Pietro Lampertico 1 , Mauro Viganò 2 , Roberta Soffredini 1 , Floriana Facchetti 1 , Eliseo Minola 3 , Giuliana Cologni 4 , Marco Rizzi 4 , Osvaldo Fracassetti 4 , Fredy Suter 4 , Serena Zaltron 5 , Andrea Vavassori 5 , Giampiero Carosi 5 , M. Puoti 5 , Elena Angeli 6 , Guido A. Gubertini 6 , Carlo Magni 6 , Angela Testa 7 , Pasquale Narciso 7 , Giorgio Antonucci 7 , Maria Vinci 8 , Giovambattista Pinzello 8 , Erika Fatta 9 , Silvia Fargion 9 , Paolo Del Poggio 10 , Barbara Coco 11 , Maurizia R. Brunetto 11 , Marco Andreoletti 12 , Agostino Colli 12 , Massimo Fasano 14 , Teresa Santantonio 13 , Guido Colloredo 15 , Luisa Pasulo 16 , Stefano Fagiuoli 16 , Alberto Eraldo Colombo 17 , Giorgio Bellati 17 , Francesco Fumagalli Maldini 18 , Maria Milanese 18 , Massimo Pozzi 19 , Natalia M. Terreni 20 , Giancarlo Spinzi 20 , Michela Quagliuolo 21 , Mauro Borzio 21 , Giovanna Lunghi 22 , Massimo Colombo 1 Maintained viral suppression and excellent safety profile of entecavir monotherapy in 418 NUC- naïve patients with chronic hepatitis B : a 4-year field practice, multicenter study 1. 1st Division of Gastroenterology, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milan, Italy. 2. U.O.Epatologia, Ospedale San Giuseppe,Università degli Studi di Milano, Milan, Italy. 3. Servizio Malattie Epatiche e Infettive, Humanitas Gavazzeni, Bergamo, Italy. 4. Infectious Diseases, Ospedali Riuniti di Bergamo, Bergamo, Italy. 5. II Divisione Malattie Infettive, Azienda Ospedaliera Spedali Civili, Brescia, Italy. 6. I and II Division Infectious Diseases, Ospedale Luigi Sacco, Milan, Italy. 7. INMI, IRCCS L. Spallanzani, Roma, Italy. 8. SC Epatologia e Gastroenterologia, Ospedale Niguarda Cà Granda, Milan, Italy. 9. Internal Medicine 1b, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milan, Italy. 10. U.O. Epatologia, Ospedale di Treviglio, Treviglio, Italy. 11. U.O. Epatologia, Azienda Ospedaliero-Universitaria Pisana, Pisa, Italy. 12. S.C. Medicina Generale, Ospedale A. Manzoni, Lecco, Italy. 13. Clinic of Infectious Diseases, Università di Foggia, Foggia, Italy. 14. Clinic of Infectious Diseases, Università di Bari, Bari, Italy. 15. Division of Medicine, Policlinico San Pietro, Ponte San Pietro, Italy. 16. Gastroenterology Unit, Liver and Lung Transplantation Center, Ospedali Riuniti di Bergamo, Bergamo, Italy. 17. Unità Operativa di Medicina, Servizio di Epatologia, Ospedale Sant'Anna, Como, Italy. 18. Liver Center, Clinica Medica, Azienda Ospedaliera San Gerardo, Università Milano Bicocca, Monza, Italy. 19. U.O. Medicina, Ospedale Fatebenefratelli, Erba, Italy. 20. U.O. Gastroenterologia, Ospedale Valduce , Como, Italy. 21. U.O. Gastroenterologia, Azienda Ospedaliera di Melegnano, Melegnano, Italy. 22. Istituto di Medicina Preventiva, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milan, Italy Background and Aim Registration trials showed Entecavir (ETV) to be a safe and effectiv e therapy for NUC-naïv e, HBeAg positiv e and negativ e patients with chronic hepatitis B. Data beyond year 1 are available for a subset of patients, only Preliminary, short-term field practice studies suggest that most patients on ETV monotherapy achiev ed HBV suppression with few treatment failures, only. The risk of HCC in cirrhotic patients under long-term suppressive therapy with ETV, is still unknown. To assess the long-term effectiveness and safety of ETV in a large cohort of NUC-naïv e patients w ith CHB Patients and Methods Study retrospective/prospective cohort, multicenter Patients 418 consecutive NUC-naiv e patients with CHB (19 Italian centers) Enrolment: 2007-2008 Treatment ETV 0.5 mg Follow -up 48 months (2-62) Assays: HBV-DNA (PCR-based assays) ETV-resistance (INNO-LiPA HBV DR V2-3) Monitoring: ev