Abstract Background Inflammatory Bowel Diseases (IBD) severely impact Quality of Life (QoL). While the “treat-to-target” strategy aims for clinical and endoscopic remission, studies indicate a discrepancy between the therapeutic goals set by patients and specialists. We aimed to explore this discordance and assess its effect on the perceived effectiveness of advanced IBD therapies in a real-world setting. Methods This prospective, multicentric, national observational study included adult Italian IBD patients (Crohn’s Disease, CD; Ulcerative Colitis, UC) initiating advanced therapy (monoclonal antibody or small molecules). At baseline (Week 0), patients and their prescribing physicians independently chose and ranked three targets from a predefined list of 18 (Figure 1). The primary aim was to assess the achievement of these goals by Week 52. Achievement of the primary target and all three selected targets was evaluated by both parties at Weeks 26 and 52. Results Two hundred eighty-eight subjects were enrolled (145 UC; 143 CD; mean age 42.7 ± 15.6 years) in 14 Italian referral centres. The Full Analysis Set (FAS) included 258 subjects. Overall, 147 patients (67.7%) achieved their primary predefined target by Week 52 (UC 68.1%, CD 67.3%). Target achievement was significantly higher when assessing the physician’s goals: 175 patients (75.4%) achieved the physician’s primary expected target at Week 52. A significant disparity emerged when assessing multi-target achievement: 137 patients (59.1%) achieved all three physician-defined targets, compared to only 101 patients (46.3%) who achieved all three of their personally selected targets (p = 0.006). Achievement of all three targets increased in patients in clinical remission 124 overall (73.4%), 69 (75.0%) in UC, 55 (71.4%) in CD. Safety analysis revealed that 102 (35.5%) subjects experienced at least one Adverse Event (total 211 events), with Serious AEs occurring in 16 (5.6%) subjects. Conclusion This study confirms important discrepancy between patients’ and physicians’ therapeutic expectations. Patients were less likely to achieve their own self-selected, QoL-focused goals compared to the clinically-driven goals set by their treating physicians. Active integration of Patient-Reported Outcomes (PROs) into formalized goal setting is essential to better align treatment targets with patient needs. Conflict of interest: Dr. Fiorino, Gionata: Personal Fees: Takeda, Johnson & Johnson, Alfasigma, AbbVie, Celltrion, Pfizer, Sandoz, Abivax, Lilly, STADA Salice, Marco: None Loy, Laura: NONE Mastronardi, Mauro: none Allocca, Mariangela: Personal Fees: consulting fees from Nikkiso Europe, Mundipharma, Janssen, Abbvie, Pfizer, Ferring, Galapagos, Sandoz, Lilly and Alfasigma Laterza, Lucrezia: Employment full time / part time None Research Grant (P.I., collaborator or consultant pending and received grants) None Other research support None Speakers Bureau / Honoraria Company name/Lecturer Abivax, Biocure Ownership interest (stock, stock-options, patent or intellectual property None Consultant / advisory board Actial Farmaceutica, Abbvie, Lilly, J & J Onali, Sara: Consultant/lecture fees to: Abbvie, Alfasigma, MSD, Takeda, J & J, Galapagos, Pfizer, Eli Lilly Testa, Anna: Consultant /Advisory board for Abbvie, J & J, Takeda, Ferring