Management and outcome of patients with chronic myeloid leukemia in blast phase in the tyrosine kinase inhibitor era - analysis of the European LeukemiaNet Blast Phase Registry
Jazyk angličtina Země Velká Británie, Anglie Médium print-electronic
Typ dokumentu časopisecké články, práce podpořená grantem
PubMed
38548962
PubMed Central
PMC11073984
DOI
10.1038/s41375-024-02204-y
PII: 10.1038/s41375-024-02204-y
Knihovny.cz E-zdroje
- MeSH
- blastická krize * patologie MeSH
- chronická myeloidní leukemie * farmakoterapie patologie terapie mortalita MeSH
- dospělí MeSH
- homologní transplantace MeSH
- inhibitory tyrosinkinasy * terapeutické užití MeSH
- lidé středního věku MeSH
- lidé MeSH
- management nemoci MeSH
- míra přežití MeSH
- mladiství MeSH
- mladý dospělý MeSH
- následné studie MeSH
- prognóza MeSH
- registrace * MeSH
- senioři nad 80 let MeSH
- senioři MeSH
- transplantace hematopoetických kmenových buněk metody MeSH
- výsledek terapie MeSH
- Check Tag
- dospělí MeSH
- lidé středního věku MeSH
- lidé MeSH
- mladiství MeSH
- mladý dospělý MeSH
- mužské pohlaví MeSH
- senioři nad 80 let MeSH
- senioři MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- práce podpořená grantem MeSH
- Geografické názvy
- Evropa MeSH
- Názvy látek
- inhibitory tyrosinkinasy * MeSH
Blast phase (BP) of chronic myeloid leukemia (CML) still represents an unmet clinical need with a dismal prognosis. Due to the rarity of the condition and the heterogeneity of the biology and clinical presentation, prospective trials and concise treatment recommendations are lacking. Here we present the analysis of the European LeukemiaNet Blast Phase Registry, an international collection of the clinical presentation, treatment and outcome of blast phases which had been diagnosed in CML patients after 2015. Data reveal the expected heterogeneity of the entity, lacking a clear treatment standard. Outcomes remain dismal, with a median overall survival of 23.8 months (median follow up 27.8 months). Allogeneic stem cell transplantation (alloSCT) increases the rate of deep molecular responses. De novo BP and BP evolving from a previous CML do show slightly different features, suggesting a different biology between the two entities. Data show that outside clinical trials and in a real-world setting treatment of blast phase is individualized according to disease- and patient-related characteristics, with the aim of blast clearance prior to allogeneic stem cell transplantation. AlloSCT should be offered to all patients eligible for this procedure.
3 Med Klinik Med Fakultät Mannheim Universität Heidelberg Mannheim Germany
Abteilung für Innere Medizin 4 Klinikum Wels Grieskirchen Wels Austria
Centre Hospitalier Annecy Genevois Annecy France
Centre Léon Bérard Hématology Départment and CRCL INSERM U590 Lyon France
Department of General Pathology Pomeranian Medical University in Szczecin Szczecin Poland
Department of Haematology University Medical Centre Ljubljana Ljubljana Slovenia
Department of Hematology Jagiellonian University Medical College Cracow Poland
Department of Hematology Jagiellonian University Medical College Krakow Poland
Department of Hematology University Hospital Uppsala Uppsala Sweden
Faculty of Pharmacy University of Ljubljana Ljubljana Slovenia
Hematology and Oncology Faculty of Medicine University of Augsburg Augsburg Germany
Hematology Center after Prof R Yeolyan Yerevan Armenia
Hematology Department Institute of Hematology and Transfusion Medicine Warsaw Poland
Hematooncology Department Copernicus Memorial Hospital Lodz Poland
Institute of Hematology and Blood Transfusion Prague Czech Republic
IRCCS Azienda Ospedaliero Universitaria di Bologna Istituto di Ematologia Seràgnoli Bologna Italy
Klinikum Passau Klinik für Onkologie Hämatologie und Palliativmedizin Passau Germany
