TREATMENT OUTCOME OF CHILDREN WITH DE NOVO AML GROUPED BASED ON RISK FEATURES AND TREATMENT RESPONSE
(Abstract release date: 05/21/15)
EHA Library. YOO K. 06/12/15; 102713; PB1650
Disclosure(s): SAMSUNG MEDICAL CENTER, SUNGKYUNKWAN UNIVERSITY SCHOOL OF MEDICINEPediatrics
KEON HEE YOO
Contributions
Contributions
Abstract
Abstract: PB1650
Type: Publication Only
Background
Approximately 80-100 children are newly diagnosed as AML in Korea. The survival rate of pediatric AML has improved due to intensified well-designed chemotherapy, better supportive care, risk-based treatment adaptation and allogeneic HSCT. However, relapse still remains the leading cause of events, which accounts for 30 to 40%. Because pediatric AML is a heterogeneous disease and only some subgroup of patients benefits from allogeneic transplantation in 1st CR, risk-adapted treatment strategy is needed.
Aims
This multicenter pilot study was aimed to evaluate the feasibility of new treatment protocol based on risk stratification for newly diagnosed pediatric de novo acute myeloid leukemia (AML).
Methods
Patients with acute promyelocytic leukemia and AML of Down syndrome were excluded. Risk features were defined as follows: 1) low risk features (LRF): inv(16)/t(16;16), t(8;21) without c-kit mutation, or with normal karyotype in the presence of NPM1 or CEBPA mutation without FLT3/ITD mutation; 2) high risk features (HRF): -5, 5q-, -7, 3q abnormalities, t(8;16), t(6;9), t(16;21), t(6;11), t(10;11), AMKL without t(1;22), complex karyotype, or FLT3/ITD mutation; 3) standard risk features (SRF): all the others. A new chemotherapy regimen was developed which consisted of 2 induction and 4 consolidation cycles. Treatment responses were determined according to the marrow response at the end of each induction as good response (CR-CR), delayed response-1 (PR [blast 5~15%]-CR), delayed response-2 (NR [blast>15%]-CR), refractory (PR/NR-PR/NR), and early relapse. Patients were allocated into either favorable (FG), intermediate (IG), or poor (PG) prognostic group considering both risk features and treatment response. Patients in FG were not allocated to transplantation.
Results
Fifty-seven patients were enrolled. There were 13 patients (22.8%) with LRF, 22 (38.6%) with SRF, and 22 (38.6%) with HRF. The overall CR rate following 2 cycles of induction was 87.5%. One patient (1.8%) died during induction therapy. Eight patients relapsed of whom 6 were in PG, 1 in IG, and 1 in FG. Twenty-three patients underwent allo-HSCT. With a median follow-up of 13 mo, the estimated 2-y overall survival and event-free survival of all patients were 91% and 71%, respectively.
Summary
Although follow-up duration is short, our new treatment protocol based on risk stratification seems effective.
Keyword(s): Acute myeloid leukemia, Children, Outcome
Session topic: Publication Only
Type: Publication Only
Background
Approximately 80-100 children are newly diagnosed as AML in Korea. The survival rate of pediatric AML has improved due to intensified well-designed chemotherapy, better supportive care, risk-based treatment adaptation and allogeneic HSCT. However, relapse still remains the leading cause of events, which accounts for 30 to 40%. Because pediatric AML is a heterogeneous disease and only some subgroup of patients benefits from allogeneic transplantation in 1st CR, risk-adapted treatment strategy is needed.
Aims
This multicenter pilot study was aimed to evaluate the feasibility of new treatment protocol based on risk stratification for newly diagnosed pediatric de novo acute myeloid leukemia (AML).
Methods
Patients with acute promyelocytic leukemia and AML of Down syndrome were excluded. Risk features were defined as follows: 1) low risk features (LRF): inv(16)/t(16;16), t(8;21) without c-kit mutation, or with normal karyotype in the presence of NPM1 or CEBPA mutation without FLT3/ITD mutation; 2) high risk features (HRF): -5, 5q-, -7, 3q abnormalities, t(8;16), t(6;9), t(16;21), t(6;11), t(10;11), AMKL without t(1;22), complex karyotype, or FLT3/ITD mutation; 3) standard risk features (SRF): all the others. A new chemotherapy regimen was developed which consisted of 2 induction and 4 consolidation cycles. Treatment responses were determined according to the marrow response at the end of each induction as good response (CR-CR), delayed response-1 (PR [blast 5~15%]-CR), delayed response-2 (NR [blast>15%]-CR), refractory (PR/NR-PR/NR), and early relapse. Patients were allocated into either favorable (FG), intermediate (IG), or poor (PG) prognostic group considering both risk features and treatment response. Patients in FG were not allocated to transplantation.
Results
Fifty-seven patients were enrolled. There were 13 patients (22.8%) with LRF, 22 (38.6%) with SRF, and 22 (38.6%) with HRF. The overall CR rate following 2 cycles of induction was 87.5%. One patient (1.8%) died during induction therapy. Eight patients relapsed of whom 6 were in PG, 1 in IG, and 1 in FG. Twenty-three patients underwent allo-HSCT. With a median follow-up of 13 mo, the estimated 2-y overall survival and event-free survival of all patients were 91% and 71%, respectively.
