Inherited marrow failure

There is often a genetic component to inherited marrow failure disorders, meaning that these rare blood conditions are associated with a family history of the disorder.

 When you bring your child with marrow failure to the MACC Fund Center for Cancer and Blood Disorders at Children’s Wisconsin, one of our first steps will be to determine whether the condition is inherited or acquired. That distinction can play a significant role in determining the patient’s treatment plan.

 If we discover the disorder is due to a family history, our expert team provides comprehensive clinical care to pediatric patients with a variety of inherited marrow failure disorders, including:

  • Amegakaryocytic thrombocytopenia (Amega), a very low number of a certain type of bone marrow cell
  • Diamond-Blackfan anemia (DBA), malfunctioning bone marrow
  • Dyskeratosis congenita (DC), a type of marrow failure that can affect fingernails and toenails, the mouth, and skin coloring
  • Fanconi anemia (FA), decreased production of blood cells
  • Pearson syndrome, problems with the development of blood-forming cells
  • Severe congenital neutropenia (SCN), a shortage of certain white blood cells
  • Shwachman-Diamond syndrome (SDS), a shortage of white blood cells caused by malfunctioning bone marrow
  • Thrombocytopenia absent radii (TAR), a platelet deficiency characterized by skeletal problems

Because patients with inherited marrow failure disorders are at high risk of developing cancer and other complications, quick and effective treatment is critical. Our expert clinical team of board-certified physicians, advanced practice providers, and nurses is experienced with all major treatment options, including blood and marrow transplant and cellular therapy. We will work with your family to determine the most appropriate treatment option for your child and guide you through every step of the treatment process.

Call (414) 266-2420 for more information or to make an appointment.

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For appointments, contact the individual programs within our MACC Fund Center for Cancer and Blood Disorders.

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