title: Outcome of children with acute myeloid leukaemia (AML) experiencing primary induction failure in the AIEOP AML 2002/01 clinical trial.
abstract: 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.
authors.affiliation: Paediatric Onco-Haematology, Stem Cell Transplantation and Cellular Therapy Division, Regina Margherita Children's Hospital, Torino, Italy.
authors.forename: Paola
authors.initials: P
authors.lastname: Quarello
authors.affiliation: Paediatric Onco-Haematology, Stem Cell Transplantation and Cellular Therapy Division, Regina Margherita Children's Hospital, Torino, Italy.
authors.forename: Franca
authors.initials: F
authors.lastname: Fagioli
authors.affiliation: Department of Woman and Child Health, Haemato-Oncology Division, University of Padova, Azienda Ospedale Padova, Padova, Italy.
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Basso
authors.affiliation: Department of Woman and Child Health, Haemato-Oncology Division, University of Padova, Azienda Ospedale Padova, Padova, Italy.
authors.forename: Maria C
authors.initials: MC
authors.lastname: Putti
authors.affiliation: Paediatric Onco-Haematology, Stem Cell Transplantation and Cellular Therapy Division, Regina Margherita Children's Hospital, Torino, Italy.
authors.forename: Massimo
authors.initials: M
authors.lastname: Berger
authors.affiliation: Department of Paediatric Haematology-Oncology, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Bambino Gesù Children's Hospital, Roma, Italy.
authors.forename: Matteo
authors.initials: M
authors.lastname: Luciani
authors.affiliation: Department of Paediatric Haematology, San Gerardo Hospital, Monza, Italy.
authors.forename: Carmelo
authors.initials: C
authors.lastname: Rizzari
authors.affiliation: Department of Paediatric Haemato-Oncology, Santobono-Pausilipon Hospital, Napoli, Italy.
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Menna
authors.affiliation: Paediatric Oncology and Haematology Unit "Lalla Seragnoli", Department of Paediatrics, University of Bologna Sant'Orsola-Malpighi Hospital, Bologna, Italy.
authors.forename: Riccardo
authors.initials: R
authors.lastname: Masetti
authors.affiliation: Department of Paediatric Haematology-Oncology, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Bambino Gesù Children's Hospital, Roma, Italy.|University of Pavia, Pavia, Italy.
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
mesh_terms: D000293:Adolescent; D064591:Allografts; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000453:epidemiology; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D015994:Incidence; D007223:Infant; D007231:Infant, Newborn; D053208:Kaplan-Meier Estimate; D015470:Leukemia, Myeloid, Acute / Q000188:drug therapy* / Q000401:mortality / Q000628:therapy; D008297:Male; D011379:Prognosis; D012008:Recurrence; D012074:Remission Induction; D019172:Transplantation Conditioning; D014182:Transplantation, Autologous; D017211:Treatment Failure
keywords: acute myeloid leukaemia; childhood leukaemia; induction failure; relapse; stem cell transplantation