https://scholars.lib.ntu.edu.tw/handle/123456789/635469
標題: | EXPLORE B: A prospective, long-term natural history study of patients with acute hepatic porphyria with chronic symptoms | 作者: | Cassiman, David Kauppinen, Raili Monroy, Susana MING-JEN LEE Bonkovsky, Herbert L Thapar, Manish Guillén-Navarro, Encarna Minder, Anna-Elisabeth Hale, Cecilia Sweetser, Marianne T Ivanova, Aneta |
關鍵字: | chronic symptoms; disease burden; hepatic complications; porphyria attack; porphyrias; prospective studies | 公開日期: | 十一月-2022 | 卷: | 45 | 期: | 6 | 起(迄)頁: | 1163 | 來源出版物: | Journal of inherited metabolic disease | 摘要: | One-year data from EXPLORE Part A showed high disease burden and impaired quality of life (QOL) in patients with acute hepatic porphyria (AHP) with recurrent attacks. We report baseline data of patients who enrolled in EXPLORE Part B for up to an additional 3 years of follow-up. EXPLORE B is a long-term, prospective study evaluating disease activity, pain intensity, and QOL in patients with AHP with ≥1 attack in the 12 months before enrollment or receiving hemin or gonadotropin-releasing hormone prophylaxis. Data were evaluated in patients with more (≥3 attacks or on prophylaxis treatment) or fewer (<3 attacks and no prophylaxis treatment) attacks. Patients in the total population (N = 136), and more (n = 110) and fewer (n = 26) attack subgroups, reported a median (range) of 3 (0-52), 4 (0-52), and 1 (0-2) acute attacks, respectively, in the 12 months prior to the baseline visit. Pain, mood/sleep, digestive/bladder, and nervous system symptoms were each experienced by ≥80% of patients; most received hemin during attacks. Almost three-quarters of patients reported chronic symptoms between attacks, including 85% of patients with fewer attacks. Pain intensity was comparable among both attack subgroups; most patients required pain medication. All groups had diminished QOL on the EuroQol visual analog scale and the European Organisation for Research and Treatment of Cancer Quality-of-life Questionnaire Core 30 versus population norms. Patients with AHP with recurrent attacks, even those having fewer attacks, experience a high disease burden, as evidenced by chronic symptoms between attacks and impaired QOL. |
URI: | https://scholars.lib.ntu.edu.tw/handle/123456789/635469 | ISSN: | 01418955 | DOI: | 10.1002/jimd.12551 |
顯示於: | 醫學院附設醫院 (臺大醫院) |
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