No abstract

No abstract

No abstract

Aim: De novo relapsed and/or refractory acute myeloid leukemia (rrAML) has limited treatment options for patients not eligible (‘unfit’) to receive intensive chemotherapy-based interventions. The authors aimed to summarize outcomes for licensed therapies in this setting. Materials & methods: A systematic literature review identified licensed therapies in this setting. A feasibility assessment was made to conduct a network meta-analysis to evaluate comparative efficacy. Results: Seven unique trials were identified. Median survival months were 13.8 for gemtuzumab ozogamicin (GO), 9.3 for gilteritinib (FLT3 mutated rrAML), 5.6 for low-dose cytarabine and 3.2 for best supportive care; transplant rates with gilteritinib and GO were 25.5% and 19%, respectively. A network meta-analysis was not feasible. Conclusion: There remains a high unmet need in de novo rrAML patients not eligible for intensive therapy, with GO and gilteritinib (only FLT3 mutated AML) providing the best current options.

cardiovascular, and rare diseases), and (b) further subdivide the publications identified by type of study (pragmatic, observational, database, registry, and other). RESULTS: The initial broad search revealed that cancer had the most publications, with a total of 1,030,609 hits, followed by cardiovascular at 395,848, and rare diseases with 91,356. In 2017, of the total publications across the three disease areas, only 0.35% (881/245,575) utilized RWE, and cardiovascular was more likely than cancer or rare diseases to feature RWE in publications (2017: 0.58% vs. 0.29% vs. 0.24%). When considering study types utilizing RWE over the period searched, the most common type for each disease area varied broadly; database was the highest for cancer, observational for rare diseases, and registry for cardiovascular. Year-on-year analysis revealed that publications utilizing RWE became more frequent across the three disease areas between 2012 and present. CONCLUSIONS: Across all three disease areas, RWE publication over time was consistent or increasing. The number of papers reporting RWE remains low, which may be reflective of the lack of guidelines for reporting, and variability in the definition of RWE. Further consensus would aid the acceptance of RWE as supportive evidence.