Isolated thyrotropin-releasing hormone deficiency

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ORPHA:238670OMIM:275120E03.1
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Overview

Isolated thyrotropin-releasing hormone (TRH) deficiency is an extremely rare endocrine disorder in which the hypothalamus does not produce enough TRH. TRH is a small hormone made in the brain's hypothalamus that signals the pituitary gland to release thyroid-stimulating hormone (TSH), which in turn tells the thyroid gland to produce thyroid hormones. When TRH is missing or severely reduced, this chain of signals breaks down, leading to a form of central hypothyroidism — meaning the thyroid gland itself is normal but does not receive the proper instructions to make enough thyroid hormone. Patients with this condition typically show signs and symptoms of hypothyroidism, which can include fatigue, weight gain, cold intolerance, constipation, dry skin, slow growth in children, and developmental delays if the condition begins early in life. Because thyroid hormones are essential for brain development, metabolism, and growth, untreated TRH deficiency in infants and children can lead to significant intellectual and physical developmental problems. The main treatment is thyroid hormone replacement therapy, usually with levothyroxine (synthetic T4), which can effectively restore normal thyroid hormone levels in the body. Early diagnosis and consistent treatment are critical to preventing long-term complications, especially in young children. Because this condition is so rare, it is often diagnosed only after other more common causes of hypothyroidism have been ruled out. It may also be referred to as tertiary hypothyroidism due to isolated TRH deficiency.

Also known as:

Key symptoms:

Fatigue and low energyWeight gain or difficulty losing weightFeeling cold when others are comfortableConstipationDry skin and hairSlow growth in childrenDelayed development or learning difficulties in childrenPuffy faceMuscle weaknessSlow heart rateDepression or low moodPoor feeding in infantsProlonged jaundice in newborns

Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Isolated thyrotropin-releasing hormone deficiency.

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No actively recruiting trials found for Isolated thyrotropin-releasing hormone deficiency at this time.

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No specialists are currently listed for Isolated thyrotropin-releasing hormone deficiency.

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Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Isolated thyrotropin-releasing hormone deficiency.

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Community

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Caregiver Resources

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Questions for your doctor

Bring these to your next appointment

  • Q1.How was this specific diagnosis confirmed, and could other causes of central hypothyroidism be involved?,What is the correct dose of levothyroxine for my (or my child's) age and weight, and how often should it be adjusted?,Should genetic testing be done to look for a specific cause?,Are there other hormone deficiencies that should be tested for?,How often should blood tests be done to monitor thyroid levels?,What signs should I watch for that might mean the medication dose needs to change?,Could this condition have been missed on newborn screening, and should my other children be tested?

Common questions about Isolated thyrotropin-releasing hormone deficiency

What is Isolated thyrotropin-releasing hormone deficiency?

Isolated thyrotropin-releasing hormone (TRH) deficiency is an extremely rare endocrine disorder in which the hypothalamus does not produce enough TRH. TRH is a small hormone made in the brain's hypothalamus that signals the pituitary gland to release thyroid-stimulating hormone (TSH), which in turn tells the thyroid gland to produce thyroid hormones. When TRH is missing or severely reduced, this chain of signals breaks down, leading to a form of central hypothyroidism — meaning the thyroid gland itself is normal but does not receive the proper instructions to make enough thyroid hormone. Pati

How is Isolated thyrotropin-releasing hormone deficiency inherited?

Isolated thyrotropin-releasing hormone deficiency follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.