Advances in the management of higher-risk myelodysplastic syndromes: future prospects

References

• Greenberg P, Cox C, LeBeau MM, et al. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood. 1997;89(6):2079–2088. doi:10.1182/blood.V89.6.2079
   PubMed Web of Science ®Google Scholar
• Greenberg PL, Tuechler H, Schanz J, et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012;120(12):2454–2465. doi:10.1182/blood-2012-03-420489
   PubMed Web of Science ®Google Scholar
• Bernard E, Tuechler H, Greenberg PL, et al. Molecular international prognostic scoring system for myelodysplastic syndromes. NEJM Evid. 2022;1(7):EVIDoa2200008. doi:10.1056/EVIDoa2200008
   PubMedGoogle Scholar
• Bejar R, Stevenson K, Abdel-Wahab O, et al. Clinical effect of point mutations in myelodysplastic syndromes. N Engl J Med. 2011;364(26):2496–2506. doi:10.1056/NEJMoa1013343
   PubMed Web of Science ®Google Scholar
• Haferlach T, Nagata Y, Grossmann V, et al. Landscape of genetic lesions in 944 patients with myelodysplastic syndromes. Leukemia. 2014;28(2):241–247. doi:10.1038/leu.2013.336
   PubMed Web of Science ®Google Scholar
• Papaemmanuil E, Gerstung M, Malcovati L, et al. Clinical and biological implications of driver mutations in myelodysplastic syndromes. Blood. 2013;122(22):3616–3627; quiz 3699. doi:10.1182/blood-2013-08-518886
   PubMed Web of Science ®Google Scholar
• Malcovati L, Stevenson K, Papaemmanuil E, et al. SF3B1-mutant MDS as a distinct disease subtype: a proposal from the international working group for the prognosis of MDS. Blood. 2020;136(2):157–170. doi:10.1182/blood.2020004850
   PubMed Web of Science ®Google Scholar
• Yu J, Li Y, Li T, et al. Gene mutational analysis by NGS and its clinical significance in patients with myelodysplastic syndrome and acute myeloid leukemia. Exp Hematol Oncol. 2020;9:2. doi:10.1186/s40164-019-0158-5
   PubMed Web of Science ®Google Scholar
• Ogawa S. Genetics of MDS. Blood. 2019;133(10):1049–1059. doi:10.1182/blood-2018-10-844621
   PubMed Web of Science ®Google Scholar
• Makishima H, Yoshizato T, Yoshida K, et al. Dynamics of clonal evolution in myelodysplastic syndromes. Nat Genet. 2017;49(2):204–212. doi:10.1038/ng.3742
   PubMed Web of Science ®Google Scholar
• Maurya N, Mohanty P, Dhangar S, et al. Comprehensive analysis of genetic factors predicting overall survival in myelodysplastic syndromes. Sci Rep. 2022;12(1):5925. doi:10.1038/s41598-022-09864-9
   PubMed Web of Science ®Google Scholar
• Urrutia S, Sasaki K, Kanagal-Shamanna R, et al. Impact of spliceosome mutations on the prognosis of myelodysplastic syndrome (MDS). Blood. 2022;140(Supplement 1):6952–6954. doi:10.1182/blood-2022-165488
   Google Scholar
• Kim T, Tyndel MS, Kim HJ, et al. The clonal origins of leukemic progression of myelodysplasia. Leukemia. 2017;31(9):1928–1935. doi:10.1038/leu.2017.17
   PubMed Web of Science ®Google Scholar
• Takahashi K, Jabbour E, Wang X, et al. Dynamic acquisition of FLT3 or RAS alterations drive a subset of patients with lower risk MDS to secondary AML. Leukemia. 2013;27(10):2081–2083. doi:10.1038/leu.2013.165
   PubMed Web of Science ®Google Scholar
• Walter MJ, Shen D, Ding L, et al. Clonal architecture of secondary acute myeloid leukemia. N Engl J Med. 2012;366(12):1090–1098. doi:10.1056/NEJMoa1106968
   PubMed Web of Science ®Google Scholar
• Bernard E. Molecular International Prognosis Scoring System for Myelodysplastic Syndromes. ASH; 2021 [cited 2022 Apr 4]. Available from: https://ash.confex.com/ash/2021/webprogram/Paper150554.html.
