Pediatric Von Willebrand Disease Treatment & Management
- Author: Suchitra S Acharya, MBBS, MD; Chief Editor: Max J Coppes, MD, PhD, MBA more...
Evidence-based guidelines for the diagnosis and management of von Willebrand disease (VWD) have been established.[10, 11]
Minor bleeding problems in patients with von Willebrand disease, such as bruising or a brief nosebleed, may not require specific treatment. For more serious bleeding, medications that can raise the von Willebrand factor (VWF) level and, thereby, limit bleeding are available. The goal of therapy is to correct the defect in platelet adhesiveness (by raising the level of effective von Willebrand factor) and the defect in blood coagulation (by raising the factor VIII [FVIII] level). In recent years, desmopressin (1-deamine-8-D-arginine vasopressin [DDAVP]) has become a mainstay of therapy for most patients with mild von Willebrand disease. At appropriate doses, DDAVP causes a 2-fold to 5-fold increase in plasma von Willebrand factor and FVIII concentrations in individuals who are healthy and patients who are responsive. DDAVP can be used to treat bleeding complications or to prepare patients with von Willebrand disease for surgery.
In general, a patient's responsiveness to DDAVP prior to its use for these purposes can be determined. Once determined, such responsiveness is generally consistent in patients over time and within families. In patients with serious bleeding, prompt treatment is important in order to decrease the possibility of complications.
Remember that in type IIB von Willebrand disease, DDAVP may cause a paradoxical drop in the platelet count and should not be used in a therapeutic setting without prior testing to see how the patient responds.
Consult a pediatric or adult hematologist.
No evidence suggests that extensive activity restrictions are necessary for most patients with mild type1 von Willebrand disease. Patients with more severe forms of von Willebrand disease should follow guidelines developed for patients with severe hemophilia.
Schneppenheim R. The pathophysiology of von Willebrand disease: therapeutic implications. Thromb Res. 2011. 128 Suppl 1:S3-7. [Medline].
Grabowski EF, Curran MA, Van Cott EM. Assessment of a cohort of primarily pediatric patients with a presumptive diagnosis of type 1 von Willebrand disease with a novel high shear rate, non-citrated blood flow device. Thromb Res. 2012 Apr. 129(4):e18-24. [Medline].
Akin M. Laboratory diagnostic approach of the parents-children relationship in differentiating low-level von Willebrand factor from mild type 1 von Willebrand disease. Blood Coagul Fibrinolysis. 2012 Jun. 23(4):351-3. [Medline].
Lindsay H, Bergstrom K, Srivaths L. Co-inheritance of mild hemophilia A and heterozygosity for type 2N von Willebrand disease: a diagnostic and therapeutic challenge. Pediatr Blood Cancer. 2014 Oct. 61(10):1888-90. [Medline].
Bowman M, Hopman WM, Rapson D, Lillicrap D, Silva M, James P. A prospective evaluation of the prevalence of symptomatic von Willebrand disease (VWD) in a pediatric primary care population. Pediatr Blood Cancer. 2010 Mar 8. [Medline].
Tosetto A, Castaman G, Rodeghiero F. Evidence-based diagnosis of type 1 von Willebrand disease: a Bayes theorem approach. Blood. 2008 Apr 15. 111(8):3998-4003. [Medline].
Sidonio RF Jr, Gunawardena S, Shaw PH, Ragni M. Predictors of von Willebrand disease in children. Pediatr Blood Cancer. 2012 May. 58(5):736-40. [Medline].
Rodriguez KD, Sun GH, Pike F, et al. Post-tonsillectomy bleeding in children with von Willebrand disease: a single-institution experience. Otolaryngol Head Neck Surg. 2010 May. 142(5):715-21. [Medline].
Loeffelbein F, Funk D, Nakamura L, Zieger B, Grohmann J, Siepe M, et al. Shear-stress induced acquired von Willebrand syndrome in children with congenital heart disease. Interact Cardiovasc Thorac Surg. 2014 Sep 16. [Medline].
[Guideline] Nichols WL, Hultin MB, James AH, et al. von Willebrand disease (VWD): evidence-based diagnosis and management guidelines, the National Heart, Lung, and Blood Institute (NHLBI) Expert Panel report (USA). Haemophilia. 2008 Mar. 14(2):171-232. [Medline].
[Guideline] Nichols WL, Rick ME, Ortel TL, et al. Clinical and laboratory diagnosis of von Willebrand disease: A synopsis of the 2008 NHLBI/NIH guidelines. Am J Hematol. 2009 Mar 16. [Medline].
Batlle J, Torea J, Rendal E, Fernandez MF. The problem of diagnosing von Willebrand's disease. J Intern Med Suppl. 1997. 740:121-8. [Medline].
Carcao MD, Blanchette VS, Dean JA, et al. The Platelet Function Analyzer (PFA-100): a novel in-vitro system for evaluation of primary haemostasis in children. Br J Haematol. 1998 Apr. 101(1):70-3. [Medline].
Federici AB, Mannucci PM. Management of inherited von Willebrand disease in 2007. Ann Med. 2007. 39(5):346-58. [Medline].
[Guideline] Lee CA, Brettler DB. Guidelines for the diagnosis and management of von Willebrand disease. Haemophilia. 1997. 3:1-25.
Nichols WC, Ginsburg D. von Willebrand disease. Medicine (Baltimore). 1997 Jan. 76(1):1-20. [Medline].
Sadler JE, Budde U, Eikenboom JC, et al. Update on the pathophysiology and classification of von Willebrand disease: a report of the Subcommittee on von Willebrand Factor. J Thromb Haemost. 2006 Oct. 4(10):2103-14. [Medline].
Werner EJ. von Willebrand disease in children and adolescents. Pediatr Clin North Am. 1996 Jun. 43(3):683-707. [Medline].
Zhang Z, Blomback M, Anvret M. Understanding von Willebrand's disease from gene defects to the patients. J Intern Med Suppl. 1997. 740:115-9. [Medline].