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Refractory Acute Myelomonocytic Leukemia

MedGen UID:
1758507
•Concept ID:
C5420825
•
Neoplastic Process

Definition

Acute myelomonocytic leukemia that does not respond to treatment. [from NCI]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVRefractory Acute Myelomonocytic Leukemia

Professional guidelines

PubMed

Primary myelofibrosis: 2013 update on diagnosis, risk-stratification, and management.
Tefferi A
Am J Hematol 2013 Feb;88(2):141-50. doi: 10.1002/ajh.23384. PMID: 23349007
Primary myelofibrosis: 2012 update on diagnosis, risk stratification, and management.
Tefferi A
Am J Hematol 2011 Dec;86(12):1017-26. doi: 10.1002/ajh.22210. PMID: 22086865
Successful combination treatment of clofarabine, cytarabine, and gemtuzumab-ozogamicin in adult refractory B-acute lymphoblastic leukemia.
Papayannidis C, Derenzini E, Iacobucci I, Curti A, Paolini S, Cilloni D, Baccarani M, Martinelli G
Am J Hematol 2009 Dec;84(12):849-50. doi: 10.1002/ajh.21548. PMID: 19844989

Recent clinical studies

Etiology

Natural history study of patients with familial platelet disorder with associated myeloid malignancy.
Cunningham L, Merguerian M, Calvo KR, Davis J, Deuitch NT, Dulau-Florea A, Patel N, Yu K, Sacco K, Bhattacharya S, Passi M, Ozkaya N, De Leon S, Chong S, Craft K, Diemer J, Bresciani E, O'Brien K, Andrews EJ, Park N, Hathaway L, Cowen EW, Heller T, Ryan K, Barochia A, Nghiem K, Niemela J, Rosenzweig S, Young DJ, Frischmeyer-Guerrerio PA, Braylan R, Liu PP
Blood 2023 Dec 21;142(25):2146-2158. doi: 10.1182/blood.2023019746. PMID: 37738626Free PMC Article
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.
Bazinet A, Darbaniyan F, Jabbour E, Montalban-Bravo G, Ohanian M, Chien K, Kadia T, Takahashi K, Masarova L, Short N, Alvarado Y, Yilmaz M, Ravandi F, Andreeff M, Kanagal-Shamanna R, Ganan-Gomez I, Colla S, Qiao W, Huang X, McCue D, Mirabella B, Kantarjian H, Garcia-Manero G
Lancet Haematol 2022 Oct;9(10):e756-e765. Epub 2022 Sep 2 doi: 10.1016/S2352-3026(22)00216-2. PMID: 36063832
10-day decitabine with venetoclax for newly diagnosed intensive chemotherapy ineligible, and relapsed or refractory acute myeloid leukaemia: a single-centre, phase 2 trial.
DiNardo CD, Maiti A, Rausch CR, Pemmaraju N, Naqvi K, Daver NG, Kadia TM, Borthakur G, Ohanian M, Alvarado Y, Issa GC, Montalban-Bravo G, Short NJ, Yilmaz M, Bose P, Jabbour EJ, Takahashi K, Burger JA, Garcia-Manero G, Jain N, Kornblau SM, Thompson PA, Estrov Z, Masarova L, Sasaki K, Verstovsek S, Ferrajoli A, Weirda WG, Wang SA, Konoplev S, Chen Z, Pierce SA, Ning J, Qiao W, Ravandi F, Andreeff M, Welch JS, Kantarjian HM, Konopleva MY
Lancet Haematol 2020 Oct;7(10):e724-e736. Epub 2020 Sep 5 doi: 10.1016/S2352-3026(20)30210-6. PMID: 32896301Free PMC Article
Decitabine/Cedazuridine: First Approval.
Dhillon S
Drugs 2020 Sep;80(13):1373-1378. doi: 10.1007/s40265-020-01389-7. PMID: 32860582Free PMC Article
Acute leukaemia in Jehovah's Witnesses.
Cullis JO, Duncombe AS, Dudley JM, Lumley HS, Apperley JF, Smith AG
Br J Haematol 1998 Mar;100(4):664-8. doi: 10.1046/j.1365-2141.1998.00634.x. PMID: 9531331

