Contemporary perspectives and initial management of pediatric ITP. William Beau Mitchell, MD Weill Cornell Medical College New York, NY USA

Transcription

1 Contemporary perspectives and initial management of pediatric ITP William Beau Mitchell, MD Weill Cornell Medical College New York, NY USA

2 Case Presentation 5 year old female Bruises on trunk, extremities Petechiae, wet purpura in mouth Otherwise well appearing WBC 7.6 Hgb 11.2 Plt 4

3 Immune Thrombocytopenia Autoimmune disorder characterized by immunologic destruction of otherwise normal platelets most commonly occurring in response to an unknown stimulus. ASH 2011 ITP Guidelines. Neunert et al. Blood: 2011: 117 (16)

4 Immune Thrombocytopenia Platelet Count < 100,00 /ul Absence of initiating/underlying cause International Consensus Report. Provan et al. Blood 2010:115 (2)

5 Basic Pathophysiology of ITP Bone marrow Peripheral blood Spleen Megakaryocyte P Platelet P Macrophage Anti platelet glycoprotein antibodies

6 Basic Evaluation Patient history Family history Physical examination Complete blood count and reticulocyte count Peripheral blood film Blood group (Rh) Direct antiglobulin test International Consensus Report. Provan et al. Blood 2010:115 (2)

7 Consider Evaluating Quantitative immunoglobulin level measurement Antithyroid antibodies and thyroid function Antiphospholipid antibodies (including anticardiolipin and lupus anticoagulant) Antinuclear antibodies Pregnancy test in women of childbearing potential Viral PCR for parvovirus and CMV Glycoprotein-specific antibody International Consensus Report. Provan et al. Blood 2010:115 (2)

8 Common Echymoses Purpura Petechiae Less common Hematuria GI Bleeding Rare Intracranial Hemorrhage Clinical History

9 Peripheral Blood Film Ash Image Bank #

10 ITP Presentation Pediatric Incidence : 2-6 per 100,000 Abrupt Onset M = F < 20% Chronic Adults Incidence : 3.3 per 100,000 Insidious Onset F > M > 50% Chronic Course Terrell et al. Am J of Hematology 2010, 85:

11 ITP Classification Newly Diagnosed Persistent (3 12 months) Chronic ( > 12 months duration) International Consensus Report. Provan et al. Blood 2010:115 (2)

12 When to Treat Bleeding/quality of life Grade 1. Minor bleeding, few petechiae ( 100 total) and/or 5 small bruises ( 3-cm diameter); no mucosal bleeding Grade 2. Mild bleeding, many petechiae (> 100 total) and/or > 5 large bruises (> 3-cm diameter); no mucosal bleeding Grade 3. Moderate bleeding, overt mucosal bleeding, troublesome lifestyle Grade 4. Mucosal bleeding or suspected internal hemorrhage Management approach Consent for observation Consent for observation or for treatment in selected children Intervention to reach grade 1/2 in selected children Intervention International Consensus Report. Provan et al. Blood 2010:115 (2)

13 Pediatric ITP Treatment Observation Intravenous Immunoglobulin Corticosteroids Intravenous Anti-D Immunoglobulin Role of Platelet Transfusion

14 Initial management of pediatric ITP ASH guidelines Observation Single dose of IVIg (0.8-1 g/kg) IVIg can be used if a more rapid increase in the platelet count is desired Short course of corticosteroids Anti-D Not advised in children with a hemoglobin concentration that is decreased due to bleeding, or with evidence of autoimmune hemolysis Must be Rh + and not splenectomized ASH 2011 ITP Guidelines. Neunert et al. Blood: 2011: 117 (16)

15 Initial management of pediatric ITP International Consensus International Consensus Report. Provan et al. Blood 2010:115 (2)

16 Watch and Wait Observation Response Time ~ 2/3 Response within 6 months Days - months Toxicity Potential bleeding Activity restriction International Consensus Report. Provan et al. Blood 2010:115 (2)

17 Dose Intravenous Immunoglobulin g/kg IV Response Time ~ 80 % Response (dose dependent) 1-2 days Toxicity Headache [Severe] Fever Chills International Consensus Report. Provan et al. Blood 2010:115 (2)

18 Corticosteroids Dose Response Time Toxicity 1-2 mg/kg up to 14 days 4 mg/kg x 3-4 days ~ 75% Response (dose dependent) 2-7 days Mood Swings Gastritis Weight Gain International Consensus Report. Provan et al. Blood 2010:115 (2)

19 Intravenous Anti-D Dose Response Time Toxicity mcg/kg IV Rh+ non-splenectomized 50-77% Response (dose dependent) > 50% Respond in 24 h Headache Fever / Chill (less common) Hemolysis Renal Failure (Rare) International Consensus Report. Provan et al. Blood 2010:115 (2)

20 Emergency Treatment Life Threatening Bleeding Event Platelet Transfusion IV Corticosteroids IVIG or IV Anti-D Consideration : Anti-Fibrinolytics Emergency Splenectomy Goal : Platelet Count & Minimize Bleeding

21 Summary of first line therapies in pediatric ITP Observation is appropriate if no or minor bleeding symptoms First line pharmacological interventions are: IVIG Corticosteroid Anti-D 80% of children will spontaneously resolve

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