Kim Ji-Won, Kim Myeong Gyu, Lee Hyun Jung, Koh Youngil, Kwon Ji-Hyun, Kim Inho, Park Seonyang, Kim Byoung Kook, Oh Jung Mi, Kim Kyung Im, Yoon Sung-Soo
Department of Internal Medicine, Seoul National University Bundang Hospital, Seongnam, Republic of Korea.
College of Pharmacy, Seoul National University, Seoul, Republic of Korea.
PLoS One. 2017 Jan 3;12(1):e0168854. doi: 10.1371/journal.pone.0168854. eCollection 2017.
The aim of this study was to evaluate the efficacy and safety of recombinant human epidermal growth factor (rhEGF) oral spray for oral mucositis (OM) induced by intensive chemotherapy with hematopoietic stem cell transplantation. In this phase 2 study, patients were randomized to either rhEGF (50 microg/mL) or placebo in a 1:1 ratio. The primary endpoint was incidence of National Cancer Institute (NCI) grade ≥2 OM. A total of 138 patients were enrolled in this study. In the intention-to-treat analysis, rhEGF did not reduce the incidence of NCI grade ≥2 OM (p = 0.717) nor reduce its duration (p = 0.725). Secondary endpoints including the day of onset and duration of NCI grade ≥2 OM, the incidence of NCI grade ≥3 OM and its duration, and patient-reported quality of life were also similar between the two groups. In the per-protocol analysis, however, the duration of opioid analgesic use was shorter in the rhEGF group (p = 0.036), and recipients in the rhEGF group required a lower cumulative dose of opioid analgesics than those in the placebo group (p = 0.046), among patients with NCI grade ≥2 OM. Adverse events were mild and transient. This study found no evidence to suggest that rhEGF oral spray reduces the incidence of OM. However, further studies are needed to investigate the effect of rhEGF on OM-induced pain reduction after intensive chemotherapy.
本研究旨在评估重组人表皮生长因子（rhEGF）口腔喷雾剂对造血干细胞移植强化化疗所致口腔黏膜炎（OM）的疗效和安全性。在这项2期研究中，患者按1:1比例随机分为rhEGF（50微克/毫升）组或安慰剂组。主要终点为美国国立癌症研究所（NCI）≥2级OM的发生率。共有138例患者纳入本研究。在意向性分析中，rhEGF未降低NCI≥2级OM的发生率（p = 0.717），也未缩短其持续时间（p = 0.725）。两组间的次要终点，包括NCI≥2级OM的发病日和持续时间、NCI≥3级OM的发生率及其持续时间，以及患者报告的生活质量也相似。然而，在符合方案分析中，在NCI≥2级OM患者中，rhEGF组的阿片类镇痛药使用时间较短（p = 0.036），且rhEGF组患者所需的阿片类镇痛药累积剂量低于安慰剂组（p = 0.046）。不良事件轻微且短暂。本研究未发现证据表明rhEGF口腔喷雾剂可降低OM的发生率。然而，需要进一步研究来探讨rhEGF对强化化疗后OM所致疼痛减轻的影响。
