Medscape is available in 5 Language Editions – Choose your Edition here.


Pediatric Von Willebrand Disease Medication

  • Author: Suchitra S Acharya, MBBS, MD; Chief Editor: Max J Coppes, MD, PhD, MBA  more...
Updated: Dec 16, 2014

Vasopressin analogues

Class Summary

Desmopressin is a synthetic analogue of antidiuretic hormone. It is considered the primary treatment for bleeding in individuals with mild von Willebrand disease (VWD). It works by causing release of von Willebrand factor (VWF) from endothelial storage sites.

Desmopressin can be administered intravenously, intranasally, or subcutaneously. The dose for hemostasis is approximately 15 times the dosage used to treat individuals with diabetes insipidus. The regular intranasal preparation (0.1 mg/mL), which is used to treat persons with diabetes insipidus, is too dilute to elicit a hemostatic response. A high-concentration intranasal preparation (ie, Stimate 1.5 mg/mL) has been licensed and has shown a similar response as the intravenous form.

The higher concentration intranasal preparation allows home treatment for bleeding symptoms; however, experience with its use in the surgical setting is limited. Most experience in treating individuals with von Willebrand disease is with intravenous infusion, with which the response is rapid (ie, peak von Willebrand factor levels in approximately 45-90 min of infusion). Doses may be repeated at intervals of 12-24 hours for continued bleeding or for postoperative use. Desmopressin has also been administered subcutaneously with a favorable response.

Desmopressin (Stimate)


Increases cellular permeability of collecting ducts, resulting in reabsorption of water by kidneys.

Test patients for response prior to usage in a bleeding episode. A 2-fold to 5-fold increase in VWF and FVIII commonly is obtained after treatment.

The higher concentration of desmopressin (ie, Stimate 1.5 mg/mL) is prescribed for VWD to provide an adequate dose.


Plasma products

Class Summary

For patients with von Willebrand disease who do not respond to desmopressin, and for individuals with the rare types 2B or 3 von Willebrand disease, plasma-derived factor VIII (FVIII) concentrates that contain von Willebrand factor in high molecular weight can be used. The product used must contain von Willebrand factor in the high–molecular weight form to be effective. However, most available FVIII concentrates do not contain sufficient von Willebrand factor to be used in von Willebrand disease. Cryoprecipitate contains multimeric von Willebrand factor; however, concerns about possible virus transmission have led many clinicians to choose FVIII products that contain multimeric von Willebrand factor and have undergone viral inactivation processes.

Only a minority of currently available FVIII products contain von Willebrand factor; the protein has been eliminated from the others. In general, the dosage of cryoprecipitate or FVIII to be used is calculated on the basis of FVIII units. Other blood products are rarely required for patients with von Willebrand disease. Platelet transfusion may benefit patients with type 3 von Willebrand disease or platelet-type von Willebrand disease who do not respond to von Willebrand factor–containing concentrates or cryoprecipitate.

Antihemophilic factor/von Willebrand Factor Complex, human (Alphanate, Humate-P, Wilate)


Some FVIII concentrates (eg, Humate-P, Alphanate, Wilate) also contain VWF in high molecular weight form. These concentrates are especially useful in types 2B and 3 vWD.

Alphanate is indicated to prevent excessive bleeding for surgical and invasive procedures in vWD in cases in which desmopressin is either ineffective or contraindicated. It is not indicated for patients with severe vWD (ie, Type 3) undergoing major surgery.

Humate-P is indicated for treatment and prevention of spontaneous and trauma-induced bleeding episodes for patients with mild-to-moderate or severe vWD.

Aminocaproic acid (Amicar)


Inhibits fibrinolysis via inhibition of plasminogen activator substances and, to a lesser degree, through antiplasmin activity. Main problem is that the thrombi that forms during treatment are not lysed and effectiveness is uncertain. Has been used to prevent recurrence of subarachnoid hemorrhage (SAH). Useful in mucous membrane bleeding.

