Transfusion-dependent Patients Research Articles

Association between HLA-DRB1*04, HLA-DQB1*03, and HLA-DQB1*06 with alloimmunization in transfusion-dependent patients with thalassemia: the first case-control study in Iran.

Transfusion therapy is crucial for treating Transfusion-dependent thalassemia (TDT) patients. However, the production of Alloantibodies presents a substantial challenge for these individuals and impacts their quality of life. The Rh and Kell blood group antigens are particularly susceptible to alloantibody development. This study aims to establish the correlation between HLA-DRB1*04, HLA-DQB1*03, and HLA-DQB1*06 alleles and alloimmunzation in thalassemia patients from Iran. 98 thalassemic patients were recruited for this study (49 alloimmunized and 49 non-alloimmunized). Alloimmunized patients developed Rh and Kell specificities alloantibodies. The two groups were compared based on the results of HLA-DRB1 and HLA-DQB1 genotyping conducted using Sequence-Specific Primers (SSP-PCR). The findings from the antibody screening revealed that the predominant alloantibody detected was Anti-K (95.9%), Anti-E (65.3%), Anti-C (30.6%), Anti-D (28.6%), Anti-c (10.2%), Anti-e (2%), and Anti-k (2%). There was a notable difference in HLA-DQB1*03 between alloimmunized and non-alloimmunized groups, 41.8% vs. 58.2%, respectively. (iP = 0.001, OR = 0.135, CI = 0.036-0.499). There was not any notable relationship between HLA-DRB1*04 and HLA-DQB1*06 alleles and alloimmunization. Our findings indicate that HLA-DQB1*03 may have a protective role in preventing alloantibody production. Thus, HLA-typing, particularly focusing on DQB1*03, can significantly enhance the screening process, leading to improved blood transfusion management, reduced rejection of hematopoietic stem cell transplantation, and minimized blood transfusion complications.

Impact of chelation therapy on serum zinc levels in transfusion dependent thalassaemia patients

Background: Children with thalassaemia major receive regular blood transfusions, which can lead to iron deposition due to the absence of excretory mechanisms. To overcome this, chelating agents like deferasirox (DFX), deferiprone (DFP), and deferoxamine (DFO) are used which affect zinc levels. Zinc deficiency in thalassaemia patients may cause immunodeficiency, aberrant haematopoiesis and delay in growth and development. Objectives: Measurement of serum zinc levels in children with transfusion-dependent thalassaemia (TDT) receiving chelating agents. Method: This cross-sectional study analysed 77 registered children with TDT in Sri Guru Ram Das Institute of Medical Sciences and Research, Sri Amritsar, India, without zinc supplementation for last 3 months. Data were collected through detailed history and chelation therapy details. Zinc levels were measured using atomic absorption spectrometry. Results: Of 73 (94.8%) children with TDT who were on chelation therapy, 35 (47.9%) were only on DFX chelation, 37 (50.7%) were receiving a combination of DFX+DFP and one (1.4%) patient was receiving a combination of DFO+DFX. In the DFX group, the mean serum zinc level was 77.1+14.13µg/dL which was significantly higher when compared to the mean serum zinc level in DFX+DFP group which was 58.3+7.38µg/dL and DFO+DFX where the mean serum zinc value was 38.4 µg/dL (p value <0.01). Zinc had significant negative correlation with the duration of chelation with correlation coefficient r=-0.231 and p value 0.049. Conclusions: Children with thalassaemia who were on more than one chelating agent had lower serum zinc levels. Zinc had significant negative correlation with the duration of chelation.

Quality of life in transfusion dependent thalassemia patients at national institute of child health.

