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Suttorp, Meinolf; Schulze, Philipp; Glauche, Ingmar; Göhring, Gudrun; Neuhoff, Nils von; Metzler, Markus; Sedlacek, Petr; Bont, Eveline S. J. M. de; Balduzzi, Adriana; Lausen, Birgitte; Aleinikova, Olga; Sufliarska, Sabina; Henze, Günter; Strauss, Gabriele; Eggert, Angelika; Kremens, Bernhard; Groll, Andreas H.; Berthold, Frank; Klein, Christoph; Gross-Wieltsch, Ute; Sykora, Karl Walter; Borkhardt, Arndt; Kulozik, Andreas E.; Schrappe, Martin; Nowasz, Christina; Krumbholz, Manuela; Tauer, Josephine T.; Claviez, Alexander; Harbott, Jochen; Kreipe, Hans H.; Schlegelberger, Brigitte; Thiede, Christian (2018): Front-line imatinib treatment in children and adolescents with chronic myeloid leukemia: results from a phase III trial. In: Leukemia, Vol. 32, No. 7: pp. 1657-1669
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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 upfront (300, 400, 500 mg/m(2), 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.