Table 2.
Values used in the model
| Source data | Value used in the model | Variation in sensitivity analysis (upper, lower) | |
|---|---|---|---|
| Assumptions | |||
| Patient weight | Average weight of men/women from BNF [56] | 63 kg | 58.00, 68.00a |
| Efficacy | |||
| Cardiac morbidity—DFP or combination therapy | Non-RCT; 4 % over 5 years [43] | 4 % | 0.00, 9.23b |
| Cardiac morbidity—DFO/DFX | Non-RCT; 21 % over 5 years [43] | 21 % | 11.78, 30.22b |
| Cardiac mortality—DFP/combination therapy | RCT: 0 deaths over 5 years [34] | 0 % at 5 years | 0, 2.49b |
| Cardiac mortality—DFO/DFX | RCT: 4.8 % mortality over 5 years [34] | 4.8 % at 5 years | 1.58, 9.71b |
| Utility associated with cardiac morbidity | 0.921 [52] | 7.9 % decrement applied to the utility value for each treatment regimen | 0.815, 1.000a |
| Adverse events | |||
| DFP/combination therapy—agranulocytosis | Product SPC: 0.6 per 100 patient-years [15] | Annual rate of 0.6 % | 0.21, 0.35c |
| DFP/combination therapy—neutropenia | Product SPC: 2.5 per 100 patient-years [15] | Annual rate of 2.5 % | 1.88, 3.13c |
| DFX—hepatitis | Product SPC: 0.7 % [17] | 0.7 % | 0.02 %, 1.58 %b |
| DFX—Fanconi syndrome | Clinical case reports: 0.8 % [51] | 0.8 % | 0.02 %, 1.63 %c |
| DFO—no AEs | – | – | – |
| Risk of mortality associated with agranulocytosis | Post-marketing data: 13.83 % | 13.83 % | 6.85, 20.81b |
| Utility associated with AEs | |||
| Agranulocytosis | Published model: 0.460 [54] | 0.460 for 7 days, 54.0 % decrement from perfect health | 0.345, 0.575c |
| Neutropenia | Published model: 0.782 [55] | 0.782 for 1 day, 21.8 % decrement from perfect health | 0.587, 0.978c |
| Hepatitis | Published model: 0.770 [53] | 0.770 for 365 days, 23.0 % decrement from perfect health | 0.710, 0.810c |
| Fanconi syndrome | No data available | No impact on QoL | No variation |
| Route of administration utility | |||
| Oral | Prior economic evaluation: 0.840 [28] | 0.840 | 0.66, 1.00d |
| SC infusion | Prior economic evaluation: 0.660 [28] |
0.66 for five times weekly DFO 0.696 for four times weekly combination therapy |
0.25b, 0.84d |
AE adverse event, BNF British National Formulary, DFO desferrioxamine, DFP deferiprone, DFX deferasirox, QoL quality of life, RCT randomized controlled trial, SC subcutaneous, SPC summary of product characteristics
aReported in source. b Calculated based on standard error reported in source. c Calculated by varying the reported value ±25 %. d Calculated value would be outside the plausible range, value set to plausible maximum (i.e., maximum SC infusion utility cannot be higher than the standard oral utility, and the minimum oral utility cannot be lower than the standard SC infusion utility)