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.

• T.I. Ionova NI Pirogov Clinic for High Medical Technology, Saint Petersburg State University, 154 Fontanki nab., Saint Petersburg, Russian Federation, 198103; Multinational Center for Quality of Life Research, 1 Artilleriiskaya ul., Saint Petersburg, Russian Federation, 191014 ; Клиника высоких медицинских технологий им. Н.И. Пирогова, ФГБОУ ВО «Санкт-Петербургский государственный университет», наб. р. Фонтанки, д. 154, Санкт-Петербург, Российская Федерация, 198103; РОО «Межнациональный центр исследования качества жизни», ул. Артиллерийская, д. 1, Санкт-Петербург, Российская Федерация, 191014 https://orcid.org/0000-0002-9431-5286 (unauthenticated)
• A.D. Kulagin RM Gorbacheva Research Institute, Pavlov University, 12 Rentgena ul., Saint Petersburg, Russian Federation, 197022 ; НИИ детской онкологии, гематологии и трансплантологии им. Р.М. Горбачевой, ФГБОУ ВО «Первый Санкт-Петербургский государственный медицинский университет им. акад. И.П. Павлова» Минздрава России, ул. Рентгена, д. 12, Санкт-Петербург, Российская Федерация, 197022 https://orcid.org/0000-0002-9589-4136 (unauthenticated)
• E.A. Lukina National Research Center for Hematology, 4 Novyi Zykovskii pr-d, Moscow, Russian Federation, 125167 ; ФГБУ «НМИЦ гематологии» Минздрава России, Новый Зыковский пр-д, д. 4, Москва, Российская Федерация, 125167 https://orcid.org/0000-0002-8774-850X (unauthenticated)
• V.V. Ptushkin Moscow Municipal Center for Hematology, SP Botkin City Clinical Hospital, 5 2-i Botkinskii pr-d, Moscow, Russian Federation, 125284 ; Московский городской гематологический центр, ГБУЗ «Городская клиническая больница им. С.П. Боткина ДЗМ», 2-й Боткинский пр-д, д. 5, Москва, Российская Федерация, 125284 https://orcid.org/0000-0002-9368-6050 (unauthenticated)
• E.G. Arshanskaya Moscow Municipal Center for Hematology, SP Botkin City Clinical Hospital, 5 2-i Botkinskii pr-d, Moscow, Russian Federation, 125284 ; Московский городской гематологический центр, ГБУЗ «Городская клиническая больница им. С.П. Боткина ДЗМ», 2-й Боткинский пр-д, д. 5, Москва, Российская Федерация, 125284 https://orcid.org/0000-0001-5906-9940 (unauthenticated)
• T.N. Babaeva Novosibirsk State Medical University, 52 Krasnyi pr-t, Novosibirsk, Russian Federation, 630091 ; ФГБОУ ВО «Новосибирский государственный медицинский университет» Минздрава России, Красный пр-т, д. 52, Новосибирск, Российская Федерация, 630091 https://orcid.org/0000-0002-2708-1133 (unauthenticated)
• B.A. Bakirov Bashkir State Medical University, 3 Lenina ul., Ufa, Republic of Bashkortostan, Russian Federation, 450008 ; ФГБОУ ВО «Башкирский государственный медицинский университет» Минздрава России, ул. Ленина, д. 3, Уфа, Республика Башкортостан, Российская Федерация, 450008 https://orcid.org/0000-0002-3297-1608 (unauthenticated)
• S.V. Voloshin Leningrad Regional Clinical Hospital, 45 korp. 2A Lunacharskogo pr-t, Saint Petersburg, Russian Federation, 194291; SM Kirov Military Medical Academy, 6 Akademika Lebedeva ul., Saint Petersburg, Russian Federation, 194044 ; ГБУЗ «Ленинградская областная клиническая больница», пр-т Луначарского, д. 45, корп. 2А, Санкт-Петербург, Российская Федерация, 194291; ФГБВОУ ВО «Военно-медицинская академия им. С.М. Кирова» Минобороны России, ул. Академика Лебедева, д. 6, Санкт-Петербург, Российская Федерация, 194044 https://orcid.org/0000-0003-1784-0375 (unauthenticated)
• G.P. Dimov Municipal Clinical Hospital No. 1, 16 Vorovskogo ul., Chelyabinsk, Russian Federation, 454048 ; ГАУЗ Ордена Трудового Красного Знамени «Городская клиническая больница № 1», ул. Воровского, д. 16, Челябинск, Российская Федерация, 454048 https://orcid.org/0000-0002-5576-1961 (unauthenticated)
• O.U. Klimova RM Gorbacheva Research Institute, Pavlov University, 12 Rentgena ul., Saint Petersburg, Russian Federation, 197022 ; НИИ детской онкологии, гематологии и трансплантологии им. Р.М. Горбачевой, ФГБОУ ВО «Первый Санкт-Петербургский государственный медицинский университет им. акад. И.П. Павлова» Минздрава России, ул. Рентгена, д. 12, Санкт-Петербург, Российская Федерация, 197022 https://orcid.org/0000-0001-7238-729X (unauthenticated)
