Highlights on the Luspatercept Treatment in Thalassemia
Abstract
:1. Introduction
2. Ineffective Erythropoiesis in β-Thalassemia
3. Modulating Erythroid Differentiation by ActRII Ligand Traps
4. Pharmacokinetics of Luspatercept
5. Clinical Studies with Luspatercept
6. Current Status, Challenges, and Future Directions of Luspatercept
7. Conclusions
Funding
Conflicts of Interest
References
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Protocol no | Phase | Planned/Treated | Dose (mg/kg) | Duration | Erythroid Response |
---|---|---|---|---|---|
NCT01432717 | 1 | 32 PM women | 0.0625–0.25 | 1 month (Q2W) | Hb ≥ 1 g/dL, 83% (0.25 mg/kg) |
NCT01749540 | 2 | 64/64 (NTDT, TDT, ≥18y) | 0.2–1.25 | 15 weeks (Q3W) | ∫ NTDT; 58% achieved Hb ≥ 1.5 g/dL for 2 weeks, 71% Hb ≥ 1.0 g/dL for 12 weeks. ∫ TDT; transfusion reduction ≥ 20, 33, and 50% at 81, 72, and 63%, respectively, for 12 weeks |
NCT02268409 | 2 | 64/51 (NTDT, TDT, ≥18y) | 0.8–1.25 | 60 months (Q3W) | |
NCT02604433 [BELIEVE] | 3 | 336/332 (TDT, ≥18y, luspatercept vs. placebo 224:112) open-label LTFU ongoing | 1.0–1.25 | 48 weeks LTFU:60 months (Q3W) | ≥33% reduction: weeks 13–24; 21.4% in luspatercept vs. 4.5% placebo ≥50% reduction: weeks 13–24; 7.6% in luspatercept vs. 1.8% placebo and ≥50% reduction: weeks 37–48; 10.3% in luspatercept vs. 0.9% placebo ≥33% reduction: any 24 weeks; 41.1% in luspatercept vs. 2.7% placebo |
NCT03342404 [BEYOND] | 2 | 150/145 (NTDT, ≥18y, luspatercept vs. placebo 96:49) | 1.0–1.25 | 48 weeks (Q3W) | Hb ≥ 1.0 g/dL: weeks 13–24; 77% in luspatercept vs. 0% placebo (p < 0.0001) Mean Hb change (g/dL) from baseline weeks 13–24; 1.48 in luspatercept vs. 0.07 placebo (p < 0.0001) Hb ≥ 1.0 g/dL: weeks 37–48; 71% in luspatercept vs. 2% placebo (p < 0.0001) |
NCT04143724 | 2 | 54/- (TDT, 6– < 18y) | 0.75–1.25 | 1 year LTFU: 5 year | Started patients’ recruitment |
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Aydinok, Y. Highlights on the Luspatercept Treatment in Thalassemia. Thalass. Rep. 2023, 13, 77-84. https://doi.org/10.3390/thalassrep13010008
Aydinok Y. Highlights on the Luspatercept Treatment in Thalassemia. Thalassemia Reports. 2023; 13(1):77-84. https://doi.org/10.3390/thalassrep13010008
Chicago/Turabian StyleAydinok, Yesim. 2023. "Highlights on the Luspatercept Treatment in Thalassemia" Thalassemia Reports 13, no. 1: 77-84. https://doi.org/10.3390/thalassrep13010008