Table 1.

Summary of the efficacy data for refractory/intolerant CP-CML

Dsatininb (32)Nilotinib (24, 33)Bosutinib (34)Ponatinib (refs. 25, 26; phase I trial)Ponatinib (27) PACE trialOmacetaxine (35)
cPatients, n101 (100%-R)321 (70%-R)288 (69%-R)4320346
Prior TKIs111≥2%–98%≥2%–98%≥2–85%
≥3%–63%≥3%–67%≥3–59%
Follow-up, y2221.91.31.6
CHR (%)93748698N/R61
MCyR (%)535953725122
CCyR (%)44444165464
MMR (%)292841c5132None
2-year PFS>80%64%79%83% MCyRa91% MCyRb7 months
55% MMR
2-year OS90%87%92%N/RN/R30.1 months

Abbreviations: CCyr, complete cytologic response; CHR, complete hematologic response; MMR, major molecular response; PFS, progression-free survival.

  • aEstimated 83% of MCyR and 55% if MMR will maintain their responses at 2 years.

  • bEstimated 91% of MCyR will maintain their responses at 1 year.

  • cReported in all treated patients regardless of the CCyR status; not measured in 4 countries participating in the trial.