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High Risk Myelodysplastic Syndrome

MedGen UID:
1623190
•Concept ID:
C4528176
•
Neoplastic Process

Definition

Myelodysplastic syndrome with more than 4.5 to 6 points, according to the revised International Prognostic Scoring System (IPSS-R). [from NCI]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVHigh Risk Myelodysplastic Syndrome

Professional guidelines

PubMed

15-days duration of venetoclax combined with azacitidine in the treatment of relapsed/refractory high-risk myelodysplastic syndromes: A retrospective single-center study.
Mei C, Ye L, Ren Y, Zhou X, Ma L, Xu G, Xu W, Lu C, Yang H, Luo Y, Jiang L, Lang W, Zhu H, Jin J, Tong H
Hematol Oncol 2023 Aug;41(3):546-554. Epub 2023 Jan 18 doi: 10.1002/hon.3112. PMID: 36516239
Outcome of high-risk myelodysplastic syndrome after azacitidine treatment failure.
Prébet T, Gore SD, Esterni B, Gardin C, Itzykson R, Thepot S, Dreyfus F, Rauzy OB, Recher C, Adès L, Quesnel B, Beach CL, Fenaux P, Vey N
J Clin Oncol 2011 Aug 20;29(24):3322-7. Epub 2011 Jul 25 doi: 10.1200/JCO.2011.35.8135. PMID: 21788559Free PMC Article
Intensive treatment strategies in patients with high-risk myelodysplastic syndrome and secondary acute myeloid leukemia.
Oosterveld M, de Witte T
Blood Rev 2000 Dec;14(4):182-9. doi: 10.1054/blre.2000.0139. PMID: 11124106

Recent clinical studies

Etiology

Clinical and molecular correlates of somatic and germline DDX41 variants in patients and families with myeloid neoplasms.
Badar T, Nanaa A, Foran JM, Viswanatha D, Al-Kali A, Lasho T, Finke C, Alkhateeb HB, He R, Gangat N, Shah M, Tefferi A, Mangaonkar AA, Litzow MR, Ongie LJ, Chlon T, Ferrer A, Patnaik MM
Haematologica 2023 Nov 1;108(11):3033-3043. doi: 10.3324/haematol.2023.282867. PMID: 37199125Free PMC Article
Olutasidenib alone or with azacitidine in IDH1-mutated acute myeloid leukaemia and myelodysplastic syndrome: phase 1 results of a phase 1/2 trial.
Watts JM, Baer MR, Yang J, Prebet T, Lee S, Schiller GJ, Dinner SN, Pigneux A, Montesinos P, Wang ES, Seiter KP, Wei AH, De Botton S, Arnan M, Donnellan W, Schwarer AP, Récher C, Jonas BA, Ferrell PB Jr, Marzac C, Kelly P, Sweeney J, Forsyth S, Guichard SM, Brevard J, Henrick P, Mohamed H, Cortes JE
Lancet Haematol 2023 Jan;10(1):e46-e58. Epub 2022 Nov 10 doi: 10.1016/S2352-3026(22)00292-7. PMID: 36370742
Molecular characterization of mutant TP53 acute myeloid leukemia and high-risk myelodysplastic syndrome.
Grob T, Al Hinai ASA, Sanders MA, Kavelaars FG, Rijken M, Gradowska PL, Biemond BJ, Breems DA, Maertens J, van Marwijk Kooy M, Pabst T, de Weerdt O, Ossenkoppele GJ, van de Loosdrecht AA, Huls GA, Cornelissen JJ, Beverloo HB, Löwenberg B, Jongen-Lavrencic M, Valk PJM
Blood 2022 Apr 14;139(15):2347-2354. doi: 10.1182/blood.2021014472. PMID: 35108372Free PMC Article
Venetoclax plus intensive chemotherapy with cladribine, idarubicin, and cytarabine in patients with newly diagnosed acute myeloid leukaemia or high-risk myelodysplastic syndrome: a cohort from a single-centre, single-arm, phase 2 trial.
Kadia TM, Reville PK, Borthakur G, Yilmaz M, Kornblau S, Alvarado Y, Dinardo CD, Daver N, Jain N, Pemmaraju N, Short N, Wang SA, Tidwell RSS, Islam R, Konopleva M, Garcia-Manero G, Ravandi F, Kantarjian HM
Lancet Haematol 2021 Aug;8(8):e552-e561. doi: 10.1016/S2352-3026(21)00192-7. PMID: 34329576Free PMC Article
Randomized controlled trial of azacitidine in patients with the myelodysplastic syndrome: a study of the cancer and leukemia group B.
Silverman LR, Demakos EP, Peterson BL, Kornblith AB, Holland JC, Odchimar-Reissig R, Stone RM, Nelson D, Powell BL, DeCastro CM, Ellerton J, Larson RA, Schiffer CA, Holland JF
J Clin Oncol 2002 May 15;20(10):2429-40. doi: 10.1200/JCO.2002.04.117. PMID: 12011120

