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Bone Marrow Failure Syndromes

Nemours Center for Cancer and Blood Disorders in the Delaware Valley and Florida

Locations & Doctors Who Treat Bone Marrow Failure

 
Delaware

Wilmington

Nemours/Alfred I. duPont Hospital for Children

1600 Rockland Road
Wilmington, DE 19803
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For Appointments: (800) 416-4441

Doctors Who Treat Bone Marrow Failure at This Location

 
New Jersey

Voorhees

Nemours duPont Pediatrics, Voorhees

443 Laurel Oak Road
Therapy Services (Suite 200)
Pediatric Specialty Services (Suite 230)
Voorhees, NJ 08043
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For Appointments: (800) 416-4441

Hours: Fourth Friday each month; 9 a.m. to 4 p.m.
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Doctors Who Treat Bone Marrow Failure at This Location

 
Pennsylvania

Philadelphia

Nemours duPont Pediatrics, Philadelphia

833 Chestnut St. E., Suite 300
Philadelphia, PA 19107
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For Appointments: (800) 416-4441

Hours: Monday–Friday: 8 a.m. to 5 p.m.
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Doctors Who Treat Bone Marrow Failure at This Location


Lancaster

Nemours duPont Pediatrics, Lancaster

2128 Embassy Drive, Suite A
Lancaster, PA 17603
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For Appointments: (800) 416-4441

Hours: Hours vary; please call
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Doctors Who Treat Bone Marrow Failure at This Location

 
Florida

Orlando

Nemours Children's Hospital, Orlando

13535 Nemours Parkway
Orlando, FL 32827
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For Appointments: (407) 650-7715

Doctors Who Treat Bone Marrow Failure at This Location


Nemours Children's Specialty Care, Orlando

1717 S. Orange Ave.
Orlando, FL 32806
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For Appointments: (407) 650-7715

Hours: Monday–Friday, 8 a.m. to 5 p.m.
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Doctors Who Treat Bone Marrow Failure at This Location

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Melbourne

Nemours Children's Specialty Care, Melbourne

1270 N. Wickham Road
Melbourne, FL 32935
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For Appointments: (407) 650-7715

Hours: Monday–Friday: 8 a.m. to 5 p.m.
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Doctors Who Treat Bone Marrow Failure at This Location

To find out which providers serve this location, please call the main number above.

Jacksonville

Nemours Children's Specialty Care, Jacksonville

807 Children’s Way
Jacksonville, FL 32207
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For Appointments: (904) 697-3789

Hours: Monday–Friday, 8 a.m. to 5 p.m.
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Doctors Who Treat Bone Marrow Failure at This Location


Pensacola

Nemours Children's Specialty Care, Pensacola

5153 N. Ninth Ave.
Pensacola, FL 32504
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For Appointments: (850) 505-4790

Hours: Monday–Friday, 8 a.m. to 5 p.m. (Central Time)
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Doctors Who Treat Bone Marrow Failure at This Location

About Bone Marrow Failure

Bone marrow failure syndromes are disorders marked by the reduced ability of the bone marrow (the red, spongy material inside the bones) to make blood. Blood has three main cell types: red blood cells (carry oxygen), white blood cells (fight infection) and platelets (clot the blood). New blood cells (called “stem cells”) begin in the bone marrow and are released to the bloodstream when mature.

Bone marrow failure results when one of these cell types is decreased (called “isolated cytopenia”) or all three cell types are decreased (called “pancytopenia”), which affects the body’s ability to function. Reduction in cell production can be caused by cell damage (aplastic anemia), defects that occur when a generic cell differentiates (or changes into) a specific type of cell (like pediatric myelodysplastic syndrome) and bone marrow infiltration (from blood cancer such as leukemia and multiple myeloma).  

Our renowned hematologists, oncologists and researchers at the Nemours Center for Cancer and Blood Disorders (NCCBD) work together with other top specialists to care for children with all kinds of bone marrow failure syndromes. 


Types of Childhood Bone Marrow Failure Syndromes We Treat

Bone marrow failure syndromes are complex and can affect the body in many different ways. Bone marrow failure can be inherited and present at birth (called “congenital”) or can develop later in life due to certain exposures (called “acquired”). In some cases, there is no known cause (referred to as “idiopathic”) for bone marrow failure.

