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Reviews

Use of endpoints in phase III randomized controlled trials for acute myeloid leukemia over the last 15 years: a systematic review

ORCID Icon, , , , , , , , , , ORCID Icon, , , & ORCID Icon show all
Pages 273-282 | Received 09 May 2022, Accepted 12 Oct 2022, Published online: 25 Oct 2022

References

  • Fleischmann M, Schnetzke U, Hochhaus A, et al. Management of acute myeloid leukemia: current treatment options and future perspectives. Cancers. 2021;13(22):5722.
  • Siegel RL, Miller KD, Fuchs HE, et al. Cancer statistics, 2022. CA Cancer J Clin. 2022;72(1):7–33.
  • Siegel RL, Miller KD, Jemal A. Cancer statistics, 2020. CA Cancer J Clin. 2020;70(1):7–30.
  • 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.
  • Burd A, Levine RL, Ruppert AS, et al. Precision medicine treatment in acute myeloid leukemia using prospective genomic profiling: feasibility and preliminary efficacy of the beat AML master trial. Nat Med. 2020;26(12):1852–1858.
  • Xu Q, He S, Yu L. Clinical benefits and safety of gemtuzumab ozogamicin in treating acute myeloid leukemia in various subgroups: an updated systematic review, meta-analysis, and network meta-analysis. Front Immunol. 2021;12:683595.
  • Lancet JE. Is the overall survival for older adults with AML finally improving? Best Pract Res Clin Haematol. 2018;31(4):387–390.
  • Cherny NI, Sullivan R, Dafni U, et al. A standardised, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated from anti-cancer therapies: the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). Ann Oncol. 2015;26(8):1547–1573.
  • Schnipper LE, Davidson NE, Wollins DS, et al. Updating the American Society of Clinical Oncology Value Framework: revisions and reflections in response to comments received. J Clin Oncol. 2016;34(24):2925–2934.
  • Estey E, Othus M, Lee SJ, et al. New drug approvals in acute myeloid leukemia: what’s the best end point? Leukemia. 2016;30(3):521–525.
  • 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.
  • Venditti A, Piciocchi A, Candoni A, et al. GIMEMA AML1310 trial of risk-adapted, MRD-directed therapy for young adults with newly diagnosed acute myeloid leukemia. Blood. 2019;134(12):935–945.
  • Jeyakumar D, O'Brien S. Minimal residual disease in acute myeloid leukemia. JAMA Oncol. 2020;6(12):1899–1900.
  • Jongen-Lavrencic M, Grob T, Hanekamp D, et al. Molecular minimal residual disease in acute myeloid leukemia. N Engl J Med. 2018;378(13):1189–1199.
  • Short NJ, Zhou S, Fu C, et al. Association of measurable residual disease with survival outcomes in patients with acute myeloid leukemia: a systematic review and meta-analysis. JAMA Oncol. 2020;6(12):1890–1899.
  • 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.
  • Appelbaum FR, Rosenblum D, Arceci RJ, et al. End points to establish the efficacy of new agents in the treatment of acute leukemia. Blood. 2007;109(5):1810–1816.
  • Medeiros BC. Interpretation of clinical endpoints in trials of acute myeloid leukemia. Leuk Res. 2018;68:32–39.
  • Mbanya Z, Chadda S. Time to second objective disease progression (PFS2): an emerging clinical trial endpoint with regulatory and reimbursement implications. Blood. 2014;124(21):6005–6005.
  • Walter RB, Appelbaum FR, Tallman MS, et al. Shortcomings in the clinical evaluation of new drugs: acute myeloid leukemia as paradigm. Blood. 2010;116(14):2420–2428.
  • Food and Drug Administration. Clinical trial endpoints for the approval of cancer drugs and biologics guidance for industry. Rockville, MD (USA): Food and Drug Administration; 2019, Available online at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/clinical-trial-endpoints-approval-cancer-drugs-and-biologics.
  • Panageas KS, Ben-Porat L, Dickler MN, et al. When you look matters: the effect of assessment schedule on progression-free survival. J Natl Cancer Inst. 2007;99:428–432.
  • Othus M, Sekeres MA, Nand S, et al. Relative survival following response to 7 + 3 versus azacytidine is similar in acute myeloid leukemia and high-risk myelodysplastic syndromes: an analysis of four SWOG studies. Leukemia. 2019;33(2):371–378.
