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Which Comes First, the Rabbit or the Horse?:

Which Comes First, the Rabbit or the Horse?:. Dr. Robert Klaassen Assistant Professor Department of Pediatrics University of Ottawa Pediatric Hematologist/Oncologist Director , Comprehensive Hemophilia Care Program Program Leader , Thalassemia and Sickle Cell Programs

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Which Comes First, the Rabbit or the Horse?:

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  1. Which Comes First, the Rabbit or the Horse?: Dr. Robert Klaassen Assistant Professor Department of Pediatrics University of Ottawa Pediatric Hematologist/Oncologist Director, Comprehensive Hemophilia Care Program Program Leader, Thalassemia and Sickle Cell Programs Department of Pediatrics, Division of Hematology/Oncology Children’s Hospital of Eastern Ontario The Treatment of Aplastic Anemia in Children

  2. Outline • Kelley’s story • What is Aplastic Anemia? • What is the Problem? • What is the Treatment? • Summary

  3. Kelley’s Story • Kelley arrived at CHEO in May 1999 when she was 9 years old covered in bruises • Her blood work showed: • anemia - hemoglobin 80 (115 - 145) • low white cells – WBC 3.7 (4.5 - 15.5) • low platelets – plt 2 (150 - 400)

  4. Kelley’s Story • She was started treatment with horse anti-thymocyte globulin and cyclosporin • She responded well to therapy and six months later she was off treatment and her blood counts were normal

  5. What is Aplastic Anemia? • Role of the bone marrow

  6. What is Aplastic Anemia? • AA appears to be caused by a problem with immune system suppressing the growth of the stem cells

  7. What is Aplastic Anemia? • Paul Ehrlich described the first patient in 1888, who was a pregnant woman • Occurs in 2 cases per million population, may be as high as 14/million in Japan • Peak incidence is at 20-25 years and again at >60

  8. What is Aplastic Anemia? • Causes • Inherited • Fanconi’s anemia • Paroxysmal Nocturnal Hemoglobinuria (PNH) • Acquired • Radiation • Drugs • Viruses – hepatitis non-A,B,C,E or G • Pregnancy • Idiopathic

  9. What is the Problem? • Low red cells • Decreased energy, pale • Strain on the heart • Low platelets • Increased bruising, bleeding • Nose bleeds, heavy menstral periods • Risk of bleeding in the brain

  10. What is the Problem? • Low white cells • Increased risk of infection • Bacteria, fungus can be life-threatening

  11. What is the Treatment? • Supportive • Transfusions for red cells and platelets • G-CSF ( a stimulating factor) for white cells

  12. What is the Treatment? • Immune Suppression • Anti-thymocyte globulin • Horse versus rabbit • Cyclosporin • Overall this approach has a 70% success rate

  13. What is the Treatment? • Bone marrow transplant • Recommended in all patients with a matched sibling • Unrelated donor transplant is reserved for patients unresponsive to immune therapy

  14. Kelley’s Story Part 2 • On routine follow up in 2004, Kelley’s platelet count was found to be 109, this slowly fell until in June of 2006 it was 10 • Retreated with cyclosporin and ATG, this time rabbit. • She has been off treatment since February of this year with normal blood counts

  15. Summary • Aplastic anemia is very rare and can be inherited or caused by drug, radiation or infection • Problems include the risk of bleeding and infection • Treatment is bone marrow transplant if there is a matched sibling or immune suppression if there is no match • Hopefully most patients can then ride off into the sunset

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