Clinical Experience of Iptacopan in Paroxysmal Nocturnal Hemoglobinuria
In brief
Iptacopan raises hemoglobin to at least 120 g/L in 76% by one year
In this retrospective study of 151 people with paroxysmal nocturnal hemoglobinuria, mean hemoglobin rose from 104 to 127 g/L by three months, and 88% became transfusion independent. Responses were similar with or without a history of bone marrow failure; no thromboses or drug-related deaths occurred, but longer follow-up is needed to clarify long-term infection risk and effectiveness.
- Journal
- American journal of hematology (Q1)
- Published
- 27 October 2026
- Study design
- Retrospective cohort
- Evidence level
- Level 3, Low (CEBM 3b)
- Authors
- Matthew Holt, Richard J Kelly, Regis Peffault de Latour, Morag Griffin, Shreyans Gandhi, Edouard Forcade, et al.
- PMID
- 42946388
- DOI
- 10.1002/ajh.70531
Why clinicians should know about it
- Picked for Hematology (paper of the day, 29 October 2026): Real-world iptacopan outcomes in PNH, including marrow failure
Abstract
Iptacopan, an oral Factor B inhibitor, is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). In clinical trials it was safe and effective but real-world data, particularly in patients with associated bone marrow failure, remain limited. A retrospective, multicenter study was conducted in patients with PNH who received iptacopan monotherapy in the UK and France between July 2021 and July 2025. Patient demographics, prior treatment and indication for switch were collected, as were hematological parameters at baseline, 3, 12 and 24 months. Outcomes included breakthrough hemolysis, infections, thrombotic events, treatment discontinuation and death. A total of 151 patients were included with a median treatment duration of 238 days. Iptacopan produced significant increases in mean hemoglobin, from 104 g/L at baseline to 127 g/L at 3 months, with improvements sustained to 24 months. A total of 76% achieved a hemoglobin ≥ 120 g/L by 12 months, compared with 21.9% at baseline, with transfusion independency in 88%. There was no statistical difference in responses between patients with and without history of bone marrow failure. Breakthrough hemolysis occurred in 6.6% of patients, with a mean hemoglobin fall of 24 g/L. Suspected bacterial infections occurred at a rate of 0.18 per patient-year, but there were no meningococcal or pneumococcal infections. There were no thromboses, and no deaths related to iptacopan. This cohort demonstrates that iptacopan produces sustained improvements in patients with PNH, irrespective of prior complement inhibition or history of bone marrow failure. Longer follow-up is needed to characterize long-term efficacy and infection risk.
Abstract as published, via PubMed.
For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.