Growth and endocrinopathies among children with ß-Thalassemia major treated at Dubai Thalassemia centre.

BACKGROUND: ß-Thalassemia major (BTM) is one of the most common hereditary anemias worldwide. Patients suffer from iron overload that results from repeated blood transfusion This in turn leads to multiple organ damage and endocrinopathies. This study aims to assess the prevalence of growth retardation, hypothyroidism, and diabetes mellitus in children and adolescents with BTM treated at Dubai Thalassemia Centre. METHODS: A total of 105 children and adolescents were included in this retrospective observational study. RESULTS: 39 children and 66 adolescents' data were analyzed. Females composed 51.3% (n = 20) of children and 53.0% (n = 35) of adolescents. Pretransfusion hemoglobin below 9 gm/dl was observed in 10.8% (n = 4) and 10.6% (n = 7) in children and adolescents, respectively. The mean age of menarche was 13.5 years. Among all study participants, 22.6% (n = 14) had normal height velocity whereas 37.1% (n = 23) had reduced height velocity in one year and 40.3% (n = 25) had reduced height velocity in two consecutive years. The proportion of children and adolescents showing reduced height velocity was significantly higher in females compared to the males (90.6% versus 63.3%, respectively, Chi-square = 6.597, p-value = 0.010). Although none of the study participants had diabetes mellitus, 26.1% (n = 12/46) had pre-diabetes. Elevated TSH was observed in 14.7% (n = 5) children and 8.1% (n = 5) adolescents while low FT4 was reported in one child and one adolescent. CONCLUSION: Of all endocrinopathies seen among children and adolescents with BTM, growth delay remains the main concern for this group of patients. Effective treatment is key to further reducing endocrinopathies. Although the sample size is limited, we postulate that the low percentage of endocrinopathies among children with BTM treated at Dubai thalassemia center and the low level of pretransfusion anemia reflect the effective transfusion and chelation at the center.

Management of transfusion-dependent ß-thalassemia (TDT): Expert insights and practical overview from the Middle East.

ß-Thalassemia is one of the most common monogenetic diseases worldwide, with a particularly high prevalence in the Middle East region. As such, we have developed long-standing experience with disease management and devising solutions to address challenges attributed to resource limitations. The region has also participated in the majority of clinical trials and development programs of iron chelators and more novel ineffective erythropoiesis-targeted therapy. In this review, we provide a practical overview of management for patients with transfusion-dependent ß-thalassemia, primarily driven by such experiences, with the aim of transferring knowledge to colleagues in other regions facing similar challenges.

ß-Thalassemia and Diabetes Mellitus: Current State and Future Directions.

ß-Thalassemia major is a congenital hemoglobin disorder that requires regular blood transfusion. The disease is often associated with iron overload and diabetes mellitus, among other complications. Pancreatic iron overload in ß-thalassemia patients disrupts ß-cell function and insulin secretion and induces insulin resistance. Several risk factors, including family history of diabetes, sedentary lifestyle, obesity, gender, and advanced age increase the risk of diabetes in ß-thalassemia patients. Precautionary measures such as blood glucose monitoring, anti-diabetic medications, and healthy living in ß-thalassemia patients notwithstanding, the prevalence of diabetes in ß-thalassemia patients continues to rise. This review aims to address the relationship between ß-thalassemia and diabetes in an attempt to understand how the pathology and management of ß-thalassemia precipitate diabetes mellitus. The possible employment of surrogate biomarkers for early prediction and intervention is discussed. More work is still needed to better understand the molecular mechanism(s) underlying the link between ß-thalassemia and diabetes and to identify novel prognostic and therapeutic targets.

Significance of Cardiac Magnetic Resonance Feature Tracking of the Right Ventricle in Predicting Subclinical Dysfunction in Patients with Thalassemia Major.

