Comprehensive Care for Bone Marrow Failure Syndromes

The Bone Marrow Failure Clinic at Cincinnati Children’s provides highly sophisticated and comprehensive multidisciplinary care for children with bone marrow failure syndromes.
Bone marrow failure happens when your bone marrow no longer makes enough blood cells. It can be caused by something that happens to you during your lifetime (such as chemotherapy or an infection) or you can be born with it. A variety of inherited diseases can cause bone marrow failure.

Thorough Evaluation, Detailed Treatment Plan

Families usually come to the Bone Marrow Failure Clinic at Cincinnati Children’s to establish a diagnosis or receive a second opinion. First, our team offers a comprehensive evaluation that typically lasts two to four days. Next, we provide treatment recommendations. Before the evaluation, the team reviews test results and other materials provided by the referring doctor. Our evaluation includes:

• A thorough history and physical examination
• Blood tests
• Other tests to assess how the disease is affecting the child’s body
• If needed, additional consultations with specialists at Cincinnati Children’s
• An appointment with a social worker who helps families connect with helpful resources

In addition, we encourage families to consult with genetic counselors at Cincinnati Children’s. These counselors can talk with families about the results of genetic tests. Genetic tests are used to help confirm diagnoses and refine the treatment plan. Our team helps schedule these consultations.

Making a Plan for the Future

After reviewing the results of the evaluation, and before the family returns home, members of the care team talk with the family about the child’s health and treatment options. The team also provides a written summary and treatment recommendations to the patient’s family and referring doctor.

Innovative Therapy for Bone Marrow Failure

We offer a broad range of therapies, including some innovative approaches not available at other children’s hospitals. Our multidisciplinary team of experts offers exceptional care.

Among the many treatment options are:

• Novel medications
• Blood transfusions
• Hematopoietic stem cell transplant

Ongoing Care

Children can receive ongoing care at Cincinnati Children’s on an inpatient or outpatient basis, or return home to the care of their referring doctor. Our team is always available to consult with families and their doctors. We see patients once a year to assess their progress and make additional treatment recommendations, if necessary.

Syndromes We Treat

The Bone Marrow Failure Clinic offers evaluation, diagnosis and treatment for all types of bone marrow failure syndromes. Among the conditions we see are:

Research That Leads to Innovation

Doctors and researchers at Cincinnati Children’s participate in numerous clinical and laboratory research projects designed to improve the lives of patients and find new therapies to treat bone marrow failure syndromes.

In our laboratories, we are working to:

• Investigate molecular, clinical and genetic mechanisms of bone marrow failure
• Find new biomarkers that predict prognosis
• Explore longitudinal changes in blood that predict bone marrow failure
• Explore novel therapeutics for certain bone marrow failure syndromes

Our doctors partner with other researchers throughout the world to further the understanding and treatment of these diseases. For instance, our team participates in the Severe Chronic Neutropenia (SCN) International Registry, which monitors the clinical course, treatment and outcomes of patients with SCN. We also participate in the Diamond Blackfan Anemia Registry, the Dyskeratosis Congenita Registry, and the Shwachman-Diamond Syndrome Registry, for which Kasiani Myers, MD, serves as the co-director.