Contributor Information and Disclosures

Suchitra S Acharya, MBBS, MD Program Head, Bleeding Disorders and Thrombosis Program, Cohen Children's Medical Center of New York; Associate Professor of Pediatrics and Pediatrics in Medicine, Hofstra North Shore-LIJ School of Medicine at Hofstra University

Suchitra S Acharya, MBBS, MD is a member of the following medical societies: American Society of Hematology, International Society on Thrombosis and Haemostasis, Hemophilia and Thrombosis Research Society, World Federation of Hemophilia

Disclosure: Nothing to disclose.

Specialty Editor Board

Mary L Windle, PharmD Adjunct Associate Professor, University of Nebraska Medical Center College of Pharmacy; Editor-in-Chief, Medscape Drug Reference

Disclosure: Nothing to disclose.

James L Harper, MD Associate Professor, Department of Pediatrics, Division of Hematology/Oncology and Bone Marrow Transplantation, Associate Chairman for Education, Department of Pediatrics, University of Nebraska Medical Center; Associate Clinical Professor, Department of Pediatrics, Creighton University School of Medicine; Director, Continuing Medical Education, Children's Memorial Hospital; Pediatric Director, Nebraska Regional Hemophilia Treatment Center

James L Harper, MD is a member of the following medical societies: American Society of Pediatric Hematology/Oncology, American Federation for Clinical Research, Council on Medical Student Education in Pediatrics, Hemophilia and Thrombosis Research Society, American Academy of Pediatrics, American Association for Cancer Research, American Society of Hematology

Disclosure: Nothing to disclose.

Chief Editor

Max J Coppes, MD, PhD, MBA Executive Vice President, Chief Medical and Academic Officer, Renown Heath

Max J Coppes, MD, PhD, MBA is a member of the following medical societies: American College of Healthcare Executives, American Society of Pediatric Hematology/Oncology, Society for Pediatric Research

Disclosure: Nothing to disclose.

Additional Contributors

J Martin Johnston, MD Associate Professor of Pediatrics, Mercer University School of Medicine; Director of Hematology/Oncology, The Children's Hospital at Memorial University Medical Center; Consulting Oncologist/Hematologist, St Damien's Pediatric Hospital

J Martin Johnston, MD is a member of the following medical societies: American Academy of Pediatrics, American Society of Pediatric Hematology/Oncology, International Society of Paediatric Oncology

Disclosure: Nothing to disclose.


John D Geil, MD Associate Professor of Pediatrics, Division of Hematology/Oncology, University of Kentucky College of Medicine; Consulting Staff, Department of Pediatric Hematology/Oncology, University of Kentucky Children's Hospital

John D Geil, MD is a member of the following medical societies: American Academy of Pediatrics, American Society of Pediatric Hematology/Oncology, and Society for Neuro-Oncology

Disclosure: Nothing to disclose.

  1. Schneppenheim R. The pathophysiology of von Willebrand disease: therapeutic implications. Thromb Res. 2011. 128 Suppl 1:S3-7. [Medline].

  2. 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].

  3. 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].

  4. 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].

  5. 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].

  6. 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].

  7. 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].

  8. 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].

  9. 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].

  10. [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].

  11. [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].

  12. 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].

  13. 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].

  14. Federici AB, Mannucci PM. Management of inherited von Willebrand disease in 2007. Ann Med. 2007. 39(5):346-58. [Medline].

  15. [Guideline] Lee CA, Brettler DB. Guidelines for the diagnosis and management of von Willebrand disease. Haemophilia. 1997. 3:1-25.

  16. Nichols WC, Ginsburg D. von Willebrand disease. Medicine (Baltimore). 1997 Jan. 76(1):1-20. [Medline].

  17. 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].

  18. Werner EJ. von Willebrand disease in children and adolescents. Pediatr Clin North Am. 1996 Jun. 43(3):683-707. [Medline].

  19. 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].

Structure and domains of von Willebrand factor.
All material on this website is protected by copyright, Copyright © 1994-2016 by WebMD LLC. This website also contains material copyrighted by 3rd parties.