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Original Articles

A phase II study of azacitidine in combination with granulocyte-macrophage colony-stimulating factor as maintenance treatment, after allogeneic blood or marrow transplantation in patients with poor-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS)

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Pages 3181-3191 | Received 23 Feb 2021, Accepted 17 Jun 2021, Published online: 21 Jul 2021

References

  • Appelbaum FR, Gundacker H, Head DR, et al. Age and acute myeloid leukemia. Blood. 2006;107(9):3481–3485.
  • Juliusson G, Antunovic P, Derolf A, et al. Age and acute myeloid leukemia: real world data on decision to treat and outcomes from the Swedish Acute Leukemia Registry. Blood. 2009;113(18):4179–4187.
  • Tallman MS, Wang ES, Altman JK, et al. Acute myeloid leukemia, version 3.2019, NCCN clinical practice guidelines in oncology. J Natl Compr Canc Netw. 2019;17(6):721–749.
  • Cornelissen JJ, van Putten WL, Verdonck LF, et al. Results of a HOVON/SAKK donor versus no-donor analysis of myeloablative HLA-identical sibling stem cell transplantation in first remission acute myeloid leukemia in young and middle-aged adults: benefits for whom? Blood. 2007;109(9):3658–3666.
  • Koreth J, Schlenk R, Kopecky KJ, et al. Allogeneic stem cell transplantation for acute myeloid leukemia in first complete remission: systematic review and meta-analysis of prospective clinical trials. JAMA. 2009;301(22):2349–2361.
  • Greenberg PL, Tuechler H, Schanz J, et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012;120(12):2454–2465.
  • Cutler CS, Lee SJ, Greenberg P, et al. A decision analysis of allogeneic bone marrow transplantation for the myelodysplastic syndromes: delayed transplantation for low-risk myelodysplasia is associated with improved outcome. Blood. 2004;104(2):579–585.
  • Nakamura R, Saber W, Martens MJ, et al. A multi-center biologic assignment trial comparing reduced intensity allogeneic hematopoietic cell transplantation to hypomethylating therapy or best supportive care in patients aged 50–75 with advanced myelodysplastic syndrome: blood and marrow transplant clinical trials network study 1102. Blood. 2020;136(Supplement 1):19–21.
  • Gale RP, Buchner T, Zhang MJ, et al. HLA-identical sibling bone marrow transplants vs chemotherapy for acute myelogenous leukemia in first remission. Leukemia. 1996;10(11):1687–1691.
  • Zittoun RA, Mandelli F, Willemze R, et al. Autologous or allogeneic bone marrow transplantation compared with intensive chemotherapy in acute myelogenous leukemia. European Organization for Research and Treatment of Cancer (EORTC) and the Gruppo Italiano Malattie Ematologiche Maligne dell'Adulto (GIMEMA) Leukemia Cooperative Groups. N Engl J Med. 1995;332(4):217–223.
  • Ciurea SO, Shah MV, Saliba RM, et al. Haploidentical transplantation for older patients with acute myeloid leukemia and myelodysplastic syndrome. Biol Blood Marrow Transplant. 2018;24(6):1232–1236.
  • Imus PH, Tsai HL, Luznik L, et al. Haploidentical transplantation using posttransplant cyclophosphamide as GVHD prophylaxis in patients over age 70. Blood Adv. 2019;3(17):2608–2616.
  • Kasamon YL, Bolanos-Meade J, Prince GT, et al. Outcomes of nonmyeloablative HLA-haploidentical blood or marrow transplantation with high-dose post-transplantation cyclophosphamide in older adults. J Clin Oncol. 2015;33(28):3152–3161.
  • Lioure B, Bene MC, Pigneux A, et al. Early matched sibling hematopoietic cell transplantation for adult AML in first remission using an age-adapted strategy: long-term results of a prospective GOELAMS study. Blood. 2012;119(12):2943–2948.
  • Passweg JR, Labopin M, Cornelissen J, et al. Conditioning intensity in middle-aged patients with AML in first CR: no advantage for myeloablative regimens irrespective of the risk group-an observational analysis by the acute leukemia working party of the EBMT. Bone Marrow Transplant. 2015;50(8):1063–1068.
  • Sorror ML, Sandmaier BM, Storer BE, et al. Long-term outcomes among older patients following nonmyeloablative conditioning and allogeneic hematopoietic cell transplantation for advanced hematologic malignancies. JAMA. 2011;306(17):1874–1883.
  • Luznik L, O'Donnell PV, Symons HJ, et al. HLA-haploidentical bone marrow transplantation for hematologic malignancies using nonmyeloablative conditioning and high-dose, posttransplantation cyclophosphamide. Biol Blood Marrow Transplant. 2008;14(6):641–650.
  • Kasamon YL, Fuchs EJ, Zahurak M, et al. Shortened-duration tacrolimus after nonmyeloablative HLA-haploidentical (NMA haplo) BMT with high-dose posttransplantation cyclophosphamide (PTCy) facilitates strategies for relapse reduction. Blood. 2016;128(22):831–831.
