Efficacy and safety of ponatinib for patients with Philadelphia chromosome-positive acute lymphoblastic leukemia: a case series from a single institute.

Szczegóły
Abstrakt

Tytuł:: Efficacy and safety of ponatinib for patients with Philadelphia chromosome-positive acute lymphoblastic leukemia: a case series from a single institute.
Autorzy:: Kidoguchi K; Division of Hematology, Respiratory Medicine and Oncology, Department of Internal Medicine, Faculty of Medicine, Saga University, 5-1-1 Nabeshima, Saga, 849-8501, Japan.
Ureshino H; Division of Hematology, Respiratory Medicine and Oncology, Department of Internal Medicine, Faculty of Medicine, Saga University, 5-1-1 Nabeshima, Saga, 849-8501, Japan. .
Kizuka-Sano H; Division of Hematology, Respiratory Medicine and Oncology, Department of Internal Medicine, Faculty of Medicine, Saga University, 5-1-1 Nabeshima, Saga, 849-8501, Japan.
Yamaguchi K; Division of Hematology, Respiratory Medicine and Oncology, Department of Internal Medicine, Faculty of Medicine, Saga University, 5-1-1 Nabeshima, Saga, 849-8501, Japan.
Katsuya H; Division of Hematology, Respiratory Medicine and Oncology, Department of Internal Medicine, Faculty of Medicine, Saga University, 5-1-1 Nabeshima, Saga, 849-8501, Japan.
Kubota Y; Division of Hematology, Respiratory Medicine and Oncology, Department of Internal Medicine, Faculty of Medicine, Saga University, 5-1-1 Nabeshima, Saga, 849-8501, Japan.; Department of Transfusion Medicine, Saga University Hospital, Saga, Japan.
Ando T; Division of Hematology, Respiratory Medicine and Oncology, Department of Internal Medicine, Faculty of Medicine, Saga University, 5-1-1 Nabeshima, Saga, 849-8501, Japan.
Miura M; Department of Pharmacy, Akita University Hospital, Akita, Japan.
Takahashi N; Department of Hematology, Nephrology and Rheumatology, Akita University Graduate School of Medicine, Akita, Japan.
Kimura S; Division of Hematology, Respiratory Medicine and Oncology, Department of Internal Medicine, Faculty of Medicine, Saga University, 5-1-1 Nabeshima, Saga, 849-8501, Japan.
Źródło:: International journal of hematology [Int J Hematol] 2021 Aug; Vol. 114 (2), pp. 199-204. Date of Electronic Publication: 2021 Apr 27.
Typ publikacji:: Case Reports; Journal Article
Język:: English
Imprint Name(s):: Publication: 2008- : Tokyo : Springer Japan
Original Publication: Amsterdam : Elsevier Science Publishers, c1991-
MeSH Terms:: Fusion Proteins, bcr-abl/*antagonists & inhibitors
Imidazoles/*therapeutic use
Precursor Cell Lymphoblastic Leukemia-Lymphoma/*drug therapy
Protein Kinase Inhibitors/*therapeutic use
Pyridazines/*therapeutic use
Aged ; Antineoplastic Combined Chemotherapy Protocols/adverse effects ; Antineoplastic Combined Chemotherapy Protocols/therapeutic use ; Combined Modality Therapy ; Disease Management ; Female ; Fusion Proteins, bcr-abl/genetics ; Humans ; Imidazoles/administration & dosage ; Imidazoles/adverse effects ; Male ; Middle Aged ; Molecular Targeted Therapy/methods ; Philadelphia Chromosome ; Precursor Cell Lymphoblastic Leukemia-Lymphoma/diagnosis ; Precursor Cell Lymphoblastic Leukemia-Lymphoma/genetics ; Protein Kinase Inhibitors/administration & dosage ; Protein Kinase Inhibitors/adverse effects ; Pyridazines/administration & dosage ; Pyridazines/adverse effects ; Retrospective Studies ; Treatment Outcome
References:: Chan LC, Karhi KK, Rayter SI, Heisterkamp N, Eridani S, Powles R, et al. A novel abl protein expressed in Philadelphia chromosome positive acute lymphoblastic leukaemia positive acute lymphoblastic leukaemia. Nature. 1987;325:635–7. (PMID: 10.1038/325635a0)
Gleissner B, Gökbuget N, Bartram CR, Janssen B, Rieder H, Janssen JW, et al. Leading prognostic relevance of the BCR-ABL translocation in adult acute B-lineage lymphoblastic leukemia: a prospective study of the German Multicenter Trial Group and confirmed polymerase chain reaction analysis. Blood. 2002;99:1536–43. (PMID: 10.1182/blood.V99.5.1536)
Ottmann OG, Druker BJ, Sawyers CL, Goldman JM, Reiffers J, Silver RT, et al. A phase 2 study of imatinib in patients with relapsed or refractory Philadelphia chromosome-positive acute lymphoid leukemias. Blood. 2002;100:1965–71. (PMID: 10.1182/blood-2001-12-0181)
Fielding AK, Rowe JM, Buck G, Foroni L, Gerrard G, Litzow MR, et al. UKALLXII/ECOG2993: addition of imatinib to a standard treatment regimen enhances long-term outcomes in Philadelphia positive acute lymphoblastic leukemia. Blood. 2014;123:843–50. (PMID: 10.1182/blood-2013-09-529008)
Mian AA, Schüll M, Zhao Z, Oancea C, Hundertmark A, Beissert T, et al. The gatekeeper mutation T315I confers resistance against small molecules by increasing or restoring the ABL-kinase activity accompanied by aberrant transphosphorylation of endogenous BCR, even in loss-of-function mutants of BCR/ABL. Leukemia. 2009;23:1614–21. (PMID: 10.1038/leu.2009.69)
Jabbour E, Short NJ, Ravandi F, Huang X, Daver N, DiNardo CD, et al. Combination of hyper-CVAD with ponatinib as first-line therapy for patients with Philadelphia chromosome-positive acute lymphoblastic leukaemia: long-term follow-up of a single-centre, phase 2 study. Lancet Haematol. 2018;5:e618–27. (PMID: 10.1016/S2352-3026(18)30176-5)
