Antiphospholipid Antibody-related Clinical Manifestations Presenting During Childhood versus Adulthood: Descriptive Results from the AntiPhospholipid Syndrome Alliance for Clinical Trials and InternatiOnal Networking (APS ACTION) Clinical Database and Repository (“Registry”)

Jheel Pandya¹, Danieli Andrade², Ann E. Clarke³, Maria Tektonidou⁴, Vittorio Pengo⁵, Massimo Radin⁶, Jose Pardos-Gea⁷, Nina Kello⁸, Diana Paredes-Ruiz⁹, Mª Angeles Aguirre-Zamorano¹⁰, H Michael Belmont¹¹, Paul Fortin¹², Flavio Victor Signorelli¹³, TATSUYA ATSUMI¹⁴, Zhuoli Zhang¹⁵, Maria Efthymiou¹⁶, David Branch¹⁷, Giulia Pazzola¹⁸, Angela Tincani¹⁹, Ali Duarte-Garcia²⁰, Esther Rodriguez-Almaraz²¹, Michelle Petri²², Ricard Cervera²³, Bahar Artim Esen²⁴, Guillermo Pons-Estel²⁵, Hui Shi²⁶, Yu Zuo²⁷, Rohan Willis²⁸, Pierluigi Meroni²⁹, Robert Roubey³⁰, Maria Laura Bertolaccini³¹, Hannah Cohen³² and Doruk Erkan¹, and on behalf of APS ACTION, ¹Hospital for Special Surgery, New York, NY, ²University of São Paulo, São Paulo, SP, Brazil, ³Division of Rheumatology, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada, ⁴National and Kapodistrian University of Athens, Athens, Greece, ⁵Thrombosis Research Laboratory, Department of Cardio-Thoracic-Vascular Sciences and Public Health, University of Padova, Padova, Italy, ⁶University of Turin, Turin, Italy, ⁷Vall d'Hebron University Hospital, Barcelona, Spain, ⁸Northwell Health, Brooklyn, NY, ⁹Autoimmune Diseases Research Unit. Biocruces Bizkaia Health Research Institute, Baracaldo, Spain, ¹⁰IMIBIC/Reina Sofia Hospital/University of Cordoba, CÓRDOBA, Andalucia, Spain, ¹¹NYU School of Medicine, New York, NY, ¹²Centre ARThrite - CHU de Québec - Université Laval, Quebec, QC, Canada, ¹³Universidade do Estado do Rio de Janeiro, Rio De Janeiro, Brazil, ¹⁴Department of Rheumatology, Endocrinology and Nephrology, Faculty of Medicine and Graduate School of Medicine, Hokkaido University, Sapporo, Japan, ¹⁵Peking University First Hospital, Beijing, China, ¹⁶University College London, London, United Kingdom, ¹⁷University of Utah and Intermountain Healthcare, Salt Lake City, UT, ¹⁸Rheumatology Unit, Azienda USL IRCCS di Reggio Emilia, Reggio Emilia, Italy, ¹⁹ASST Spedali Civili-University of Brescia, Brescia, Italy, ²⁰Mayo Clinic, Rochester, MN, ²¹Hospital Universitario 12 de Octubre, Madrid, Spain, ²²Johns Hopkins University School of Medicine, Timonium, MD, ²³Hospital Clinic de Barcelona, Barcelona, Spain, ²⁴Istanbul University, Istanbul Faculty of Medicine, Division of Rheumatology, Istanbul, Turkey, ²⁵CREAR, Rosario, Argentina, ²⁶Department of Rheumatology and lmmunology, Ruijin Hospital, School of Medicine, Shanghai Jiao Tong University, Shanghai, China, Shanghai, China (People's Republic), ²⁷University of Michigan, Ann Arbor, MI, ²⁸University of Texas Medical Branch, Galveston, TX, ²⁹IRCCS Istituto Auxologico Italiano 100%, Cusano Milanino, Milan, Milan, Italy, ³⁰Division of Rheumatology, Allergy, and Immunology, University of North Carolina, Chapel Hill, NC, ³¹King's College London, London, United Kingdom, ³²University College London Hospitals NHS Foundation Trust, London, United Kingdom