Diagnosis

Myelomonocytic and monocytic acute myeloid leukemia demonstrate comparable poor outcomes with venetoclax-based treatment: a monocentric real-world study.
Zhao L, Yang J, Chen M, Xiang X, Ma H, Niu T, Gong Y, Chen X, Liu J, Wu Y
Ann Hematol 2024 Apr;103(4):1197-1209. Epub 2024 Feb 8 doi: 10.1007/s00277-024-05646-7. PMID: 38329487
Natural history study of patients with familial platelet disorder with associated myeloid malignancy.
Cunningham L, Merguerian M, Calvo KR, Davis J, Deuitch NT, Dulau-Florea A, Patel N, Yu K, Sacco K, Bhattacharya S, Passi M, Ozkaya N, De Leon S, Chong S, Craft K, Diemer J, Bresciani E, O'Brien K, Andrews EJ, Park N, Hathaway L, Cowen EW, Heller T, Ryan K, Barochia A, Nghiem K, Niemela J, Rosenzweig S, Young DJ, Frischmeyer-Guerrerio PA, Braylan R, Liu PP
Blood 2023 Dec 21;142(25):2146-2158. doi: 10.1182/blood.2023019746. PMID: 37738626Free PMC Article
10-day decitabine with venetoclax for newly diagnosed intensive chemotherapy ineligible, and relapsed or refractory acute myeloid leukaemia: a single-centre, phase 2 trial.
DiNardo CD, Maiti A, Rausch CR, Pemmaraju N, Naqvi K, Daver NG, Kadia TM, Borthakur G, Ohanian M, Alvarado Y, Issa GC, Montalban-Bravo G, Short NJ, Yilmaz M, Bose P, Jabbour EJ, Takahashi K, Burger JA, Garcia-Manero G, Jain N, Kornblau SM, Thompson PA, Estrov Z, Masarova L, Sasaki K, Verstovsek S, Ferrajoli A, Weirda WG, Wang SA, Konoplev S, Chen Z, Pierce SA, Ning J, Qiao W, Ravandi F, Andreeff M, Welch JS, Kantarjian HM, Konopleva MY
Lancet Haematol 2020 Oct;7(10):e724-e736. Epub 2020 Sep 5 doi: 10.1016/S2352-3026(20)30210-6. PMID: 32896301Free PMC Article
Chromosomal change during 6-mercaptopurine (6-MP) therapy in juvenile myelomonocytic leukemia: the growth of a 6-MP-refractory clone that already exists at onset.
Matsuda K, Matsuzaki S, Miki J, Hidaka E, Yanagisawa R, Nakazawa Y, Sakashita K, Kamijo T, Asami K, Sano K, Koike K
Leukemia 2006 Mar;20(3):485-90. doi: 10.1038/sj.leu.2404106. PMID: 16424864
Classification of acute leukemia.
Ann Intern Med 1977 Dec;87(6):740-53. doi: 10.7326/0003-4819-87-6-740. PMID: 337870

