Guidelines for Steroids in Children with Lupus

狼疮儿童类固醇指南

• 批准号: 6604503
• 负责人: Hermine I Brunner
• 金额: $ 7.4万
• 依托单位: CINCINNATI CHILDRENS HOSP MED CTR
• 依托单位国家: 美国
• 财政年份: 2003
• 资助国家: 美国
• 起止时间: 2003-05-08 至 2006-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6604503
• 关键词: artificial immunosuppression; children; clinical research; connective tissue disorder chemotherapy; connective tissue pharmacology; decision making; dosage; drug adverse effect; drug screening /evaluation; health care cost /financing; human subject; human therapy evaluation; longitudinal human study; outcomes research; patient care planning; patient oriented research; pharmacokinetics; quality of life; questionnaires; statistics /biometry; steroids; systemic lupus erythematosus

DESCRIPTION (provided by applicant): BACKGROUND: This is a pilot study to evaluate the use of steroids and other medications in children diagnosed with SLE (cSLE). After having revolutionized the prognosis of lupus in the 1950s, steroids remain the mainstay of therapy of cSLE. Recent studies suggest that, despite their proven benefits, steroids contribute to the development of permanent disease damage in both adult and pediatric SLE patients. 10-year patient survival is only at 85%. Preliminary data support that there is a considerable degree of practice variation among pediatric rheumatologists treating cSLE and that these differences in treatment approach may have an impact on patient outcomes. There are no published guidelines of how to best treat cSLE, especially how to use steroids for its the treatment and when to introduce other steroid-sparing medications. GOALS: 1) To document treatment patterns of pediatric rheumatologists for patients with cSLE in order a) To identify key factor that prompt physicians to choose a certain steroid dose and document the factors that make physicians change a given dose of steroids; b) To identify the key variables that prompt physicians to introduce of immunosuppressive therapies for patients diagnosed with cSLE. 2) To measure quality of life and specific outcomes (damage, costs) associated with the treatment of children and adolescents diagnosed with cSLE. STUDY DESIGN: A cohort of consecutively sampled patients treated for cSLE (n=70) at 4 pediatric US and Canadian Rheumatology Centers (Chicago, Cincinnati, Minneapolis, Toronto) will be assessed in at least tri-monthly intervals over an 18-month period regarding their disease course (disease activity, number of flares, infection and hospitalization), treatments, and outcomes (damage, quality of life). Key determinants that prompt the physicians to use certain therapies will be recorded. Relevant retrospective patient information will be obtained by chart review. Patient quality of life and treatment costs will be measured. Correlation, regression, multivariable modeling including repeated measure analysis will be used to analyze the relationship of cSLE therapies to outcomes (damage, quality of life, costs) and to key determinants of treatment decisions in cSLE. SIGNIFICANCE: The proposed pilot study will provide information regarding physician treatment patterns, cost of cSLE and patient quality of life. Data will be collected to support that there are important differences in the approach to cSLE therapy that have a significant impact on patient outcomes. Results of the study will be used to generate hypotheses towards improved treatment approaches for cSLE. The proposed study constitutes a first step towards the development of evidence-based guidelines. Information on HRQL and costs of patients diagnosed with cSLE is required for future cost-effectiveness analyses of treatments for cSLE.

描述（由申请人提供）：背景：这是一项试点研究，用于评估被诊断为SLE（CSLE）的儿童中类固醇和其他药物的使用。在1950年代彻底改变了狼疮的预后后，类固醇仍然是CSLE治疗的支柱。最近的研究表明，尽管具有证明的好处，但类固醇有助于成人和小儿SLE患者的永久性疾病损害的发展。 10年患者的生存仅为85％。初步数据支持的是，小儿风湿病学家治疗CSLE的实践差异很大，并且治疗方法上的这些差异可能会影响患者的结果。没有关于如何最好地治疗CSLE的公开准则，尤其是如何使用类固醇进行治疗以及何时引入其他药物。 目标：1）记录CSLE患者儿科风湿病学家的治疗模式 a）确定促使医生选择某种类固醇剂量的关键因素，并记录导致的因素 医生会改变给定的类固醇。 b）确定促使医生为被诊断为CSLE的患者引入免疫抑制疗法的关键变量。 2）衡量与诊断为CSLE的儿童和青少年的治疗相关的生活质量和特定结果（损害，成本）。 研究设计：在美国和加拿大4个儿科和加拿大风湿病中心（芝加哥，辛辛那提，明尼阿波利斯，多伦多）在4个儿科和加拿大风湿中心进行CSLE（n = 70）的一组群体，将在至少每一个月度的疾病病程中评估（至少在三个月内的疾病疗法）（近期疾病）的疾病及其数量，数量及其疾病活动，数量，且数量的疾病，数量，数量，数量，数量，数量，数量，并将评估。 （损害，生活质量）。促使医生使用某些疗法的关键决定因素将记录下来。相关的回顾性患者信息将通过图表审查获得。将测量患者的生活质量和治疗费用。相关性，回归，多变量建模（包括重复度量分析）将用于分析CSLE疗法与结果（损害，生活质量，成本）以及CSLE治疗决策的关键决定因素的关系。 意义：拟议的试点研究将提供有关医师治疗模式，CSLE成本和患者生活质量的信息。将收集数据以支持CSLE治疗方法对患者预后产生重大影响。该研究的结果将用于产生假设，以改善CSLE的治疗方法。拟议的研究构成了朝着基于证据的指南制定的第一步。对于CSLE治疗的将来的成本效益分析，需要有关HRQL的信息和被诊断为CSLE的患者的成本。