Oral iptacopan monotherapy in paroxysmal nocturnal haemoglobinuria: final 48-week results from the open-label, randomised, phase 3 APPLY-PNH trial in anti-C5-treated patients and the open-label, single-arm, phase 3 APPOINT-PNH trial in patients previously untreated with complement inhibitors.

Risitano AM, Kulasekararaj AG, Scheinberg P, Röth A, Han B, Maciejewski JP, Ueda Y, de Castro CM, Di Bona E, Fu R, Zhang L, Griffin M, Langemeijer SMC, Panse J, Schrezenmeier H, Barcellini W, Mauad VAQ, Schafhausen P, Tavitian S, Beggiato E, Chew LP, Gaya A, Huang WH, Jang JH, Kitawaki T, Kutlar A, Notaro R, Pullarkat V, Schubert J, Terriou L, Uchiyama M, Lee LWL, Yap ES, Frieri C, Marano L, de Fontbrune FS, Gandhi S, Trikha R, Alashkar F, Yang C, Liu H, Kelly RJ, Höchsmann B, Lawniczek T, Mahajan N, Solar-Yohay S, Kerloëguen C, Ferber P, Kumar R, Wang Z, Thorburn C, Maitra S, Li S, Verles A, Dahlke M, de Latour RP.

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DOI: 10.1016/s2352-3026(25)00081-x
Published: 2025-06-01
Container: Lancet Haematol
Publisher: Not recorded
Open access: no

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Cite this work

BibTeX

@article{allodium:10.1016/s2352-3026-25-00081-x,
  title = {Oral iptacopan monotherapy in paroxysmal nocturnal haemoglobinuria: final 48-week results from the open-label, randomised, phase 3 APPLY-PNH trial in anti-C5-treated patients and the open-label, single-arm, phase 3 APPOINT-PNH trial in patients previously untreated with complement inhibitors.},
  author = {Risitano AM and  Kulasekararaj AG and  Scheinberg P and  Röth A and  Han B and  Maciejewski JP and  Ueda Y and  de Castro CM and  Di Bona E and  Fu R and  Zhang L and  Griffin M and  Langemeijer SMC and  Panse J and  Schrezenmeier H and  Barcellini W and  Mauad VAQ and  Schafhausen P and  Tavitian S and  Beggiato E and  Chew LP and  Gaya A and  Huang WH and  Jang JH and  Kitawaki T and  Kutlar A and  Notaro R and  Pullarkat V and  Schubert J and  Terriou L and  Uchiyama M and  Lee LWL and  Yap ES and  Frieri C and  Marano L and  de Fontbrune FS and  Gandhi S and  Trikha R and  Alashkar F and  Yang C and  Liu H and  Kelly RJ and  Höchsmann B and  Lawniczek T and  Mahajan N and  Solar-Yohay S and  Kerloëguen C and  Ferber P and  Kumar R and  Wang Z and  Thorburn C and  Maitra S and  Li S and  Verles A and  Dahlke M and  de Latour RP.},
  year = {2025},
  journal = {Lancet Haematol},
  doi = {10.1016/s2352-3026(25)00081-x},
  url = {https://doi.org/10.1016/s2352-3026(25)00081-x}
}

RIS

TY  - JOUR
TI  - Oral iptacopan monotherapy in paroxysmal nocturnal haemoglobinuria: final 48-week results from the open-label, randomised, phase 3 APPLY-PNH trial in anti-C5-treated patients and the open-label, single-arm, phase 3 APPOINT-PNH trial in patients previously untreated with complement inhibitors.
AU  - Risitano AM
AU  -  Kulasekararaj AG
AU  -  Scheinberg P
AU  -  Röth A
AU  -  Han B
AU  -  Maciejewski JP
AU  -  Ueda Y
AU  -  de Castro CM
AU  -  Di Bona E
AU  -  Fu R
AU  -  Zhang L
AU  -  Griffin M
AU  -  Langemeijer SMC
AU  -  Panse J
AU  -  Schrezenmeier H
AU  -  Barcellini W
AU  -  Mauad VAQ
AU  -  Schafhausen P
AU  -  Tavitian S
AU  -  Beggiato E
AU  -  Chew LP
AU  -  Gaya A
AU  -  Huang WH
AU  -  Jang JH
AU  -  Kitawaki T
AU  -  Kutlar A
AU  -  Notaro R
AU  -  Pullarkat V
AU  -  Schubert J
AU  -  Terriou L
AU  -  Uchiyama M
AU  -  Lee LWL
AU  -  Yap ES
AU  -  Frieri C
AU  -  Marano L
AU  -  de Fontbrune FS
AU  -  Gandhi S
AU  -  Trikha R
AU  -  Alashkar F
AU  -  Yang C
AU  -  Liu H
AU  -  Kelly RJ
AU  -  Höchsmann B
AU  -  Lawniczek T
AU  -  Mahajan N
AU  -  Solar-Yohay S
AU  -  Kerloëguen C
AU  -  Ferber P
AU  -  Kumar R
AU  -  Wang Z
AU  -  Thorburn C
AU  -  Maitra S
AU  -  Li S
AU  -  Verles A
AU  -  Dahlke M
AU  -  de Latour RP.
PY  - 2025
JO  - Lancet Haematol
DO  - 10.1016/s2352-3026(25)00081-x
UR  - https://doi.org/10.1016/s2352-3026(25)00081-x
ER  -

APA

AM, R., AG, K., P, S., A, R., B, H., JP, M., Y, U., CM, D. C., E, D. B., R, F., L, Z., M, G., SMC, L., J, P., H, S., W, B., VAQ, M., P, S., S, T., E, B., LP, C., A, G., WH, H., JH, J., T, K., A, K., R, N., V, P., J, S., L, T., M, U., LWL, L., ES, Y., C, F., L, M., FS, D. F., S, G., R, T., F, A., C, Y., H, L., RJ, K., B, H., T, L., N, M., S, S., C, K., P, F., R, K., Z, W., C, T., S, M., S, L., A, V., M, D., & RP., D. L. (2025). Oral iptacopan monotherapy in paroxysmal nocturnal haemoglobinuria: final 48-week results from the open-label, randomised, phase 3 APPLY-PNH trial in anti-C5-treated patients and the open-label, single-arm, phase 3 APPOINT-PNH trial in patients previously untreated with complement inhibitors.. Lancet Haematol. https://doi.org/10.1016/s2352-3026(25)00081-x

Source records

europe-pmc · retrieved 2026-09-26T18:24:50.232Z
hal · retrieved 2026-09-26T18:24:50.254Z