Hypotrichosis-lymphedema-telangiectasia-renal defect syndrome

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ORPHA:69735OMIM:137940Q82.0
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Overview

Hypotrichosis-lymphedema-telangiectasia-renal defect syndrome (also called HLTRS or sometimes referred to by the gene involved, SOX18-related syndrome) is a very rare inherited condition that affects several parts of the body at the same time. The name describes its four main features: hypotrichosis (very sparse or thin hair), lymphedema (swelling caused by a buildup of fluid, usually in the legs or arms), telangiectasias (small, widened blood vessels visible near the skin surface, often appearing as red or purple spider-like marks), and renal defects (problems with how the kidneys work or are formed). This condition is caused by changes (mutations) in the SOX18 gene, which plays an important role in the development of blood vessels, lymphatic vessels, and hair follicles. When this gene does not work properly, the body's systems that rely on it do not develop or function as they should. Symptoms can vary quite a bit from person to person, even within the same family. There is currently no cure for this syndrome. Treatment focuses on managing each symptom separately. For example, compression garments and physical therapy can help with lymphedema, while kidney problems may need monitoring or specialist care. Because this condition is so rare, most care is provided by a team of specialists working together. Early diagnosis is important so that complications, especially kidney problems, can be caught and managed as soon as possible.

Also known as:

Key symptoms:

Very sparse, thin, or absent hair on the scalp and body (hypotrichosis)Swelling of the legs, arms, or other body parts due to fluid buildup (lymphedema)Small red or purple spider-like blood vessels visible on the skin (telangiectasias)Kidney abnormalities or reduced kidney functionSwelling present from birth or early infancySparse or absent eyebrows and eyelashesSkin changes related to abnormal blood vesselsPossible protein in the urine (a sign of kidney stress)Recurrent swelling that may worsen over timePossible abnormal lymph vessel development

Clinical phenotype terms (16)— hover any for plain English
Absent eyelashesHP:0000561Predominantly lower limb lymphedemaHP:0003550Abnormality of the lymphatic systemHP:0100763Palmar telangiectasiaHP:0100869Plantar telangiectasiaHP:0100870Hydrocele testisHP:0000034Palpebral edemaHP:0100540
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 Hypotrichosis-lymphedema-telangiectasia-renal defect syndrome.

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No actively recruiting trials found for Hypotrichosis-lymphedema-telangiectasia-renal defect syndrome at this time.

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Search ClinicalTrials.gov ↗Join the Hypotrichosis-lymphedema-telangiectasia-renal defect syndrome community →

No specialists are currently listed for Hypotrichosis-lymphedema-telangiectasia-renal defect syndrome.

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 Hypotrichosis-lymphedema-telangiectasia-renal defect syndrome.

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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.How severely are my (or my child's) kidneys affected, and what monitoring schedule do you recommend?,What is the best way to manage the lymphedema, and should we see a specialist lymphedema therapist?,Are there any other family members who should be tested for this condition?,What signs of worsening should prompt an urgent visit or emergency care?,Are there any clinical trials or research studies we could participate in?,What dietary or lifestyle changes would help protect kidney function?,Which specialists should be part of our long-term care team?

Common questions about Hypotrichosis-lymphedema-telangiectasia-renal defect syndrome

What is Hypotrichosis-lymphedema-telangiectasia-renal defect syndrome?

Hypotrichosis-lymphedema-telangiectasia-renal defect syndrome (also called HLTRS or sometimes referred to by the gene involved, SOX18-related syndrome) is a very rare inherited condition that affects several parts of the body at the same time. The name describes its four main features: hypotrichosis (very sparse or thin hair), lymphedema (swelling caused by a buildup of fluid, usually in the legs or arms), telangiectasias (small, widened blood vessels visible near the skin surface, often appearing as red or purple spider-like marks), and renal defects (problems with how the kidneys work or are

How is Hypotrichosis-lymphedema-telangiectasia-renal defect syndrome inherited?

Hypotrichosis-lymphedema-telangiectasia-renal defect syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Hypotrichosis-lymphedema-telangiectasia-renal defect syndrome typically begin?

Typical onset of Hypotrichosis-lymphedema-telangiectasia-renal defect syndrome is neonatal. Age of onset can vary across affected individuals.