Acquired neutropenia

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

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

2 events
Jun 2024A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

X4 Pharmaceuticals — PHASE3

TrialRECRUITING
Jan 2010A Phase I Study of Mozobil in the Treatment of Patients With WHIMS

National Institute of Allergy and Infectious Diseases (NIAID) — PHASE1, PHASE2

TrialRECRUITING

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

Treatments

1 available

Azithromycin

AZITHROMYCIN· Preferred Pharmaceuticals Inc.

Community-acquired pneumonia in adults and pediatric patients (6 months of age and older)

Clinical Trials

2 recruitingView all trials with filters →
Phase 31 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
Phase 3
Actively Recruiting
PI: Vice President Global Head of Clinical Development and Safety (X4 Pharmaceuticals) · Sites: Phoenix, Arizona; Irvine, California +107 more · Age: 1299 yrs

Specialists

10 foundView all specialists →
JC
Joanna Cichy
Specialist
1 Acquired neutropenia publication
CP
Christina Maria Perraki
Specialist
1 Acquired neutropenia publication
BK
Brice Korkmaz
Specialist
1 Acquired neutropenia publication
HP
Helen A Papadaki
Specialist
1 Acquired neutropenia publication
AM
Angelika Mazur
Specialist
1 Acquired neutropenia publication
JS
Joanna Skrzeczynska-Moncznik
Specialist
1 Acquired neutropenia publication
IM
Irene Mavroudi
Specialist
1 Acquired neutropenia publication
GD
Gere S diZerega
LOS ANGELES, CA
Specialist
PI on 1 active trial
AT
Andrew Talal
BUFFALO, NY
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

Financial Resources

1 resources
Azithromycin(AZITHROMYCIN)Preferred Pharmaceuticals Inc.

Travel Grants

No travel grants are currently matched to Acquired neutropenia.

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Community

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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

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.

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.