Chemotherapy Before HSCT

In an attempt to cytoreduce leukemic burden, the combination of fludarabine and cytarabine with isotretinoin alone was given for two cycles before planned HSCT in 34 of 87 evaluable children with JMML in the Children's Oncology Group (COG) AAML0122 trial.[3]

In an attempt to cytoreduce leukemic burden, the combination of fludarabine and cytarabine with isotretinoin alone was given for two cycles before planned HSCT in 34 of 87 evaluable children with JMML in the Children's Oncology Group (COG) AAML0122 trial.[3]

In this group, the overall response rate (partial response [PR] and complete response [CR]) was 68%. However, achieving a CR before HSCT did not significantly improve overall survival, event-free survival (EFS), or relapse risk.

A phase II, single-arm, open-label trial included 18 children with newly diagnosed JMML who received single-agent azacitidine, given for 7 days in 28-day cycles.[4] Partially based on this trial, the U.S. Food and Drug Administration expanded the approved indications for azacitidine to include children with newly diagnosed JMML.

A phase II, single-arm, open-label trial included 18 children with newly diagnosed JMML who received single-agent azacitidine, given for 7 days in 28-day cycles.[4] Partially based on this trial, the U.S. Food and Drug Administration expanded the approved indications for azacitidine to include children with newly diagnosed JMML.

The study found that 61% of patients had partial remissions after three cycles of treatment.

A phase II, single-arm, open-label trial included 18 children with newly diagnosed JMML who received single-agent azacitidine, given for 7 days in 28-day cycles.[4] Partially based on this trial, the U.S. Food and Drug Administration expanded the approved indications for azacitidine to include children with newly diagnosed JMML.

Responding patients, defined using the International JMML response criteria,[5] tended to be younger and had low-to-medium methylation classifications.

A phase II, single-arm, open-label trial included 18 children with newly diagnosed JMML who received single-agent azacitidine, given for 7 days in 28-day cycles.[4] Partially based on this trial, the U.S. Food and Drug Administration expanded the approved indications for azacitidine to include children with newly diagnosed JMML.

Six patients became platelet-transfusion independent, and all responders had reductions in splenomegaly.

A phase II, single-arm, open-label trial included 18 children with newly diagnosed JMML who received single-agent azacitidine, given for 7 days in 28-day cycles.[4] Partially based on this trial, the U.S. Food and Drug Administration expanded the approved indications for azacitidine to include children with newly diagnosed JMML.

Seventeen of the 18 patients received an HSCT at a median of 5.5 months after diagnosis. Of these 17 patients, 14 remained leukemia-free at last follow-up (median, 23.8 months after HSCT).[4]