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Association details:
| Biomarker: | NPM1 mutation |
|---|---|
| Cancer: | Acute Myelogenous Leukemia |
| Drug: | azacitidine (DNMT inhibitor) |
| Direction: | Sensitive |
Evidence:
Source:
Title:
Prognostic impact of NPM1 and FLT3 mutations in patients with AML in first remission treated with oral azacitidine
Published date:
10/13/2022
Excerpt:
Among patients with NPM1mut, OS and RFS were improved with Oral-AZA by 37% (hazard ratio [HR], 0.63; 95% confidence interval [CI], 0.41-0.98) and 45% (HR, 0.55; 95% CI, 0.35-0.84), respectively, vs placebo. Median OS was improved numerically with Oral-AZA among patients with NPM1mut whether without MRD (48.6 months vs 31.4 months with placebo) or with MRD (46.1 months vs 10.0 months with placebo) post-IC. Among patients with FLT3mut, Oral-AZA improved OS and RFS by 37% (HR, 0.63; 95% CI, 0.35-1.12) and 49% (HR, 0.51; 95% CI, 0.27-0.95), respectively, vs placebo.
DOI:
10.1182/blood.2022016293
Trial ID:
Source:
Title:
Long-Term Overall Survival (OS) with Oral Azacitidine (Oral-AZA) in Patients with Acute Myeloid Leukemia (AML) in First Remission after Intensive Chemotherapy (IC): Updated Results from the Phase 3 QUAZAR AML-001 Trial
Published date:
12/24/2021
Excerpt:
In all, 472 pts were randomized to Oral-AZA (n = 238) or PBO (n = 234)….those in the LT Survivors group were more likely to have intermediate-risk cytogenetics (95% vs. 82%) and an NPM1 mutation (45% vs. 9%) at AML Dx, and less likely to be MRD+ at BL (33% vs. 52%)....Intermediate-risk cytogenetics and NPM1 mutations at AML Dx, and absence of detectable MRD post-IC, were associated with long-term survival in QUAZAR AML-001.
DOI:
10.1182/blood-2021-147501
Trial ID:
Source:
Title:
A Study of Safety, Efficacy and Pharmacodynamics of Azacitidine in Children and Young Adults With Acute Myeloid Leukemia.
Excerpt:
...NPM1 mutation 5....
Trial ID:
Source:
Title:
Treatment of MDS/AML patients with an impending hematological relapse with azacitidine alone or in combination with pevonedistat Behandlung von MDS/AML Patienten mit drohendem hämatologischem Rezidiv mit Azacytidin alleine oder Azacytidin in Kombination mit Pevonedistat
Excerpt:
...- AML or MDS- continuing first CR after conventional intensive chemotherapy OR continuing CR after alloSCT- MRD positivity (assessed by local lab) as defined by:o NPM1mut status >1% in peripheral blood or bone marrow in NPM1 mutated patients at diagnosis oro Patients after allogeneic transplantation, who were NPM1 unmutated at diagnosis and have a blood or marrow CD34/CD117 chimerism <80%...
Trial ID:
Less C2 evidence
Source:
Title:
Prognostic Impact of NPM1 and FLT3 Mutations at Diagnosis and Presence of Measurable Residual Disease (MRD) after Intensive Chemotherapy (IC) for Patients with Acute Myeloid Leukemia (AML) in Remission: Outcomes from the QUAZAR AML-001 Trial of Oral Azacitidine (Oral-AZA) Maintenance
Published date:
11/04/2021
Excerpt:
In QUAZAR AML-001, pts aged ≥55 years with AML and NCCN intermediate or poor-risk cytogenetics at Dx were randomized 1:1 to receive Oral-AZA 300 mg or PBO QD within 4 mo after attaining first CR/CRi with IC (induction ± consolidation)....In MV analyses, Oral-AZA significantly improved OS vs PBO when adjusted for other variables (P = 0.035); NPM1 status (P = 0.001), FLT3 status (P = 0.035), and cytogenetic risk at Dx (P < 0.001) were each also significantly predictive of OS, as was post-IC MRD status (P < 0.001).
DOI:
10.1182/blood-2021-147465
Trial ID:
Source:
Title:
SURVIVAL OUTCOMES FROM THE QUAZAR AML-001 TRIAL WITH ORAL AZACITIDINE FOR PATIENTS WITH ACUTE MYELOID LEUKEMIA IN REMISSION BY DISEASE SUBTYPE, CYTOGENETIC RISK, AND NPM1 MUTATION STATUS AT DIAGNOSIS
Published date:
05/12/2021
Excerpt:
...pts with mutNPM1 in the Oral-AZA arm derived an extended OS benefit of more than 2.5 years vs PBO...
Source:
Title:
Azacitidine to Consolidate and Deepen the Therapeutic Response Achieved by Intensive Induction Treatment in a Young Patient Affected by NPM1mut-AML Who Has Become Ineligible for High-Dose Consolidation
Published date:
01/12/2022
Excerpt:
We report the case of a young patient with NPM1mut-AML who underwent a first cycle of intensive induction treatment, achieving a complete remission...Due to the ineligibility to further lines of intensive chemotherapy, we decided to consolidate the response with azacitidine, administered according to the regular schedule. The minimal residual disease (MRD), monitored through the NPM1 mutation at diagnosis, progressively decreased and became undetectable after 36 cycles of hypomethylating therapy.
DOI:
10.1159/000520205
