Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study.

NIBD/IRB-236/21-2021.

The goal of this study is to compare the effectiveness and safety of two hemoglobin F inducer. This is single centered interventional pilot study is to compare the efficacy and safety parameters in beta thalassemia patients. As this is a pilot study, the investigator took a small number of patients. The Sample size was calculated by the World health organization sample size calculator. After screening 39 patients and 24 patients were eligible for enrollment in this study. The main objective was to evaluate safety of both drugs in genetic disorder like thalassemia. for safety evaluation, hematological parameters were evaluated that includes total bilirubin , indirect bilirubin, Serum Glutamate Pyruvate Transaminase, serum glutamic-oxaloacetic transaminase, urea, creatinine and lactate dehydrogenase were monitored . Moreover to evaluate the efficacy of drug, hematological parameters that includes hemoglobin, red blood cells , nucleated red blood cells , reticulocytes count, Red blood cells indices ( mean corpuscular hemoglobin, mean corpuscular volume and mean corpuscular hemoglobin concentration) , white blood cells and platelets were done. Another important parameters to evaluate the efficacy of hemoglobin F inducer is transfusion frequency. Test were done at baseline and after completion of study means after 06 months.

2021-09-01

2021-12-01

2022-03-03

Completed

Early phase I

24

Inclusion Criteria: * Know case of beta thalassemia age between 10-30 years Exclusion Criteria: * Patients on any other haemoglobin F inducer or erythropoietin. * Patients with co-morbidities like cardiopulmonary and neurological disease * Pregnant, lactating women. * Patient did not willing to take contraceptive measures. * Participants with history of thrombosis.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Patients were divided into 2 groups, one received hydroxyurea second group received thalidomide.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 24, 'type': 'ACTUAL'}}

2024-03-12