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

Phase 2
Completed
Conditions
Beta-Thalassemia
Interventions
Registration Number
NCT06239389
Lead Sponsor
National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Brief Summary

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.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
hydroxyureaThalidomide 100mgHydroxyurea is available in oral dosage form which is capsule. frequency: once a day duration: at every 24 hours dose: 15/mg/kg/day.
ThalidomideHydroxyureaThalidomide is available in oral dosage form which is capsule. frequency: once a day at night duration: at every 24 hours dose:50 mg / day (in patients \>10-13 years) while the adult dose was 100 mg /day (age \>13 Years)
Primary Outcome Measures
NameTimeMethod
comparison of hemoglobin06 months

Hemoglobin

comparison of red blood cells06 months

Red blood cells

comparison of Mean Corpuscular Hemoglobin concentration06 months

Mean Corpuscular Hemoglobin concentration

comparison of Mean corpuscular volume06 months

Mean corpuscular volume

comparison of Mean Corpuscular Hemoglobin06 months

Mean Corpuscular Hemoglobin

comparison of Indirect bilirubin06 months

Indirect bilirubin

comparison of Total Bilirubin06 months

Total Bilirubin

comparison of Serum glutamic pyruvic transaminase06 months

Serum glutamic pyruvic transaminase

comparison of glutamic-oxaloacetic transaminase06 months

glutamic-oxaloacetic transaminase

comparison of white blood cells06 months

white blood cells

comparison of platelets06 months

Platelets

comparison of urea06 months

Urea

comparison of creatinine06 months

Creatinine

comparison of Lactate dehydrogenase baseline and after the completion of study.06 months

Lactate dehydrogenase

comparison of tranfusion frequency baseline and after the completion of study.06 months

Transfusion interval

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (1)

National Institute of Blood Diseases and Bone Marrow Transplantation

🇵🇰

Karachi, Sindh, Pakistan

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