   Google Scholar
• Merz AMA, Sébert M, Sonntag J, et al. Phase to phase: navigating drug combinations with hypomethylating agents in higher-risk MDS trials for optimal outcomes. Cancer Treat Rev. 2024;123:102673. doi:10.1016/j.ctrv.2023.102673
   PubMed Web of Science ®Google Scholar
• Khoury JD, Solary E, Abla O, et al. The 5th edition of the world health organization classification of haematolymphoid tumours: myeloid and histiocytic/dendritic neoplasms. Leukemia. 2022;36(7):1703–1719. doi:10.1038/s41375-022-01613-1
   PubMed Web of Science ®Google Scholar
• Arber DA, Orazi A, Hasserjian RP, et al. International consensus classification of myeloid neoplasms and acute leukemias: integrating morphologic, clinical, and genomic data. Blood. 2022;140(11):1200–1228. doi:10.1182/blood.2022015850
   PubMed Web of Science ®Google Scholar
• Levine AJ. p53: 800 million years of evolution and 40 years of discovery. Nat Rev Cancer. 2020;20(8):471–480. 2020/05/15 ed. doi:10.1038/s41568-020-0262-1
   PubMed Web of Science ®Google Scholar
• Vousden KH, Lu X. Live or let die: the cell’s response to p53. Nat Rev Cancer. 2002;2(8):594–604. doi:10.1038/nrc864
   PubMed Web of Science ®Google Scholar
• Bernard E, Nannya Y, Hasserjian RP, et al. Implications of TP53 allelic state for genome stability, clinical presentation and outcomes in myelodysplastic syndromes. Nat Med. 2020;26(10):1549–1556. doi:10.1038/s41591-020-1008-z
   PubMed Web of Science ®Google Scholar
• Haase D, Stevenson KE, Neuberg D, et al. TP53 mutation status divides myelodysplastic syndromes with complex karyotypes into distinct prognostic subgroups. Leukemia. 2019;33(7):1747–1758. doi:10.1038/s41375-018-0351-2
   PubMed Web of Science ®Google Scholar
• Montalban-Bravo G, Takahashi K, Patel K, et al. Impact of the number of mutations in survival and response outcomes to hypomethylating agents in patients with myelodysplastic syndromes or myelodysplastic/myeloproliferative neoplasms. Oncotarget. 2018;9(11):9714–9727. doi:10.18632/oncotarget.23882
   PubMedGoogle Scholar
• Guess T, Potts CR, Bhat P, et al. Distinct patterns of clonal evolution drive myelodysplastic syndrome progression to secondary acute myeloid leukemia. Blood Cancer Discov. 2022;3(4):316–329. doi:10.1158/2643-3230.BCD-21-0128
   PubMedGoogle Scholar
• Fenaux P, Mufti GJ, Hellstrom-Lindberg E, et al. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009;10(3):223–232. doi:10.1016/S1470-2045(09)70003-8
   PubMed Web of Science ®Google Scholar
• Lübbert M, Suciu S, Baila L, et al. Low-Dose decitabine versus best supportive care in elderly patients with intermediate- or high-risk myelodysplastic syndrome (MDS) ineligible for intensive chemotherapy: final results of the randomized phase III study of the European organisation for research and treatment of cancer leukemia group and the German MDS study group. J Clin Oncol. 2011;29(15):1987–1996. doi:10.1200/JCO.2010.30.9245
   PubMed Web of Science ®Google Scholar
• Diesch J, Zwick A, Garz AK, et al. A clinical-molecular update on azanucleoside-based therapy for the treatment of hematologic cancers. Clin Epigenetics. 2016;8(1):71. doi:10.1186/s13148-016-0237-y
   PubMedGoogle Scholar
• Garcia JS, Swords RT, Roboz GJ, et al. A systematic review of higher-risk myelodysplastic syndromes clinical trials to determine the benchmark of azacitidine and explore alternative endpoints for overall survival. Leuk Res. 2021;104:106555. doi:10.1016/j.leukres.2021.106555
   PubMed Web of Science ®Google Scholar
• Silverman LR, Fenaux P, Mufti GJ, et al. Continued azacitidine therapy beyond time of first response improves quality of response in patients with higher-risk myelodysplastic syndromes. Cancer. 2011;117(12):2697–2702. doi:10.1002/cncr.25774
   PubMed Web of Science ®Google Scholar
• Garcia-Manero G, Griffiths EA, Steensma DP, et al. Oral cedazuridine/decitabine for MDS and CMML: a phase 2 pharmacokinetic/pharmacodynamic randomized crossover study. Blood. 2020;136(6):674–683. doi:10.1182/blood.2019004143