Diagnosis

Clinical and molecular correlates of somatic and germline DDX41 variants in patients and families with myeloid neoplasms.
Badar T, Nanaa A, Foran JM, Viswanatha D, Al-Kali A, Lasho T, Finke C, Alkhateeb HB, He R, Gangat N, Shah M, Tefferi A, Mangaonkar AA, Litzow MR, Ongie LJ, Chlon T, Ferrer A, Patnaik MM
Haematologica 2023 Nov 1;108(11):3033-3043. doi: 10.3324/haematol.2023.282867. PMID: 37199125Free PMC Article
Venetoclax combined with induction chemotherapy in patients with newly diagnosed acute myeloid leukaemia: a post-hoc, propensity score-matched, cohort study.
Lachowiez CA, Reville PK, Kantarjian H, Jabbour E, Borthakur G, Daver N, Loghavi S, Furudate K, Xiao L, Pierce S, Short NJ, Maiti A, Yilmaz M, Sasaki K, Takahashi K, Konopleva M, Pemmaraju N, Popat U, Shpall E, Garcia-Manero G, Ravandi F, DiNardo CD, Kadia TM
Lancet Haematol 2022 May;9(5):e350-e360. doi: 10.1016/S2352-3026(22)00076-X. PMID: 35483396Free PMC Article
Molecular characterization of mutant TP53 acute myeloid leukemia and high-risk myelodysplastic syndrome.
Grob T, Al Hinai ASA, Sanders MA, Kavelaars FG, Rijken M, Gradowska PL, Biemond BJ, Breems DA, Maertens J, van Marwijk Kooy M, Pabst T, de Weerdt O, Ossenkoppele GJ, van de Loosdrecht AA, Huls GA, Cornelissen JJ, Beverloo HB, Löwenberg B, Jongen-Lavrencic M, Valk PJM
Blood 2022 Apr 14;139(15):2347-2354. doi: 10.1182/blood.2021014472. PMID: 35108372Free PMC Article
Venetoclax plus intensive chemotherapy with cladribine, idarubicin, and cytarabine in patients with newly diagnosed acute myeloid leukaemia or high-risk myelodysplastic syndrome: a cohort from a single-centre, single-arm, phase 2 trial.
Kadia TM, Reville PK, Borthakur G, Yilmaz M, Kornblau S, Alvarado Y, Dinardo CD, Daver N, Jain N, Pemmaraju N, Short N, Wang SA, Tidwell RSS, Islam R, Konopleva M, Garcia-Manero G, Ravandi F, Kantarjian HM
Lancet Haematol 2021 Aug;8(8):e552-e561. doi: 10.1016/S2352-3026(21)00192-7. PMID: 34329576Free PMC Article
Isavuconazole as Primary Antifungal Prophylaxis in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome: An Open-label, Prospective, Phase 2 Study.
Bose P, McCue D, Wurster S, Wiederhold NP, Konopleva M, Kadia TM, Borthakur G, Ravandi F, Masarova L, Takahashi K, Estrov Z, Yilmaz M, Daver N, Pemmaraju N, Naqvi K, Rausch CR, Marx KR, Qiao W, Huang X, Bivins CA, Pierce SA, Kantarjian HM, Kontoyiannis DP
Clin Infect Dis 2021 May 18;72(10):1755-1763. doi: 10.1093/cid/ciaa358. PMID: 32236406Free PMC Article

