Contributions
Abstract: PB2502
Type: Publication Only
Background
Β-Thalassemia is the most common hemoglobinophaty in Georgia. Carrier rate is 3,79%, and annual affected births of homozygote β-thalassemia are about 20 new births per year. One of the most important problems of treatment of this pathology is excretion excess iron with iron chelator therapy. In recent years for this purpose, usage of Exjade, but its efficiency criteria’s and distant results not known yet.
Aims
This study was undertaken to evaluate the effectiveness of Exjade in patients with transfusion-dependent β-thalassemia major during 3 years in Georgia
Methods
16 children, aged 3 to 17 years, with transfusion-dependent β-thalassemia phenotype, were enrolled in a trial. The starting dose of Exjade was 20-30mg/kg per day given orally once a day. Baseline and serial laboratory tests were performed: detailed history and physical examination, complete blood count with red blood cell indices, reticulocyte count, hemoglobin electrophoresis, and ferritin level measurement monthly, liver and renal function tests, bilirubin, urine analysis. Response to therapy was evaluated after 6 month, 1, 2, 3 years of treatment.
Results
No adverse effects were observed at a dose of 20-30mg/kg day. Positive clinical-laboratory effect was noticed in 13 patients: the general condition of children improved, increased intervals between hemotransfusions after 6 month of treatment. The decrease in ferritin levels started in 4-5 months of treatment. Taking into account the ferritin level, in some cases, treatment was temporarily stopped, or, dose of Exjade was decreased after a year of treatment. In 3 cases, the treatment was ineffective, despite Exjade dose was increased
Conclusion
Accurate assessment of excessive iron stores is essential to optimal therapy. In our cases the Exjade is safe and effective in 81,2% of patients with transfusion-dependent β-thalassemia major. It is noted, that the result is achieved with continuous, regular Exjade therapy and such patients are under monthly monitoring of serum ferritin level. We also should consider that Exjade is characterized by a later effect (after 4-5 months of treatment). Exjade therapy should start as soon as the patient becomes significantly iron overloaded, this correlates with the serum ferritin>1,000 ng/ml. In some cases, his ineffectiveness is due by irregular/late start of treatment (the level of ferritin exceeds 2,000 ng/ml).Combination chelation may be an effective treatment strategy for patients with insufficient response to monotherapy. In rare cases, determining the causes of Exjade inefficiency is very difficult, that’s why further studies in this direction are needed.
Session topic: 28. Thalassemias
Keyword(s): Beta thalassemia, Chelation
Abstract: PB2502
Type: Publication Only
Background
Β-Thalassemia is the most common hemoglobinophaty in Georgia. Carrier rate is 3,79%, and annual affected births of homozygote β-thalassemia are about 20 new births per year. One of the most important problems of treatment of this pathology is excretion excess iron with iron chelator therapy. In recent years for this purpose, usage of Exjade, but its efficiency criteria’s and distant results not known yet.
Aims
This study was undertaken to evaluate the effectiveness of Exjade in patients with transfusion-dependent β-thalassemia major during 3 years in Georgia
Methods
16 children, aged 3 to 17 years, with transfusion-dependent β-thalassemia phenotype, were enrolled in a trial. The starting dose of Exjade was 20-30mg/kg per day given orally once a day. Baseline and serial laboratory tests were performed: detailed history and physical examination, complete blood count with red blood cell indices, reticulocyte count, hemoglobin electrophoresis, and ferritin level measurement monthly, liver and renal function tests, bilirubin, urine analysis. Response to therapy was evaluated after 6 month, 1, 2, 3 years of treatment.
Results
No adverse effects were observed at a dose of 20-30mg/kg day. Positive clinical-laboratory effect was noticed in 13 patients: the general condition of children improved, increased intervals between hemotransfusions after 6 month of treatment. The decrease in ferritin levels started in 4-5 months of treatment. Taking into account the ferritin level, in some cases, treatment was temporarily stopped, or, dose of Exjade was decreased after a year of treatment. In 3 cases, the treatment was ineffective, despite Exjade dose was increased
Conclusion
Accurate assessment of excessive iron stores is essential to optimal therapy. In our cases the Exjade is safe and effective in 81,2% of patients with transfusion-dependent β-thalassemia major. It is noted, that the result is achieved with continuous, regular Exjade therapy and such patients are under monthly monitoring of serum ferritin level. We also should consider that Exjade is characterized by a later effect (after 4-5 months of treatment). Exjade therapy should start as soon as the patient becomes significantly iron overloaded, this correlates with the serum ferritin>1,000 ng/ml. In some cases, his ineffectiveness is due by irregular/late start of treatment (the level of ferritin exceeds 2,000 ng/ml).Combination chelation may be an effective treatment strategy for patients with insufficient response to monotherapy. In rare cases, determining the causes of Exjade inefficiency is very difficult, that’s why further studies in this direction are needed.
Session topic: 28. Thalassemias
Keyword(s): Beta thalassemia, Chelation