  • DeZern AE, Zahurak M, Bolanos-Meade J, et al. Shortened immunosuppression following peripheral blood (PB) haploidentical (haplo) transplantation with post-transplant cyclophosphamide (PTCy) is associated with tolerable rates of graft-vs-host disease (GVHD). Blood. 2019;134(Supplement_1):3320–3320.
  • Armand P, Kim HT, Zhang MJ, et al. Classifying cytogenetics in patients with acute myelogenous leukemia in complete remission undergoing allogeneic transplantation: A center for international blood and marrow transplant research study. Biol Blood Marrow Transplant. 2012;18(2):280–288.
  • Litzow MR, Tarima S, Perez WS, et al. Allogeneic transplantation for therapy-related myelodysplastic syndrome and acute myeloid leukemia. Blood. 2010;115(9):1850–1857.
  • Sengsayadeth S, Gatwood KS, Boumendil A, et al. Conditioning intensity in secondary AML with prior myelodysplastic syndrome/myeloproliferative disorders: an EBMT ALWP study. Blood Adv. 2018;2(16):2127–2135.
  • Sengsayadeth SM, Jagasia M, Engelhardt BG, et al. Allo-SCT for high-risk AML-CR1 in the molecular era: impact of FLT3/ITD outweighs the conventional markers. Bone Marrow Transplant. 2012;47(12):1535–1537.
  • Zhou Y, Othus M, Araki D, et al. Pre- and post-transplant quantification of measurable ('minimal') residual disease via multiparameter flow cytometry in adult acute myeloid leukemia. Leukemia. 2016;30(7):1456–1464.
  • Frassoni F, Barrett AJ, Granena A, et al. Relapse after allogeneic bone marrow transplantation for acute leukaemia: a survey by the E.B.M.T. of 117 cases. Br J Haematol. 1988;70(3):317–320.
  • Chen YB, Li S, Lane AA, et al. Phase I trial of maintenance sorafenib after allogeneic hematopoietic stem cell transplantation for fms-like tyrosine kinase 3 internal tandem duplication acute myeloid leukemia. Biol Blood Marrow Transplant. 2014;20(12):2042–2048.
  • Pratz KW, Rudek MA, Smith BD, et al. A prospective study of peritransplant sorafenib for patients with FLT3-ITD acute myeloid leukemia undergoing allogeneic transplantation. Biol Blood Marrow Transplant. 2020;26(2):300–306.
  • Gerber JM, Smith BD, Ngwang B, et al. A clinically relevant population of leukemic CD34(+)CD38(–) cells in acute myeloid leukemia. Blood. 2012;119(15):3571–3577.
  • Gerber JM, Zeidner JF, Morse S, et al. Association of acute myeloid leukemia's most immature phenotype with risk groups and outcomes. Haematologica. 2016;101(5):607–616.
  • Curik N, Burda P, Vargova K, et al. 5-Azacitidine in aggressive myelodysplastic syndromes regulates chromatin structure at PU.1 gene and cell differentiation capacity. Leukemia. 2012;26(8):1804–1811.
  • Taylor SM, Jones PA. Multiple new phenotypes induced in 10T1/2 and 3T3 cells treated with 5-azacytidine. Cell. 1979;17(4):771–779.
  • Laufer CB, Roberts O. Differentiation syndrome in acute myeloid leukemia after treatment with azacitidine. Eur J Haematol. 2015;95(5):484–485.
  • Bolanos-Meade J, Smith BD, Gore SD, et al. 5-Azacytidine as salvage treatment in relapsed myeloid tumors after allogeneic bone marrow transplantation. Biol Blood Marrow Transplant. 2011;17(5):754–758.
  • Smith BD, Jones RJ, Cho E, et al. Differentiation therapy in poor risk myeloid malignancies: results of a dose finding study of the combination bryostatin-1 and GM-CSF. Leuk Res. 2011;35(1):87–94.
  • Matsui WH, Gladstone DE, Vala MS, et al. The role of growth factors in the activity of pharmacological differentiation agents. Cell Growth Differ. 2002;13(6):275–283.
  • Matsui W, Smith BD, Vala M, et al. Requirement for myeloid growth factors in the differentiation of acute promyelocytic leukaemia. Br J Haematol. 2005;128(6):853–862.
  • Przepiorka D, Weisdorf D, Martin P, et al. consensus conference on acute GVHD grading. Bone Marrow Transplant. 1995;15(6):825–828.
  • Filipovich AH, Weisdorf D, Pavletic S, et al. National institutes of health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. diagnosis and staging working group report. Biol Blood Marrow Transplant. 2005;11(12):945–956.
  • Ringden O, Labopin M, Ehninger G, et al. Reduced intensity conditioning compared with myeloablative conditioning using unrelated donor transplants in patients with acute myeloid leukemia. J Clin Oncol. 2009;27(27):4570–4577.
  • Armand P, Kim HT, Logan BR, et al. Validation and refinement of the disease risk index for allogeneic stem cell transplantation. Blood. 2014;123(23):3664–3671.
  • Carpenter PA, Snyder DS, Flowers ME, et al. Prophylactic administration of imatinib after hematopoietic cell transplantation for high-risk Philadelphia chromosome-positive leukemia. Blood. 2007;109(7):2791–2793.
