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Table 4 Baseline characteristics and disposition at end-of-study3

From: Retrospective analysis of arterial occlusive events in the PACE trial by an independent adjudication committee

 

CP-CML

n = 270

Total

N = 449

Characteristic at baseline

Median age (range), y

60 (18–94)

59 (18–94)

Female, n (%)

126 (47)

211 (47)

Previous use of approved TKIs, n (%)a

 ≥ 2 drugs

251 (93)

417 (93)

 ≥ 3 drugs

154 (57)

250 (56)

Median duration of previous treatment with approved TKIs (range), ya

5.4 (0.4–13.3)

4.6 (0.1–13.3)

Resistant or intolerant to dasatinib or nilotinib, n (%)

 Resistant

215 (80)

375 (84)

 Intolerant only

39 (14)

49 (11)

 Both resistant and intolerant

52 (19)

81 (18)

Mutation status, n (%)b

 No mutation detected

138 (51)

198 (44)

 BCR::ABL1T315I

64 (24)

128 (29)

Best response of MMR or better to most recent regimen containing dasatinib or nilotinib, n (%)c

8 (3)

16 (4)

Baseline cardiovascular risk factorsd

 Arterial hypertension

NA

240 (53)

 Hypercholesterolemia

NA

219 (49)

 Obesity

NA

109 (24)

 Diabetes mellitus

NA

72 (16)

Baseline history of cardiovascular disease

 Non-ischemic cardiac disease

NA

193 (43)

 Ischemic disease

NA

102 (23)

Patient disposition at end of study

Median duration of treatment, mo (range)

32.1 (0.1–73.0)

16.7 (0.03–73.0)

Median follow-up, mo (range)

56.8 (0.1–73.1)

37.3 (0.1–73.1)

Median dose intensity, mg/d (range)

27.2 (5–45)

ND

Primary reason for discontinuation, n (%)

 Disease progression

29 (11)

105 (23)

 Adverse event

57 (21)

79 (18)

 Patient request

31 (11)

42 (9)

 Lack of efficacy

15 (6)

26 (6)

 Deathe

9 (3)

26 (6)

 Investigator decision

11 (4)

17 (4)

 Lost to follow-up

0

3 (< 1)

 Non-compliance

3 (1)

4 (< 1)

 Protocol violation

2 (< 1)

2 (< 1)

 Study closuref

90 (33)

107 (24)

 Otherf,g

14 (5)

28 (6)

  1. CML chronic myeloid leukemia, CP chronic phase, MMR major molecular response, ND not determined, TKI tyrosine kinase inhibitor
  2. aApproved TKIs were imatinib, nilotinib, dasatinib, and bosutinib. Previous investigational TKIs received by at least 1% of patients included radotinib (received by 2% of patients), bafetinib (2%), rebastinib (2%), and XL-228 (2%)
  3. bAssessed by conventional Sanger sequencing at baseline
  4. cPercentages were calculated according to the number of patients who received previous dasatinib or nilotinib: 256 patients with CP-CML, 80 patients with AP-CML, 61 patients with BP-CML, and 30 patients with Ph+ ALL
  5. dSmoking and family history were not collected as part of the trial. Patients with significant or active cardiovascular disease, including myocardial infarction, unstable angina or congestive heart failure (in prior 3 months), or history of clinically significant atrial or ventricular arrhythmia were excluded from the trial
  6. eSeven deaths were assessed by investigators as possibly or probably related to ponatinib (CP-CML: pneumonia, acute myocardial infarction; AP-CML: fungal pneumonia, gastrointestinal hemorrhage; BP-CML: hemorrhagic gastritis; Ph+ ALL: cardiac arrest, mesenteric arterial occlusion)
  7. fPatients who continued to derive clinical benefit from their treatment had the option to receive ponatinib through alternative mechanisms
  8. gThis category includes stem cell transplantation (in 11 patients with CP-CML, 5 with AP-CML, 6 with BP-CML, and 1 with Ph+ ALL). The 9 CP-CML patients and 1 AP-CML patient who remained on study at the time of last response assessment are not included in this category.3