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Refractory Myeloid Neoplasm

MedGen UID:
1764933
•Concept ID:
C5419564
•
Neoplastic Process

Definition

A myeloid neoplasm that does not respond to treatment. [from NCI]

Professional guidelines

PubMed

Myelodysplastic syndromes: 2023 update on diagnosis, risk-stratification, and management.
Garcia-Manero G
Am J Hematol 2023 Aug;98(8):1307-1325. Epub 2023 Jun 8 doi: 10.1002/ajh.26984. PMID: 37288607
Waldenström macroglobulinemia: 2023 update on diagnosis, risk stratification, and management.
Gertz MA
Am J Hematol 2023 Feb;98(2):348-358. Epub 2023 Jan 1 doi: 10.1002/ajh.26796. PMID: 36588395Free PMC Article
Diagnosis and management of Aspergillus diseases: executive summary of the 2017 ESCMID-ECMM-ERS guideline.
Ullmann AJ, Aguado JM, Arikan-Akdagli S, Denning DW, Groll AH, Lagrou K, Lass-Flörl C, Lewis RE, Munoz P, Verweij PE, Warris A, Ader F, Akova M, Arendrup MC, Barnes RA, Beigelman-Aubry C, Blot S, Bouza E, Brüggemann RJM, Buchheidt D, Cadranel J, Castagnola E, Chakrabarti A, Cuenca-Estrella M, Dimopoulos G, Fortun J, Gangneux JP, Garbino J, Heinz WJ, Herbrecht R, Heussel CP, Kibbler CC, Klimko N, Kullberg BJ, Lange C, Lehrnbecher T, Löffler J, Lortholary O, Maertens J, Marchetti O, Meis JF, Pagano L, Ribaud P, Richardson M, Roilides E, Ruhnke M, Sanguinetti M, Sheppard DC, Sinkó J, Skiada A, Vehreschild MJGT, Viscoli C, Cornely OA
Clin Microbiol Infect 2018 May;24 Suppl 1:e1-e38. Epub 2018 Mar 12 doi: 10.1016/j.cmi.2018.01.002. PMID: 29544767

Recent clinical studies

Etiology

Myelodysplastic syndromes: 2023 update on diagnosis, risk-stratification, and management.
Garcia-Manero G
Am J Hematol 2023 Aug;98(8):1307-1325. Epub 2023 Jun 8 doi: 10.1002/ajh.26984. PMID: 37288607
The International Consensus Classification (ICC) of hematologic neoplasms with germline predisposition, pediatric myelodysplastic syndrome, and juvenile myelomonocytic leukemia.
Rudelius M, Weinberg OK, Niemeyer CM, Shimamura A, Calvo KR
Virchows Arch 2023 Jan;482(1):113-130. Epub 2022 Nov 29 doi: 10.1007/s00428-022-03447-9. PMID: 36445482
Management of Acute Myeloid Leukemia: A Review for General Practitioners in Oncology.
Stubbins RJ, Francis A, Kuchenbauer F, Sanford D
Curr Oncol 2022 Aug 30;29(9):6245-6259. doi: 10.3390/curroncol29090491. PMID: 36135060Free PMC Article
Venetoclax Combined With FLAG-IDA Induction and Consolidation in Newly Diagnosed and Relapsed or Refractory Acute Myeloid Leukemia.
DiNardo CD, Lachowiez CA, Takahashi K, Loghavi S, Xiao L, Kadia T, Daver N, Adeoti M, Short NJ, Sasaki K, Wang S, Borthakur G, Issa G, Maiti A, Alvarado Y, Pemmaraju N, Montalban Bravo G, Masarova L, Yilmaz M, Jain N, Andreeff M, Jabbour E, Garcia-Manero G, Kornblau S, Ravandi F, Konopleva MY, Kantarjian HM
J Clin Oncol 2021 Sep 1;39(25):2768-2778. Epub 2021 May 27 doi: 10.1200/JCO.20.03736. PMID: 34043428Free PMC Article
Gilteritinib or Chemotherapy for Relapsed or Refractory FLT3-Mutated AML.
Perl AE, Martinelli G, Cortes JE, Neubauer A, Berman E, Paolini S, Montesinos P, Baer MR, Larson RA, Ustun C, Fabbiano F, Erba HP, Di Stasi A, Stuart R, Olin R, Kasner M, Ciceri F, Chou WC, Podoltsev N, Recher C, Yokoyama H, Hosono N, Yoon SS, Lee JH, Pardee T, Fathi AT, Liu C, Hasabou N, Liu X, Bahceci E, Levis MJ
N Engl J Med 2019 Oct 31;381(18):1728-1740. doi: 10.1056/NEJMoa1902688. PMID: 31665578