  • Castaigne S, Pautas C, Terré C, et al. Effect of gemtuzumab ozogamicin on survival of adult patients with de-novo acute myeloid leukaemia (ALFA-0701): a randomised, open-label, phase 3 study. Lancet. 2012;379(9825):1508–1516.
  • Burnett AK, Russell NH, Hunter AE, et al. Clofarabine doubles the response rate in older patients with acute myeloid leukemia but does not improve survival. Blood. 2013;122(8):1384–1394.
  • Stone RM, Mandrekar SJ, Sanford BL, et al. Midostaurin plus chemotherapy for acute myeloid leukemia with a FLT3 mutation. N Engl J Med. 2017;377(5):454–464.
  • Voso MT, Ottone T, Lavorgna S, et al. MRD in AML: the role of new techniques. Front Oncol. 2019;9:655.
  • Lo-Coco F, Cuneo A, Pane F, et al. Prognostic impact of genetic characterization in the GIMEMA LAM99P multicenter study for newly diagnosed acute myeloid leukemia. Haematologica. 2008;93(7):1017–1024.
  • Pollyea DA, Bixby D, Perl A, et al. NCCN guidelines insights: acute myeloid leukemia, version 2.2021. J Natl Compr Canc Netw. 2021;19(1):16–27.
  • Falini B, Sciabolacci S, Falini L, et al. Diagnostic and therapeutic pitfalls in NPM1-mutated AML: notes from the field. Leukemia. 2021;35:3113–3126.
  • Dillon R, Hills R, Freeman S, et al. Molecular MRD status and outcome after transplantation in NPM1-mutated AML. Blood. 2020;135(9):680–688.
  • Kapp-Schwoerer S, Weber D, Corbacioglu A, et al. Impact of gemtuzumab ozogamicin on MRD and relapse risk in patients with NPM1-mutated AML: results from the AMLSG 09-09 trial. Blood. 2020;136(26):3041–3050.
  • Evaluation of Measurable Residual Disease in Patients With Acute Myeloid Leukemia as Surrogate Endpoint for Survival (PERDAM). Available at: https://clinicaltrials.gov/ct2/show/study/NCT03549351
  • Chen X, Xie H, Wood BL, et al. Relation of clinical response and minimal residual disease and their prognostic impact on outcome in acute myeloid leukemia. J Clin Oncol. 2015;33(11):1258–1264.
  • US Food and Drug Administration. Hematologic malignancies: regulatory considerations for use of minimal residual disease in development of drug and biological products for treatment. FDA; 2020. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/hematologic-malignancies-regulatory-considerations-use-minimal-residual-disease-development-drug-and
  • Abbasi S, Diab O, Mohyuddin GR, et al. In-Hospital morbidity and mortality among acute myeloid leukemia patients admitted for hematopoietic stem cell transplants between 2002–2014. Blood. 2019;134:2147–2147.
  • Alibhai SM, Breunis H, Timilshina N, et al. Quality of life and physical function in adults treated with intensive chemotherapy for acute myeloid leukemia improve over time independent of age. J Geriatr Oncol. 2015;6(4):262–271.
  • Ellis LM, Bernstein DS, Voest EE, et al. American Society of Clinical Oncology Perspective: raising the bar for clinical trials by defining clinically meaningful outcomes. J Clin Oncol. 2014;32(12):1277–1280.
  • Stewart DJ, Batist G, Kantarjian HM, et al. The urgent need for clinical research reform to permit faster, less expensive access to new therapies for lethal diseases. Clin Cancer Res. 2015;21(20):4561–4568.
  • Roboz GJ, DiNardo CD, Stein EM, et al. Ivosidenib induces deep durable remissions in patients with newly diagnosed IDH1-mutant acute myeloid leukemia. Blood. 2020;135(7):463–471.
  • DiNardo CD, Stein EM, de Botton S, et al. Durable remissions with ivosidenib in IDH1-mutated relapsed or refractory AML. N Engl J Med. 2018;378(25):2386–2398.
  • Stein EM, DiNardo CD, Pollyea DA, et al. Enasidenib in mutant IDH2 relapsed or refractory acute myeloid leukemia. Blood. 2017;130(6):722–731.
  • 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.
  • Cortes JE, Heidel FH, Hellmann A, et al. Randomized comparison of low dose cytarabine with or without glasdegib in patients with newly diagnosed acute myeloid leukemia or high-risk myelodysplastic syndrome. Leukemia. 2019;33(2):379–389.

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