Experience with ponatinib in paediatric patients with leukaemia.
Philadelphia chromosome
acute lymphoblastic leukaemia
chronic myeloid leukaemia
paediatrics
ponatinib
Journal
British journal of haematology
ISSN: 1365-2141
Titre abrégé: Br J Haematol
Pays: England
ID NLM: 0372544
Informations de publication
Date de publication:
04 2020
04 2020
Historique:
received:
23
06
2019
accepted:
21
08
2019
pubmed:
25
1
2020
medline:
15
12
2020
entrez:
25
1
2020
Statut:
ppublish
Résumé
Ponatinib has proven to be effective in adults with Philadelphia chromosome-positive leukaemias, but data in paediatrics are scarce. Among paediatric patients with chronic myeloid leukaemia (n = 9) or acute lymphoblastic leukaemia (n = 12) treated with varying doses of ponatinib in 13 centres, 71% showed a decrease in disease burden after a median of three months. Ponatinib was well tolerated, with grade 3 toxicities occurring in 29% of patients. Toxicities were similar to those reported in adults, with the exception of arterial thrombotic events, which were not observed. Ponatinib has a favourable safety profile in this paediatric cohort, but dose-finding studies are needed.
Substances chimiques
Antineoplastic Agents
0
Imidazoles
0
Pyridazines
0
ponatinib
4340891KFS
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
363-368Informations de copyright
© 2020 British Society for Haematology and John Wiley & Sons Ltd.
Références
Champagne, M.A., Fu, C.H., Chang, M., Chen, H., Gerbing, R.B., Alonzo, T.A., Cooley, L.D., Heerema, N.A., Oehler, V., Wood, C., French, M.E., Arceci, R.J., Smith, F.O. & Bernstein, M.L. (2011) Higher dose imatinib for children with de novo chronic phase chronic myelogenous leukemia: a report from the Children's Oncology Group. Pediatric Blood & Cancer, 57, 56-62.
Cortes, J.E., Kim, D.W., Pinilla-Ibarz, J., le Coutre, P., Paquette, R., Chuah, C., Nicolini, F.E., Apperley, J.F., Khoury, H.J., Talpaz, M., DiPersio, J., DeAngelo, D.J., Abruzzese, E., Rea, D., Baccarani, M., Müller, M.C., Gambacorti-Passerini, C., Wong, S., Lustgarten, S., Rivera, V.M., Clackson, T., Turner, C.D., Haluska, F.G., Guilhot, F., Deininger, M.W., Hochhaus, A., Hughes, T., Goldman, J.M., Shah, N.P., Kantarjian, H.;PACE Investigators. (2013) A phase 2 trial of ponatinib in Philadelphia chromosome-positive leukemias. New England Journal of Medicine, 369, 1783-1796.
Cortes, J.E., Kim, D.-W., Pinilla-Ibarz, J., Le Coutre, P., Paquette, R., Chuah, C., Nicolini, F.E., Apperley, J.F., Khoury, H.J., Talpaz, M., DiPersio, J.F., DeAngelo, D.J., Abruzzese, E., Rea, D., Baccarani, M., Müller, M.C., Gambacorti-Passerini, C., Lustgarten, S., Rivera, V.M., Clackson, T., Turner, C.D., Haluska, F.G., Guilhot, F., Deininger, M.W., Hochhaus, A., Hughes, T.P., Shah, N.P. & Kantarjian, H.M. (2014) Long-term follow-up of ponatinib efficacy and safety in the phase 2 PACE trial. Blood, 124, 3135.
Gore, L., Kearns, P.R., de Martino Lee, M.L., De Souza, C.A., Bertrand, Y., Hijiya, N., Stork, L.C., Chung, N.-G., Cardenas-Cardos, R., Saikia, T., Fagioli, F., Seo, J.J., Landman-Parker, J., Lancaster, D., Place, A.E., Rabin, K.R., Sacchi, M., Swanink, R. & Zwaan, C.M. (2018) Dasatinib in pediatric patients with chronic myeloid leukemia in chronic phase: results from a phase II trial. Journal of Clinical Oncology, 36, 1330-1338.
