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Pediatrics

Contributed by: Benjamin Marsh, M.D., Assistant Professor of Anesthesiology; Pediatric & Pediatric Cardiac Anesthesiologist, Northwell Health / Zucker School of Medicine at Hofstra

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Introduction

In pediatric populations, coagulopathy encompasses a broad spectrum of congenital and acquired disorders. It is essential for clinicians to understand age-related differences in coagulation and clinical presentation in pediatric patients in order to tailor diagnostic and therapeutic care.

Hemostasis in Children

Hemostasis in children differs significantly from that in adults, particularly in neonates and infants. During early life, the levels and functional activity of many coagulation factors, anticoagulants, and fibrinolytic proteins vary by age. In healthy term neonates, vitamin K–dependent clotting factors (II, VII, IX, X), contact factors (XI, XII, prekallikrein), and natural anticoagulants (protein C, protein S, antithrombin) are present at lower concentrations compared to adults. However, this does not typically result in spontaneous bleeding, as the neonatal hemostatic system remains relatively balanced. Platelet function and fibrinolytic activity also differ from those in older children and adults, which can influence the presentation and management of coagulopathies in younger age groups.

Coagulopathy in Children: Inherited and Acquired

Coagulopathy in children can be categorized broadly into congenital and acquired causes. Inherited bleeding disorders often present in infancy or early childhood, either with spontaneous bleeding or following minor trauma or surgical interventions. Major congenital coagulopathies include: Hemophilia A and B, von Willebrand Disease, rare factor deficiencies, and inherited platelet function disorders. Hemophilia A (factor VIII deficiency) and Hemophilia B (factor IX deficiency) are X-linked recessive disorders. These typically present with deep tissue bleeding, including hemarthroses, intramuscular hematomas, and prolonged bleeding after injury or procedures. Severity is related to the residual activity level of the deficient factor. Von Willibrand Disease is the most common inherited bleeding disorder and is caused by a deficiency or dysfunction of von Willebrand factor, which plays a critical role in platelet adhesion and stabilizing factor VIII. Patients often experience mucocutaneous bleeding, such as epistaxis, gingival bleeding, and menorrhagia in adolescent females. Rare factor deficiencies include deficiencies of factors I (fibrinogen), II (prothrombin), V, VII, X, XI, and XIII. They are generally autosomal recessive and more common in consanguineous populations. The clinical phenotype varies significantly, from mild bleeding to life-threatening hemorrhage. Disorders such as Glanzmann thrombasthenia and Bernard-Soulier syndrome result from defects in platelet aggregation or adhesion. These rare conditions present with mucocutaneous bleeding and may be diagnosed during early childhood.

Acquired coagulopathies are more prevalent in pediatric practice and may be transient or associated with serious underlying pathology. Vitamin K is essential for the synthesis of factors II, VII, IX, and X. Newborns are especially vulnerable due to limited vitamin K stores, poor placental transfer, and an immature gut flora. This is the basis for routine vitamin K administration at birth. Deficiency may also occur due to malabsorption, prolonged antibiotic therapy, or poor dietary intake. The liver synthesizes most clotting factors and anticoagulants. Liver dysfunction can impair both clotting and fibrinolysis, leading to complex coagulopathies. While conventional tests such as PT and INR are often abnormal, they do not always correlate with bleeding risk. Disseminated intravascular coagulation is a systemic process involving widespread activation of coagulation pathways, leading to consumption of clotting factors and platelets, and secondary fibrinolysis. It is triggered by conditions such as sepsis, severe trauma, malignancy, and shock. Laboratory findings include elevated PT, aPTT, D-dimer, and low fibrinogen and platelets. Immune thrombocytopenia is an autoimmune condition characterized by isolated thrombocytopenia and mucocutaneous bleeding. It commonly follows a viral illness in young children and is usually self-limited. Anticoagulant medications, chemotherapy, or extracorporeal therapies can cause or exacerbate coagulopathy via consumption or dilution of coagulation factors. Large-volume transfusions without balanced replacement of clotting factors and platelets may result in dilutional coagulopathy, particularly in trauma or surgical settings.

The clinical manifestations of coagulopathy depend on the underlying cause and severity. Presentations can include: cutaneous bleeding (petechiae, purpura, easy bruising), mucosal bleeding (epistaxis, gum bleeding, gastrointestinal bleeding, menorrhagia, deep tissue hemorrhage (hemarthroses, muscular hematomas, intracranial bleeding), and prolonged bleeding (after minor trauma, surgery, or venipuncture). In neonates, signs may be subtle and nonspecific, such as irritability, pallor, or bulging fontanelle in the case of intracranial hemorrhage.

A careful history and physical examination are essential to guide further workup. Important historical factors include: family history of bleeding disorders; nature, frequency, and site of bleeding, any perinatal events (for neonates); and exposure to medications, infections, or recent illnesses. Initial laboratory evaluation often includes complete blood count with platelet count, prothrombin time, activated partial thromboplastin time, fibrinogen level, D-dimer, mixing studies, specific factor assays, and/or von Willebrand panel.

Treatment

Treatment of coagulopathy in children is cause-specific but shares common principles. Supportive care includes volume resuscitation, minimizing trauma, and avoiding unnecessary procedures. Replacement therapy can be in the form of recombinant or plasma-derived factor concentrates for hemophilia, desmopressin in mild hemophilia A or von Willebrand disease, fresh frozen plasma and cryoprecipitate for multiple factor deficiencies or hypofibrinogenemia, platelet transfusions for thrombocytopenia or platelet function defects, and vitamin K for suspected or confirmed deficiency. Disease-specific therapy may include IVIG or steroids for immune thrombocytopenia, antibiotics and supportive care for sepsis-related disseminated intravascular coagulation, or management of underlying liver disease or nutritional deficiencies.

Congenital bleeding disorders are lifelong but can be managed effectively with prophylactic factor replacement, education, and coordinated care through hematology specialists. Acquired coagulopathies generally resolve with treatment of the underlying condition, though some may require ongoing surveillance or intervention.

Coagulopathy in pediatric populations represents a complex and varied group of disorders. A nuanced understanding of developmental hemostasis, coupled with a systematic diagnostic and therapeutic approach, is essential to provide effective care. Prompt recognition and targeted management can prevent serious bleeding complications and improve outcomes in children with both congenital and acquired bleeding disorders.

References

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2. World Federation of Hemophilia. Guidelines for the Management of Hemophilia. 3rd ed. WFH; 2020. https://www1.wfh.org/publications/files/pdf-1574.pdf
3. American Society of Hematology. Clinical practice guidelines on inherited bleeding disorders. https://www.hematology.org/education/clinicians/guidelines-and-quality-care
4. Valentine SL, Bateman ST, Spinella PC, et al. Recommendations on transfusion and bleeding management in critically ill children. Pediatr Crit Care Med. 2018;19(9S Suppl 1):S137-S151.
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