   PubMed Web of Science ®Google Scholar
• Garcia-Manero G, McCloskey J, Scott BL, et al. Development of oral azacitidine with cedazuridine for myelodysplastic syndrome (MDS) and myeloproliferative neoplasms (MPN) including CMML (chronic myelomonocytic leukemia) by targeting pharmacokinetic AUC equivalence Vs subcutaneous azacitidine. Blood. 2023;142(Supplement 1):3245–3245. doi:10.1182/blood-2023-178024
   Google Scholar
• Garcia-Manero G, Sasaki K, Montalban-Bravo G, et al. A phase II study of nivolumab or ipilimumab with or without azacitidine for patients with myelodysplastic syndrome (MDS). Blood. 2018;132(Supplement 1):465–465. doi:10.1182/blood-2018-99-119424
   PubMed Web of Science ®Google Scholar
• Ades L, Watts JM, Radinoff A, et al. Phase II study of pevonedistat (P) + azacitidine (A) versus a in patients (pts) with higher-risk myelodysplastic syndromes (MDS)/chronic myelomonocytic leukemia (CMML), or low-blast acute myelogenous leukemia (LB AML) (NCT02610777). J Clin Oncol. 2020;38(15_suppl):7506–7506. doi:10.1200/JCO.2020.38.15_suppl.7506
   Web of Science ®Google Scholar
• Zeidan AM, Ando K, Rauzy O, et al. Sabatolimab plus hypomethylating agents in previously untreated patients with higher-risk myelodysplastic syndromes (STIMULUS-MDS1): a randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Haematol. 2024;11(1)2023/12/09 ed.:e38–e50. doi:10.1016/S2352-3026(23)00333-2
   PubMed Web of Science ®Google Scholar
• Sallman DA, Al Malki MM, Asch AS, et al. Magrolimab in combination with azacitidine in patients with higher-risk myelodysplastic syndromes: final results of a phase Ib study. J Clin Oncol. 2023;41(15):2815–2826. doi:10.1200/JCO.22.01794
   PubMed Web of Science ®Google Scholar
• Garcia-Manero G. Current status of phase 3 clinical trials in high-risk myelodysplastic syndromes: pitfalls and recommendations. Lancet Haematol. 2023;10(1):e71–e78. doi:10.1016/S2352-3026(22)00265-4
   PubMed Web of Science ®Google Scholar
• Bazinet A, Bravo GM. New approaches to myelodysplastic syndrome treatment. Curr Treat Options Oncol. 2022;23(5):668–687. doi:10.1007/s11864-022-00965-1
   PubMed Web of Science ®Google Scholar
• Tsao T, Shi Y, Kornblau S, et al. Concomitant inhibition of DNA methyltransferase and BCL-2 protein function synergistically induce mitochondrial apoptosis in acute myelogenous leukemia cells. Ann Hematol. 2012;91(12):1861–1870. doi:10.1007/s00277-012-1537-8
   PubMed Web of Science ®Google Scholar
• Konopleva M, Letai A. BCL-2 inhibition in AML: an unexpected bonus? Blood. 2018;132(10):1007–1012. doi:10.1182/blood-2018-03-828269
   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. doi:10.1056/NEJMoa2012971
   PubMed Web of Science ®Google Scholar
• Bazinet A, Darbaniyan F, Jabbour E, et al. Azacitidine plus venetoclax in patients with high-risk myelodysplastic syndromes or chronic myelomonocytic leukaemia: phase 1 results of a single-Centre, dose-escalation, dose-expansion, phase 1-2 study. Lancet Haematol. 2022;9(10):e756–e765. doi:10.1016/S2352-3026(22)00216-2
   PubMed Web of Science ®Google Scholar
• Garcia JS, Platzbecker U, Odenike O, et al. Efficacy and safety of venetoclax in combination with azacitidine for the treatment of patients with treatment-naive, higher-risk myelodysplastic syndromes. Blood. 2023;142(Supplement 1):319–319. doi:10.1182/blood-2023-189446
   Google Scholar
• Bataller A, Montalban-Bravo G, Bazinet A, et al. Oral decitabine plus cedazuridine and venetoclax in patients with higher-risk myelodysplastic syndromes or chronic myelomonocytic leukaemia: a single-Centre, phase 1/2 study. Lancet Haematol. 2024;11;0. Available from: (3)[Internet].:e186–e195. https://www.thelancet.com/journals/lanhae/article/PIIS2352-3026(/fulltext. doi:10.1016/S2352-3026(23)00367-8
   PubMed Web of Science ®Google Scholar
• Zeidan AM, Garcia JS, Fenaux P, et al. Phase 3 VERONA study of venetoclax with azacitidine to assess change in complete remission and overall survival in treatment-naïve higher-risk myelodysplastic syndromes. J Clin Oncol. 2021;39(15_suppl):TPS7054–TPS7054. doi:10.1200/JCO.2021.39.15_suppl.TPS7054
