Contributions
Abstract: EP1191
Type: E-Poster Presentation
Session title: Quality of life, palliative care, ethics and health economics
Background
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired and potentially life-threatening hematologic disease, characterized by hemolytic anemia, thrombosis and impaired bone marrow function. Treatment with the C5 inhibitors has resulted in a reduction in intravascular hemolysis and improvements in morbidity and mortality. Despite the clinical benefit for PNH patients (pts), real world evidence studies indicate that pts treated with C5 inhibitors eculizumab (ECU) and ravulizumab (RAV) experience continued impairment in productivity and overall quality-of-life (QoL), and that a significant proportion of pts, besides being anemic continues to experience fatigue and other symptoms.1
Aims
Assess the burden of illness in European PNH pts currently treated with C5 inhibitors, from a clinical, humanistic and economic perspective. Here, we present interim results to specifically, assess the impact of PNH on QoL and work productivity in a subgroup of pts receiving ECU in Germany, France, and the United Kingdom (UK), or RAV (Germany only).
Methods
A cross-sectional online survey administered to pts with self-reported PNH diagnosis. The survey, initiated in February 2021, aims to recruit 90 pts. Following a Central Institutional Review Board exemption, convenience sampling was used to recruit pts through patient advocacy groups in each country. Inclusion criteria required a self-reported PNH diagnosis, age ≥18 years, current treatment with ECU or RAV and agreement to informed consent and adverse event reporting. To assess the burden of illness in these pts, Work Productivity and Activity Impairment–General Health questionnaire, Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale (total score range: 0-52), and European Organization for Research and Treatment of Cancer QoL Questionnaire (EORTC-QLQ-C30; total score range: 0-100) were analyzed. For the interim analyses reported here, descriptive statistics are presented.
Results
In February 2021, 37 pts were included in the study, among which 21 were residing in Germany, 4 in France, and 12 in the UK. Pts with self-reported PNH diagnosis had a median age of 43 years (range 19-74) and most were female (70%). There were 32% pts on ECU, and 68% pts on RAV; all pts had been on treatment for at least 3 months and most for at least 1 year (96% ECU and 83% RAV). The FACIT-Fatigue total score (mean[±SD]: 35[±13]) was lower than the general population mean (~44, determined in a German population); and the EORTC-QLQ-C30 global health status score (67[±18]) was also below the mean of the European general population (~76). Furthermore, the EORTC-QLC-C30 physical functioning score mean (84[±15]) averaged lower than the general population mean (~93). Pts also reported 35%[±29] impairment in normal daily activities in the prior 7 days (37%[±31] ECU, 32%[±26] RAV). The 22 pts (60%) employed at the time of the study reported 6%[±22] absenteeism (proportion of work time lost due to being absent; ECU: 10% [±28], RAV: 1%[±4]) and 20%[±24] presenteeism (productivity impairment while working; ECU: 21%±[26], RAV: 19%[±23]). Total work productivity impairment was 29%[±32] (ECU: 48%[±34], RAV: 19%[±23]).
Conclusion
This burden of illness interim analysis among European PNH pts indicates a substantial unmet need in regards to economic burden and QoL as the results show relevant work-related productivity loss, diminished ability to work, limitations in daily life activities, and reduced QoL, despite treatment with ECU or RAV.
1Levy AR et al. Blood. 2019;134(Supplement_1):4803
Keyword(s): Fatigue, Inhibitor, Paroxysmal nocturnal hemoglobinuria (PNH), Quality of life
Abstract: EP1191
Type: E-Poster Presentation
Session title: Quality of life, palliative care, ethics and health economics
Background
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired and potentially life-threatening hematologic disease, characterized by hemolytic anemia, thrombosis and impaired bone marrow function. Treatment with the C5 inhibitors has resulted in a reduction in intravascular hemolysis and improvements in morbidity and mortality. Despite the clinical benefit for PNH patients (pts), real world evidence studies indicate that pts treated with C5 inhibitors eculizumab (ECU) and ravulizumab (RAV) experience continued impairment in productivity and overall quality-of-life (QoL), and that a significant proportion of pts, besides being anemic continues to experience fatigue and other symptoms.1
Aims
Assess the burden of illness in European PNH pts currently treated with C5 inhibitors, from a clinical, humanistic and economic perspective. Here, we present interim results to specifically, assess the impact of PNH on QoL and work productivity in a subgroup of pts receiving ECU in Germany, France, and the United Kingdom (UK), or RAV (Germany only).
Methods
A cross-sectional online survey administered to pts with self-reported PNH diagnosis. The survey, initiated in February 2021, aims to recruit 90 pts. Following a Central Institutional Review Board exemption, convenience sampling was used to recruit pts through patient advocacy groups in each country. Inclusion criteria required a self-reported PNH diagnosis, age ≥18 years, current treatment with ECU or RAV and agreement to informed consent and adverse event reporting. To assess the burden of illness in these pts, Work Productivity and Activity Impairment–General Health questionnaire, Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale (total score range: 0-52), and European Organization for Research and Treatment of Cancer QoL Questionnaire (EORTC-QLQ-C30; total score range: 0-100) were analyzed. For the interim analyses reported here, descriptive statistics are presented.
Results
In February 2021, 37 pts were included in the study, among which 21 were residing in Germany, 4 in France, and 12 in the UK. Pts with self-reported PNH diagnosis had a median age of 43 years (range 19-74) and most were female (70%). There were 32% pts on ECU, and 68% pts on RAV; all pts had been on treatment for at least 3 months and most for at least 1 year (96% ECU and 83% RAV). The FACIT-Fatigue total score (mean[±SD]: 35[±13]) was lower than the general population mean (~44, determined in a German population); and the EORTC-QLQ-C30 global health status score (67[±18]) was also below the mean of the European general population (~76). Furthermore, the EORTC-QLC-C30 physical functioning score mean (84[±15]) averaged lower than the general population mean (~93). Pts also reported 35%[±29] impairment in normal daily activities in the prior 7 days (37%[±31] ECU, 32%[±26] RAV). The 22 pts (60%) employed at the time of the study reported 6%[±22] absenteeism (proportion of work time lost due to being absent; ECU: 10% [±28], RAV: 1%[±4]) and 20%[±24] presenteeism (productivity impairment while working; ECU: 21%±[26], RAV: 19%[±23]). Total work productivity impairment was 29%[±32] (ECU: 48%[±34], RAV: 19%[±23]).
Conclusion
This burden of illness interim analysis among European PNH pts indicates a substantial unmet need in regards to economic burden and QoL as the results show relevant work-related productivity loss, diminished ability to work, limitations in daily life activities, and reduced QoL, despite treatment with ECU or RAV.
1Levy AR et al. Blood. 2019;134(Supplement_1):4803
Keyword(s): Fatigue, Inhibitor, Paroxysmal nocturnal hemoglobinuria (PNH), Quality of life