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If your child has trouble fighting off infections or has bleeding or clotting issues, they may have a bone marrow failure disorder.
Bone marrow is a soft tissue inside the body's bones. Within this tissue are stem cells.
Your body uses stem cells to make:
All of these blood cells are vital to your health. When bone marrow doesn't make enough of these cells, doctors call it bone marrow failure.
Bone marrow failure might also make damaged cells.
Bone marrow failure disorders are a group of conditions. In other words, there's more than one type of bone marrow failure disorder.
Some types are genetic, meaning parents pass them to their children through genes. Also known as inherited bone marrow failure syndromes, they're more common in kids.
Acquired bone marrow failure disorders come from:
Children with bone marrow failure disorders are at risk of certain cancers:
Bone marrow failure disorders are rare diseases.
Our experts at UPMC Children's Hospital of Pittsburgh treat a range of genetic and acquired bone marrow failure disorders.
Aplastic anemia is when damaged stem cells keep the body from making enough new blood cells. This disorder can be genetic or acquired.
MDS results from stem cells changing in abnormal ways, causing them to make fewer blood cells. This syndrome is a type of cancer.
FA is an inherited aplastic anemia that impacts the bone marrow's production of all blood cells.
SA occurs when red blood cells have too much iron and don't form correctly. These anemias are either genetic or acquired, often as part of MDS.
Since there are many types of bone marrow failure disorders, symptoms vary.
Some symptoms your child might have include:
Talk with your doctor if you notice changes in your child or have concerns about something you see.
Bone marrow failure disorders are rare.
To make a diagnosis, doctors gather data and use tests such as:
Sometimes, the doctor may suggest a bone marrow aspiration or biopsy to test the bone marrow itself. They will see if the cells that give rise to red and white blood cells and platelets look normal.
An aspiration removes bone marrow in liquid form. A biopsy removes intact bone marrow tissue.
Doctors use a needle to remove the liquid or tissue from the hip bone while your child is under anesthesia.
How doctors treat your child's bone marrow failure disorder depends on the type they have.
The Bone Marrow Failure Disorders Program experts will work with you and your family to manage your child's care.
Doctors insert a needle into a vein to transfer donated blood from a family member or an unknown blood donor. They take a few hours to complete.
Blood transfusions help prevent anemia, and platelet transfusions stop severe event bleeding.
These are drugs that help keep the immune system from attacking the body's bone marrow. That way, the bone marrow can make more blood cells.
The most common drugs include anti-thymocyte globulin (ATG) and cyclophosphamide.
Called hematopoietic growth factor, these drugs trigger a child's bone marrow to make more blood cells.
Doctors will remove damaged or sick bone marrow and replace it with healthy stem cells.
Depending on the transplant type, healthy cells come from your child, a donor, or umbilical cord blood.
Your child might need chemo or radiation to get ready for a bone marrow transplant.
Your care team will discuss which treatments are best for your child.
You can count on our doctors to partner with you and your family in managing your child's illness.
When you come for your first visit, you'll talk with a doctor or other team member, based on your child's needs.
They'll talk about any tests your child might need, such as blood tests or a bone marrow biopsy.
They might want to take blood samples from your child during this first visit.
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
With myCHP, you can request appointments, review test results, and more.
For questions about a hospital bill call:
To pay your bill online, please visit UPMC's online bill payment system.
Interested in giving to Children's Hospital? Support the hospital by making a donation online, joining our Heroes in Healing monthly donor program, or visiting our site to learn about the other ways you can give back.