   Web of Science ®Google Scholar
• Estey E, Thall P, Beran M, et al. Effect of diagnosis (refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, or acute myeloid leukemia [AML]) on outcome of AML-type chemotherapy. Blood. 1997;90(8):2969–2977. doi:10.1182/blood.V90.8.2969
   PubMed Web of Science ®Google Scholar
• Peterlin P, Le Bris Y, Turlure P, et al. CPX-351 in higher risk myelodysplastic syndrome and chronic myelomonocytic leukaemia: a multicentre, single-arm, phase 2 study. Lancet Haematol. 2023;10(7):e521–e529. doi:10.1016/S2352-3026(23)00090-X
   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. doi:10.1182/blood-2016-03-643544
   PubMed Web of Science ®Google Scholar
• DiNardo CD, Jabbour E, Ravandi F, et al. IDH1 and IDH2 mutations in myelodysplastic syndromes and role in disease progression. Leukemia. 2016;30(4):980–984. doi:10.1038/leu.2015.211
   PubMed Web of Science ®Google Scholar
• Patnaik MM, Hanson CA, Hodnefield JM, et al. Differential prognostic effect of IDH1 versus IDH2 mutations in myelodysplastic syndromes: a Mayo clinic study of 277 patients. Leukemia. 2012;26(1):101–105. doi:10.1038/leu.2011.298
   PubMed Web of Science ®Google Scholar
• Dinardo C, Roboz G, M. Watts J, et al. P724: updated substudy results for ivosidenib in idh1-mutant relapsed/refractory myelodysplastic syndrome. Hemasphere. 2023;7(S3):e75740ab. doi:10.1097/01.HS9.0000969800.75740.ab
   Google Scholar
• Stein EM, Fathi AT, DiNardo CD, et al. Enasidenib in patients with mutant IDH2 myelodysplastic syndromes: a phase 1 subgroup analysis of the multicentre, AG221-C-001 trial. Lancet Haematol. 2020;7(4):e309–e319. doi:10.1016/S2352-3026(19)30284-4
   PubMed Web of Science ®Google Scholar
• DiNardo CD, Venugopal S, Lachowiez C, et al. Targeted therapy with the mutant IDH2 inhibitor enasidenib for high-risk IDH2-mutant myelodysplastic syndrome. Blood Adv. 2023;7(11):2378–2387. doi:10.1182/bloodadvances.2022008378
   PubMed Web of Science ®Google Scholar
• Daver N, Strati P, Jabbour E, et al. FLT3 mutations in myelodysplastic syndrome and chronic myelomonocytic leukemia. Am J Hematol. 2013;88(1):56–59. doi:10.1002/ajh.23345
   PubMed Web of Science ®Google Scholar
• Bains A, Luthra R, Medeiros LJ, et al. FLT3 and NPM1 mutations in myelodysplastic syndromes. Am J Clin Pathol. 2011;135(1):62–69. doi:10.1309/AJCPEI9XU8PYBCIO
   PubMed Web of Science ®Google Scholar
• Issa GC, Aldoss I, DiPersio J, et al. The menin inhibitor revumenib in KMT2A-rearranged or NPM1-mutant leukaemia. Nature. 2023;615(7954):920–924. doi:10.1038/s41586-023-05812-3
   PubMed Web of Science ®Google Scholar
• Montalban-Bravo G, Kanagal-Shamanna R, Benton CB, et al. Genomic context and TP53 allele frequency define clinical outcomes in TP53-mutated myelodysplastic syndromes. Blood Adv. 2020;4(3):482–495. doi:10.1182/bloodadvances.2019001101
   PubMed Web of Science ®Google Scholar
• Lehmann S, Bykov VJN, Ali D, et al. Targeting p53 in vivo: a first-in-Human study with p53-Targeting compound APR-246 in refractory hematologic malignancies and prostate cancer. J Clin Oncol. 2012;30(29):3633–3639. doi:10.1200/JCO.2011.40.7783
   PubMed Web of Science ®Google Scholar
• Sallman DA, DeZern AE, Garcia-Manero G, et al. Eprenetapopt (APR-246) and azacitidine in TP53-Mutant myelodysplastic syndromes. J Clin Oncol. 2021;39(14):1584–1594. doi:10.1200/JCO.20.02341
   PubMed Web of Science ®Google Scholar
• Cluzeau T, Sebert M, Rahmé R, et al. Eprenetapopt plus azacitidine in TP53-Mutated myelodysplastic syndromes and acute myeloid leukemia: a phase II study by the groupe francophone des myélodysplasies (GFM). J Clin Oncol. 2021;39(14):1575–1583. doi:10.1200/JCO.20.02342
   PubMed Web of Science ®Google Scholar
• Bolon YT, Atshan R, Allbee-Johnson M, et al. Current use and outcome of hematopoietic stem cell transplantation: CIBMTR summary slides, The US Summary Slides. 2022.