Macaluso, Fabio Salvatore: Advisory board and/or lecture fees for: AbbVie, MSD Galapagos, Sandoz, Takeda, Janssen, Eli-Lilly, Pfizer, Alfasigma, Giuliani Variola, Angela: Consultant for Abbvie, Alfasigma, Celltrion, Eli-Lilly, Johnson and Johnson, Takeda, Pfizer Speaker fee for Abbvie, Alfasigma, Celltrion, Eli-Lilly, Johnson and Johnson, Ferring, Takeda, Pfizer, Zamboni Viola, Anna: None Cosintino, Rocco: None Ribaldone, Davide Giuseppe: Davide Giuseppe Ribaldone declares the following paid consultancies, lecture fees for the past two years: Johnson & Johnson, Takeda, Celltrion, Alfasigma, Pfizer, Eli Lilly, Abbvie, Sandoz Conforti, Francesco Simone: none Carmagnola, Stefania: None Caprioli, Flavio: Flavio Caprioli served as consultant to: Abbvie, Amgen, MSD, Takeda, Janssen, Roche, Celgene, Bristol-Meyers Squibb, Galapagos, Gllead, Pfizer, Mundipharma, Biogen, Ferring, Eli-Lilly, Nestlè, Lionhealth, AlfaSigma, Dr Falk, Celltrion, Abivax. He received lecture fees from Abbvie, Ferring, Takeda, Allergy Therapeutics, Janssen, Pfizer, Biogen, Sandoz, Tillotts Pharma, Vifor Pharma, AlfaSigma, Celltrion, Eli-Lilly. and unrestricted research grants from Giuliani, Sofar, MSD, Takeda, Abbvie, Celltrion, Pfizer, Actial. Orlando, Ambrogio: Advisory board and/or lecture fees for: AbbVie, Galapagos, Celltrion, MAD, Sofar, Pfizer, Takeda, Cadigroup, Sandoz, Janssen, Eli-Lilly, Alfasigma Castiglione, Fabiana: Honoraria from: Takeda, AbbVie, Celltrion, Johnsson Johnsson, Cadigroup, Sandoz, Pfizer, Lilly, Lionhealth, Nestlè Fantini, Massimo Claudio: MCF has acted as a consultant for: AbbVie, AlfaSigma, Celgene, Celltrion, Gilead, Pfizer, MSD, Bristol-Meyer, Takeda, Johnson & Johnson, Roche, Galapagos, Biogen, Sandoz, Eli-Lilly, Lionhealth, Teva, Giuliani, Dr Falk Pharma, Sanofi he has received financial support for research from Johnson & Johnson and Pfizer. Danese, Silvio: Personal Fees: AbbVie, Alimentiv, Allergan, Amgen, Applied Molecular Transport, AstraZeneca, Athos Therapeutics, Biogen, Boehringer Ingelheim, Bristol Myers Squibb, Celgene, Celltrion, Dr Falk Pharma, Eli Lilly, Enthera, Ferring Pharmaceuticals Inc., Gilead, Hospira, Inotrem, Janssen, Johnson & Johnson, Morphic, MSD, Mundipharma, Mylan, Pfizer, Roche, Sandoz, Sublimity Therapeutics, Takeda, Teladoc Health, TiGenix, UCB Inc., Vial, Vifor Lecture fees from Abbvie, Amgen, Ferring Pharmaceuticals Inc., Gilead, Janssen, Mylan, Pfizer, Takeda Armuzzi, Alessandro: Consulting fees from AbbVie, Abivax, Alfa Sigma, Astra Zeneca, Biogen, Boehringer Ingelheim, Bristol-Myers Squibb, Celltrion, Eli-Lilly, Enthera, Ferring, Galapagos, Gilead, Giuliani, Janssen, Lionhealth, MSD, Nestlé, Pfizer, Protagonist Therapeutics, Roche, Samsung Bioepis, Sanofi, Sandoz, Takeda, Teva Pharmaceuticals, Tillots Pharma Speaker’s fees from AbbVie, Abivax, AG Pharma, Alfa Sigma, Biogen, Bristol-Myers Squibb, Celltrion, Eli-Lilly, Ferring, Galapagos, Gilead, Janssen, Lionhealth, MSD, Novartis, Pfizer, Roche, Samsung Bioepis, Sandoz, Takeda, Teva Pharmaceuticals Research support from Biogen, MSD, Takeda, and Pfizer Non-financial support from Abbvie, Janssen, MSD, Pfizer, Takeda Gionchetti, Paolo: Personal Fees: Takeda, Johnson & Johnson, Alfasigma, AbbVie, Celltrion, Pfizer, Sandoz, Abivax, Lilly, STADA
Fiorino et al. (Thu,) studied this question.