Specialties & Conditions Bone Marrow Failure
Bone Marrow Failure in Children
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What Causes Bone Marrow Failure Disorders in Children?
These disorders can be inherited or acquired.
Inherited Disorders
These involve genetic mutations (changes to genes) passed down in families. Children are born with genetic changes that affect bone marrow function. These bone marrow failure disorders can appear even without a known family history.
Acquired Disorders
These disorders typically occur during adolescence and adulthood. Potential causes include:
- Exposure to environmental triggers, including certain chemicals or radiation
- Immune system mistakenly attacking its own bone marrow
- Reactions to some medications
- Viral infections (like certain types of hepatitis)
Sometimes, doctors can't identify the exact cause. But keep in mind that these disorders are not caused by anything a parent did or didn’t do.
What Are the Symptoms of Bone Marrow Failure Disorders?
Symptoms depend on the affected blood cell type:
- Platelets (thrombocytopenia): Easy bruising, frequent or prolonged nosebleeds, petechiae (tiny red skin spots) and prolonged bleeding from cuts
- Red blood cells (anemia): Fatigue, pale skin, dizziness, headaches and shortness of breath
- White blood cells (leukopenia): Recurrent infections, mouth sores and unexplained fevers
How Do Doctors Diagnose Bone Marrow Failure Disorders?
Accurate diagnosis is important for effective treatment. Doctors order many tests to identify causes.
It typically begins with asking about your child’s medical history. From there, your care team will use various tests to make a diagnosis and determine the next steps. These tests may include:
- Blood tests, to evaluate blood cell counts
- Bone marrow biopsy, to analyze cell structure and function.
- Cancer screening, because some bone marrow disorders increase the risk of some cancers, including leukemia
- Genetic testing, which can determine if the disorder is inherited
- Viral studies, which can identify infections that may have triggered the condition
What Happens During a Bone Marrow Biopsy?
One of our skilled proceduralists will insert a thin hollow needle into the hip bone to collect a small bone marrow sample. Imagine poking an apple with a straw. The piece of apple that comes up is like the tiny sample of bone marrow we collect. We perform the procedure under general anesthesia to ensure your child is comfortable and doesn't feel any pain. (However, your child may feel some pain after the procedure.)
This sample helps us determine the cause of bone marrow failure by giving us important clues about why your child has low blood cell counts. A hematopathologist will assess the bone marrow sample. This helps us to better understand the possible cause of failure or identify signs of increased cell production.
It will also identify signs of increased cell production. If blood cells are being destroyed elsewhere in the body, the bone marrow often tries to compensate by making more.
How Do Doctors Treat Bone Marrow Failure Disorders?
Your child’s treatment depends on the severity and cause of the disorder.
- Blood transfusions can increase blood cell counts (red cells and platelets) and manage symptoms.
- Immunosuppressive therapy can calm an overactive immune response in conditions such as aplastic anemia.
- Stem cell transplantation may be an option. If your doctor determines this is the best approach, they will refer your child to the Stem Cell Transplant team. These specialists will determine whether transplantation is an option for your child.
Because bone marrow disorders can affect different parts of the body, Lurie Children’s provides care from a wide range of specialists.
Collaborative Care for Bone Marrow Failure Disorders
Bone marrow disorders are complex and can affect various aspects of your child’s health. It requires a team approach, and that’s what we provide.
At Lurie Children’s, we provide a highly collaborative approach to bone marrow failure disorders. Depending on your child’s specific diagnosis, they may see several specialists in different fields, including:
- Hematology and oncology
- Endocrinology
- Gastroenterology
- Genetics
- Stem cell transplantation
The close collaboration among these experts allows for faster coordination of appointments, streamlined communication between providers, and personalized treatment strategies. Your doctor will also be coordinating with support teams, including:
- Education liaisons for school accommodations
- Genetic counselors to explain hereditary conditions
- Psychologists to help children manage the emotional impact of their diagnosis and treatment
- Social workers for family support and coping strategies
- The Cancer Predisposition Clinic for certain bone marrow failure disorders
Long-Term Consequences of Bone Marrow Failure Disorders
The long-term impact depends on both the specific diagnosis and the type of treatment your child receives. Here are a few:
- Cancer risk: Some disorders make it more likely for children to get leukemia, requiring ongoing screening.
- Iron overload: This is a particular concern for children who need frequent transfusions.
- Stem cell transplant monitoring and management: Transplants can sometimes cure bone marrow failure disorders but carry risks. For example, transplanted immune cells can attack the recipient's healthy tissue. Children who receive stem cell transplants will likely need ongoing, even lifelong, follow-up care and supportive therapies.
The Center for Cancer and Blood Disorders at Lurie Children’s
Lurie Children’s Center for Cancer and Blood Disorders provides the region’s most comprehensive care for pediatric blood disorders. Our family-centered approach addresses each child's unique physical, emotional and social needs within a nurturing, child-friendly environment.
We coordinate care across specialties, making it easier to access expert treatment. We also collaborate with The Robert H. Lurie Comprehensive Cancer Center of Northwestern University to offer the latest advancements in care. This approach ensures your child receives the most advanced and personalized care possible.
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