Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.

Phase 2

• Conditions: Thalassemia Intermedia
• Interventions: Drug: Hydroxyurea ,Epiao; Drug: hydroxyurea, blood transfusion

• Registration Number: NCT01624038
• Lead Sponsor: Ain Shams University

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.

Study Timeline

• Study First Submitted: 2012-04-13
• Status Verified: 2012-06
• Study Start: 2012-06
• Study First Submitted QC: 2012-06-18
• Last Update Submitted: 2012-06-18
• Study First Posted: 2012-06-20
• Last Update Posted: 2012-06-20
• Primary Completion: 2012-12
• Study Completion: 2012-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 40

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Hydroxyurea, Epiao	Hydroxyurea ,Epiao	* Hydroxyurea (Myers-Squibb, USA) 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. White cell count and platelet count were determined on a monthly basis. Side effects such as nausea, vomiting, diarrhea, rashes, and malaise, experienced during the first 6 h after taking the HU will be considered as clinical toxicity. * Erythropiotien therapy (rHuEPO - Epiao) from 250 to 500 IU/kg rHuEPO subcutaneously three times a week.
Hydroxyurea,blood transfusion	hydroxyurea, blood transfusion	Hydroxyurea (Myers-Squibb, USA) was administered in dosages ranging from 15 up to 35 mg/kg/day orally over 7 days/week.

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

hematology clinic ,pediatrics hospital, Ain Shams University hospital; 🇪🇬 Cairo, Egypt