Table 6.

Published clinical trials of single-agent THAL in MMM

Authors and
reference no.
No. of
patients
MinMaxDropout/DMAnemia responsePlatelet responseSpleen response
Elliott et al24 15  506-150 400 12 (80%) 3/15 (20%) 4/5 (80%) 1/15 (7%) 
Barosi et al22 21 100 400 19 (91%) 3/7 (43%) 2/3 (66.7%) 4/20 (20%) 
Canepa et al23 10 200 800 3/10 (30%) 3/10 (30%) 3/10 (30%) 
Pozzato et al25 100 NR NR 3/6 (50%) NR 0/6 
Piccaluga et al26 12 100 600 11/12 (92%) 3/12 (25%) 3/12 (25%) 2/12 (16.7%) 
Merup et al27 15 200 800 6/15 (40%) 
Authors and
reference no.
No. of
patients
MinMaxDropout/DMAnemia responsePlatelet responseSpleen response
Elliott et al24 15  506-150 400 12 (80%) 3/15 (20%) 4/5 (80%) 1/15 (7%) 
Barosi et al22 21 100 400 19 (91%) 3/7 (43%) 2/3 (66.7%) 4/20 (20%) 
Canepa et al23 10 200 800 3/10 (30%) 3/10 (30%) 3/10 (30%) 
Pozzato et al25 100 NR NR 3/6 (50%) NR 0/6 
Piccaluga et al26 12 100 600 11/12 (92%) 3/12 (25%) 3/12 (25%) 2/12 (16.7%) 
Merup et al27 15 200 800 6/15 (40%) 

Min indicates minimum THAL dose; Max, maximum THAL dose; Dropout/DM, number (percentage) of patients in the trial with early dropout or necessitating dose modification; NR, not reported.

F6-150

The 50-mg dose was given only as a dose modification after initial toxicity.

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