Beta-Thalassemia
Conditions
Keywords
thalidomide,, hydroxyurea, hemoglobin F inducers, beta thalassemia
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.
Interventions
Thalidomide is available in capsulated form in the strength of 100 mg and 50 mg. It was given according to the weight of the patient. Due to somnolence, it was advised to give at night only.
DRUGHydroxyurea
Hydroxyurea is available in capsulated form under the brand of hydrea. Its available strength is 500 mg . Dosing depends upon weight of patient, that is 15mg/kg/day. so patients unable to take full capsule due to low weight, was advised to take hydroxyurea syrup.
Sponsors
National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Patients were divided into 2 groups, one received hydroxyurea second group received thalidomide.
Eligibility
Sex/Gender
ALL
Age
10 Years to 30 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| comparison of hemoglobin | 06 months | Hemoglobin |
| comparison of red blood cells | 06 months | Red blood cells |
| comparison of Mean corpuscular volume | 06 months | Mean corpuscular volume |
| comparison of Mean Corpuscular Hemoglobin | 06 months | Mean Corpuscular Hemoglobin |
| comparison of Mean Corpuscular Hemoglobin concentration | 06 months | Mean Corpuscular Hemoglobin concentration |
| comparison of Indirect bilirubin | 06 months | Indirect bilirubin |
| comparison of Total Bilirubin | 06 months | Total Bilirubin |
| comparison of Serum glutamic pyruvic transaminase | 06 months | Serum glutamic pyruvic transaminase |
| comparison of glutamic-oxaloacetic transaminase | 06 months | glutamic-oxaloacetic transaminase |
| comparison of white blood cells | 06 months | white blood cells |
| comparison of platelets | 06 months | Platelets |
| comparison of urea | 06 months | Urea |
| comparison of creatinine | 06 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 |
Countries
Pakistan
Outcome results
None listed