Overview
Idiopathic aplastic anemia (IAA) is a rare and serious blood disorder where the bone marrow — the spongy tissue inside your bones — stops making enough blood cells. 'Idiopathic' means doctors cannot find a clear cause. The bone marrow contains stem cells that normally produce red blood cells, white blood cells, and platelets. In aplastic anemia, the immune system mistakenly attacks these stem cells, leaving the bone marrow empty or nearly empty. This is sometimes called 'bone marrow failure.' Because the body cannot make enough blood cells, people with this condition experience a range of serious symptoms. Low red blood cells cause fatigue and paleness. Low white blood cells make it hard to fight infections. Low platelets lead to easy bruising and bleeding that is difficult to stop. These problems can range from mild to life-threatening depending on how severe the bone marrow failure is. Treatment has improved greatly over the past few decades. Younger patients who have a matched donor may be cured with a bone marrow transplant (also called a stem cell transplant). For those who cannot have a transplant, immunosuppressive therapy — medicines that calm the immune system — is the main treatment. Newer drugs like eltrombopag have also improved outcomes. With proper treatment, many patients can achieve remission and live full lives.
Also known as:
Key symptoms:
Extreme tiredness and lack of energyShortness of breath, even with light activityPale or yellowish skinFrequent or severe infectionsUnexplained bruisingBleeding that takes a long time to stopSmall red or purple spots on the skin (petechiae)Bleeding gums or nosebleedsRapid or irregular heartbeatDizziness or lightheadednessHeadachesFever without an obvious causeHeavy menstrual bleeding in women
Clinical phenotype terms (12)— hover any for plain English
Sporadic
Usually appears on its own, not inherited from a parent
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
5 eventsInstitute of Hematology & Blood Diseases Hospital, China — NA
National Heart, Lung, and Blood Institute (NHLBI) — PHASE1
Assistance Publique - Hôpitaux de Paris — PHASE2
National Heart, Lung, and Blood Institute (NHLBI) — NA
Paul Szabolcs — PHASE1, PHASE2
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
No FDA-approved treatments are currently listed for Idiopathic aplastic anemia.
4 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.
View clinical trials →Rare Disease Specialist
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Rare Disease Specialist
Treatment Centers
8 centersCHU Angers
📍 Angers
👤 Julien Rossignol, Dr
CHU bordeaux
📍 Bordeau
👤 Léo RAZAKAMAMANTSOA
👤 Sebastien HASCOËT
CHU Montpellier
📍 Montpellier
👤 Léo RAZAKAMAMANTSOA
CHU Grenoble
📍 Grenoble
👤 Sebastien HASCOËT
👤 Julien Rossignol, Dr
CHU Lille
📍 Lille
👤 Sebastien HASCOËT
Harvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
Travel Grants
No travel grants are currently matched to Idiopathic aplastic anemia.
Community
No community posts yet. Be the first to share your experience with Idiopathic aplastic anemia.
Start the conversation →Latest news about Idiopathic aplastic anemia
Disease timeline:
New recruiting trial: Lung and Bone Marrow Transplantation for Lung and Bone Marrow Failure
A new clinical trial is recruiting patients for Idiopathic aplastic anemia
New trial: Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophospham
Phase PHASE2 trial recruiting. Allogenic transplantation
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 aplastic anemia, and what does that mean for my treatment options?,Am I a candidate for a bone marrow transplant, and how do we find a matching donor?,What are the risks and benefits of immunosuppressive therapy compared to transplant for my situation?,Should I or my family members be tested for inherited bone marrow failure syndromes?,What infections should I be most worried about, and how do I protect myself?,What activities or exposures should I avoid during treatment?,What are the chances of relapse, and what would happen if I relapse?
Common questions about Idiopathic aplastic anemia
What is Idiopathic aplastic anemia?
Idiopathic aplastic anemia (IAA) is a rare and serious blood disorder where the bone marrow — the spongy tissue inside your bones — stops making enough blood cells. 'Idiopathic' means doctors cannot find a clear cause. The bone marrow contains stem cells that normally produce red blood cells, white blood cells, and platelets. In aplastic anemia, the immune system mistakenly attacks these stem cells, leaving the bone marrow empty or nearly empty. This is sometimes called 'bone marrow failure.' Because the body cannot make enough blood cells, people with this condition experience a range of ser
How is Idiopathic aplastic anemia inherited?
Idiopathic aplastic anemia follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
Are there clinical trials for Idiopathic aplastic anemia?
Yes — 4 recruiting clinical trials are currently listed for Idiopathic aplastic anemia on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Idiopathic aplastic anemia?
25 specialists and care centers treating Idiopathic aplastic anemia are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.