Validation of the Children's International Mucositis Evaluation Scale (ChIMES)

Sung， Lillian Preamble：口腔黏膜炎是接受化疗和干细胞移植（SCT）的儿童癌症治疗中最令人痛苦的副作用之一。为了确定最有效的预防和治疗策略，我们需要可行的、心理测量学上健全的和临床有用的口腔黏膜炎结果测量。我们的团队已经制定了一项研究计划，以开发儿童口腔粘膜炎的特定测量方法，在这项拨款申请中，我们计划通过评估儿童国际粘膜炎评估量表（ChIMES）的心理测量特性来扩展我们之前的研究。a)通过证明在第13天和第14天检查总ChIMES评分时，对于报告粘膜炎没有变化的患者，类内相关系数（ICC）为0.7来评估重测信度；b)通过证明在第14天父母和12岁儿童之间比较总ChIMES得分时的ICC e为0.5来评估评估者间的信度；c)通过期望Cronbach's alpha e0.7来评估内部一致性。a)通过预测SCT的总ChIMES评分高于化疗来评估判别效度；b)通过预测总ChIMES评分与其他粘膜炎指标，即视觉模拟量表（VAS）、美国国家癌症研究所共同毒性标准（NCI-CTC）、世界卫生组织量表（WHO）和口腔粘膜炎每日问题（OMDQ）的中度相关来评估收敛效度，预测Spearman相关系数为0.35；c)通过证明在第14天报告口腔黏膜炎的儿童从基线到第14天的总ChIMES评分显着增加来评估反应性。方法：这是一项多中心前瞻性研究，将在美国、加拿大和英国的5个中心招募患者。我们将包括0 - 18岁的儿童，他们接受与粘膜炎高风险相关的化疗，以及接受任何SCT的儿童。我们将在化疗开始或SCT调理后的第7至17天，在基线和每天使用ChIMES测量粘膜炎。其他评估粘膜炎的方法包括VAS、NCI-CTC、WHO和OMDQ。对于年龄较大的儿童（12岁及以上），儿童将作为答辩人，而对于年龄较小的儿童（12岁以下），父母将作为答辩人。我们计划在2年内招募200名受试者，这将足以满足研究目标。意义：口腔黏膜炎是化疗最严重的副作用之一。为了评估干预措施是否具有有意义的影响并促进临床护理，需要可行的、心理测量学上合理的和临床有用的结果测量。这个研究项目，为口腔黏膜炎开发这样一个结果测量，对于改善接受化疗和SCT治疗的癌症儿童的生活质量至关重要。

DESCRIPTION (provided by applicant): Validation of the Children's International Mucositis Evaluation Scale (ChIMES) Sung, Lillian Preamble: Oral mucositis is one of the most distressing side effects of treatment for children with cancer receiving chemotherapy and stem cell transplantation (SCT). In order to determine the most effective prevention and treatment strategies, we need feasible, psychometrically sound and clinically useful outcome measures of oral mucositis. Our group has developed a program of research in developing a pediatric specific oral mucositis measure and in this grant proposal, we plan to extend our previous research by evaluating the psychometric properties of the Children's International Mucositis Evaluation Scale (ChIMES). Specific Aims: Aim 1 a) To evaluate test-retest reliability by demonstrating an intraclass correlation coefficient (ICC) e 0.7 when the total ChIMES scores are examined on days 13 and 14 for those who report no change in mucositis; b) To evaluate inter-rater reliability by demonstrating an ICC e 0.5 when the total ChIMES scores are compared between parents and children e 12 years of age on day 14; and c) To evaluate internal consistency by expecting Cronbach's alpha e 0.7. Aim 2 a) To evaluate discriminant validity by anticipating total ChIMES scores will be higher with SCT compared to chemotherapy; b) To evaluate convergent validity by anticipating total ChIMES score will be moderately correlated with other measures of mucositis namely visual analogue scale (VAS), National Cancer Institute Common Toxicity Criteria (NCI-CTC), World Health Organization scale (WHO) and Oral Mucositis Daily Question (OMDQ) by anticipating a Spearman correlation e 0.35; and c) To evaluate responsiveness by demonstrating a significant increase in total ChIMES score from baseline to day 14 in children who report oral mucositis on day 14. Methods: This is a multi-center prospective study that will enroll patients in 5 centers in the United States, Canada and the United Kingdom. We will include children 0 to 18 years of age receiving chemotherapy associated with a higher risk of mucositis and those receiving any SCT. We will measure mucositis using ChIMES at baseline and daily from days 7 to 17 following initiation of chemotherapy or conditioning for SCT. Other measures of mucositis will be VAS, NCI-CTC, WHO and OMDQ. For older children (12 years and older), the children will be the respondent while for younger children (less than 12 years), the parent will be the respondent. We intend to enroll 200 subjects over 2 years, which will be sufficient to meet the study aims. Significance: Oral mucositis is one of the most debilitating side effects of chemotherapy. In order to assess whether interventions have a meaningful impact and to facilitate clinical care, feasible, psychometrically sound and clinically useful outcome measures are required. This program of research, to develop such an outcome measure for oral mucositis, is critically important to improve quality of life during treatment for children with cancer who receive chemotherapy and SCT. PUBLIC HEALTH RELEVANCE: Validation of the Children's International Mucositis Evaluation Scale (ChIMES) Sung, Lillian Mouth sores are one of the most distressing and painful side effects of chemotherapy and stem cell transplantation. In order to study treatments to prevent or treat them, we need good instruments to measure mucositis suitable for children and thus, we have developed the Children's International Mucositis Evaluation Scale (ChIMES). This grant proposal is designed to determine whether this instrument is good at measuring the effects of mucositis and thus, our study is critically important in order to study ways to reduce or prevent mouth sores and improve quality of life for children with cancer.