Summary
Although follow-up duration is short, our new treatment protocol based on risk stratification seems effective.
Keyword(s): Acute myeloid leukemia, Children, Outcome
Session topic: Publication Only
Abstract: PB1650
Type: Publication Only
Background
Approximately 80-100 children are newly diagnosed as AML in Korea. The survival rate of pediatric AML has improved due to intensified well-designed chemotherapy, better supportive care, risk-based treatment adaptation and allogeneic HSCT. However, relapse still remains the leading cause of events, which accounts for 30 to 40%. Because pediatric AML is a heterogeneous disease and only some subgroup of patients benefits from allogeneic transplantation in 1st CR, risk-adapted treatment strategy is needed.
Aims
This multicenter pilot study was aimed to evaluate the feasibility of new treatment protocol based on risk stratification for newly diagnosed pediatric de novo acute myeloid leukemia (AML).
Methods
Patients with acute promyelocytic leukemia and AML of Down syndrome were excluded. Risk features were defined as follows: 1) low risk features (LRF): inv(16)/t(16;16), t(8;21) without c-kit mutation, or with normal karyotype in the presence of NPM1 or CEBPA mutation without FLT3/ITD mutation; 2) high risk features (HRF): -5, 5q-, -7, 3q abnormalities, t(8;16), t(6;9), t(16;21), t(6;11), t(10;11), AMKL without t(1;22), complex karyotype, or FLT3/ITD mutation; 3) standard risk features (SRF): all the others. A new chemotherapy regimen was developed which consisted of 2 induction and 4 consolidation cycles. Treatment responses were determined according to the marrow response at the end of each induction as good response (CR-CR), delayed response-1 (PR [blast 5~15%]-CR), delayed response-2 (NR [blast>15%]-CR), refractory (PR/NR-PR/NR), and early relapse. Patients were allocated into either favorable (FG), intermediate (IG), or poor (PG) prognostic group considering both risk features and treatment response. Patients in FG were not allocated to transplantation.
Results
Fifty-seven patients were enrolled. There were 13 patients (22.8%) with LRF, 22 (38.6%) with SRF, and 22 (38.6%) with HRF. The overall CR rate following 2 cycles of induction was 87.5%. One patient (1.8%) died during induction therapy. Eight patients relapsed of whom 6 were in PG, 1 in IG, and 1 in FG. Twenty-three patients underwent allo-HSCT. With a median follow-up of 13 mo, the estimated 2-y overall survival and event-free survival of all patients were 91% and 71%, respectively.
Summary
Although follow-up duration is short, our new treatment protocol based on risk stratification seems effective.
Keyword(s): Acute myeloid leukemia, Children, Outcome
Session topic: Publication Only
Type: Publication Only
Background
Approximately 80-100 children are newly diagnosed as AML in Korea. The survival rate of pediatric AML has improved due to intensified well-designed chemotherapy, better supportive care, risk-based treatment adaptation and allogeneic HSCT. However, relapse still remains the leading cause of events, which accounts for 30 to 40%. Because pediatric AML is a heterogeneous disease and only some subgroup of patients benefits from allogeneic transplantation in 1st CR, risk-adapted treatment strategy is needed.
Aims
This multicenter pilot study was aimed to evaluate the feasibility of new treatment protocol based on risk stratification for newly diagnosed pediatric de novo acute myeloid leukemia (AML).
Methods
Patients with acute promyelocytic leukemia and AML of Down syndrome were excluded. Risk features were defined as follows: 1) low risk features (LRF): inv(16)/t(16;16), t(8;21) without c-kit mutation, or with normal karyotype in the presence of NPM1 or CEBPA mutation without FLT3/ITD mutation; 2) high risk features (HRF): -5, 5q-, -7, 3q abnormalities, t(8;16), t(6;9), t(16;21), t(6;11), t(10;11), AMKL without t(1;22), complex karyotype, or FLT3/ITD mutation; 3) standard risk features (SRF): all the others. A new chemotherapy regimen was developed which consisted of 2 induction and 4 consolidation cycles. Treatment responses were determined according to the marrow response at the end of each induction as good response (CR-CR), delayed response-1 (PR [blast 5~15%]-CR), delayed response-2 (NR [blast>15%]-CR), refractory (PR/NR-PR/NR), and early relapse. Patients were allocated into either favorable (FG), intermediate (IG), or poor (PG) prognostic group considering both risk features and treatment response. Patients in FG were not allocated to transplantation.
Results
Fifty-seven patients were enrolled. There were 13 patients (22.8%) with LRF, 22 (38.6%) with SRF, and 22 (38.6%) with HRF. The overall CR rate following 2 cycles of induction was 87.5%. One patient (1.8%) died during induction therapy. Eight patients relapsed of whom 6 were in PG, 1 in IG, and 1 in FG. Twenty-three patients underwent allo-HSCT. With a median follow-up of 13 mo, the estimated 2-y overall survival and event-free survival of all patients were 91% and 71%, respectively.
Summary
Although follow-up duration is short, our new treatment protocol based on risk stratification seems effective.
Keyword(s): Acute myeloid leukemia, Children, Outcome
Session topic: Publication Only
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