. Author manuscript; available in PMC: 2023 Dec 5.

Published in final edited form as: Pharmacoeconomics. 2021 Aug 9;39(11):1225–1241. doi: 10.1007/s40273-021-01072-z

Table 3.

Results of the base case scenario in included economic evaluations of interventions for sickle cell disease

Author, Year	Study Population	Time Horizon	Intervention vs. Comparator	Currency (fiscal year); Effectiveness Measure	Costs (per person) of Intervention vs. Comparator^*	Effectiveness (per person) of Intervention vs. Comparator^*	ICER as Reported by Authors of Original Publication
*Blood Transfusion*
Cherry et al., 2012[22]	• Children with SCD • Had no prior history of stroke	• Lifetime	• Intervention: TCD scans followed by blood transfusion where the scan revealed a blood velocity of > 200 cm/second • Comparator: TCD scans only	• £(2010); QALY	• 52,472 • 38,720	• 14.87 • 14.30	• £24,075 (US$37,316) per QALY gained
Spackman et al., 2014[23]	• SCD patients • Undergoing low- or medium-risk surgery	• 1 year	• Intervention: Preoperative transfusion • Comparator: No transfusion	• £(2011); QALY	• 1,706 • 2,442	• 0.71 • 0.70	• Less costly, more effective
*Transplantation*
Arnold et al., 2015[24]	• Children with SCD	• 1 year	• Intervention: alloHCT • Comparator: Referred but without alloHCT	• US$(N/A); QALY	• 430,861 • 8,245	• 0.78 • 0.91	• More costly, less effective^†
Arnold et al., 2015[24]	• Children with SCD	• Lifetime		• US$(N/A); QALY	• N/A • N/A	• N/A • N/A	• N/A^†
*Pharmaceuticals*
Cunningham-Myrie et al., 2015[25]	• Children with SCD • Had a first clinical stroke	Mean study duration • 3.82 years for intervention group • 4.47 years for control group	• Intervention: Hydroxyurea • Comparator: No hydroxyurea	• J$(2009); stroke	• 86,710 • 69,941	• 0.03 • 0.13	• J$169,238 (US$1,917) per Stroke averted
Cunningham-Myrie et al., 2015[25]	• Children with SCD • Had a first clinical stroke		• Intervention: Hydroxyurea • Comparator: No hydroxyurea	• J$(2009); death	• 86,710 • 69,941	• 0.00 • 0.00	• J$635,843 (US$7,203) per death averted
Bradt et al., 2020 2020[26]	• SCD patients • Had a baseline rate of 3 acute pain crises per year	• Lifetime	• Intervention: L-glutamine • Comparator: Optimal usual care alone	• US$(2019); LY	• 1,414,000 • 1,174,000	• 15.35 • 14.34	• US$238,000 per LY gained
				• US$(2019); evLYG		• 8.96 • 8.07	• US$270,000 per evLYG gained
				• US$(2019); QALY		• 8.47 • 8.07	• US$604,000 per QALY gained
			• Intervention: Crizanlizumab • Comparator: Optimal usual care alone	• US$(2019); LY	• 2,046,000 • 1,174,000	• 16.36 • 14.34	• US$432,000 per LY gained
				• US$(2019); evLYG		• 9.78 • 8.07	• US$509,000 per evLYG gained
				• US$(2019); QALY		• 8.87 • 8.07	• US$1,086,000 per QALY gained
			• Intervention: Voxelotor • Comparator: Optimal usual care alone	• US$(2019); LY	• 2,291,000 • 1,174,000	• 16.37 • 14.34	• US$550,000 per LY gained
				• US$(2019); evLYG		• 9.96 • 8.07	• US$589,000 per evLYG gained
				• US$(2019); QALY		• 9.10 • 8.07	• US$1,082,000 per QALY gained
*Hypothetical cell or genetic therapy*
Salcedo et al, 2021[27]	• Newborns with SCD	• Lifetime	• Intervention: Hypothetical cell or genetic therapy • Comparator: Standard of care	• US$(2018); LY	• 2,372,482 • 1,175,566	• 29.9 • 26.2	• N/A^‡
Salcedo et al, 2021[27]	• Newborns with SCD	• Lifetime		• US$(2018); QALY	• 2,372,482 • 1,175,566	• 26.4 • 17.9	• US$140,877 per QALY gained
*Screening*
Castilla-Rodríguez et al., 2016[28]	• Newborns	• 10 years	• Intervention: Newborn screening program • Comparator: No screening	• €(2013); LY	• 5 • 6	• 8.66 • 8.66	• €34,169(US$45,445) per LY gained
Kuznik et al., 2016[29]	• Newborns	• Lifetime	• Intervention: Newborn screening and prophylactic intervention • Comparator: No screening	• US$(2014); DALY	• 16 • 0	N/A^§ N/A^§	• US$213 per DALY averted
McGann et al., 2015[30]	• Newborns	• Lifetime	• Intervention: Newborn screening and treatment program for sickle cell anemia • Comparator: No screening	• US$(N/A); HLY	• 44 • 0	N/A^‖• N/A^‖	• US$2,214 per HLY gained – US$2,824 per HLY gained
Bryan et al., 2011[31]	• Pregnant women (biological mothers • Their partners (biological fathers)	• Pregnancy to conclusion	• Intervention: Primary care sequential • Comparator: Midwife care	• £(2010); woman screened	• 18 • 15	• 0.29 • 0.03	• £13(US$20) per woman screened
			• Intervention: Primary care parallel • Comparator: Midwife care		• 20 • 15	• 0.26 • 0.03	• £25(US$39) per woman screened
			• Intervention: Primary care parallel • Comparator: Primary care sequential		• 20 • 20	• 0.26 • 0.29	• More costly, less effective
