Sociomedical Profile and Quality of Life of Paroxysmal Nocturnal Hemoglobinuria Patients with Suboptimal Response to Pathogenetic Therapy

BACKGROUND. Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired clonal non-neoplastic disorder of hematopoietic tissue characterized by complement-mediated hemolysis, bone marrow dysfunction, and higher risk of thrombotic and organic complications. This disease is associated with rapid rate of progression as well as worsening life expectancy and quality of life. Despite high efficacy of anti-complement treatment, a considerable proportion of patients show suboptimal response to pathogenetic therapy. Problems that patients encounter in everyday life, their quality of life, burden of disease, and limitations in professional life have not been adequately addressed in literature. AIM. To study sociomedical profile and quality-of-life indicators of PNH patients with suboptimal response to pathogenetic therapy and to assess the FACIT-Fatigue Scale in terms of its clinical information value and practical significance for analyzing the overall treatment success. MATERIALS & METHODS. As part of the multicenter observational clinical study, PNH patients with suboptimal response to pathogenetic therapy completed the EQ-5D and FACIT-Fatigue questionnaires as well as a checklist of main PNH symptoms, their disease, treatment, and professional life. The method of descriptive statistics as well as the Mann-Whitney test, the χ2 test, and correlation analysis were used to analyze the data. RESULTS. The study was based on the data from 45 PNH patients treated in 12 health centers in Russia (classic PNH accounted for 64.4 %). The age median was 41 years, 62 % of patients were women. The mean duration of suboptimal response to pathogenetic therapy was 3.2 years. Most patients had mobility issues (55.6 %), anxiety/depression (62.2 %), and hardships in their daily activities (68.9 %). In the vast majority of them, the disease had a moderate or considerable impact on their daily activities and social/family life. In 61 % of professionally active patients, the underlying disease moderately or considerably limited their professional life. Most patients reported fatigue (96 %), headache (86 %), dyspnea (83 %), concentration issues and sleep disorders (82 %), rapid heartbeat (77 %), yellowness of the skin (76 %), and back pain (71 %). Pronounced fatigue (with FACIT-Fatigue score of < 40) was registered in 70 % of patients. A significant correlation was established between FACIT-Fatigue and EQ-5D VAS scores as well as a relationship with the severity of dyspnea, tachycardia, and concentration loss. CONCLUSION. This clinical study demonstrated a considerably reduced quality of life of PNH patients with suboptimal response to pathogenetic therapy. It also confirmed the information value of the FACIT-Fatigue Scale for assessing fatigue and its impact on everyday life. This study clearly identified outstanding issues related to PNH patients with suboptimal response to pathogenetic therapy. That can contribute to bringing more patient-oriented care to this challenging population of hematologic patients.