Neonatal autoimmune hemolytic anemia

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:398109D59.1
Who is this for?
Show terms as
1FDA treatments9Specialists8Treatment centers1Financial resources

Where are you in your journey?

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

Overview

Neonatal autoimmune hemolytic anemia (NAIHA) is a rare blood condition that affects newborn babies, usually within the first few days or weeks of life. In this disease, the baby's immune system mistakenly produces antibodies that attack and destroy its own red blood cells. This process is called hemolysis. Red blood cells are essential for carrying oxygen throughout the body, so when they are destroyed too quickly, the baby can develop anemia (low red blood cell count), jaundice (yellowing of the skin and eyes due to a buildup of bilirubin), and other complications. This condition is different from hemolytic disease of the newborn caused by maternal antibodies (such as Rh incompatibility), because in NAIHA the baby's own immune system is producing the harmful antibodies. The exact cause is not fully understood, but it is considered an autoimmune process rather than a purely inherited genetic disorder. Symptoms can range from mild to severe. Babies may appear pale, yellow, or lethargic and may have difficulty feeding. In severe cases, dangerously high bilirubin levels can lead to brain damage (kernicterus) if not treated promptly. Treatment typically involves supportive care such as phototherapy for jaundice, blood transfusions for severe anemia, and in some cases immunosuppressive therapies like corticosteroids or intravenous immunoglobulin (IVIG). Most babies respond well to treatment, and the condition often resolves over weeks to months as the immune system matures.

Also known as:

Key symptoms:

Yellowing of the skin and eyes (jaundice)Pale skin or pallorFatigue or excessive sleepinessPoor feeding or difficulty feedingRapid heartbeatRapid breathingDark-colored urineEnlarged spleenEnlarged liverIrritability or fussinessSwelling of the body (in severe cases)Low red blood cell count (anemia)

Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

FDA & Trial Timeline

2 events
Jan 2023Evaluation of a Smartphone-based Screening Tool (Picterus JP) for Neonatal Jaundice (Chicago)

Picterus AS — NA

TrialRECRUITING
Feb 2022Enjaymo: FDA approved

To decrease the need for red blood cell (RBC) transfusion due to hemolysis in adults with cold agglutinin disease (CAD)

FDAcompleted

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

Treatments

1 available

Enjaymo

SUTIMLIMAB-JOME· Recordati Rare Diseases, Inc.Orphan Drug

To decrease the need for red blood cell (RBC) transfusion due to hemolysis in adults with cold agglutinin disease (CAD)

No actively recruiting trials found for Neonatal autoimmune hemolytic anemia at this time.

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

Search ClinicalTrials.gov ↗Join the Neonatal autoimmune hemolytic anemia community →

Specialists

9 foundView all specialists →
PM
Paul Szabolcs, MD
Pittsburgh, Pennsylvania
Specialist

Rare Disease Specialist

PI on 7 active trials
DM
David T. Teachey, MD
PHILADELPHIA, PA
Specialist
PI on 1 active trial
YM
Yi-Bin A Chen, M.D.
BOSTON, MA
Specialist
PI on 1 active trial
AM
Adela Cardones, MD
DURHAM, NC
Specialist
PI on 2 active trials
JM
Jeanmarie Schied, MD
Chicago, Illinois
Specialist

Rare Disease Specialist

Treatment Centers

8 centers
⚗️ Trial Site

University of Chicago

📍 Chicago, Illinois

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🔬 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

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

Financial Resources

1 resources
Enjaymo(SUTIMLIMAB-JOME)Recordati Rare Diseases, Inc.

Travel Grants

No travel grants are currently matched to Neonatal autoimmune hemolytic anemia.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Neonatal autoimmune hemolytic anemiaForum →

No community posts yet. Be the first to share your experience with Neonatal autoimmune hemolytic anemia.

Start the conversation →

Latest news about Neonatal autoimmune hemolytic anemia

Disease timeline:

New trial: Evaluation of a Smartphone-based Screening Tool (Picterus JP) for Neonatal Jaundice (Chicago)

Phase NA trial recruiting. Picterus Jaundice Pro (JP)

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.How severe is my baby's anemia, and what is the current treatment plan?,Will my baby need blood transfusions, and if so, how many might be expected?,What are the risks of the treatments being used, such as IVIG or steroids?,How long is this condition expected to last before it resolves?,What warning signs should I watch for at home that would require emergency care?,Will this condition affect my baby's long-term development or health?,Is there any chance this could happen again in future pregnancies?

Common questions about Neonatal autoimmune hemolytic anemia

What is Neonatal autoimmune hemolytic anemia?

Neonatal autoimmune hemolytic anemia (NAIHA) is a rare blood condition that affects newborn babies, usually within the first few days or weeks of life. In this disease, the baby's immune system mistakenly produces antibodies that attack and destroy its own red blood cells. This process is called hemolysis. Red blood cells are essential for carrying oxygen throughout the body, so when they are destroyed too quickly, the baby can develop anemia (low red blood cell count), jaundice (yellowing of the skin and eyes due to a buildup of bilirubin), and other complications. This condition is differen

How is Neonatal autoimmune hemolytic anemia inherited?

Neonatal autoimmune hemolytic anemia follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Neonatal autoimmune hemolytic anemia typically begin?

Typical onset of Neonatal autoimmune hemolytic anemia is neonatal. Age of onset can vary across affected individuals.

Which specialists treat Neonatal autoimmune hemolytic anemia?

9 specialists and care centers treating Neonatal autoimmune hemolytic anemia are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.