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Outcome of children with acute myeloid leukaemia ( AML ) experiencing primary induction failure in the AIEOP AML 2002/01 clinical trial
Author(s) -
Quarello Paola,
Fagioli Franca,
Basso Giuseppe,
Putti Maria C.,
Berger Massimo,
Luciani Matteo,
Rizzari Carmelo,
Menna Giuseppe,
Masetti Riccardo,
Locatelli Franco
Publication year - 2015
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.13611
Subject(s) - medicine , hematopoietic stem cell transplantation , refractory (planetary science) , transplantation , disease , myeloid leukaemia , clinical trial , population , induction chemotherapy , chemotherapy , oncology , physics , environmental health , astrobiology
Summary Paediatric patients with acute myeloid leukaemia ( AML ) who fail induction due to primary resistance to chemotherapy account for a significant proportion of cases and have a particularly dismal prognosis. We report the clinical and biological data, and final outcome of 48 paediatric patients with primary‐resistant AML enrolled in the Associazione Italiana di Ematologia e Oncologia Pediatrica AML 2002/01 clinical trial. These patients had a significantly higher white blood cell count at diagnosis compared to other AML patients. Cytogenetic and molecular features did not differ between patients with primary induction failure and patients allocated to the high‐risk group. For the whole patient population, the probability of overall survival, event‐free survival ( EFS ) and disease‐free survival ( DFS ) was 21·8% ± 6·2, 20·4% ± 5·9, and 49·5% ± 11·3, respectively. Twenty‐eight (58%) patients received haematopoietic stem cell transplantation ( HSCT ); 3 were autologous and 25 were allogeneic. Patients who underwent HSCT had improved EFS (31·2% vs. 5%, P < 0·0001). Only one of the 20 patients who did not receive HSCT is alive and disease free. The 19 patients in complete remission at time of HSCT showed significantly better DFS than the 9 with active disease ( 46% vs. 0%, P = 0·02). This study represents one of the largest series with long‐term follow up of paediatric AML patients with primary refractory disease. Children who underwent transplantation had an encouraging long‐term outcome. Disease recurrence remains the major cause of treatment failure; a better understanding of the disease biology is desirable to develop more effective treatment strategies.