Claims analyses are also unable to capture specific data related to the patient perspective and QoL. represents the number of individuals that had at least experienced a thrombotic event/transfusion once in their lifetime and who were on C5i treatment (ECU or RAV) for one or more years. b Most recent patient-reported Hb levels. represents the number of survey participants that reported their most recent Rusalatide acetate Hb levels (overall, for ECU users; for RAV users; em PNH /em , paroxysmal nocturnal hemoglobinuria; em RAV /em , ravulizumab; em Tx /em , treatment The survey showed that among participants who provided Hb levels ( em n /em ?=?114), most C5i-treated individuals reported Hb levels??12?g/dL (ECU, 87.5%, em n /em ?=?28/32; RAV, 82.9%, em n /em ?=?68/82) (Fig.?1b). More than half of the survey participants who provided Hb levels reported levels? ?10.5?g/dL despite treatment with ECU or RAV (ECU, 62.5%, em n /em ?=?20/32; RAV, 57.3%, em n /em ?=?47/82) (Fig.?1b). Figure?1c shows the most common and current PNH symptoms reported by at least 35% of the total survey respondents. These PNH symptoms include fatigue, breakthrough hemolysis, shortness of breath, headaches, difficulty focusing, sleeping difficulties, and back pain. The most common symptom reported was fatigue (ECU, 88.6%, em n /em ?=?31/35; RAV, 74.7%, em n /em ?=?65/87) (Fig.?1c). Further analysis demonstrated IL5R that individuals with Hb levels? ?10.5?g/dL reported significantly higher frequency of fatigue (86.6%, em n /em ?=?58/67, em p /em ?=?0.004) and breakthrough hemolysis (52.2%, em n /em ?=?35/67, em p /em ? ?0.001) as compared to patients with Hb levels??10.5?g/dL (fatigue: 63.8%, em n /em ?=?30/47; breakthrough hemolysis: 19.1%, Rusalatide acetate em n /em ?=?9/47). Quality of life analysis The mean FACIT-Fatigue scores recorded from the survey participants receiving ECU or RAV therapy were lower (ECU, 29.3??14.0; RAV, 33.3??13.0) compared to what has been reported for the general US population (43.6) (Fig.?2) [24]. Participants receiving ECU and RAV reported an average score of 62.4 (?21.1) and 67.2 (?19.0) for global health status on the EORTC QLQ-C30, respectively, compared to a general population score of 75.7 [23] (Fig.?2). The physical functioning scores were 76.4 (?17.5) for ECU users and 76.7 (?20.3) for RAV users, which were lower than the reported average of the general population at 91.0 (Fig.?2) [23]. Participants also reported scores lower than the population average for functioning related to role, emotional, cognitive, and social parameters of the EORTC QLQ-C30 (Online Resource 1 Figure). Open in a separate window Fig. 2 FACIT-Fatigue and EORTC QLQ-C30 scores. Mean FACIT-Fatigue score gathered from patients with?PNH receiving C5i therapy ( em N /em ?=?122) compared to FACIT-Fatigue score for the general US population [24]. Rusalatide acetate Mean EORTC-QLQ-C30 scores for global health status and physical functioning recorded from C5i-treated patients with?PNH ( em N /em ?=?122) compared to the EORTC QLQ-C30 scores representative of the general population [23]. em Abbreviations /em : em C5i /em , C5-inhibitor; em ECU /em , eculizumab; em EORTC QLQ-C30 /em , European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30; Rusalatide acetate em FACIT /em , Functional Assessment of Chronic Illness Therapy; em RAV /em , ravulizumab Healthcare resource utilization related to PNH Among those survey respondents treated with ECU or RAV who had visited the ER (ECU, em n /em ?=?19; RAV, em n /em ?=?37) or had been hospitalized (ECU, em n /em ?=?7; RAV, em n /em ?=?25), an average of 1.05 (?1.03) or 1.22 (?1.08) PNH-related ER visits and 0.57 (?0.53) or 1.36 (?1.22) hospitalizations, respectively, were reported (Table ?(Table2).2). Common reasons (experienced by at least 25% of total responders) for PNH-related ER visits and hospitalizations included fatigue, breakthrough hemolysis, abdominal pain, and shortness of breath. Survey data also revealed that nearly all patients with PNH-related ER visits due to fatigue were anemic (Hb levels??12?g/dL, em n /em ?=?16/17, 1 patient did not know their Hb level). Table 2 PNH-related healthcare resource utilization (HCRU) within the past 12?months, among patients who had reported all-cause HCRU thead th align=”left” rowspan=”1″ colspan=”1″ Number of PNH-related eventsa /th th align=”left” Rusalatide acetate rowspan=”1″ colspan=”1″ Total br / Mean (SD) /th th align=”left” rowspan=”1″ colspan=”1″ Eculizumab br / Mean (SD) /th th align=”left” rowspan=”1″ colspan=”1″ Ravulizumab br / Mean.