Which Comes First, the Rabbit or the Horse?:

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Which Comes First, the Rabbit or the Horse?

• The Treatment of Aplastic Anemia in Children

Dr. Robert Klaassen Assistant Professor Departmen
t of Pediatrics University of Ottawa Pediat
ric Hematologist/Oncologist Director,
Comprehensive Hemophilia Care Program Program
Leader, Thalassemia and Sickle Cell
Programs Department of Pediatrics, Division of
Hematology/Oncology Childrens Hospital of
Eastern Ontario
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Outline

• Kelleys story
• What is Aplastic Anemia?
• What is the Problem?
• What is the Treatment?
• Summary

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Kelleys 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)

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Kelleys 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

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What is Aplastic Anemia?

• Role of the bone marrow

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What is Aplastic Anemia?

• AA appears to be caused by a problem with immune
  system suppressing the growth of the stem cells

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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 gt60

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What is Aplastic Anemia?

• Causes
• Inherited
• Fanconis anemia
• Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Acquired
• Radiation
• Drugs
• Viruses hepatitis non-A,B,C,E or G
• Pregnancy
• Idiopathic

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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

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What is the Problem?

• Low white cells
• Increased risk of infection
• Bacteria, fungus can be life-threatening

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What is the Treatment?

• Supportive
• Transfusions for red cells and platelets
• G-CSF ( a stimulating factor) for white cells

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What is the Treatment?

• Immune Suppression
• Anti-thymocyte globulin
• Horse versus rabbit
• Cyclosporin
• Overall this approach has a 70 success rate

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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

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Kelleys Story Part 2

• On routine follow up in 2004, Kelleys 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

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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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