Diagnosis

Myelodysplastic syndromes: 2023 update on diagnosis, risk-stratification, and management.
Garcia-Manero G
Am J Hematol 2023 Aug;98(8):1307-1325. Epub 2023 Jun 8 doi: 10.1002/ajh.26984. PMID: 37288607
The menin inhibitor revumenib in KMT2A-rearranged or NPM1-mutant leukaemia.
Issa GC, Aldoss I, DiPersio J, Cuglievan B, Stone R, Arellano M, Thirman MJ, Patel MR, Dickens DS, Shenoy S, Shukla N, Kantarjian H, Armstrong SA, Perner F, Perry JA, Rosen G, Bagley RG, Meyers ML, Ordentlich P, Gu Y, Kumar V, Smith S, McGeehan GM, Stein EM
Nature 2023 Mar;615(7954):920-924. Epub 2023 Mar 15 doi: 10.1038/s41586-023-05812-3. PMID: 36922593Free PMC Article
The International Consensus Classification (ICC) of hematologic neoplasms with germline predisposition, pediatric myelodysplastic syndrome, and juvenile myelomonocytic leukemia.
Rudelius M, Weinberg OK, Niemeyer CM, Shimamura A, Calvo KR
Virchows Arch 2023 Jan;482(1):113-130. Epub 2022 Nov 29 doi: 10.1007/s00428-022-03447-9. PMID: 36445482
FLT3 mutated acute myeloid leukemia: 2021 treatment algorithm.
Daver N, Venugopal S, Ravandi F
Blood Cancer J 2021 May 27;11(5):104. doi: 10.1038/s41408-021-00495-3. PMID: 34045454Free PMC Article
Venetoclax Combined With FLAG-IDA Induction and Consolidation in Newly Diagnosed and Relapsed or Refractory Acute Myeloid Leukemia.
DiNardo CD, Lachowiez CA, Takahashi K, Loghavi S, Xiao L, Kadia T, Daver N, Adeoti M, Short NJ, Sasaki K, Wang S, Borthakur G, Issa G, Maiti A, Alvarado Y, Pemmaraju N, Montalban Bravo G, Masarova L, Yilmaz M, Jain N, Andreeff M, Jabbour E, Garcia-Manero G, Kornblau S, Ravandi F, Konopleva MY, Kantarjian HM
J Clin Oncol 2021 Sep 1;39(25):2768-2778. Epub 2021 May 27 doi: 10.1200/JCO.20.03736. PMID: 34043428Free PMC Article

