Real-world adjunctive use of an alpha-lactalbumin/sodium butyrate Food for Special Medical Purposes in drug-resistant epilepsy: the multicentre retrospective ARMILLA study
Abstract
The purpose of this study was to describe real-world adjunctive use, tolerability, and hypothesis-generating seizure and multidomain outcomes of LALBAAY, an alpha-lactalbumin/sodium butyrate Food for Special Medical Purposes (FSMP), in patients with drug-resistant epilepsy (DRE). This retrospective national multicentre observational study (17 centres) analysed 60 patients with DRE treated with adjunctive LALBAAY. Seizure outcomes, individual treatment duration, clinician-documented gastrointestinal (GI) and behavioural changes, and tolerability were descriptively analysed. GI and behavioural domains were routinely monitored in clinical practice, but no standardised scales were used. Exploratory association analyses evaluated selected clinically plausible baseline variables potentially associated with ≥ 50% seizure reduction using Fisher exact tests and unadjusted odds ratios; all analyses were considered hypothesis-generating. The cohort included 30 patients (50.0%) with developmental and epileptic encephalopathy, 23 (38.3%) with focal epilepsy, and 7 (11.7%) with generalised epilepsy. Median age was 18.0 years (IQR 11.8–32.2). Any seizure reduction was documented in 35/60 patients (58.3%), ≥50% seizure reduction in 20/60 (33.3%; Wilson 95% CI 22.7–45.9%), and seizure freedom in 8/60 (13.3%). Two additional patients had resolution of clinician-designated major seizure events without complete seizure freedom. Favourable GI and behavioural changes were documented in 12/60 (20.0%) and 13/60 (21.7%) patients, respectively. Adverse events were documented in 4/60 patients (6.7%); 55/60 remained on treatment and 5/60 discontinued. Exploratory association analyses did not identify a statistically robust response-associated profile. In this retrospective real-world DRE cohort, adjunctive LALBAAY use was accompanied by descriptively favourable seizure findings and few documented adverse events. GI and behavioural observations were clinician-documented descriptive signals rather than standardised efficacy outcomes. Exploratory associations require confirmation in larger prospective studies.
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Authors: Emilio Russo, Antonella Riva, Federica Cristofalo, Carmen De Caro, Valeria Guarneri, Irene Bagnasco, Pier Antonio Battistella, Vincenzo Belcastro, Silvia Boeri, Marta Brichetto, Barbara Federici, Gianfranco Di Gennaro, Claudio Liguori, Greta Macorig, Francesca Marchese, Lorenzo Perilli, Benedetta Piccolo, Giulia Prato, Eleonora Rosati, Rosita Ruta, Alberto Spalice, Marilena Vecchi, Alberto Verrotti, Maurizio Viri, Paolo Mainardi, Pasquale Striano, Roberta Roberti
Institutions: University of Padua, University College London, Azienda Ospedaliero-Universitaria Careggi, Istituti di Ricovero e Cura a Carattere Scientifico, University of Bologna, University of Parma, Sapienza University of Rome, University of Naples Federico II, Magna Graecia University, Università degli Studi del Piemonte Orientale “Amedeo Avogadro”, Azienda Ospedaliera Ospedale Maggiore, IRCCS Eugenio Medea, University of Genoa, University of Rome Tor Vergata, University of Perugia, National Hospital for Neurology and Neurosurgery, Istituto Giannina Gaslini, Policlinico Tor Vergata, Ospedale Martini, Azienda Ospedaliero Universitaria Maggiore della Carita, Nuovo Ospedale San Giovanni di Dio, UCL Queen Square Institute of Neurology, Aziende Socio Sanitarie Territoriale Lodi, Istituto di Sociologia Internazionale di Gorizia