   Google Scholar
• D'Souza A, Lee S, Zhu X, et al. Current use and trends in hematopoietic cell transplantation in the United States. Biol Blood Marrow Transplant. 2017;23(9):1417–1421. doi:10.1016/j.bbmt.2017.05.035
   PubMed Web of Science ®Google Scholar
• 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. doi:10.1182/blood-2004-01-0338
   PubMed Web of Science ®Google Scholar
• Koreth J, Pidala J, Perez WS, et al. Role of reduced-intensity conditioning allogeneic hematopoietic stem-cell transplantation in older patients with de novo myelodysplastic syndromes: an international collaborative decision analysis. J Clin Oncol. 2013;31(21):2662–2670. doi:10.1200/JCO.2012.46.8652
   PubMed Web of Science ®Google Scholar
• Robin M, Porcher R, Ruggeri A, et al. HLA-Mismatched donors in patients with myelodysplastic syndrome: an EBMT registry analysis. Biol Blood Marrow Transplant. 2019;25(1):114–120. doi:10.1016/j.bbmt.2018.08.026
   PubMed Web of Science ®Google Scholar
• Kröger N, Iacobelli S, Franke GN, et al. Dose-reduced versus standard conditioning followed by allogeneic stem-cell transplantation for patients with myelodysplastic syndrome: a prospective randomized phase III study of the EBMT (RICMAC trial). J Clin Oncol. 2017;35(19):2157–2164. doi:10.1200/JCO.2016.70.7349
   PubMed Web of Science ®Google Scholar
• Nakamura R, Saber W, Martens MJ, et al. Biologic assignment trial of reduced-intensity hematopoietic cell transplantation based on donor availability in patients 50-75 years of age with advanced myelodysplastic syndrome. J Clin Oncol. 2021;39(30):3328–3339. doi:10.1200/JCO.20.03380
   PubMed Web of Science ®Google Scholar
• Muffly L, Pasquini MC, Martens M, et al. Increasing use of allogeneic hematopoietic cell transplantation in patients aged 70 years and older in the United States. Blood. 2017;130(9):1156–1164. doi:10.1182/blood-2017-03-772368
   PubMed Web of Science ®Google Scholar
• Carré M, Porcher R, Finke J, et al. Role of age and hematopoietic cell transplantation-specific comorbidity index in myelodysplastic patients undergoing an allotransplant: a retrospective study from the chronic malignancies working party of the European group for blood and marrow transplantation. Biol Blood Marrow Transplant. 2020;26(3):451–457. doi:10.1016/j.bbmt.2019.10.015
   PubMed Web of Science ®Google Scholar
• McClune BL, Weisdorf DJ, Pedersen TL, et al. Effect of age on outcome of reduced-intensity hematopoietic cell transplantation for older patients with acute myeloid leukemia in first complete remission or with myelodysplastic syndrome. J Clin Oncol. 2010;28(11):1878–1887. doi:10.1200/JCO.2009.25.4821
   PubMed Web of Science ®Google Scholar
• Modi D, Deol A, Kim S, et al. Age does not adversely influence outcomes among patients older than 60 years who undergo allogeneic hematopoietic stem cell transplant for AML and myelodysplastic syndrome. Bone Marrow Transplant. 2017;52(11):1530–1536. doi:10.1038/bmt.2017.182
   PubMed Web of Science ®Google Scholar
• Itonaga H, Ishiyama K, Aoki K, et al. Increased opportunity for prolonged survival after allogeneic hematopoietic stem cell transplantation in patients aged 60–69 years with myelodysplastic syndrome. Ann Hematol. 2019;98(6):1367–1381. doi:10.1007/s00277-019-03653-7
   PubMed Web of Science ®Google Scholar
• Sorror ML, Maris MB, Storb R, et al. Hematopoietic cell transplantation (HCT)-specific comorbidity index: a new tool for risk assessment before allogeneic HCT. Blood. 2005;106(8):2912–2919. doi:10.1182/blood-2005-05-2004
   PubMed Web of Science ®Google Scholar
• Penack O, Peczynski C, Mohty M, et al. Association of pre-existing comorbidities with outcome of allogeneic hematopoietic cell transplantation. A retrospective analysis from the EBMT. Bone Marrow Transplant. 2022;57(2):183–190. doi:10.1038/s41409-021-01502-8
   PubMed Web of Science ®Google Scholar
