PIEZO1-related generalized lymphatic dysplasia with non-immune hydrops fetalis

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ORPHA:568062OMIM:616843Q82.0
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Overview

PIEZO1-related generalized lymphatic dysplasia with non-immune hydrops fetalis (sometimes called PIEZO1-GLD or Hennekam-like syndrome) is a rare genetic condition that affects the lymphatic system — the network of vessels and tissues that help move fluid throughout the body and support the immune system. When the PIEZO1 gene does not work properly, the lymphatic vessels fail to develop or function normally. This causes fluid to build up in tissues and body cavities, a condition known as lymphedema. In the most severe form, this fluid buildup begins before birth and is called non-immune hydrops fetalis, meaning the baby accumulates dangerous amounts of fluid in the womb without an immune-related cause. Babies affected by this condition may be born with swelling throughout the body, fluid around the heart and lungs, and swelling of the abdomen. Some pregnancies end in miscarriage or stillbirth due to the severity of the fluid buildup. Infants who survive may continue to experience chronic lymphedema — persistent swelling, especially in the limbs — along with repeated infections, breathing difficulties, and problems with the intestines absorbing nutrients properly. There is currently no cure for this condition. Treatment focuses on managing symptoms, reducing swelling through compression therapy and physical therapy, preventing infections, and supporting nutrition. A team of specialists is usually needed to care for affected individuals throughout their lives. Early diagnosis and careful ongoing management can improve quality of life and help reduce complications.

Also known as:

Key symptoms:

Severe whole-body swelling (generalized lymphedema) present from birth or before birthFluid buildup around the lungs (pleural effusion) making breathing difficultFluid buildup around the heart (pericardial effusion)Swollen abdomen due to fluid in the belly cavity (ascites)Persistent swelling of the arms and legs throughout lifeRepeated skin infections (cellulitis) in swollen areasDifficulty absorbing nutrients from food due to lymphatic problems in the intestinesLow protein levels in the blood from poor absorptionBreathing problems in newbornsThickened, hardened skin over swollen areas over timeIncreased risk of serious infections due to immune system involvementPossible intellectual or developmental delays in some children

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 PIEZO1-related generalized lymphatic dysplasia with non-immune hydrops fetalis.

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No actively recruiting trials found for PIEZO1-related generalized lymphatic dysplasia with non-immune hydrops fetalis at this time.

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No specialists are currently listed for PIEZO1-related generalized lymphatic dysplasia with non-immune hydrops fetalis.

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 PIEZO1-related generalized lymphatic dysplasia with non-immune hydrops fetalis.

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Community

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Latest news about PIEZO1-related generalized lymphatic dysplasia with non-immune hydrops fetalis

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Caregiver Resources

NORD 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 specific change was found in the PIEZO1 gene, and what does it mean for my child's health?,Should other family members be tested for this gene change?,What is the best lymphedema management plan for my child's age and severity?,Are there any clinical trials or research studies we should consider joining?,What signs of infection or complications should prompt an emergency room visit?,Does my child need any special diet, and should we see a dietitian?,What support services — such as physical therapy, school accommodations, or social work — are available to us?

Common questions about PIEZO1-related generalized lymphatic dysplasia with non-immune hydrops fetalis

What is PIEZO1-related generalized lymphatic dysplasia with non-immune hydrops fetalis?

PIEZO1-related generalized lymphatic dysplasia with non-immune hydrops fetalis (sometimes called PIEZO1-GLD or Hennekam-like syndrome) is a rare genetic condition that affects the lymphatic system — the network of vessels and tissues that help move fluid throughout the body and support the immune system. When the PIEZO1 gene does not work properly, the lymphatic vessels fail to develop or function normally. This causes fluid to build up in tissues and body cavities, a condition known as lymphedema. In the most severe form, this fluid buildup begins before birth and is called non-immune hydrops

How is PIEZO1-related generalized lymphatic dysplasia with non-immune hydrops fetalis inherited?

PIEZO1-related generalized lymphatic dysplasia with non-immune hydrops fetalis follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does PIEZO1-related generalized lymphatic dysplasia with non-immune hydrops fetalis typically begin?

Typical onset of PIEZO1-related generalized lymphatic dysplasia with non-immune hydrops fetalis is neonatal. Age of onset can vary across affected individuals.