MSK is one of the world’s leading centers for treating children and adults with bone marrow failure. Our program is one of the oldest programs of its kind, internationally recognized for advancing care and research. We provide access to cutting-edge treatments for bone marrow failure and other cancer predisposition (genetic) syndromes. Our experts care for people with bone marrow failure at every stage, from diagnosis to treatment and long-term monitoring. At MSK, you have a team of experts all working together to care for you.
Contact MSK’s Inherited Bone Marrow Failure Program
Our specialists see children and adults with bone marrow failure, or those who think they may have bone marrow failure. Make an appointment today.
What is bone marrow failure?
Bone marrow failure happens when the body doesn’t make enough red blood cells, white blood cells, or platelets. Blood cells and platelets are made in your bone marrow, which is the soft, spongy tissue found inside bones. Blood cells and platelets carry oxygen to your body parts through your blood. Oxygen in the blood helps to fight infections and heal wounds. Having bone marrow failure can increase your risk of getting cancer.
The 2 types of bone marrow failure are:
- Inherited. This type is caused by a genetic change (mutation or variant) that is passed down from a parent to a child or can happen spontaneously.
- Acquired. This type may happen over time. Sometimes as a side effect of treatment for another condition or from other factors in life that may affect your health. There are also times when the cause is not known.
What is the MSK Inherited Bone Marrow Failure Program?
Inherited bone marrow failure disorders are rare, and every case is unique. It’s important to get care from a team that has expertise in treating all forms of the disease.
MSK’s Inherited Bone Marrow Failure Program has skilled and supportive experts to care for adults and children with inherited bone marrow failure. Even if you suspect you may have bone marrow failure, we can help. We bring together experts in blood disorders, stem cell transplantation, genetic testing and counseling, cancer surveillance, and more. Our team takes a comprehensive approach to your care, which means we treat the medical issue and support your overall health.
Though inherited bone marrow failure is more common in children, adults can also have it. Inherited bone marrow failure disorders we treat include:
- Fanconi anemia
- Congenital amegakaryocytic thrombocytopenia (CAMT)
- Diamond-Blackfan anemia (DBA)
- GATA2 deficiency
- Shwachman Diamond syndrome
- Telomere biology disorders, such as Dyskeratosis congenita
- Thrombocytopenia absent radii (TAR Syndrome)
- SAMD9/SAMD9L-associated predisposition to myeloid neoplasms
- Other bone marrow failure syndromes
Who is this program for?
MSK’s Inherited Bone Marrow Failure Program is for adults, adolescents, and children with suspected or diagnosed bone marrow failure disorders. Our doctors can also see adults and children if they have:
- Unexplained or very low levels of red blood cells, white blood cells, or platelets (cytopenias).
- Early-onset (starting at a younger age than usual) myelodysplastic syndromes or acute myeloid leukemia.
- Early-onset head and neck cancers.
- Unexplained increased toxicity to chemotherapy or radiation.
What services does MSK’s Inherited Bone Marrow Failure Program offer?
Diagnosis and genetic testing
When you have bone marrow failure, it’s important to know which kind so you can get the best treatment. Our program provides:
- Accurate diagnosis of bone marrow failure using genetic tests.
- Genetic counseling to address concerns about you or your family’s risk of cancer. You can learn more about genetic testing and counseling at MSK.
Treatment and management
Our team creates a tailored plan to treat you with fewer side effects and manage your disease. MSK provides multidisciplinary care. This means different specialists, such as doctors, nurses, genetic counselors and other experts work together as one team to support all aspects of your health. Treatment from our multidisciplinary team for inherited bone marrow failure disorders may include:
- Active surveillance (watching over time). This is also called watchful waiting or watch-and-wait.
- A stem cell transplant.
- Blood transfusions.
- Chemotherapy.
- Medications that suppress the immune system.
- Other new and cutting-edge therapies (as available through clinical trials).
People with inherited bone marrow failure often need long-term monitoring (management) and other types of support. This is because they are at high risk of getting certain cancers or other blood disorders. Long-term monitoring from our multidisciplinary team may include:
- Regular check-ups.
- A cancer screening schedule to check for signs of cancer early.
- Ongoing follow-up care, which means getting treatment when you need it or a diagnosis if you’re having new symptoms.
- Treating side effects, if needed.
- Mental health support.
Clinical trials and other research
MSK is a leader in bone marrow failure treatment and research. We offer patients access to clinical trials that test new treatments, often before they are available anywhere else. Talk with our experts about whether a clinical trial is right for you.
MSK is also recognized as a Fanconi Anemia Care Center by the Fanconi Cancer Foundation. Only programs that have expertise in diagnosing, treating, and monitoring this rare inherited condition have the recognition.
MSK lab research on inherited bone marrow failure
MSK is leading the way in Fanconi anemia research and treatment. Learn more about how MSK’s pioneering research and collaborations with scientists at Rockefeller University are deepening our understanding of the condition.
MSK’s Inherited Bone Marrow Failure experts
Hematologist oncologists specializing in blood and bone marrow disorders
- María Cancio, MD, Pediatric Hematologist-Oncologist & Bone Marrow Transplant Specialist
- Rekha Parameswaran, MD, Hematologist-Oncologist
- Jaap-Jan Boelens, MD, PhD, Pediatric Hematologist-Oncologist & Bone Marrow Transplant Specialist
- Alexandre G. Troullioud Lucas, MD, MSc, Pediatric Hematologist-Oncologist
- Roni Tamari, MD, Adult Bone Marrow Transplant Specialist
Head and neck surgeons
- Richard J. Wong, MD, FACS, Head and Neck Cancer Surgeon
- Babak Givi, MD, MHPE, Head and Neck Cancer Surgeon
Genetic counselors and specialists
- Chimene Kesserwan, MD, Clinical Geneticist and pathologist
- Lauren Banaszak, MD, Leukemia Specialist
- Jennifer Kennedy, MS, CGC, Genetic Counselor
- Alicia Latham, MD, MS, Clinical Geneticist and Family Medicine Physician
Psychiatrist
- Julia A. Kearney, MD, Pediatric Psychiatrist
Endocrinologist
- Zoltan Antal, MD, Pediatric Endocrinologist
Fertility
- Lauren Martino, MSN, RN, CPNP, Fertility Advanced Practice Provider
- Anne Casson, MSN, RN, CNP, Pediatric and Adolescent and Young Adult Fertility Advanced Practice Provider
Adolescent and Young Adult Transition
- Emily Sarro, MSN, RN, FNP-BC, CPHON, Adolescent and Young Adult Nurse Practitioner
Gastrointestinal specialists
- Ghassan K. Abou-Alfa, MD, JD, MBA, PhD (hc), Gastrointestinal Medical Oncologist
- Karen Queliza, MD, Pediatric Gastroenterologist
- Stanley Cho, MD, Pediatric Gastroenterologist
Dermatologist
- Alina Markova, MD, Dermatologist
Gynecologic surgeon
- Deborah J. Goldfrank, MD, FACOG, Gynecologic Surgeon
Research
- Jennifer Bieler, BSN, RN, CPHON, Clinical Trials Nurse, MSK Kids Stem Cell Transplant and Cellular Therapies Service
Common questions
If I receive treatment for bone marrow failure at MSK and get cancer later in life, can my MSK doctor continue to care for me?
Yes. Our program is designed to provide care for you at any stage of bone marrow failure. This includes treatment for future cancer if you get it. Children who are treated for bone marrow failure can keep seeing MSK experts into their adult life.