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
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.