Bone marrow transplant may be answer to incurable, fatal blood disease

September 4, 2014 | by Darrin Joy

Even its name is daunting. Systemic mastocytosis is a fatal disease of the blood with no known cure. But a new study suggests a bone marrow transplant may be the answer for some patients.

Nakamura and Pullarkat Systemic mastocytosis is currently considered a fatal blood disorder. City of Hope researchers Ryotaro Nakamura, left, and Vinod Pullarkat have found that bone marrow transplantation could offer a cure.

While rare, systemic mastocytosis is resistant to treatment with drugs and, when aggressive, can be fatal within four years of diagnosis. No standard therapy currently exists for the disease, which is caused by an overabundance of mast cells in the blood and bone marrow.

Mast cells are a type of white blood cell that normally helps in wound healing and in defending against infectious disease agents. The cells also are involved in allergic reactions including anaphylaxis, a severe reaction that can sometimes lead to death.

Patients with mastocytosis tend to develop hives and itching and are prone to anaphylactic shock. They also can develop other blood cancers such as leukemia and myelodysplasia together with systemic mastocytosis.

A multinational, multi-institutional group of researchers that included associate professors Ryotaro Nakamura, M.D., and Vinod Pullarkat, M.D., in the Department of Hematology & Hematopoietic Cell Transplantation, found that many patients with systemic mastocytosis responded favorably to allogeneic bone marrow transplantation (transplant using blood stem cells from a donor).

Study first author Celalettin Ustun, M.D., from the University of Minnesota, and the researchers examined the records of nearly 60 mastocytosis patients who underwent transplantation and found that almost three-quarters had a favorable response. More than a quarter went into complete remission. Overall, the three-year survival rate was 57 percent.

While the results are promising, the study is retrospective, meaning it examined data from the records of patients treated in the past. The researchers cautioned that more study is needed, including a clinical trial specifically designed to test the benefits of transplantation for systemic mastocytosis patients.

“We would like to see a prospective clinical study to see if our findings hold up to more scrutiny,” said Nakamura. “If they do, we might finally have a way to improve outcomes for these patients.”

The systemic mastocytosis study appears online in the Journal of Clinical Oncology.

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