Moslehi JJ, Deininger M. Tyrosine kinase inhibitor-associated cardiovascular toxicity in chronic myeloid leukemia. J Clin Oncol. 2015;33:4210–8. (PMID: 10.1200/JCO.2015.62.4718)
Tojo A, Kyo T, Yamamoto K, Nakamae H, Takahashi N, Kobayashi Y, et al. Ponatinib in Japanese patients with Philadelphia chromosome-positive leukemia, a phase 1/2 study. Int J Hematol. 2017;106:385–97. (PMID: 10.1007/s12185-017-2238-9)
Tanaka R, Kimura S, Ashihara E, Yoshimura M, Takahashi N, Wakita H, et al. Rapid automated detection of ABL kinase domain mutations in imatinib-resistant patients. Cancer Lett. 2011;312:228–34. (PMID: 10.1016/j.canlet.2011.08.009)
Rousselot P, Coudé MM, Gokbuget N, Gambacorti Passerini C, Hayette S, Cayuela JM, et al. Dasatinib and low-intensity chemotherapy in elderly patients with Philadelphia chromosome-positive ALL. Blood. 2016;128:774–82. (PMID: 10.1182/blood-2016-02-700153)
Sasaki K, Jabbour EJ, Ravandi F, Short NJ, Thomas DA, Garcia-Manero G, et al. Hyper-CVAD plus ponatinib versus hyper-CVAD plus dasatinib as frontline therapy for patients with Philadelphia chromosome-positive acute lymphoblastic leukemia: a propensity score analysis. Cancer. 2016;122:3650–6. (PMID: 10.1002/cncr.30231)
Cortes JE, Kim DW, Pinilla-Ibarz J, le Coutre PD, Paquette R, Chuah C, et al. Ponatinib efficacy and safety in Philadelphia chromosome-positive leukemia: final 5-year results of the phase 2 PACE trial. Blood. 2018;132:393–404. (PMID: 10.1182/blood-2016-09-739086)
Cortes JE, Lomaia E, Turkina A, Turkina A, Moiraghi B, Undurraga M, et al. Interim analysis (IA) of OPTIC: A dose-ranging study of three ponatinib (PON) starting doses. J Clin Oncol. 2020;38:7502–7502. (PMID: 10.1200/JCO.2020.38.15_suppl.7502)
Abumiya M, Miura M, Takahashi N. Therapeutic drug monitoring of ponatinib using a simple high-performance liquid chromatography method in Japanese patients. Leuk Res. 2018;64:42–5. (PMID: 10.1016/j.leukres.2017.11.012)
Webster JA, Luznik L, Tsai HL, Imus PH, DeZern AE, Pratz KW, et al. Allogeneic transplantation for Ph+ acute lymphoblastic leukemia with posttransplantation cyclophosphamide. Blood Adv. 2020;4:5078–88. (PMID: 10.1182/bloodadvances.2020002945)
Copland M. Is there a role for dose modification of TKI therapy in CML? Curr Hematol Malig Rep. 2019;14:337–45. (PMID: 10.1007/s11899-019-00524-w)
Caldemeyer L, Dugan M, Edwards J, Akard L, Dugan M, Edwards J. Long-term side effects of tyrosine kinase inhibitors in chronic myeloid leukemia. Curr Hematol Malig Rep. 2016;11:71–9. (PMID: 10.1007/s11899-016-0309-2)
Hochhaus A, Cortes JE, Kim DW, Pinilla-Ibarz J, le Coutre PD, Paquette R, et al. Efficacy and safety of ponatinib following failure of dasatinib in patients (pts) with chronic phase chronic myeloid leukemia (CP-CML) in the PACE trial. Blood. 2013;122:1498. (PMID: 10.1182/blood.V122.21.1498.1498)
Porkka K, Koskenvesa P, Lundán T, Rimpiläinen J, Mustjoki S, Smykla R, et al. Dasatinib crosses the blood-brain barrier and is an efficient therapy for central nervous system philadelphia chromosome positive leukemia. Blood. 2008;112:1005–12. (PMID: 10.1182/blood-2008-02-140665)
Lai SW, Huang TC, Chen JH, Wu YY, Chang PY. Dasatinib as the salvage therapy for chronic myeloid leukemia with blast crisis and central nervous system involvement: A case report. Oncol Lett. 2015;9:1957–61. (PMID: 10.3892/ol.2015.2928)
Abid MB, De Mel S. Does ponatinib cross the blood-brain barrier? Br J Haematol. 2017;179:497–8. (PMID: 10.1111/bjh.14222)
Masuda K, Nakazato T, Nishiyama-Fujita Y, Ito C, Ogura S, Mizuno K, et al. Successful treatment with ponatinib for central nervous system relapse of Philadelphia chromosome-positive B-cell acute lymphoblastic leukaemia. Intern Med J. 2019;49:1332–4. (PMID: 10.1111/imj.14451)
O’Hare T, Shakespeare WC, Zhu X, Eide CA, Rivera VM, Wang F, et al. AP24534, a pan-BCR-ABL inhibitor for chronic myeloid leukemia, potently inhibits the T315I mutant and overcomes mutation-based resistance. Cancer Cell. 2009;16:401–12. (PMID: 10.1016/j.ccr.2009.09.028)
Gaur S, Torbi AR, Corral J. Isolated central nervous system relapse in two patients with BCR-ABL-positive acute leukemia while receiving a next-generation tyrosine kinase inhibitor. Vivo. 2014;28:1149–53.
Lazarus HM, Richards SM, Chopra R, Litzow MR, Burnett AK, Wiernik PH, et al. Central nervous system involvement in adult acute lymphoblastic leukemia at diagnosis: results from the international ALL trial MRC UKALL XII/ECOG E2993. Blood. 2006;108:465–72. (PMID: 10.1182/blood-2005-11-4666)
Contributed Indexing:: Keywords: ABL1 tyrosine kinase inhibitor; Acute lymphoblastic leukemia; Philadelphia chromosome; Ponatinib; T315I mutation
Substance Nomenclature:: 0 (Imidazoles)
0 (Protein Kinase Inhibitors)
0 (Pyridazines)
4340891KFS (ponatinib)
EC 2.7.10.2 (Fusion Proteins, bcr-abl)
Entry Date(s):: Date Created: 20210428 Date Completed: 20210809 Latest Revision: 20210809
Update Code:: 20240104
DOI:: 10.1007/s12185-021-03156-0
PMID:: 33907977
: Czasopismo naukowe