Objective: To determine the quality of life of children suffering from transfusion dependent thalassemia. Study Design: Case-control study. Setting: Department of Pediatrics, National Institute of Child Health, Karachi, Pakistan. Period: March 2024 to August 2024. Methods: Children of either gender, aged between 6 months to 14 years, and having transfusion dependent thalassemia were included as cases (n=95). For controls (n=95), healthy volunteers without chronic disease, matched in terms of gender and age were included. At the time of enrollment, demographical data like age, gender, height, and weight were noted. Relevant laboratory investigations were performed and evaluated. Children of both groups along with their parents were interviewed about quality of life administering WHOQOL-BREF questionnaire. Results: In a total of 190 children (95 children in each group), 104 (54.7%) were male and 86 (45.3%) female. Physical health scores were 35.62±6.68 in children with transfusion dependent thalassemia vs. 74.92±7.92 in controls (p<0.001). Psychological health scores were 38.16±8.12 vs. 76.68±6.66 (p<0.001) among transfusion dependent thalassemia children versus controls, respectively. Social relationships were 41.19±6.14 vs. 77.50±7.01 (p<0.001) among transfusion dependent thalassemia children versus controls, respectively. Environmental scores were 43.51±6.93 vs. 71.59±6.06 (p<0.001), among transfusion dependent thalassemia children versus controls, respectively. Conclusion: Our study highlights the significant negative impact of transfusion-dependent thalassemia on the quality of life of affected children across all domains of the WHOQOL-BREF questionnaire.

Blood use and alloimmunization in myelodysplastic syndrome patients: A study of a hospital transfusion experience.

Blood use and alloimmunization in myelodysplastic syndrome patients: A study of a hospital transfusion experience.

Long-term complications, survival and mortality in splenectomised adult transfusion-dependent thalassemia patients.

Long-term complications, survival and mortality in splenectomised adult transfusion-dependent thalassemia patients.

Correlation study between magnetic resonance imaging-quantitated cardiac iron deposition and left ventricular function in patients with β-thalassemia major in China.

To explore the correlation between cardiac iron deposition and left ventricular function indicators, such as left ventricular end-diastolic volume index, left ventricular end-systolic volume index, and left ventricular ejection fraction, and to evaluate the accuracy of predicting patients with cardiac iron deposition by using left ventricular ejection fraction as an index. This quantitative cross-sectional study involved one hundred and fifty transfusion-dependent patients with β-thalassemia major who were evaluated by magnetic resonance imaging to obtain T2* values, left ventricular end-diastolic volume index, left ventricular end-systolic volume index, and left ventricular ejection fraction. The relationship between cardiac R2* values and left ventricular end-diastolic volume index, left ventricular end-systolic volume index, and left ventricular ejection fraction was analyzed. Out of the 150 patients, cardiac iron overload was not observed for 92 patients, 42 patients exhibited mild to moderate cardiac iron overload, and 16 patients were severe cardiac iron overloaded. A linear correlation was not observed between the cardiac R2* values and left ventricular end-diastolic volume index, left ventricular end-systolic volume index, and left ventricular ejection fraction (P > 0.05). For the left ventricular ejection fraction index, the sensibility, specificity, positive predictive value, negative predictive value, and the agreement rate were 83.3%, 63.2%, 8.6%, 98.9%, and 64.0%, respectively; also, the area under the receiver operating characteristic curve was 0.348. A linear correlation was not observed between cardiac R2* values and left ventricular end-diastolic volume index, left ventricular end-systolic volume index, and left ventricular ejection fraction in patients with β-thalassemia major. Therefore, using left ventricular ejection fraction as an indirect index to predict cardiac iron deposition may be not reliable in clinical practice.

First Ever Pediatric Allogeneic Hematopoietic Stem Cell Transplantation In A Transfusion Dependent Thalassemia Patient In Bangladesh- A Case Report With 17 Months Post-Transplant Follow Up

