TY - JOUR
T1 - The long-term efficacy and safety of nilotinib in pediatric patients with CML
T2 - a 5-year update of the DIALOG study
AU - Hijiya, Nobuko
AU - Maschan, Alexey
AU - Rizzari, Carmelo
AU - Shimada, Hiroyuki
AU - Dufour, Carlo
AU - Goto, Hiroaki
AU - Kang, Hyoung Jin
AU - Guinipero, Terri
AU - Karakas, Zeynep
AU - Bautista, Francisco
AU - Ducassou, Stéphane
AU - Yoo, Keon Hee
AU - Zwaan, Christian Michel
AU - Millot, Frédéric
AU - Patterson, Briana C.
AU - Samis, Jill
AU - Izquierdo, Miguel
AU - Titorenko, Ksenia
AU - Li, Sai
AU - Sosothikul, Darintr
N1 - Publisher Copyright:
© 2023 by The American Society of Hematology.
PY - 2023/12/1
Y1 - 2023/12/1
N2 - The efficacy and safety of nilotinib in pediatric patients with imatinib/dasatinib resistant/ intolerant (R/I) or newly diagnosed (ND) Philadelphia chromosome–positive (Ph+) chronic myeloid leukemia in chronic phase (CML-CP) was demonstrated in the phase 2, open-label DIALOG study. In this final analysis, long-term efficacy and safety are presented for patients who completed 66 cycles (of 28 days) of treatment with nilotinib (230 mg/m2 twice daily) or discontinued early. Overall, 59 patients were enrolled and 58 were treated (R/I, n = 33; ND, n = 25; median time on treatment: 60.5 and 51.9 months, respectively). In the R/I cohort, the cumulative major molecular response (MMR; BCR::ABL1 international scale [IS] ≤ 0.1%) rate was 60.6%, and no patients had a confirmed loss of MMR. Among ND patients, the best overall MMR rate was 76.0%; 3 patients had a confirmed loss of MMR. The cumulative molecular response MR4 (BCR::ABL1IS ≤ 0.01%) and MR4.5 (BCR::ABL1IS ≤ 0.0032%) rates by 66 cycles were 27.3% and 12.1% in the R/I cohort, and 56.0% and 44.0% in the ND cohort, respectively. The safety profile of nilotinib was consistent with those of earlier reports. No on-treatment deaths occurred. These long-term (up to ~5 years) data support the efficacy and safety of nilotinib in pediatric patients with Ph+ CML-CP. This trial was registered at www.clinicaltrials.gov.uk as #NCT01844765.
AB - The efficacy and safety of nilotinib in pediatric patients with imatinib/dasatinib resistant/ intolerant (R/I) or newly diagnosed (ND) Philadelphia chromosome–positive (Ph+) chronic myeloid leukemia in chronic phase (CML-CP) was demonstrated in the phase 2, open-label DIALOG study. In this final analysis, long-term efficacy and safety are presented for patients who completed 66 cycles (of 28 days) of treatment with nilotinib (230 mg/m2 twice daily) or discontinued early. Overall, 59 patients were enrolled and 58 were treated (R/I, n = 33; ND, n = 25; median time on treatment: 60.5 and 51.9 months, respectively). In the R/I cohort, the cumulative major molecular response (MMR; BCR::ABL1 international scale [IS] ≤ 0.1%) rate was 60.6%, and no patients had a confirmed loss of MMR. Among ND patients, the best overall MMR rate was 76.0%; 3 patients had a confirmed loss of MMR. The cumulative molecular response MR4 (BCR::ABL1IS ≤ 0.01%) and MR4.5 (BCR::ABL1IS ≤ 0.0032%) rates by 66 cycles were 27.3% and 12.1% in the R/I cohort, and 56.0% and 44.0% in the ND cohort, respectively. The safety profile of nilotinib was consistent with those of earlier reports. No on-treatment deaths occurred. These long-term (up to ~5 years) data support the efficacy and safety of nilotinib in pediatric patients with Ph+ CML-CP. This trial was registered at www.clinicaltrials.gov.uk as #NCT01844765.
UR - http://www.scopus.com/inward/record.url?scp=85179609439&partnerID=8YFLogxK
U2 - 10.1182/bloodadvances.2023010122
DO - 10.1182/bloodadvances.2023010122
M3 - Article
C2 - 37738125
AN - SCOPUS:85179609439
SN - 2473-9529
VL - 7
SP - 7279
EP - 7289
JO - Blood Advances
JF - Blood Advances
IS - 23
ER -