• DeFilipp Z, Ciurea SO, Cutler C, et al. Hematopoietic cell transplantation in the management of myelodysplastic syndrome: an evidence-based review from the American society for transplantation and cellular therapy committee on practice guidelines. Transplant Cell Ther. 2023;29(2):71–81. doi:10.1016/j.jtct.2022.11.014
   PubMedGoogle Scholar
• Bejanyan N, Zhang M, Bo-Subait K, et al. Myeloablative conditioning for allogeneic transplantation results in superior disease-free survival for acute myelogenous leukemia and myelodysplastic syndromes with low/intermediate but not high disease risk index: a center for international blood and marrow transplant research study. Transplant Cell Ther. 2021;27(1):68.e1-68–e9. doi:10.1016/j.bbmt.2020.09.026
   Google Scholar
• Martino R, Henseler A, van Lint M, et al. Long-term follow-up of a retrospective comparison of reduced-intensity conditioning and conventional high-dose conditioning for allogeneic transplantation from matched related donors in myelodysplastic syndromes. Bone Marrow Transplant. 2017;52(8):1107–1112. doi:10.1038/bmt.2017.19
   PubMed Web of Science ®Google Scholar
• Luger SM, Ringdén O, Zhang MJ, et al. Similar outcomes using myeloablative vs reduced-intensity allogeneic transplant preparative regimens for AML or MDS. Bone Marrow Transplant. 2011;47(2):203–211. doi:10.1038/bmt.2011.69
   PubMed Web of Science ®Google Scholar
• Scott BL, Pasquini MC, Fei M, et al. Myeloablative versus reduced-intensity conditioning for hematopoietic cell transplantation in acute myelogenous leukemia and myelodysplastic syndromes—long-term follow-up of the BMT CTN 0901 clinical trial. Transplant Cell Ther. 2021;27(6):483.e1-483–e6. doi:10.1016/j.jtct.2021.02.031
   Google Scholar
• Saber W, Cutler CS, Nakamura R, et al. Impact of donor source on hematopoietic cell transplantation outcomes for patients with myelodysplastic syndromes (MDS). Blood. 2013;122(11):1974–1982. doi:10.1182/blood-2013-04-496778
   PubMed Web of Science ®Google Scholar
• Grunwald MR, Zhang MJ, Elmariah H, et al. Alternative donor transplantation for myelodysplastic syndromes: haploidentical relative and matched unrelated donors. Blood Adv. 2021;5(4):975–983. doi:10.1182/bloodadvances.2020003654
   PubMed Web of Science ®Google Scholar
• Raj K, Eikema D-J, Sheth V, et al. Comparison of outcomes for HLA-matched sibling and haplo-identical donors in myelodysplastic syndromes: report from the chronic malignancies working party of EBMT. Blood Cancer J. 2022;12(9):140–140. doi:10.1038/s41408-022-00729-y
   PubMed Web of Science ®Google Scholar
• Wang Y, Wang H-X, Lai Y-R, et al. Haploidentical transplant for myelodysplastic syndrome: registry-based comparison with identical sibling transplant. Leukemia. 2016;30(10):2055–2063. doi:10.1038/leu.2016.110
   PubMed Web of Science ®Google Scholar
• Nazha A, Komrokji R, Meggendorfer M, et al. Personalized prediction model to risk stratify patients with myelodysplastic syndromes. J Clin Oncol. 2021;39(33):3737–3746. doi:10.1200/JCO.20.02810
   PubMed Web of Science ®Google Scholar
• Bersanelli M, Travaglino E, Meggendorfer M, et al. Classification and personalized prognostic assessment on the basis of clinical and genomic features in myelodysplastic syndromes. J Clin Oncol. 2021;39(11):1223–1233. doi:10.1200/JCO.20.01659
   PubMed Web of Science ®Google Scholar
• 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. doi:10.1056/NEJMoa1611604
   PubMed Web of Science ®Google Scholar
• Bejar R, Stevenson KE, Caughey B, et al. Somatic mutations predict poor outcome in patients with myelodysplastic syndrome after hematopoietic stem-cell transplantation. J Clin Oncol. 2014;32(25):2691–2698. doi:10.1200/JCO.2013.52.3381
   PubMed Web of Science ®Google Scholar
• Della Porta MG, Gallì A, Bacigalupo A, et al. Clinical effects of driver somatic mutations on the outcomes of patients with myelodysplastic syndromes treated with allogeneic hematopoietic stem-cell transplantation. J Clin Oncol. 2016;34(30):3627–3637. doi:10.1200/JCO.2016.67.3616
