Research priorities in childhood-onset lupus: results of a multidisciplinary prioritization exercise
Research priorities in childhood-onset lupus: results of a multidisciplinary prioritization exercise
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DOI：
10.1186/s12969-019-0327-4
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发表时间：
2019-07-01
影响因子：
2.5
通讯作者：
Hersh, Aimee O.
中科院分区：
文献类型：
--
作者：
Ardoin, Stacy P.;Daly, R. Paola;Hersh, Aimee O.
研究背景儿童期发病的系统性红斑狼疮（systemic erythematosus lupus，cSLE）与成人期发病的SLE相比，具有病情重、器官受累广、病死率高的特点。然而，cSLE在很大程度上资金不足，无法进行必要的研究以推进该领域的发展。很少有常用的SLE药物在儿童中进行了研究，并且存在关于流行病学、遗传学、病理生理学和cSLE.MethodsIn最佳治疗的重要知识缺口，为了评估最高的cSLE研究优先领域，美国狼疮基金会（LFA）和儿童关节炎和风湿病研究联盟（CARRA）向儿科风湿病学家进行了cSLE研究优先调查，皮肤科医生和肾脏科医生的专家在狼疮。LFA和CARRA的SLE委员会的成员确定了一个cSLE研究领域的列表，并开发了一个17项分层，基于网络的调查，要求受访者将研究领域分为高，中，低优先领域。对于被确定为高优先级的领域，受访者将研究主题列为该类别。例如，对于肾炎的域，受访者排名的重要性：流行病学，生物标志物，长期的结果，质量改进，等。该调查被分配到CARRA，中西部儿科肾病联盟（MWPNC）和儿科皮肤病研究联盟（佩德拉）结缔组织疾病group.ResultsThe的整体响应率为256/752（34%）的成员。最优先的研究领域是：肾炎、临床试验、生物标志物、神经精神疾病和难治性皮肤病。值得注意的是，肾炎，临床试验和生物标志物在所有组中排名前五。在每个研究领域内，所有组都表现出一致的重要重点领域：确定最佳治疗方法，生物标志物/病理生理学，药物发现/新的治疗方法，了解长期的结果，并细化供应商报告的质量measures.ConclusionThis调查确定了最高的cSLE研究优先级领先的风湿病学，皮肤病学和肾脏病学的临床医生和研究人员从事护理儿童狼疮。迫切需要推进多学科合作，参与调查的利益攸关方认为这一点非常重要。这些调查结果应作为指导cSLE资金和具体研究计划的路线图，以解决这一人群中迫切的，未满足的需求。
BackgroundChildhood-onset systemic erythematosus lupus (cSLE) is characterized by more severe disease, widespread organ involvement and higher mortality compared to adult-onset SLE. However, cSLE is largely underfunded to carry out necessary research to advance the field. Few commonly used SLE medications have been studied in children, and important knowledge gaps exist concerning epidemiology, genetics, pathophysiology and optimal treatments for cSLE.MethodsIn order to assess highest cSLE research priority areas, the Lupus Foundation of America (LFA) and Childhood Arthritis and Rheumatology Research Alliance (CARRA) administered a cSLE research prioritization survey to pediatric rheumatologists, dermatologists and nephrologists with expertise in lupus. Members of LFA and CARRA's SLE Committee identified a list of cSLE research domains and developed a 17-item tiered, web-based survey asking respondents to categorize the research domains into high, medium, or low priority areas. For domains identified as high priority, respondents ranked research topics within that category. For example, for the domain of nephritis, respondents ranked importance of: epidemiology, biomarkers, long-term outcomes, quality improvement, etc. The survey was distributed to members of CARRA, Midwestern Pediatric Nephrology Consortium (MWPNC) and Pediatric Dermatology Research Alliance (PeDRA) Connective Tissue Disease group.ResultsThe overall response rate was 256/752 (34%). The highest prioritized research domains were: nephritis, clinical trials, biomarkers, neuropsychiatric disease and refractory skin disease. Notably, nephritis, clinical trials and biomarkers were ranked in the top five by all groups. Within each research domain, all groups showed agreement in identifying the following as important focus areas: determining best treatments, biomarkers/pathophysiology, drug discovery/novel treatments, understanding long term outcomes, and refining provider reported quality measures.ConclusionThis survey identified the highest cSLE research priorities among leading rheumatology, dermatology and nephrology clinicians and investigators engaged in care of children with lupus. There is a strong need for multidisciplinary collaboration moving forward, which was indicated as highly important among stakeholders involved in the survey. These survey results should be used as a roadmap to guide funding and specific research programs in cSLE to address urgent, unmet needs among this population.