Arginine Vasopressin Deficiency

Understanding a Rare and Serious Condition

Arginine Vasopressin Deficiency (AVP-D), formerly known as central diabetes insipidus (DI), is a rare disorder characterized by excessive thirst and the production of large volumes of diluted urine. Unlike diabetes mellitus, which involves insulin and blood sugar regulation, AVP-D involves a deficiency of arginine vasopressin (also known as antidiuretic hormone or ADH). To reduce confusion between the two conditions, a name change was proposed in 2022 by experts across endocrinology, nephrology, and pediatrics.

AVP-D occurs when the brain fails to produce enough vasopressin, a hormone made in the hypothalamus and released by the pituitary gland. Vasopressin plays a critical role in maintaining the body’s water balance by regulating reabsorption in the kidneys. Without sufficient vasopressin, the kidneys cannot concentrate urine, leading to rapid fluid loss and a risk of severe dehydration.

AVP-D is often caused by damage to the hypothalamus or pituitary gland commonly due to tumors, surgery, trauma, or other conditions affecting this area of the brain.

What Causes AVP-D?

Arginine Vasopressin Deficiency (AVP-D) occurs when the hypothalamus or pituitary gland is damaged and can no longer produce or release enough vasopressin (ADH). This damage disrupts the body’s ability to regulate fluid balance.

Common causes include:

• Brain tumors, especially hypothalamic-pituitary tumors such as craniopharyngioma
• Surgical removal of tumors or lesions near the pituitary gland
• Traumatic brain injury
• Infections affecting the brain (e.g., meningitis, encephalitis)
• Genetic conditions or congenital abnormalities
• Autoimmune diseases or inflammatory disorders

At RAWF, we focus specifically on hypothalamic-pituitary brain tumors and the lifelong challenges survivors face, including AVP-D.

Symptoms of Diabetes Insipidus

The most common symptoms include:

• Excessive thirst (polydipsia)
• Excessive urine output (polyuria)

Patients may urinate frequently and pass large volumes of pale, diluted urine. If fluid loss isn’t adequately replaced, it can quickly lead to dehydration and dangerous electrolyte imbalances, including hypernatremia (high sodium levels).

In some cases using desmopressin, the synthetic form of vasopressin, a hormone naturally produced by the brain to help the body regulate water balance., patients may develop hyponatremia (low sodium levels). This condition can be life-threatening if not recognized early and managed properly. Symptoms of hyponatremia may include headache, nausea, confusion, seizures, or in severe cases, coma.

Some individuals with hypothalamic damage also experience adipsia—a lack of thirst—even when dehydrated, making the condition especially difficult to manage without careful sodium and fluid monitoring.

Treatment

Diagnosing AVP-D involves blood and urine tests, including water deprivation testing, to evaluate the body’s ability to concentrate urine and maintain sodium balance.

Treatment focuses on hormone replacement and fluid management:

• Desmopressin, a synthetic form of vasopressin, is used to restore water balance.
• Routine sodium monitoring is critical, especially for patients with adipsia or those newly diagnosed, to avoid both dehydration and overcorrection (low sodium).

For patients with adipsia or are newly diagnosed, routine blood sodium testing may be required to ensure sodium levels are within a safe range and to reach a proper dosage of desmopressin.

Desmopressin is available in various forms, including:

1. Oral Tablets: Desmopressin is available in tablet form, which can be taken orally. This form is often used to treat conditions like diabetes insipidus and nocturnal enuresis. The tablets are usually taken with water.
2. Nasal Spray: This is one of the more common forms of desmopressin. The medication is administered by spraying it into the nostrils. It is often used to treat conditions such as diabetes insipidus and nocturnal enuresis. The nasal spray is absorbed through the nasal mucosa and enters the bloodstream.
3. Nasal Solution: Similar to the nasal spray, the nasal solution is also administered through the nostrils. It’s usually used for similar indications as the nasal spray, and the liquid form allows for controlled dosing.
4. Injectable Form: Desmopressin can also be administered via injection, either intramuscularly or subcutaneously. Injectable forms are often used in cases where rapid onset of action is needed, such as in emergency situations or medical procedures.
5. On the Horizon: ET-600 Oral Liquid Desmopressin
   ET-600 is a new pediatric-focused, FDA-reviewed oral liquid form of desmopressin for AVP-D. If approved in early 2026, it would be the first FDA-approved oral liquid option, offering more precise dosing than tablets or compounded versions.

The choice of the appropriate form of desmopressin depends on the patient’s condition, the severity of the condition, the desired speed of onset, and individual preferences. The prescribing healthcare professional will determine the most suitable form and dosage based on the patient’s medical history and needs. It’s important for patients to follow their healthcare provider’s instructions carefully and to monitor their response to the medication.

Home Sodium Monitoring: A Breakthrough in Progress

Managing Arginine Vasopressin Deficiency (AVP-D) especially in children with adipsic diabetes insipidus requires vigilant control of fluid balance and blood sodium levels. Yet, there is no FDA-approved home testing mechanism for sodium, leaving families reliant on lab visits or guesswork to manage serious risks like seizures, dehydration, and hospitalization.

The Raymond A. Wood Foundation is leading efforts to change that.

In partnership with Giner Labs, a global leader in electrochemical innovation, RAWF is developing a home-use blood sodium meter, a simple fingerstick device that allows patients and caregivers to monitor sodium levels in real time.

This patient-driven project began through collaboration with medical experts at Children’s Hospital of Philadelphia (CHOP) and is rooted in the lived experience of families in our community.

Why it matters:

• Detects dangerous sodium swings early
• Supports safer desmopressin use
• Reduces emergency visits and hospitalizations
• Empowers families with real-time data

RAWF has provided nearly $200,000 in additional funding beyond initial NIH support and is now actively raising funds to launch Phase 2 development which will be critical to advancing the prototype toward FDA approval and broader access. Find out more

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