Effect sizes are an important component of experimental design, data analysis, and interpretation of statistical results. In some situations, an effect size of clinical or practical importance may be unknown to the researcher. In other situations, the researcher may be interested in comparing observed effect sizes to known standards to quantify clinical importance. In these cases, the notion of relative effect sizes (small, medium, large) can be useful as benchmarks. Although there is generally an extensive literature on relative effect sizes for continuous data, little of this research has focused on relative effect sizes for measures of risk that are common in epidemiological or biomedical studies. The aim of this paper, therefore, is to extend existing relative effect sizes to the relative risk, odds ratio, hazard ratio, rate ratio, and Mantel–Haenszel odds ratio for related samples. In most scenarios with equal group allocation, effect sizes of 1.22, 1.86, and 3.00 can be taken as small, medium, and large, respectively. The odds ratio for a non rare event is a notable exception and modified relative effect sizes are 1.32, 2.38, and 4.70 in that situation.

Sample size calculations are an important part of research to balance the use of resources and to avoid undue harm to participants. Effect sizes are an integral part of these calculations and meaningful values are often unknown to the researcher. General recommendations for effect sizes have been proposed for several commonly used statistical procedures. For the analysis of tables, recommendations have been given for the correlation coefficient for binary data; however, it is well known that suffers from poor statistical properties. The odds ratio is not problematic, although recommendations based on objective reasoning do not exist. This paper proposes odds ratio recommendations that are anchored to for fixed marginal probabilities. It will further be demonstrated that the marginal assumptions can be relaxed resulting in more general results.

Myelodysplastic syndromes and chronic myelomonocytic leukemia are blood disorders characterized by ineffective hematopoiesis and progressive marrow failure that can transform into acute leukemia. The DNA methyltransferase inhibitor 5-azacytidine (AZA) is the most effective pharmacological option, but only ∼50% of patients respond. A response only manifests after many months of treatment and is transient. The reasons underlying AZA resistance are unknown, and few alternatives exist for non-responders. Here, we show that AZA responders have more hematopoietic progenitor cells (HPCs) in the cell cycle. Non-responder HPC quiescence is mediated by integrin α5 (ITGA5) signaling and their hematopoietic potential improved by combining AZA with an ITGA5 inhibitor. AZA response is associated with the induction of an inflammatory response in HPCs in vivo. By molecular bar coding and tracking individual clones, we found that, although AZA alters the sub-clonal contribution to different lineages, founder clones are not eliminated and continue to drive hematopoiesis even in complete responders.