) and the total number of antiradical units per 1g of raw fruits (TAU515). Results: The fruit extracts exhibited significant higher ferrous ion chelating and free radical scavenging capacity compared to synthetic antioxidants (standard). Fruit extracts in early ripening stage (ERS) exhibited stronger ion chelation, nitric oxide and hydroxyl radical scavenging ability with low effective fruit extract concentration required to reduce free radicals by 50% (EC50) that were 14, 25 and 30µg/ml respectively than the late ripening stage (LRS) extracts. The fruit extracts also showed high AU515, EAU515 and TAU515. The values of AU515 ranged from 0.93 to 0.95, while EAU515 from 78.30 to 79.34 and TAU515 ranged from 19762.46 to 23821.23 of the extracts in LRS and ERS respectively. Conclusion: Based on these observations Ximenia caffra is potentially beneficial to human health due to its strong ability to scavenge free radicals. Its utilization can potentially reduce the risk of degenerative diseases to human beings.

Study of iron overload assessment by T2* magnetic resonance imaging in patients with myelodysplastic syndromes / 中华血液学杂志

Objectives: To analyze the cardiac T2* value, liver iron concentration (LIC) , and related laboratory parameters in myelodysplastic syndrome (MDS) with iron overload and evaluate the changes of organ functions after iron chelation therapy. To explore the value of magnetic resonance imaging (MRI) T2* in making early diagnosis and assessing organs iron overload. Methods: Retrospective investigation was used to observe the cardiac T2* value, LIC, iron metabolism parameters and related laboratory parameters of 85 MDS patients from Nov 2014 to Jan 2018. Among them, 7 MDS patients with Low/Int-1 have received iron chelation therapy for 6 months during two MRI examinations. The above parameters were collected before and after iron chelation therapy for comparison. Results: Correlations were found between heart T2* value and age (rs=-0.290, P=0.007) and left ventricular ejection fraction (LVEF) (rs=0.265, P=0.009) . There was a significant negative correlation between heart T2* value and blood transfusion units (rs=-0.701, P<0.001) . There was a significant positive correlation between LIC and serum ferritin (SF) (rs=0.577, P<0.001) . There was also a correlation between LIC and ALT (rs=0.268, P=0.014) and blood transfusion units (rs=0.244, P=0.034) . There was no correlation between heart T2* and pro-BNP, SF (all P>0.05) , and no correlation between LIC and age (P>0.05) . The increase of heart T2* between the normal and abnormal groups was statistically significant (P=0.005) , but the iron overload ratio of the heart T2*<20 ms was not significant between the two groups. There was statistical significance in the proportion of severe liver iron overload (LIC>15 mg/g DW) (P=0.045) . After iron chelation therapy, the values of SF, transferrin saturation, ALT, AST, pro-BNP and LIC of 7 patients were decreased compared with values before iron chelation therapy, and the peripheral blood cell level was increased. However, the changes of LVEF and T2* values after iron chelation were not obvious. Conclusion: MRI T2* may be a predictor of iron overload in patients with MDS in early stage, and may be more valuable compare with LVEF, SF and other laboratory indicators. The safety and repeatability of MRI cardiac T2* examination are recognized, and it can be used as an ideal detection for patients with iron overload.