TY - JOUR
T1 - Discontinuation of imatinib in children with chronic myeloid leukaemia in sustained deep molecular remission
T2 - results of the STOP IMAPED study
AU - de Bruijn, Clara M A
AU - Millot, Frédéric
AU - Suttorp, Meinolf
AU - Borisevich, Marina
AU - Brons, Paul
AU - Lausen, Birgitte
AU - de Bont, Eveline S J M
N1 - © 2019 British Society for Haematology and John Wiley & Sons Ltd.
PY - 2019
Y1 - 2019
N2 - This international study aimed to assess the effect of imatinib discontinuation in paediatric patients with chronic myeloid leukaemia (CML) after deep molecular remission (DMR) had been achieved and maintained for at least 2 years. The primary endpoint of this analysis was the molecular relapse-free survival, estimated by the non-parametric Kaplan-Meier method. Major endpoint was the estimated rate of patients without molecular relapse at 6 months. Fourteen patients were enrolled; 4 patients maintained DMR with a follow-up of 24 (two patients), 34 and 66 months, respectively, whereas 10 patients relapsed. All molecular relapses occurred within 6 months (median 3 months, range 1-6) after imatinib discontinuation. The overall probability of maintaining DMR at 6 months was 28·6%. No parameters associated with molecular relapse could be identified. Keeping in mind the rarity of paediatric CML, which contributed to the small size of the cohort, our findings illustrate that imatinib cessation after sustained DMR is successful in only limited numbers of patients, whereas much higher rates are reported in adult patients. Further research is needed to extend the cohort of paediatric CML patients who might achieve treatment-free remission with an ideal prerequisite of predicting the occurrence of molecular relapse l after imatinib cessation.
AB - This international study aimed to assess the effect of imatinib discontinuation in paediatric patients with chronic myeloid leukaemia (CML) after deep molecular remission (DMR) had been achieved and maintained for at least 2 years. The primary endpoint of this analysis was the molecular relapse-free survival, estimated by the non-parametric Kaplan-Meier method. Major endpoint was the estimated rate of patients without molecular relapse at 6 months. Fourteen patients were enrolled; 4 patients maintained DMR with a follow-up of 24 (two patients), 34 and 66 months, respectively, whereas 10 patients relapsed. All molecular relapses occurred within 6 months (median 3 months, range 1-6) after imatinib discontinuation. The overall probability of maintaining DMR at 6 months was 28·6%. No parameters associated with molecular relapse could be identified. Keeping in mind the rarity of paediatric CML, which contributed to the small size of the cohort, our findings illustrate that imatinib cessation after sustained DMR is successful in only limited numbers of patients, whereas much higher rates are reported in adult patients. Further research is needed to extend the cohort of paediatric CML patients who might achieve treatment-free remission with an ideal prerequisite of predicting the occurrence of molecular relapse l after imatinib cessation.
U2 - https://doi.org/10.1111/bjh.15826
DO - https://doi.org/10.1111/bjh.15826
M3 - Article
C2 - 30843196
SN - 0007-1048
VL - 185
SP - 718
EP - 724
JO - British journal of haematology
JF - British journal of haematology
IS - 4
ER -