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Management of bleeding disorders in children
1The Children's Hospital of Philadelphia, 34th & Civic Center Blvd., Rm. 9518 Main Bldg., Philadelphia, PA 19104, USA. manno@email.chop.edu
Insights
Pediatric bleeding disorders like neonatal alloimmune thrombocytopenia, hemophilia, and immune-mediated thrombocytopenic purpura require specialized care. Management strategies are evolving, focusing on prophylaxis for hemophilia and careful observation for ITP in children.
Area of Science:
- Pediatric Hematology
- Hemostasis and Thrombosis
- Immunology
Background:
- Pediatric hemostasis differs from adult physiology.
- Bleeding disorders in children have unique natural courses.
- Neonatal alloimmune thrombocytopenia (NAIT), hemophilia, and immune-mediated thrombocytopenic purpura (ITP) are key pediatric hematologic concerns.
Purpose of the Study:
- To review the diagnosis and management of three critical pediatric bleeding disorders.
- To outline current therapeutic approaches for NAIT, hemophilia, and ITP.
- To highlight the distinct aspects of pediatric hemostasis and disease progression.
Main Methods:
- Review of current literature and management strategies for NAIT, hemophilia, and ITP.
- Discussion of pediatric hemostatic physiology.
- Analysis of treatment outcomes and side effects.
Main Results:
- Neonatal immune thrombocytopenia due to NAIT is a significant cause of infant morbidity and mortality.
- Prophylaxis has become the standard for hemophilia management, preventing arthropathy.
- Inhibitor development in hemophilia necessitates understanding of bypassing agents and immune tolerance.
- ITP management involves various treatments, but observation is a viable option due to side effects of interventions.
Conclusions:
- Effective management of pediatric bleeding disorders demands specialized knowledge of pediatric hemostasis.
- Shifting treatment paradigms, such as prophylaxis in hemophilia, improve patient outcomes.
- Careful consideration of treatment risks versus benefits is crucial, particularly in ITP management.
Abstract:
Diagnosis and management of congenital and acquired bleeding disorders in children requires not only an understanding of the unique characteristics of pediatric hemostasis but also the natural course of bleeding disorders in children, which may differ substantially from the course observed in adult patients. In this article, three bleeding disorders of great importance to the pediatric hematologist are reviewed: neonatal alloimmune thrombocytopenia (NAIT), hemophilia and immune-mediated thrombocytopenic purpura (ITP). Current aspects of management are outlined. The unique physiology of transplacental transfer of maternally derived anti-platelet antibodies can result in neonatal immune thrombocytopenia, a significant cause of morbidity and mortality from bleeding in affected infants. For patients with hemophilia, approaches to treatment have shifted over the past decade from on-demand therapy to prophylaxis, either primary of secondary, resulting in delay of onset or complete avoidance of hemophilic arthropathy. Hemophilic inhibitors often develop in young children, prompting the need for a thorough understanding of the use of bypassing agents as well as immune tolerance induction in the young child. Finally, although several management strategies for ITP of childhood have been shown to improve the platelet count, side effects associated with corticosteroids, IVIg, anti-D and splenectomy force the practitioner to also consider the option of carefully observing, but not treating, the child with ITP.
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