Table 1.

Reported clinical trials.

Primary Investigator	Indication	Phase	No. of patients	Treatment regimen	Response	Overall Survival
Goebeler et al. 201623	Adult R/R B-NHL	I	76	Continuous infusion, escalating doses. MTD 60µg/m2/day given in n = 35	At MTD: ORR FL = 80% MCL = 71% DLBCL = 55% Other = 50%	Not available (Median response duration 404 days)
Viardot et al. 201624	Adult R/R DLBCL	II	25	Continuous infusion with weekly dose escalation, to target 112 µg/day	ORR 43%	5.0 months
Topp et al. 201125	Adult MRD positive B-ALL	II	21	Continuous infusion 15 µg/m2/day	80% MRD response	Not available (At median follow-up 50.8 months 50% still in remission)
Gökbuget et al. 201728	Adult MRD positive B-ALL	II	116	Continuous infusion 15 µg/m2/day	78% MRD response post cycle 1, 80% MRD response overall	36.4 months
Topp et al. 201429	Adult R/R B-ALL	II	36	5–30 µg/m2/day	69% CR/CRh (88% MRD response post cycle 1)	9.8 months
Topp et al. 201531	Adult R/R B-ALL	II	189	9 µg/day for first week cycle 1, 28 µg/day thereafter	43% CR/CRh (82% MRD response)	6.1 months
Martinelli et al. 201535	Adult Ph-positive R/R B-ALL	II	45	9 µg/day for first week cycle 1, 28 µg/day thereafter	CR/CRh 36% (86% MRD response)	7.1 months
Kantarjian et al. 201733	Adult R/R B-ALL	III	405 (B: 271, SOC 134)	9 µg/day for first week cycle 1, 28 µg/day thereafter	B: 46% CR/CRh SOC: 28% CR/CRh	B: 7.8 months SOC: 4.0 months
Von Stackelberg et al. 201636	Paediatric R/R B-ALL	I/II	70 (at recommended dosage)	In phase II: 5 µg/m2/day for first week, 15 µg/m2/day thereafter	38.6% CR	7.5 months

B-NHL: B-cell non Hodgkin lymphoma, B-ALL: B-cell precursor acute lymphoblastic leukaemia; FL: follicular lymphoma; MCL: mantle cell lymphoma; DLBCL: diffuse large B-cell lymphoma; R/R: relapsed or refractory; MRD: minimal residual disease; Ph: Philadelphia chromosome; ORR: overall response rate; CR: complete response; CRh: complete response with incomplete haematologic recovery; SOC: standard of care;