Hyperinsulinism due to short chain 3-hydroxylacyl-CoA dehydrogenase deficiency

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ORPHA:71212OMIM:231530E71.3
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Overview

Hyperinsulinism due to short chain 3-hydroxylacyl-CoA dehydrogenase deficiency, also known as SCHAD deficiency or HAD deficiency, is a rare inherited metabolic disorder that causes the body to produce too much insulin. Insulin is the hormone that lowers blood sugar, so when too much is made, blood sugar levels can drop dangerously low — a condition called hypoglycemia. This happens because the SCHAD enzyme, which normally helps break down certain fats for energy, also plays an important role in controlling insulin release from the pancreas. Without this enzyme working properly, insulin is released in an uncontrolled way. The most serious concern with this condition is recurrent low blood sugar episodes, which can begin in the newborn period or early infancy. If low blood sugar is not treated quickly, it can cause seizures, brain injury, or other serious complications. Babies may appear floppy, feed poorly, or have seizures as early warning signs. Fortunately, many children with SCHAD deficiency respond well to treatment with a medication called diazoxide, which helps reduce insulin release. With early diagnosis and proper management, many children can achieve good blood sugar control and lead healthy lives. However, ongoing monitoring and dietary management are important parts of long-term care.

Also known as:

Key symptoms:

Low blood sugar (hypoglycemia) episodesSeizures caused by low blood sugarPoor feeding in newborns or infantsUnusual sleepiness or difficulty wakingLimpness or poor muscle tone (floppiness) in babiesSweating, shakiness, or pallor during low blood sugar episodesIrritability or unusual cryingElevated levels of certain fatty acid breakdown products in blood and urineElevated blood ammonia levels in some casesAbnormal liver function in some patients

Clinical phenotype terms (36)— hover any for plain English
Decreased 3-hydroxyacyl-CoA dehydrogenase levelHP:0100950Hypoketotic hypoglycemiaHP:0001985Neonatal hypoglycemiaHP:0001998Hypoglycemic seizuresHP:0002173Dicarboxylic aciduriaHP:0003215Proportionate short statureHP:0003508Hypoglycemic encephalopathyHP:0006929Fasting hyperinsulinemiaHP:0008283Abnormal circulating acylcarnitine concentrationHP:0012071Increased circulating free fatty acid levelHP:0030781Increased C-peptide levelHP:0030796
Inheritance

Autosomal recessive

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

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Hyperinsulinism due to short chain 3-hydroxylacyl-CoA dehydrogenase deficiency.

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No specialists are currently listed for Hyperinsulinism due to short chain 3-hydroxylacyl-CoA dehydrogenase 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

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Questions for your doctor

Bring these to your next appointment

  • Q1.How often should we check blood sugar at home, and what levels should concern us?,What is our emergency plan if my child has a severe low blood sugar episode?,How long will my child need to take diazoxide, and what side effects should we watch for?,Are there any dietary restrictions or foods we should avoid?,Should other family members be tested for this condition?,What developmental monitoring or support services should we consider?,Are there any clinical trials or new treatments we should know about?

Common questions about Hyperinsulinism due to short chain 3-hydroxylacyl-CoA dehydrogenase deficiency

What is Hyperinsulinism due to short chain 3-hydroxylacyl-CoA dehydrogenase deficiency?

Hyperinsulinism due to short chain 3-hydroxylacyl-CoA dehydrogenase deficiency, also known as SCHAD deficiency or HAD deficiency, is a rare inherited metabolic disorder that causes the body to produce too much insulin. Insulin is the hormone that lowers blood sugar, so when too much is made, blood sugar levels can drop dangerously low — a condition called hypoglycemia. This happens because the SCHAD enzyme, which normally helps break down certain fats for energy, also plays an important role in controlling insulin release from the pancreas. Without this enzyme working properly, insulin is rele

How is Hyperinsulinism due to short chain 3-hydroxylacyl-CoA dehydrogenase deficiency inherited?

Hyperinsulinism due to short chain 3-hydroxylacyl-CoA dehydrogenase deficiency follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Hyperinsulinism due to short chain 3-hydroxylacyl-CoA dehydrogenase deficiency typically begin?

Typical onset of Hyperinsulinism due to short chain 3-hydroxylacyl-CoA dehydrogenase deficiency is neonatal. Age of onset can vary across affected individuals.