Acute myeloid leukemia patients with NUP98::NSD1 showing initially poor treatment response can benefit from FLT3 inhibitors and venetoclax as well as HSCT

Sun, Haimin; Yan, Han; Yan, Zeying; Zhu, Yong‐Mei; Yang, Guang; Zhang, Sujiang

doi:10.1007/s00277-022-05047-8

Cited by 6 publications

(3 citation statements)

References 9 publications

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“…Venetoclax combined with decitabine has been reported as a valuable method for NUP98-rearranged leukemia. 9 Considering the nature of AML with NUP98 rearrangement, 10 the presence of additional genetic risk factors such as TP53 mutation and complex karyotype, the young age, and allo-HSCT was highly recommended for this patient.…”

Section: Dear Editorsmentioning

confidence: 99%

Novel ZFPL1::NUP98 fusion gene identified in an adult acute myeloid leukemia patient

Zhu,

Chen,

Zhang

et al. 2024

Int J Lab Hematology

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Section: Dear Editorsmentioning

confidence: 99%

Novel ZFPL1::NUP98 fusion gene identified in an adult acute myeloid leukemia patient

Zhu,

Chen,

Zhang

et al. 2024

Int J Lab Hematology

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“…Over 50% of children with chemotherapyresistant AML bear NUP98 protein 21 . Similarly, adult AML patients with normal karyotype and NUP98 rearrangement showed a poor response to conventional intensive chemotherapy 13,17 , although most of them are reported from small cohorts 22 . In addition, there are no effective targeted drugs for these patients so far.…”

Section: Introductionmentioning

confidence: 99%

Allogeneic hematopoietic stem cell transplantation for adult acute myeloid leukemia patients with nucleoporin 98 (NUP98) gene rearrangements: a real-world study in China

Cao,

Zhang,

Zhao

et al. 2023

Bone Marrow Transplant

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Nucleoporin 98 (NUP98) gene rearrangements comprise a family of rare recurrent alterations in acute myeloid leukemia (AML), and conferred dismal outcomes. The e cacy and safety of allogeneic hematopoietic stem cell transplantation (allo-HSCT) were still unclear. In this retrospective, multicenter, realworld study, we enrolled 26 de novo adult AML patients with NUP98 rearrangements who received rst allo-HSCT. The 100-day cumulative incidence of grade II-IV acute graft-versus-host disease (GVHD) after allo-HSCT was 19.2% (95% CI, 3.8-34.7%) and the 2-year cumulative incidences of moderate to severe chronic GVHD after allo-HSCT was 47.5% (95% CI, 17.0-77.9%). Ten (38.5%) patient received maintenance therapies after allo-HSCT. Among the 24 patients with MRD monitoring regularly, all of them achieved MRD negative after allo-HSCT, and 21 (87.5%) achieved persistent MRD negative until the last follow-up. The 2-year cumulative incidence of relapse and non-relapse mortality after allo-HSCT was 17.2% (95% CI, 1.4-33.1%) and 4.6% (95% CI, 0-13.7%), respectively. The 2-year probabilities of leukemia-free survival and overall survival after allo-HSCT were 78.2% (95%CI, 62.8-97.2%) and 86.3% (95%CI, 73.0-100%), respectively. In summary, we rstly identify the e cacy and safety of allo-HSCT in adult AML patients with NUP98 rearrangement, which should be further con rmed in prospective cohorts with a longer follow-up.

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“…In our previous study, we have demonstrated that NUP98::NSD1 positive AML showing initially poor treatment response can benefit from FLT3 inhibitors (FLT3i) and venetoclax (VEN) as well as hematopoietic stem cell transplantation (HSCT), suggesting the important treatment role of FLT3i or/and VEN therapy and HSCT in this subgroup patients [ 8 ]. Recent studies also indicated that NUP98r in leukemogenesis may be associated with their interaction of menin with KMT2A [ 9 ].…”

mentioning

confidence: 99%

The landscape of NUP98 rearrangements clinical characteristics and treatment response from 1491 acute leukemia patients

Tian,

Zhu,

et al. 2024

Blood Cancer J.

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Acute myeloid leukemia patients with NUP98::NSD1 showing initially poor treatment response can benefit from FLT3 inhibitors and venetoclax as well as HSCT

Cited by 6 publications

References 9 publications

Novel ZFPL1::NUP98 fusion gene identified in an adult acute myeloid leukemia patient

Novel ZFPL1::NUP98 fusion gene identified in an adult acute myeloid leukemia patient

Allogeneic hematopoietic stem cell transplantation for adult acute myeloid leukemia patients with nucleoporin 98 (NUP98) gene rearrangements: a real-world study in China

The landscape of NUP98 rearrangements clinical characteristics and treatment response from 1491 acute leukemia patients

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