TY - JOUR
T1 - Front-line imatinib treatment in children and adolescents with chronic myeloid leukemia
T2 - results from a phase III trial
AU - Suttorp, Meinolf
AU - Schulze, Philipp
AU - Glauche, Ingmar
AU - Göhring, Gudrun
AU - von Neuhoff, Nils
AU - Metzler, Markus
AU - Sedlacek, Petr
AU - de Bont, Eveline S J M
AU - Balduzzi, Adriana
AU - Lausen, Birgitte
AU - Aleinikova, Olga
AU - Sufliarska, Sabina
AU - Henze, Günter
AU - Strauss, Gabriele
AU - Eggert, Angelika
AU - Kremens, Bernhard
AU - Groll, Andreas H
AU - Berthold, Frank
AU - Klein, Christoph
AU - Groß-Wieltsch, Ute
AU - Sykora, Karl Walter
AU - Borkhardt, Arndt
AU - Kulozik, Andreas E
AU - Schrappe, Martin
AU - Nowasz, Christina
AU - Krumbholz, Manuela
AU - Tauer, Josephine T
AU - Claviez, Alexander
AU - Harbott, Jochen
AU - Kreipe, Hans H
AU - Schlegelberger, Brigitte
AU - Thiede, Christian
PY - 2018/7
Y1 - 2018/7
N2 - A total of 156 patients (age range 1.3-18.0 years, median 13.2 years; 91 (58.3%) male) with newly diagnosed CML (N = 146 chronic phase (CML-CP), N = 3 accelerated phase (CML-AP), N = 7 blastic phase (CML-BP)) received imatinib up-front (300, 400, 500 mg/m2, respectively) within a prospective phase III trial. Therapy response, progression-free survival, causes of treatment failure, and side effects were analyzed in 148 children and adolescents with complete data. Event-free survival rate by 18 months for patients in CML-CP (median follow-up time 25 months, range: 1-120) was 97% (95% CI, 94.2-99.9%). According to the 2006 ELN-criteria complete hematologic response by month 3, complete cytogenetic response (CCyR) by month 12, and major molecular response (MMR) by month 18 were achieved in 98, 63, and 59% of the patients, respectively. By month 36, 86% of the patients achieved CCyR and 74% achieved MMR. Thirty-eight patients (27%) experienced imatinib failure because of unsatisfactory response or intolerance (N = 9). In all, 28/148 patients (19%) underwent stem cell transplantation (SCT). In the SCT sub-cohort 2/23 patients diagnosed in CML-CP, 0/1 in CML-AP, and 2/4 in CML-BP, respectively, died of relapse (N = 3) or SCT-related complications (N = 2). This large pediatric trial extends and confirms data from smaller series that first-line imatinib in children is highly effective.
AB - A total of 156 patients (age range 1.3-18.0 years, median 13.2 years; 91 (58.3%) male) with newly diagnosed CML (N = 146 chronic phase (CML-CP), N = 3 accelerated phase (CML-AP), N = 7 blastic phase (CML-BP)) received imatinib up-front (300, 400, 500 mg/m2, respectively) within a prospective phase III trial. Therapy response, progression-free survival, causes of treatment failure, and side effects were analyzed in 148 children and adolescents with complete data. Event-free survival rate by 18 months for patients in CML-CP (median follow-up time 25 months, range: 1-120) was 97% (95% CI, 94.2-99.9%). According to the 2006 ELN-criteria complete hematologic response by month 3, complete cytogenetic response (CCyR) by month 12, and major molecular response (MMR) by month 18 were achieved in 98, 63, and 59% of the patients, respectively. By month 36, 86% of the patients achieved CCyR and 74% achieved MMR. Thirty-eight patients (27%) experienced imatinib failure because of unsatisfactory response or intolerance (N = 9). In all, 28/148 patients (19%) underwent stem cell transplantation (SCT). In the SCT sub-cohort 2/23 patients diagnosed in CML-CP, 0/1 in CML-AP, and 2/4 in CML-BP, respectively, died of relapse (N = 3) or SCT-related complications (N = 2). This large pediatric trial extends and confirms data from smaller series that first-line imatinib in children is highly effective.
U2 - 10.1038/s41375-018-0179-9
DO - 10.1038/s41375-018-0179-9
M3 - Journal article
C2 - 29925908
SN - 0887-6924
VL - 32
SP - 1657
EP - 1669
JO - Leukemia
JF - Leukemia
IS - 7
ER -