An Algorithm to Start Iron Chelation in Minimally Transfused Young Beta-thalassemia Major Patients

• Status: Unknown status
• Trial end date: 2015-07-01
• Target enrollment: 0
• Participant gender: All

Summary

A prospective randomized study on Safety, Tolerability and Efficacy of oral Low dose DFP (50 mg/kg/day) in minimally transfused B-TM after 5 transfusions when SF reaches 500 ng/m and with either appearance of LPI > 0.2 or TSAT reaches 50% compared with non treatment arm.

So the aim of this study:

1. To determine the time as well as amount of transfused iron ( calculated in mg iron/kg ) which lead to Serum ferritin reaches 500 ng /ml and LPI appearance >0.2 as well as TSAT reaches 50 % .

2. Tolerability and safety of early low dose DFP 50mg/kg and effectiveness to postpone or prevent SF from reaching 1000 ng/ml or LPI >0.6 or TSAT >70% in comparison to patients not starting chelation therapy

3. Determine adverse events whether drug or non drug related

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ain Shams University
• Treatments: Deferiprone; Iron

Criteria

Inclusion Criteria:

1. Young beta thalassemia major patients (diagnosed by HPLC, CBC) who started transfusion therapy who received 5-7transfusions or less, aged more than 6 months.

2. Pre-transfusional Hb should be >9 g/dL.

3. Serum ferritin should be ≤ 500ng/ml, transferrin saturation ≤ 50%.

Exclusion Criteria:

• 1. Beta thalassemia intermedia patients, patients with other transfusion dependent anemias (myelodysplasia, other chronic hemolytic anemias , pure red cell aplasia , aplastic anemia ) 2. Patients with levels of ALT >5 the upper limit of normal (ULN), serum creatinine > ULN on 2 measurements.

3. Patients with history of agranulocytosis [absolute neutrophil count (ANC) <0.5×109/L].

4. Non complaint patients acknowledged by reviewing the patient's records.