Palifermin reduces patient-reported mouth and throat soreness and improves patient functioning in the hematopoietic stem-cell transplantation setting
Palifermin reduces patient-reported mouth and throat soreness and improves patient functioning in the hematopoietic stem-cell transplantation setting
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DOI：
10.1200/jco.2005.02.8340
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发表时间：
2006-11-20
影响因子：
45.3
通讯作者：
Spielberger, Ricardo
中科院分区：
文献类型：
--
作者：
Stiff, Patrick J.;Emmanouilides, Christos;Spielberger, Ricardo
目的描述患者报告的口腔和咽喉疼痛（MTS）和相关后遗症对日常活动的影响，这些结果来自一项在自体造血干细胞移植（HSCT）环境中进行的帕利佛明III期研究，并使用客观量表比较患者自我评估与临床医生对口腔粘膜炎的评估。在全身照射（12戈伊）前3天、依托泊苷60 mg/kg和环磷酰胺100 mg/kg以及HSCT后3天，给药60 μ g/kg/d或安慰剂。患者完成每日问卷（口腔粘膜炎每日问卷[OMDQ]），评价MTS严重程度及其对日常功能活动的影响。患者的自我评估数据进行了比较与临床医生的口腔粘膜炎的评估使用客观scales.Results帕利弗明降低了严重的口腔粘膜炎的发病率和持续时间，由临床医生和患者评估。患者和临床医生评估之间的比较表明，粘膜炎等级和主观（MTS）工具之间的平均每日评分相似，尽管患者报告的MTS发作、峰值和消退早于临床医生评估（1至3天）。接受帕利佛明治疗的患者报告日常功能活动（吞咽、饮水、进食、说话、睡眠）有统计学显著改善（P < .001），需要的麻醉性阿片类药物显著减少（P < .001）;还报告了患者整体身体和功能健康的改善。这是证实了癌症治疗questionnaire.Conclusion功能评估的结果支持帕利弗明在HSCT设置的临床效益，提供证据表明，患者的自我评估工具（OMDQ）可以作为一种替代工具，以评估口腔粘膜炎的严重程度在临床试验。
Purpose To describe patient-reported outcomes of mouth and throat soreness (MTS) and related sequelae on daily activities from a phase III study of palifermin in the autologous hematopoietic stem-cell transplantation (HSCT) setting and to compare patient self-evaluations with clinicians' assessments of oral mucositis using objective scales.Patients and Methods Patients (n = 212) received palifermin (60 mu g/kg/d) or placebo for 3 days before total-body irradiation (12 Gy), etoposide 60 mg/kg, and cyclophosphamide 100 mg/kg, and 3 days after HSCT. Patients completed a daily questionnaire (Oral Mucositis Daily Questionnaire [OMDQ]) evaluating MTS severity and its effects on daily functional activities. Patients' self-assessment data were compared with clinicians' assessments of oral mucositis using the objective scales.Results Palifermin reduced the incidence and duration of severe oral mucositis, as assessed by both clinicians and patients. Comparisons between patient and clinician assessments demonstrated that the average daily scores between mucositis grade and subjective (MTS) instruments were similar, although patients reported MTS onset, peak and resolution earlier (1 to 3 days) than, clinicians' assessments. Patients receiving palifermin reported statistically significant improvements (P < .001) in daily functioning activities (swallowing, drinking, eating, talking, sleeping) and required significantly less narcotic opioids (P < .001); improvement in the patient's overall physical and functional well-being was also reported. This was confirmed by the results of the Functional Assessment of Cancer Treatment questionnaire.Conclusion These results support the clinical benefit of palifermin in the HSCT setting, providing evidence that a patient's self-assessment instrument (OMDQ) may serve as an alternative tool to assess oral mucositis severity in clinical trials.