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Desmopressin (DDAVP) in bleeding disorders of childhood
1Universitäts-Kinderklinik, Freiburg, Germany.
Insights
Desmopressin (DDAVP) effectively treats bleeding disorders in children, including mild hemophilia and von Willebrand disease (vWD). Caution is advised for infants due to potential water intoxication risks.
Area of Science:
- Pediatric Hematology
- Pharmacology
Background:
- Desmopressin (DDAVP) is a synthetic analog of vasopressin used for bleeding disorder management in children.
- It is a primary treatment for mild hemophilia and type 1 von Willebrand disease (vWD).
Observation:
- DDAVP demonstrates efficacy in various vWD variants and platelet function defects.
- It mitigates bleeding risks associated with uremia and drug-induced complications.
- A test dose is crucial to predict hemostatic response, measured by bleeding time and PTT, indicating increased Factor VIII or von Willebrand factor levels.
Findings:
- Mild, transient side effects like headache and flushing can occur but are minimized with appropriate dosing (≤0.3 mcg/kg) and infusion duration (20-30 minutes).
- The antidiuretic effect requires careful monitoring in infants under 18 months to prevent water intoxication, especially with fluid substitution.
- DDAVP offers significant advantages, including reduced reliance on plasma concentrates, minimizing infection/immunological risks, and cost savings.
Implications:
- DDAVP is a valuable therapeutic option for pediatric bleeding disorders, particularly type 1 vWD.
- Its use can decrease exposure to blood products and associated complications.
- Careful patient selection and monitoring are essential for safe and effective DDAVP administration in young children.
Abstract:
As in adults, desmopressin (DDAVP) can be used in children for prophylaxis of bleeding and to stop bleeding in many hereditary and acquired bleeding disorders. DDAVP is the treatment of choice in children with mild hemophilia and type 1 von Willebrand's disease (vWD). It is effective in some variants of vWD and in many patients with platelet function defects. It reduces the bleeding diathesis of children with uremia and drug-induced bleeding complications. In any case, a test dose of DDAVP has to be given to the patient to predict the hemostatic effect before relying on this drug for treatment. The response can be measured by shortening of the bleeding time (BT) and of partial thromboplastin time (PTT), indicating a rise of Factor (F) VIII or von Willebrand factor (vWF). Side effects such as facial flushing, transient headache, increased pulse rate, and drop in systolic blood pressure are mild and transient. They can be minimized when the dose is not exceeding 0.3 microg/kg body weight, and the infusion lasts at least 20 to 30 minutes. The strong antidiuretic action of DDAVP has some potential problems that are negligible in adults and older children when water intake is restricted. In infants and small children under the age of 18 months, however, DDAVP should be used with caution and with close surveillance in order to prevent water intoxication and electrolyte imbalance. The danger is increased when the patients receive parenteral fluid substitution. The advantages of DDAVP include the reduction in the use of plasma factor concentrates, thereby minimizing the danger of immunological or infectious complications, as well as the considerable reduction of costs realized by treatment with this form of medication. Fortunately, it can be applied successfully in the most frequent hereditary bleeding disorder, namely vWD type 1.
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