Congenital Aplastic Anemia
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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
Pediatrics | Pediatric Hematology Oncology
Elite in Congenital Aplastic Anemia
Pediatrics | Pediatric Hematology Oncology
Referral may be required

M Health Fairview Center For Pediatric Blood And Marrow Transplant And Celluar Therapy

Journey Clinic Riverside East 9th, Floor 2450 Riverside Ave, 
Minneapolis, MN 
Languages Spoken:
English
Accepting New Patients

Margaret Macmillan is a Pediatrics specialist and a Pediatric Hematologist Oncology provider practicing medicine in Minneapolis, Minnesota. Dr. Macmillan is rated as an Elite provider by MediFind in the treatment of Congenital Aplastic Anemia. She is also highly rated in 14 other conditions, according to our data. Her clinical expertise encompasses Congenital Aplastic Anemia, Fanconi Anemia, Graft Versus Host Disease (GvHD), Aplastic Anemia, and Bone Marrow Transplant. Dr. Macmillan is currently accepting new patients.

Elite in Congenital Aplastic Anemia
Hematology | Oncology
Elite in Congenital Aplastic Anemia
Hematology | Oncology
Referral may be required

Office

3333 Burnet Ave # 7015, 
Cincinnati, OH 
Languages Spoken:
English

Stella Davies is a Hematologist and an Oncologist practicing medicine in Cincinnati, Ohio. Dr. Davies is rated as an Elite provider by MediFind in the treatment of Congenital Aplastic Anemia. She is also highly rated in 22 other conditions, according to our data. Her clinical expertise encompasses Congenital Aplastic Anemia, Fanconi Anemia, Shwachman-Diamond Syndrome, Bone Marrow Transplant, and Bone Graft.

 
 
 
 
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Elite in Congenital Aplastic Anemia
Hematology | Oncology
Elite in Congenital Aplastic Anemia
Hematology | Oncology
Referral may be required

UCI Health Chao Family Comprehensive Cancer Center

101 The City Drive South, Building 23, 
Orange, CA 
Languages Spoken:
English, Spanish

Dr. Miguel A. Villalona-Calero is a UCI Health medical oncologist who specializes in lung cancer, early phase clinical trials, the use of genomic-driven therapies and immune system treatments to target cancer.An internationally regarded expert in lung cancer and developmental therapeutics, Villalona-Calero serves as deputy director of the UCI Health Chao Family Comprehensive cancer center. As chief of the UC Irvine School of Medicine's Division of Hematology-Oncology, he leads a faculty of more than 70 hematologists and oncologists, including nationally know specialists in many types of cancer.He earned his medical degree from Universidad Nacional Pedro Henriquez Urena in the Dominican Republic, followed by an internship and internal medicine residency at Kings County Hospital Center and the State University of New York Health Science Center in Brooklyn. He completed fellowships in medical oncology at the University of Minnesota Hospitals & Clinics in Minneapolis and the Roswell Park Cancer Institute in Buffalo, N.Y.He also received fellowship training at the University of Texas’ Institute for Drug Development in San Antonio and in health policy leadership at New York University’s Wagner Graduate School of Public Service in New York City. Before joining UCI Health, he served as director of an early phase therapeutics program at City of Hope in Duarte, Calif.Villalona-Calero is widely known for his work in lung cancer and developmental therapeutics, including the design of more effective anticancer agents. His main area of expertise is in translating laboratory science developments into clinical trials for cancer patients. Most recently, his focus has been on targeting cellular deficiencies in DNA repair and finding new ways to use the immune system to attack cancer cells.The author or co-author of more than 140 peer-reviewed publications, Villalona-Calero has been inducted into the American Association for the Advancement of Science and is a fellow of the American College of Physicians. He has led dozens of clinical trials, including early phase studies funded by the National Cancer Institute (NCI) and the National Institutes of Health (NIH). He currently leads a phase 1 clinical trial for non-small cell lung cancer using enhanced natural killer cells, part of a prestigious NCI and NIH R01 grant for developmental cell therapeutics.Villalona, who is fluent in English and Spanish, sees patients inIrvineandOrange. Dr. Villalona is rated as an Elite provider by MediFind in the treatment of Congenital Aplastic Anemia. He is also highly rated in 13 other conditions, according to our data. His clinical expertise encompasses Fanconi Anemia, Congenital Aplastic Anemia, Lung Cancer, and Non-Small Cell Lung Cancer (NSCLC). Dr. Villalona is board certified in American Board Of Internal Medicine - Medical Oncology.

What are the latest Congenital Aplastic Anemia Clinical Trials?
The Experience and Management of Cancer Screening-Related Anxiety in Fanconi Anemia: an Ethnographic Study

Background: Fanconi anemia (FA) is a rare, inherited cancer syndrome. FA causes a range of physical issues. Children with FA may have abnormal features; these may include a small head and eyes and issues with their internal organs. Young adults have a much higher risk of cancer. To screen for these cancers, people with FA may need to pursue many visits with different doctors. This constant need for cancer scr...

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Familial Investigations of Childhood Cancer Predisposition

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...