Front-line imatinib treatment in children and adolescents with chronic myeloid leukemia: results from a phase III trial
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 I...
Saved in:
| Main Authors: | , |
|---|---|
| Format: | Article (Journal) |
| Language: | English |
| Published: |
20 June 2018
|
| In: |
Leukemia
Year: 2018, Volume: 32, Issue: 7, Pages: 1657-1669 |
| ISSN: | 1476-5551 |
| DOI: | 10.1038/s41375-018-0179-9 |
| Online Access: | Verlag, Volltext: https://doi.org/10.1038/s41375-018-0179-9 Verlag, Volltext: https://www.nature.com/articles/s41375-018-0179-9 
|
| Author Notes: | Meinolf Suttorp, Philipp Schulze, Ingmar Glauche, Gudrun Göhring, Nils von Neuhoff, Markus Metzler, Petr Sedlacek, Eveline S. J. M. de Bont, Adriana Balduzzi, Birgitte Lausen, Olga Aleinikova, Sabina Sufliarska, Günter Henze, Gabriele Strauss, Angelika Eggert, Bernhard Kremens, Andreas H. Groll, Frank Berthold, Christoph Klein, Ute Groß-Wieltsch, Karl Walter Sykora, Arndt Borkhardt, Andreas E. Kulozik, Martin Schrappe, Christina Nowasz, Manuela Krumbholz, Josephine T. Tauer, Alexander Claviez, Jochen Harbott, Hans H. Kreipe, Brigitte Schlegelberger, Christian Thiede |
| Summary: | 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. |
|---|---|
| Item Description: | Gesehen am 30.04.2019 |
| Physical Description: | Online Resource |
| ISSN: | 1476-5551 |
| DOI: | 10.1038/s41375-018-0179-9 |