Meeting: ACR Convergence 2024

Keywords: antiphospholipid syndrome, Pediatric rheumatology

Session Information

Date: Sunday, November 17, 2024

Title: Pediatric Rheumatology – Clinical Poster II

Session Type: Poster Session B

Session Time: 10:30AM-12:30PM

Background/Purpose: APS ACTION Registry studies long-term outcomes in persistently antiphospholipid antibody (aPL)-positive patients with/without other systemic autoimmune rheumatic diseases. Given autoimmune diseases present differently in different age groups, and the limited number of studies comparing pediatric to adult antiphospholipid syndrome (APS) patients, our objective was to analyze the characteristics of patients presenting with aPL-related clinical manifestations during childhood (pediatric-onset) versus adulthood (adult-onset).

Methods: A web-based data capture system is used to store patient demographics and aPL-related medical history. Inclusion criteria are patients between 18 to 75 years-old (yo), and positive aPL based on Revised Sapporo Classification Criteria. For this retrospective analysis, we retrieved baseline demographic, clinical (including the first aPL-related event type/date), and laboratory (including the first positive aPL date) characteristics of patients. Events were grouped as: vascular (macro/micro); and non-vascular (immune thrombocytopenia, autoimmune hemolytic anemia, cardiac valve disease, and/or cognitive dysfunction). First, we compared the frequency of vascular and non-vascular events between patients with pediatric (0-17 yo)- and adult-onset (18-75 yo) aPL-related manifestations. Secondly, we descriptively analyzed the characteristics of patients in smaller age intervals, and the timeline between the first aPL-related clinical event and first aPL positivity.

Results: Of 1,122 patients recruited as of January 2023, 335 (30%) were excluded due to no history of an aPL-related event. Of 787 remaining (mean age at registry entry 46 + 14), 70% (547) were female; 61% (484) had primary aPL-positivity with/without APS classification, 32% (248) had lupus classification, 447/787 (57%) had only vascular events, 108 (14%) had only non-vascular events, and 232 (29%) had both. Pediatric-onset patients presented more commonly with a non-vascular event (as the first event), compared to adult-onset patients (49% vs 19%, p: 0.0001) (Table 1); the percentage of patients presenting with a non-vascular event mostly decreased with each increasing age group (Table 2, Figure 1) A subgroup analysis of 484 primary aPL/APS patients, revealed no significant change in the results; 47% of pediatric-onset and 20% of adult-onset patients presented with a non-vascular event (p: 0.0007, full data not shown). Timeline analysis between the first aPL event and aPL positivity demonstrated, 317 (40%) patients had a positive aPL test within the same calendar year (c-y) of the first clinical event, 207 (26%) within (+/-) 1 to 3 c-y, and 263 (33%) more than (+/-) 3 c-y.

Conclusion: Our analysis of a large-scale international multi-center registry for persistently aPL-positive patients demonstrates that patients with pediatric-onset aPL-related manifestations more commonly present with non-vascular events. These results, despite the relatively small number of pediatric-onset aPL-related manifestations, highlight the importance of understanding the clinical differences between pediatric and adult APS patients, which have diagnostic, therapeutic, and research implications.

