Paraneoplastic pemphigus

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:63455L10.8
Who is this for?
Show terms as
1Specialists8Treatment centers

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
Report missing data

Overview

Paraneoplastic pemphigus (PNP), also sometimes called paraneoplastic autoimmune multiorgan syndrome (PAMS), is a rare and serious blistering disease that happens when the immune system attacks the skin and mucous membranes in response to an underlying tumor or cancer. The word 'paraneoplastic' means the condition is triggered by a cancer elsewhere in the body, not by a direct infection or injury to the skin itself. The immune system makes abnormal proteins called autoantibodies that mistakenly attack the tissues that hold skin cells together, causing painful blisters, sores, and peeling. The disease most commonly affects the mouth, lips, throat, and eyes, making it very hard to eat, drink, or swallow. Blisters can also appear on the skin of the body. In many cases, the lung lining becomes inflamed, which can cause serious breathing problems. The condition is strongly linked to blood cancers such as non-Hodgkin lymphoma, chronic lymphocytic leukemia, and Castleman disease, though other tumors can also trigger it. Treatment focuses on two main goals: treating the underlying cancer and calming the overactive immune system. Doctors use corticosteroids, immunosuppressive drugs, and sometimes rituximab to reduce the immune attack. Even with treatment, paraneoplastic pemphigus is a very serious condition with a high risk of life-threatening complications, especially lung disease. Early diagnosis and prompt treatment of the associated tumor offer the best chance of improvement.

Key symptoms:

Painful sores and blisters in the mouth and on the lipsBlisters or raw areas on the skin of the bodyDifficulty swallowing due to sores in the throatRed, inflamed, or crusted eyelids and eye irritationShortness of breath or difficulty breathingPeeling or sloughing of the skinSores inside the nose or on the genitalsFatigue and general feeling of being unwellWeight loss linked to difficulty eatingHoarse voice or throat pain

Clinical phenotype terms (7)— hover any for plain English
B-cell lymphomaHP:0012191SarcomaHP:0100242ThymomaHP:0100522
Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Paraneoplastic pemphigus.

View clinical trials →

No actively recruiting trials found for Paraneoplastic pemphigus at this time.

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

Search ClinicalTrials.gov ↗Join the Paraneoplastic pemphigus community →

Specialists

1 foundView all specialists →

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 Paraneoplastic pemphigus.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Paraneoplastic pemphigusForum →

No community posts yet. Be the first to share your experience with Paraneoplastic pemphigus.

Start the conversation →

Latest news about Paraneoplastic pemphigus

No recent news articles for Paraneoplastic pemphigus.

Follow this condition to be notified when news becomes available.

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 of tumor or cancer is causing my paraneoplastic pemphigus, and what are my treatment options for it?,Which autoantibodies were found in my blood tests, and what do they tell us about my prognosis?,What is my risk of developing lung disease (bronchiolitis obliterans), and how will you monitor for it?,Which immunosuppressive medications do you recommend for me, and what are the main side effects I should watch for?,If the cancer is treated successfully, is there a chance my skin and mouth symptoms will improve?,Are there any clinical trials for paraneoplastic pemphigus that I might be eligible for?,What warning signs should prompt me to go to the emergency room immediately?

Common questions about Paraneoplastic pemphigus

What is Paraneoplastic pemphigus?

Paraneoplastic pemphigus (PNP), also sometimes called paraneoplastic autoimmune multiorgan syndrome (PAMS), is a rare and serious blistering disease that happens when the immune system attacks the skin and mucous membranes in response to an underlying tumor or cancer. The word 'paraneoplastic' means the condition is triggered by a cancer elsewhere in the body, not by a direct infection or injury to the skin itself. The immune system makes abnormal proteins called autoantibodies that mistakenly attack the tissues that hold skin cells together, causing painful blisters, sores, and peeling. The

How is Paraneoplastic pemphigus inherited?

Paraneoplastic pemphigus follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Paraneoplastic pemphigus typically begin?

Typical onset of Paraneoplastic pemphigus is adult. Age of onset can vary across affected individuals.

Which specialists treat Paraneoplastic pemphigus?

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