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Childhood Acute Myeloid Leukemia with NUP98 Rearrangement

MedGen UID:
884951
•Concept ID:
C4055248
•
Neoplastic Process

Definition

An acute myeloid leukemia that occurs in childhood and is characterized by the rearrangement of the NUP98 gene. [from NCI]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVChildhood Acute Myeloid Leukemia with NUP98 Rearrangement

Professional guidelines

PubMed

Combination of menin and kinase inhibitors as an effective treatment for leukemia with NUP98 translocations.
Miao H, Chen D, Ropa J, Purohit T, Kim E, Sulis ML, Ferrando A, Cierpicki T, Grembecka J
Leukemia 2024 Aug;38(8):1674-1687. Epub 2024 Jun 18 doi: 10.1038/s41375-024-02312-9. PMID: 38890447

Recent clinical studies

Etiology

Acute myeloid leukemias with UBTF tandem duplications are sensitive to menin inhibitors.
Barajas JM, Rasouli M, Umeda M, Hiltenbrand R, Abdelhamed S, Mohnani R, Arthur B, Westover T, Thomas ME 3rd, Ashtiani M, Janke LJ, Xu B, Chang TC, Rosikiewicz W, Xiong E, Rolle C, Low J, Krishan R, Song G, Walsh MP, Ma J, Rubnitz JE, Iacobucci I, Chen T, Krippner-Heidenreich A, Zwaan CM, Heidenreich O, Klco JM
Blood 2024 Feb 15;143(7):619-630. doi: 10.1182/blood.2023021359. PMID: 37890156Free PMC Article
A global study for acute myeloid leukemia with RARG rearrangement.
Zhu HH, Qin YZ, Zhang ZL, Liu YJ, Wen LJ, You MJ, Zhang C, Such E, Luo H, Yuan HJ, Zhou HS, Liu HX, Xu R, Li J, Li JH, Hao JP, Jin J, Yu L, Zhang JY, Liu LP, Zhang LP, Huang RB, Shen SH, Gao SJ, Wang W, Yan XJ, Zhang XY, Du X, Chu XX, Yu YF, Wang Y, Mi YC, Lu Y, Cai Z, Su Z, Taussig DC, MacMahon S, Ball ED, Wang HY, Welch JS, Yin CC, Borthakur G, Sanz MA, Kantarjian HM, Huang JY, Hu J, Chen SN
Blood Adv 2023 Jul 11;7(13):2972-2982. doi: 10.1182/bloodadvances.2022008364. PMID: 36799929Free PMC Article
Genomic subtyping and therapeutic targeting of acute erythroleukemia.
Iacobucci I, Wen J, Meggendorfer M, Choi JK, Shi L, Pounds SB, Carmichael CL, Masih KE, Morris SM, Lindsley RC, Janke LJ, Alexander TB, Song G, Qu C, Li Y, Payne-Turner D, Tomizawa D, Kiyokawa N, Valentine M, Valentine V, Basso G, Locatelli F, Enemark EJ, Kham SKY, Yeoh AEJ, Ma X, Zhou X, Sioson E, Rusch M, Ries RE, Stieglitz E, Hunger SP, Wei AH, To LB, Lewis ID, D'Andrea RJ, Kile BT, Brown AL, Scott HS, Hahn CN, Marlton P, Pei D, Cheng C, Loh ML, Ebert BL, Meshinchi S, Haferlach T, Mullighan CG
Nat Genet 2019 Apr;51(4):694-704. Epub 2019 Mar 29 doi: 10.1038/s41588-019-0375-1. PMID: 30926971Free PMC Article
Recurrent abnormalities can be used for risk group stratification in pediatric AMKL: a retrospective intergroup study.
de Rooij JD, Masetti R, van den Heuvel-Eibrink MM, Cayuela JM, Trka J, Reinhardt D, Rasche M, Sonneveld E, Alonzo TA, Fornerod M, Zimmermann M, Pigazzi M, Pieters R, Meshinchi S, Zwaan CM, Locatelli F
Blood 2016 Jun 30;127(26):3424-30. Epub 2016 Apr 25 doi: 10.1182/blood-2016-01-695551. PMID: 27114462Free PMC Article
11p15 translocations involving the NUP98 gene in childhood therapy-related acute myeloid leukemia/myelodysplastic syndrome.
Nishiyama M, Arai Y, Tsunematsu Y, Kobayashi H, Asami K, Yabe M, Kato S, Oda M, Eguchi H, Ohki M, Kaneko Y
Genes Chromosomes Cancer 1999 Nov;26(3):215-20. PMID: 10502319

