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New Strategies for PNH

CE / CME

Clinical Focus in Paroxysmal Nocturnal Hemoglobinuria: New Strategies for Evading the Complement Cascade

ABIM MOC: maximum of 0.75 Medical Knowledge MOC point

Physicians: Maximum of 0.75 AMA PRA Category 1 Credit

Released: March 10, 2026

Expiration: September 09, 2026

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References

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  8. Eculizumab [prescribing information]. Cheshire, CT: Alexion; February 2025.
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  10. Lee JW, Sicre de Fontbrune F, Wong Lee Lee L, et al. Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study. Blood. 2019;133:530-539.
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  14. de Latour R, Roeth A, Kulasekararaj A, et al. Oral monotherapy with iptacopan, a proximal complement inhibitor of Factor B, has superior efficacy to intravenous terminal complement inhibition with standard of care eculizumab or ravulizumab and favorable safety in patients with paroxysmal nocturnal hemoglobinuria and residual anemia: results from the randomized, active-comparator-controlled, open-label, multicenter, phase III APPLY-PNH study. Blood. 2022;140(supple 2):LBA-2.
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  17. Danicopan [prescribing information]. Boston, MA: Alexion; March 2024.
  18. Kulasekararaj A, Griffin M, Piatek CI, et al. Danicopan as add-on therapy to ravulizumab or eculizumab versus placebo in patients with paroxysmal nocturnal hemoglobinuria and clinically significant extravascular hemolysis: phase 3 long-term data. Blood. 2023;142(suppl 1):576.
  19. Kulasekararaj A, Griffin M, Piatek C, et al. Long-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant EVH. Blood. 2025;145:811-822.
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  25. Nishimura J, Ando K, Masuko M, et al. Tesidolumab (LFG316) for treatment of C5-variant patients with paroxysmal nocturnal hemoglobinuria. Haematologica. 2022;107:1483-1488.
  26. Ueda Y, Obara N, Ueno S, et al. Treatment burden in patients with paroxysmal nocturnal hemoglobinuria: an in-depth interview survey. Ann Hematol. 2025;104:3575-3584.
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