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Refractory Myelodysplastic/Myeloproliferative Neoplasm

MedGen UID:
1754843
•Concept ID:
C5419091
•
Neoplastic Process

Definition

A myelodysplastic/myeloproliferative neoplasm that is resistant to treatment. [from NCI]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVRefractory Myelodysplastic/Myeloproliferative Neoplasm

Professional guidelines

PubMed

Myeloid progenitors with PTPN11 and nonRAS pathway gene mutations are refractory to treatment with 6-mercaptopurine in juvenile myelomonocytic leukemia.
Matsuda K, Nakazawa Y, Iwashita C, Kurata T, Hirabayashi K, Saito S, Tanaka M, Yoshikawa K, Yanagisawa R, Sakashita K, Sasaki S, Honda T, Koike K
Leukemia 2014 Jul;28(7):1545-8. Epub 2014 Feb 5 doi: 10.1038/leu.2014.58. PMID: 24496301
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
The myelodysplastic syndromes: diagnostic criteria and laboratory evaluation.
Ho PJ, Gibson J, Vincent P, Joshua D
Pathology 1993 Jul;25(3):297-304. doi: 10.3109/00313029309066593. PMID: 8265251

Recent clinical studies

Etiology

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
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
Allogeneic stem cell transplantation for trisomy 8-positive myelodysplastic syndrome or myelodysplastic/myeloproliferative disease with refractory Behçet's disease: Case report and the review of literature.
Onaka T, Nakano K, Uemoto Y, Miyakawa N, Otsuka Y, Ogura-Kato A, Iwai F, Tanaka Y, Yonezawa A
Mod Rheumatol Case Rep 2022 Jun 24;6(2):273-277. doi: 10.1093/mrcr/rxac032. PMID: 35365826
Decitabine/Cedazuridine: First Approval.
Dhillon S
Drugs 2020 Sep;80(13):1373-1378. doi: 10.1007/s40265-020-01389-7. PMID: 32860582Free PMC Article
Challenges in Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPN).
Smith BN, Savona M, Komrokji RS
Clin Lymphoma Myeloma Leuk 2019 Jan;19(1):1-8. Epub 2018 Nov 23 doi: 10.1016/j.clml.2018.11.019. PMID: 30555034Free PMC Article

Diagnosis

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
Allogeneic stem cell transplantation for trisomy 8-positive myelodysplastic syndrome or myelodysplastic/myeloproliferative disease with refractory Behçet's disease: Case report and the review of literature.
Onaka T, Nakano K, Uemoto Y, Miyakawa N, Otsuka Y, Ogura-Kato A, Iwai F, Tanaka Y, Yonezawa A
Mod Rheumatol Case Rep 2022 Jun 24;6(2):273-277. doi: 10.1093/mrcr/rxac032. PMID: 35365826
Challenges in Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPN).
Smith BN, Savona M, Komrokji RS
Clin Lymphoma Myeloma Leuk 2019 Jan;19(1):1-8. Epub 2018 Nov 23 doi: 10.1016/j.clml.2018.11.019. PMID: 30555034Free PMC Article
Myelodysplastic/myeloproliferative neoplasms.
Hyjek E, Vardiman JW
Semin Diagn Pathol 2011 Nov;28(4):283-97. doi: 10.1053/j.semdp.2011.07.002. PMID: 22195406
Myelodysplastic/myeloproliferative neoplasms.
Cazzola M, Malcovati L, Invernizzi R
Hematology Am Soc Hematol Educ Program 2011;2011:264-72. doi: 10.1182/asheducation-2011.1.264. PMID: 22160044

Therapy

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
Allogeneic stem cell transplantation for trisomy 8-positive myelodysplastic syndrome or myelodysplastic/myeloproliferative disease with refractory Behçet's disease: Case report and the review of literature.
Onaka T, Nakano K, Uemoto Y, Miyakawa N, Otsuka Y, Ogura-Kato A, Iwai F, Tanaka Y, Yonezawa A
Mod Rheumatol Case Rep 2022 Jun 24;6(2):273-277. doi: 10.1093/mrcr/rxac032. PMID: 35365826
Decitabine/Cedazuridine: First Approval.
Dhillon S
Drugs 2020 Sep;80(13):1373-1378. doi: 10.1007/s40265-020-01389-7. PMID: 32860582Free PMC Article
Focus on myeloproliferative diseases and myelodysplastic syndromes.
Van Etten RA, Shannon KM
Cancer Cell 2004 Dec;6(6):547-52. doi: 10.1016/j.ccr.2004.12.004. PMID: 15607959
New treatment approaches for myelodysplastic syndrome and secondary leukaemias.
De Witte T
Ann Oncol 1994 May;5(5):401-8. doi: 10.1093/oxfordjournals.annonc.a058870. PMID: 8075047