Oncology Centre of the Podkarpackie Province Department of Hematooncology Brzozow Poland
Onkologische Schwerpunktpraxis Anhut Kronach Germany
Servicio de Hematología Hospital General Universitario de Toledo Toledo Spain
Uniklinik Erlangen Medizinische Klinik 5 Erlangen Germany
Zentrum für Innere Medizin Hämatologie Onkologie Stauferklinikum Schwäbisch Gmünd Mutlangen Germany
Zobrazit více v PubMed
Hochhaus A, Larson RA, Guilhot F, Radich JP, Branford S, Hughes TP, et al. Long-term outcomes of imatinib treatment for chronic myeloid leukemia. N Engl J Med. 2017;376:917–27. doi: 10.1056/NEJMoa1609324. PubMed DOI PMC
Hochhaus A, Burchert A, Saussele S, Baerlocher GM, Mayer J, Brümmendorf TH, et al. Treatment free remission after nilotinib plus peg-interferon alpha induction and peg-interferon alpha maintenance therapy for newly diagnosed chronic myeloid leukemia patients; the tiger trial. Blood. 2023;142:446. doi: 10.1182/blood-2023-182792. PubMed DOI
Saussele S, Richter J, Guilhot J, Gruber FX, Hjorth-Hansen H, Almeida A, et al. Discontinuation of tyrosine kinase inhibitor therapy in chronic myeloid leukaemia (EURO-SKI): a prespecified interim analysis of a prospective, multicentre, non-randomised, trial. Lancet Oncol. 2018;19:747–57. doi: 10.1016/S1470-2045(18)30192-X. PubMed DOI
Hochhaus A, Masszi T, Giles FJ, Radich JP, Ross DM, Gómez Casares MT, et al. Treatment-free remission following frontline nilotinib in patients with chronic myeloid leukemia in chronic phase: results from the ENESTfreedom study. Leukemia. 2017;31:1525–31. doi: 10.1038/leu.2017.63. PubMed DOI PMC
Mahon F-X, Réa D, Guilhot J, Guilhot F, Huguet F, Nicolini F, et al. Discontinuation of imatinib in patients with chronic myeloid leukaemia who have maintained complete molecular remission for at least 2 years: the prospective, multicentre Stop Imatinib (STIM) trial. Lancet Oncol. 2010;11:1029–35. doi: 10.1016/S1470-2045(10)70233-3. PubMed DOI
Rea D, Nicolini FE, Tulliez M, Guilhot F, Guilhot J, Guerci-Bresler A, et al. Discontinuation of dasatinib or nilotinib in chronic myeloid leukemia: interim analysis of the STOP 2G-TKI study. Blood. 2017;129:846–54. doi: 10.1182/blood-2016-09-742205. PubMed DOI
Etienne G, Guilhot J, Rea D, Rigal-Huguet F, Nicolini F, Charbonnier A, et al. Long-term follow-up of the French stop imatinib (STIM1) study in patients with chronic myeloid leukemia. J Clin Oncol. 2017;35:298–305. doi: 10.1200/JCO.2016.68.2914. PubMed DOI
Ross DM, Branford S, Seymour JF, Schwarer AP, Arthur C, Yeung DT, et al. Safety and efficacy of imatinib cessation for CML patients with stable undetectable minimal residual disease: results from the TWISTER study. Blood. 2013;122:515–22. doi: 10.1182/blood-2013-02-483750. PubMed DOI
Takahashi N, Nishiwaki K, Nakaseko C, Aotsuka N, Sano K, Ohwada C, et al. Treatment-free remission after two-year consolidation therapy with nilotinib in patients with chronic myeloid leukemia: STAT2 trial in Japan. Haematologica. 2018;103:1835–42. doi: 10.3324/haematol.2018.194894. PubMed DOI PMC
Hehlmann R, Saußele S, Voskanyan A, Silver RT. Management of CML-blast crisis. Best Pract Res Clin Haematol. 2016;29:295–307. doi: 10.1016/j.beha.2016.10.005. PubMed DOI
Copland M. Treatment of blast phase chronic myeloid leukaemia: a rare and challenging entity. Br J Haematol. 2022;199:665–78. doi: 10.1111/bjh.18370. PubMed DOI PMC
Senapati J, Jabbour E, Kantarjian H, Short NJ. Pathogenesis and management of accelerated and blast phases of chronic myeloid leukemia. Leukemia. 2023;37:5–17. doi: 10.1038/s41375-022-01736-5. PubMed DOI
Hehlmann R. How I treat CML blast crisis. Blood. 2012;120:737–47. doi: 10.1182/blood-2012-03-380147. PubMed DOI
Sokal JE. Evaluation of survival data for chronic myelocytic leukemia. Am J Hematol. 1976;1:493–500. doi: 10.1002/ajh.2830010414. PubMed DOI
