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Bone Marrow Failure and Acute Leukemia: More Mutations, More Problems

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Constitutional aplastic anemia Fanconi anemia, dyskeratosis congenita. We perform a complete medical work-up of all patients, including blood work for genetic and other tests, bone marrow testing, and tests specific to each of the inherited bone marrow failure syndromes. Management of these diseases includes periodic check-ups, blood work, and other testing. Our multidisciplinary care team includes experts in endocrinology, head and neck oncology , gynecology, and orthopedics to monitor for any additional health problems.

We base the treatment for each child on his or her current condition. Therefore, treatments can vary, and include supportive care, blood transfusions, blood growth factors, immune-based medications, chelation therapy, and allogeneic hematopoietic stem cell transplantation HSCT , which involves the replenishment of blood-forming stem cells from a donor. Around the world, there are patient registries for some of the inherited bone marrow failure syndromes to keep track of all patients with a particular diagnosis, study features of the disease, and record the treatment and outcomes of patients to inform future treatments.

Investigators at Memorial Sloan Kettering are in contact with these registries as well as with investigators worldwide to stay up-to-date about the latest research studies and treatments for the different disorders. Our experts have also been involved in developing clinical care guidelines, such as indications for Fanconi anemia. Though inherited bone marrow failure syndromes themselves cannot be cured, the failure of bone marrow to produce the required blood cells can be treated and cured, most often with allogeneic stem cell transplantation. The timing of the treatment depends on whether a stem cell or bone marrow match is found as well as the age of your child children under age 10 tend to have better outcomes.

An allogeneic stem cell transplant is also referred to as a peripheral blood stem cell transplant, bone marrow transplant, or cord blood transplant, depending on the origin of the transplanted cells. The particular type of transplantation used to treat your child depends on the type of syndrome. Regardless of the type of transplantation, the goals are the same:. Our investigators have been performing stem cell transplants in young patients with bone marrow failure syndromes for decades.

One of our major advantages is that we are able to perform transplants from any type of donor — related or unrelated, matched or mismatched.

Inherited Bone Marrow Failure Syndromes Study Literature

The advantages of our program are that for a large number of patients we are able to perform transplants without using total body irradiation. The actual stem cell transplant typically takes place a day or two after conditioning is completed. The donor stem cells or bone marrow are given to your child through a catheter or central venous line inserted in the chest or the arm into large veins close to the heart, in a process similar to that of a blood transfusion.

The procedure is painless and takes between five and 20 minutes. We ask our patients to remain in the New York area to receive care for about three to six months.

After that time, children can return to their primary doctor for most of their care. After one year post transplant, the patient returns for an appointment each year with the internationally recognized experts on our pediatric long-term follow-up team or adult long-term follow-up team.

Inherited Bone Marrow Failure Syndromes (IBMFS)

Some young patients can subsist with borderline blood counts that do not require therapy for many weeks, months, or even years. When treatment is necessary, therapies for managing Fanconi anemia can include transfusions of red blood cells and platelets, and oral administration of male hormones known as androgens to help increase the blood counts. If a child develops a dependence on transfusions or develops a blood cancer such as leukemia or myelodysplastic syndrome , a stem cell transplant is necessary and is the only curative treatment.

Memorial Sloan Kettering is one of the leading centers in the United States and is known for its success in performing allogeneic stem cell transplants in children with Fanconi anemia. The process can be difficult because young patients with Fanconi anemia have genetic defects that result in fragile DNA and chromosomes that break easily. Through a careful combination of transplantation and supportive care measures designed by our experts, many children with Fanconi anemia can begin to produce healthy blood cells.

Bone Marrow Failure Disorders Program - The Johns Hopkins Kimmel Cancer Center

These high numbers of stem cells help prevent the child from rejecting the transplant. Our center has been the leading institution of a multicenter trial for transplantation of patients with Fanconi anemia. This trial has involved five different centers and has been recognized and partially funded by the Fanconi Anemia Research Fund. Children with dyskeratosis congenita have a number of organ issues or damage. They are also at risk for developing aplastic anemia, myelodysplastic syndrome , or acute myelogenous leukemia.

Patients are often treated with stem cell transplantation. However, we recognize that children with this disease have especially fragile skin, lungs, and livers.


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  • Inherited Bone Marrow Failure Syndromes (IBMFS)?

We aim to cure the bone marrow failure and blood disorder with transplantation, but at the same time minimize the risks of both graft-versus-host disease that can affect these organs and toxicity to these organs from the chemotherapy delivered prior to transplantation. We have transplanted several patients with this disease using this approach — low dose chemotherapy and T-cell depleted transplant — and the results have been promising.