This phase 2 study evaluates the sequential combination of decitabine then midostaurin for the treatment of newly-diagnosed acute myeloid leukemia (AML) in older patients.
Treatment with decitabine, a cytidine analog, then midostaurin, a multi-target protein kinase inhibitor (PKI), may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
13
Given IV
Given PO
Stanford University School of Medicine
Stanford, California, United States
Complete Remission (CR) Rate
The complete remission (CR) rate, or complete response rate, is reported as the sum and proportion of participants that achieved CR or CR with incomplete blood count recovery (CRi), within 12 months of starting midostaurin treatment. * Complete remission (CR): Bone marrow blasts \< 5%; absence of blasts with Auer rods; absence of extramedullary disease; absolute neutrophil count (ANC) \> 1000/μL; platelet count \> 100,000/μL; independence of red cell transfusions. * CR with incomplete recovery (CRi): All CR criteria except for ANC \< 1000/μL or platelet count \< 100,000/μL. * Partial remission (PR): All hematologic criteria of CR; except decrease of bone marrow blast percentage to 5% to 25%; and decrease of pretreatment bone marrow blast percentage by at least 50%.
Time frame: Up to 1 year
Overall Response Rate (ORR)
Overall response rate (ORR) was assessed as the number and proportion of participants who received midostaurin and achieved a partial response (PR), complete response (CR), or complete response with incomplete blood count recovery (CRi). * Complete remission (CR): Bone marrow blasts \< 5%; absence of blasts with Auer rods; absence of extramedullary disease; absolute neutrophil count (ANC) \> 1000/μL; platelet count \> 100,000/μL; independence of red cell transfusions. * CR with incomplete recovery (CRi): All CR criteria except for ANC \< 1000/μL or platelet count \< 100,000/μL. * Partial remission (PR): All hematologic criteria of CR; except decrease of bone marrow blast percentage to 5% to 25%; and decrease of pretreatment bone marrow blast percentage by at least 50%.
Time frame: up to 1 year
Median Duration of Response (DoR)
Response was assessed by evaluations conducted every 3 cycles (12 weeks). Once documented as partial response (PR), complete response (CR), or complete response with incomplete blood count recover (CRi), response status was confirmed every 12 weeks. In responding participants, duration of response was assessed from the start of treatment through the last documented response before documented progressive disease or death. The outcome is reported as the median value for duration of response, with full range. * CR: Bone marrow blasts \< 5%; absence of blasts with Auer rods; absence of extramedullary disease; ANC \> 1000/μL; platelet \> 100,000/μL; independence of red cell transfusions. * CRi: All CR criteria except ANC \< 1000/μL or platelet count \< 100,000/μL. * PR: All hematologic criteria of CR; except decrease of bone marrow blast percentage to 5% to 25%; \& decrease of pretreatment bone marrow blast percentage by at least 50%.
Time frame: Up to 1 year
Progression-free Survival (PFS)
Progression-free survival (PFS) is reported as the number and proportion of participants who did not receive hematopoietic cell transplantation, and who did not experience disease progression or death for any reason within 2 years after starting midostaurin treatment. Progressive disease: Bone marrow blasts ≥ 5%; or reappearance of blasts in the blood; or development of extramedullary disease.
Time frame: Up to 2 years
Overall Survival (OS)
Survival is reported as the number and proportion of participants that received midostaurin who remained alive 2 years after starting midostaurin treatment.
Time frame: Up to 2 years
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