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Table 4 Long-term results of TKI-based clinical trials for adult Ph+ (patients in complete hematologic remission), with emphasis on allogeneic stem cell transplantation (SCT)

From: Treatment and monitoring of Philadelphia chromosome-positive leukemia patients: recent advances and remaining challenges

Study (author, ref.)

No. of patients

TKI-based therapy

Treatment outcome (%)

General

SCT

No SCT

No.

Outcome

TRM

No.

Outcome

P (vs. SCT)

Pfeifer (2010) [63]

247

IM/CT

40–50 (OS, 4 years)

180

57 (OS, 3 years) 72 (DFS)

21–26

–

14 (OS, 3 years)

NR

Bassan (2010) [64]

53

IM/CT

38 (OS, 5 years)

39 (DFS)

34

46 (DFS, 5 years)

17

19

30 (OS, 2 years)

0.019 (DFS)

Ribera (2012) [65]

56

IM/CT

37–63 (EFS, 2 years)

32

NR

31

24

NR

NR

Thyagu (2012) [66]

30

IM/CT

53 (OS, 3 years)

50 (EFS)

16

56 (OS, 3 years)

70 (EFS)

37.5

14

50 (OS, 3 years)

45 (EFS, 3 years)

0.34 (OS)

0.51 (DFS)

Fielding (2014) [67]

161

IM/CT

38 (OS, 4 years)

50 (DFS)

33 (EFS)

93

52 (OS, 4 years)

72 (DFS)

49 (EFS)

NR

44

19 (OS, 4 years)

14 (DFS)

14 (EFS)

NR

Chalandon (2015) [68]

254

IM/CT

45.6 (OS, 5 years)

37.1 (EFS)

148

56.7 (OS, 5 years)

48.3 (DFS)

25.8

106

35 (OS, 5 years)

28 (DFS)

0.02 (OS)

0.03 (DFS)

Daver (2015) [69]

39

IM/CT

43 (OS. 5 years)

43 (DFS)

16

63 (DFS, 5 years)

NR

23

43 (DFS, 5 years)

0.52 (DFS)

Lim (2015) [70]

82

IM/CT

39(OS, 5 years)

33 (DFS)

56

53 (OS, 5 years)

43 (DFS)

30

26

NR

NR

Chiaretti (2016) [72]

47

IM/CT

48.8 (OS, 5 years)

45.5 (DFS, 5 years)

23

NR

13

24

NR

0.03 (OS)

Wang (2018) [71]

136

IM/CT

69.2 (OS, 4 years)

61 (DFS)

77

82.6 (OS, 4 years)

71.3 (DFS)

10

56

45.6 (OS-4 years)

43.9 (DFS)

< 0.001 (OS, DFS)

Chiaretti (2015) [58]

60

DAS/CT

58 (OS, 3 years)

49 (DFS, 3 years)

NR

NR

NR

NR

NR

NR

Ravandi (2016) [74]

83

DAS/CT

69 (OS, 3 years)

62 (DFS)

55 (EFS)

41

76 (DFS, 3 years)

0

53*

56 (OS, 3 years)

51 (DFS)

0.037 (OS)

0.038 (DFS)

Kim (2015) [73]

82

NIL/CT

72 (OS, 2 years)

57

78 (DFS, 2 years)

19

25

49 (DFS, 2 years)

0.045 (DFS)

Ottmann (2018) [78]

68

NIL/CT

47 (OS, 4 years)

42 (EFS)

24

61 (OS, 4 years)

25

44

39 (OS, 4 years)

NS

Jabbour (2018) [75]

76

PON/CT

71 (OS, 5 years)

83 (DFS, 3 years)

67 (EFS)

15

70 (OS, 3 years)

20

61

87 (OS, 3 years)

0.32 (OS)

  1. *Including eight no-protocol SCT patients
  2. Abbreviations: OS overall survival; DFS disease-free survival; EFS event-free survival, shown is a long-term estimate at 3+ years (length of follow-up); CT chemotherapy; TRM transplant-related mortality; NR not reported; NS not significant
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Journal of Hematology & Oncology

ISSN: 1756-8722

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