Abstract
The aim of the study was to assess the outcome of thalidomide in patients with Thalassemia. Method: We identified the outcome of thalidomide in 51 patients with thalassemia needing blood transfusion. We enlisted thalassemia patients, who received treatment with thalidomide between 01 July 2017 and 29 Feb 2020 in Combined Military Hospital, Dhaka. Thalidomide was used in a reduced dose (2 mg/ kg to 5 mg/kg). Thalidomide had been stopped in those patients who developed complications. The following factors were recorded: age and sex, hemoglobin (Hb) levels, ferritin level, blood transfusion interval before and after treatment with thalidomide. Furthermore, we documented blood transfusion dependence and the complications subsequently. All data were studied using SPSS v 24.0 (SPSS Inc. Chicago, IL, U.S.A.). For patients, a paired sample t-test was used to compare the changes in the mean Hb level, ferritin level and the difference in blood transfusion interval before and after treatment with thalidomide. P < 0.05 was considered statistically significant. Results: The study included 51 patients comprised of 23 females (45.1%) and 28 males (54.9%) with an age ranging from 3 to 24 years (9.75±4.11 years). Amongst them 18% (n=9) were beta-thalassemia and 82% (n=42) were Hb E beta-thalassemia. Data of Hb and ferritin levels before and after treatment were recorded in all 51 patients. Ferritin levels reduced to 20.52% (3258.11±2291.91 ng/ml to 2589.65± 2072.74 ng/ml). Hb level increased by 19.26% (7.04±0.53 g/dL to 8.39±1.44g/dL). We found 18 (35.3%) patients with no requirement of blood transfusion after the treatment. 63% (n=32) of the patients showed no adverse effect of thalidomide after the treatment. 16% (n=8) of the patients showed high ALT (Alaline Aminotransferase) and 6% (n=3) showed excessive sleepiness. Other effects included- high TSH, acute urticaria, restlessness, edema, facial puffiness, palpitation, and vomiting. Conclusion: Treatment with thalidomide considerably improved Hb level, increased blood transfusion interval even resulted to non-dependence on blood transfusion and reduced ferritin levels. More studies are necessary to clarify the possible use of thalidomide in the treatment of thalassemia.
Highlights
Thalassemia is recognized as a genetic condition of blood defined by reduced or deficiency of the globin chain synthesis
In India only the thalassemia carrier ranges from 1% to 17% i.e. 1 in every 25 people in India are the carrier of thalassemia. [2, 3] Beta thalassemia is a type of thalassemia, Maksuda Begum et al.: Outcome of Treatment with Thalidomide in Transfusion Dependent Thalassemia Patients: A Prospective Study in a Thalassemia Center, Dhaka, Bangladesh in which Beta globin chain is compromised
The highest number of patients belongs to the age of 6-10-year-old (54.9%) followed by 11- 15-year-old (27.5%). (Table 1) Hemoglobin and serum ferritin levels and blood transfusion intervals have been measured in all patients
Summary
Thalassemia is recognized as a genetic condition of blood defined by reduced or deficiency of the globin chain synthesis. [1] Thalassemia was first identified in the Mediterranean population. A person in such state is incapable to produce normal oxygen-carrying components of blood. This condition is more pronounced in the thalassemia belt region of the world. A Prospective Study in a Thalassemia Center, Dhaka, Bangladesh in which Beta globin chain is compromised. E Beta thalassemia is a type in which Hb E is produced alongside with the compromised beta chain. Apoptosis of cell is a result of excess development of α-chain which initiates during erythropoiesis and reaches maximum concentration at polychromatophilic erythroblasts. [4]
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