In patients with thalassemia major (TM), cardiac magnetic resonance feature-tracking (CMR-FT) has been shown to be an effective method for diagnosing subclinical left ventricular (LV) dysfunction. This study aimed to determine whether CMR-FT could detect abnormal RV dysfunction in patients with a normal right ventricular ejection fraction (RVEF). We performed a retrospective analysis of TM patients admitted to Dubai's Rashid Hospital between July 2019 and March 2021. The inclusion criteria were TM patients with SSFP cine with T2* (T2*-weighted imaging), while exclusion criteria included any other cardiovascular disease. When there was no myocardial iron overload (MIO) (T2* ≥ 20 ms) and when there was significant MIO (T2* < 20 ms), the CMR-FT was used to correlate with EF. Among the 89 participants, there were 46 men (51.7%) and 43 women (48.3%), with a mean age of 26.14 ± 7.4 years (range from 10 to 48 years). Forty-six patients (51.69%) did not have MIO, while 43 individuals did (48.31%). Thirty-nine patients (32.6%) were diagnosed with severe MIO, while seventeen (19.1%) were diagnosed with mild to moderate MIO. A significant correlation existed between RVEF and T2* values (r = 0.274, p = 0.014) and between left ventricular ejection fraction (LVEF) and T2* values (r = 0.256, p = 0.022). Using a multiple logistic regression model with predictors such as right ventricular longitudinal strain (RVGLS), LV ejection fraction (LV EF), and hemoglobin, abnormal myocardial iron overload can be predicted. This model demonstrates an AUC of 78.3%, a sensitivity of 72%, and a specificity of 76%. In the group with preserved RVEF > 53%, the left ventricular radial strain (LVGRS) (p = 0.001), right ventricular radial strain (RVGRS) (p = 0.000), and right ventricular basal circumferential strain (RVGCS-basal) (p = 0.000) CMR-FT strain values are significantly lower than those of the control group (p > 0.05). There was no significant correlation between the LVGLS and T2*. RVGLS was ranked among the most accurate predictors of abnormal myocardial iron overload. The LVGRS, RVGRS, and RVGCS-basal CMR-FT strain values were the best predictors of subclinical RV dysfunction in the group with preserved RVEF. The most accurate way to diagnose MIO is still T2*, but FT-strain can help us figure out how MIO affects the myocardium from a pathophysiological point of view.

Therapeutic plasma exchange followed by convalescent plasma transfusion in critical COVID-19-An exploratory study.

The pathophysiology of severe coronavirus disease 2019 (COVID-19) is primarily a host immune interplay to virus invasion. The therapeutic options have been explored either against hyperinflammation from dysregulated adaptive immunity or direct virus neutralization using antibodies from convalescent plasma (CP) of a recovered patient. The therapeutic plasma exchange (TPE) for removal of excessive inflammatory cytokines has been tried with success in COVID-19. We undertook this exploratory study to evaluate safety and efficacy of TPE followed by CP transfusion in 14 patients with critical COVID-19 requiring invasive mechanical ventilation (IMV). All patients showed improvement in symptoms and decrease of inflammatory markers especially CRP (p = 0.03). 10 patients were liberated from IMV after a median of 5.5 (3-36) days, post sequential therapy. Day 7 and Day 28 mortality was 21.4% and 28.6% respectively. The median duration ICU and hospital LOS were 12 (5-42) days and 18 (12-47) days respectively. No patient developed transfusion-associated complications, but three patients developed secondary bacterial sepsis within 14 days of therapy, and one died. This case series demonstrated the sequential use of TPE followed by CP transfusion as a therapeutic option in critical COVID-19.

Prevalence of iron overload complications among patients with b-thalassemia major treated at Dubai Thalassemia Centre.

BACKGROUND AND OBJECTIVES: Authors and team members of the Dubai Thalassemia Centre obtained data on the prevalence of iron overload complications among patients with b-thalassemia major (b-TM) and compared it to international data to improve patient care and evaluate the effectiveness of earlier used treatment modalities. The information obtained is also expected to be useful in genetic counseling. DESIGN AND SETTING: Cross-sectional study of all living transfusion-dependent b-TM patients registered at the Thalassemia Centre in Dubai, United Arab Emirates, until the end of 2007 (n=382). PATIENTS AND METHODS: Diagnosis of TM was based on clinical history and laboratory confirmation by hemoglobin electrophoresis and DNA testing. All were uniformly treated with desferrioxamine and monitored by serial serum ferritin. results: The mean (SD) age of patients was 15.4 (7.6) years, with 50.5% males. Mean (SD) serum ferritin was 2597.2 (1976.8) micro g/L. The frequency of iron overload complications were as follows: hypogonadism (n=99, 52.7%), hypoparathyroidism (n=40, 10.5%), diabetes mellitus (n=40, 10.5%), hypothyroidism (n=24, 6.5%) and cardiomyopathies (n=7, 1.8%). Hypogonadism was the most common endocrine abnormality in our study and other reported series. However, cardiomyopathies were less prevalent among our patients with higher rates of diabetes and hypoparathyroidism compared to rates reported internationally. Females had statistically significant lower serum ferritin (2530.8 (1931.2), P < .05) with a lower cardiomyopathies rate. CONCLUSION: Iron overload related complications among our patients with thalassemia major were different from those reported internationally. Studying the genetic status of patients from our area may uncover the underlying genetic modifiers of iron overload mediated organs injury.