Session: 906-Outcomes Research—Malignant Conditions (Myeloid Disease): Real World Management And Outcome
Hematology Disease Topics & Pathways:
Therapies, Clinically relevant
Methods: NNT is an established and easily interpretable measure to assess the effectiveness of healthcare interventions. The clinical event estimates (ie, CR/CRh rate, CRc rate, 1-year OS, and HSCT rate) of gilteritinib and salvage chemotherapy among R/R FLT3mut+ AML patients were obtained from the ADMIRAL trial. CR/CRh was defined as the combined rate of CR and CRh. CRc was defined as the combination of CR, CR with incomplete hematologic recovery, and CR with incomplete platelet recovery. OS was defined as time from randomization to death due to any cause. The NNT is calculated as the inverse of the absolute rate difference between the event rates of gilteritinib and salvage chemotherapy. Positive NNT values represent treatment benefit, with lower values indicating greater benefit of gilteritinib over salvage chemotherapy. The 95% confidence interval (CI) of the NNT was derived from the 95% CI of the event rate difference.
Results: In the ADMIRAL trial, patients assigned to gilteritinib had significantly higher CR/CRh rates (34.0% vs 15.3%) and CRc rates (54.3% vs 21.8%) than patients assigned to salvage chemotherapy. The NNT for CR/CRh and CRc was 5.35 (95% CI: 3.66, 9.98) and 3.08 (95% CI: 2.38, 4.36), suggesting that treating five and three patients with gilteritinib instead of salvage chemotherapy would result in one additional patient achieving CR/CRh and CRc, respectively. With respect to the survival outcome, patients randomized to gilteritinib had significantly prolonged OS compared to those randomized to salvage chemotherapy (median OS: 9.3 vs 5.6 months; hazard ratio: 0.64); rates of 1-year survival were 37.1% versus 16.7%, respectively. The NNT comparing gilteritinib with salvage chemotherapy was 4.90 (95% CI: 3.29, 9.64) for 1-year OS, which suggests that treating approximately five patients with gilteritinib instead of salvage chemotherapy would lead to one additional survivor at the end of the first year. Lastly, more patients underwent HSCT in the gilteritinib arm versus the salvage chemotherapy arm (25.5% vs 15.3%); the corresponding NNT was estimated at 9.82 (95% CI: 5.40, 54.59) for gilteritinib versus salvage chemotherapy.
Conclusion: The results demonstrated that treatment with gilteritinib compared with salvage chemotherapy leads to more R/R FLT3mut+ AML patients achieving CR/CRh, CRc, and proceeding to HSCT, as well as more patients remaining alive at 1 year. This NNT analysis supports the superior clinical benefit of gilteritinib versus salvage chemotherapy in R/R FLT3mut+ AML patients.
Disclosures: Pandya: Astellas Pharma, Inc.: Current Employment. Qi: BMS: Other: Employee of Analysis Group Inc., which received consulting fees; Astellas Pharma, Inc.: Research Funding. Yang: Analysis Group Inc.: Current Employment; Takeda Pharmaceutical Company Ltd: Research Funding. Garnham: Astellas Pharma, Inc.: Current Employment. Shah: Astellas: Current Employment. Zeidan: Taiho: Consultancy, Honoraria; Celgene / BMS: Consultancy, Honoraria, Research Funding; Cardinal Health: Consultancy, Honoraria; Pfizer: Consultancy, Honoraria, Research Funding; Abbvie: Consultancy, Honoraria, Research Funding; Trovagene: Consultancy, Honoraria, Research Funding; Otsuka: Consultancy, Honoraria; CCITLA: Other; ADC Therapeutics: Research Funding; Seattle Genetics: Consultancy, Honoraria; Aprea: Research Funding; MedImmune/Astrazeneca: Research Funding; Astex: Research Funding; Daiichi Sankyo: Consultancy, Honoraria; Astellas: Consultancy, Honoraria; Acceleron: Consultancy, Honoraria; Novartis: Consultancy, Honoraria, Research Funding; Incyte: Consultancy, Honoraria, Research Funding; Jazz: Consultancy, Honoraria; Agios: Consultancy, Honoraria; Boehringer-Ingelheim: Consultancy, Honoraria, Research Funding; Epizyme: Consultancy, Honoraria; Leukemia and Lymphoma Society: Other; Ionis: Consultancy, Honoraria; Takeda: Consultancy, Honoraria, Research Funding; BeyondSpring: Consultancy, Honoraria; Cardiff Oncology: Consultancy, Honoraria, Other.
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