Current Treatment of Macrophage Activation Syndrome Worldwide: The METAPHOR Project, a PReS/PRINTO Real-life International Survey

Francesca Minoia¹, Francesco Baldo², Remco Erkens³, Greta Rogani³, Claudia Bracaglia⁴, Dirk Foell⁵, Marco Gattorno⁶, Marija Jelusic⁷, Jordi Anton⁸, Paul Brogan⁹, Scott Canna¹⁰, Shanmuganathan Chandrakasan¹¹, Randy Cron¹², Fabrizio De Benedetti⁴, Alexei Grom¹³, Merav Heshin Bekenstein¹⁴, AnnaCarin Horne¹⁵, Raju Khubchandani¹⁶, Mao Mizuta¹⁷, Seza Ozen¹⁸, PIERRE QUARTIER¹⁹, Angelo Ravelli⁶, Masaki Shimizu²⁰, grant schulert¹³, Christiaan Scott²¹, Rashmi Sinha²², Nicolino Ruperto²³, Joost Swart³ and Sebastiaan Vastert³, and the PReS MAS/sJIA Working Party and Pediatric Rheumatology International Trial Organization., ¹Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milano, Milan, Italy, ²Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy, ³University Medical Center Utrecht, Utrecht, Netherlands, ⁴IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy, ⁵University Hospital Muenster, Muenster, Germany, ⁶IRCCS Giannina Gaslini, Genoa, Italy, ⁷University of Zagreb School of Medicine, University Hospital Centre Zagreb, Zagreb, Zagreb, Croatia, ⁸Hospital Sant Joan de Déu. Universitat de Barcelona, Esplugues de Llobregat (Barcelona), Spain, ⁹UCL Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom, ¹⁰Children's Hospital of Philadelphia, Philadelphia, PA, ¹¹Aflac Cancer and Blood Disorders Center Children's Healthcare of Atlanta Emory University School of Medicine, Atlanta, GA, ¹²University of Alabama at Birmingham, Birmingham, AL, ¹³Cincinnati Children's Hospital Medical Center, Cincinnati, OH, ¹⁴Dana Dwek Children's Hospital, Tel Aviv Medical Center, Tel Aviv University, Binyamina, Israel, ¹⁵Karolinska Institute, Sollentuna, Sweden, ¹⁶SRCC Childrens Hospital, Mumbai, India, ¹⁷Department of Pediatric Rheumatology, Hyogo Prefectural Kobe Children's Hospital, Kanazawa, Hyogo, Japan, ¹⁸Department of Pediatrics, Hacettepe University, Ankara, Turkey, ¹⁹Université Paris-Cite, IMAGINE Institute, Necker Children's Hospital, Paris Cedex 15, France, ²⁰Tokyo Medical and Dental University, Bunkyo-ku, Tokyo, Japan, ²¹University of Cape Town, Cape Town, South Africa, ²²Systemic JIA Foundation, Cincinnati, OH, ²³IRCCS Giannina Gaslini, Genova, Italy

Meeting: ACR Convergence 2024

Keywords: macrophage activation syndrome, Surveys

Session Information

Date: Monday, November 18, 2024

Title: Pediatric Rheumatology – Clinical Poster III

Session Type: Poster Session C

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

Background/Purpose: Despite significant improvement in its management, macrophage activation syndrome (MAS) treatment is still not standardized, due to lack of robust evidence and differences in access to medications, especially for MAS related to rheumatologic conditions other than systemic juvenile idiopathic arthritis (sJIA). We aimed to capture the current treatment of MAS worldwide and to evaluate major unmet needs

Methods: In context of the METAPHOR project, a PReS/PRINTO initiative to optimize treatment in sJIA and MAS,
a survey on MAS treatment was developed. Topics addressed were based on a systematic literature review, and selected by a panel of 22 experts, including 1 patient representative and 2 pediatric hematologists. The survey consisted of: a) demographic section, including country of practice; b) clinical section, investigating real-life approaches to MAS; c) patient focused section, developed with patient representatives and exploring major unmet needs. Physicians part of the PReS/PRINTO network and of the Histiocyte Society were invited to anonymously complete the web-survey from Oct 5^th to Dec 15^th2023

