2,652
Views
2
CrossRef citations to date
0
Altmetric
Hematology

Efficacy and safety of combination therapies vs monotherapy of hypomethylating agents in accelerated or blast phase of Philadelphia negative myeloproliferative neoplasms: a systematic review and meta-analysis

ORCID Icon, , &
Pages 348-360 | Received 08 Aug 2022, Accepted 28 Dec 2022, Published online: 16 Jan 2023

References

  • 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.
  • Yogarajah M, Tefferi A. Leukemic transformation in myeloproliferative neoplasms: a literature review on risk, characteristics, and outcome. Mayo Clin Proc. 2017;92(7):1118–1128.
  • Tam CS, Nussenzveig RM, Popat U, et al. The natural history and treatment outcome of blast phase BCR-ABL- myeloproliferative neoplasms. Blood. 2008;112(5):1628–1637.
  • Mesa RA, Verstovsek S, Cervantes F, et al. Primary myelofibrosis (PMF), post polycythemia vera myelofibrosis (post-PV MF), post essential thrombocythemia myelofibrosis (post-ET MF), blast phase PMF (PMF-BP): consensus on terminology by the international working group for myelofibrosis research and treatment (IWG-MRT). Leuk Res. 2007;31(6):737–740.
  • Palandri F, Breccia M, Tiribelli M, et al. Risk factors for progression to blast phase and outcome in 589 patients with myelofibrosis treated with ruxolitinib: real-world data. Hematol Oncol. 2020;38(3):372–380.
  • Takagi S, Masuoka K, Uchida N, et al. Allogeneic hematopoietic cell transplantation for leukemic transformation preceded by Philadelphia chromosome-negative myeloproliferative neoplasms: a nationwide survey by the Adult Acute Myeloid Leukemia Working Group of the Japan Society for Hematopoietic Cell Transplantation. Biol Blood Marrow Transplant. 2016;22(12):2208–2213.
  • Gowin K, Ballen K, Ahn KW, et al. Survival following allogeneic transplant in patients with myelofibrosis. Blood Adv. 2020;4(9):1965–1973.
  • Andrew H, Wei PM, Ivanov V, et al. Venetoclax plus LDAC for patients with untreated AML ineligible for intensive chemotherapy- phase 3 randomized placebo controlled trial. Blood. 2020;135(24):2137–2145.
  • Dohner 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.
  • Zhou S, Tremblay D, Hoffman R, et al. Clinical benefit derived from decitabine therapy for advanced phases of myeloproliferative neoplasms. Acta Haematol. 2021;144(1):48–57.
  • Thepot S, Itzykson R, Seegers V, 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.
  • Rampal RK, Mascarenhas JO, Kosiorek HE, et al. Safety and efficacy of combined ruxolitinib and decitabine in accelerated and blast-phase myeloproliferative neoplasms. Blood Adv. 2018;2(24):3572–3580.
  • 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.
  • Liberati A, Altman DG, Tetzlaff J, et al. The PRISMA statement for reporting systematic reviews and meta-analyses of studies that evaluate healthcare interventions: explanation and elaboration. BMJ. 2009;339:b2700.
  • Slim K, Nini E, Forestier D, et al. Methodological index for non-randomized studies (minors): development and validation of a new instrument. ANZ J Surg. 2003;73(9):712–716.
  • Mascarenhas J, Heaney ML, Najfeld V, 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.
  • Cheson BD, Bennett JM, Kopecky KJ, et al. Revised recommendations of the international working group for diagnosis, standardization of response criteria, treatment outcomes, and reporting standards for therapeutic trials in acute myeloid leukemia. J Clin Oncol. 2003;21(24):4642–4649.
  • Bose P, Verstovsek S, Gasior Y, et al. Phase I/II study of ruxolitinib (RUX) with decitabine (DAC) in patients with Post-Myeloproliferative neoplasm acute myeloid leukemia (post-MPN AML): phase I results [abstract]. Blood. 2016;128(22):4262–4262.
  • Rampal RK, Mascarenhas JO, Kosiorek HE, et al. Safety and efficacy of combined ruxolitinib and decitabine in patients with Blast-Phase MPN and Post-MPN AML: results of a phase I study (myeloproliferative disorders research consortium 109 trial) [abstract]. Blood. 2016;128(22):1124–1124.
  • Assi R, Bose P, Verstovsek S, et al. The combination of ruxolitinib (RUX) with decitabine (DAC) in patients (pts) with post-myeloproliferative neoplasm acute myeloid leukemia (post-MPN AML): interim report of a phase I/II trial [abstract]. Blood. 2017;130(Suppl_1):1379.
  • Drummond MW, Harrison CN, Vicente SM, et al. A phase Ib study to assess the safety and tolerability of ruxolitinib in combination with azacitidine in patients with advanced phase myeloproliferative neoplasms (MPN), including myelodysplastic syndromes (MDS) or ACUTE MYELOID LEUKAEMIA (AML) arising from MPN (the bloodwise/TAP PHAZAR study on behalf of the UK MPN CSG) [conference abstract]. Blood. 2017;130(Suppl_1):1649.
