Hereditary leiomyomatosis and renal cell cancer

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ORPHA:523OMIM:150800C64
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6Specialists8Treatment centers

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Overview

Hereditary leiomyomatosis and renal cell cancer (HLRCC) is a rare inherited condition that causes benign smooth muscle tumors called leiomyomas to grow in the skin and, in women, the uterus. It also significantly raises the risk of a specific and aggressive type of kidney cancer. HLRCC is sometimes called Reed syndrome, especially when referring to the skin and uterine features. The condition is caused by a change (mutation) in a gene called FH, which normally helps cells produce energy. When this gene does not work properly, cells can grow in abnormal ways. People with HLRCC often first notice firm, sometimes painful bumps on their skin, usually on the arms, legs, chest, or back. Women frequently develop uterine fibroids (leiomyomas) at a younger age than usual, and these can be larger and more numerous than typical fibroids, often causing heavy periods and pelvic pain. The kidney cancer associated with HLRCC — called type 2 papillary renal cell carcinoma — tends to be aggressive and can spread early, so regular kidney surveillance is very important. Treatment focuses on managing symptoms and monitoring for cancer. Skin lesions can be treated with medications or minor procedures. Uterine fibroids may require surgery. Kidney tumors, if found, are usually treated surgically. There is no cure for the underlying genetic condition, but careful surveillance can catch kidney cancer early when it is most treatable. Genetic counseling is strongly recommended for patients and their families.

Also known as:

Key symptoms:

Firm, sometimes painful skin bumps (cutaneous leiomyomas), often on the arms, legs, chest, or backSkin bumps that may hurt more in cold temperatures or when touchedUterine fibroids (benign muscle tumors in the womb) appearing at a younger age than usualHeavy or prolonged menstrual periodsPelvic pain or pressureNeeding surgery for uterine fibroids at a young ageA lump or mass in the kidney found on imagingBlood in the urineBack or side pain near the kidneysUnexplained weight loss (if kidney cancer is present)Fatigue

Clinical phenotype terms (11)— hover any for plain English
Uterine leiomyomaHP:0000131Uterine leiomyosarcomaHP:0002891Papillary renal cell carcinoma type 2HP:0006732Multiple cutaneous leiomyomasHP:0007437Cutaneous leiomyomaHP:0007620Barrett esophagusHP:0100580Vaginal neoplasmHP:0100650Esophageal neoplasmHP:0100751
Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Hereditary leiomyomatosis and renal cell cancer.

View clinical trials →

No actively recruiting trials found for Hereditary leiomyomatosis and renal cell cancer at this time.

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

Search ClinicalTrials.gov ↗Join the Hereditary leiomyomatosis and renal cell cancer community →

Specialists

6 foundView all specialists →
JM
Joshua Mann, MPH
Specialist
PI on 1 active trial
RS
Ramaprasad Srinivasan
BETHESDA, MD
Specialist
PI on 1 active trial28 Hereditary leiomyomatosis and renal cell cancer publications
WM
W. Marston Linehan, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 6 active trials
EM
Edward W Cowen, M.D.
SILVER SPRING, MD
Specialist
PI on 2 active trials

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 Hereditary leiomyomatosis and renal cell cancer.

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Community

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Latest news about Hereditary leiomyomatosis and renal cell cancer

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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.How often should I have kidney MRI scans, and what exactly are you looking for?,Should my children, siblings, and parents be tested for the FH gene mutation?,What are my options for managing the pain from my skin leiomyomas?,If I develop a kidney tumor, what are my treatment options and how quickly does it need to be treated?,Are there any clinical trials I should know about for HLRCC-related kidney cancer?,What should I do if I notice new or changing skin bumps or symptoms?,Should I see a gynecologist who specializes in HLRCC, and what monitoring do I need for uterine fibroids?

Common questions about Hereditary leiomyomatosis and renal cell cancer

What is Hereditary leiomyomatosis and renal cell cancer?

Hereditary leiomyomatosis and renal cell cancer (HLRCC) is a rare inherited condition that causes benign smooth muscle tumors called leiomyomas to grow in the skin and, in women, the uterus. It also significantly raises the risk of a specific and aggressive type of kidney cancer. HLRCC is sometimes called Reed syndrome, especially when referring to the skin and uterine features. The condition is caused by a change (mutation) in a gene called FH, which normally helps cells produce energy. When this gene does not work properly, cells can grow in abnormal ways. People with HLRCC often first noti

How is Hereditary leiomyomatosis and renal cell cancer inherited?

Hereditary leiomyomatosis and renal cell cancer follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Hereditary leiomyomatosis and renal cell cancer typically begin?

Typical onset of Hereditary leiomyomatosis and renal cell cancer is adult. Age of onset can vary across affected individuals.

Which specialists treat Hereditary leiomyomatosis and renal cell cancer?

6 specialists and care centers treating Hereditary leiomyomatosis and renal cell cancer are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.