Comparing study populations and outcomes
| Study . | Drug/dose . | Genotype . | Other therapies . | Outcome . |
|---|---|---|---|---|
| Niihara et al (2018)2 : placebo-controlled RCT (230 patients, 5-58 y) | L-glutamine 0.3 mg/kg/dose BID for 48 weeks | Hgb SS/Hgb Sβ0 thalassemia | Excluded patients on transfusions Two-thirds on HU in each group | Median VOC: 3 (L-glutamine), 4 (placebo) Hospitalizations: 2 (L-glutamine), 3 (placebo) |
| HOPE trial (2019)4 : placebo-controlled RCT (274 patients, 12-65 y) | 1500 mg voxelotor (vox), 900 mg vox, placebo daily for up to 72 weeks | Hgb SS, Hgb SC, Hgb Sβ0, Hgb SB+, other variants | Excluded patients on transfusions Two-thirds on HU | Hgb increase >1 g/dL from baseline: 51% (1500 mg vox) vs 7% (placebo) Decreased indirect bilirubin: –29.3% (1500 mg vox) vs −3.2% (placebo) |
| Zaidi et al (2020)5 : real-world use of vox retrospective (1275 patients, 6-month follow-up) | 17 patients on chronic transfusions | After vox initiation: 55% patients had Hgb increase >1 g/dL Annualized VOC rate decreased 3.86 to 3.64 | ||
| SUSTAIN trial (2017)6 : placebo-controlled RCT (198 patients, 16-63 y) | 2.5 mg/kg crizanlizumab (criz), 5 mg/kg criz, placebo, 14 doses over 52 weeks | Hgb SS, Hgb SC, Hgb Sβ0, Hgb SB+, other variants | Excluded patients on chronic transfusions Two-thirds on HU in each group | Median annual VOC: 1.63 (high-dose criz) vs 2.98 (placebo) Median time to first VOC: 4.07 months (high-dose criz) vs 1.38 months (placebo) |
| Kutlar et al (2019)7 : SUSTAIN subgroup analysis | 5 mg/kg criz, placebo | Hgb SS vs other genotypes | Two-thirds on HU | In patients with 5-10 VOCs in prior year, concomitant HU use, OR more clinically severe HbSS genotype: criz increased likelihood of being VOC free vs placebo |
| Kanter et al (2021)8 : real-world use of criz retrospective (238 patients with >1 dose) | 5 mg/kg criz | Hgb SS, Hgb SC, Hgb Sβ0, Hgb SB+, other variants | 72 patients (32%) discontinued criz due to perceived lack of improvement, infusion-related reactions, logistics 32 patients (55%) with ≥12 infusions had significant decrease in ED use and hospitalizations |
| Study . | Drug/dose . | Genotype . | Other therapies . | Outcome . |
|---|---|---|---|---|
| Niihara et al (2018)2 : placebo-controlled RCT (230 patients, 5-58 y) | L-glutamine 0.3 mg/kg/dose BID for 48 weeks | Hgb SS/Hgb Sβ0 thalassemia | Excluded patients on transfusions Two-thirds on HU in each group | Median VOC: 3 (L-glutamine), 4 (placebo) Hospitalizations: 2 (L-glutamine), 3 (placebo) |
| HOPE trial (2019)4 : placebo-controlled RCT (274 patients, 12-65 y) | 1500 mg voxelotor (vox), 900 mg vox, placebo daily for up to 72 weeks | Hgb SS, Hgb SC, Hgb Sβ0, Hgb SB+, other variants | Excluded patients on transfusions Two-thirds on HU | Hgb increase >1 g/dL from baseline: 51% (1500 mg vox) vs 7% (placebo) Decreased indirect bilirubin: –29.3% (1500 mg vox) vs −3.2% (placebo) |
| Zaidi et al (2020)5 : real-world use of vox retrospective (1275 patients, 6-month follow-up) | 17 patients on chronic transfusions | After vox initiation: 55% patients had Hgb increase >1 g/dL Annualized VOC rate decreased 3.86 to 3.64 | ||
| SUSTAIN trial (2017)6 : placebo-controlled RCT (198 patients, 16-63 y) | 2.5 mg/kg crizanlizumab (criz), 5 mg/kg criz, placebo, 14 doses over 52 weeks | Hgb SS, Hgb SC, Hgb Sβ0, Hgb SB+, other variants | Excluded patients on chronic transfusions Two-thirds on HU in each group | Median annual VOC: 1.63 (high-dose criz) vs 2.98 (placebo) Median time to first VOC: 4.07 months (high-dose criz) vs 1.38 months (placebo) |
| Kutlar et al (2019)7 : SUSTAIN subgroup analysis | 5 mg/kg criz, placebo | Hgb SS vs other genotypes | Two-thirds on HU | In patients with 5-10 VOCs in prior year, concomitant HU use, OR more clinically severe HbSS genotype: criz increased likelihood of being VOC free vs placebo |
| Kanter et al (2021)8 : real-world use of criz retrospective (238 patients with >1 dose) | 5 mg/kg criz | Hgb SS, Hgb SC, Hgb Sβ0, Hgb SB+, other variants | 72 patients (32%) discontinued criz due to perceived lack of improvement, infusion-related reactions, logistics 32 patients (55%) with ≥12 infusions had significant decrease in ED use and hospitalizations |
BID, twice a day; ED, emergency department; HU, hydroxyurea; RCD, randomized controlled trial.