Results: A total of 287 replies from 64 countries worldwide were collected. Respondents were mostly pediatric rheumatologists (90%), while 10% were pediatric hematologists. Methylprednisolone (MPN) pulses were the commonest glucocorticoid (GC) in all subtypes of MAS. In addition to GC, ciclosporin (CsA) and anakinra were the cornerstones of treatment in sJIA-MAS, used by 91% and 74% of physicians, respectively. Anakinra was the most selected agent beside GC as 1^st line, and only 2% of respondents indicated not to consider the use anakinra in sJIA-MAS. However, access to anakinra is a major gap across countries: almost all physicians would use it in North America and Western Europe, but it is still unavailable for more than 65% and 70% of physicians in Asia and South America (Table 1). Systemic erythematosus lupus (SLE)-associated MAS represents the condition with the highest heterogeneity of therapeutic approaches across centres and countries. Besides MPN, which was largely the 1^st line choice worldwide (98%), 58% of physicians would use anakinra as 1^st line in North America and 63% as 1^st/2^nd line in Western Europe, while CsA and immunoglobulin would be the medications of choice in all the other countries (Table 2). Etoposide was globally the agent most frequently selected as 3^rd line/rescue, followed by JAK-inhibitors, mainly ruxolitinib. Emapalumab was potentially chosen as 2^nd/3^rd line treatment across all subtypes of MAS; however, its access is still drastically limited in all countries, except North America and partially Western Europe

Conclusion: A wide heterogeneity in the approach to MAS still exists, with relevant discrepancies in its management worldwide. An international effort is needed to optimize therapeutic options, reduce gaps in access to medications and harmonize treatment

Table 1. Treatment approaches to sJIA-MAS worldwide

Table 2. Treatment approaches to SLE-MAS worldwide

Disclosures: F. Minoia: Novartis, 6, SOBI, 6; F. Baldo: None; R. Erkens: None; G. Rogani: None; C. Bracaglia: GlaxoSmithKlein(GSK), 6, Novartis, 2, Sobi, 6; D. Foell: Boehringer-Ingelheim, 2, Novartis, 2, SOBI, 2; M. Gattorno: None; M. Jelusic: None; J. Anton: None; P. Brogan: None; S. Canna: Apollo Therapeutics, 2, Bristol-Myers Squibb(BMS), 6, Novartis, 5, PracticePoint CME, 6, Simcha Therapeutics, 5; S. Chandrakasan: None; R. Cron: AbbVie/Abbott, 1, Pfizer, 1, Sobi, 1, 5; F. De Benedetti: None; A. Grom: None; M. Heshin Bekenstein: None; A. Horne: Novartis, 6, SOBI, 6; R. Khubchandani: None; M. Mizuta: None; S. Ozen: Novartis, 2, 6, SOBI, 2, 6; P. QUARTIER: Abbvie, 2, 5, 6, BMS, 2, 5, 6, Chugai-Roche, 2, 5, 6, Novartis, 2, 5, 6, Pfizer, 2, 5, 6, Sweedish Orphan Biovitrum, 2, 5, 6; A. Ravelli: AbbVie/Abbott, 2, 6, Alexion, 6, Galapagos, 2, Novartis, 2, 5, 6, Pfizer, 2, 6, SOBI, 6; M. Shimizu: None; g. schulert: Boehringer-Ingelheim, 2, IpiNovyx, 5, SOBI, 2; C. Scott: None; R. Sinha: None; N. Ruperto: Ablynx, 2, Amgen, 2, Astrazeneca-Medimmune, 2, Aurinia, 2, Bayer, 2, Bristol Myers and Squibb, 2, Cambridge Healthcare Research (CHR), 2, Celgene, 2, Domain therapeutic, 2, Eli Lilly and Company, 2, 6, EMD Serono, 2, Glaxo Smith and Kline, 2, 6, Idorsia, 2, Janssen, 2, Novartis, 2, Pfizer, 2, 6, Sobi, 2, 6, UCB, 2, 6; J. Swart: None; S. Vastert: Novartis, 2, Sobi, 2.

To cite this abstract in AMA style:

Minoia F, Baldo F, Erkens R, Rogani G, Bracaglia C, Foell D, Gattorno M, Jelusic M, Anton J, Brogan P, Canna S, Chandrakasan S, Cron R, De Benedetti F, Grom A, Heshin Bekenstein M, Horne A, Khubchandani R, Mizuta M, Ozen S, QUARTIER P, Ravelli A, Shimizu M, schulert g, Scott C, Sinha R, Ruperto N, Swart J, Vastert S. Current Treatment of Macrophage Activation Syndrome Worldwide: The METAPHOR Project, a PReS/PRINTO Real-life International Survey [abstract]. Arthritis Rheumatol. 2024; 76 (suppl 9). https://acrabstracts.org/abstract/current-treatment-of-macrophage-activation-syndrome-worldwide-the-metaphor-project-a-pres-printo-real-life-international-survey/. Accessed .

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