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

Kimberly Legault¹, Christopher Hillis¹, Cindy Yeung¹, Elie Akl², Marc Carrier³, Ricard Cervera⁴, Mark Crowther¹, Francesco Dentali⁵, Doruk Erkan⁶, Gerard Espinosa⁷, Munther A Khamashta⁸, Joerg Meerpohl⁹, Karen Moffatt¹⁰, Sarah O'Brien¹¹, Vittorio Pengo¹², Jacob Rand¹³, Ignasi Rodriguez¹⁴, Lisa Thom¹⁵, Holger Schunemann¹ and Alfonso Iorio¹, ¹McMaster University, Hamilton, ON, Canada, ²American University of Beirut, Beirut, Lebanon, ³University of Ottawa, Ottawa, ON, Canada, ⁴Department of Autoimmune Diseases, Institut Clínic de Medicina i Dermatologia, Hospital Clínic de Barcelona, Barcelona, Spain, ⁵Insubria University, Insubria, Italy, ⁶Rheumatology, Hospital for Special Surgery- Weill Cornell Medicine, New York, NY, ⁷Autoimmune Diseases Department. Hospital Clínic de Barcelona, Barcelona, Spain, ⁸Graham Hughes Lupus Research Laboratory, The Rayne Institute, London, United Kingdom, ⁹University of Freiburg, Freiburg, Germany, ¹⁰Hamilton Health Sciences, Hamilton, ON, Canada, ¹¹Nationwide Children's, Columbus, OH, ¹²Azienda Ospedaliera of Padova, University of Padova, Padova, Italy, ¹³NY Presbyterian Hospital, New York, NY, ¹⁴Rheumatology, Hospital Clinica, Barcelona, Spain, ¹⁵None (patient representative), Oxford, United Kingdom

Meeting: 2017 ACR/ARHP Annual Meeting

Date of first publication: September 18, 2017

Keywords: Antiphospholipid antibodies, antiphospholipid syndrome, catastrophic antiphospholipid syndrome and clinical practice guidelines

Session Information

Date: Sunday, November 5, 2017

Title: Antiphospholipid Syndrome Poster

Session Type: ACR Poster Session A

Session Time: 9:00AM-11:00AM

Background/Purpose:

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.

Methods:

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.

Results:

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.

Conclusion:

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.

Disclosure: K. Legault, Bayer, 9; C. Hillis, None; C. Yeung, None; E. Akl, None; M. Carrier, Leo Pharma, 5,Bayer, 5,Pfizer Inc, 5,Leo Pharma, 9,BMS, 9; R. Cervera, None; M. Crowther, Bayer, 2; F. Dentali, None; D. Erkan, Alexion Pharmaceuticals, Inc., 5; G. Espinosa, None; M. A. Khamashta, None; J. Meerpohl, None; K. Moffatt, None; S. O'Brien, None; V. Pengo, None; J. Rand, None; I. Rodriguez, None; L. Thom, None; H. Schunemann, None; A. Iorio, None.

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 .

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