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The Bone Marrow Failure and Myelodysplastic Syndrome Clinic gives the highest quality of care for children and adolescents who have disorders that affect the bone marrow.
The Bone Marrow Failure and Myelodysplastic Syndromes (BMF/MDS) Clinic at St. Jude Children’s Research Hospital provides specialized care for children who have disorders that affect how their bone marrow works.
Bone marrow is the soft, spongy tissue inside most bones. It contains stem cells, which make new blood cells such as white blood cells, red blood cells, and platelets.
BMF and MDS can have different causes. In some children, these conditions are caused by genetic changes passed down from a parent (inherited). In other children, BMF or MDS develops later and is acquired (not inherited). For example, acquired aplastic anemia is often caused by an overactive immune system that damages the bone marrow.
BMF and MDS can cause very low blood cell counts and, in some cases, increase the risk of developing leukemia.
Patients must be referred to the BMF/MDS Clinic by their primary clinical team or by a St. Jude affiliate site.
The BMF/MDS Clinic brings together specialists from different areas of medicine and uses advanced genomic (gene) testing. The team diagnoses patients, guides treatment choices, and watches patients for early signs that the condition may be getting worse.
Our highly trained experts provide complete care and management of a child’s condition. They support patients and families by:
The clinic also offers virtual consultations for clinicians who would like to discuss care of their patients.
The BMF/MDS Clinic is staffed by medical professionals and bone marrow failure specialists, including:
This experienced team works together to diagnose inherited and acquired BMF/MDS disorders.
They also help manage children’s care and support them throughout their treatment.
Some members of the BMF/MDS team also do research to develop new treatments, known as personalized medicine, for these rare disorders.
During your visit:
Depending on your child’s condition, they may also get a bone marrow evaluation, genetic testing, and imaging studies.
Follow-up visits may include testing to watch for acquired (pre-leukemic) genetic changes (changes in genes). These test results will help your doctor suggest the best treatment plan for your child.
St. Jude provides the highest quality of care with BMF/MDS. Along with standard medical treatments, St. Jude offers clinical trials and research studies to treat related medical problems.
Learn more about clinical research at St. Jude.
Study goal:
To study mavorixafor as an oral treatment to raise neutrophil counts and reduce infections in chronic neutropenia.
Age:
12 years and older
Study goal:
To study iptacopan (LNP023) in children and adolescents with paroxysmal nocturnal hemoglobinuria (PNH) to understand drug exposure and evaluate safety and tolerability.
Age:
2–17 years
Study goal:
The main goal of this trial is to collect DNA from individuals with non-cancerous blood diseases and their family members. Researchers will use the DNA to study how genes cause and influence these diseases. All research data will be confidential.
Study goal:
The main goal of this study is to learn more about the effects (good and bad) of this treatment in children and young adults with high-risk blood disorders.
Age:
21 years old and younger
For questions or referrals, please email the BMF/MDS Clinic at BMFreferrals@stjude.org.