Moving toward Individual Treatment Goals with Pegcetacoplan in Patients with PNH and Impaired Bone Marrow Function
- Author(s)
- Szer, J; Panse, J; Kulasekararaj, A; Oliver, M; Fattizzo, B; Nishimura, JI; Horneff, R; Szamosi, J; Peffault de Latour, R;
- Details
- Publication Year 2024-08-06,Volume 25,Issue #16,Page 8591
- Journal Title
- International Journal of Molecular Sciences
- Publication Type
- Research article
- Abstract
- Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, potentially life-threatening haematological disease characterised by chronic complement-mediated haemolysis with multiple clinical consequences that impair quality of life. This post hoc analysis assessed haematological and clinical responses to the first targeted complement C3 inhibitor pegcetacoplan in patients with PNH and impaired bone marrow function in the PEGASUS (NCT03500549) and PRINCE (NCT04085601) studies. For patients with impaired bone marrow function, defined herein as haemoglobin <10 g/dL and absolute neutrophil count <1.5 × 10(9) cells/L, normalisation of the parameters may be difficult. Indeed, 20% and 43% had normalised haemoglobin in PEGASUS and PRINCE, respectively; 60% and 57% had normalised LDH, and 40% and 29% had normalised fatigue scores. A new set of parameters was applied using changes associated with clinically meaningful improvements, namely an increase in haemoglobin to ≥2 g/dL above baseline, decrease in LDH to ≤1.5× the upper limit of normal, and an increase in fatigue scores to ≥5 points above baseline. With these new parameters, 40% and 71% of PEGASUS and PRINCE patients had improved haemoglobin; 60% and 71% had an improvement in LDH, and 60% and 43% had an improvement in fatigue scores. Thus, even patients with impaired bone marrow function may achieve clinically meaningful improvements with pegcetacoplan.
- Publisher
- MDPI
- Keywords
- Humans; *Hemoglobinuria, Paroxysmal; Male; Female; Middle Aged; *Bone Marrow/metabolism; Adult; Hemoglobins/metabolism; Aged; Complement C3/metabolism; Treatment Outcome; Quality of Life; L-Lactate Dehydrogenase/blood/metabolism; Pnh; bone marrow dysfunction; clinical response; haematological response; pegcetacoplan
- Department(s)
- Clinical Haematology
- Publisher's Version
- https://doi.org/10.3390/ijms25168591
- Open Access at Publisher's Site
https://doi.org/10.3390/ijms25168591- Terms of Use/Rights Notice
- Refer to copyright notice on published article.
Creation Date: 2024-09-24 02:20:16
Last Modified: 2024-09-24 02:22:35