Bone Marrow Failure and Myelodysplastic Syndrome (MDS) Program in the
Dana-Farber/Boston Children's Cancer and Blood Disorders Center is recognized
as one of the nation’s best pediatric treatment and research programs for bone
marrow failure, MDS, and related conditions.
Our patients have access to
advanced diagnostic evaluations and treatments, including individually tailored stem cell
transplantation and ongoing clinical trials that are investigating new treatments.
Our program also has been designated a “Center of Excellence” from the MDS
Foundation – the only large pediatric center in the country awarded this
Our program has the expertise to diagnose and treat a wide range of disorders including:
We also specialize in the evaluation of undefined bone marrow
failure and MDS disorders and offer cutting edge diagnostic testing and
Children’s offers one of the largest and most renowned pediatric bone marrow
failure and MDS programs in the world. Here are a few of the reasons to choose
and accurate diagnosis of bone marrow failure or MDS allows optimal medical
management and treatment to improve the health of children, adolescents, and
young adults with these disorders. Dana-Farber/Boston Children's is one of
only a few centers in the country with hematopathologists specifically trained
to evaluate these disorders in children.
Our doctors use a variety of diagnostic tests, including blood tests, bone marrow aspiration and biopsy, chromosomal analysis, and the newest genetic tests available. We also provide MMC/DEB testing (also called chromosome breakage testing), which tests for mitomycin C (MMC) and diepoxybutane (DEB). This is the standard, definitive test for Fanconi anemia, a blood disorder that is characterized by an inability to repair DNA damage caused by agents like MMC and DEB.
on the type of bone marrow failure a child has, we offer a range of
comprehensive treatments, including:
Children's patients have access to the broadest set of pediatric and
hematologic expertise available. The breadth of our expertise allows us to assemble
a team of specialists to meet the specific needs of each child.
core of our treatment teams are pediatric hematologists and bone marrow failure
specialists, as well as pathologists with specialized training in pediatric
hematopathology, pediatric oncologists, geneticists, and nurse practitioners. Many
of our physicians are also active researchers, so our patients have access to
the very best and up-to-date diagnostic tests and treatments available.
From there, we build a team that is best suited
to carry out your child’s treatment plan. Sub-specialists may include
cardiologists, dental surgeons, endocrinologists, gastroenterologists,
pulmonologists, immunologists, hand surgeons, nephrologists, orthopedists, and
otolaryngologists – all with expertise with pediatric bone marrow failure and
Dana-Farber/Boston Children's has a long history of research and innovation in conditions
associated with bone marrow failure. Our basic science and clinical research
program offers unique access to clinical trials in which children can receive
the newest bone marrow failure diagnostic evaluations and treatments.
Our physicians and scientists are working on a number of
initiatives, including collecting blood and bone marrow from patients to better
understand the causes of bone marrow failure syndromes and MDS; exploring
possible genetic factors that guide medical care; collecting blood and bone
marrow to identify specific proteins that are faulty and how these defects
relate to clinical complications and outcomes; and clinical trials to improve stem
cell transplants for disorders such as dyskeratosis congenita. Another study
is testing whether unrelated donor stem cell transplants can provide better
long-term outcomes than traditional immunosuppressive therapy (IST) for children with severe
With the support of funding
from the National Institutes of Health, we established a nationwide
Pediatric Bone Marrow Failure and MDS Registry. This allows researchers to
collect clinical information and tissue samples to help better understand and
ultimately identify new therapies for these conditions. The registry also
offers second opinion pathology consultation as a resource for the hematology
We are also
leading members of the North American Pediatric Aplastic Anemia Consortium (NAPAAC) and
the Shwachman-Diamond Syndrome (SDS) Registry.
More about clinical trials
For many children with rare or hard-to-treat conditions, clinical trials
provide new options.