Disclosures: J. Pandya: None; D. Andrade: None; A. Clarke: AstraZeneca, 2, 5, 6, Bristol Myers Squibb, 2, GSK, 2, 5, 6, Otsuka Pharmaceutical, 1, Roche, 1; M. Tektonidou: None; V. Pengo: None; M. Radin: None; J. Pardos-Gea: None; N. Kello: None; D. Paredes-Ruiz: None; M. Aguirre-Zamorano: None; H. Belmont: Alexion, 1, Aurinia, 6; P. Fortin: AstraZeneca, 2, 6, GlaxoSmithKlein(GSK), 2, 6, Moderna, 2; F. Signorelli: None; T. ATSUMI: None; Z. Zhang: None; M. Efthymiou: None; D. Branch: UCB Pharma Inc, 5; G. Pazzola: None; A. Tincani: None; A. Duarte-Garcia: None; E. Rodriguez-Almaraz: None; M. Petri: Amgen, 2, AnaptysBio, 2, Annexon Bio, 2, Arthros-FocusMedEd, 6, AstraZeneca, 2, 5, Atara Biosciences, 2, Aurinia, 5, 6, Autolus, 2, Avoro Ventures, 2, Biocryst, 2, Boxer Capital, 2, Cabaletto Bio, 2, Caribou Biosciences, 2, CTI, 1, CVS Health, 1, Eli Lilly, 2, 5, Emergent Biosolutions, 1, Ermiium, 2, Escient Pharmaceuticals, 2, Exagen, 5, Exo Therapeutics, 2, Gentibio, 2, GlaxoSmithKlein(GSK), 2, 5, iCell Gene Therapeutics, 2, Innovaderm Research, 2, IQVIA, 1, Janssen, 5, Kira Pharmaceuticals, 2, Merck/EMD Serono, 1, Nexstone Immunology, 2, Nimbus Lakshmi, 2, Novartis, 2, PPD Development, 2, Precision Biosciences, 2, Proviant, 2, Regeneron Pharmaceuticals, 2, Sanofi, 2, Seismic Therapeutic, 2, Senti Bioscienes, 2, Sinomab Biosciences, 2, Takeda, 2, Tenet Medicines Inc, 2, TG Therapeutics, 2, UCB, 2, Vertex Pharmaceuticals, 2, Worldwide Clinical Trials, 1, Zydus, 2; R. Cervera: None; B. Artim Esen: None; G. Pons-Estel: AbbVie/Abbott, 1, AstraZeneca, 1, 6, Boehringer-Ingelheim, 6, GlaxoSmithKlein(GSK), 1, 5, 6, Janssen, 1, 5, 6, Werfen/Inova, 6; H. Shi: None; Y. Zuo: None; R. Willis: Louisville APL Diagnostics Inc, 2, 8; P. Meroni: None; R. Roubey: None; M. Bertolaccini: None; H. Cohen: argenx, 1, GlaxoSmithKlein(GSK), 6, Roche, 1, Technoclone (paid to Univ Coll London Hosp Charity), 6, UCB (paid to Univ College London Hosp Charity), 2; D. Erkan: ACR, 5, APS ACTION, 4, Argenx, 1, Cadrenal, 2, Chugai, 1, EULAR, 5, Exagen, 5, GlaxoSmithKlein(GSK), 2, 5, 6, Kyverna, 1, NIH, 5, Otsuka/Visterra, 1, Up-To-Date, 9.

To cite this abstract in AMA style:

Pandya J, Andrade D, Clarke A, Tektonidou M, Pengo V, Radin M, Pardos-Gea J, Kello N, Paredes-Ruiz D, Aguirre-Zamorano M, Belmont H, Fortin P, Signorelli F, ATSUMI T, Zhang Z, Efthymiou M, Branch D, Pazzola G, Tincani A, Duarte-Garcia A, Rodriguez-Almaraz E, Petri M, Cervera R, Artim Esen B, Pons-Estel G, Shi H, Zuo Y, Willis R, Meroni P, Roubey R, Bertolaccini M, Cohen H, Erkan D. Antiphospholipid Antibody-related Clinical Manifestations Presenting During Childhood versus Adulthood: Descriptive Results from the AntiPhospholipid Syndrome Alliance for Clinical Trials and InternatiOnal Networking (APS ACTION) Clinical Database and Repository (“Registry”) [abstract]. Arthritis Rheumatol. 2024; 76 (suppl 9). https://acrabstracts.org/abstract/antiphospholipid-antibody-related-clinical-manifestations-presenting-during-childhood-versus-adulthood-descriptive-results-from-the-antiphospholipid-syndrome-alliance-for-clinical-trials-and-internat/. Accessed .

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