   PubMed Web of Science ®Google Scholar
• Versluis J, Saber W, Tsai HK, et al. Allogeneic hematopoietic cell transplantation improves outcome in myelodysplastic syndrome across high-risk genetic subgroups: genetic analysis of the blood and marrow transplant clinical trials network 1102 study. J Clin Oncol. 2023;41(28):4497–4510. JCO.23.00866. doi:10.1200/JCO.23.00866
   PubMed Web of Science ®Google Scholar
• Liu L, Jia M, Sun L, et al. Meta-analysis of the benefit of hypomethylating agents before allogeneic hematopoietic stem cell transplantation in myelodysplastic syndromes. Clin Exp Med. 2021;21(4):537–543. doi:10.1007/s10238-021-00712-0
   PubMed Web of Science ®Google Scholar
• Schroeder T, Wegener N, Lauseker M, et al. Comparison between upfront transplantation and different pretransplant cytoreductive treatment approaches in patients with high-risk myelodysplastic syndrome and secondary acute myelogenous leukemia. Biol Blood Marrow Transplant. 2019;25(8):1550–1559. doi:10.1016/j.bbmt.2019.03.011
   PubMed Web of Science ®Google Scholar
• Potter VT, Iacobelli S, van Biezen A, et al. Comparison of intensive chemotherapy and hypomethylating agents before allogeneic stem cell transplantation for advanced myelodysplastic syndromes: a study of the myelodysplastic syndrome subcommittee of the chronic malignancies working party of the European society for blood and marrow transplant research. Biol Blood Marrow Transplant. 2016;22(9):1615–1620. doi:10.1016/j.bbmt.2016.05.026
   PubMed Web of Science ®Google Scholar
• 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. Cancer. 2010;116(23):5420–5431. doi:10.1002/cncr.25500
   PubMed Web of Science ®Google Scholar
• 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. doi:10.1182/bloodadvances.2020002544
   PubMed Web of Science ®Google Scholar
• 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. doi:10.1016/j.bbmt.2015.05.026
   PubMed Web of Science ®Google Scholar
• Kungwankiattichai S, Ponvilawan B, Roy C, et al. Maintenance with hypomethylating agents after allogeneic stem cell transplantation in acute myeloid leukemia and myelodysplastic syndrome: a systematic review and meta-analysis. Front Med (Lausanne). 2022;9:801632–801632. doi:10.3389/fmed.2022.801632
   PubMed Web of Science ®Google Scholar
• Garcia-Manero G, Fenaux P, Al-Kali A, et al. Rigosertib versus best supportive care for patients with high-risk myelodysplastic syndromes after failure of hypomethylating drugs (ONTIME): a randomised, controlled, phase 3 trial. Lancet Oncol. 2016;17(4):496–508. doi:10.1016/S1470-2045(16)00009-7
   PubMed Web of Science ®Google Scholar
• Zeidan AM, Platzbecker U, Bewersdorf JP, et al. Consensus proposal for revised international working group response criteria for higher risk myelodysplastic syndromes. Blood. 2023;141(17):2047–2061. blood.2022018604. doi:10.1182/blood.2022018604
   PubMed Web of Science ®Google Scholar
• Urrutia S, Bose P, Alvarado Y, et al. Prospective performance of the international working group 2023 and the international prognostic scoring system - molecular in a phase II trial of guadecitabine in higher-risk myelodysplastic syndrome or chronic myelomonocytic leukemia. Blood Neoplasia. 2024;1(1):100008. doi:10.1016/j.bneo.2024.100008
   Google Scholar
• Greenberg PL, Stone RM, Al-Kali A, et al. NCCN guidelines® insights: myelodysplastic syndromes, version 3.2022: featured updates to the NCCN guidelines. J Natl Compr Canc Netw. 2022;20(2):106–117. doi:10.6004/jnccn.2022.0009
   PubMed Web of Science ®Google Scholar
• Beran M, Shen Y, Kantarjian H, et al. High-dose chemotherapy in high-risk myelodysplastic syndrome. Cancer. 2001;92(8):1999–2015. doi:10.1002/1097-0142(20011015)92:8<1999::AID-CNCR1538>3.0.CO;2-B
   PubMed Web of Science ®Google Scholar