Background: Hematopoietic stem cell transplantation (HSCT) is the mainstay of treatment for many disease conditions. The first allogeneic HSCT was done by E. Donnall Thomas which was reported in the New England Journal of Medicine in September 1957. In that study six patients of Acute Leukemia were treated with radiation and chemotherapy followed by intravenous infusion of marrow from a normal donor where only two patients were engrafted but all died within 100 days of transplantation. In 1979 Thomas applied allogeneic HSCT earlier in the course of acute leukemia and reported a 50% cure rate in AML patients transplanted after first remission. In Bangladesh there was no scope of HSCT for thalassemia patients. The first ever pediatric allogeneic HSCT in a transfusion dependent thalassemia patient was performed in BMT center of CMH Dhaka on 30 November 2021. Here the case is reported with 17 months post-transplant follow up. Case Report: Arfin a 3 years 11 months old boy first reported in November 2018 with the history of repeated blood transfusion since 6 months of age and was diagnosed as a case of Hb-E Beta thalassemia. On initial presentation he had hepatomegaly, splenomegaly, stunting, high serum ferritin level, and high liver iron concentration and was treated with regular irradiated and filtered PRBC with multiple iron chelating agents. On 30 November 2021 allogeneic HSCT of that patient was successfully completed. Donor was his elder brother who was Hb-E trait and had 10/10 HLA full matches. In April 2023 after 17 months during post-transplant follow up his Hb was 10.3 gm/dl, serum ferritin level was normal, had normal growth and required no further blood transfusion after transplantation. Conclusion: In transfusion dependent thalassemia patients allogeneic HSCT is curative. This is the first and successful case of allogeneic HSCT in thalassemia patients in Bangladesh. J Bangladesh Coll Phys Surg 2025; 43: 77-80

Transfusion efficacy of leucoreduced packed red blood cells prepared by two different methods: A randomized controlled trial in transfusion-dependent thalassaemia patients (FUEL trial).

Great variations may be observed in the haemoglobin (Hb) content of packed red blood cell (PRBC) units prepared by different methods. This study aimed to assess the Hb increment in thalassaemia major patients transfused with leucoreduced PRBCs (LPRBCs) prepared by two different methods: (i) standard leucoreduced PRBCs (SLPRBCs) and (ii) leucoreduced PRBCs prepared by a new method where leucoreduction of whole blood is done first (NLPRBCs). This prospective, randomized, controlled trial included 80 adult thalassaemia major patients who were randomized into two groups of 40 each. Group I patients received SLPRBC and those of Group II received NLPRBC transfusions for 3 months. SLPRBCs had a mean (±SD) volume of 275.50 ± 17.07 mL, while it was 316.46 ± 1.42 mL for NLPRBCs (p < 0.001). The mean Hb content of SLPRBCs was 50.60 ± 5.12 g, while that of NLPRBCs was 56.98 ± 5.92 g (p < 0.001). The mean Hb increment in Group I patients was 2.11 ± 0.89 g/dL, while that of Group II patients was 2.48 ± 0.88 g/dL (p < 0.001). The mean transfusion interval for Group I patients was 20.30 ± 3.75 days, while it was 21.34 ± 5.13 days for Group II patients (p < 0.045). A significant positive correlation was observed between the Hb dose transfused and the Hb increment with both SLPRBC (ρ = 0.4, p < 0.001) and NLPRBC (ρ = 0.19, p = 0.011) transfusions. NLPRBCs had significantly higher Hb content than the SLPRBCs, leading to a better Hb increment post transfusion, which may potentially prolong the transfusion interval in thalassaemia major patients.

Efficacy and safety of hydroxyurea therapy on patients with β-thalassemia: a systematic review and meta-analysis.

