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Thrombocytopenia
Thrombocytopenia
AND TREATMENT OF
THROMBOCYTOPENIA
Petechiae
Remove Antigen: Rx Inciting
Agent = Fix “ITP”
HIV
Hepatitis C
Helicobacter pylori
WHEN TO DO A BONE
MARROW IN THE
THROMBOCYTOPENIC
PATIENT?
ITP: A SIMPLE DISEASE
• Patients make auto-antibodies directed
against their own platelets
• These platelets are rapidly destroyed
• If the platelet count becomes low enough,
bleeding symptoms may ensue
• Bleeding is rarely serious, ie an intracranial
hemorrhage, even at very low counts
ITP: A COMPLICATED DISEASE
800
600
400
200
1 2 3 1 2 3 4 5 6 7 8 9
Hours Days
Harrington WJ, et al. J. Lab Clin Med. 1951;38:1-10.
ITP: what tests are helpful
• Complete CBC---not just the platelets
• Bone marrow---not in all/most cases
• Blood type & DAT-prognostic re hemolysis
• PT-PTT, Thyroid, Ig’s, lupus, SMA
• Anti-phospholipid antibodies
• Platelet turnover (estimates): platelet
retics, thrombopoietin, large platelets
Who Needs Treatment with ITP?
At What Platelet Count ?
Needs to be individualized:
job
physical trauma ie sports
access to care
anxiety
effect on fatigue
Acute Platelet Increase
• gold standard: IVIG at 1 gm/kg
• IV anti-D: as fast as IVIG at 75 mcg/kg
• Steroids: IV solumedrol 30/kg, high dose
dexamethasone or Prednisone 2-4/kg
• Platelet transfusions
• Combinations including Steroids, IVIG, IV
anti-D and/or vincristine
Advantages and Disadvantages of
Treatment for Children with ITP
Advantages Disadvantages
• Steroids: oral, continuous so much toxicity
often works with any usage
• IVIG: rapid substantial blood product,
platelet increase headache, 4-6hrs
• IV anti-D: 5-15 minute, at fever-chill, hemo-
75 mcg/kg=IVIG lysis, IVH, blood
STUDY TREATMENTS
ARM - A
D D D D
days
1 2 3 4 7 14 21 28
ARM - B
D D D D RTX RTX RTX RTX
days
1 2 3 4 7 14 21 28
D: Dexamethasone 40 mg po daily x 4
Thrombo-
poietin
P
Peripheral blood
P
Bone marrow
Platelet
Megakaryocyte
TPO Agonists in
Thrombocytopenic States: Focus
on ITP
Newer agents that will probably
revolutionize our approach to
thrombocytopenia in many
conditions, not only ITP
rhTPO and PEG-rHUMGDF
rhTPO PEG-rHuMGDF
• Glycosylated • Not glycosylated
• Full length • Truncated
COOH Polyethylene • Additional polyethylene
terminal glycol glycol moiety
domain
60 60
10
40 38.1 40
5
20 20
Thrombopoietin Eltrombopag
MW 64,000 MW 442
Primary Endpoint: Percentage of
Patients With Platelets ≥50,000/µL at
100
Day 43 Visit†
80
P <0.001‡
Responders (%)
40
20
0
Placebo§ Eltrombopag
300
250
Platelet count (Gi/L)
200
150
100
50
Baseline 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20
Number of subjects:
Week
Placebo
Median Platelet Count
200
30 mg
50 mg
150 75 mg
100
50
INITIATION MAINTENANCE
0
0 14 28 42 56 70 84 98 112
Study Day
McHutchison, NEJM 2007