2019
DOI: 10.1182/bloodadvances.2018026989
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NPM1 mutations define a specific subgroup of MDS and MDS/MPN patients with favorable outcomes with intensive chemotherapy

Abstract: Nucleophosmin (NPM1) mutations are common in acute myeloid leukemia and are associated with high remission rates and prolonged survival with intensive chemotherapy. NPM1 mutations are rare in myelodysplastic syndromes (MDS) or myelodysplastic/myeloproliferative neoplasm (MDS/MPN), and the clinical outcomes of these patients, when treated with intensive chemotherapy, are unknown. We retrospectively evaluated the clinicopathologic characteristics and the impact of therapy in 31 patients with MDS or MDS/MPN and N… Show more

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Cited by 101 publications
(105 citation statements)
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References 40 publications
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“…Montaban‐Bravo et al recently reported significantly higher CR rates by intensive chemotherapy when compared to HMA therapy in patients with NPM1 mutations 6 . However, in this study, three out of the five patients (with NPM1 mut DNMT3A mut ) did not show any response to at least three cycles of subsequent chemotherapy after unsuccessful decitabine therapy.…”
Section: Figurecontrasting
confidence: 68%
“…Montaban‐Bravo et al recently reported significantly higher CR rates by intensive chemotherapy when compared to HMA therapy in patients with NPM1 mutations 6 . However, in this study, three out of the five patients (with NPM1 mut DNMT3A mut ) did not show any response to at least three cycles of subsequent chemotherapy after unsuccessful decitabine therapy.…”
Section: Figurecontrasting
confidence: 68%
“…These are summarized in Table 5. One consists of the combination of azacitidine with the BCL‐2 inhibitor venetoclax, following successful data with this combination in AML 163 . Data from a multicenter trial of azacitidine and venetoclax was presented at the 2019 ASH Annual Meeting 156 .…”
Section: Risk Adapted Therapymentioning
confidence: 99%
“… b Low, low allelic ratio (<0.5); high, high allelic ratio (at least 0.5); semi‐quantitative assessment of FLT3 ‐ITD allelic ratio (using DNA fragment analysis) is determined as ratio of the area under the curve “ FLT3 ‐ITD” divided by area under the curve “ FLT3 ‐ wild type”; recent studies indicate that AML with NPM1 mutation and FLT3 ‐ITD low allelic ratio may also have a more favorable prognosis and patients should not routinely be assigned to allogeneic HCT 57‐59,61 …”
Section: Therapy Issuesmentioning
confidence: 99%
“…The same may apply when deciding on intensity of induction. For example, Montalban‐Bravo et al have reported that in patients with MDS and NPM1 mutations induction with higher doses of cytarabine, rather than HMA, was associated with longer survival and EFS 61 …”
Section: Therapy Issuesmentioning
confidence: 99%