Our aim is to review the safety and efficacy of hydroxyurea (HU) on β-thalassemia patients. Studies that evaluated the safety and efficacy of HU on β-thalassemia patients were searched in Pub-Med, Cochrane Databases, Web of Science, China-Biology-Medicine, CNKI, Embase, VIP, and WanFang data. The proportions of response rate (RR) (50% fall in transfusion need in transfusion-dependent β-thalassemia patients, or 1 g/dL elevate in hemoglobin (Hb) levels in transfusion-independent β-thalassemia patients) and good RR (transfusion-free in transfusion-dependent β-thalassemia patients or 2 g/dL elevate in Hb levels in transfusion-independent β-thalassemia patients) were utilized to evaluate the effect size (ES). The secondary outcomes were the adverse events incidence rates of HU in β-thalassemia patients. Two randomized controlled trials (RCTs) and 25 single-armed observational studies with typically 1,748 individuals were involved in our analysis. All 27 clinical trials were reported with fair quality. HU, in transfusion-dependent β-thalassemia patients, was related to a significant decrease in transfusion requirements [a pooled RR of 0.37 and a pooled good RR of 0.65 (95% CI, 0.53-0.76)]; in transfusion-independent β-thalassemia patients, it was correlated to an excellent raise in Hb levels [a pooled RR of 0.20 (95% CI, 0.08-0.35) and a pooled good RR of 0.53 (95% CI, 0.41-0.65)]. Neutropenia and leucopenia were the most prevalent adverse events in β-thalassemia patients treated with HU, while the incidence rates of other side effects were relatively lower. Our findings demonstrated that β-thalassemia patients tolerated and responded well to HU. Due to the control arms absence in the involved studies, more double-masked RCTs are essential for proving the safety and efficacy of HU in β-thalassemia patients.

The Effect of Single Nucleotide Polymorphisms on Clinical Phenotypes of Sabahan Transfusion-Dependent β-Thalassemia Patients with Homozygous Filipino β0-Deletion

Sabah has the highest prevalence of β-thalassemia in Malaysia, with the Filipino β0-deletion as the predominant mutation. Patients with the homozygous Filipino β0-deletion exhibit phenotypic heterogeneity due to various genetic modifiers, yet the effects of these modifiers on the clinical phenotype remain poorly understood. This study investigated the effects of the coinheritance of α-thalassemia, XmnI-Gγ rs7482144, BCL11A rs766432, and 5’HS4 rs16912979 polymorphisms on the clinical phenotype of homozygous Filipino β0-deletion patients in Sabah. Molecular analyses were performed on 124 homozygous Filipino β0-deletion patients using gap-PCR, PCR-RFLP, multiplex PCR, ARMS-PCR, gel electrophoresis, and DNA sequencing. Data showed that the coinheritance of the –α3.7 deletion significantly affected the clinical phenotypes of homozygous Filipino β0-deletion patients (p < 0.05). Patients with the –α3.7/–α3.7 genotype (5.6%) had a less severe clinical phenotype compared to those with the αα/αα (71.8%) and –α3.7/αα (22.6%) genotypes. Our data further revealed that the MAFs of the XmnI-Gγ rs7482144 and BCL11A rs766432 polymorphisms in these patients were 0.032 and 0.194, respectively. Interestingly, none of these single nucleotide polymorphisms significantly influenced the clinical phenotype of the patients. The effect of the 5’HS4 rs16912979 polymorphism on the clinical phenotype could not be assessed due to its rarity (1.6%). However, a novel 5’HS4 c.733+G mutation was identified, warranting further investigation of its potential impact on β-thalassemia pathogenesis. Our findings indicate that the clinical phenotype of patients with the homozygous Filipino β0-deletion is strongly influenced by the coinheritance of the –α3.7 deletion, but not by the XmnI-Gγ rs7482144 and BCL11A rs766432 polymorphisms.

Inflammatory Monocyte Subsets Correlation with Iron Levels in Low Vitamin D Pediatric Transfusion-Dependent Thalassemia.