Diagnosis

Combination of menin and kinase inhibitors as an effective treatment for leukemia with NUP98 translocations.
Miao H, Chen D, Ropa J, Purohit T, Kim E, Sulis ML, Ferrando A, Cierpicki T, Grembecka J
Leukemia 2024 Aug;38(8):1674-1687. Epub 2024 Jun 18 doi: 10.1038/s41375-024-02312-9. PMID: 38890447
Acute myeloid leukemias with UBTF tandem duplications are sensitive to menin inhibitors.
Barajas JM, Rasouli M, Umeda M, Hiltenbrand R, Abdelhamed S, Mohnani R, Arthur B, Westover T, Thomas ME 3rd, Ashtiani M, Janke LJ, Xu B, Chang TC, Rosikiewicz W, Xiong E, Rolle C, Low J, Krishan R, Song G, Walsh MP, Ma J, Rubnitz JE, Iacobucci I, Chen T, Krippner-Heidenreich A, Zwaan CM, Heidenreich O, Klco JM
Blood 2024 Feb 15;143(7):619-630. doi: 10.1182/blood.2023021359. PMID: 37890156Free PMC Article
A global study for acute myeloid leukemia with RARG rearrangement.
Zhu HH, Qin YZ, Zhang ZL, Liu YJ, Wen LJ, You MJ, Zhang C, Such E, Luo H, Yuan HJ, Zhou HS, Liu HX, Xu R, Li J, Li JH, Hao JP, Jin J, Yu L, Zhang JY, Liu LP, Zhang LP, Huang RB, Shen SH, Gao SJ, Wang W, Yan XJ, Zhang XY, Du X, Chu XX, Yu YF, Wang Y, Mi YC, Lu Y, Cai Z, Su Z, Taussig DC, MacMahon S, Ball ED, Wang HY, Welch JS, Yin CC, Borthakur G, Sanz MA, Kantarjian HM, Huang JY, Hu J, Chen SN
Blood Adv 2023 Jul 11;7(13):2972-2982. doi: 10.1182/bloodadvances.2022008364. PMID: 36799929Free PMC Article
Genomic subtyping and therapeutic targeting of acute erythroleukemia.
Iacobucci I, Wen J, Meggendorfer M, Choi JK, Shi L, Pounds SB, Carmichael CL, Masih KE, Morris SM, Lindsley RC, Janke LJ, Alexander TB, Song G, Qu C, Li Y, Payne-Turner D, Tomizawa D, Kiyokawa N, Valentine M, Valentine V, Basso G, Locatelli F, Enemark EJ, Kham SKY, Yeoh AEJ, Ma X, Zhou X, Sioson E, Rusch M, Ries RE, Stieglitz E, Hunger SP, Wei AH, To LB, Lewis ID, D'Andrea RJ, Kile BT, Brown AL, Scott HS, Hahn CN, Marlton P, Pei D, Cheng C, Loh ML, Ebert BL, Meshinchi S, Haferlach T, Mullighan CG
Nat Genet 2019 Apr;51(4):694-704. Epub 2019 Mar 29 doi: 10.1038/s41588-019-0375-1. PMID: 30926971Free PMC Article
Pediatric non-Down syndrome acute megakaryoblastic leukemia is characterized by distinct genomic subsets with varying outcomes.
de Rooij JD, Branstetter C, Ma J, Li Y, Walsh MP, Cheng J, Obulkasim A, Dang J, Easton J, Verboon LJ, Mulder HL, Zimmermann M, Koss C, Gupta P, Edmonson M, Rusch M, Lim JY, Reinhardt K, Pigazzi M, Song G, Yeoh AE, Shih LY, Liang DC, Halene S, Krause DS, Zhang J, Downing JR, Locatelli F, Reinhardt D, van den Heuvel-Eibrink MM, Zwaan CM, Fornerod M, Gruber TA
Nat Genet 2017 Mar;49(3):451-456. Epub 2017 Jan 23 doi: 10.1038/ng.3772. PMID: 28112737Free PMC Article

Therapy

Combination of menin and kinase inhibitors as an effective treatment for leukemia with NUP98 translocations.
Miao H, Chen D, Ropa J, Purohit T, Kim E, Sulis ML, Ferrando A, Cierpicki T, Grembecka J
Leukemia 2024 Aug;38(8):1674-1687. Epub 2024 Jun 18 doi: 10.1038/s41375-024-02312-9. PMID: 38890447
A global study for acute myeloid leukemia with RARG rearrangement.
Zhu HH, Qin YZ, Zhang ZL, Liu YJ, Wen LJ, You MJ, Zhang C, Such E, Luo H, Yuan HJ, Zhou HS, Liu HX, Xu R, Li J, Li JH, Hao JP, Jin J, Yu L, Zhang JY, Liu LP, Zhang LP, Huang RB, Shen SH, Gao SJ, Wang W, Yan XJ, Zhang XY, Du X, Chu XX, Yu YF, Wang Y, Mi YC, Lu Y, Cai Z, Su Z, Taussig DC, MacMahon S, Ball ED, Wang HY, Welch JS, Yin CC, Borthakur G, Sanz MA, Kantarjian HM, Huang JY, Hu J, Chen SN
Blood Adv 2023 Jul 11;7(13):2972-2982. doi: 10.1182/bloodadvances.2022008364. PMID: 36799929Free PMC Article
A Novel NUP98/RARG Gene Fusion in Pediatric Acute Myeloid Leukemia Resembling Acute Promyelocytic Leukemia.
Zhang J, Shen H, Song H, Shen D, Liao C, Fang M, Wang Y, Tang Y, Zhu H
J Pediatr Hematol Oncol 2022 Apr 1;44(3):e665-e671. doi: 10.1097/MPH.0000000000002331. PMID: 35319505
Screening for NUP98 rearrangements in hematopoietic malignancies by fluorescence in situ hybridization.
Nebral K, König M, Schmidt HH, Lutz D, Sperr WR, Kalwak K, Brugger S, Dworzak MN, Haas OA, Strehl S
Haematologica 2005 Jun;90(6):746-52. PMID: 15951287
11p15 translocations involving the NUP98 gene in childhood therapy-related acute myeloid leukemia/myelodysplastic syndrome.
Nishiyama M, Arai Y, Tsunematsu Y, Kobayashi H, Asami K, Yabe M, Kato S, Oda M, Eguchi H, Ohki M, Kaneko Y
Genes Chromosomes Cancer 1999 Nov;26(3):215-20. PMID: 10502319

