Abstract Topic: 35. Quality of life and palliative care
Background: Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare hematological disease with an estimated global prevalence of 15.9 individuals per 1 million people1. Due to the rarity of the condition, in many countries PNH patients often do not receive adequate services from the healthcare system and socio-economic wellbeing of this population remains largely out of focus of attention of social services, donors and communities. At best, PNH is seen collectively within the rare diseases framework, without any specificity.
Patient organizations help to improve the visibility of the disease in the care space. In 2021, Ukrainian PNH patients formed their own community organization - “PNH Ukraine”, that united patients living with the disease and their families. The organization facilitated access to treatment (mainly BMT and C5 inhibitors) and clinical trials for its members and, with the start of the Russian invasion to Ukraine, coordinated evacuations of patients to European countries.
Aims: After one year of the brutal war in Ukraine, “PNH Ukraine” took a closer look at its impact on the quality of life and the health status its constituents. A small survey was conducted among patients to characterize and measure this impact.
Methods: Patients living with PNH were invited to respond to an online questionnaire of 28 questions addressing their basic needs, health status and the level of care and support that patients received during the past year. All this information was meant to approximate the change in the quality of life and was supplemented by the health status assessments (patient-reported) using EQ-5D-5L scale before the war (historic data) and one year into the war. The validity of this evaluation is limited by a small number of respondents and lack of adjustments for possible confounders but overall illustrates well the effects of the war.
Results: 12 patients responded to the survey, all Ukrainians diagnosed with confirmed PNH, mean age 38.
Analysis of the provided responses showed in the Table 1. It demonstrates that the at least half of the respondents experienced severe disruptions of all aspects of socio-economic wellbeing, including safety, housing, employment, access to healthcare and welfare support with two thirds having lived endangerment, displacement, and lack of medicines or discontinuation of treatments. At that, 86% of the displaced respondents want to return to Ukraine and 14% plan to do it.
Paradoxically, 42% of respondents were able to access treatment with C5 inhibitors earlier unavailable in Ukraine due to war-induced emigration to Europe and 50% started therapy within clinical trials. Therefore while overall impact that the Russian aggression has on the the quality of life of Ukrainian PNH patients is estimated as negative by 91% of patients; their mean EQ-5D-5L health status score increased by 15.49 points within last year from 54.6 in early 2022 (n=5) to 70.09 in February 2023 (n=12).
Summary/Conclusion: While the war has affected the quality of life of Ukrainian PNH patients in an extremely negative way, it also opened the door to receiving pathogenetic treatment in EU to many of the patients, which significantly improved the group’s mean HRQoL score. This leads to a substantiated assumption that the lack of treatment affects the lives of PNH patients in way commensurate with the effects of war.
1https://www.rarediseaseadvisor.com/disease-info-pages/paroxysmal-nocturnal-hemoglobinuria-epidemiology/
Keywords: Quality of life, Paroxysmal nocturnal hemoglobinuria (PNH)