Table 2.
Summary of components of included cost-effectiveness analyses
| Author, Year | Study population | Mean Age (years) | Intervention | Time Horizon | Model Type | Cost Type | Effectiveness Measure | Source of Health Utility | Discounting |
|---|---|---|---|---|---|---|---|---|---|
| Cherry et al., 2012[22] | • Children with SCD • Had no prior history of stroke |
• 2 | • TCD scans followed by blood transfusion where the scan revealed a blood velocity of > 200 cm/second | • Lifetime | • Markov model | Costs within the formal healthcare sector • Costs of the intervention • Costs of consequences due to the intervention |
• QALY | • Empirically elicited for non-SCD specific population • Assumed by the authors without referencing empirical study. |
• 3.5% |
| Spackman et al., 2014[23] | • SCD patients • Undergoing low- or medium-risk surgery |
• 17.3 | • Preoperative transfusion | • 1 year | • N/A | Costs within the formal healthcare sector • Costs of the intervention • Costs of consequences due to the intervention |
• QALY | • Empirically elicited from SCD population • Assumed by the authors without referencing empirical study. |
• Not discounted |
| Arnold et al., 2015[24] | • Children with SCD | • Intervention: 9.10 (SD: 6.25) • Comparator: 4.23 (SD: 3.74) |
• alloHCT | • 1 year • Lifetime |
• Not a modeling study | Costs within the formal healthcare sector • Costs of the intervention • Costs of consequences due to the intervention |
• QALY | • Empirically elicited for SCD population • Empirically elicited for non-SCD specific population |
• Not discounted |
| Cunningham-Myrie et al., 2015[25] | • Children with SCD • Had a first clinical stroke |
• N/A | • Hydroxyurea | Mean study duration • 3.82 years for intervention group • 4.47 years for control group |
• Not a modeling study | Costs within the formal healthcare sector • Costs of the intervention • Costs of consequences due to the intervention |
• Stroke • Death |
• Utilities not used | • Not discounted |
| Bradt et al., 2020[26] | • SCD patients • Had a baseline rate of 3 acute pain crises per year |
• 24 | • Voxelotor • Crizanlizumab • L-glutamine |
Lifetime | • Markov model | Costs within the formal healthcare sector • Costs of the intervention • Costs of consequences due to the intervention Costs within the informal health care sector • Caregiver-time costs Costs within the non-health care sector • Productivity • Education |
• LY • QALY • evLYG |
• Empirically elicited for SCD population • Empirically elicited for non-SCD specific population • Assumed by the authors without referencing empirical study. |
• 3% |
| Salcedo et al., 2021[27] | • Newborns with SCD | • At birth | • Hypothetical cell or genetic therapy | • Lifetime | • Markov model | Costs within the formal healthcare sector • Costs of the intervention • Costs of consequences due to the intervention |
• LY • QALY |
• Empirically elicited for SCD specific population • Empirically elicited for non-SCD specific population |
• 3% |
| Castilla-Rodríguez et al., 2016[28] | • Newborns | • At birth | • Newborn screening program | • 10 years | • Discrete event simulation model | Costs within the formal healthcare sector • Costs of the intervention • Costs of consequences due to the intervention |
• LY | • Utilities not used | • 3% |
| Kuznik et al., 2016[29] | • Newborns | • At birth | • Newborn screening program | • Lifetime | • Markov model | Costs within the formal healthcare sector • Costs of the intervention • Costs of consequences due to the intervention |
• DALY | • Utilities not used | • 3% |
| McGann et al. 2015[30] | • Newborns | • At birth | • Newborn screening program | • Lifetime | • Life table | Costs within the formal healthcare sector • Costs of the intervention • Costs of consequences due to the intervention |
• HLY | • Utilities not used | • 3% |
| Bryan et al., 2011[31] | • Pregnant women (biological mothers) • Their partners (biological fathers) |
• N/A | • Primary care parallel • Primary care sequential |
• Pregnancy to conclusion | • N/A | Costs within the formal healthcare sector • Costs of the intervention • Costs of consequences due to the intervention |
• Woman screened | • Utilities not used | • Not discounted |
| Kacker et al., 2014 (a)[32] | • SCD patients • Undergoing chronic blood transfusion |
• Equivalent to mean age of the U.S. population | • Prospective antigen-matching • Perfectly informed antigen-matching • Imperfectly informed antigen-matching |
• 10 years • 20 years |
• Markov model | Costs within the formal healthcare sector • Costs of the intervention • Costs of consequences due to the intervention |
• Alloimmunization event | • Utilities not used | • 3% |
| Kacker et al., 2014 (b)[33] | • SCD patients • Undergoing chronic blood transfusion |
• N/A | • Prospective limited matching • Prospective extensive matching |
• 10 years • 20 years |
• Markov model | Costs within the formal healthcare sector • Costs of the intervention • Costs of consequences due to the intervention |
• Alloimmunization event | • Utilities not used | • 3% |
| McLeod et al., 2009[34] | • Patients with beta-thalassemia major or SCD | • Stratified by age ranging from 2 to 18+ | • Deferasirox | • 1 year | • N/A | Costs within the formal healthcare sector • Cost of interventions |
• QALY | • Empirically elicited for non-SCD specific population • Assumed by the authors without referencing empirical study |
• Not discounted |
alloHCT = Allogeneic hematopoietic cell transplantation; evLYG = equal value of life year gain; DALY = disability-adjusted life year; HLY = health life year; ICER = incremental cost-effectiveness ratio; LY = life year; N/A = not available; QALY = quality-adjusted life year; SCD = sickle cell disease; SD = standard deviation; TCD = transcranial doppler ultrasound