Patients with transfusion-dependent thalassemia experience iron dysregulation, which affects the immune response. Surface proteins such as FcγRIII (CD16), lipopolysaccharide receptor (CD14), and human leukocyte antigen (HLA-DR) on monocytes are crucial for innate and adaptive responses. Blood monocytes, identified by their CD14 and CD16 expression, show functional diversity during injury or inflammation. Considering the mechanisms of vitamin D activation and its potential interaction with monocytes, further investigation of its immunomodulatory role in transfusion-dependent thalassemia is essential. This study evaluated monocyte subsets, population, and surface receptor expression (CD14, CD16, and HLA-DR), and their association with iron status and vitamin D levels in patients with transfusion-dependent thalassemia. Fifty lysed erythrocyte-heparinized whole blood samples from transfusion-dependent thalassemia patients were analyzed by flow cytometry and classified into three monocyte subsets: CD14++CD16- (classical), CD14++CD16+ (intermediate), and CD14+CD16++ (non-classical). Cell percentage referred to the monocyte subset population. Median fluorescence intensity (MFI) indicated surface protein expression. The 25(OH)vitamin D level was used to measure vitamin D levels. Iron status was assessed using ferritin and serum iron levels. A correlational study was performed. We did not find a correlation between low vitamin D levels (22.9 ng/mL ± 3.9) and monocyte characteristics, iron status, or hematology profile. However, we observed a negative correlation between the percentage of intermediate and non-classical monocytes and hemoglobin and ferritin levels (P = 0.02, r = -0.3; P = 0.04, r = -0.3). Additionally, we found a positive correlation between the median fluorescence intensity (MFI) of CD14 in non-classical monocytes and serum iron (P = 0.04, r = 0.3). Our findings suggest that iron overload and anemia may influence the function of inflammatory monocyte subsets. Considering the immunomodulatory role of vitamin D through monocyte modulation during pathogen insult, further research utilizing a whole-blood stimulation assay is imperative.

Luspatercept: a treatment for ineffective erythropoiesis in thalassemia.

Patients with β-thalassemia continue to have several unmet needs. In non-transfusion-dependent patients, untreated ineffective erythropoiesis and anemia have been associated with a variety of clinical sequelae, with no treatment currently available beyond supportive transfusions. In transfusion-dependent forms, lifelong transfusion and iron chelation therapy are associated with considerable clinical, psychological, and economic burden on the patient and health care system. Luspatercept is a novel disease-modifying agent targeting ineffective erythropoiesis that became recently available for patients with β-thalassemia. Data from randomized clinical trials confirmed its efficacy and safety in reducing transfusion burden in transfusion-dependent patients and increasing total hemoglobin level in non-transfusion-dependent patients. Secondary clinical benefits in patient-reported outcomes and iron overload were also observed on long-term therapy, and further data from real-world evidence studies are awaited.

Challenges in hospice and end-of-life care in the transfusion-dependent patient.

Despite promising advances leading to improved survival, many patients with hematologic malignancies end up dying from their underlying disease. Their end-of-life (EOL) care experience is often marked by worsening symptoms, late conversations about patient values, increased healthcare utilization, and infrequent involvement of palliative care and hospice services. There are several challenges to the delivery of high-quality EOL care that span across disease, patient, clinician, and system levels. These barriers include an unpredictable prognosis, the patient's prognostic misunderstandings and preference to focus on the immediate future, and the oncologist's hesitancy to initiate EOL conversations. Additionally, many patients with hematologic malignancies have increasing transfusion requirements at the end of life. The hospice model often does not support ongoing blood transfusions for patients, creating an additional and substantial hurdle to hospice utilization. Ultimately, patients who are transfusion-dependent and elect to enroll in hospice do so often within a limited time frame to benefit from hospice services. Strategies to overcome challenges in EOL care include encouraging repeated patient-clinician conversations that set expectations and incorporate the patient's goals and preferences and promoting multidisciplinary team collaboration in patient care. Ultimately, policy-level changes are required to improve EOL care for patients who are transfusion-dependent. Many research efforts to improve the care of patients with hematologic malignancies at the end of life are underway, including studies directed toward patients dependent on transfusions.

Early Engraftment and Immune Kinetics Following Allogeneic Transplant Using a Novel Reduced-Toxicity Transplant Strategy in Children/Adolescents with High-Risk Transfusion-Dependent Thalassemia: Early Results of the ThalFAbS Trial.

Early Engraftment and Immune Kinetics Following Allogeneic Transplant Using a Novel Reduced-Toxicity Transplant Strategy in Children/Adolescents with High-Risk Transfusion-Dependent Thalassemia: Early Results of the ThalFAbS Trial.

