Overview
Acquired neutropenia is a condition characterized by an abnormally low number of neutrophils (a type of white blood cell essential for fighting infections) in the blood, where the neutrophil count falls below 1,500 cells per microliter. Unlike congenital or inherited forms of neutropenia, acquired neutropenia develops after birth due to external or secondary causes. It can affect individuals at any age and may be acute or chronic in nature. The condition primarily impacts the immune system, leaving affected individuals vulnerable to bacterial and fungal infections, particularly of the skin, mucous membranes, gums, and respiratory tract. Acquired neutropenia can arise from a wide range of causes, including medications (such as chemotherapy agents, certain antibiotics, antithyroid drugs, and anticonvulsants), autoimmune disorders (where antibodies target and destroy neutrophils), viral infections (including HIV, hepatitis, and Epstein-Barr virus), nutritional deficiencies (particularly vitamin B12, folate, or copper deficiency), bone marrow infiltration by malignancies, or overwhelming sepsis. Autoimmune neutropenia is a notable subtype, particularly common in infants and young children, where autoantibodies directed against neutrophil surface antigens lead to accelerated neutrophil destruction. Key symptoms include recurrent infections, fever, mouth ulcers (oral stomatitis), gingivitis, skin abscesses, and in severe cases, life-threatening sepsis. The severity of clinical manifestations generally correlates with the degree of neutropenia: mild neutropenia (1,000–1,500 cells/μL) may be asymptomatic, while severe neutropenia (below 500 cells/μL) carries a high risk of serious infections. Treatment depends on the underlying cause and severity. Management strategies include identifying and removing offending agents, treating underlying conditions, administering granulocyte colony-stimulating factor (G-CSF) to stimulate neutrophil production, and using prophylactic antibiotics in severe cases. In many instances, particularly drug-induced or autoimmune neutropenia in children, the condition is reversible once the causative factor is addressed.
Also known as:
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
2 eventsX4 Pharmaceuticals — PHASE3
National Institute of Allergy and Infectious Diseases (NIAID) — PHASE1, PHASE2
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
1 availableAzithromycin
Community-acquired pneumonia in adults and pediatric patients (6 months of age and older)
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Financial Resources
1 resourcesTravel Grants
No travel grants are currently matched to Acquired neutropenia.
Community
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Start the conversation →Latest news about Acquired neutropenia
Disease timeline:
New recruiting trial: A Phase I Study of Mozobil in the Treatment of Patients With WHIMS
A new clinical trial is recruiting patients for Acquired neutropenia
New recruiting trial: A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
A new clinical trial is recruiting patients for Acquired neutropenia
Caregiver Resources
NORD Caregiver Resources
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Mental Health Support
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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.
Common questions about Acquired neutropenia
What is Acquired neutropenia?
Acquired neutropenia is a condition characterized by an abnormally low number of neutrophils (a type of white blood cell essential for fighting infections) in the blood, where the neutrophil count falls below 1,500 cells per microliter. Unlike congenital or inherited forms of neutropenia, acquired neutropenia develops after birth due to external or secondary causes. It can affect individuals at any age and may be acute or chronic in nature. The condition primarily impacts the immune system, leaving affected individuals vulnerable to bacterial and fungal infections, particularly of the skin, mu
Are there clinical trials for Acquired neutropenia?
Yes — 2 recruiting clinical trials are currently listed for Acquired neutropenia on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Acquired neutropenia?
10 specialists and care centers treating Acquired neutropenia are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Acquired neutropenia?
1 patient support program are currently tracked on UniteRare for Acquired neutropenia. See the treatments and support programs sections for copay assistance, eligibility, and contact details.