Kantarjian H, Cortes JE. Complete cytogenetic response, not deep molecular response, is associated with survival in chronic myeloid leukemia. J Clin Oncol. 2014;32:3077. doi: 10.1200/JCO.2014.56.0904. PubMed DOI
DeFilipp Z, Khoury HJ. Management of advanced-phase chronic myeloid leukemia. Curr Hematol Malig Rep. 2015;10:173–81. doi: 10.1007/s11899-015-0249-2. PubMed DOI
Kantarjian HM, Cortes J, O’Brien S, Giles FJ, Albitar M, Rios MB, et al. Imatinib mesylate (STI571) therapy for Philadelphia chromosome-positive chronic myelogenous leukemia in blast phase. Blood. 2002;99:3547–53. doi: 10.1182/blood.V99.10.3547. PubMed DOI
Silver RT, Cortes J, Waltzman R, Mone M, Kantarjian H. Sustained durability of responses and improved progression-free and overall survival with imatinib treatment for accelerated phase and blast crisis chronic myeloid leukemia: long-term follow-up of the STI571 0102 and 0109 trials. Haematologica. 2009;94:743–4. doi: 10.3324/haematol.2009.006999. PubMed DOI PMC
Cortes J, Kim D-W, Raffoux E, Martinelli G, Ritchie E, Roy L, et al. Efficacy and safety of dasatinib in imatinib-resistant or -intolerant patients with chronic myeloid leukemia in blast phase. Leukemia. 2008;22:2176–83. doi: 10.1038/leu.2008.221. PubMed DOI
Giles FJ, Kantarjian HM, le Coutre PD, Baccarani M, Mahon F-X, Blakesley RE, et al. Nilotinib is effective in imatinib-resistant or -intolerant patients with chronic myeloid leukemia in blastic phase. Leukemia. 2012;26:959–62. doi: 10.1038/leu.2011.355. PubMed DOI
Cortes JE, Kim D-W, Pinilla-Ibarz J, le Coutre P, Paquette R, Chuah C, et al. A phase 2 trial of ponatinib in Philadelphia chromosome-positive leukemias. N Engl J Med. 2013;369:1783–96. doi: 10.1056/NEJMoa1306494. PubMed DOI PMC
Gambacorti-Passerini C, Kantarjian HM, Kim D-W, Khoury HJ, Turkina AG, Brümmendorf TH, et al. Long-term efficacy and safety of bosutinib in patients with advanced leukemia following resistance/intolerance to imatinib and other tyrosine kinase inhibitors. Am J Hematol. 2015;90:755–68. doi: 10.1002/ajh.24034. PubMed DOI PMC
Sembill S, Ampatzidou M, Chaudhury S, Dworzak M, Kalwak K, Karow A, et al. Management of children and adolescents with chronic myeloid leukemia in blast phase: International pediatric CML expert panel recommendations. Leukemia. 2023;37:505–17. doi: 10.1038/s41375-023-01822-2. PubMed DOI PMC
Baccarani M, Saglio G, Goldman J, Hochhaus A, Simonsson B, Appelbaum F, et al. Evolving concepts in the management of chronic myeloid leukemia: recommendations from an expert panel on behalf of the European LeukemiaNet. Blood. 2006;108:1809–20. doi: 10.1182/blood-2006-02-005686. PubMed DOI
Khoury JD, Solary E, Abla O, Akkari Y, Alaggio R, Apperley JF, et al. The 5th edition of the world health organization classification of haematolymphoid tumours: myeloid and histiocytic/dendritic neoplasms. Leukemia. 2022;36:1703–19. doi: 10.1038/s41375-022-01613-1. PubMed DOI PMC
Lauseker M, Bachl K, Turkina A, Faber E, Prejzner W, Olsson-Strömberg U, et al. Prognosis of patients with chronic myeloid leukemia presenting in advanced phase is defined mainly by blast count, but also by age, chromosomal aberrations and hemoglobin. Am J Hematol. 2019;94:1236–43. doi: 10.1002/ajh.25628. PubMed DOI
Hehlmann R, Voskanyan A, Lauseker M, Pfirrmann M, Kalmanti L, Rinaldetti S, et al. High-risk additional chromosomal abnormalities at low blast counts herald death by CML. Leukemia. 2020;34:2074–86. doi: 10.1038/s41375-020-0826-9. PubMed DOI PMC
Clark RE, Apperley JF, Copland M, Cicconi S. Additional chromosomal abnormalities at chronic myeloid leukemia diagnosis predict an increased risk of progression. Blood Adv. 2021;5:1102–9. doi: 10.1182/bloodadvances.2020003570. PubMed DOI PMC