  • Lancman G, Brunner A, Hoffman R, et al. Outcomes and predictors of survival in blast phase myeloproliferative neoplasms. Leuk Res. 2018;70:49–55.
  • Bose P, Verstovsek S, Cortes JE, et al. A phase 1/2 study of ruxolitinib and decitabine in patients with post-myeloproliferative neoplasm acute myeloid leukemia. Leukemia. 2020;34(9):2489–2492.
  • Gangat N, Morsia E, Foran JM, et al. Venetoclax plus hypomethylating agent in blast-phase myeloproliferative neoplasm: preliminary experience with 12 patients. Br J Haematol. 2020;191(5):e120–e124.
  • Mascarenhas JO, Rampal RK, Kosiorek HE, et al. Phase 2 study of ruxolitinib and decitabine in patients with myeloproliferative neoplasm in accelerated and blast phase. Blood Adv. 2020;4(20):5246–5256.
  • 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.
  • 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.
  • 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. 2022;97(1):E7–E10.
  • Yoon JJ, Benitez LL, Bixby DL, et al. Efficacy of HMA +/- venetoclax or intensive chemotherapy in blast-phase myeloproliferative neoplasms [conference abstract]. Blood. 2021;138(Supplement 1):2569–2569.
  • Andriani A, Montanaro M, Voso MT, et al. The experience of Gruppo Laziale for the study of Ph-, chronic myeloproliferative syndromes in the treatment of myeloproliferative neoplasms in blastic phase with azacytidine. Blood. 2014;124(21):3164–3164.
  • Badar T, Kantarjian HM, Ravandi F, et al. Decitabine therapy for myeloproliferative neoplasm in accelerated (MPN-AP) or mastic (acute myeloid leukemia; MPN-AML) phase. J Clin Oncol. 2014;32(15_suppl):e18006.
  • 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.
  • Andriani A, Latagliata R, Voso MT, et al. Azacitidine in the treatment of PH-myeloproliferative neoplasms in blastic phase - the experience of Gruppo Laziale for the study of PH-SMPC [abstract]. Blood. 2016;122(21):5270.
  • Chihara D, Kantarjian HM, Newberry KJ, et al. Survival outcome of patients with acute myeloid leukemia transformed from myeloproliferative neoplasms [abstract]. Blood. 2016;128(22):1940–1940.
  • Andriani A, Montanaro M, Breccia M, et al. Treatment of myeloproliferative neoplasms in blastic phase with azacytidine. Clinical results and identification of prognostic factors [abstract]. Blood. 2017;130(Suppl_1):1635.
  • Dumas PY, Bertoli S, Bérard E, et al. Azacitidine or intensive chemotherapy for older patients with secondary or therapy-related acute myeloid leukemia. Oncotarget. 2017;8(45):79126–79136.
  • 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.
  • Mollard LM, Chauveau A, Boyer-Perrard F, et al. Outcome of Ph negative myeloproliferative neoplasms transforming to accelerated or leukemic phase. Leuk Lymphoma. 2018;59(12):2812–2820.
  • Tefferi A, Mudireddy M, Mannelli F, et al. Blast phase myeloproliferative neoplasm: mayo-AGIMM study of 410 patients from two separate cohorts. Leukemia. 2018;32(5):1200–1210.
  • Andriani A, Elli E, Trapè G, et al. Treatment of Philadelphia-negative myeloproliferative neoplasms in accelerated/blastic phase with azacytidine. Clinical results and identification of prognostic factors. Hematol Oncol. 2019;37(3):291–295.
  • Morsia E, Gangat N, Foran JM, et al. Efficacy of venetoclax plus hypomethylating agent in blast phase myeloproliferative neoplasm [conference abstract]. Blood. 2020;136(Supplement 1):21.
  • Wei AH, Strickland SA Jr, Hou JZ, et al. Venetoclax combined with low-dose cytarabine for previously untreated patients with acute myeloid leukemia: results from a phase Ib/II study. J Clin Oncol. 2019;37(15):1277–1284.
  • DiNardo CD, Pratz K, Pullarkat V, et al. Venetoclax combined with decitabine or azacitidine in treatment-naive, elderly patients with acute myeloid leukemia. Blood. 2019;133(1):7–17.
  • Ma J, Ge Z. Comparison between decitabine and azacitidine for patients with acute myeloid leukemia and higher-risk myelodysplastic syndrome: a systematic review and network meta-analysis. Front Pharmacol. 2021;12:701690.
  • Maiti A, Qiao W, Sasaki K, et al. Venetoclax with decitabine vs intensive chemotherapy in acute myeloid leukemia: a propensity score matched analysis stratified by risk of treatment-related mortality. Am J Hematol. 2021;96(3):282–291.
  • Apel A, Moshe Y, Ofran Y, et al. Venetoclax combinations induce high response rates in newly diagnosed acute myeloid leukemia patients ineligible for intensive chemotherapy in routine practice. Am J Hematol. 2021;96(7):790–795.