Regularity of blood transfusion influences the severity of systemic iron burden, cognitive decline, and gut dysbiosis in thalassemia patients

AbstractBackgroundThalassemia is a hereditary disease with impaired red blood cell production, resulting in cumulative systemic iron burden. The life‐long therapeutic blood transfusion with or without iron chelators in those patients leads to the development of early‐onset neurocognitive decline. However, the effects of regularity of blood transfusion on the severity of iron burden, cognitive decline, and gut dysbiosis in thalassemia patients are still unclear.MethodSixty participants, including 20 transfusion‐dependent (TDT) patients, 20 non‐transfusion‐dependent (NTDT) patients, and 20 healthy controls, underwent evaluation for neurocognitive function using the Montreal Cognitive Assessment (MoCA). The current systemic iron burden was determined by the levels of serum ferritin. Amplicon‐based sequencing of the bacterial 16s rRNA V3‐V4 regions of stool DNA was employed to delineate profiles of gut microbiota.ResultThe TDT patients showed greater severity of systemic iron burden, cognitive decline and gut dysbiosis than the NTDT patients. The TDT group was particularly afflicted with systemic iron burden as indicated by the highest levels of serum ferritin (Fig. 1A). Compared to the controls, the TDT and NTDT groups had significantly lower MoCA scores (Fig. 1B). In addition, there was a negative correlation between MoCA scores and the levels of serum ferritin (Fig. 1C). The thalassemia patients also exhibited gut dysbiosis, with microbial compositions among the three groups being distinct from each other (Fig. 1D and 1E). Interestingly, an increase in abundance of Paraclostridium was associated with cognitive decline.ConclusionThese findings suggest that the severity of systemic iron burden, based on the regularity of blood transfusion, impacts on the magnitude of cognitive decline and gut dysbiosis in thalassemia patients. It is possible that systemic iron potentially serves as a dose‐dependent factor in the pathogenesis of neurodegeneration through gut dysbiosis. Therefore, therapeutic approaches via well‐control of systemic iron levels and balanced gut microbiota could prevent early‐onset cognitive decline in thalassemia patients.

Association of Serum Ferritin With Growth and Endocrine Function in Thalassemia Major Children in North India: An Observational Study.

Background Thalassemia is the most common form of hereditary anemia caused by the impaired synthesis of one of the two globin chains in hemoglobin. A decrease in beta-globin chains occurs in beta-thalassemia, resulting in a relative excess of alpha-globin chains. Thalassemia major is the severe form of thalassemia, which requires frequent blood transfusions for survival. Consequently, the natural course of the disease is affected by blood transfusion-related side effects. Repeated blood transfusions lead to the accumulation of iron in tissues such as the liver, heart, and endocrine glands. Serum ferritin is a biomarker of iron overload. Endocrinopathies are among the most frequently observed complications in thalassemia. Early recognition and treatment are important in order to prevent late irreversible sequelae and improve the quality of life of these patients. This study was conducted to evaluate growth parameters and endocrine function in children with thalassemia major and their relation with serum ferritin. Methods This prospective observational study included all patients between the age groups six months and 14 years with transfusion-dependent thalassemia. We included 62 children admitted during the study period fulfilling eligibility criteria. The data was analyzed using descriptive statistics and making comparisons among various groups. Spearman correlation analysis was done to assess the correlation between serum ferritin and thyroid hormone. The difference of means across the groups was tested with the Mann-Whitney U test for two groups and the Kruskal-Wallis test for more than two groups. Results The mean age of the study participants was 5.66 ± 3.77 years, with the largest group consisting of children aged one to three years, comprising 40.3% of the participants. The majority of participants were boys. This study showed a high prevalence of endocrinopathies in transfusion-dependent thalassemic patients. The most common endocrinopathywas short stature(37.1%), followed by impaired glucose tolerance (28.6%), subclinical hypothyroidism (14.5%), and parathyroid dysfunction (14.5%). Overt diabetes and pubertal delay were not seen. A statistically significant association of ferritin was found with age (p < 0.001), stature (p = 0.001), thryroid-stimulating hormone (TSH) (p = 0.004), and parathyroid function (p = 0.006). Conclusions The prevalence of endocrinopathies in present transfusion-dependent thalassemic cohorts was considerably high, presenting as short stature, impaired glucose tolerance, hypoparathyroidism, and subclinical hypothyroidism. The study showed a weak positive correlation of endocrinopathies with serum ferritin levels. Hence, irrespective of serum ferritin levels, patients with transfusion-dependent thalassemia can have a considerably high prevalence of endocrine complications.

