Bone marrow is the soft tissue inside the body’s large bones. Bone makes 95% of the body’s blood cells, including red cells, white cells and platelets. These cells help carry oxygen, fight infection and stop bleeding.

A bone marrow disorder is when it doesn’t make enough healthy blood cells or makes cells that don’t work right. This can lead to tiredness, infections or bruising. Your child might have low white blood cell counts or anemia.

There are many causes of bone marrow failure. Some bone marrow disorders are genetic and passed down from parents. Others are due to exposures or changes over time and develop later in life.

Inherited Marrow Failure Syndromes

Genetic changes can disrupt the bone marrow, causing inherited bone marrow diseases. They’re present at birth. They often affect how your body makes blood cells.

• Fanconi anemia: Genetic changes affect the bone marrow, causing low blood cell counts. It can also affect growth and increase cancer risk.
• Shwachman-Diamond syndrome: A genetic disorder that affects the bone marrow, pancreas and bones. It can lead to low white blood cells and frequent infections.
• Diamond-Blackfan anemia: Mostly affects red blood cells. Kids with this condition may look pale or feel tired.
• Severe congenital neutropenia: Kids with this disorder have very low levels of neutrophils, a type of white blood cell. This makes it harder to fight infections.
• Dyskeratosis congenita: Affects many body parts, including skin, nails and bone marrow. It can lead to low blood cell counts and other health issues.
• Amegakaryocytic thrombocytopenia (Amega): Kids have very few or no megakaryocytes. These cells help make platelets. It causes easy bruising or bleeding.
• Pearson syndrome: A rare condition that affects energy production in cells. It causes problems in the bone marrow and pancreas.

Acquired Marrow Failure

These disorders develop over time and are not inherited.

• Aplastic anemia: The bone marrow stops making enough new blood cells. This can cause fatigue, infections and bleeding.
• Paroxysmal nocturnal hemoglobinuria (PNH): A rare disease where red blood cells break down too early. This can lead to blood clots, dark urine and other serious issues.
• Acquired neutropenia: Low levels of neutrophils caused by illness, medicine or other reasons. It raises the risk of infections.
• Viral infections: Some viruses can affect the bone marrow. This may be temporary or permanent. These include Epstein-Barr virus, cytomegalovirus, hepatitis viruses, parvovirus B19 and HIV.
• Toxins and medicines: Certain chemicals, pesticides and medicines can damage the bone marrow and lower blood cell counts.

Your child’s care plan depends on:

• Their diagnosis
• Their age
• Their overall health
• How their body responds to treatment

Some treatments aim to fix the problem, while others help manage symptoms and keep your child safe and comfortable.

Medicines

Medicines help the bone marrow work better and make more blood cells.

• Growth factors: Medicines can help the body make more white and red blood cells.
• Immunosuppressive therapy: These medicines calm the immune system. Used in aplastic anemia.
• Antibiotics and antivirals: Help prevent or treat infections.

Supportive Care

We offer care that supports your child’s comfort, nutrition and safety.

• Infection care: We use medicines, protective steps and regular checkups to lower the risk of infection.
• Diet support: Our care team works with your family to ensure your child gets the nutrition they need.
• Vitamins: Nutrients to support blood cell growth and overall health.

Procedures

These procedures replace or support the bone marrow when it isn’t working well.

• Blood transfusions: Replace missing red blood cells or platelets. Helps treat symptoms like fatigue, weakness or bleeding.
• Bone marrow transplant: A possible cure for some bone marrow disorders. We replace defective bone marrow with healthy marrow from a donor.
• Clinical trials: We have active open clinical trials to treat patients with aplastic anemia for both front immunosuppresive treatment protocols and novel transplant protocols.

When your child comes to Children’s Wisconsin for a bone marrow disorder, you’ll get expert care. Your care team treats your family like their own.

We’ll work closely with you to learn about your child’s symptoms and make an accurate diagnosis.

We use a team approach. Your child’s care team includes experts in:

• Blood disorders
• Immunology
• Infectious disease
• Genetics
• Nutrition

We all work together to build a care plan tailored to your child. We meet often to review your child’s progress and adjust the plan as needed.

Here’s what you can expect:

• Tailored testing: We begin with advanced testing to find out exactly what’s causing the problem. Our experts study your child’s blood counts, bone marrow samples and genetic results.
• Support at every step: From the first visit through treatment and follow-up care, we’re by your side at each step. We help you understand your child’s condition, make decisions together and support your family in every way.
• Advanced care options: We offer the latest therapies designed for kids. We’ll walk you through your options. We also provide pain care, mental health support and other therapies to help your child feel as strong as possible.
• Long-term follow-up: Many bone marrow disorders need regular monitoring. We stay with you for the long haul. You’ll have regular visits to check labs, adjusting care plan and watch for any changes in your child’s health.