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Pediatric Hematology Unit Ha’Emek Medical Center Afula , Israel

Pediatric Hematology Unit Ha’Emek Medical Center Afula , Israel. Levin C . MD, Koren A. MD, Grinberg B. RN, Mary M.SW. Our patients. 64 patients with ß-thalassemia major and/or intermedia Age range: 18 month to 42 years old 37 patients receiving regular blood transfusions

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Pediatric Hematology Unit Ha’Emek Medical Center Afula , Israel

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  1. Pediatric Hematology Unit Ha’Emek Medical Center Afula, Israel Levin C. MD, Koren A. MD, Grinberg B. RN, Mary M.SW

  2. Our patients 64 patients with ß-thalassemia major and/or intermedia Age range: 18 month to 42 years old 37 patients receiving regular blood transfusions 36 patients actually receiving chelation therapy

  3. Deferasirox experience in ß Thalassemia patients 37 patients Age: 2-18 years: 19 >18 years: 18 Male: 19 Female: 18 Treatment period: 4 – 29 month No previous chelation therapy 5 patients

  4. Deferasirox experience in ß Thalassemia patients • Treatment discontinued in 7 patients • Pat. 1 20 year old female, ß-thalassemia major patient- Post splenectomy- Chronic active hepatitis - Desferal therapy replaced by Exjade 20 mg/kg; One month later - patient started on a new therapeutic course for the HCV infection. Progressive increase in serum iron and ferritin levels, clinical deterioration and cardiac decompensation.The patient dies 1 year later from congestive heart failure.

  5. Deferasirox experience in ß Thalassemia patients • Treatment discontinuation in 7 patients • Pat. 2 23 year old male ß-thalassemia intermedia patientPost splenectomyNon transfusion dependent after Hydroxyurea Tx. Exjade dose 20 mg/kg=>15mg/kg - Serum ferritin levels: initially 2000 ng/dl. after 4 months of treatment stable at ± 500 ng/dl. Adverse events: drop of Hb level 1.5 gr/dl from base line.

  6. Deferasirox experience in ß Thalassemia patients • Treatment discontinuation in 7 patients • Pat. 3; 4 20 and 13 year ß-thalassemia major siblings. Previous Tx: Desferioxamine and Deferiprone combination therapy. Deferasirox dose 20 mg/kg=>25mg/kg =>30mg/kg Serum ferritin levels: initially ± 7000 ng/ml after 16 month of treatment ferritin ± 10000 ng/ml No adverse events related to chelation treatment. Compliance problem !!!

  7. Deferasirox experience in ß Thalassemia patients • Treatment discontinuation in 7 patients • Pat. 5 13 year old female ß-thalassemia major patient- Growth hormone therapy Deferasirox 20 mg/kg=>25mg/kg =>30mg/kg Serum ferritin levels: initially 1000 ng/ml after 12 month 1500 ng/ml after 24 month 3000 ng/ml No adverse events related to chelation treatment. Compliance vs GH replacement Tx !!!

  8. Deferasirox experience in ß Thalassemia patients • Treatment discontinuation in 7 patients • Pat. 6 25 year old female - ß-thalassemia major Post splenectomy Small VSD Deferasirox 20 mg/kg=>25mg/kg Serum ferritin levels: initially 6500 ng/ml after 12 month 10000 ng/ml No adverse events related to chelation treatment. Compliance problem !!!

  9. Deferasirox experience in ß Thalassemia patients • Treatment discontinuation in 7 patients • Pat. 7 42 year old female ß-thalassemia major patientChronic active HCV infection Deferasirox 20 mg/kg=>25mg/kg =>30mg/kg Serum ferritin levels: initially 3500 ng/ml after 18 month 6000 ng/ml No adverse events related to chelation treatment. CAH activity ?Liver disease / Deferasirox metabolism?

  10. Deferasirox experience in ß Thalassemia patients • Adverse events: Rash: 3 patients resolved with antihistaminic treatment Gastro intestinal symptoms: 4 patients - Abdominal pain 3, nausea 1,vomiting 1- Change time of drug administration Low serum Phosphor levels: 2 patients after several months of treatment, asymptomatic, resolved without drug discontinuation. Acute appendicitis 2 patients !!?? No significant serum creatinine increase. Transient hepatic liver enzymes elevations from base line.

  11. Deferasirox experience in ß Thalassemia patients • Dose: 15mg/kg: 2 patients 20 mg/kg: 7 patients 25mg/kg: 10 patients 30mg/kg: 10 patients 35mg/kg: 1 patient

  12. Deferasirox experience in ß Thalassemia patients 34 patients follow up 18 month

  13. Deferasirox experience in ß Thalassemia patients 34 patients follow up 18 month

  14. Deferasirox experience in ß Thalassemia patients 34 patients follow up 18 month

  15. Deferasirox experience in ß Thalassemia patients 34 patients follow up 18 month

  16. Deferasirox experience in ß Thalassemia patients • Impact in quality of life: - Patients reported greater comfort and satisfaction with the treatment compared with previous S.C. treatment. - Allows patients greater freedom during the daily activities and less inconvenience at night. - Improvement of compliance. • Due to greater awareness parent and patients are more involved in treatment decisions.

  17. Thanks to the staff for all the hard work!

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