The following article features coverage from the 62nd Annual Scientific Meeting of the British Society for Haematology. Click here to read more of Hematology Advisor‘s conference coverage.

A long-term analysis of patients with paroxysmal nocturnal hemoglobinuria (PNH) suggests that treatment with eculizumab is associated with survival benefits. This is according to study results presented at the 62nd Annual Scientific Meeting of the British Society for Haematology by Morag Griffin, MBChB, FRCPath, of Leeds Teaching Hospitals NHS Trust in Leeds, UK, and colleagues.

The study was based on data from the International PNH Registry ( Identifier: NCT01374360) involving eculizumab-treated patients (“ever-treated” patients) and patients who had never received eculizumab (“never-treated” patients). Those in the ever-treated group had received eculizumab for at least 35 days during enrollment in the Registry.

This analysis primarily focused on patients’ baseline characteristics and overall survival results. Baseline was defined as the start of eculizumab treatment for ever-treated patients, and it was defined as enrollment into the Registry for never-treated patients. Patients were also evaluated based on presence or absence of baseline high disease activity (HDA), which was based on a lactate dehydrogenase (LDH) ratio ≥1.5 times the upper limit of normal and 1 or more factors from a set of several possible disease-related features.

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A total of 4627 patients were included in this study, with 1892 patients in the ever-treated group and 2735 patients in the never-treated group. The mean age at the start of disease was 40.2 years across the population, and the mean age at enrollment was 45.7 years.

Most (90%) of the ever-treated patients showed an LDH ratio of ≥1.5 times the upper limit of normal, while 35% of the never-treated population did. Only 3% of patients in the ever-treated group had a granulocyte clone size of <10%, compared with 57% of patients in the never-treated group. Also, fewer (45%) patients in the ever-treated group had a history of bone marrow failure, compared with the never-treated group (76%).

Ever-treated patients showed a significantly lower mortality risk than never-treated patients did (hazard ratio [HR], 0.48; 95% CI, 0.39-0.60; P <.0001). In the ever-treated group, the reduction in mortality risk appeared strongest for patients who had evidence of HDA at baseline (HR, 0.46; 95% CI, 0.33-0.64). Patients in the ever-treated group who did not have HDA also showed reduced mortality (HR, 0.65; 95% CI, 0.39-1.10), as did patients with unknown HDA status at baseline (HR, 0.50; 95% CI, 0.32-0.76).

The study investigators reported that through 20 years of follow-up, patients in the ever-treated group had a 20-year survival probability of 82%, compared with 69% in the never-treated population. Although patients with baseline HDA showed the greatest long-term survival probability, the investigators reported that patients in all HDA categories showed long-term survival benefits in the ever-treated group.

The study investigators concluded that survival benefits were seen with eculizumab, regardless of baseline HDA status. However, survival benefits with this agent appeared greater for those with baseline HDA than for those without HDA.

Disclosures: Some authors have declared affiliations with or received grant support from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.

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Griffin M, Terriou L, Patriquin CJ, et al. Long-term survival benefit of eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria: data from the International PNH Registry. Presented at: 62nd Annual Scientific Meeting of the British Society for Haematology; April 3-5, 2022. Abstract OR06.