A phase 2 clinical trial of luspatercept in non-transfusion-dependent patients with myelodysplastic syndromes

Luspatercept has shown durable clinical efficacy for the treatment of anemia in transfusion-dependent patients with lower-risk myelodysplastic syndromes (LR-MDS). We report the results of a prespecified primary analysis of a phase 2 trial of luspatercept in non-transfusion-dependent (NTD) Japanese patients with anemia due to LR-MDS. Luspatercept (starting dose 1.0 mg/kg) was administered subcutaneously once every 3 weeks. The primary endpoint was the proportion of patients who achieved hematological improvement-erythroid (HI-E) response (≥ 1.5 g/dL increase in hemoglobin level for 8 weeks) without transfusions within the first 24 weeks of treatment. At the primary analysis data cutoff, 21 patients had been enrolled/treated; 17 and 10 patients had completed 24 and 48 weeks of treatment, respectively. HI-E response occurred within 24 weeks in 10 patients (47.6%; 95% confidence interval, 25.7–70.2; P < 0.0001), which was significantly higher than the predefined threshold (10%). By week 48, HI-E response occurred in 12 patients (57.1%) and 17 patients (81.0%) remained NTD. Luspatercept was well tolerated. Three patients (14.3%) had grade 3–4 treatment-related treatment-emergent adverse events. Luspatercept resulted in statistically and clinically significant improvements in hemoglobin levels, and may help delay the need for transfusions in NTD patients with LR-MDS.

Role of Malondialdehyde Levels in The Occurrence of Hypogonadism in Transfusiondependent Thalassemia Male Patients

Role of Malondialdehyde Levels in The Occurrence of Hypogonadism in Transfusiondependent Thalassemia Male Patients

Real-world impact of luspatercept on red blood cell transfusions among patients with myelodysplastic syndromes: A United States healthcare claims database study

Real-world impact of luspatercept on red blood cell transfusions among patients with myelodysplastic syndromes: A United States healthcare claims database study

Intravenous administration of iron dextran as a potential inducer for hemochromatosis: Development of an iron overload animal model.

Iron overload in transfusion-dependent thalassemia patients represents a significant public health challenge due to its high mortality rate and risks of severe complications. Therefore, developing safe and effective therapeutic modalities for managing iron overload is critical, as current animal models inadequately replicate human conditions. The aim of this study was to investigate the effects of intravenous iron dextran on hepatocyte morphology, liver iron concentration, and serum iron profile changes as a model for hemochromatosis. An experimental design with a post-test-only control group method was conducted using animal models. Fifty rats were used and divided into ten groups, nine received different intravenous doses of iron dextran: 10, 20, 30, 40, 50, 60, 80, 100, and 120 mg/kg body weight (BW) and a control group received no treatment. The results showed that intravenous iron dextran starting at a dose of 10 mg/kg BW caused significant changes in liver iron concentration while starting at 20 mg/kg BW significantly affected hepatocyte morphology, transferrin levels, unsaturated iron binding capacity, serum iron levels, and transferrin saturation. Intravenous iron dextran starting at 40 mg/kg BW resulted significant changes in the level of total iron binding capacity compared to control group. In conclusion, intravenous iron dextran significantly altered hepatocyte morphology, increased liver iron concentration, and modified the serum iron profile, reflecting changes that might be observed in patients with transfusion-dependent thalassemia.