  • Jedlickova Z, Schmid C, Koenecke C, et al. Long-term results of adjuvant donor lymphocyte transfusion in AML after allogeneic stem cell transplantation. Bone Marrow Transplant. 2016;51(5):663–667.
  • Ali N, Tomlinson B, Metheny L, et al. Conditioning regimen intensity and low-dose azacitidine maintenance after allogeneic hematopoietic cell transplantation for acute myeloid leukemia. Leuk Lymphoma. 2020;61(12):2839–2849.
  • Maples KT, Sabo RT, McCarty JM, et al. Maintenance azacitidine after myeloablative allogeneic hematopoietic cell transplantation for myeloid malignancies. Leuk Lymphoma. 2018;59(12):2836–2841.
  • de Lima M, Oran B, Champlin RE, et al. CC-486 maintenance after stem cell transplantation in patients with acute myeloid leukemia or myelodysplastic syndromes. Biol Blood Marrow Transplant. 2018;24(10):2017–2024.
  • Vij R, Le-Rademacher J, Laumann K, et al. A phase II multicenter study of the addition of azacitidine to reduced-intensity conditioning allogeneic transplant for high-risk myelodysplasia (and older patients with acute myeloid leukemia): results of CALGB 100801 (alliance). Biol Blood Marrow Transplant. 2019;25(10):1984–1992.
  • Webster JA, Luznik L, Tsai HL, et al. Allogeneic transplantation for ph + acute lymphoblastic leukemia with posttransplantation cyclophosphamide. Blood Adv. 2020;4(20):5078–5088.
  • Wei AH, Döhner H, Pocock C, et al. The QUAZAR AML-001 maintenance trial: Results of a phase III international, randomized, double-blind, placebo-controlled study of CC-486 (oral formulation of azacitidine) in patients with acute myeloid leukemia (AML) in first remission. Blood. 2019;134(Supplement_2):LBA-3–LBA-3.
  • Huls G, Chitu DA, Havelange V, et al. Azacitidine maintenance after intensive chemotherapy improves DFS in older AML patients. Blood. 2019;133(13):1457–1464.
  • Levis M, Small D. FLT3: ITDoes matter in leukemia. Leukemia. 2003;17(9):1738–1752.
  • Hourigan CS, Dillon LW, Gui G, et al. Impact of conditioning intensity of allogeneic transplantation for acute myeloid leukemia with genomic evidence of residual disease. J Clin Oncol. 2020;38(12):1273–1283.
  • Craddock C, Jilani N, Siddique S, et al. Tolerability and clinical activity of post-transplantation azacitidine in patients allografted for acute myeloid leukemia treated on the RICAZA trial. Biol Blood Marrow Transplant. 2016;22(2):385–390.
  • Pusic I, Choi J, Fiala MA, et al. Maintenance therapy with decitabine after allogeneic stem cell transplantation for acute myelogenous leukemia and myelodysplastic syndrome. Biol Blood Marrow Transplant. 2015;21(10):1761–1769.
  • Platzbecker U, Wermke M, Radke J, et al. Azacitidine for treatment of imminent relapse in MDS or AML patients after allogeneic HSCT: results of the RELAZA trial. Leukemia. 2012;26(3):381–389.
  • de Lima M, Giralt S, Thall PF, et al. Maintenance therapy with low-dose azacitidine after allogeneic hematopoietic stem cell transplantation for recurrent acute myelogenous leukemia or myelodysplastic syndrome: a dose and schedule finding study. Cancer. 2010;116(23):5420–5431.
  • McCurdy SR, Kasamon YL, Kanakry CG, et al. Comparable composite endpoints after HLA-matched and HLA-haploidentical transplantation with post-transplantation cyclophosphamide. Haematologica. 2017;102(2):391–400.
  • Dombret H, Seymour JF, Butrym A, et al. International phase 3 study of azacitidine vs conventional care regimens in older patients with newly diagnosed AML with >30% blasts. Blood. 2015;126(3):291–299.
  • Silverman LR, Demakos EP, Peterson BL, et al. Randomized controlled trial of azacitidine in patients with the myelodysplastic syndrome: a study of the cancer and leukemia group B. J Clin Oncol. 2002;20(10):2429–2440.
  • Oran B, de Lima M, Garcia-Manero G, et al. A phase 3 randomized study of 5-azacitidine maintenance vs observation after transplant in high-risk AML and MDS patients. Blood Adv. 2020;4(21):5580–5588.
  • Levis MJ, Chen Y-B, Hamadani M, et al. FLT3 inhibitor maintenance after allogeneic transplantation: is a placebo-controlled, randomized trial ethical? J Clin Oncol. 2019;37(19):1604–1607.
  • Lindsley RC, Saber W, Mar BG, et al. Prognostic mutations in myelodysplastic syndrome after stem-cell transplantation. N Engl J Med. 2017;376(6):536–547.
  • Norsworthy KJ, DeZern AE, Tsai HL, et al. Timed sequential therapy for acute myelogenous leukemia: results of a retrospective study of 301 patients and review of the literature. Leuk Res. 2017;61:25–32.

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