Therapy

RAS-mutant leukaemia stem cells drive clinical resistance to venetoclax.
Sango J, Carcamo S, Sirenko M, Maiti A, Mansour H, Ulukaya G, Tomalin LE, Cruz-Rodriguez N, Wang T, Olszewska M, Olivier E, Jaud M, Nadorp B, Kroger B, Hu F, Silverman L, Chung SS, Wagenblast E, Chaligne R, Eisfeld AK, Demircioglu D, Landau DA, Lito P, Papaemmanuil E, DiNardo CD, Hasson D, Konopleva M, Papapetrou EP
Nature 2024 Dec;636(8041):241-250. Epub 2024 Oct 30 doi: 10.1038/s41586-024-08137-x. PMID: 39478230Free PMC Article
Natural history study of patients with familial platelet disorder with associated myeloid malignancy.
Cunningham L, Merguerian M, Calvo KR, Davis J, Deuitch NT, Dulau-Florea A, Patel N, Yu K, Sacco K, Bhattacharya S, Passi M, Ozkaya N, De Leon S, Chong S, Craft K, Diemer J, Bresciani E, O'Brien K, Andrews EJ, Park N, Hathaway L, Cowen EW, Heller T, Ryan K, Barochia A, Nghiem K, Niemela J, Rosenzweig S, Young DJ, Frischmeyer-Guerrerio PA, Braylan R, Liu PP
Blood 2023 Dec 21;142(25):2146-2158. doi: 10.1182/blood.2023019746. PMID: 37738626Free PMC Article
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.
Bazinet A, Darbaniyan F, Jabbour E, Montalban-Bravo G, Ohanian M, Chien K, Kadia T, Takahashi K, Masarova L, Short N, Alvarado Y, Yilmaz M, Ravandi F, Andreeff M, Kanagal-Shamanna R, Ganan-Gomez I, Colla S, Qiao W, Huang X, McCue D, Mirabella B, Kantarjian H, Garcia-Manero G
Lancet Haematol 2022 Oct;9(10):e756-e765. Epub 2022 Sep 2 doi: 10.1016/S2352-3026(22)00216-2. PMID: 36063832
10-day decitabine with venetoclax for newly diagnosed intensive chemotherapy ineligible, and relapsed or refractory acute myeloid leukaemia: a single-centre, phase 2 trial.
DiNardo CD, Maiti A, Rausch CR, Pemmaraju N, Naqvi K, Daver NG, Kadia TM, Borthakur G, Ohanian M, Alvarado Y, Issa GC, Montalban-Bravo G, Short NJ, Yilmaz M, Bose P, Jabbour EJ, Takahashi K, Burger JA, Garcia-Manero G, Jain N, Kornblau SM, Thompson PA, Estrov Z, Masarova L, Sasaki K, Verstovsek S, Ferrajoli A, Weirda WG, Wang SA, Konoplev S, Chen Z, Pierce SA, Ning J, Qiao W, Ravandi F, Andreeff M, Welch JS, Kantarjian HM, Konopleva MY
Lancet Haematol 2020 Oct;7(10):e724-e736. Epub 2020 Sep 5 doi: 10.1016/S2352-3026(20)30210-6. PMID: 32896301Free PMC Article
Decitabine/Cedazuridine: First Approval.
Dhillon S
Drugs 2020 Sep;80(13):1373-1378. doi: 10.1007/s40265-020-01389-7. PMID: 32860582Free PMC Article

Prognosis

RAS-mutant leukaemia stem cells drive clinical resistance to venetoclax.
Sango J, Carcamo S, Sirenko M, Maiti A, Mansour H, Ulukaya G, Tomalin LE, Cruz-Rodriguez N, Wang T, Olszewska M, Olivier E, Jaud M, Nadorp B, Kroger B, Hu F, Silverman L, Chung SS, Wagenblast E, Chaligne R, Eisfeld AK, Demircioglu D, Landau DA, Lito P, Papaemmanuil E, DiNardo CD, Hasson D, Konopleva M, Papapetrou EP
Nature 2024 Dec;636(8041):241-250. Epub 2024 Oct 30 doi: 10.1038/s41586-024-08137-x. PMID: 39478230Free PMC Article
Natural history study of patients with familial platelet disorder with associated myeloid malignancy.
Cunningham L, Merguerian M, Calvo KR, Davis J, Deuitch NT, Dulau-Florea A, Patel N, Yu K, Sacco K, Bhattacharya S, Passi M, Ozkaya N, De Leon S, Chong S, Craft K, Diemer J, Bresciani E, O'Brien K, Andrews EJ, Park N, Hathaway L, Cowen EW, Heller T, Ryan K, Barochia A, Nghiem K, Niemela J, Rosenzweig S, Young DJ, Frischmeyer-Guerrerio PA, Braylan R, Liu PP
Blood 2023 Dec 21;142(25):2146-2158. doi: 10.1182/blood.2023019746. PMID: 37738626Free PMC Article
Decitabine/Cedazuridine: First Approval.
Dhillon S
Drugs 2020 Sep;80(13):1373-1378. doi: 10.1007/s40265-020-01389-7. PMID: 32860582Free PMC Article
Acute leukaemia in Jehovah's Witnesses.
Cullis JO, Duncombe AS, Dudley JM, Lumley HS, Apperley JF, Smith AG
Br J Haematol 1998 Mar;100(4):664-8. doi: 10.1046/j.1365-2141.1998.00634.x. PMID: 9531331
Mitoxantrone and etoposide in patients with relapsed and refractory acute nonlymphocytic leukemia.
Rowe JM, Mazza JJ, Hines JD, Cassileth PA, Oken MM, Bennett JM, Andersen J
Haematol Blood Transfus 1990;33:326-9. doi: 10.1007/978-3-642-74643-7_61. PMID: 2182427

