In patients with paroxysmal nocturnal hemoglobinuria (PNH), 5-level EQ-5D version (EQ-5D-5L) data were necessary to conduct a cost-utility analysis (CUA) of treatment with pegcetacoplan, according to findings from a direct mapping study published in the Journal of Comparative Effectiveness Research.

PNH is associated with intravascular hemolysis, extravascular hemolysis, thrombocytosis, and bone marrow failure (eg, aplastic anemia), which are caused by genetic mutations in hematopoietic stem cells. The novel C3 inhibitor pegcetacoplan was recently approved by the US Food and Drug Administration and the European Medicines Agency for the treatment of adult patients with PNH who continue to exhibit anemia following treatment with a C5 inhibitor, such as eculizumab, for 3 months or longer.

Recognizing that to date no relevant algorithm had been published, the researchers sought to establish a mapping algorithm to estimate EQ-5D-5L health-state utilities from the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) data among individuals with PNH.

The authors collected data from an online cross-sectional PNH patient survey performed in France, Germany, and the United Kingdom between January 1, 2021, and March 31, 2021. Participants presented data on their health-related quality of life (HRQoL), according to findings from the EORTC QLQ-C30 and EQ-5D-5L, along with demographic and baseline characteristics, such as hemoglobin (Hb) level and any history of packed red blood cell transfusions.   

The algorithm was validated with use of patient data from the phase 3, randomized, controlled PEGASUS trial of pegcetacoplan vs eculizumab in adults with PNH. After the mapping exercise was conducted, EQ-5D-5L health-state utilities were computed with patient-level EORTC QLQ-C30 data.

As the source measure used for the mapping, the EORTC QLQ-C30 evaluates important domains of functioning (eg, physical functioning, cognitive functioning, emotional functioning, role functioning, social functioning); common symptoms (eg, nausea/vomiting, constipation, diarrhea, loss of appetite, fatigue, pain, insomnia, dyspnea); and other outcomes (eg, financial difficulties, global QoL associated with cancer).

The EQ-5D assesses 5 dimensions of HRQoL: mobility, self-care, usual activities, pain/discomfort, anxiety/depression. Based on the version of EQ-5D used, the severity of each of the dimensions can be evaluated with use of a 3-level (ie, EQ-5D-3L or EQ-5D-Y-3L [youth version]) or a 5-level (ie, EQ-5D-5L) response scale.

Learn about experimental therapies for paroxysmal nocturnal hemoglobinuria

A total of 71 participants—20 from France, 31 from Germany, and 20 from the United Kingdom—completed the survey. The mean patient age was 43.0±13.1 years. Overall, 66.2% of the survey sample were women. Mean participant age at diagnosis of PNH was 29.8±11.6 years. The mean serum Hb level was 10.2±2.0 g/dL. Aplastic anemia was identified in 36 patients and was the most common PNH-linked comorbidity.

Study results showed that utilities mapped with use of the EQ-5D-5L French value set ranged from 0.58±0.42 to 0.89±0.10, thus exhibiting the best predictive validity.

“The PNH EQ-5D-5L direct mapping model . . . enables calculation of reliable health-state utility data required for CUA evaluating treatments of PNH,” the researchers concluded.


Wojciechowski P, Wdowiak M, Hakimi Z, et al. Mapping the EORTC QLQ-C30 onto the EQ-5D-5L index for patients with paroxysmal nocturnal hemoglobinuria in France. J Comp Eff Res. Published online April 13, 2023. doi:10.57264/cer-2022-0178