TY - JOUR T1 - Childhood high-risk acute lymphoblastic leukemia in first remission: results after chemotherapy or transplant from the AIEOP ALL 2000 study. JF - Blood Y1 - 2014 A1 - Conter, Valentino A1 - Valsecchi, Maria Grazia A1 - Parasole, Rosanna A1 - Putti, Maria Caterina A1 - Locatelli, Franco A1 - Barisone, Elena A1 - Lo Nigro, Luca A1 - Santoro, Nicola A1 - Aricò, Maurizio A1 - Ziino, Ottavio A1 - Pession, Andrea A1 - Testi, Anna Maria A1 - Micalizzi, Concetta A1 - Casale, Fiorina A1 - Zecca, Marco A1 - Casazza, Gabriella A1 - Tamaro, Paolo A1 - La Barba, Gaetano A1 - Notarangelo, Lucia Dora A1 - Silvestri, Daniela A1 - Colombini, Antonella A1 - Rizzari, Carmelo A1 - Biondi, Andrea A1 - Masera, Giuseppe A1 - Basso, Giuseppe KW - Adolescent KW - Antineoplastic Combined Chemotherapy Protocols KW - Child KW - Child, Preschool KW - Combined Modality Therapy KW - Female KW - Hematopoietic Stem Cell Transplantation KW - Humans KW - Infant KW - Male KW - Neoplasm, Residual KW - Precursor Cell Lymphoblastic Leukemia-Lymphoma KW - Radiotherapy KW - Remission Induction KW - Treatment Outcome AB -

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.

VL - 123 IS - 10 U1 - http://www.ncbi.nlm.nih.gov/pubmed/24415536?dopt=Abstract ER -