 
Acquired Bone Marrow Failure Syndromes
Aplastic Anemia

Aplastic anemia in children occurs when the bone marrow fails to produce enough of all three blood cell types: red, white and platelets. Aplastic anemia commonly arises when the immune system suppresses stem cells. Although most of the time (in 50–75 percent of cases) aplastic anemia has no known cause (this is called “idiopathic aplastic anemia”), some are the result of infections (post-infectious aplastic anemia), medications (including chemotherapy), or environmental toxins, and exposure to radiation and autoimmune diseases (which cause the body’s immune system to attack healthy cells) can contribute.

Aplastic anemia in children is classified as “moderate,” “severe” or “very severe.” 

Pediatric Myelodysplastic Syndrome (MDS)

Myelodysplastic syndrome (MDS) is rare blood disease that occurs when the bone marrow fails to develop all three blood cell types: red, white and platelets. In myelodysplastic syndrome, the blood cell maturation process is disturbed. Whether it’s due to too few mature blood cells being produced, or too many immature blood cells (called “blasts”) crowding out the mature ones, pediatric myelodysplastic disease can sometimes turn into leukemia (in 30 percent of the cases). 

In primary myelodysplastic disease, there is no known cause. Secondary myelodysplastic disease can result from inherited bone marrow failure syndromes, cancer treatments (radiation therapy and chemotherapy) and genetic causes (called “familial MDS”).

There are different types of myelodysplastic syndrome based on how the cells look under the microscope, and by the amount of blasts present.

These include:

  • refractory cytopenia of childhood (RCC)
  • refractory anemia with excess blasts (RAEB)
  • RAEB in transformation
 
Inherited Bone Marrow Failure Syndromes
  • Fanconi anemia (affects the genes that repair damaged DNA in stem cells)
  • Diamond Blackfan anemia (affects ribosomes, or cellular structures that helps make the protein in red blood cells)
  • dyskeratosis congenita (affects multiple body systems, skin, nails and mouth)
  • Shwachman-Diamond syndrome (affects the white blood cells causing pancreatic and skeletal defects)
  • severe congenital neutropenia (also called “Kostmann syndrome,” affects the white blood cell maturation process causing recurring infections)
  • congenital amegakaryocytic thrombocytopenia (affects the cells that make platelets)
  • thrombocytopenia absent radius (or “TAR syndrome,” affects the platelets causing missing radius bones in the forearms)

Diagnosing Bone Marrow Failure in Children

Most often, bone marrow failure syndromes that are inherited and only affect one blood cell type (including Diamond Blackfan anemia, severe congenital neutropenia and congenital amegakaryocytic thrombocytopenia) are diagnosed in infancy. Bone marrow syndromes that affect more than one blood cell type (aplastic anemia, Fanconi anemia and dyskeratosis congenital) are diagnosed in later childhood and adolescence. 

Signs & Symptoms of Bone Marrow Failure

Symptoms of bone marrow failure in children vary widely depending on the disorder, but can include:

  • pale skin (pallor)
  • energy loss
  • shortness of breath
  • small red dots under the skin (petechiae)
  • unexplained and/or recurring infections
  • easy bruising
  • fatigue
  • difficulty stopping bleeding (with minor wounds, nosebleeds)

Physical defects such as skin pigmentation, short stature and abnormalities in the thumbs may also be present.


Testing & Evaluations

If your child has a possible bone failure syndrome such as aplastic anemia or myelodysplastic syndrome, Nemours’ experienced cancer and blood disorder care team works together to quickly and appropriately diagnose the disease through a variety of tests and procedures, including:

  • thorough physical examination and medical history
  • blood tests
  • bone marrow aspiration and biopsy

Treating & Caring for Children With Bone Marrow Failure

Your Child's Comprehensive Bone Marrow Failure Care Team

If we discover a bone marrow failure syndrome, our multidisciplinary (collaborative) team plans the most effective treatment personalized to your child’s needs. In addition to an experienced, compassionate nurse coordinator (who provides your child and family support, education and care coordination), Nemours’ pediatric blood disorder teams include:

