Table 2.

Clinical outcomes of patients with newly diagnosed CML-CP who achieved deep molecular responses with TKI therapy

Outcomes for patients with or without deep molecular response, %
Trial descriptionNMedian f/u, moComparatorsEFS ratePFS rateOS rateTFS rate
Etienne et al. (30): first-line IM 400 mg26653.2CCyR + MMR + CMRa CCyR + MMR − CMRa CCyR − MMR956528988256OS was not different among the 3 groupsNRNRNR
Falchi et al. (31): first-line IM (400 mg, n = 83; 800 mg, n = 204), NIL (n = 106), or DAS (n = 102)49573No MR at 18 moMMR at 18 moMR4 at 18 moMR4.5 at 18 moUndetectable BCR-ABLb at 18 moNo MR at 24 moMMR at 24 moMR4 at 24 moMR4.5 at 24 mo78 (6 y)94 (6 y)97 (6 y)93 (6 y)100 (6 y)80 (6 y)90 (6 y)97 (6 y)95 (6 y)NRNRNRNRNRNRNRNRNR93 (6 y) 98 (6 y) 97 (6 y) 95 (6 y) 100 (6 y) 92 (6 y) 97 (6 y) 100 (6 y) 97 (6 y)90 (6 y) 100 (6 y) 100 (6 y) 100 (6 y) 100 (6 y) 90 (6 y) 100 (6 y) 100 (6 y) 100 (6 y)
Undetectable BCR-ABLb at 24 mo100 (6 y)NR100 (6 y)100 (6 y)
CML study IV (32): first-line IM 400 mg, IM 400 mg + IFN-α, IM 400 mg + AraC, IM after IFN-α failure, or IM 800 mg1,52567.5After a median duration of MR4 of 3.7 y, only 4 of 792 patients with CMR4 (0.5%) progressed; life expectancy with MR4 and MR4.5 was identical to that of the age-matched population
Kantarjian et al. (28): retrospective analysis. IM (400 mg, n = 71; 800 mg, n = 205)27648Durable CMRb (≥6 mo) Nondurable CMRb (<6 mo)NR NR100 (5 y) 98 (5 y)NR NRNR NR
Verma et al. (29): retrospective analysis. First-line IM (400 mg, n = 73; 800 mg, n = 208)28165CCyR + MMR + CMRb at 2 y CCyR + MMR − CMRb at 2 y CCyR − MMR − CMRb at 2 y100 (5 y) 96 (5 y) 86 (5 y)NR NR NRNR NR NR100 (5 y) 96 (5 y) 91 (5 y)
Press et al. (77): all patients achieved CCyR on IM and had ≥2 measurements of BCR–ABL1 level after achieving CCyR9049MMR + CMRc MMR − CMRcNR NRNot reachedd 44 modNR NRNR NR

Abbreviations: AraC, cytarabine; DAS, dasatinib; f/u, follow-up; IM, imatinib; MR, molecular response; NIL, nilotinib; NR, not reported.

  • aCMR was defined as undetectable BCR–ABL1 transcripts with a sensitivity of ≥4.5 logs on 2 consecutive analyses ≥2 mo apart.

  • bMinimum sensitivity 4.5 logs.

  • cMinimum sensitivity 4 logs.

  • dData shown are median relapse-free survival, defined as progression to accelerated phase/blast crisis, loss of complete hematologic response, or loss of CCyR.