Hochhaus A, Baccarani M, Silver RT, Schiffer C, Apperley JF, Cervantes F, et al. European LeukemiaNet 2020 recommendations for treating chronic myeloid leukemia. Leukemia. 2020;34:966–84. doi: 10.1038/s41375-020-0776-2. PubMed DOI PMC
Pfirrmann M, Clark RE, Prejzner W, Lauseker M, Baccarani M, Saussele S, et al. The EUTOS long-term survival (ELTS) score is superior to the Sokal score for predicting survival in chronic myeloid leukemia. Leukemia. 2020;34:2138–49. doi: 10.1038/s41375-020-0931-9. PubMed DOI PMC
Jain P, Kantarjian HM, Ghorab A, Sasaki K, Jabbour EJ, Nogueras Gonzalez G et al. Prognostic factors and survival outcomes in patients with chronic myeloid leukemia in blast phase in the tyrosine kinase inhibitor era: cohort study of 477 patients. Cancer. 2017. 10.1002/cncr.30864. PubMed PMC
Saxena K, Jabbour E, Issa G, Sasaki K, Ravandi F, Maiti A, et al. Impact of frontline treatment approach on outcomes of myeloid blast phase CML. J Hematol Oncol. 2021;14:94. doi: 10.1186/s13045-021-01106-1. PubMed DOI PMC
Chen Z, Medeiros LJ, Kantajian HM, Zheng L, Gong Z, Patel KP, et al. Differential depth of treatment response required for optimal outcome in patients with blast phase versus chronic phase of chronic myeloid leukemia. Blood Cancer J. 2017;7:e521. doi: 10.1038/bcj.2017.4. PubMed DOI PMC
Strati P, Kantarjian H, Thomas D, O’Brien S, Konoplev S, Jorgensen JL, et al. HCVAD plus imatinib or dasatinib in lymphoid blastic phase chronic myeloid leukemia. Cancer. 2014;120:373–80. doi: 10.1002/cncr.28433. PubMed DOI PMC
Radujkovic A, Dietrich S, Blok H-J, Nagler A, Ayuk F, Finke J, et al. Allogeneic stem cell transplantation for blast crisis chronic myeloid leukemia in the era of tyrosine kinase inhibitors: a retrospective study by the EBMT chronic malignancies working party. Biol Blood Marrow Transpl. 2019;25:2008–16. doi: 10.1016/j.bbmt.2019.06.028. PubMed DOI
Kim T, Tyndel MS, Kim HJ, Ahn J-S, Choi SH, Park HJ, et al. Spectrum of somatic mutation dynamics in chronic myeloid leukemia following tyrosine kinase inhibitor therapy. Blood. 2017;129:38–47. doi: 10.1182/blood-2016-04-708560. PubMed DOI
Branford S, Wang P, Yeung DT, Thomson D, Purins A, Wadham C, et al. Integrative genomic analysis reveals cancer-associated mutations at diagnosis of CML in patients with high-risk disease. Blood. 2018;132:948–61. doi: 10.1182/blood-2018-02-832253. PubMed DOI
Schönfeld L, Rinke J, Hinze A, Nagel SN, Schäfer V, Schenk T, et al. ASXL1 mutations predict inferior molecular response to nilotinib treatment in chronic myeloid leukemia. Leukemia. 2022;36:2242–9. doi: 10.1038/s41375-022-01648-4. PubMed DOI PMC
Ochi Y, Yoshida K, Huang Y-J, Kuo M-C, Nannya Y, Sasaki K, et al. Clonal evolution and clinical implications of genetic abnormalities in blastic transformation of chronic myeloid leukaemia. Nat Commun. 2021;12:2833. doi: 10.1038/s41467-021-23097-w. PubMed DOI PMC
CML-1: Understanding the clonal hierarchy in Chronic Myeloid Leukemia to help improve patient outcomes. https://www.harmony-alliance.eu/projects/research-project/cml-understanding-the-clonal-hierarchy-in-chronic-myeloid-leukemia-to-help-improve-patient-outcomes-1655723411 (accessed 11 Feb2024).
Copland M, Slade D, McIlroy G, Horne G, Byrne JL, Rothwell K, et al. Ponatinib with fludarabine, cytarabine, idarubicin, and granulocyte colony-stimulating factor chemotherapy for patients with blast-phase chronic myeloid leukaemia (MATCHPOINT): a single-arm, multicentre, phase 1/2 trial. Lancet Haematol. 2022;9:e121–e132. doi: 10.1016/S2352-3026(21)00370-7. PubMed DOI
Deau B, Nicolini FE, Guilhot J, Huguet F, Guerci A, Legros L, et al. The addition of daunorubicin to imatinib mesylate in combination with cytarabine improves the response rate and the survival of patients with myeloid blast crisis chronic myelogenous leukemia (AFR01 study) Leuk Res. 2011;35:777–82. doi: 10.1016/j.leukres.2010.11.004. PubMed DOI