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Table 3 Effects of disulfiram on progression-free survival and overall survival from human studies

From: Anticancer effects of disulfiram: a systematic review of in vitro, animal, and human studies

Reference

Country

Study design

Study participants

Study protocol

OS

PFS

Adverse events

Huang, et al.

USA

Phase II, open-label, single-arm study

Recurrent GBM who had developed unequivocal progression after RT and concurrent TMZ as per the RANO criteria while receiving adjuvant TMZ or within 3 months from the last dose of TMZ”

DSF 80 mg and Cu Gluconate1.5 mg TID by mouth approximately 4–8h apart.

7.1 months (95% CI 5.8–8.5)

1.7 months (95% CI 1.4–1.9)

Nausea/vomiting (17%) followed by dizziness (9% grade). Only one patient (4%) had a possible DLT with grade 3 elevated alanine transaminase on day 31, which required study therapy to be held. The liver function test subsequently recovered after 4 weeks.

Huang, et al.

USA

Phase I, open-label, single-arm, single-institution study

Adjuvant TMZ in newly diagnosed adult GBM patients after standard chemoradiotherapy

7 patients at DSF 500 mg per day 5 patients at DSF 1000 mg per day, 6 patients at DSF 500 mg per day with Cu 2 mg

14.0 months (95% CI 8.3–19.6)

4.5 months (95% CI 0.8–8.2)

One with delirium after 1.6 months (without Cu), one with motor neuropathy after 2.6 months (without Cu) and one with diarrhea and nausea after 0.5 months (with Cu). All symptoms resolved shortly after dose reduction.

Nechushtan, et al.

Israel

Phase II, multicenter randomized double-blinded study

Newly diagnosed NSCLC patients were recruited. Patients with either stage IV or what was considered at the time “wet IIIb” (since 2009, these patients have been considered stage IV) were recruited. The patients were treated with only chemotherapy, and none were treated with either surgery or chemoradiation.

controls: six cycles of cisplatin and vinorelbine (plus placebo tablets), experimental groups: the same plus disulfiram (40mg three times daily).

10.0 versus 7.1 months

5.9 versus 4.9 months

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  1. Abbreviations: GBM Glioblastoma, NSCLC Non-small cell lung cancer, TMZ Temozolomide, TID Three times per day, DLT Dose-limiting toxicity, RANO Radiologic Assessment in Neuro-Oncology