Leniolisib and rapamycin in Activated PI3-Kinase-δ-syndrome: a retrospective ESID registry-based analysis.
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Rivalta Beatrice, Wolkewitz Martin, Kusters Maaike, Uhlmann Annette, Guarnieri Valentina, Abou-Chahla Wadih, Adjaoud Dalila, Aladjidi Nathalie, Armari-Alla Corinne, Azarnoush Saba, Baris Safa, Barlogis Vincent, Barzaghi Federica, Baumann Ulrich, Bloomfield Marketa, Bodet Damien, Boutboul David, Boz Valentina, Bozkurt Selcen, Bucciol Giorgia, Cancrini Caterina, Catherinot Emilie, Cavazzana Marina, Cheminant Morgane, Chinello Matteo, Ciznar Peter, Conti Francesca, Costagliola Giorgio, Dalm Virgil, D'Aveni-Piney Maud, Ekwall Olov, Eren Efrem, Eric Zelimir, Farmaki Evangelia, Fasth Anders, Fieschi Claire, Fischer Alain, Fournier Benjamin, Galicier Lionel, Ghosh Sujal, Gkantaras Antonios, Hatchuel Yves, Hilfanova Anna, Hoenig Manfred, Jeziorski Eric, Jolles Stephen, Karakoc-Aydiner Elif, Kilic Sara S, Kiykim Ayca, Körholz Julia, Le Guenno Guillaume, Licciardi Francesco, Lodi Lorenzo, Lougaris Vassilios, Lucena José Manuel, Malphettes Marion, Marzollo Antonio, Masmas Tania N, Meinhardt Andrea, Merlin Etienne, Meyts Isabelle, Milota Tomas, Moratti Mattia, Moussouni Nabila, Neth Olaf, Nunes-Gomes Christopher, Ocak Süheyla, Olbrich Peter, Yücel Esra Özek, Özen Ahmet, Schmid Jana Pachlopnik, Palma Paolo, Pellier-Landreau Isabelle, Perlat Antoinette, Picard Capucine, Prader Seraina, Ricci Silvia, Riviere Jacques, Santilli Veronica, Sarrot-Reynauld Francoise, Schleinitz Nicolas, Schrijvers Rik, Schulz Ansgar, Schuetz Catharina, Sediva Anna, Semeraro Michaela, Sharapova Svetlana, Sogkas Georgios, Soomann Maarja, Palacín Pere Soler, Suarez Felipe, Tessarin Giulio, Tommasini Alberto, Trück Johannes, van Aerde Koen, van Montfrans Joris, Vermont Clementien, Wustrau Katharina, Zharankova Yulia, Moshous Despina, Neven Bénédicte, Seidel Markus G, Chandra Anita, Thalhammer Julian, Kracker Sven, Ehl Stephan, Mahlaoui Nizar, Elfeky Reem, Maccari Maria Elena, ESID-APDS Registry Working Party
Аннотация
OBJECTIVE: To assess real-world treatment indications, clinical course, immunological changes, and tolerability in patients with APDS treated with rapamycin and leniolisib.
METHODS: We performed a retrospective analysis of the ESID-APDS registry including 95 APDS patients treated with rapamycin (81) and/or leniolisib (28) through a Managed Access Program. Clinical manifestations, immunological parameters, and treatment-associated complications were assessed.
RESULTS: Lymphoproliferation was the predominant indication (81% and 52% respectively) for treatment initiation. Overall responses (complete or partial) were observed in 67% of rapamycin- and 64% of leniolisib-treated patients. Across other disease manifestations, responses were documented in 35% and 50% of patients. Leniolisib was initiated for recurrent infections in 3 patients, whereas recurrent infections were not a primary indication for rapamycin. Treatment interruption due to adverse events occurred only in the rapamycin group. Lymphoma cases were observed during follow-up.
CLINICAL IMPLICATION: In this real-world registry cohort, both rapamycin and leniolisib were associated with improvement of lymphoproliferation in APDS. Treatment-limiting adverse events were reported under rapamycin. Prospective long-term studies are needed to better define the role of these therapies and their long-term impact on disease outcome, including malignancy risk.
CLINICAL IMPLICATION: In a real-world APDS cohort, leniolisib and rapamycin reduced benign lymphoproliferation, with treatment-limiting adverse events reported only under rapamycin.