Autosomal recessive severe congenital neutropenia due to CXCR2 deficiency

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:420699OMIM:619407D70
Who is this for?
Show terms as
8Treatment centers

Where are you in your journey?

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

Overview

Autosomal recessive severe congenital neutropenia due to CXCR2 deficiency is a very rare inherited blood disorder. It is sometimes called CXCR2-deficient severe congenital neutropenia or SCN due to CXCR2 deficiency. In this condition, the body cannot make enough working neutrophils — a type of white blood cell that fights bacterial and fungal infections. Neutrophils are your immune system's first responders. Without enough of them, even minor infections can become life-threatening. The root cause is a change (mutation) in both copies of the CXCR2 gene. This gene gives instructions for making a protein that helps neutrophils move from the bone marrow into the bloodstream where they are needed. When this protein does not work properly, neutrophils get 'stuck' and cannot do their job. As a result, the body is left very vulnerable to serious infections from birth. Symptoms usually appear in the first weeks or months of life and include repeated serious bacterial infections, mouth sores, skin infections, and delayed healing of wounds. Treatment focuses on preventing and treating infections, and may include a medication called G-CSF (granulocyte colony-stimulating factor) to boost neutrophil production. In some cases, a bone marrow transplant may be considered as a more permanent solution. Early diagnosis and careful medical management are essential to improving outcomes for affected children.

Key symptoms:

Very low levels of neutrophils (infection-fighting white blood cells) in the bloodRepeated serious bacterial infections starting in early infancySkin infections and abscessesMouth sores and gum infectionsLung infections (pneumonia)Belly infectionsSlow or poor wound healingFever that keeps coming backSwollen lymph nodesFailure to gain weight or grow normally

Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Autosomal recessive severe congenital neutropenia due to CXCR2 deficiency.

View clinical trials →

No actively recruiting trials found for Autosomal recessive severe congenital neutropenia due to CXCR2 deficiency at this time.

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

Search ClinicalTrials.gov ↗Join the Autosomal recessive severe congenital neutropenia due to CXCR2 deficiency community →

No specialists are currently listed for Autosomal recessive severe congenital neutropenia due to CXCR2 deficiency.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

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 Autosomal recessive severe congenital neutropenia due to CXCR2 deficiency.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Autosomal recessive severe congenital neutropenia due to CXCR2 deficiencyForum →

No community posts yet. Be the first to share your experience with Autosomal recessive severe congenital neutropenia due to CXCR2 deficiency.

Start the conversation →

Latest news about Autosomal recessive severe congenital neutropenia due to CXCR2 deficiency

No recent news articles for Autosomal recessive severe congenital neutropenia due to CXCR2 deficiency.

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 neutrophil count level is safe for my child, and when should I be worried?,How often does my child need blood tests, and what are we watching for?,Is my child a candidate for bone marrow transplantation, and what would that involve?,What infections should I be most worried about, and how do I recognize them early?,Are there any vaccines my child should or should not receive?,What is the long-term risk of leukemia with G-CSF treatment, and how is that monitored?,Should other family members be tested for the CXCR2 gene mutation?

Common questions about Autosomal recessive severe congenital neutropenia due to CXCR2 deficiency

What is Autosomal recessive severe congenital neutropenia due to CXCR2 deficiency?

Autosomal recessive severe congenital neutropenia due to CXCR2 deficiency is a very rare inherited blood disorder. It is sometimes called CXCR2-deficient severe congenital neutropenia or SCN due to CXCR2 deficiency. In this condition, the body cannot make enough working neutrophils — a type of white blood cell that fights bacterial and fungal infections. Neutrophils are your immune system's first responders. Without enough of them, even minor infections can become life-threatening. The root cause is a change (mutation) in both copies of the CXCR2 gene. This gene gives instructions for making

How is Autosomal recessive severe congenital neutropenia due to CXCR2 deficiency inherited?

Autosomal recessive severe congenital neutropenia due to CXCR2 deficiency follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Autosomal recessive severe congenital neutropenia due to CXCR2 deficiency typically begin?

Typical onset of Autosomal recessive severe congenital neutropenia due to CXCR2 deficiency is neonatal. Age of onset can vary across affected individuals.