Cutaneous mastocytosis

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ORPHA:66646
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1FDA treatments1Active trials44Specialists8Treatment centers

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Overview

Cutaneous mastocytosis (also called skin mastocytosis) is a condition where too many mast cells — a type of immune cell — build up in the skin. Mast cells normally help protect the body from infections and allergies, but when there are too many of them, they can release chemicals that cause a wide range of symptoms. The most common form in children is called urticaria pigmentosa, which causes brownish or reddish spots on the skin that itch and swell when rubbed or scratched. Other forms include a single skin lesion called a mastocytoma, and a more widespread form called diffuse cutaneous mastocytosis. The condition mainly affects the skin, causing itching, hives, blistering, and flushing. In some cases, the chemicals released by mast cells can affect other parts of the body, causing stomach pain, diarrhea, low blood pressure, or even a severe allergic reaction called anaphylaxis. Triggers like heat, friction, certain foods, insect stings, or medications can make symptoms flare up. Cutaneous mastocytosis is most often diagnosed in young children and tends to improve or even go away on its own by puberty in many cases. Adults can also develop the condition, though it is less likely to resolve on its own in adults. Treatment focuses on managing symptoms and avoiding triggers. Antihistamines are the main treatment, and other medications are available for more severe symptoms. The outlook for most children with this condition is very good.

Key symptoms:

Brownish or reddish spots or patches on the skinItching, especially after rubbing or scratching the skinSkin that swells or forms a hive when rubbed (called Darier's sign)Blistering or fluid-filled bumps on the skin, especially in young childrenFlushing or redness of the skinHives (urticaria)Stomach pain or crampingDiarrhea or nauseaHeadachesLow blood pressure or dizzinessRapid heartbeatSevere allergic reaction (anaphylaxis) in some casesBone pain (less common)

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

2 events
Jan 2024Mastocytosis From Pediatric Age to Adulthood: Local Registry of Cutaneous and Systemic Mastocytosis

Fondazione IRCCS Policlinico San Matteo di Pavia

TrialRECRUITING
Dec 1989

Gastrocrom: FDA approved

Cutaneous mastocytosis

FDAcompleted

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

Treatments

1 available

Gastrocrom

Cromolyn sodium· Fisons CorporationOrphan Drug

Clinical Trials

1 recruitingView all trials with filters →
Other1 trial
Mastocytosis From Pediatric Age to Adulthood: Local Registry of Cutaneous and Systemic Mastocytosis
Actively Recruiting
· Sites: Pavia, Pavia · Age: 699 yrs

Specialists

Showing 25 of 44View all specialists →
OP
Olivier Lortholary, MD, PhD
Specialist
PI on 5 active trials
MF
Maryam Fouladi
COLUMBUS, OH
Specialist
PI on 7 active trials2 Cutaneous mastocytosis publications
RN
Ryotaro Nakamura
Duarte, California
Specialist

Rare Disease Specialist

PI on 6 active trials
NM
Neal Flomenberg, MD
PHILADELPHIA, PA
Specialist
PI on 5 active trials
DC
Dolores Grosso, DNP, CRNP
Specialist
PI on 4 active trials
RP
Rachael Easton, MD, Ph.D.
TROY, MI
Specialist
PI on 1 active trial
MP
Marcus Maurer, Prof
PUEBLO, CO
Specialist
PI on 1 active trial
JM
Jeffrey J. Raizer, MD
CHICAGO, IL
Specialist
PI on 7 active trials
SM
Sheila Galbraith, MD
EAU CLAIRE, WI
Specialist
PI on 1 active trial
CM
Cristina Livideanu, MD
Specialist
PI on 1 active trial
MM
Melody C Carter, M.D.
Specialist
PI on 3 active trials
AP
Anthony L Boral, MD, PhD
CAMBRIDGE, MA
Specialist
PI on 1 active trial
CM
Cristina Bulai Livideanu, MD
Specialist
PI on 3 active trials
AW
Ann Woolfrey
SEATTLE, WA
Specialist
PI on 5 active trials
TM
Tom R. Fitch, M.D.
GLENDALE, AZ
Specialist
PI on 1 active trial
MM
Mary Ann Knovich, MD
CHARLOTTE, NC
Specialist
PI on 1 active trial
AM
Adam J. Esbenshade, MD
NASHVILLE, TN
Specialist
PI on 2 active trials
EM
Elizabeth C. Dees, MD
CHAPEL HILL, NC
Specialist
PI on 1 active trial
RP
Robert Z. Orlowski, MD, PhD
HOUSTON, TX
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 Cutaneous mastocytosis.

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Caregiver Resources

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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 cutaneous mastocytosis does my child or I have, and what does that mean for the future?,Should I or my child carry an epinephrine auto-injector, and when should it be used?,What are my specific triggers, and how can I best avoid them?,Is genetic testing recommended, and what would the results tell us?,How often should we have follow-up appointments, and what signs should prompt an earlier visit?,Is there a risk that this condition will spread to other organs, and how would we know?,Are there any clinical trials or new treatments I should know about?

Common questions about Cutaneous mastocytosis

What is Cutaneous mastocytosis?

Cutaneous mastocytosis (also called skin mastocytosis) is a condition where too many mast cells — a type of immune cell — build up in the skin. Mast cells normally help protect the body from infections and allergies, but when there are too many of them, they can release chemicals that cause a wide range of symptoms. The most common form in children is called urticaria pigmentosa, which causes brownish or reddish spots on the skin that itch and swell when rubbed or scratched. Other forms include a single skin lesion called a mastocytoma, and a more widespread form called diffuse cutaneous masto

Are there clinical trials for Cutaneous mastocytosis?

Yes — 1 recruiting clinical trial is currently listed for Cutaneous mastocytosis on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Cutaneous mastocytosis?

25 specialists and care centers treating Cutaneous mastocytosis are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Cutaneous mastocytosis?

1 patient support program are currently tracked on UniteRare for Cutaneous mastocytosis. See the treatments and support programs sections for copay assistance, eligibility, and contact details.