Childhood high-risk acute lymphoblastic leukemia in first remission: results after chemotherapy or transplant from the AIEOP ALL 2000 study
Valentino Conter, Maria Grazia Valsecchi, Rosanna Parasole, Maria Caterina Putti, Franco Locatelli, Elena Barisone, Luca Lo Nigro, Nicola Santoro, Maurizio Aricò, Ottavio Ziino, Andrea Pession, Anna Maria Testi, Concetta Micalizzi, Fiorina Casale, Marco Zecca, Gabriella Casazza, Paolo Tamaro, Gaetano La Barba, Lucia Dora Notarangelo, Daniela Silvestri, Antonella Colombini, Carmelo Rizzari, Andrea Biondi, Giuseppe Masera, Giuseppe Basso, Valentino Conter, Maria Grazia Valsecchi, Rosanna Parasole, Maria Caterina Putti, Franco Locatelli, Elena Barisone, Luca Lo Nigro, Nicola Santoro, Maurizio Aricò, Ottavio Ziino, Andrea Pession, Anna Maria Testi, Concetta Micalizzi, Fiorina Casale, Marco Zecca, Gabriella Casazza, Paolo Tamaro, Gaetano La Barba, Lucia Dora Notarangelo, Daniela Silvestri, Antonella Colombini, Carmelo Rizzari, Andrea Biondi, Giuseppe Masera, Giuseppe Basso
Abstract
The outcome of high-risk (HR) acute lymphoblastic leukemia patients enrolled in the AIEOP-BFM ALL 2000 study in Italy is described. HR criteria were minimal residual disease (MRD) levels ≥10(-3) at day 78 (MRD-HR), no complete remission (CR) at day 33, t(4;11) translocation, and prednisone poor response (PPR). Treatment (2 years) included protocol I, 3 polychemotherapy blocks, delayed intensification (protocol IIx2 or IIIx3), cranial radiotherapy, and maintenance. A total of 312 HR patients had a 5-year event-free survival (EFS) of 58.9% (standard error [SE] = 2.8) and an overall survival of 68.9% (SE = 2.6). In hierarchical order, EFS was 45.9% (4.4) in 132 MRD-HR patients, 41.2% (11.9) in 17 patients with no CR at day 33, 36.4% (14.5) in 11 patients with t(4;11), and 74.0% (3.6) in 152 HR patients only for PPR. No statistically significant difference was found for disease-free survival in patients with very HR features [MRD-HR, no CR at day 33, t(4;11) translocation], given hematopoietic stem cell transplantation (HSCT) (n = 66) or chemotherapy only (n = 88), after adjusting for waiting time to HSCT (5.7 months). Patients at HR only for PPR have a favorable outcome. MRD-HR is associated with poor outcome despite intensive treatment and/or HSCT and may qualify for innovative therapies. The study was registered at www.clinicaltrials.gov as #NCT00613457.
Source: PubMed