Pancreatic triacylglycerol lipase deficiency

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ORPHA:309031OMIM:614338K90.3
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Overview

Pancreatic triacylglycerol lipase deficiency, also known as pancreatic lipase deficiency or congenital pancreatic lipase deficiency, is a rare condition in which the pancreas does not produce enough of a specific enzyme called pancreatic triglyceride lipase (PTL). This enzyme is essential for breaking down fats (triglycerides) from the food you eat so your body can absorb them properly. Without enough of this enzyme, dietary fats pass through the digestive system without being fully digested or absorbed. The main symptom is steatorrhea, which means fatty, greasy, foul-smelling stools. People with this condition may also experience bloating, abdominal discomfort, diarrhea, and over time may develop deficiencies in fat-soluble vitamins (A, D, E, and K) because these vitamins need fat to be absorbed. In children, poor fat absorption can lead to failure to thrive and growth problems. Unlike some other causes of fat malabsorption, this condition is isolated to the lipase enzyme, meaning the rest of the pancreas typically functions normally. Treatment focuses on replacing the missing enzyme. Pancreatic enzyme replacement therapy (PERT) taken with meals can help the body digest and absorb fats more effectively. Dietary adjustments, such as reducing very high-fat meals or supplementing with medium-chain triglycerides (MCTs) that are easier to absorb, may also be recommended. With proper management, most people can lead relatively normal lives, though lifelong enzyme supplementation is usually needed.

Also known as:

Key symptoms:

Greasy, fatty, foul-smelling stools (steatorrhea)DiarrheaBloating and gasAbdominal pain or discomfortPoor weight gain in childrenFailure to thrive in infantsVitamin A deficiency (night blindness, dry skin)Vitamin D deficiency (weak bones)Vitamin E deficiency (nerve problems)Vitamin K deficiency (easy bruising or bleeding)Oily or floating stoolsFatigue from poor nutrition

Clinical phenotype terms (21)— hover any for plain English
Exocrine pancreatic insufficiencyHP:0001738SteatorrheaHP:0002570Reduced circulating vitamin A concentrationHP:0004905Decreased circulating vitamin K concentrationHP:0011892Decreased circulating vitamin D concentrationHP:0100512Decreased circulating vitamin E concentrationHP:0100513RicketsHP:0002748OsteomalaciaHP:0002749HemeralopiaHP:0012047
Inheritance

Autosomal recessive

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

Age of Onset

Infantile

Begins in infancy, roughly 1 month to 2 years old

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Pancreatic triacylglycerol lipase deficiency.

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No actively recruiting trials found for Pancreatic triacylglycerol lipase deficiency at this time.

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No specialists are currently listed for Pancreatic triacylglycerol lipase deficiency.

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 Pancreatic triacylglycerol lipase deficiency.

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Community

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Caregiver Resources

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.What dose of pancreatic enzyme replacement therapy does my child (or I) need, and how should it be adjusted?,How often should we check fat-soluble vitamin levels and nutritional status?,Are there specific dietary changes that can help improve fat absorption?,How can we tell if the enzyme replacement therapy is working well enough?,Should other family members be tested for this condition?,What should we do if symptoms return or worsen despite taking enzymes?,Are there any new treatments or clinical trials available for this condition?

Common questions about Pancreatic triacylglycerol lipase deficiency

What is Pancreatic triacylglycerol lipase deficiency?

Pancreatic triacylglycerol lipase deficiency, also known as pancreatic lipase deficiency or congenital pancreatic lipase deficiency, is a rare condition in which the pancreas does not produce enough of a specific enzyme called pancreatic triglyceride lipase (PTL). This enzyme is essential for breaking down fats (triglycerides) from the food you eat so your body can absorb them properly. Without enough of this enzyme, dietary fats pass through the digestive system without being fully digested or absorbed. The main symptom is steatorrhea, which means fatty, greasy, foul-smelling stools. People

How is Pancreatic triacylglycerol lipase deficiency inherited?

Pancreatic triacylglycerol lipase deficiency follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Pancreatic triacylglycerol lipase deficiency typically begin?

Typical onset of Pancreatic triacylglycerol lipase deficiency is infantile. Age of onset can vary across affected individuals.