Table 4 Comparison of the core outcome set for clinical trials in type 2 diabetes and outcomes in pre-clinical studies
Core outcome | Domain | Outcomes reported in pre-clinical in vivo studies | Number of pre-clinical studies reporting the outcome (%) | Number of phase 3/4 clinical trials reporting the outcome (%) |
|---|---|---|---|---|
Overall survival—how long someone lives | Death | Mortality, survival | 7 (2.5) | 2 (1.4)a |
Death from a diabetes related cause such as heart disease | Death | No study specifically described death from a diabetes related cause | 0 (0) | 1 (0.7) |
Glycaemic control—how well someone’s blood glucose is controlled | Physiological/clinical | Glycated haemoglobin (HbA1c), glycated serum proteins, euglycaemic duration, fructosamine | 40 (14.3) | 93 (67.4) |
Body weight—how much someone weighs | Physiological/clinical | Body weight | 165 (58.9) | 54 (39.1) |
Kidney function—how well someone’s kidneys are working | Physiological/clinical | Blood urea nitrogen (BUN), creatinine, electrolytes, renal fructose, renal sorbitol, total protein, urine volume, kidney functionb | 23 (8.2) | 25 (18.1) |
Hyperglycaemia—how often someone has high blood glucose | Physiological/clinical | Blood glucose | 253 (90.4) | 64 (46.4) |
Hypoglycaemia—how often someone has low blood glucose levels | Physiological/clinical | Hypoglycaemic and hypoglycaemic durationc | 3 (1.1) | 45 (32.6) |
Visual deterioration or blindness—if someone’s eyesight gets worse or if they have loss of vision including blindness | Physiological/clinical | No studies measured this outcome | 0 (0) | 2 (1.4) |
Neuropathy—damage to the nerves caused by high glucose. This can lead to tingling and pain or numbness in the feet or legs. It can also affect bowel control; stomach emptying and sexual function | Physiological/clinical | Hyperalgesia, long term potentiation, pain threshold | 5 (1.8) | 2 (1.4) |
Having gangrene or having an amputation of the leg, foot or took | Physiological/clinical | No studies measured this outcome | 0 (0) | 0 (0) |
Nonfatal myocardial infarction—having a heart attack that is not fatal | Physiological/clinical | No studies measured this outcome | 0 (0) | 2 (1.4) |
Heart failure | Physiological/clinical | Heart weight, appearance of the heart, heart histology | 6 (2.1) | 11 (7.9) |
Cerebrovascular disease—including stroke, subarachnoid haemorrhage, transient ischaemic attack and vascular dementia | Physiological/clinical | No studies measured this outcome | 0 (0) | 0 (0) |
Hyperglycaemic emergencies (to include diabetic ketoacidosis and hyperosmolar hyperglycaemic state) | Physiological/clinical | Ketone bodies, lactate, β-hydroxybutyrate | 8 (2.9) | 11 (7.9) |
Global quality of life—someone’s overall quality of life including physical, mental and social wellbeing | Life impact | Object recognition, anxiety, mental status, food or water intaked | 111 (39.6) | 7 (5.1) |
Object recognition | 2 (0.7) | – | ||
Anxiety/mental status | 3 (1.1) | – | ||
Food or water intake | 110 (39.3) | – | ||
Activities of daily living - being able to complete usual everyday tasks and activities including those related to personal care; house hold tasks or community based tasks | Life impact | Behaviour, locomotor activity, open field assessment, exploratory activity, ambulatory activity, physical activity, external appearance, coat fur, hair colour | 18 (6.4) | 2 (1.4) |
How often someone is admitted to hospital because of their diabetes | Resource use | Not applicable | N/A | 3 (2.2) |
Side effects of treatment- any unwanted effects of the treatment | Adverse events | Organ toxicity, adverse effects, side effects, toxicity | 20 (7.1) | 34 (26.6) |