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J. Koreth et al.
rates in arm A vs. B and arm A vs. C in parallel. We projected the incidence of grade II-IV aGvHD as 40% in arm A and 15% in arm B and in arm C. With the sample size of 46 per arm, there is an 80% power to detect a 25% difference in grade II-IV aGvHD rate between two arms. This power calculation is based on Fisher’s exact test at one-sided type I error rate of 0.05. The primary analy- sis was performed per the modified intent-to-treat principle (mITT), i.e., all patients who were randomized and received any
Table 1. Baseline characteristics of the study cohorts: by treatment arms
amount of the study treatment were included in the primary analysis. Cumulative incidence of aGvHD and cGvHD, relapse and NRM were estimated in the competing risk framework. Relapse with or without death was considered a competing risk for NRM, and similarly NRM for relapse. Death or relapse without developing GvHD was a competing event for GvHD. GvHD inci- dences occurring after relapse, in the setting of relapse interven- tions (e.g., immunosuppression [IS] taper, donor lymphocyte infu-
A, B, C, and overall.
Arm A (N=46) Arm B (N=45)
(Tac/Mtx) (Bort/Tac/Mtx) (Bort/Sir/Tac)
Arm C (N=47) All (N=138) N%N%N%N%P
Age, median (range) Patient Sex
Male
Female Donor Sex
Male
Female
Patient-Donor Sex Match
MM MF FM FF
ECOG Performance Status 0
1
2
Primary Disease
AML
CLL/SLL/PLL
CML
Hodgkin Disease
ALL
MDS
MPD
Mixed MDS/MPD
NHL
Other Acute Leukemia
HLA Type 8/8 match 7/8 match
Patient-Donor CMV Serostatus Positive
Graft Source PB
Disease Risk Index Low
Intermediate
High/Very high
65 (29, 74)
28 60.9 18 39.1
32 69.6 14 30.4
20 43.5 8 17.4 12 26.1 6 13.0
7 15.2 21 45.7 18 39.1
19 41.3
5 10.9
- -
2 4.4
3 6.5 - - 3
12 26.1 12 26.7 9 2 4.4 1 2.2 - - - 1 2.2 - 2 4.4 9 20.0 9
65 (30, 75)
32 71.1 13 28.9
33 73.3 12 26.7
26 57.8 6 13.3 7 15.6 6 13.3
10 22.2 20 44.4 15 33.3
62 (24, 73)
51.1 48.9
76.6 23.4
44.7 6.4 31.9 17.0
21.3 63.8 14.9
64 (24, 75) 0.13 0.15
24 23
36 11
21 3 15 8
10 30 7
84 60.9 54 39.1
101 73.2 37 26.8
67 48.6 17 12.3 34 24.6 20 14.5
27 19.6 71 51.4 40 29.0
53 38.4 11 8.0 3 2.2 7 5.1 6 4.4 33 23.9 - 3 2.2 - 1 0.7
0.75
0.39
0.11
0.48
16 35.6
3 6.7
1 2.2 2 2 4.4 3
18 3
38.3 6.4 4.3 6.4 6.4 19.2
1 2.2
34 73.9 12 26.1
36 78.3 46 100
5 10.9 33 71.7 8 17.4
- -
-
32 15
25
47
19.2 20 14.5 - 1 0.7
68.1 98 71.0 31.9 40 29.0
53.2 96 69.6 100 138 100
32 71.1 13 28.9
35 77.8 45 100
0.83
0.01 NA 0.94
4 8.9 5 30 66.7 33 11 24.4 9
10.6 14 70.2 96 19.2 28
10.1 69.6 20.3
524
Tac: tacrolimus; Mtx: methotrexate; Bort: bortezomib; Sir: sirolimus; MM: male patient/male donor; MF: male patient/female donor; FM: female patient/male donor; FF: female patient/female donor; AML: acute myeloid leukemia; CLL/SLL/PLL: chronic lymphocytic leukemia/small lymphocytic lymphoma/prolymphocytic leukemia; CML: chronic myeloid leukemia; MDS: myelodysplastic syndromes; MPD: myeloproliferative syndromes; NHL: non-Hodgkin lymphoma; PB: peripheral blood; ALL: acute lymphoblastic leukemia; HLA: human leukocyte antigen; CMV: cytomegalovirus.
haematologica | 2018; 103(3)