Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia

Transfusion frequency will be assessed by recording the number of blood transfusions a patient receives over a specified period, such as weekly, monthly, or annually. This data will be collected from patient medical records and/or transfusion logs, ensuring accurate tracking of each transfusion event

Hemoglobin levels will be assessed using a complete blood count (CBC) test, measured in grams per deciliter (g/dL) of blood. This test is conducted through a venous blood sample, which is then analyzed using an automated hematology analyzer to determine the hemoglobin concentration.

Leukocyte count will be assessed using a complete blood count (CBC) test, measured in thousands of cells per microliter (thousand cells/µL) of blood. This test involves analyzing a venous blood sample with an automated hematology analyzer to determine the total number of white blood cells present.

Reticulocyte count will be assessed using a complete blood count (CBC) test, measured as a percentage of the total red blood cells or as an absolute number per microliter (µL) of blood. This test involves analyzing a venous blood sample with an automated hematology analyzer, which identifies and quantifies reticulocytes using specific staining techniques.

Platelet count will be assessed using a complete blood count (CBC) test, measured in thousands of cells per microliter (thousand cells/µL) of blood. This test involves analyzing a venous blood sample with an automated hematology analyzer to determine the number of platelets.

Genetic modifiers will be assessed through genetic testing E.g thalassemia genetic profile, focusing on specific genes or genetic variations known to influence the expression or function of proteins related to the condition under study. This analysis helps identify how genetic factors may modify disease progression, treatment response, or other relevant outcomes.

Spleen size will be assessed using imaging techniques such as ultrasound, computed tomography (CT) scan, or magnetic resonance imaging (MRI). Measurements will be reported in centimeters (cm).

Serum Glutamate Pyruvate Transaminase, also known as alanine aminotransferase (ALT), will be assessed through a blood test, measured in units per liter (U/L). This test analyzes a venous blood sample to determine the concentration of ALT, an enzyme primarily found in the liver cells.

Serum ferritin levels will be assessed using a blood test, measured in nanograms per milliliter (ng/mL). A venous blood sample will be analyzed in a clinical laboratory using immunoassay techniques to determine the concentration of ferritin, providing an indicator of the body's iron stores.

Lactate dehydrogenase (LDH) levels will be assessed through a blood test, measured in units per liter (U/L). This test analyzes a venous blood sample to determine the concentration of LDH, an enzyme involved in cellular metabolism. Elevated LDH levels may indicate tissue damage or disease,

Nucleated red blood cells (NRBCs) will be assessed using a complete blood count (CBC) test with a differential, measured as the number of NRBCs per 100 white blood cells (WBCs) or as an absolute number per microliter (µL) of blood. This test involves analyzing a venous blood sample with an automated hematology analyzer, which can differentiate and count NRBCs to provide insights into bone marrow function and potential hematologic disorders.

D-dimer levels will be assessed through a blood test, typically measured in nanograms per milliliter (ng/mL) or micrograms per liter (µg/L). This test analyzes a venous blood sample to determine the concentration of D-dimer, a fibrin degradation product.

Total bilirubin (TBIL) levels will be assessed through a blood test, measured in milligrams per deciliter (mg/dL). This test analyzes a venous blood sample to determine the total concentration of bilirubin, including both direct (conjugated) and indirect (unconjugated) bilirubin.

Indirect bilirubin (IBIL) levels will be assessed through a blood test, measured in milligrams per deciliter (mg/dL). This test analyzes a venous blood sample to determine the concentration of unconjugated bilirubin, which is a byproduct of red blood cell breakdown before it is processed by the liver.

FibroScan will be used to assess liver stiffness and steatosis, which can be indicative of liver iron overload. Elevated serum ferritin levels may suggest iron accumulation in the liver, and FibroScan provides a non-invasive method to evaluate the extent of liver fibrosis and fat content.

T2* (T2 star) magnetic resonance imaging (MRI) will be used to assess liver iron concentration in relation to serum ferritin levels. This imaging technique measures the rate of signal decay in liver tissue, which is inversely correlated with iron content. A shorter T2* value indicates higher iron concentration. T2* MRI provides a non-invasive, quantitative assessment of liver iron overload.

Transfusion frequency will be assessed by recording the number of blood transfusions a patient receives over a specified period, such as weekly, monthly, or annually. This data will be collected from patient medical records and/or transfusion logs, ensuring accurate tracking of each transfusion event

The safety profile will be assessed by monitoring and documenting adverse events, side effects, and any other safety-related outcomes throughout the study period. This includes recording and analyzing data on patient-reported symptoms, clinical observations, laboratory test results (including those for the parameters previously discussed like liver enzymes, kidney function tests, etc.), and any other relevant safety assessments specified in the study protocol.