Session Type: ACR Poster Session A
Session Time: 9:00AM-11:00AM
Catastrophic antiphospholipid syndrome (CAPS), a rare disease, is characterized by the rapid onset of widespread thrombosis associated with multi-organ failure in patients meeting the serological criteria for antiphospholipid syndrome. To date, the diagnosis and treatment for CAPS has been based on expert consensus and case-series, not evidence-based guidelines developed with rigorous methodology. One of the main obstacles to guideline development in rare diseases is the sparsity and very low certainty (or quality) of the available evidence. As a prototypical rare disease, CAPS was selected to pilot the application of a guideline development process to a rare disease.
The RARE-BestPractices project group in partnership with McMaster University developed a clinical practice guideline on diagnosis and management of patients with CAPS, bringing together a panel of international experts, and using the GIN-McMaster Guideline Development checklist. The evidence for the CAPS guideline was summarized using the GRADE methodology framework. Two novel methods, systematic observation reporting forms and ad hoc registry data analysis, were implemented to supplement the available evidence.
Systematic observation reporting involved collecting standardized contributions from each panel member prior to the panel meeting regarding characteristics and clinical course of previous CAPS patients. The goal was to systematize the process of expert input based on actual experience rather than opinions or anecdotes. Ad hoc analysis of registry data involved calculating raw data for survival estimates for each therapy question from the ‘CAPS Registry’, an international database that has over 500 CAPS patients enrolled.
Question generation yielded 47 questions, which were prioritized. The top 10 questions were selected by the panel for the guideline process, 3 diagnosis and 7 therapy. The therapy search identified 671 references, of which 8 articles were included in the systematic reviews. The diagnosis search identified 519 references, of which 1 article was included. All of the included studies were considered to be at high risk of bias. Eleven of the 20 panel members submitted systematic observation forms, representing 55 patient cases. The CAPS registry was used to calculate raw mortality for 7 recommendations and impacted the panel’s decision for a treatment recommendation related to rituximab.
The CAPS guideline initiative met the objective of successful development of a clinical practice guideline in a rare disease using GRADE methodology. The novel methodology was found to be useful for complementing literature-based evidence in informing the recommendations.
To cite this abstract in AMA style:Legault K, Hillis C, Yeung C, Akl E, Carrier M, Cervera R, Crowther M, Dentali F, Erkan D, Espinosa G, Khamashta MA, Meerpohl J, Moffatt K, O'Brien S, Pengo V, Rand J, Rodriguez I, Thom L, Schunemann H, Iorio A. Methodology and Systematic Review of the Literature for the Mcmaster RARE-Best Practice Clinical Practice Guideline on Diagnosis and Management of the Catastrophic Antiphospholipid Syndrome [abstract]. Arthritis Rheumatol. 2017; 69 (suppl 10). https://acrabstracts.org/abstract/methodology-and-systematic-review-of-the-literature-for-the-mcmaster-rare-best-practice-clinical-practice-guideline-on-diagnosis-and-management-of-the-catastrophic-antiphospholipid-syndrome/. Accessed September 24, 2021.
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