ery 3-6 months Baseline clinical and virological characteristics Num ber 418 Age, yr* 58 (18-82) Male 316 (76%) HBe Ag neg 347 (83%) HBV-DNA, log IU/ml* 6.0 (1.5-9) ALT, U/ml* 92 (11-2241) Genotype D 84/93 (90%) Cirrhosis 202 (49%) BMI > 25 Kg/m 2 168/365 (46%) Concomitant diseases 228 (56%) * Median (range) 0 20 40 60 80 100 0% 6 67% Patients % 85% 12 Virological response through month 52 (undetectable HBV DNA) Baseline 405 418 391 Patients on f-up 95% 344 Months 24 96% 288 36 98% 188 48 100% 81 54 Virological response by HBeAg status (undetectable HBV DNA) 0 20 40 60 80 100 6 41% Patients % 66% 12 67 66 88% 48 Months 24 0 20 40 60 80 100 6 74% Patients % 90% 12 338 327 Patients on f-up 96% 296 24 HBeAg positive HBeAg negative 87% 35 36 98% 260 36 94% 29 48 99% 167 48 100% 12 54 100% 58 54 0 20 40 60 80 100 0 6 12 18 24 30 36 42 48 Months Patients : - at risk HBeAg seroconversion rates* 71 67 54 56% 45 - on f-up 71 67 64 55 47 38 43 27 HBeAg seroconversion: 27 patients * Kaplan-Meier estimates 35 17 20 9 10 4 0 10 20 30 40 50 0 6 12 18 24 30 36 42 48 Months Patients : - at risk HBsAg loss in HBeAg-positive patients* 21% HBsAg loss: 12 patients * Kaplan-Meier estimates 71 70 65 54 - on f-up 71 67 64 55 47 48 43 38 35 25 20 13 10 6 0 1 2 3 4 5 6 0 6 12 18 24 30 36 42 48 HBV-DNA log UI/ml Months of treatment ETV 0.5 mg/die TDF 245 mg LLQ < 6 UI/ml L180M M204V S202G One patient developed ETV resistance* *Innolipa assay 0 20 40 60 80 100 16% 6 69% Patients % 81% 24 12 Biochemical response (normal ALT) 86% Baseline 401 418 390 347 Patients on fup 88% 301 Months 36 89% 188 48 93% 81 54 0 10 20 30 40 50 0 6 12 18 24 30 36 42 48 Months Patients at risk HCC rates in cirrhotic patients* 10% HCC: 12 patients * Kaplan-Meier estimates 164 161 154 148 136 117 84 41 13 *2 (0.2%) patients had to reduce ETV dose due to eGFR decline: a 78-year old women with AH; a 48-year olt renal-transplanted woman with compensate dcirrhosis ** median (range) ***one patient only Renal safety* Variables Baseline Month 12 Month 24 Month 36 Month 48 Creatinine**, mg/dl 0.90 (0.6-9.0) 0.90 (0.5-2.2) 0.90 (0.5-2.0) 0.90 (0.5-2.0) 0.90 (0.5-2.0) Serum creatinine >1.5 mg/dl 2% 2% 1% 1% 3% *** Phosphorus* *, mg/dl Na 3.4 (2.4-4.3) 3.2 (2.2-4.4) 3.1 (1.9-4.5) 3.1 (2.4-4.2) Phosphorus <2.0 mg/dl Na 0% 0% 2% 0% Proteinuria (30 mg by dip stick) Na 0% 2% 0% 3% *** 0 20 40 60 80 100 18 33% % Patients with TmPO4/GFR ratio 24 TmPO4/GFR ratio over time* months 30 36 20% 36% 18% 37% 20% 51% 21% TmPO4/GFR < 0.80 mmol/L TmPO4/GFR < 0.70 mmol/L No. patients studied 42 43% 21% 15 39 54 42 53 *No data available from baseline to month 12 Patients withdrawing from the study OUTCOME No. (%) REASON (No. patients) Dead 28 (7%) HCC (16) decom (2), extraheaptic (9) unclassified (1) OLT 11 (3%) HCC* (10), ESLD (1) Lost to follow-up 39 (9%) na TDF* rescue 20 (5%) HBV DNA positive at week 48-96 Peg-IFN 8 (2%) 3 HBeAg pos, 5 HBeAg negative Stop ETV 12 (3%) HBsAg loss Overall 118 (28%) * HCC was diagnosed before ETV in 9 patients ** TDF+ETV Summary Virological responses increased over time in both HBeAg positive and negative patients, with more than 90% achieving undetectable HBV DNA HBeAg and HBsAg seroconversion rates increased over time. Twelve patients stopped ETV successfully. One patient (0.2%) developed ETV resistance at month 42 Most patients achieved persistently normal ALT levels HCC developed at 2.5% yearly rates in cirrhotics No safety issues were recorded, none of the patients had to stop the medication. Two patients (<1%) had to reduce the dose Conclusions ETV monotherapy was safe and effective up to 4 years in clinical practice, as it provided high rates of virological suppression and increasing rates of HBeAg and HBsAg seroconversion