{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,26]],"date-time":"2026-06-26T10:50:13Z","timestamp":1782471013904,"version":"3.54.5"},"reference-count":32,"publisher":"Springer Science and Business Media LLC","issue":"6","license":[{"start":{"date-parts":[[2026,2,11]],"date-time":"2026-02-11T00:00:00Z","timestamp":1770768000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2026,2,11]],"date-time":"2026-02-11T00:00:00Z","timestamp":1770768000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Int J Hematol"],"published-print":{"date-parts":[[2026,6]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>This study aimed to determine the clinical profile and disease burden of patients with paroxysmal nocturnal hemoglobinuria (PNH) in Japan using real-world data from the Adelphi Real World PNH Disease Specific Programme (DSP)\u2122, a cross-sectional survey conducted between January and December 2022. Data included demographics, treatment, and clinical values (hemoglobin [Hb] and lactate dehydrogenase [LDH]). Patient-reported outcome measures included the EQ-5D-5L and the FACIT-Fatigue. Seventeen physicians provided information on 45 patients, among whom 86.7% were receiving treatment with complement 5 inhibitors (C5i). Median (IQR) age was 65.0 (54.5, 73.0) years; 55.6% were male. Among C5i-treated patients (n\u2009=\u200939), 51.3% received ravulizumab and 48.7% eculizumab, for a median (IQR) duration of 1.6 (1.0, 2.7) years. Median (IQR) Hb level was 7.2 (7.0, 8.1) g\/dL at diagnosis and 10.0 (8.8, 10.6) g\/dL at survey; 91.4% had LDH levels exceeding 1.5 times the upper limit of normal at diagnosis, 11.4% at survey. Twelve patients returned a self-completed questionnaire. Patient-reported symptoms included tiredness (83.3%), shortness of breath (75.0%), and anemia (58.3%). Mean (SD) EQ-5D-5L and FACIT-Fatigue scores were 0.73 (0.16) and 32.3 (7.1). Even C5i-treated patients continued to experience substantial disease burden, highlighting the need for more effective treatments to improve quality of life.<\/jats:p>","DOI":"10.1007\/s12185-026-04170-w","type":"journal-article","created":{"date-parts":[[2026,2,11]],"date-time":"2026-02-11T03:11:54Z","timestamp":1770779514000},"page":"837-847","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Disease burden and treatment patterns of paroxysmal nocturnal hemoglobinuria in Japan: a real-world survey"],"prefix":"10.1007","volume":"123","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6687-4136","authenticated-orcid":false,"given":"Masatoshi","family":"Sakurai","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yasutaka","family":"Ueda","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Naoshi","family":"Obara","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michel","family":"Kroes","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Takeo","family":"Dochi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anggie","family":"Wiyani","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria-Magdalena","family":"Balp","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olivier","family":"Somenzi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yasmin","family":"Taylor","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tatsuya","family":"Kawaguchi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jun-ichi","family":"Nishimura","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2026,2,11]]},"reference":[{"issue":"18","key":"4170_CR1","doi-asserted-by":"publisher","first-page":"2804","DOI":"10.1182\/blood-2014-02-522128","volume":"124","author":"RA Brodsky","year":"2014","unstructured":"Brodsky RA. 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YU has received honoraria from Alexion, Chugai, Incyte, Kaken, Nippon Shinyaku, Novartis, Sanofi, and Sobi; consultancy fees from Alexion, Asahi Kasei, Chugai, Janssen, Johnson and Johnson, Novartis, Ono, Sanofi and Sobi; research funding from Chugai; and serves as an editor for the International Journal of Hematology. NO has received personal fees and research grants from Novartis, Kyowa Kirin, Chugai, Roche, and Alexion. MK is an employee of Novartis Pharma AG TD is an employee of Novartis Pharma Tokyo. AW is an employee of Novartis Pharmaceuticals UK Ltd. MMB is an employee of Novartis Pharma AG. OS is an employee of Novartis Pharma AG. YT is an employee of Adelphi Real World. TK has received fees from Alexion and Chugai. JN has received fees from Alexion, Chugai, Roche, Novartis, Asahi Kasei Pharma, and Sobi.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}}]}}