Prognosis

Decitabine/Cedazuridine: First Approval.
Dhillon S
Drugs 2020 Sep;80(13):1373-1378. doi: 10.1007/s40265-020-01389-7. PMID: 32860582Free PMC Article
der(5;17)(p10;q10) is a recurrent but rare whole-arm translocation in patients with hematological neoplasms: a report of three cases.
Manabe M, Okita J, Tarakuwa T, Harada N, Aoyama Y, Kumura T, Ohta T, Furukawa Y, Mugitani A
Acta Haematol 2014;132(2):134-9. doi: 10.1159/000357111. PMID: 24556628
Myelodysplastic/myeloproliferative neoplasms.
Cazzola M, Malcovati L, Invernizzi R
Hematology Am Soc Hematol Educ Program 2011;2011:264-72. doi: 10.1182/asheducation-2011.1.264. PMID: 22160044
Trisomy 14 in myeloid malignancies: report of two cases and review of the literature.
Horton YM, Johnson PR
Cancer Genet Cytogenet 2001 Jan 15;124(2):172-4. doi: 10.1016/s0165-4608(00)00345-9. PMID: 11172913
Oncogene mutation and prognosis in the myelodysplastic syndromes.
Padua RA, West RR
Br J Haematol 2000 Dec;111(3):873-4. PMID: 11122149

Clinical prediction guides

Clinical Application for Diagnosis of Myelodysplatic/Myeloproliferative Neoplasm with Ring Sideroblasts and Thrombocytosis.
Lee MY, Jang S, Park CJ, Cho YU
Clin Lab 2022 Apr 1;68(4) doi: 10.7754/Clin.Lab.2021.210738. PMID: 35443589
Myelodysplastic/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T): Mayo-Moffitt collaborative study of 158 patients.
Mangaonkar AA, Lasho TL, Ketterling RP, Reichard KK, Gangat N, Al-Kali A, Begna KH, Pardanani A, Al Ali NH, Talati C, Sallman D, Padron E, Patnaik MM, Tefferi A, Komrokji R
Blood Cancer J 2022 Feb 1;12(2):26. doi: 10.1038/s41408-022-00622-8. PMID: 35105856Free 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
Splicing factor mutations in MDS RARS and MDS/MPN-RS-T.
Yoshimi A, Abdel-Wahab O
Int J Hematol 2017 Jun;105(6):720-731. Epub 2017 May 2 doi: 10.1007/s12185-017-2242-0. PMID: 28466384
The genomic landscape of juvenile myelomonocytic leukemia.
Stieglitz E, Taylor-Weiner AN, Chang TY, Gelston LC, Wang YD, Mazor T, Esquivel E, Yu A, Seepo S, Olsen S, Rosenberg M, Archambeault SL, Abusin G, Beckman K, Brown PA, Briones M, Carcamo B, Cooper T, Dahl GV, Emanuel PD, Fluchel MN, Goyal RK, Hayashi RJ, Hitzler J, Hugge C, Liu YL, Messinger YH, Mahoney DH Jr, Monteleone P, Nemecek ER, Roehrs PA, Schore RJ, Stine KC, Takemoto CM, Toretsky JA, Costello JF, Olshen AB, Stewart C, Li Y, Ma J, Gerbing RB, Alonzo TA, Getz G, Gruber T, Golub T, Stegmaier K, Loh ML
Nat Genet 2015 Nov;47(11):1326-1333. Epub 2015 Oct 12 doi: 10.1038/ng.3400. PMID: 26457647Free PMC Article

Recent systematic reviews

Comparison of a restrictive versus liberal red cell transfusion policy for patients with myelodysplasia, aplastic anaemia, and other congenital bone marrow failure disorders.
Gu Y, Estcourt LJ, Doree C, Hopewell S, Vyas P
Cochrane Database Syst Rev 2015 Oct 5;2015(10):CD011577. doi: 10.1002/14651858.CD011577.pub2. PMID: 26436602Free PMC Article

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