Combined pancreatic lipase-colipase deficiency

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ORPHA:309111OMIM:614338K90.3
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Overview

Combined pancreatic lipase-colipase deficiency is an extremely rare inherited condition that affects the body's ability to digest dietary fats. The pancreas normally produces two important proteins — pancreatic lipase and colipase — that work together to break down fats from the food you eat. In this condition, both of these proteins are missing or do not work properly. Without them, fat passes through the digestive system without being absorbed, leading to a condition called fat malabsorption or steatorrhea. The main symptoms typically appear in infancy or early childhood and include greasy, foul-smelling stools (steatorrhea), poor weight gain, failure to thrive, and possible deficiencies in fat-soluble vitamins (A, D, E, and K). Children may have bloating, abdominal discomfort, and diarrhea after eating fatty foods. Because fat is an important source of calories and essential fatty acids, untreated children may experience growth delays and nutritional problems. Treatment focuses on replacing the missing enzymes using pancreatic enzyme replacement therapy (PERT), which is taken with meals and snacks. Dietary modifications, including adjusting fat intake and supplementing fat-soluble vitamins, are also important parts of management. With proper treatment, most patients can lead relatively normal lives, though lifelong enzyme supplementation is typically required.

Key symptoms:

Greasy, oily, foul-smelling stoolsDiarrhea after eating fatty foodsPoor weight gain in infancy and childhoodFailure to thriveBloating and abdominal discomfortExcessive gasGrowth delaysFat-soluble vitamin deficiencies (vitamins A, D, E, K)Dry skin or skin problems from vitamin deficiencyEasy bruising from vitamin K deficiencyBone weakness from vitamin D deficiencyFatigue and low energyLarge, bulky stools that may float

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 Combined pancreatic lipase-colipase deficiency.

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No actively recruiting trials found for Combined pancreatic lipase-colipase deficiency at this time.

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No specialists are currently listed for Combined pancreatic lipase-colipase 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 Combined pancreatic lipase-colipase deficiency.

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Community

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Caregiver Resources

NORD Caregiver Resources

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Mental Health Support

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Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.What is the correct dose of pancreatic enzymes for my child's age and weight?,How should we adjust enzyme doses as my child grows or eats different amounts of fat?,Which fat-soluble vitamins need to be supplemented, and how often should levels be checked?,Are there any dietary changes that can help manage symptoms better?,How often should growth and nutritional status be monitored?,Should other family members be tested for this condition?,Are there any new treatments or clinical trials available for this condition?

Common questions about Combined pancreatic lipase-colipase deficiency

What is Combined pancreatic lipase-colipase deficiency?

Combined pancreatic lipase-colipase deficiency is an extremely rare inherited condition that affects the body's ability to digest dietary fats. The pancreas normally produces two important proteins — pancreatic lipase and colipase — that work together to break down fats from the food you eat. In this condition, both of these proteins are missing or do not work properly. Without them, fat passes through the digestive system without being absorbed, leading to a condition called fat malabsorption or steatorrhea. The main symptoms typically appear in infancy or early childhood and include greasy,

How is Combined pancreatic lipase-colipase deficiency inherited?

Combined pancreatic lipase-colipase deficiency follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Combined pancreatic lipase-colipase deficiency typically begin?

Typical onset of Combined pancreatic lipase-colipase deficiency is infantile. Age of onset can vary across affected individuals.