• E.Yu. Komarceva Rostov Regional Clinical Hospital, 170 Blagodatnaya ul., West Residential Area, Rostov-on-Don, Russian Federation, 344015 ; ГБУ РО «Ростовская областная клиническая больница», ул. Благодатная, д. 170, Западный жилой массив, Ростов-на-Дону, Российская Федерация, 344015 https://orcid.org/0000-0002-8952-2643 (unauthenticated)
• O.L. Panteleeva ; ГБУЗ ВО «Областная клиническая больница», Судогодское ш., д. 41, Владимир, Российская Федерация, 600023 https://orcid.org/0000-0002-6616-656X (unauthenticated)
• R.V. Ponomarev National Research Center for Hematology, 4 Novyi Zykovskii pr-d, Moscow, Russian Federation, 125167 ; ФГБУ «НМИЦ гематологии» Минздрава России, Новый Зыковский пр-д, д. 4, Москва, Российская Федерация, 125167 https://orcid.org/0000-0002-1218-0796 (unauthenticated)
• T.I. Pospelova Novosibirsk State Medical University, 52 Krasnyi pr-t, Novosibirsk, Russian Federation, 630091 ; ФГБОУ ВО «Новосибирский государственный медицинский университет» Минздрава России, Красный пр-т, д. 52, Новосибирск, Российская Федерация, 630091 https://orcid.org/0000-0002-1261-5470 (unauthenticated)
• S.V. Samarina Kirov Research Institute of Hematology and Transfusiology, 72 Krasnoarmeiskaya ul., Kirov, Russian Federation, 610027 ; ФГБУН «Кировский НИИ гематологии и переливания крови ФМБА», ул. Красноармейская, д. 72, Киров, Российская Федерация, 610027 https://orcid.org/0000-0001-8639-719X (unauthenticated)
• N.O. Saraeva Irkutsk State Medical University, 1 Krasnogo Vosstaniya ul., Irkutsk, Russian Federation, 664003 ; ФГБУ ВО «Иркутский государственный медицинский университет» Минздрава России, ул. Красного Восстания, д. 1, Иркутск, Российская Федерация, 664003 https://orcid.org/0009-0004-9849-3137 (unauthenticated)
• K.O. Farizova Leningrad Regional Clinical Hospital, 45 korp. 2A Lunacharskogo pr-t, Saint Petersburg, Russian Federation, 194291 ; ГБУЗ «Ленинградская областная клиническая больница», пр-т Луначарского, д. 45, корп. 2А, Санкт-Петербург, Российская Федерация, 194291 https://orcid.org/0000-0002-7016-0750 (unauthenticated)
• M.N. Finyakina National Research Center for Hematology, 4 Novyi Zykovskii pr-d, Moscow, Russian Federation, 125167 ; ФГБУ «НМИЦ гематологии» Минздрава России, Новый Зыковский пр-д, д. 4, Москва, Российская Федерация, 125167 https://orcid.org/0009-0001-3317-5718 (unauthenticated)
• A.A. Shutylev Perm Krai Clinical Hospital, 85 Pushkina ul., Perm, Russian Federation, 614990 ; ГБУЗ ПК Ордена «Знак Почета» «Пермская краевая клиническая больница», ул. Пушкина, д. 85, Пермь, Российская Федерация, 614990 https://orcid.org/0000-0003-2859-4229 (unauthenticated)
• D.A. Lipatova Multinational Center for Quality of Life Research, 1 Artilleriiskaya ul., Saint Petersburg, Russian Federation, 191014 ; РОО «Межнациональный центр исследования качества жизни», ул. Артиллерийская, д. 1, Санкт-Петербург, Российская Федерация, 191014 https://orcid.org/0009-0009-0007-6105 (unauthenticated)
• N.M. Porfirieva Multinational Center for Quality of Life Research, 1 Artilleriiskaya ul., Saint Petersburg, Russian Federation, 191014 ; РОО «Межнациональный центр исследования качества жизни», ул. Артиллерийская, д. 1, Санкт-Петербург, Российская Федерация, 191014 https://orcid.org/0000-0003-3329-2743 (unauthenticated)
• Tatyana Pavlovna Nikitina NI Pirogov Clinic for High Medical Technology, Saint Petersburg State University, 154 Fontanki nab., Saint Petersburg, Russian Federation, 198103; Multinational Center for Quality of Life Research, 1 Artilleriiskaya ul., Saint Petersburg, Russian Federation, 191014 ; Клиника высоких медицинских технологий им. Н.И. Пирогова, ФГБОУ ВО «Санкт-Петербургский государственный университет», наб. р. Фонтанки, д. 154, Санкт-Петербург, Российская Федерация, 198103; РОО «Межнациональный центр исследования качества жизни», ул. Артиллерийская, д. 1, Санкт-Петербург, Российская Федерация, 191014 https://orcid.org/0000-0002-8279-8129 (unauthenticated)

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