Table 2. Design and results of all RCTs in hematological cancer published during 2014–2017.
| All RCTs n = 124 |
MM n = 31 |
Acute leukemia n = 20 |
Lymphoma NHL/HL n = 42 |
CLL n = 20 |
CML n = 8 |
MDS/MPN n = 3 |
p-value | |
|---|---|---|---|---|---|---|---|---|
| N (%) | ||||||||
| Primary endpoint OS DFS/EFS/RFS PFS/TTF QOL/toxicity RR Other Industry funding Yes No Sample size Median (IQR) IF Median (IQR) Primary EP met Yes No |
15 (12) 24 (19) 58 (47) 4 (3) 20 (16) 3 (2) 97 (78) 27 (22) 373 (236, 544) 13 (7–27) 69 (56) 55 (44) |
2 (7) 1 (3) 25 (81) 0 (0) 3 (10) 0 (0) 23 (74) 8 (26) 498 (271, 668) 12 (7–48) 21 (68) 10 (32) |
9 (45) 7 (35) 0 (0) 2 (10) 2 (10) 0 (0) 11 (55) 9 (45) 349 (243, 535) 26 (13–27) 8 (40) 12 (60) |
3 (7) 13 (31) 17 (41) 2 (5) 5 (12) 2 (5) 34 (81) 8 (19) 363 (223, 531) 18 (6–34) 21 (50) 21 (50) |
1 (5) 0 (0) 16 (80) 0 (0) 3 (15) 0 (0) 18 (90) 2 (10) 298 (230, 468) 11 (4–32) 13 (65) 7 (35) |
0 (0) 2 (25) 0 (0) 0 (0) 6 (75) 0 (0) 8 (100) 0 (0) 287 (216, 515) 11 (8–21) 4 (50) 4 (50) |
0 (0) 1 (33) 0 (0) 0 (0) 1 (33) 1 (33) 3 (100) 0 (0) 149 (129, n/a) 26 (8, n/a) 2 (67) 1 (33) |
<0.001 0.042 0.201 0.499 0.391 |
Percentages may not total 100 due to rounding; p-values are based on the Fisher’s exact test or the Kruskal-Wallis test
RCTs = randomized controlled trial, OS = overall survival, DFS = disease free survival, EFS = event free survival, RFS = relapse free survival, PFS = progression free survival, TTF = time to treatment failure; QOL = quality of life; RR = response rate; IQR = inter-quartile range; EP = endpoint; MM = multiple myeloma; NHL = Non Hodgkin lymphoma; HL = Hodgkin lymphoma; CLL = chronic lymphocytic leukemia; CML = chronic myeloid leukemia;
MDS = myelodysplastic syndromes; MPN = myeloproliferative neoplasm