Therapy

Myelodysplastic syndromes: 2023 update on diagnosis, risk-stratification, and management.
Garcia-Manero G
Am J Hematol 2023 Aug;98(8):1307-1325. Epub 2023 Jun 8 doi: 10.1002/ajh.26984. PMID: 37288607
The menin inhibitor revumenib in KMT2A-rearranged or NPM1-mutant leukaemia.
Issa GC, Aldoss I, DiPersio J, Cuglievan B, Stone R, Arellano M, Thirman MJ, Patel MR, Dickens DS, Shenoy S, Shukla N, Kantarjian H, Armstrong SA, Perner F, Perry JA, Rosen G, Bagley RG, Meyers ML, Ordentlich P, Gu Y, Kumar V, Smith S, McGeehan GM, Stein EM
Nature 2023 Mar;615(7954):920-924. Epub 2023 Mar 15 doi: 10.1038/s41586-023-05812-3. PMID: 36922593Free PMC Article
The emerging therapeutic landscape of relapsed/refractory multiple myeloma.
Tanenbaum B, Miett T, Patel SA
Ann Hematol 2023 Jan;102(1):1-11. Epub 2022 Dec 3 doi: 10.1007/s00277-022-05058-5. PMID: 36462062Free PMC Article
Venetoclax Combined With FLAG-IDA Induction and Consolidation in Newly Diagnosed and Relapsed or Refractory Acute Myeloid Leukemia.
DiNardo CD, Lachowiez CA, Takahashi K, Loghavi S, Xiao L, Kadia T, Daver N, Adeoti M, Short NJ, Sasaki K, Wang S, Borthakur G, Issa G, Maiti A, Alvarado Y, Pemmaraju N, Montalban Bravo G, Masarova L, Yilmaz M, Jain N, Andreeff M, Jabbour E, Garcia-Manero G, Kornblau S, Ravandi F, Konopleva MY, Kantarjian HM
J Clin Oncol 2021 Sep 1;39(25):2768-2778. Epub 2021 May 27 doi: 10.1200/JCO.20.03736. PMID: 34043428Free PMC Article
Emerging Immunotherapy for Acute Myeloid Leukemia.
Tabata R, Chi S, Yuda J, Minami Y
Int J Mol Sci 2021 Feb 16;22(4) doi: 10.3390/ijms22041944. PMID: 33669431Free PMC Article

Prognosis

Three-Year Follow-Up of KTE-X19 in Patients With Relapsed/Refractory Mantle Cell Lymphoma, Including High-Risk Subgroups, in the ZUMA-2 Study.
Wang M, Munoz J, Goy A, Locke FL, Jacobson CA, Hill BT, Timmerman JM, Holmes H, Jaglowski S, Flinn IW, McSweeney PA, Miklos DB, Pagel JM, Kersten MJ, Bouabdallah K, Khanal R, Topp MS, Houot R, Beitinjaneh A, Peng W, Fang X, Shen RR, Siddiqi R, Kloos I, Reagan PM
J Clin Oncol 2023 Jan 20;41(3):555-567. Epub 2022 Jun 4 doi: 10.1200/JCO.21.02370. PMID: 35658525Free PMC Article
Venetoclax Combined With FLAG-IDA Induction and Consolidation in Newly Diagnosed and Relapsed or Refractory Acute Myeloid Leukemia.
DiNardo CD, Lachowiez CA, Takahashi K, Loghavi S, Xiao L, Kadia T, Daver N, Adeoti M, Short NJ, Sasaki K, Wang S, Borthakur G, Issa G, Maiti A, Alvarado Y, Pemmaraju N, Montalban Bravo G, Masarova L, Yilmaz M, Jain N, Andreeff M, Jabbour E, Garcia-Manero G, Kornblau S, Ravandi F, Konopleva MY, Kantarjian HM
J Clin Oncol 2021 Sep 1;39(25):2768-2778. Epub 2021 May 27 doi: 10.1200/JCO.20.03736. PMID: 34043428Free PMC Article
Emerging Immunotherapy for Acute Myeloid Leukemia.
Tabata R, Chi S, Yuda J, Minami Y
Int J Mol Sci 2021 Feb 16;22(4) doi: 10.3390/ijms22041944. PMID: 33669431Free PMC Article
Myelodysplastic syndromes: 2021 update on diagnosis, risk stratification and management.
Garcia-Manero G, Chien KS, Montalban-Bravo G
Am J Hematol 2020 Nov;95(11):1399-1420. doi: 10.1002/ajh.25950. PMID: 32744763
Gilteritinib or Chemotherapy for Relapsed or Refractory FLT3-Mutated AML.
Perl AE, Martinelli G, Cortes JE, Neubauer A, Berman E, Paolini S, Montesinos P, Baer MR, Larson RA, Ustun C, Fabbiano F, Erba HP, Di Stasi A, Stuart R, Olin R, Kasner M, Ciceri F, Chou WC, Podoltsev N, Recher C, Yokoyama H, Hosono N, Yoon SS, Lee JH, Pardee T, Fathi AT, Liu C, Hasabou N, Liu X, Bahceci E, Levis MJ
N Engl J Med 2019 Oct 31;381(18):1728-1740. doi: 10.1056/NEJMoa1902688. PMID: 31665578

