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. 2024 May 9;331(21):1814–1823. doi: 10.1001/jama.2024.4783

Table 2. Minimum Residual Disease Negativity With Complete Response at End of Induction: Response Rates and Durability of Responsea.

Outcomes Ponatinib (n = 154) Imatinib (n = 78) Risk difference (95% CI) Relative risk (95% CI) P value
Primary end point: MRD-negative complete remission at end of induction, No. (%)b 53 (34.4) 13 (16.7) 0.18 (0.06-0.29) 2.06 (1.19-3.56)c .002c
MRD negativity (MR4) at end of induction, No. (%) 61 (43.0)d 15 (22.1)d 0.21 (0.08-0.34) 1.94 (1.19-3.17)c .002c
Duration of MRD-negative complete remission n = 53 n = 13
Loss-of-response events, No. (%) 9 (17.0) 6 (46.2)
Duration, median (95% CI), mo NE (16.6-NE) 18.0 (8.4-27.8) NA NA

Abbreviations: MRD, minimal residual disease; NA, not available; NE, not estimable.

a

Outcomes were evaluated in the population of patients in the intention-to-treat population with p190/p210 dominant isoforms confirmed by the central laboratory.

b

The composite primary end point required central laboratory–reported MRD negativity (defined as ≤0.01% BCR::ABL1IS [MR4]) and investigator-reported complete remission (bone marrow blast response with hematologic recovery as measured by absolute neutrophil and platelet counts) for ≥4 weeks at the end of induction.

c

Adjusted relative risks, 95% CIs, and P values were calculated based on a Cochran-Mantel-Haenszel χ2 test, with stratification according to randomization strata (ages 18 to <45 years, ≥45 to <60 years, and ≥60 years).

d

Among patients with evaluable samples at end of trial cycle 3 (n = 142 for ponatinib and n = 68 for imatinib).