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Review
. 2022 Jun 17:13:327-335.
doi: 10.2147/JBM.S339660. eCollection 2022.

Managing Fatigue in Patients with Paroxysmal Nocturnal Hemoglobinuria: A Patient-Focused Perspective

Bruno Fattizzo  1   2 , Francesca Cavallaro  1   2 , Esther Natalie Oliva  3 , Wilma Barcellini  1
Affiliations
Review

Managing Fatigue in Patients with Paroxysmal Nocturnal Hemoglobinuria: A Patient-Focused Perspective

Bruno Fattizzo et al. J Blood Med. .

Abstract

The most frequently reported symptom in patients with paroxysmal nocturnal hemoglobinuria (PNH), a disease characterized by complement mediated hemolysis and chronic anemia, is "fatigue". The latter seems the best word to communicate patient' perception of personal health status and disease impact on daily living, namely quality of life (QoL). Objectivating QoL and grading patient's fatigue is one of the most difficult medical tasks given the highly heterogeneous communication skills of patients and caregivers and the multitude of meanings that might be attributed to this term. Along with anemia, QoL in PNH is also affected by the emotional burden of a chronic life-long disease with heterogeneous treatment requirement, risk of hemolytic exacerbations (breakthrough hemolysis) and of thrombosis. In the last decade, structured surveys and scores have been adapted from cancer settings to evaluate fatigue and QoL in patients with PNH, and to assess the benefit of complement inhibitors in this setting. Eculizumab was the first drug utilized and was shown to improve QoL scores in the registrative trials. However, the intravenous fortnightly administration, the presence of residual anemia, and the risk of extravascular hemolysis are some of the unmet needs impacting QoL under eculizumab. Several novel drugs have been designed to improve patients' convenience and alleviate anemia and fatigue. In this review, we focus on available studies that evaluated fatigue and QoL in PNH patients, and the effect of old and new therapeutic strategies.

Keywords: eculizumab; fatigue; novel drugs; paroxysmal nocturnal hemoglobinuria; quality of life.

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Conflict of interest statement

Dr Bruno Fattizzo received consultancy honoraria and speaker bureau from Alexion, Amgegen, Annexon, Jannsen, and Novartis. Dr Esther Natalie Oliva reports personal fees from Celgene, Janssen, personal fees, Daiichi, and Alexion, outside the submitted work. Dr Wilma Barcellini received consultancy honoraria and speaker bureau from Agios, Alexion, Amgegen, Annexon, Jannsen, and Novartis. Dr Francesca Cavallaro declares no conflicts of interest to disclose.

References

    1. US Food and Drug Administration. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents.... Accessed May 24, 2022. - PMC - PubMed
    1. Walton MK, Powers JH 3rd, Hobart J, et al. International society for pharmacoeconomics and outcomes research task force for clinical outcomes assessment. clinical outcome assessments: conceptual foundation-report of the ISPOR clinical outcomes assessment - emerging good practices for outcomes research task force. Value Health. 2015;18(6):741–752. doi:10.1016/j.jval.2015.08.006 - DOI - PMC - PubMed
    1. Parker CJ. Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria. Hematol Am Soc Hematol Educ Program. 2016;2016(1):208–216. doi:10.1182/asheducation-2016.1.208 - DOI - PMC - PubMed
    1. Kinoshita T, Inoue N, Takeda J. Defective glycosyl phosphatidylinositol anchor synthesis and paroxysmal nocturnal hemoglobinuria. Adv Immunol. 1995;60:57–103. - PubMed
    1. Parker CJ. Hemolysis in PNH. In: Young NS, Moss J, editors. Paroxysmal Nocturnal Hemoglobinuria and the Glycosylphosphatidylinositol-Linked Proteins. San Diego: Academic Press; 2000:49–100.