Prognosis

A global study for acute myeloid leukemia with RARG rearrangement.
Zhu HH, Qin YZ, Zhang ZL, Liu YJ, Wen LJ, You MJ, Zhang C, Such E, Luo H, Yuan HJ, Zhou HS, Liu HX, Xu R, Li J, Li JH, Hao JP, Jin J, Yu L, Zhang JY, Liu LP, Zhang LP, Huang RB, Shen SH, Gao SJ, Wang W, Yan XJ, Zhang XY, Du X, Chu XX, Yu YF, Wang Y, Mi YC, Lu Y, Cai Z, Su Z, Taussig DC, MacMahon S, Ball ED, Wang HY, Welch JS, Yin CC, Borthakur G, Sanz MA, Kantarjian HM, Huang JY, Hu J, Chen SN
Blood Adv 2023 Jul 11;7(13):2972-2982. doi: 10.1182/bloodadvances.2022008364. PMID: 36799929Free PMC Article
Genomic subtyping and therapeutic targeting of acute erythroleukemia.
Iacobucci I, Wen J, Meggendorfer M, Choi JK, Shi L, Pounds SB, Carmichael CL, Masih KE, Morris SM, Lindsley RC, Janke LJ, Alexander TB, Song G, Qu C, Li Y, Payne-Turner D, Tomizawa D, Kiyokawa N, Valentine M, Valentine V, Basso G, Locatelli F, Enemark EJ, Kham SKY, Yeoh AEJ, Ma X, Zhou X, Sioson E, Rusch M, Ries RE, Stieglitz E, Hunger SP, Wei AH, To LB, Lewis ID, D'Andrea RJ, Kile BT, Brown AL, Scott HS, Hahn CN, Marlton P, Pei D, Cheng C, Loh ML, Ebert BL, Meshinchi S, Haferlach T, Mullighan CG
Nat Genet 2019 Apr;51(4):694-704. Epub 2019 Mar 29 doi: 10.1038/s41588-019-0375-1. PMID: 30926971Free PMC Article
Recurrent abnormalities can be used for risk group stratification in pediatric AMKL: a retrospective intergroup study.
de Rooij JD, Masetti R, van den Heuvel-Eibrink MM, Cayuela JM, Trka J, Reinhardt D, Rasche M, Sonneveld E, Alonzo TA, Fornerod M, Zimmermann M, Pigazzi M, Pieters R, Meshinchi S, Zwaan CM, Locatelli F
Blood 2016 Jun 30;127(26):3424-30. Epub 2016 Apr 25 doi: 10.1182/blood-2016-01-695551. PMID: 27114462Free PMC Article

Clinical prediction guides

Recurrent abnormalities can be used for risk group stratification in pediatric AMKL: a retrospective intergroup study.
de Rooij JD, Masetti R, van den Heuvel-Eibrink MM, Cayuela JM, Trka J, Reinhardt D, Rasche M, Sonneveld E, Alonzo TA, Fornerod M, Zimmermann M, Pigazzi M, Pieters R, Meshinchi S, Zwaan CM, Locatelli F
Blood 2016 Jun 30;127(26):3424-30. Epub 2016 Apr 25 doi: 10.1182/blood-2016-01-695551. PMID: 27114462Free PMC Article
Functional analysis of the NUP98-CCDC28A fusion protein.
Petit A, Ragu C, Soler G, Ottolenghi C, Schluth C, Radford-Weiss I, Schneider-Maunoury S, Callebaut I, Dastugue N, Drabkin HA, Bernard OA, Romana S, Penard-Lacronique V
Haematologica 2012 Mar;97(3):379-87. Epub 2011 Nov 4 doi: 10.3324/haematol.2011.047969. PMID: 22058212Free PMC Article
Screening for NUP98 rearrangements in hematopoietic malignancies by fluorescence in situ hybridization.
Nebral K, König M, Schmidt HH, Lutz D, Sperr WR, Kalwak K, Brugger S, Dworzak MN, Haas OA, Strehl S
Haematologica 2005 Jun;90(6):746-52. PMID: 15951287

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