Therapy

Olutasidenib alone or with azacitidine in IDH1-mutated acute myeloid leukaemia and myelodysplastic syndrome: phase 1 results of a phase 1/2 trial.
Watts JM, Baer MR, Yang J, Prebet T, Lee S, Schiller GJ, Dinner SN, Pigneux A, Montesinos P, Wang ES, Seiter KP, Wei AH, De Botton S, Arnan M, Donnellan W, Schwarer AP, Récher C, Jonas BA, Ferrell PB Jr, Marzac C, Kelly P, Sweeney J, Forsyth S, Guichard SM, Brevard J, Henrick P, Mohamed H, Cortes JE
Lancet Haematol 2023 Jan;10(1):e46-e58. Epub 2022 Nov 10 doi: 10.1016/S2352-3026(22)00292-7. PMID: 36370742
Venetoclax combined with induction chemotherapy in patients with newly diagnosed acute myeloid leukaemia: a post-hoc, propensity score-matched, cohort study.
Lachowiez CA, Reville PK, Kantarjian H, Jabbour E, Borthakur G, Daver N, Loghavi S, Furudate K, Xiao L, Pierce S, Short NJ, Maiti A, Yilmaz M, Sasaki K, Takahashi K, Konopleva M, Pemmaraju N, Popat U, Shpall E, Garcia-Manero G, Ravandi F, DiNardo CD, Kadia TM
Lancet Haematol 2022 May;9(5):e350-e360. doi: 10.1016/S2352-3026(22)00076-X. PMID: 35483396Free PMC Article
Venetoclax plus intensive chemotherapy with cladribine, idarubicin, and cytarabine in patients with newly diagnosed acute myeloid leukaemia or high-risk myelodysplastic syndrome: a cohort from a single-centre, single-arm, phase 2 trial.
Kadia TM, Reville PK, Borthakur G, Yilmaz M, Kornblau S, Alvarado Y, Dinardo CD, Daver N, Jain N, Pemmaraju N, Short N, Wang SA, Tidwell RSS, Islam R, Konopleva M, Garcia-Manero G, Ravandi F, Kantarjian HM
Lancet Haematol 2021 Aug;8(8):e552-e561. doi: 10.1016/S2352-3026(21)00192-7. PMID: 34329576Free PMC Article
Isavuconazole as Primary Antifungal Prophylaxis in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome: An Open-label, Prospective, Phase 2 Study.
Bose P, McCue D, Wurster S, Wiederhold NP, Konopleva M, Kadia TM, Borthakur G, Ravandi F, Masarova L, Takahashi K, Estrov Z, Yilmaz M, Daver N, Pemmaraju N, Naqvi K, Rausch CR, Marx KR, Qiao W, Huang X, Bivins CA, Pierce SA, Kantarjian HM, Kontoyiannis DP
Clin Infect Dis 2021 May 18;72(10):1755-1763. doi: 10.1093/cid/ciaa358. PMID: 32236406Free PMC Article
Randomized controlled trial of azacitidine in patients with the myelodysplastic syndrome: a study of the cancer and leukemia group B.
Silverman LR, Demakos EP, Peterson BL, Kornblith AB, Holland JC, Odchimar-Reissig R, Stone RM, Nelson D, Powell BL, DeCastro CM, Ellerton J, Larson RA, Schiffer CA, Holland JF
J Clin Oncol 2002 May 15;20(10):2429-40. doi: 10.1200/JCO.2002.04.117. PMID: 12011120

Prognosis

Risk Stratification in Older Intensively Treated Patients With AML.
Versluis J, Metzner M, Wang A, Gradowska P, Thomas A, Jakobsen NA, Kennedy A, Moore R, Boertjes E, Vonk CM, Kavelaars FG, Rijken M, Gilkes A, Schwab C, Beverloo HB, Manz M, Visser O, Van Elssen CHMJ, de Weerdt O, Tick LW, Biemond BJ, Vekemans MC, Freeman SD, Harrison CJ, Cook JA, Dennis M, Knapper S, Thomas I, Craddock C, Ossenkoppele GJ, Löwenberg B, Russell N, Valk PJM, Vyas P
J Clin Oncol 2024 Dec;42(34):4084-4094. Epub 2024 Sep 4 doi: 10.1200/JCO.23.02631. PMID: 39231389Free PMC Article
Understanding the Continuum between High-Risk Myelodysplastic Syndrome and Acute Myeloid Leukemia.
Zavras PD, Sinanidis I, Tsakiroglou P, Karantanos T
Int J Mol Sci 2023 Mar 6;24(5) doi: 10.3390/ijms24055018. PMID: 36902450Free PMC Article
Molecular characterization of mutant TP53 acute myeloid leukemia and high-risk myelodysplastic syndrome.
Grob T, Al Hinai ASA, Sanders MA, Kavelaars FG, Rijken M, Gradowska PL, Biemond BJ, Breems DA, Maertens J, van Marwijk Kooy M, Pabst T, de Weerdt O, Ossenkoppele GJ, van de Loosdrecht AA, Huls GA, Cornelissen JJ, Beverloo HB, Löwenberg B, Jongen-Lavrencic M, Valk PJM
Blood 2022 Apr 14;139(15):2347-2354. doi: 10.1182/blood.2021014472. PMID: 35108372Free PMC Article
Venetoclax plus intensive chemotherapy with cladribine, idarubicin, and cytarabine in patients with newly diagnosed acute myeloid leukaemia or high-risk myelodysplastic syndrome: a cohort from a single-centre, single-arm, phase 2 trial.
Kadia TM, Reville PK, Borthakur G, Yilmaz M, Kornblau S, Alvarado Y, Dinardo CD, Daver N, Jain N, Pemmaraju N, Short N, Wang SA, Tidwell RSS, Islam R, Konopleva M, Garcia-Manero G, Ravandi F, Kantarjian HM
Lancet Haematol 2021 Aug;8(8):e552-e561. doi: 10.1016/S2352-3026(21)00192-7. PMID: 34329576Free PMC Article
Novel therapies in low- and high-risk myelodysplastic syndrome.
Germing U, Schroeder T, Kaivers J, Kündgen A, Kobbe G, Gattermann N
Expert Rev Hematol 2019 Oct;12(10):893-908. Epub 2019 Jul 31 doi: 10.1080/17474086.2019.1647778. PMID: 31353975

