Thalassemia Intermedia
Conditions
Keywords
Hydroxyurea, Erythropoitin therapy, Thalassemia intermedia
Brief summary
The study hypothesis that treatment with Erythropoietin (EPO) combined with Human Erythropoietin (HUO) therapy will result in hematologic improvement in thalassemia intermedia patients. Second is to determine whether any of the following correlate with improved hematologic response: A decrease in hemolysis, as assayed by a decrease in LDH, compared to baseline levels,baseline Erythropoietin levels,baseline hemoglobin levels and baseline reticulocyte counts (or % circulating nucleated erythroblasts/100 WBCs). Goal: The aim is to assess the possibility of steady increase of hemoglobin levels in thalassemia intermedia patients by at least 1g/dl above baseline levels during therapy using Hydroxyurea and Erythropoietin, growth evaluation,quality of life (QoL) and decline transfusion requirements during study period. Also to report and compare adverse events with other published data regarding.
Detailed description
To determine whether any of the following correlate with improved hematologic response: A decrease in hemolysis, as assayed by a decrease in LDH, compared to baseline levels,baseline Erythropoietin levels,baseline hemoglobin level and baseline reticulocyte counts (or % circulating nucleated erythroblasts/100 WBCs). To assess the possibility of steady increase of hemoglobin levels in thalassemia intermedia patients by at least 1g/dl above baseline levels during therapy using Hydroxyurea and Erythropoietin, growth evaluation , quality of life ( QoL ) and decline transfusion requirements during study period. Also to report and compare adverse events with other published data regarding. THE following criteria are used when including the patient in the study: Patients with thalassemia intermedia.Diagnosis based on genetic mutations, hemoglobin electrophoresis and characteristic clinical data at presentation. Patients requiring different transfusion requirements and not transfusion dependent.Patients having a baseline hemoglobin of less than or equal to 6-8g/dl.Patients with normal renal and liver function.
Interventions
DRUGHydroxyurea ,Epiao
Hydroxyurea (Myers-Squibb, USA) was administered in dosages ranging from 15 up to 35 mg/kg/day orally over 7 days/week. Erythropiotien therapy (rHuEPO - Epiao) from 250 to 500 IU/kg rHuEPO subcutaneously three times a week.
DRUGhydroxyurea, blood transfusion
* Hydroxyurea was administered in dosages ranging from 15 up to 35 mg/kg/day orally over 7 days/week. Hydroxyurea toxicity was defined as a white cell count of less than 2500/μL or a platelet count of less than 100,000/μL, in which case the drug was discontinued.
Sponsors
Ain Shams University
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* Patients with thalassemia intermedia. Diagnosis based on genetic mutations, hemoglobin electrophoresis and characteristic clinical data at presentation. * Require different transfusion requirements and not transfusion dependent. * Have a baseline hemoglobin of less than or equal to 6-8g/dl. * Patients with normal renal and liver function.
Exclusion criteria
* Evidence of active hepatitis (ALT \> 5 times above ULN). * Evidence of renal impairment (serum creatinine \> ULN). * Patients who are dependent on red blood cell transfusions.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in baseline transfusion frequency with increase of pre-transfusion hemoglobin | baseline and 6 month hemoglobin level and transfusion frequency | Decrease in baseline transfusion frequency with increase of pre-transfusion hemoglobin by calculation of transfusion index and mean hemoglobin level |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in baseline quality of life assessment. | baseline and 6 month QOL questionaire | Quality of life assessment using (QOL questionaire) at the begining and at 6 month |
Countries
Egypt
Contacts
Primary ContactAmira A M Adly, Asst. prof.
Outcome results
None listed