Congenital Aplastic Anemia
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Congenital Aplastic Anemia Overview

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Learn About Congenital Aplastic Anemia

View Main Condition: Anemia

What is the definition of Congenital Aplastic Anemia?
Congenital aplastic anemia is a rare, serious condition where an infant is born with an abnormality of the bone marrow that leads to inadequate production of red and white blood cells (erythrocytes and granulocytes) and platelets (cell fragments that produce blood clotting). Infants born with congenital aplastic anemia often have several other congenital abnormalities, such as small heads, kidney abnormalities, abnormalities of the forearms and thumbs, other skeletal abnormalities, heart and lung defects, abnormalities of the urinary and genital tracts, and an increased risk of developing cancers, such as gastrointestinal malignancies and leukemia.
What are the symptoms of Congenital Aplastic Anemia?
Symptoms of congenital aplastic anemia include fatigue, pale skin, shortness of breath, rapid or irregular heart rate, headache, dizziness, fever, frequent or prolonged infections, rash, bruising easily, nosebleeds and bleeding gums, and uncontrolled bleeding. In severe cases, congenital aplastic anemia can be fatal.
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What are the current treatments for Congenital Aplastic Anemia?
While there is no cure for congenital aplastic anemia, treatment includes red blood cell and platelet transfusions; immunosuppressant medications, such as cyclosporine (Gengraf, Neoral, and Sandimmune) and anti-thymocyte globulin; corticosteroids; and bone marrow stimulants, such as the colony-stimulating factors, sargramostim (Leukine), filgrastim (Neupogen), pegfilgrastim (Neulasta), epoetin alfa (Epogen/Procrit), and eltrombopag (Promacta). Antibiotics or antivirals may be administered for infections. Some selected individuals with severe aplastic anemia may be considered for stem cell transplant.
Who are the top Congenital Aplastic Anemia Local Doctors?
Elite in Congenital Aplastic Anemia
Dr. Stella Davies
Hematology | Oncology
Elite in Congenital Aplastic Anemia
Dr. Stella Davies
Hematology | Oncology

Office

3333 Burnet Ave # 7015, 
Cincinnati, OH 
513-636-4266
Languages Spoken:
English
See accepted insurances

Stella Davies is a Hematologist and an Oncologist in Cincinnati, Ohio. Dr. Davies is rated as an Elite provider by MediFind in the treatment of Congenital Aplastic Anemia. Her top areas of expertise are Congenital Aplastic Anemia, Fanconi Anemia, Shwachman-Diamond Syndrome, Bone Marrow Transplant, and Bone Graft.

Elite in Congenital Aplastic Anemia
Dr. Parinda Mehta
Hematology | Oncology
Elite in Congenital Aplastic Anemia
Dr. Parinda Mehta
Hematology | Oncology

Cincinnati Children's Hospital Medical Center

3333 Burnet Ave, 
Cincinnati, OH 
513-636-4266
Languages Spoken:
English
See accepted insurances

Parinda Mehta is a Hematologist and an Oncologist in Cincinnati, Ohio. Dr. Mehta is rated as an Elite provider by MediFind in the treatment of Congenital Aplastic Anemia. Her top areas of expertise are Congenital Aplastic Anemia, Fanconi Anemia, Aplastic Anemia, Anemia, and Bone Marrow Transplant.

 
 
 
 
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Elite in Congenital Aplastic Anemia
Dr. Kasiani Myers
Hematology | Oncology
Elite in Congenital Aplastic Anemia
Dr. Kasiani Myers
Hematology | Oncology

Cincinnati Children's Hospital Medical Center

3333 Burnet Ave, 
Cincinnati, OH 
513-636-4266
Languages Spoken:
English
See accepted insurances

Kasiani Myers is a Hematologist and an Oncologist in Cincinnati, Ohio. Dr. Myers is rated as an Elite provider by MediFind in the treatment of Congenital Aplastic Anemia. Her top areas of expertise are Shwachman-Diamond Syndrome, Congenital Aplastic Anemia, Fanconi Anemia, Aplastic Anemia, and Bone Marrow Transplant.

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What are the latest Congenital Aplastic Anemia Clinical Trials?
Familial Investigations of Childhood Cancer Predisposition
Familial Investigations of Childhood Cancer Predisposition
Enrollment Status: Recruiting
Publish Date: February 09, 2026

Summary: NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll. If you are interested in clinical testing please consider seeing a local genetic counselor or other genetics professional. If you have already had clinical genetic testing and meet eligibility...

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Etiologic Investigation of Cancer Susceptibility in Inherited Bone Marrow Failure Syndromes: A Natural History Study
Etiologic Investigation of Cancer Susceptibility in Inherited Bone Marrow Failure Syndromes: A Natural History Study
Enrollment Status: Recruiting
Publish Date: February 05, 2026

Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders. Pathogenic variant(s) in IBMFS genes are relevant to carcinogenesis in sporadic cancers. Patients with IBMFS who develop cancer differ in their genetic and/or environmental features from patients with IBMFS who do not develop cancer. These cancer-...

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