Von Willebrand Disease in the PICU

In this episode of "PICU Doc on Call," Drs. Pradip Kamat and Rahul Damania dive into a pediatric ICU case involving a 4-year-old girl who presents with severe anemia and bleeding, ultimately diagnosed with von Willebrand disease (VWD). They chat about the causes and different types of VWD, walk through the key clinical features, and break down how to diagnose and manage this condition. Drs. Kamat and Damania highlight the important roles of desmopressin and factor concentrates in treatment. Throughout the episode, they stress the need to recognize VWD in kids who have mucosal bleeding and offer practical tips for intensivists on lab evaluation and treatment strategies for this common inherited bleeding disorder.

Show Nighlights:

1. Clinical case discussion of a 4-year-old girl with severe anemia and bleeding symptoms
2. Diagnosis of von Willebrand disease (VWD) and its significance in pediatric critical care
3. Etiology and pathogenesis of von Willebrand disease
4. Classification of von Willebrand disease into types (Type 1, Type 2 with subtypes, Type 3)
5. Clinical manifestations and symptoms associated with VWD
6. Diagnostic approach for identifying von Willebrand disease, including laboratory tests
7. Management strategies for VWD, including desmopressin and von Willebrand factor concentrates
8. Role of adjunctive therapies such as antifibrinolytics and hormonal treatments
9. Importance of multidisciplinary collaboration in managing complex bleeding disorders
10. Overview of the pathophysiology of von Willebrand factor and its role in hemostasis

References:

1. Fuhrman & Zimmerman - Textbook of Pediatric Critical Care Chapter ***.
2. Reference 1: Leebeek FW, Eikenboom JC. Von Willebrand's Disease. N Engl J Med. 2016 Nov 24;375(21):2067-2080.
3. Reference 2: Ng C, Motto DG, Di Paola J. Diagnostic approach to von Willebrand disease. Blood. 2015 Mar 26;125(13):2029-37.
4. Platton S, Baker P, Bowyer A, et al. Guideline for laboratory diagnosis and monitoring of von Willebrand disease: A joint guideline from the United Kingdom Haemophilia Centre Doctors' Organisation and the British Society for Hematology. Br J Haematol 2024 May;204(5):1714-1731.
5. Mohinani A, Patel S, Tan V, Kartika T, Olson S, DeLoughery TG, Shatzel J. Desmopressin as a hemostatic and blood-sparing agent in bleeding disorders. Eur J Haematol. 2023 May;110(5):470-479. doi: 10.1111/ejh.13930. Epub 2023 Feb 12. PMID: 36656570; PMCID: PMC10073345.