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- Title
Observation and treatment in DDX41-mutated acute myeloid leukemia and myelodysplastic syndrome.
- Authors
Al-Kali, Aref; Nanaa, Ahmad; Viswanatha, David; He, Rong; Nguyen, Phuong; Jevremovic, Dragan; Foran, James M.; Yi, Cecelia Arana; Greipp, Patricia T.; Gangat, Naseema; Patnaik, Mrinal; Tefferi, Ayalew; Litzow, Mark R.; Mangaonkar, Abhishek A.; Shah, Mithun Vinod; Badar, Talha; Alkhateeb, Hassan B.
- Abstract
Overall, 15 (38%) patients had germline testing (13 patients were proven to harbor at least one germline I DDX41 i -varient and two patients were proven to be somatic variants). We performed survival analysis on MDS/AML sub-group for homogeneity purpose (we excluded one MDS patient from the analysis, who was lost to follow up within 1.5 months) and found significantly better OS in observation group compared to treatment group with 5-year OS of 100% in observation group compared to median OS of 41 months in treatment group ( I p i = 0.0069) (Fig. Only 3 AML patients in treatment group were adverse risk European Leukemia Net 2022 (ELN) and all other were intermediate-risk including one AML patient in observation group (Table 1). Of the 2 MDS patients who progressed to AML under observation, one patient started on HMA + Venetoclax (VEN) therapy after progression, while the other one received induction with Daunorubicin/Cytarabine 24 months after progression.
- Subjects
ACUTE myeloid leukemia; MYELODYSPLASTIC syndromes; NUCLEOTIDE sequencing
- Publication
Blood Cancer Journal, 2023, Vol 13, Issue 1, p1
- ISSN
2044-5385
- Publication type
Article
- DOI
10.1038/s41408-023-00818-6