*Intervention for SCD complications*
Kacker et al., 2014 (a)[32]	• SCD patients • Undergoing chronic blood transfusion	• 10 years	• Intervention: Prospective antigen-matching • Comparator: History-based antigen-matching	• US$(2012); alloimmunization event	• 247,201 • 162,623	• 0.25 • 0.46	• US$412,132 per alloimmunization event averted
			• Intervention: Perfectly informed antigen-matching • Comparator: History-based antigen-matching		• 164,866 • 162,623	• 0.25 • 0.46	• US$10,934 per alloimmunization event averted
			• Intervention: Imperfectly informed antigen-matching • Comparator: History-based antigen-matching		• 164,556 – 185,700 • 162,623	• 0.25 – 0.31 • 0.46	• US$12,558 per alloimmunization event averted – US$147,915 per alloimmunization event averted
		• 20 years	• Intervention: Prospective antigen-matching • Comparator: History-based antigen-matching		• 433,064 • 287,313	• 0.41 • 0.60	• US$759,799 per alloimmunization event averted
			• Intervention: Perfectly informed antigen-matching • Comparator: History-based antigen-matching		• 288,721 • 287,313	• 0.41 • 0.60	• US$7,344 per alloimmunization event averted
			• Intervention: Imperfectly informed antigen-matching • Comparator: History-based antigen-matching		• 288,619 – 325,057 • 287,313	• 0.41 – 0.46 • 0.60	• US$9,082 per alloimmunization event averted – US$261,638 per alloimmunization event averted
Kacker et al., 2014 (b)[33]	• SCD patients • Undergoing chronic blood transfusion • Had been transfusion naive initially	• 10 years	• Intervention: Prospective limited matching • Comparator: History-based limited matching	• US$(2012); alloimmunization event	• 456,376 • 236,978	• 0.08 • 0.36	• US$369,479 per alloimmunization event averted
		• 10 years	• Intervention: Prospective extensive matching • Comparator: History-based extensive matching		• 286,025 • 195,959	• 1.06 • 1.30	• US$769,344 per alloimmunization event averted
		• 20 years	• Intervention: Prospective limited matching • Comparator: History-based limited matching		• 502,938 • 344,544	• 1.86 • 2.10	• US$640,814 per alloimmunization event averted
		• 20 years	• Intervention: Prospective extensive matching • Comparator: History-based extensive matching		• 799,565 • 15,649	• 0.13 • 0.41	• US$1,364,247 per alloimmunization event averted
	• SCD patients • Undergoing chronic blood transfusion • Included patients with a prior history of transfusion and possible alloimmunization	• 10 years	• Intervention: Prospective limited matching • Comparator: History-based limited matching		• 169,720 • 127,562	• 0.64 • 0.81	• US$252,708 per alloimmunization event averted
		• 10 years	• Intervention: Prospective extensive matching • Comparator: History-based extensive matching		• 271,488 • 162,016	• 0.04 • 0.25	• US$541,939 per alloimmunization event averted
		• 20 years	• Intervention: Prospective limited matching • Comparator: History-based limited matching		• 271,272 • 201,192	• 1.05 • 1.25	• US$355,630 per alloimmunization event averted
		• 20 years	• Intervention: Prospective extensive matching • Comparator: History-based extensive matching		• 433,806 • 254,993	• 0.07 • 0.30	• US$775,069 per alloimmunization event averted
McLeod et al., 2009[34]	• Patients with beta-thalassemia major or SCD • Stratified by age ranging from 2 to 18+ years	• 1 year	• Intervention: Deferasirox • Comparator: Deferoxamine/desferrioxamine	• £(2007); QALY	• 4,386 – 18,594 • 2,733 – 7,219	• 0.84 • 0.66	• £9,232 (US$18,464) per QALY gained – • £63,195 (US$126,390) per QALY gained
McLeod et al., 2009[34]		• 1 year	• Intervention: Deferasirox • Comparator: Deferiprone	• £(2007); QALY	• 4,386 – 18,594 • 2,194 – 5,565	• 0.84 • 0.66	• £12,224(US$24,448) per QALY – • £72,386(US$144,772) per QALY gained

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; N/A = not available; SCD = sickle cell disease; TCD = transcranial doppler ultrasound

The costs and effectiveness were converted to per person value if not reported. The original number from the references can be found in Appendix 5.

^†

The ICER provided by Arnold et al. was not calculated as incremental costs divided by incremental effectiveness. The original ICER can be found in Appendix 5.

^‡

Salcedo et al. provided the ICER measured as costs per QALY gained but did not provide the ICER measured as costs per LY gained.

^§

Kuznik et al. provided the DALYs averted but did not provide the mean DALYs for intervention and for comparator.

^‖

McGann et al. provided the HLYs gained but did not provide the mean HLYs for intervention and for comparator.