Clinical prediction guides

Understanding the Continuum between High-Risk Myelodysplastic Syndrome and Acute Myeloid Leukemia.
Zavras PD, Sinanidis I, Tsakiroglou P, Karantanos T
Int J Mol Sci 2023 Mar 6;24(5) doi: 10.3390/ijms24055018. PMID: 36902450Free PMC Article
Venetoclax combined with induction chemotherapy in patients with newly diagnosed acute myeloid leukaemia: a post-hoc, propensity score-matched, cohort study.
Lachowiez CA, Reville PK, Kantarjian H, Jabbour E, Borthakur G, Daver N, Loghavi S, Furudate K, Xiao L, Pierce S, Short NJ, Maiti A, Yilmaz M, Sasaki K, Takahashi K, Konopleva M, Pemmaraju N, Popat U, Shpall E, Garcia-Manero G, Ravandi F, DiNardo CD, Kadia TM
Lancet Haematol 2022 May;9(5):e350-e360. doi: 10.1016/S2352-3026(22)00076-X. PMID: 35483396Free PMC Article
Molecular characterization of mutant TP53 acute myeloid leukemia and high-risk myelodysplastic syndrome.
Grob T, Al Hinai ASA, Sanders MA, Kavelaars FG, Rijken M, Gradowska PL, Biemond BJ, Breems DA, Maertens J, van Marwijk Kooy M, Pabst T, de Weerdt O, Ossenkoppele GJ, van de Loosdrecht AA, Huls GA, Cornelissen JJ, Beverloo HB, Löwenberg B, Jongen-Lavrencic M, Valk PJM
Blood 2022 Apr 14;139(15):2347-2354. doi: 10.1182/blood.2021014472. PMID: 35108372Free PMC Article
Venetoclax plus intensive chemotherapy with cladribine, idarubicin, and cytarabine in patients with newly diagnosed acute myeloid leukaemia or high-risk myelodysplastic syndrome: a cohort from a single-centre, single-arm, phase 2 trial.
Kadia TM, Reville PK, Borthakur G, Yilmaz M, Kornblau S, Alvarado Y, Dinardo CD, Daver N, Jain N, Pemmaraju N, Short N, Wang SA, Tidwell RSS, Islam R, Konopleva M, Garcia-Manero G, Ravandi F, Kantarjian HM
Lancet Haematol 2021 Aug;8(8):e552-e561. doi: 10.1016/S2352-3026(21)00192-7. PMID: 34329576Free PMC Article
Novel therapies in low- and high-risk myelodysplastic syndrome.
Germing U, Schroeder T, Kaivers J, Kündgen A, Kobbe G, Gattermann N
Expert Rev Hematol 2019 Oct;12(10):893-908. Epub 2019 Jul 31 doi: 10.1080/17474086.2019.1647778. PMID: 31353975

Recent systematic reviews

A systematic review and meta-analysis of the efficacy and adverse events of azacitidine-plus-lenalidomide treatment for patients with acute myeloid leukemia, myelodysplastic syndromes and chronic myelomonocytic leukemia (1).
Kunacheewa C, Thongthang P, Ungprasert P, Utchariyaprasit E, Owattanapanich W
Hematology 2019 Dec;24(1):498-506. doi: 10.1080/16078454.2019.1631425. PMID: 31221030
5-azacitidine prolongs overall survival in patients with myelodysplastic syndrome--a systematic review and meta-analysis.
Gurion R, Vidal L, Gafter-Gvili A, Belnik Y, Yeshurun M, Raanani P, Shpilberg O
Haematologica 2010 Feb;95(2):303-10. Epub 2009 Sep 22 doi: 10.3324/haematol.2009.010611. PMID: 19773261Free PMC Article

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