Gruner, S., Sayeed, H., Suarez-Ferguson, L., Fisher, K., Miller, M., Rouce, R. & Rau, R. (2018) Ponatinib use in pediatric/adolescent and young adult patients with relapsed Ph+ ALL: a case series and review of the literature. Pediatric Blood & Cancer, 65, S59-S60.
Hijiya, N. & Suttorp, M. (2019) How I treat chronic myeloid leukemia in children and adolescents. Blood, 133, 2374-2384.
Hijiya, N., Maschan, A., Rizzari, C., Shimada, H., Dufour, C., Goto, H., Kang, H.J., Guinipero, T., Karakas, Z., Bautista, F., Ducassou, S., Yoo, K.H., Zwaan, C.M., Millot, F., Aimone, P., Allepuz, A., Quenez, S., Hourcade-Potelleret, F., Hertle, S., & Sosothikul, D. (2019) Phase 2 study of nilotinib in pediatric patients with Philadelphia chromosome-positive chronic myeloid leukemia. Blood, 134, 2036-2045.
Jabbour, E. & Kantarjian, H. (2016) Chronic myeloid leukemia: 2016 update on diagnosis, therapy, and monitoring. American Journal of Hematology, 91, 252-65.
Jain, P., Kantarjian, H., Boddu, P.C., Nogueras-González, G.M., Verstovsek, S., Garcia-Manero, G., Borthakur, G., Sasaki, K., Kadia, T.M., Sam, P., Ahaneku, H., O’Brien, S., Estrov, Z., Ravandi, F., Jabbour, E. & Cortes, J.E. (2019) Analysis of cardiovascular and arteriothrombotic adverse events in chronic-phase CML patients after frontline TKIs. Blood Advances, 3, 851-861.
Lipton, J.H., Chuah, C., Guerci-Bresler, A., Rosti, G., Simpson, D., Assouline, S., Etienne, G., Nicolini, F.E., le Coutre, P., Clark, R.E., Stenke, L., Andorsky, D., Oehler, V., Lustgarten, S., Rivera, V.M., Clackson, T., Haluska, F.G., Baccarani, M., Cortes, J.E., Guilhot, F., Hochhaus, A., Hughes, T., Kantarjian, H.M., Shah, N.P., Talpaz, M. & Deininger, M.W. (2016) Ponatinib versus imatinib for newly diagnosed chronic myeloid leukaemia: an international, randomised, open-label, phase 3 trial. The Lancet Oncology, 17, 612-621.
Medeiros, B.C., Possick, J. & Fradley, M. (2018) Cardiovascular, pulmonary, and metabolic toxicities complicating tyrosine kinase inhibitor therapy in chronic myeloid leukemia: Strategies for monitoring, detecting, and managing. Blood Reviews, 32, 289-299.
Nickel, R.S., Daves, M. & Keller, F. (2015) Treatment of an adolescent with chronic myeloid leukemia and the T315I mutation with ponatinib. Pediatric Blood & Cancer, 62, 2050-2051.
Samis, J., Lee, P., Zimmerman, D., Arceci, R.J., Suttorp, M. & Hijiya, N. (2016) Recognizing endocrinopathies associated with tyrosine kinase inhibitor therapy in children with chronic myelogenous leukemia. Pediatric Blood & Cancer, 63, 1332-1338.
Schultz, K.R., Carroll, A., Heerema, N.A., Bowman, W.P., Aledo, A., Slayton, W.B., Sather, H., Devidas, M., Zheng, H.W., Davies, S.M., Gaynon, P.S., Trigg, M., Rutledge, R., Jorstad, D., Winick, N., Borowitz, M.J., Hunger, S.P., Carroll, W.L. & Camitta, B. (2014) Long-term follow-up of imatinib in pediatric Philadelphia chromosome-positive acute lymphoblastic leukemia: Children's Oncology Group study AALL0031. Leukemia, 28, 1467-1471.
Yamamoto, M., Hori, T., Igarashi, K., Shimada, H. & Tsutsumi, H. (2018) Response to ponatinib before hematopoietic stem cell transplantation in a child with relapsed Philadelphia chromosome-positive acute lymphoblastic leukemia. Pediatrics International, 60, 85-87.