Pediatric Hematology Unit H’Eamek Medical Center Afula , Israel

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

2. Chronic hematological patients • Coagulation disorders: Bleeding, Thrombotic diseases • Thrombocytopenia: ITP, Fanconi’s Aplastic Anemia • Neutropenia: Kostmman’s, Fanconi’s Aplastic Anemia • Chronic Anemia: Sickle Cell Disease, Congenital Spherocytosis, Thalassemia

3. ßThalassemia is a genetic disease, that starts merely as hemolytic anemia but becomes a chronic disease with multisystem involvement impairing organ and tissue function.

4. ßThalassemia Prevention • Autosomal recessive disease • Prevention of the disease is possible throughgenetic counseling

5. ßThalassemia Treatment • BMT is the only definitive cure, limited by HLA matching donor and associated complications • In best conditions still associated with 5% mortality and 10% morbidity • Conservative treatment: Blood transfusions and Iron chelation, Fetal Hemoglobin inducers

6. ßThalassemia Treatment • Regular Blood transfusions every 2-4 weeks for life • Transfusions begin from the first year of life (Pre-transfusion Hb level of 9.5g/dl)

7. ßThalassemia Treatment • Iron Chelation therapy for life • Access to new drugs improve quality of life

8. ßThalassemia Treatment • Iron Chelation therapy for life • Access to new drugs improve quality of life

9. ßThalassemia Complications • Most of the complications are related to Iron overload • Can be prevented by adequate iron chelation therapy

10. ßThalassemia Complications • Cardiac disease: cardiomyopathy, arrhythmia Continues to be the most frequent cause of death • Infection: high susceptibility due to excessive Iron availability and splenectomy

11. ßThalassemia Complications • Liver disease: related to iron overload and/or HCV infection • Thrombotic disorders • Osteoporosis • Pulmonary hypertension

12. ßThalassemia Complications • Endocrine Abnormalities: delayed growth and puberty, fertility problems, hypothyroidism, hypoparathyroidism, diabetes • Bone deformities due to bone marrow hyperplasia

13. Living longer, living better • 68% of patients live beyond age 35 • Quality of life improved dramatically

14. ßThalassemia Coping • Intensive and demanding treatment • Growth retardation, sexual immaturity • Facial deformities • Congenital nature of the disease • Fear of death • Social acceptance • Cultural and family expectations

15. Medical care Psychologist Transfusion unit Hematologist team Cardiologist Nutritionist Endocrinologist Thalassemic patient Genetic counseling Occupational therapist Gynecologist/ fertility Inpatient unit adult/ pediatric Osteoporosis team Blood bank and Laboratory facilities

16. Psychosocialsupport Education Economic support Self help groups Occupation Thalassemic patient Social worker Caregivers support Activities/ sport Family support

17. Our patients 64 patients with ß-thalassemia major and/or intermedia Age range: 1year to 42 years old 37 patients receiving regular blood transfusions 35 patients receiving chelation therapy

18. Patients social characteristics No Adult patients working

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