Study 200 efficacy and study design

In a phase 1/2 trial in adults with CP, AP, or BP CML resistant or intolerant to prior therapy
Proven efficacy in patients who have had prior TKI therapy
BOSULIF helped patients achieve a durable response in the 2nd- and 3rd-line settings1
2nd line
BOSULIF helped patients achieve MCyR after treatment with imatinib (n=262 evaluable)*
65% of responders had an MCyR lasting at least 18 months
43% of responders had an MCyR lasting at least 54 months
Median duration of MCyR was not reached at the time of analysis1
62% (n=96) of patients who achieved MCyR at any time (n=156) stayed on BOSULIF for at least 5 years1
Treatment with BOSULIF helps keep transformation rates low2†
95% of patients remained in CP while taking BOSULIF (n=269 out of 284)
– 5% of patients in CP had confirmed disease transformation to AP or BP disease while taking BOSULIF (n=15 out of 284)
*Median duration of treatment was 26 months for evaluable patients.
Transformation rates for CP 2nd-line patients.
AP=accelerated phase; BP=blast phase; CI=confidence interval; MCyR=major cytogenetic response; Ph+=Philadelphia chromosome–positive;
TKI=tyrosine kinase inhibitor.
3rd line
BOSULIF helped patients achieve MCyR after treatment with imatinib followed by dasatinib or nilotinib (n=112 evaluable)1*
64% of responders had an MCyR lasting at least 9 months
36% of responders had an MCyR lasting at least 42 months
Median duration of MCyR was not reached at the time of analysis1
Treatment with BOSULIF helps keep transformation rates low2†
96% of patients remained in CP while taking BOSULIF (n=114 out of 119)
– 4% of patients in CP had confirmed disease transformation to AP or BP disease while taking BOSULIF (n=5 out of 119)
*Median duration of treatment was 9 months for evaluable patients.
Transformation rates for CP 3rd-line patients.
AP=accelerated phase; BP=blast phase; CI=confidence interval; MCyR=major cytogenetic response
Advanced phase
Proven hematologic response rates with BOSULIF (n=132 evaluable)
Treatment with BOSULIF helps keep transformation rates low2
96% of patients in AP taking BOSULIF did not have confirmed disease transformation to BP
– 4% of patients had confirmed disease transformation to BP while taking BOSULIF (n=3 out of 79)
OHR was defined as MHR (CHR + no evidence of leukemia) or RCP. All responses were confirmed after 4 weeks. CHR for AP and BP CML: WBC count ≤ institutional ULN, platelets ≥100,000/mm3, and <450,000/mm3. ANC ≥1.0 × 109/L, no blasts or promyelocytes in peripheral blood, <5% myelocytes + metamyelocytes in bone marrow, <20% basophils in peripheral blood, and no extramedullary involvement. No evidence of leukemia: meets all other criteria for CHR except may have thrombocytopenia (platelets ≥20,000/mm3 and <100,000/mm3) and/or neutropenia (ANC ≥0.5 × 109/L and <1.0 × 109/L). RCP was defined as disappearance of features defining AP or BP but still in CP.1
ANC=absolute neutrophil count; AP=accelerated phase; BP=blast phase; CHR=complete hematologic response; MHR=major hematologic response;
OHR=overall hematologic response; Ph+=Philadelphia chromosome–positive; RCP=return to chronic phase; ULN=upper limit of normal; WBC=white blood cell.
Study design
The efficacy and safety of BOSULIF as 2nd- and 3rd-line treatment were evaluated in a long-term follow-up study1
The long-term analysis was based on a minimum of 60 months of follow-up for patients with CP CML treated with 1 prior TKI (imatinib) and a minimum of 48 months of follow-up for patients with CP CML treated with imatinib and at least 1 additional TKI (nilotinib and/or dasatinib).
BOSULIF was studied in 546 patients with Ph+ CML with resistance or intolerance to previous therapy1
BOSULIF was studied in a single-arm, phase 1/2, open-label, multicenter trial in 546 patients with CP, AP, and BP CML who had developed resistance or intolerance to either 1st-line imatinib or 1st-line imatinib and then dasatinib and/or nilotinib.
Patients were treated with BOSULIF 500 mg once daily
Of the 546 treated patients, 506 were considered evaluable for efficacy. Patients were evaluable for efficacy if they had received at least 1 dose of BOSULIF and had a valid baseline efficacy assessment
AdvP=advanced phase; AP=accelerated phase; BP=blast phase; CHR=complete hematologic response; MCyR=major cytogenetic response; OHR=overall hematologic response; Ph+=Philadelphia chromosome–positive; TKI=tyrosine kinase inhibitor.
References
1.
BOSULIF Prescribing Information. New York, NY: Pfizer Inc.
2.
Data on file. Pfizer Inc., New York, NY.