Author:
Kayser Sabine,Martínez-Cuadrón David,Hanoun Maher,Stölzel Friedrich,Gil Cristina,Reinhardt H. Christian,Aguiar Eliana,Schäfer-Eckart Kerstin,Burgues Juan Miguel Bergua,Steffen Björn,Bernal Teresa,Krause Stefan W.,Riaza Rosalía,Schliemann Christoph,Cervera Jose,Kaufmann Martin,Torres-Miñana Laura,Hänel Mathias,Acuña-Cruz Evelyn,Jost Edgar,Algarra Jesus Lorenzo,Crysandt Martina,Fransecky Lars,Cornago-Navascues Javier,Kraus Sabrina,Martinez-Lopez Joaquin,Einsele Hermann,Niemann Dirk,Neubauer Andreas,Seggewiß-Bernhardt Ruth,Scholl Sebastian,Klein Stefan A.,Schmid Christoph,Schaich Markus,Schmidt-Hieber Martin,Zukunft Sven,Ho Anthony D.,Platzbecker Uwe,Baldus Claudia D.,Müller-Tidow Carsten,Thiede Christian,Bornhäuser Martin,Serve Hubert,Levis Mark,Montesinos Pau,Röllig Christoph,Schlenk Richard F.
Abstract
We retrospectively studied 125 patients with acute myeloid leukemia and trisomy 4 (median age at diagnosis, 58 years; range, 16-77 years) treated between 2000 and 2019 within a multicenter study. Trisomy 4 was the sole abnormality in 28 (22%) patients and additional abnormalities were present in 97 (78%) patients. Twenty-two (22%) and 15 (15%) of 101 tested patients harbored NPM1 and FLT3-ITD mutations. Two (3%) of 72 tested patients had double CEBPA mutations. Data on response to intensive anthracycline-based induction therapy were available for 119 patients. Complete remission was achieved in 67% (n=80) and the early death rate was 5% (n=6). Notably, patients with trisomy 4 as sole abnormality had a complete remission rate of 89%. Allogeneic hematopoietic cell transplantation was performed in 40 (34%) patients, of whom 19 were transplanted in first complete remission. The median follow-up of the intensively treated cohort was 5.76 years (95% confidence interval [95% CI]: 2.99-7.61 years). The 5-year overall survival and relapse-free survival rates were 30% (95% CI: 22-41%) and 27% (95% CI: 18-41%), respectively. An Andersen-Gill regression model on overall survival revealed that favorable-risk according to the European LeukemiaNet classification (hazard ratio [HR]=0.34; P=0.006) and trisomy 4 as sole abnormality (HR=0.41; P=0.01) were favorable factors, whereas age with a difference of 10 years (HR=1.15; P=0.11), female gender (HR=0.74; P=0.20) and allogeneic hematopoietic cell transplantation (HR=0.64; P=0.14) did not have an significant impact. In our cohort, patients with trisomy 4 as their sole abnormality had a high complete remission rate and favorable clinical outcome. Allogeneic hematopoietic cell transplantation did not seem to improve overall survival.
Publisher
Ferrata Storti Foundation (Haematologica)