Acitretin/etretinate embryopathy

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Overview

Acitretin/etretinate embryopathy is a rare condition that occurs when a baby is exposed to certain vitamin A-related medications — specifically acitretin or etretinate — while still in the womb. These drugs are powerful treatments for severe skin conditions like psoriasis, but they can cause serious birth defects if a pregnant person takes them during pregnancy. The condition is also sometimes called retinoid embryopathy when caused by this class of drugs. When a developing baby is exposed to these medications, especially during the first trimester, it can affect the formation of many body systems. The most commonly affected areas include the brain, face, ears, heart, and limbs. Babies may be born with structural differences in the skull and brain, hearing problems, heart defects, and differences in how their arms or legs developed. There is no cure for the birth defects caused by this exposure. Treatment focuses on managing each individual problem the child has — for example, surgery for heart defects, hearing aids or cochlear implants for hearing loss, and therapies to support development. Prevention is the most important strategy, which is why acitretin and etretinate come with strict pregnancy prevention programs. Women who have taken etretinate may carry the drug in their body fat for years, meaning the risk can persist long after stopping the medication.

Also known as:

Key symptoms:

Abnormal skull shape or underdevelopment of the skull bonesBrain malformations, including problems with brain structureSmall or absent ears, or ears that are not formed correctlyHearing loss or deafnessHeart defects present at birthCleft palate or differences in facial structureShort or missing limb bonesExtra fingers or toes, or fused fingers or toesEye abnormalitiesIntellectual disability or developmental delaysSpinal cord or vertebral abnormalitiesThymus gland problems affecting the immune system

Clinical phenotype terms (27)— hover any for plain English
Maternal teratogenic exposureHP:0011438Cupped earHP:0000378Abnormal retinal morphologyHP:0000479Hypoplasia of the thymusHP:0000778Conotruncal defectHP:0001710Hypoplastic nasal septumHP:0005104Atrioventricular canal defectHP:0006695Aplasia/Hypoplasia of the optic nerveHP:0008058Abnormality of the calcaneusHP:0008364Bilateral sensorineural hearing impairmentHP:0008619Median cleft palateHP:0009099
Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Acitretin/etretinate embryopathy.

View clinical trials →

No actively recruiting trials found for Acitretin/etretinate embryopathy at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Acitretin/etretinate embryopathy community →

No specialists are currently listed for Acitretin/etretinate embryopathy.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

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 Acitretin/etretinate embryopathy.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Acitretin/etretinate embryopathy

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Caregiver Resources

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Questions for your doctor

Bring these to your next appointment

  • Q1.Which organs or body systems are affected in my child, and how severe is each problem?,What specialists does my child need to see, and how often?,Are there surgeries or procedures that could correct any of my child's structural differences?,What early intervention or therapy services should we start right away?,What should I watch for at home that would mean my child needs emergency care?,What is the likely long-term outlook for my child's development and independence?,Are there support groups or resources for families dealing with retinoid embryopathy?

Common questions about Acitretin/etretinate embryopathy

What is Acitretin/etretinate embryopathy?

Acitretin/etretinate embryopathy is a rare condition that occurs when a baby is exposed to certain vitamin A-related medications — specifically acitretin or etretinate — while still in the womb. These drugs are powerful treatments for severe skin conditions like psoriasis, but they can cause serious birth defects if a pregnant person takes them during pregnancy. The condition is also sometimes called retinoid embryopathy when caused by this class of drugs. When a developing baby is exposed to these medications, especially during the first trimester, it can affect the formation of many body sy

How is Acitretin/etretinate embryopathy inherited?

Acitretin/etretinate embryopathy follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Acitretin/etretinate embryopathy typically begin?

Typical onset of Acitretin/etretinate embryopathy is neonatal. Age of onset can vary across affected individuals.