Venetoclax in combination with hypomethylating agent for the treatment of advanced myeloproliferative neoplasms and acute myeloid leukemia with extramedullary disease

References

• Jain T, Rampal RK. Accelerated and blast phase myeloproliferative neoplasms. Hematol Oncol Clin North Am. 2021;35(2):325–335.
   PubMed Web of Science ®Google Scholar
• Kennedy JA, Atenafu EG, Messner HA, et al. Treatment outcomes following leukemic transformation in philadelphia-negative myeloproliferative neoplasms. Blood. 2013;121(14):2725–2733.
   PubMed Web of Science ®Google Scholar
• Cherington C, Slack JL, Leis J, et al. Allogeneic stem cell transplantation for myeloproliferative neoplasm in blast phase. Leuk Res. 2012;36(9):1147–1151.
   PubMed Web of Science ®Google Scholar
• Dinardo CD, Jonas BA, Pullarkat V, et al. Azacitidine and venetoclax in previously untreated acute myeloid leukemia. N Engl J Med. 2020;383(7):617–629.
   PubMed Web of Science ®Google Scholar
• Gangat N, Guglielmelli P, Szuber N, et al. Venetoclax with azacitidine or decitabine in blast‐phase myeloproliferative neoplasm: a multicenter series of 32 consecutive cases. Am J Hematol. 2021;96(7):781–789.
   PubMed Web of Science ®Google Scholar
• King AC, Weis TM, Derkach A, et al. Multicenter evaluation of efficacy and toxicity of venetoclax‐based combinations in patients with accelerated and blast phase myeloproliferative neoplasms. Am J Hematol. 2021;97(1):E7–E10.
   PubMed Web of Science ®Google Scholar
• Masarova L, Dinardo CD, Bose P, et al. Single-center experience with venetoclax combinations in patients with newly diagnosed and relapsed AML evolving from MPNs. Blood Adv. 2021;5(8):2156–2164.
   PubMed Web of Science ®Google Scholar
• Otoukesh S, Zhang J, Nakamura R, et al. The efficacy of venetoclax and hypomethylating agents in acute myeloid leukemia with extramedullary involvement. Leuk Lymphoma. 2020;61(8):2020–2023.
   PubMed Web of Science ®Google Scholar
• Chang H, Brandwein J, Yi Q-L, et al. Extramedullary infiltrates of AML are associated with CD56 expression, 11q23 abnormalities and inferior clinical outcome. Leuk Res. 2004;28(10):1007–1011.
   PubMed Web of Science ®Google Scholar
• Arber DA, Orazi A, Hasserjian R, et al. The 2016 revision to the world health organization classification of myeloid neoplasms and acute leukemia. Blood. 2016;127(20):2391–2405.
   PubMed Web of Science ®Google Scholar
• Mascarenhas J, Heaney ML, Najfeld V, Post-myeloproliferative neoplasm acute myeloid leukemia consortium, et al. Proposed criteria for response assessment in patients treated in clinical trials for myeloproliferative neoplasms in blast phase (MPN-BP): formal recommendations from the post-myeloproliferative neoplasm acute myeloid leukemia consortium. Leuk Res. 2012;36(12):1500–1504.
   PubMed Web of Science ®Google Scholar
• Döhner H, Estey E, Grimwade D, et al. Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel. Blood. 2017;129(4):424–447.
   PubMed Web of Science ®Google Scholar
• Tremblay D, Feld J, Dougherty M, et al. Venetoclax and hypomethylating agent combination therapy in acute myeloid leukemia secondary to a myeloproliferative neoplasm. Leuk Res. 2020;98(106456):106456.
   PubMedGoogle Scholar
• Winters AC, Gutman JA, Purev E, et al. Real-world experience of venetoclax with azacitidine for untreated patients with acute myeloid leukemia. Blood Adv. 2019;3(20):2911–2919.
   PubMed Web of Science ®Google Scholar
• Venton G, Courtier F, Charbonnier A, et al. Impact of gene mutations on treatment response and prognosis of acute myeloid leukemia secondary to myeloproliferative neoplasms. Am J Hematol. 2018;93(3):330–338.
   PubMed Web of Science ®Google Scholar
• Chihara D, Kantarjian HM, Newberry KJ, et al. Survival Outcome of patients with acute myeloid leukemia transformed from myeloproliferative neoplasms. Blood. 2016;128(22):1940–1940.
   PubMed Web of Science ®Google Scholar
• Andriani A, Montanaro M, Voso MT, et al. Azacytidine for the treatment of retrospective analysis from the gruppo laziale for the study of Ph-negative MPN. Leuk Res. 2015;39(8):801–804.
