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Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 19, 2013
Intensive therapy for children with acute lymphoblastic leukaemia and unfavourable presenting features. Early
P S Gaynon1, P G Steinherz, W A Bleyer
1Wyler Children's Hospital, Chicago.
Insights
Intensive chemotherapy regimens significantly improved event-free survival for children with high-risk acute lymphoblastic leukaemia (ALL). These new treatments reduced treatment failure by 2.7 times compared to the control regimen.
Area of Science:
- Pediatric Oncology
- Hematology
- Clinical Trials
Background:
- Children with acute lymphoblastic leukaemia (ALL) and high-risk features historically faced poor treatment outcomes.
- Previous studies indicated a 3-year event-free survival (EFS) of 47% for this patient group before 1981.
Purpose of the Study:
- To evaluate the efficacy of two intensive chemotherapy regimens (Berlin-Frankfurt-Munster [BFM] 76/79 and New York [NY]) compared to a standard control regimen.
- To determine if intensive therapies improve event-free survival in high-risk pediatric ALL patients.
Main Methods:
- A randomized study involving 229 children diagnosed with high-risk acute lymphoblastic leukaemia.
- Comparison of three treatment regimens: BFM 76/79, NY, and a control regimen.
- Analysis of data from 214 patients (93.4% of the total cohort).
Main Results:
- The 3-year event-free survival (EFS) was significantly higher for the intensive BFM and NY regimens (78%) compared to the control regimen (49%).
- These survival differences remained consistent across various patient subgroups, including age, sex, white blood cell count, and bone marrow blast morphology.
- Patients receiving the control regimen were 2.7 times more likely to experience induction failure, death, or relapse than those on intensive regimens.
Conclusions:
- Intensive chemotherapy regimens, specifically the BFM 76/79 and NY protocols, demonstrate superior efficacy in improving event-free survival for children with high-risk acute lymphoblastic leukaemia.
- These findings support the use of intensive treatment strategies to enhance outcomes for pediatric patients with ALL who are at high risk of treatment failure.
Abstract:
229 children with acute lymphoblastic leukaemia (ALL) and with clinical and laboratory features associated with a high risk of treatment failure entered a randomised study of three treatment regimens. Before 1981, such patients had a 3-year event-free survival (EFS) of 47%. Two intensive therapies, the Berlin-Frankfurt-Munster (BFM) 76/79 regimen and the New York (NY) regimen were compared with a control regimen that had achieved the best outcome in previous Trials. Data on 214 cases (93.4%) were analysed. The 3-year EFS was 78% for the BFM and NY regimens and 49% for the control regimen, a significant difference. The differences persisted after stratification by age at onset, sex, white blood cell count at diagnosis, and marrow blast morphology. Control patients were 2.7 times more likely to fail induction, to die, or to relapse than were patients on the intensive regimens.
