. Author manuscript; available in PMC: 2021 Dec 10.

Published in final edited form as: Clin Cancer Res. 2020 Jan 14;26(9):2104–2110. doi: 10.1158/1078-0432.CCR-19-2591

Table 5A.

Comparison of frequency of grade ≥3 cytopenias between radium-223 + enzalutamide arm with ALSYMPCA trial.

AEs	Agarwal et al N=35	95% CI	Parker et al [4] (ALSYMPCA) N=600	P-value (one sided)
Hematologic, N (%) ^a	4 (13.8%)	4.2% - 30.5%	128 (21.3%)	0.91
Neutropenia, N (%) ^b	3 (8.6%)	1.8% - 23.1%	13 (2.2%)	0.041
Anemia, N (%) ^b	0 (0%)	0% - 10%	76 (12.7%)	1.00
Thrombocytopenia, N (%) ^b	1 (2.8%)	0% - 14.9%	39 (6.5%)	0.90

Hematologic AEs includes all neutropenia, anemia, and thrombocytopenia events and is used for comparison to the ALSYMPCA trial [5, 6]. For the comparison of 4/35 events in the Agarwal et al study versus 128/600 events in the ALSYMPCA trial [5, 6], a maximum likelihood test from a Poisson model was used. With a safety population of 35 evaluable patients, ≥13 events of grade ≥3 hematologic AEs would indicate an unacceptable rate of hematologic AEs.

An exact binomial test was performed comparing the proportion of neutropenia, anemia, or thrombocytopenia subjects with grade ≥3 events in the Agarwal et al study to the proportions of neutropenia, anemia, or thrombocytopenia subjects in the ALSYMPCA trial [5, 6].