BAP1-related tumor predisposition syndrome

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ORPHA:289539OMIM:614327D23.9
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1Active trials5Specialists8Treatment centers

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Overview

BAP1-related tumor predisposition syndrome (also called BAP1 tumor predisposition syndrome or BAP1-TPDS) is a rare inherited condition that increases a person's risk of developing certain types of cancer throughout their lifetime. It is caused by changes (mutations) in the BAP1 gene, which normally acts as a tumor suppressor — meaning it helps prevent cells from growing out of control. When one copy of this gene is not working properly, the body's ability to keep certain cancers in check is reduced. People with this syndrome have a higher-than-normal risk of developing several specific cancers. The most commonly associated cancers include uveal melanoma (a cancer of the eye), malignant mesothelioma (a cancer of the lining of the lungs or abdomen, often linked to asbestos exposure in the general population but occurring even without asbestos exposure in BAP1 carriers), kidney cancer (specifically clear cell renal cell carcinoma), and a distinctive type of skin lesion called a BAP1-inactivated melanocytic tumor (sometimes called atypical Spitz tumor). Some families also show increased rates of cutaneous melanoma (skin melanoma) and other cancers. There is currently no cure for the syndrome itself, but early detection and regular cancer screening are the cornerstones of management. When cancers are caught early through surveillance programs, treatment outcomes are generally much better. Treatment depends on which cancer develops and may include surgery, radiation therapy, immunotherapy, or targeted therapies. Genetic counseling is strongly recommended for affected individuals and their family members.

Also known as:

Key symptoms:

Melanoma of the eye (uveal melanoma)Mesothelioma (cancer of the lining of the lungs or abdomen)Kidney cancer (clear cell renal cell carcinoma)Unusual skin moles or growths (BAP1-inactivated melanocytic tumors)Skin melanomaMultiple cancers occurring in one person over their lifetimeFamily history of multiple cancers across generationsCholangiocarcinoma (bile duct cancer) in some familiesBasal cell carcinoma in some individualsThyroid cancer in some families

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 ↗

FDA & Trial Timeline

1 event
Mar 2021Prospective Evaluation of High Resolution Dual Energy Computed Tomographic Imaging, Noninvasive (Liquid) Biopsies, and Minimally Invasive Surgical Surveillance for Early Detection of Mesotheliomas in Patients With BAP1 Tumor Predisposition Syndrome

National Cancer Institute (NCI)

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for BAP1-related tumor predisposition syndrome.

1 clinical trialare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

1 recruitingView all trials with filters →
Other1 trial
Prospective Evaluation of High Resolution Dual Energy Computed Tomographic Imaging, Noninvasive (Liquid) Biopsies, and Minimally Invasive Surgical Surveillance for Early Detection of Mesotheliomas in Patients With BAP1 Tumor Predisposition Syndrome
Actively Recruiting
PI: David S Schrump, M.D. (National Cancer Institute (NCI)) · Sites: Bethesda, Maryland · Age: 3099 yrs

Specialists

5 foundView all specialists →
BM
Brigitte C Widemann, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 18 active trials
KM
Kim E. Nichols, MD
Memphis, Tennessee
Specialist

Rare Disease Specialist

PI on 2 active trials
DM
David S Schrump, M.D.
BETHESDA, MD
Specialist
PI on 19 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 BAP1-related tumor predisposition syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about BAP1-related tumor predisposition syndrome

Disease timeline:

New recruiting trial: Prospective Evaluation of High Resolution Dual Energy Computed Tomographic Imaging, Noninvasive (Liquid) Biopsies, and Minimally Invasive Surgical Surveillance for Early Detection of Mesotheliomas in Patients With BAP1 Tumor Predisposition Syndrome

A new clinical trial is recruiting patients for BAP1-related tumor predisposition syndrome

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 specific cancers am I at highest risk for based on my BAP1 mutation and family history?,What screening schedule do you recommend for me, and how often should I have each type of exam?,Should my children or other family members be tested for this mutation, and at what age?,Are there any clinical trials or new treatments being studied for people with BAP1 mutations?,What symptoms should prompt me to seek immediate medical attention between screening visits?,Can you refer me to a genetic counselor and to specialists experienced with this syndrome?,Are there any lifestyle changes I can make to reduce my cancer risk?

Common questions about BAP1-related tumor predisposition syndrome

What is BAP1-related tumor predisposition syndrome?

BAP1-related tumor predisposition syndrome (also called BAP1 tumor predisposition syndrome or BAP1-TPDS) is a rare inherited condition that increases a person's risk of developing certain types of cancer throughout their lifetime. It is caused by changes (mutations) in the BAP1 gene, which normally acts as a tumor suppressor — meaning it helps prevent cells from growing out of control. When one copy of this gene is not working properly, the body's ability to keep certain cancers in check is reduced. People with this syndrome have a higher-than-normal risk of developing several specific cancer

How is BAP1-related tumor predisposition syndrome inherited?

BAP1-related tumor predisposition syndrome follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does BAP1-related tumor predisposition syndrome typically begin?

Typical onset of BAP1-related tumor predisposition syndrome is adult. Age of onset can vary across affected individuals.

Are there clinical trials for BAP1-related tumor predisposition syndrome?

Yes — 1 recruiting clinical trial is currently listed for BAP1-related tumor predisposition syndrome on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat BAP1-related tumor predisposition syndrome?

5 specialists and care centers treating BAP1-related tumor predisposition syndrome are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.