Clinical prediction guides

RAS-mutant leukaemia stem cells drive clinical resistance to venetoclax.
Sango J, Carcamo S, Sirenko M, Maiti A, Mansour H, Ulukaya G, Tomalin LE, Cruz-Rodriguez N, Wang T, Olszewska M, Olivier E, Jaud M, Nadorp B, Kroger B, Hu F, Silverman L, Chung SS, Wagenblast E, Chaligne R, Eisfeld AK, Demircioglu D, Landau DA, Lito P, Papaemmanuil E, DiNardo CD, Hasson D, Konopleva M, Papapetrou EP
Nature 2024 Dec;636(8041):241-250. Epub 2024 Oct 30 doi: 10.1038/s41586-024-08137-x. PMID: 39478230Free PMC Article
Myelomonocytic and monocytic acute myeloid leukemia demonstrate comparable poor outcomes with venetoclax-based treatment: a monocentric real-world study.
Zhao L, Yang J, Chen M, Xiang X, Ma H, Niu T, Gong Y, Chen X, Liu J, Wu Y
Ann Hematol 2024 Apr;103(4):1197-1209. Epub 2024 Feb 8 doi: 10.1007/s00277-024-05646-7. PMID: 38329487
Preclinical characterization and clinical trial of CFI-400945, a polo-like kinase 4 inhibitor, in patients with relapsed/refractory acute myeloid leukemia and higher-risk myelodysplastic neoplasms.
Murphy T, Mason JM, Leber B, Bray MR, Chan SM, Gupta V, Khalaf D, Maze D, McNamara CJ, Schimmer AD, Schuh AC, Sibai H, Trus M, Valiquette D, Martin K, Nguyen L, Li X, Mak TW, Minden MD, Yee KWL
Leukemia 2024 Mar;38(3):502-512. Epub 2023 Dec 19 doi: 10.1038/s41375-023-02110-9. PMID: 38114624
Natural history study of patients with familial platelet disorder with associated myeloid malignancy.
Cunningham L, Merguerian M, Calvo KR, Davis J, Deuitch NT, Dulau-Florea A, Patel N, Yu K, Sacco K, Bhattacharya S, Passi M, Ozkaya N, De Leon S, Chong S, Craft K, Diemer J, Bresciani E, O'Brien K, Andrews EJ, Park N, Hathaway L, Cowen EW, Heller T, Ryan K, Barochia A, Nghiem K, Niemela J, Rosenzweig S, Young DJ, Frischmeyer-Guerrerio PA, Braylan R, Liu PP
Blood 2023 Dec 21;142(25):2146-2158. doi: 10.1182/blood.2023019746. PMID: 37738626Free PMC Article
Decitabine/Cedazuridine: First Approval.
Dhillon S
Drugs 2020 Sep;80(13):1373-1378. doi: 10.1007/s40265-020-01389-7. PMID: 32860582Free PMC Article

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