Hyaline fibromatosis syndrome

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Overview

Hyaline fibromatosis syndrome (HFS) is a rare inherited condition caused by a buildup of a waxy, protein-like material called hyaline in the skin, joints, and other tissues throughout the body. It was previously known by two separate names — infantile systemic hyalinosis (ISH) and juvenile hyaline fibromatosis (JHF) — which are now understood to be different points on the same disease spectrum. ISH is generally the more severe form, appearing at birth or in early infancy, while JHF tends to be milder and may appear slightly later in childhood. The condition causes firm, pearly skin growths (called papules or nodules), painful and stiff joints that become difficult to move, and thickening of the gums. In more severe cases, the internal organs can also be affected, leading to serious problems with the gut, immune system, and overall growth. Children with HFS often experience significant pain, especially when being moved or handled. There is currently no cure for hyaline fibromatosis syndrome. Treatment focuses on managing symptoms, reducing pain, improving joint movement through physical therapy, and addressing complications as they arise. A team of specialists is usually needed to provide the best care.

Key symptoms:

Firm, pearly or flesh-colored skin bumps or nodules, especially on the face, scalp, ears, and neckThickened, overgrown gums (gingival hyperplasia)Stiff, painful joints that are hard to bend or straightenContractures — joints that become permanently fixed in a bent positionSoft tissue swellings around the jointsPersistent or recurrent diarrhea and poor absorption of nutrientsProtein-losing enteropathy (the gut loses important proteins, causing swelling and poor growth)Frequent infections due to a weakened immune systemSlow growth and failure to thrive in infantsSevere pain when being picked up or movedSkin thickening, especially over the scalp and backEnlarged lymph nodesBone changes visible on X-ray

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 ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Hyaline fibromatosis syndrome.

View clinical trials →

No actively recruiting trials found for Hyaline fibromatosis syndrome at this time.

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Specialists

1 foundView all specialists →
JM
James Cantrell, MD
Specialist
PI on 1 active trial

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 Hyaline fibromatosis syndrome.

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Community

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Latest news about Hyaline fibromatosis syndrome

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Caregiver Resources

NORD Caregiver Resources

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

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

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 type and severity of hyaline fibromatosis syndrome does my child have, and what does that mean for their future?,Which specialists should be part of my child's care team, and how often should we see each one?,What are the best ways to manage my child's pain during daily care activities?,Are there any clinical trials or experimental treatments we should know about?,What signs should prompt me to seek emergency care?,Should other family members be tested, and what are the chances of this happening in a future pregnancy?,What support services — such as physical therapy, nutritional support, or social services — are available to us?

Common questions about Hyaline fibromatosis syndrome

What is Hyaline fibromatosis syndrome?

Hyaline fibromatosis syndrome (HFS) is a rare inherited condition caused by a buildup of a waxy, protein-like material called hyaline in the skin, joints, and other tissues throughout the body. It was previously known by two separate names — infantile systemic hyalinosis (ISH) and juvenile hyaline fibromatosis (JHF) — which are now understood to be different points on the same disease spectrum. ISH is generally the more severe form, appearing at birth or in early infancy, while JHF tends to be milder and may appear slightly later in childhood. The condition causes firm, pearly skin growths (c

How is Hyaline fibromatosis syndrome inherited?

Hyaline fibromatosis syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Hyaline fibromatosis syndrome typically begin?

Typical onset of Hyaline fibromatosis syndrome is infantile. Age of onset can vary across affected individuals.

Which specialists treat Hyaline fibromatosis syndrome?

1 specialists and care centers treating Hyaline fibromatosis syndrome are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.