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MOLECULAR INTERNATIONAL PROGNOSTIC SCORING SYSTEM FOR MYELODYSPLASTIC SYNDROMES: A MONOCENTRIC EXPERIENCE

Diral, E. ; Bergonzi, G. ; Mastaglio, S. ; Tresoldi, C. ; Ronchi, P. ; Ponzoni, M. ; Cristante, M. ; Clerici, D. ; Vago, L. ; Bernardi, M. ; Ciceri, F.

Leukemia research reports, 2024, Vol.21, p.100431, Article 100431 [Periódico revisado por pares]

Elsevier Ltd

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  • Título:
    MOLECULAR INTERNATIONAL PROGNOSTIC SCORING SYSTEM FOR MYELODYSPLASTIC SYNDROMES: A MONOCENTRIC EXPERIENCE
  • Autor: Diral, E. ; Bergonzi, G. ; Mastaglio, S. ; Tresoldi, C. ; Ronchi, P. ; Ponzoni, M. ; Cristante, M. ; Clerici, D. ; Vago, L. ; Bernardi, M. ; Ciceri, F.
  • É parte de: Leukemia research reports, 2024, Vol.21, p.100431, Article 100431
  • Descrição: Allogeneic HSCT (aHSCT) is the only curative treatment, reserved for IPSS-R higher risk (HR, > 3.5) MDS. Molecular data have been integrated within the recently validated IPSS-Mol score system, in order to better predict clinical outcome. However, IPSS-Mol is not still used to guide clinical decisions. We aim to investigate IPSS-Mol significance in a cohort of MDS patients transplanted at our center. We retrospectively analyzed a cohort of 74 MDS patients undergoing aHSCT between 2010-2022 at our center according to IPSS-R risk score. All patients received treosulfan-based conditioning regimens and PBSC as stem cell source from matched related/unrelated or haploidentical donors. NGS testing for somatic myeloid mutations was performed retrospectively on cryopreserved marrow cells at diagnosis and MDS risk score was then re-calculated according to IPSS-Mol. 27 patients (36%) were lower risk (LR) at diagnosis and underwent aHSCT for disease progression. All the other patients were HR (IPSS-R > 3.5) and received aHSCT as upfront or consolidation treatment. At least one oncogenic mutation was found in 86.5% of cases by NGS testing. With IPSS-Mol 10 LR patients (37%) were re-stratified as HR (of note, 6/12 patients with intermediate IPSS-R ≤ 3.5), while 7 HR patients (15%) were re-stratified as LR. aHSCT remains the only curative strategy in HR MDS. NGS testing and application of IPSS-Mol allow to better prognosticate MDS, mostly in patients with LR MDS and specifically in intermediate risk (≤3.5) group. This could help in guiding treatment and specifically optimizing the use of aHSCT in MDS.
  • Editor: Elsevier Ltd
  • Idioma: Inglês
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  • Realização: ABCD-USP USP   Logos de Redes Sociais: Freepik

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