Neutropenia-monocytopenia-deafness syndrome

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Overview

Neutropenia-monocytopenia-deafness syndrome is a very rare inherited condition that affects the blood and hearing. The name describes its three main features: neutropenia (too few neutrophils, which are white blood cells that fight bacterial infections), monocytopenia (too few monocytes, another type of immune cell), and sensorineural hearing loss (damage to the inner ear or hearing nerve that causes permanent hearing difficulty). Together, these problems mean the body has a harder time fighting off infections and the person experiences significant hearing loss. People with this syndrome are at higher risk for repeated or serious bacterial infections because their immune system is weakened. The hearing loss is typically present from birth or early childhood and does not improve on its own. The exact cause at the genetic level is not fully understood in all cases, and the syndrome is sometimes referred to by its Orphanet classification or described as a combined immunodeficiency with deafness. Treatment focuses on managing infections, supporting the immune system, and addressing hearing loss with hearing aids or cochlear implants. There is currently no cure, but careful medical monitoring and early intervention can significantly improve quality of life.

Also known as:

Key symptoms:

Very low levels of infection-fighting white blood cells (neutropenia)Low levels of monocytes (another type of immune cell)Permanent hearing loss, usually in both earsFrequent or severe bacterial infectionsRecurrent ear infections or respiratory infectionsFatigue due to repeated illnessDelayed recovery from common infections

Clinical phenotype terms (4)— hover any for plain English
Abnormality of neutrophilsHP:0001874Abnormal macrophage morphologyHP:0004311
Inheritance

Autosomal dominant

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

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Neutropenia-monocytopenia-deafness syndrome.

View clinical trials →

No actively recruiting trials found for Neutropenia-monocytopenia-deafness syndrome at this time.

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No specialists are currently listed for Neutropenia-monocytopenia-deafness syndrome.

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 Neutropenia-monocytopenia-deafness syndrome.

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Community

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Caregiver Resources

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Questions for your doctor

Bring these to your next appointment

  • Q1.What is causing my (or my child's) low white blood cell counts, and has a specific gene been identified?,How often should blood counts be checked, and what levels should prompt an emergency visit?,Should we use preventive antibiotics, and if so, which ones and for how long?,Is a bone marrow transplant something we should consider, and what are the risks and benefits?,What type of hearing support is best — hearing aids or cochlear implants — and when should we start?,Are there any vaccines that are not safe given the immune deficiency?,Are there any clinical trials or research studies we could participate in?

Common questions about Neutropenia-monocytopenia-deafness syndrome

What is Neutropenia-monocytopenia-deafness syndrome?

Neutropenia-monocytopenia-deafness syndrome is a very rare inherited condition that affects the blood and hearing. The name describes its three main features: neutropenia (too few neutrophils, which are white blood cells that fight bacterial infections), monocytopenia (too few monocytes, another type of immune cell), and sensorineural hearing loss (damage to the inner ear or hearing nerve that causes permanent hearing difficulty). Together, these problems mean the body has a harder time fighting off infections and the person experiences significant hearing loss. People with this syndrome are

How is Neutropenia-monocytopenia-deafness syndrome inherited?

Neutropenia-monocytopenia-deafness syndrome follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Neutropenia-monocytopenia-deafness syndrome typically begin?

Typical onset of Neutropenia-monocytopenia-deafness syndrome is childhood. Age of onset can vary across affected individuals.