  • hematologists (blood disorder specialists)
  • oncologists (cancer doctors)
  • gastroenterologists (digestive health specialists)
  • pulmonologists (lung specialists) 
  • endocrinologists (specialists in hormone and growth disorders)
  • immunologists (autoimmune disorder specialists)
  • dermatologists (skin doctors)
  • pathologists (diagnose diseases by examining body tissues, fluids and organs)
  • geneticists (perform genetic counseling and testing)
  • interventional radiologists (perform image-guided minimally-invasive procedures)
  • behavioral health specialists (psychologists and psychiatrists)
  • registered dietitians (nutrition specialists)
  • hematology nurses (registered nurses specializing in childhood blood disorders)  

Your care team is here to provide your family with ongoing support and follow-up. We always include you in the entire process, because we truly believe in the healing power of families, and that you know your child best.

Learn More About Nemours’ Pediatric Hematology Care Teams in Your Area »


Innovative Childhood Bone Marrow Failure Treatment at Nemours

Bone marrow failure syndromes in children are complex and require expert, efficient treatment, close monitoring and follow-up care. Treatments depend on the symptoms and resulting medical problems caused by or related to the disease. Treatment might include:

  • blood transfusion (adding blood cells and platelets intravenously — through the veins)
  • antibiotics (for infections that cause the condition)
  • immunosuppressive therapy (to keep the immune system from attacking healthy cells) 
  • granulocyte-colony stimulating growth factor (or “G-CFS,” to stimulate the bone marrow to produce blood cells, particularly with severe congenital neutropenia) 
  • steroid therapy (prednisone or cortisone to help increase hemoglobin or red blood cells)
  • targeted drug therapies (targets genetic abnormalities)
  • blood and bone marrow transplant (also called a “BBMT,” “BMT” or “stem cell transplant”)
  • hormone therapy (to improve blood counts, particularly with Fanconi anemia)
  • chemotherapy (cancer-killing medicines)
For your child’s overall health and wellness, we also prescribe:

Learn More About Pediatric Hematology Treatment at Nemours »

Additional Resources & Support for Families

 
Nemours Support Services

Dealing with a complex medical condition is difficult for your child and family. But you don’t have to go through it alone. Nemours provides an array of support services that begin on the very first day we meet, and continues throughout your journey because at Nemours, your child and family become part of our own.

Our services include: 
  • patient and family education
  • social work
  • mental health counseling
  • Child Life services
  • creative therapy
  • support groups

Get Comprehensive Patient Education & Support Resources »

 
Informative Articles & Videos for Parents From Nemours’ KidsHealth

Learn More From Nemours' KidsHealth.org

General Information
Testing and Diagnosis
Treatments
 
Trusted National Bone Marrow Failure Syndrome Websites

Diamond Blackfan Anemia Foundation: This foundation’s website is designed to direct you to information and resources for patients, families and research.

National Heart, Lung, and Blood Institute: The institute is part of the Department of Health and Human Services, and promotes the research, prevention and treatment of heart, lung and blood diseases.

Platelet Disorder Support Association: This organization is dedicated to enhancing the lives of people with immune thrombocytopenia (ITP) and other platelet disorders through education, advocacy, research and support.

 
Blood & Bone Marrow Resources

Blood & Marrow Transplant Information Network: BMTinfonet.org provides information and support for patients and families before, during and after transplant.

The Bone Marrow Foundation: They offer information on financial assistance and resources to bone marrow/stem cell transplant patients and their families. 

Center for International Blood & Marrow Transplant Research (CIBMTR):The center disseminates information on blood and marrow transplant research.

National Marrow Donor Program: The site contains info about marrow and cord blood donation and transplantation.

National Bone Marrow Transplant Link: The organization helps patients, caregivers and families cope with the social and emotional challenges of bone marrow/stem cell transplantation, from diagnosis through survivorship. They provide information and personalized support services.

Appointments & Referrals

Delaware, Pennsylvania & New Jersey

(800) 416-4441


Florida
Jacksonville Area

(904) 697-3789

Orlando Area

(407) 650-7715

Pensacola Area

(850) 505-4790

Learn About Our Locations »


International
Patients

+1 (302) 651-4993

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