Ueda Yasutaka, Obara Naoshi, Ueno Shikiko, Sakurai Masatoshi, Hosokawa Kohei, Murata Tatsunori, Fukuoka Yuta, Hayama Nozomi, Yamashita Masami, Ogawa Yoshiaki, Usuki Kensuke, Ikezoe Takayuki, Kawaguchi Tatsuya, Hosen Naoki, Kanakura Yuzuru, Gotoh Akihiko, Nishimura Jun-Ichi
Department of Hematology and Oncology, The University of Osaka Graduate School of Medicine, Osaka, Japan.
Department of Hematology, University of Tsukuba Hospital, Ibaraki, Japan.
Ann Hematol. 2025 Jul;104(7):3575-3584. doi: 10.1007/s00277-025-06486-9. Epub 2025 Jul 18.
Paroxysmal nocturnal hemoglobinuria (PNH) is a lifelong, clonal hematologic disease posing life-threatening risks if untreated. The prognosis for PNH has improved with the advent of C5 inhibitors, which are now the standard of care where available. As treatment options continue to expand, healthcare providers can better address both PNH management and the impact of treatment on patients' daily lives. To investigate the burden of disease and key factors taken into consideration when patients with PNH choose their preferred treatment, we conducted an in-depth patient interview survey. Of survey participants (N = 30), 70.0% were receiving intravenous C5 inhibitors. Notably, 56.7% of all patients reported needing ≥ 1 hospital visit per month, and 46.7% required a day off from work or school for visits (33.3% among those receiving intravenous C5 inhibitors). A frequently reported burden of PNH treatment was financial concern, including the cost of treatment, hospital visits, and the negative impact on income. Additionally, burden related to waiting times and distance from the hospital and the overall time spent on outpatient PNH care were identified with similar results for patients receiving intravenous C5 inhibitors. These results suggest that the time and effort to get to treatment centers and the time required to receive treatment for PNH were critical unmet needs in PNH care. Our study indicates that persistent burdens associated with current PNH care should be taken into account alongside therapeutic effectiveness when making treatment decisions. Further analysis with a larger sample size is required to confirm these findings.
阵发性睡眠性血红蛋白尿（PNH）是一种终身性的克隆性血液疾病，若不治疗会带来危及生命的风险。随着C5抑制剂的出现，PNH的预后有所改善，在有条件的情况下，C5抑制剂现已成为标准治疗方法。随着治疗选择的不断扩展，医疗服务提供者能够更好地应对PNH的管理以及治疗对患者日常生活的影响。为了调查疾病负担以及PNH患者选择其首选治疗方法时考虑的关键因素，我们开展了一项深入的患者访谈调查。在调查参与者（N = 30）中，70.0%正在接受静脉注射C5抑制剂治疗。值得注意的是，所有患者中有56.7%报告每月需要≥1次医院就诊，46.7%的患者因就诊需要请假（接受静脉注射C5抑制剂治疗的患者中这一比例为33.3%）。PNH治疗中经常被报告的负担是经济方面的担忧，包括治疗费用、医院就诊费用以及对收入的负面影响。此外，对于接受静脉注射C5抑制剂治疗的患者，与等待时间、距离医院的远近以及门诊PNH护理所花费的总时间相关的负担也得到了类似的结果。这些结果表明，前往治疗中心所需的时间和精力以及接受PNH治疗所需的时间是PNH护理中尚未得到满足的关键需求。我们的研究表明，在做出治疗决策时，除了治疗效果外，还应考虑当前PNH护理持续存在的负担。需要更大样本量的进一步分析来证实这些发现。
