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Baffling High-Risk Leukemia Solved

Published: 2018-09-12 |

Source: St. Jude Children's Research Hospital

St. Jude Children's Research Hospital investigators have unraveled the origins and identified mutations with a perplexing form of acute leukemia, The landmark study appears today as an advance online publication in the journal Nature and lays the foundation for more effective treatment of patients with the high-risk cancer.

The research focused on mixed phenotype acute leukemia (MPAL), a subtype of acute leukemia that accounts for about 3 percent of the estimated 3,500 pediatric cases of acute leukemia diagnosed annually in the U.S. MPAL also occurs in adults. Their treatment is complicated because MPAL does not fit cleanly into a single diagnosis, but includes features of both acute lymphoblastic leukemia and acute myeloid leukemia, These markers, which help determine treatment, sometimes change with time or treatment, in some cases enough to change the diagnosis from MPAL to AML or vise versa.

"ALL and AML have very different treatments. But MPAL has features of both, so the question of how best to treat patients with MPAL has been challenging the leukemia community worldwide--and long-term survival of patients has been poor, " said Charles Mullighan, MBBS, M.D., a member of the St. Jude Department of Pathology. He and Hiroto Inaba, M.D., Ph.D., an associate member of the St. Jude Department of Oncology, are the study's corresponding authors. Long-term survival for young MPAL patients is 47 to 75 percent, compared to more than 90 percent for young ALL patients and 65 to 75 percent for AML.

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