L-ferritin deficiency

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ORPHA:440731OMIM:615604E83.1
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Overview

L-ferritin deficiency is a rare inherited condition that affects how the body stores and manages iron. Ferritin is a protein that acts like a storage container for iron inside your cells. There are two main types of ferritin: H-ferritin and L-ferritin. L-ferritin, made by the FTL gene, is especially important in the liver, spleen, and red blood cells. When the FTL gene does not work properly, the body cannot make enough L-ferritin, which disrupts normal iron storage throughout the body. This condition is also sometimes called hereditary hyperferritinemia-cataract syndrome (HHCS) when a different type of FTL mutation causes too much ferritin to build up — but true L-ferritin deficiency refers to having too little functional L-ferritin. People with L-ferritin deficiency may have very low ferritin levels in their blood, which can be mistaken for iron deficiency anemia. However, the underlying cause is genetic, not a lack of dietary iron. Symptoms can include fatigue, weakness, and problems related to abnormal iron handling in the body. Because this condition is so rare and can look like common iron deficiency, it is often misdiagnosed or diagnosed late. Treatment is mainly focused on managing symptoms, since there is no cure. Genetic testing is the most reliable way to confirm the diagnosis. Patients are usually followed by a hematologist and a clinical geneticist.

Key symptoms:

Very low ferritin levels in the bloodFatigue and low energyWeaknessAnemia-like symptoms despite normal or near-normal iron levelsPale skinShortness of breath with activityDifficulty concentratingDizziness or lightheadednessPoor exercise tolerance

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 L-ferritin deficiency.

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No actively recruiting trials found for L-ferritin deficiency at this time.

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

Search ClinicalTrials.gov ↗Join the L-ferritin deficiency community →

No specialists are currently listed for L-ferritin 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 L-ferritin deficiency.

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Community

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

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.Is my low ferritin definitely caused by the FTL gene, and should other family members be tested?,Should I avoid iron supplements, and are there any foods or vitamins I should limit?,How often do I need blood tests to monitor my iron and ferritin levels?,Are there any clinical trials or research studies I could join?,What symptoms should prompt me to seek urgent medical attention?,Should I see a hematologist regularly, and are there specialists who have experience with this specific condition?,How might this condition affect me as I get older, and what should I watch for long-term?

Common questions about L-ferritin deficiency

What is L-ferritin deficiency?

L-ferritin deficiency is a rare inherited condition that affects how the body stores and manages iron. Ferritin is a protein that acts like a storage container for iron inside your cells. There are two main types of ferritin: H-ferritin and L-ferritin. L-ferritin, made by the FTL gene, is especially important in the liver, spleen, and red blood cells. When the FTL gene does not work properly, the body cannot make enough L-ferritin, which disrupts normal iron storage throughout the body. This condition is also sometimes called hereditary hyperferritinemia-cataract syndrome (HHCS) when a differ

How is L-ferritin deficiency inherited?

L-ferritin deficiency follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.