• Wattel E, Preudhomme C, Hecquet B, et al. p53 mutations are associated with resistance to chemotherapy and short survival in hematologic malignancies. Blood. 1994;84(9):3148–3157. doi:10.1182/blood.V84.9.3148.3148
   PubMed Web of Science ®Google Scholar
• Takahashi K, Patel K, Bueso-Ramos C, et al. Clinical implications of TP53 mutations in myelodysplastic syndromes treated with hypomethylating agents. Oncotarget. 2016;7(12):14172–14187. doi:10.18632/oncotarget.7290
   PubMedGoogle Scholar
• Welch JS, Petti AA, Miller CA, et al. TP53 and decitabine in acute myeloid leukemia and myelodysplastic syndromes. N Engl J Med. 2016;375(21):2023–2036. doi:10.1056/NEJMoa1605949
   PubMed Web of Science ®Google Scholar
• Jung S-H, Kim Y-J, Yim S-H, et al. Somatic mutations predict outcomes of hypomethylating therapy in patients with myelodysplastic syndrome. Oncotarget. 2016;7(34):55264–55275. doi:10.18632/oncotarget.10526
   PubMedGoogle Scholar
• Zeidan AM, Bewersdorf JP, Hasle V, et al. Prognostic implications of Mono-hit and multi-hit TP53 alterations in patients with acute myeloid leukemia and higher risk myelodysplastic syndromes treated with azacitidine-based therapy. Leukemia. 2023;37(1):240–243. doi:10.1038/s41375-022-01766-z
   PubMed Web of Science ®Google Scholar
• Urrutia S, Chien KS, Li Z, et al. Performance of IPSS-M in patients with myelodysplastic syndrome after hypomethylating agent failure. Am J Hematol. 2023;98(10):E281–E284. doi:10.1002/ajh.27043
   PubMed Web of Science ®Google Scholar
• Mossner M, Jann J-C, Wittig J, et al. Mutational hierarchies in myelodysplastic syndromes dynamically adapt and evolve upon therapy response and failure. Blood. 2016;128(9):1246–1259. doi:10.1182/blood-2015-11-679167
   PubMed Web of Science ®Google Scholar
• Ganan-Gomez I, Yang H, Ma F, et al. Stem cell architecture drives myelodysplastic syndrome progression and predicts response to venetoclax-based therapy. Nat Med. 2022;28(3):557–567. doi:10.1038/s41591-022-01696-4
   PubMed Web of Science ®Google Scholar
• Rodriguez-Sevilla JJ, Adema V, Garcia-Manero G, et al. Emerging treatments for myelodysplastic syndromes: biological rationales and clinical translation. Cell Rep Med. 2023;4(2):100940. doi:10.1016/j.xcrm.2023.100940
   PubMedGoogle Scholar
• Gener-Ricos G, Bataller A, Almanza-Huante E, et al. NPM1-Mutated myeloid neoplasms: updated outcomes with high-intensive chemotherapy regardless of the blast percentage. Blood. 2023;142(Supplement 1):957–957. doi:10.1182/blood-2023-187676
   Google Scholar
• Montalban-Bravo G, Kanagal-Shamanna R, Sasaki K, et al. NPM1 mutations define a specific subgroup of MDS and MDS/MPN patients with favorable outcomes with intensive chemotherapy. Blood Adv. 2019;3(6):922–933. doi:10.1182/bloodadvances.2018026989
   PubMed Web of Science ®Google Scholar
• Jabbour E, Faderl S, Sasaki K, et al. Phase 2 study of low-dose clofarabine plus cytarabine for patients with higher-risk myelodysplastic syndrome who have relapsed or are refractory to hypomethylating agents. Cancer. 2017;123(4):629–637. doi:10.1002/cncr.30383
   PubMed Web of Science ®Google Scholar
• Zeidan AM, Borate U, Pollyea DA, et al. A phase 1b study of venetoclax and azacitidine combination in patients with relapsed or refractory myelodysplastic syndromes. Am J Hematol. 2023;98(2):272–281. doi:10.1002/ajh.26771
   PubMed Web of Science ®Google Scholar
• Rahmani NE, Ramachandra N, Sahu S, et al. ASXL1 mutations are associated with distinct epigenomic alterations that lead to sensitivity to venetoclax and azacytidine. Blood Cancer J. 2021;11(9):157. doi:10.1038/s41408-021-00541-0
   PubMed Web of Science ®Google Scholar
• Festuccia M, Baker K, Gooley TA, et al. Hematopoietic cell transplantation in myelodysplastic syndromes after treatment with hypomethylating agents. Biol Blood Marrow Transplant. 2017;23(9):1509–1514. doi:10.1016/j.bbmt.2017.05.034
   PubMed Web of Science ®Google Scholar