49 The Drug Rediscovery Protocol Extended Data Table 3. Rates of response and clinical benefit Rates of clinical benefit and response in the first 215 patients who started study treatment. Clinical benefit is defined as a complete or partial response or absence of disease progression at ≥16 weeks; it must be measured 2 or more times ≥28 days apart (defined as a confirmed response). Given that 29 patients had ongoing clinical benefit at the time of analyses, the actual mean duration is expected to exceed the current mean duration. The reasons for early withdrawal from study (other than progressive disease) included death (n = 9 patients), adverse events (n = 5 patients), patient preference (n = 3 patients) or were unknown (n = 7 patients). n In total, over 215 patients Median duration in months (95% CI) Clinical benefit Complete response (confirmed) 1 34% 9 (8 – 11) Partial response (confirmed) 32 Stable disease ≥16 weeks (confirmed) 41 No clinical benefit Stable disease <16 weeks / non-confirmed stable disease only 117 66% Early study withdrawal, for other reasons than progressive disease 24 Extended Data Table 4. Adverse events in the first 215 patients Rates of clinical benefit and response in the first 215 patients who started study treatment. Clinical benefit is defined as a complete or partial response or absence of disease progression at ≥16 weeks; it must be measured 2 or more times ≥28 days apart (defined as a confirmed response). Given that 29 patients had ongoing clinical benefit at the time of analyses, the actual mean duration is expected to exceed the current mean duration. The reasons for early withdrawal from study (other than progressive disease) included death (n = 9 patients), adverse events (n = 5 patients), patient preference (n = 3 patients) or were unknown (n = 7 patients). Adverse event Grade 3 Grade 4 Grade 5 Abdominal infection 1 Abscess 2 Adrenal insufficiency 1 AF ↑ 5 ALT ↑ 3 AST ↑ 6 Amylase ↑ 1 Anal fistula or ulcer 2 Anemia 8 Arteritis 1 Atrioventricular block 1 2
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