Full Text Finder

Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph + ALL) is an aggressive leukemia that occurs in 20-40% of adult patients. Ph + ALL is caused by the Philadelphia chromosome (Ph), which consists of a t(9;22)(q34;q11) reciprocal translocation leading to the formation of a BCR-ABL1 fusion gene. The disease is treated with targeted therapy comprising ABL1 tyrosine kinase inhibitors (TKIs). Ponatinib is a third generation TKI that demonstrates higher binding affinity for ABL1 than first/second generation TKIs. Although intensive combined immunotherapy with ponatinib greatly improves the prognosis of Ph + ALL, the safety and efficacy profiles of ponatinib in Japanese patients are unclear. This retrospective study investigated five cases of Ph + ALL at a single institute to evaluate safety and efficacy profiles. Three patients achieved a deep molecular response (DMR) following combined intensive treatment with ponatinib as induction chemotherapy. Four patients received consolidative allogenic stem cell transplantation (allo-SCT) during their first complete response. Three of the four experienced early relapse within 100 days; they subsequently received ponatinib, and one of the three achieved a DMR. No patient experienced severe cardiovascular events. This case series suggests that ponatinib at a concentration of least 30 mg exhibits anti-leukemia effects in Japanese patients with Ph + ALL.
(© 2021. Japanese Society of Hematology.)

Informacja