Clinical prediction guides

Myelodysplastic syndromes: 2023 update on diagnosis, risk-stratification, and management.
Garcia-Manero G
Am J Hematol 2023 Aug;98(8):1307-1325. Epub 2023 Jun 8 doi: 10.1002/ajh.26984. PMID: 37288607
Longitudinal single-cell profiling of chemotherapy response in acute myeloid leukemia.
Naldini MM, Casirati G, Barcella M, Rancoita PMV, Cosentino A, Caserta C, Pavesi F, Zonari E, Desantis G, Gilioli D, Carrabba MG, Vago L, Bernardi M, Di Micco R, Di Serio C, Merelli I, Volpin M, Montini E, Ciceri F, Gentner B
Nat Commun 2023 Mar 8;14(1):1285. doi: 10.1038/s41467-023-36969-0. PMID: 36890137Free PMC Article
Myelodysplastic syndromes: 2021 update on diagnosis, risk stratification and management.
Garcia-Manero G, Chien KS, Montalban-Bravo G
Am J Hematol 2020 Nov;95(11):1399-1420. doi: 10.1002/ajh.25950. PMID: 32744763
The leukaemia stem cell: similarities, differences and clinical prospects in CML and AML.
Vetrie D, Helgason GV, Copland M
Nat Rev Cancer 2020 Mar;20(3):158-173. Epub 2020 Jan 6 doi: 10.1038/s41568-019-0230-9. PMID: 31907378
How I treat myelodysplastic syndromes of childhood.
Locatelli F, Strahm B
Blood 2018 Mar 29;131(13):1406-1414. Epub 2018 Feb 8 doi: 10.1182/blood-2017-09-765214. PMID: 29438960

Recent systematic reviews

Efficacy and safety of FDA-approved IDH inhibitors in the treatment of IDH mutated acute myeloid leukemia: a systematic review and meta-analysis.
Chen X, Xing H, Xie X, Kou L, Li J, Li Y
Clin Epigenetics 2023 Jul 11;15(1):113. doi: 10.1186/s13148-023-01529-2. PMID: 37434249Free PMC Article
Outcomes with chimeric antigen receptor t-cell therapy in relapsed or refractory acute myeloid leukemia: a systematic review and meta-analysis.
Shahzad M, Nguyen A, Hussain A, Ammad-Ud-Din M, Faisal MS, Tariq E, Ali F, Butt A, Anwar I, Chaudhary SG, Lutfi F, Ahmed N, Singh AK, Hematti P, McGuirk JP, Mushtaq MU
Front Immunol 2023;14:1152457. Epub 2023 Apr 24 doi: 10.3389/fimmu.2023.1152457. PMID: 37168849Free PMC Article
Efficacy and tolerability of isocitrate dehydrogenase inhibitors in patients with acute myeloid leukemia: A systematic review of clinical trials.
Aiman W, Ali MA, Basit MA, Omar Z, Suleman M, Hassan M, Jamil T, Anwar MS, Shafique Z, Dhanesar G, Faisal MS, Akerman MJ, Maroules M, Anwer F
Leuk Res 2023 Jun;129:107077. Epub 2023 Apr 14 doi: 10.1016/j.leukres.2023.107077. PMID: 37100025
The efficacy and safety of venetoclax and azacytidine combination treatment in patients with acute myeloid leukemia and myelodysplastic syndrome: systematic review and meta-analysis.
Du Y, Li C, Yan J
Hematology 2023 Dec;28(1):2198098. doi: 10.1080/16078454.2023.2198098. PMID: 37036307
Antifungal prophylaxis in adult patients with acute myeloid leukaemia treated with novel targeted therapies: a systematic review and expert consensus recommendation from the European Hematology Association.
Stemler J, de Jonge N, Skoetz N, Sinkó J, Brüggemann RJ, Busca A, Ben-Ami R, Ráčil Z, Piechotta V, Lewis R, Cornely OA
Lancet Haematol 2022 May;9(5):e361-e373. doi: 10.1016/S2352-3026(22)00073-4. PMID: 35483397

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