What are the indications for desmopressin (antidiuretic hormone (ADH) analogue)?

Desmopressin Indications

Desmopressin is FDA-approved for three primary indications: central diabetes insipidus (as antidiuretic replacement therapy), hemophilia A (in patients with factor VIII levels >5%), and mild-to-moderate von Willebrand disease Type I (with factor VIII levels >5%), with additional established uses in nocturnal enuresis and diagnostic testing of renal tubular function. 1

FDA-Approved Indications

Central Diabetes Insipidus

• Desmopressin serves as antidiuretic replacement therapy for central (cranial) diabetes insipidus, recognized as the drug of choice due to its selective antidiuretic activity without adverse pressor effects 1, 2
• Also indicated for managing temporary polyuria and polydipsia following head trauma or pituitary surgery 1
• Critical limitation: Desmopressin is ineffective and NOT indicated for nephrogenic diabetes insipidus 1

Hemophilia A

• Indicated for patients with factor VIII coagulant activity levels greater than 5% to maintain hemostasis during surgical procedures and postoperatively 1
• Used to reduce bleeding with episodes of spontaneous or traumatic injuries including hemarthroses, intramuscular hematomas, or mucosal bleeding 1
• Works by promoting release of von Willebrand factor and factor VIII from endothelial storage sites 3, 4

Von Willebrand Disease (Type I)

• Approved for patients with mild-to-moderate Type I disease with factor VIII levels greater than 5% 1
• Maintains hemostasis during surgical procedures or traumatic injuries such as hemarthroses, intramuscular hematomas, or mucosal bleeding 1
• Critical limitation: NOT indicated for severe Type I von Willebrand disease or when abnormal molecular forms of factor VIII antigen are present 1
• Important warning: Use in Type IIB von Willebrand disease may cause thrombosis due to platelet aggregation 1

Established Off-Label Uses

Nocturnal Enuresis (Bedwetting)

• Desmopressin is an evidence-based first-line pharmacological treatment for nocturnal enuresis, reducing wet nights by approximately 30-40% during active treatment 5, 6
• Approximately 30% of children become completely dry (full responders) and 40% achieve partial response 5, 6
• Works best in children with nocturnal polyuria (nighttime urine production >130% of expected bladder capacity) and normal bladder function 5
• Also the drug of choice for nocturnal enuresis in adults, particularly when associated with nocturnal polyuria, with effectiveness rates of 10-65% 7

Diagnostic Testing

• Used as a diagnostic test to distinguish ACTH-dependent Cushing's syndrome from pseudo-Cushing's syndrome 3
• The desmopressin test has high specificity for Cushing's disease, based on ACTH-secreting adenomas expressing vasopressin V1b (V3) receptors, producing a rise in plasma ACTH after desmopressin injection 3
• Less complex and expensive than the Dex-CRH test with good diagnostic performance 3

Other Bleeding Disorders

• Used in uremic bleeding, dental extractions, epistaxis, and menstrual bleeding 4
• May reduce blood loss during surgical operations with unusually large blood losses 8
• Can shorten prolonged bleeding time in patients with uremia, liver cirrhosis, and platelet dysfunctions 8
• Important note: Prophylactic use in cardiac surgery patients without platelet hemostatic defects shows no benefit and is not recommended 3

Critical Safety Contraindications

Absolute Contraindications

• Hyponatremia or history of hyponatremia 1
• Moderate to severe renal impairment (creatinine clearance <50 mL/min) 1
• Known or suspected syndrome of inappropriate antidiuretic hormone (SIADH) secretion 1
• Polydipsia (excessive thirst/drinking) - this is an absolute contraindication 5, 6, 1
• Concomitant use with loop diuretics or systemic/inhaled glucocorticoids 1
• During illnesses that can cause fluid or electrolyte imbalance 1
• Heart failure or uncontrolled hypertension 1

Mandatory Safety Measures

• Fluid restriction is essential: Evening fluid intake must be limited to 200 mL (6 ounces) or less with no drinking until morning to prevent water intoxication with hyponatremia and convulsions 5, 6, 7, 1
• Ensure serum sodium is normal before starting therapy 1
• Measure serum sodium within 1 week and approximately 1 month after starting, then periodically during treatment 1
• More frequent sodium monitoring required in patients ≥65 years and those at increased risk of hyponatremia 1
• Nasal spray formulations are strongly discouraged due to higher risk of water intoxication with hyponatremia and convulsions 5, 6, 7

Common Clinical Pitfalls

• Not screening for polydipsia before initiating therapy - this is a critical mistake as it represents an absolute contraindication 5, 6
• Inadequate fluid restriction counseling can lead to life-threatening water intoxication 5, 6, 7
• Using nasal spray formulation increases hyponatremia risk compared to oral formulations 5, 6, 7
• Continuing desmopressin indefinitely without regular short drug holidays prevents assessment of ongoing need 5, 6
• Attempting to use desmopressin in nephrogenic diabetes insipidus where it is completely ineffective 1