   PubMed Web of Science ®Google Scholar
• Badar T, Kantarjian HM, Ravandi F, et al. Therapeutic benefit of decitabine, a hypomethylating agent, in patients with high-risk primary myelofibrosis and myeloproliferative neoplasm in accelerated or blastic/acute myeloid leukemia phase. Leuk Res. 2015;39(9):950–956.
   PubMed Web of Science ®Google Scholar
• Mascarenhas J, Navada S, Malone A, et al. Therapeutic options for patients with myelofibrosis in blast phase. Leuk Res. 2010;34(9):1246–1249.
   PubMed Web of Science ®Google Scholar
• Thepot S, Itzykson R, Seegers V, Groupe Francophone des Myelodysplasies (GFM), et al. Treatment of progression of philadelphia-negative myeloproliferative neoplasms to myelodysplastic syndrome or acute myeloid leukemia by azacitidine: a report on 54 cases on the behalf of the groupe francophone des myelodysplasies (GFM). Blood. 2010;116(19):3735–3742.
   PubMed Web of Science ®Google Scholar
• Dunbar AJ, Rampal RK, Levine R. Leukemia secondary to myeloproliferative neoplasms. Blood. 2020;136(1):61–70.
   PubMed Web of Science ®Google Scholar
• Karantanos T, Tsai H-L, Gondek LP, et al. Genomic landscape of myelodysplastic/myeloproliferative neoplasm can predict response to hypomethylating agent therapy. Leuk Lymphoma. 2022;63(8):1942–1948.
   PubMed Web of Science ®Google Scholar
• Dinardo CD, Tiong IS, Quaglieri A, et al. Molecular patterns of response and treatment failure after frontline venetoclax combinations in older patients with AML. Blood. 2020;135(11):791–803.
   PubMed Web of Science ®Google Scholar
• Pollyea DA, Pratz KW, Wei AH, et al. Outcomes in patients with poor-risk cytogenetics with or without TP53 mutations treated with venetoclax and azacitidine. Clin Cancer Res. 2022;28(24):5272–5279. Epub 20220825.
   PubMed Web of Science ®Google Scholar
• Stahl M, Menghrajani K, Derkach A, et al. Clinical and molecular predictors of response and survival following venetoclax therapy in relapsed/refractory AML. Blood Adv. 2021;5(5):1552–1564.
   PubMed Web of Science ®Google Scholar
• Chua CC, Roberts AW, Reynolds J, et al. Chemotherapy and venetoclax in elderly acute myeloid leukemia trial (CAVEAT): a phase Ib Dose-Escalation study of venetoclax combined with modified intensive chemotherapy. J Clin Oncol. 2020;38(30):3506–3517.
   PubMed Web of Science ®Google Scholar
• Wang Y-W, Tsai C-H, Lin C-C, et al. Cytogenetics and mutations could predict outcome in relapsed and refractory acute myeloid leukemia patients receiving BCL-2 inhibitor venetoclax. Ann Hematol. 2020;99(3):501–511.
   PubMed Web of Science ®Google Scholar
• Aldoss I, Yang D, Aribi A, et al. Efficacy of the combination of venetoclax and hypomethylating agents in relapsed/refractory acute myeloid leukemia. Haematologica. 2018;103(9):e404–e7.
   PubMed Web of Science ®Google Scholar
• Pollyea DA, Stevens BM, Jones CL, et al. Venetoclax with azacitidine disrupts energy metabolism and targets leukemia stem cells in patients with acute myeloid leukemia. Nat Med. 2018;24(12):1859–1866.
   PubMed Web of Science ®Google Scholar
• Chan SM, Thomas D, Corces-Zimmerman MR, et al. Isocitrate dehydrogenase 1 and 2 mutations induce BCL-2 dependence in acute myeloid leukemia. Nat Med. 2015;21(2):178–184.
   PubMed Web of Science ®Google Scholar
• Lachowiez CA, Loghavi S, Furudate K, et al. Impact of splicing mutations in acute myeloid leukemia treated with hypomethylating agents combined with venetoclax. Blood Adv. 2021;5(8):2173–2183.
   PubMed Web of Science ®Google Scholar
• Yoshimi A, Lin K-T, Wiseman DH, et al. Coordinated alterations in RNA splicing and epigenetic regulation drive leukaemogenesis. Nature. 2019;574(7777):273–277.
   PubMed Web of Science ®Google Scholar