EASL 2013 Ivan Gardini

MY PERSONAL VIETNAM

20 YEARS OF CHRONIC HEPATITS 5 TREATMENTS FAIELD 3 CIRRHOSIS 2 LIVER TRANSPLANT

A virus that has stolen 20 years of my life Kidnapping mind, body and spirit

NO RE-TRANSPLANT= MONTHS LEAVING Post OLT diagnosis beginning 2011 CHOLESTATIC AND AGGRESSIV HCV RECURRENCY First transplant, cirrhosis due HCV: December 1997, 34 year old Second transplant, due to HCV recurrency: July 2009, 46 years old

CLINICAL PROFILE beginning 2011  PARTIAL RESPONDER  IL28 CT  WIDESPREAD CIRRHOSIS  PLATELET (Limits for compassionate use: )  DIABETIC  IMMUNOSOPPRESSIVE TREATMENT MAINTENANCE THERAPY INF PEG + RIBA

February 2011 INF PEG alfa + RBV Copie/mL

boceprevir INF PEG alfa + RBV Lead in: 18 weeks Febbraio 11Maggio 11 Copie/mL

boceprevir Week 2 4 log decrease INF PEG alfa + RBV + BOCEPREVIR February 11 May 11 boceprevir Copie/mL

boceprevir INF ALFA + RBV SOC + BOC boceprevir INF PEG alfa + RBV + BOCEPREVIR Febbraio 11 Maggio 11 Week 4 5 log decrease Copie/mL

boceprevir INF ALFA + RBV SOC + BOC Week 6 HCV RNA negative INF PEG alfa + RBV + BOCEPREVIR boceprevir February 11 May 11 Copie/mL

INF ALFA + RBV SOC + BOCINF PEG alfa + RBV + BOCEPREVIR – 48 WEEKS May 11 May 12 HCV RNA negative

LAST HCV RNA (FEBRUARY 2013) SVR 36 !

WAS IT EASY? Side effects Anemia = Erithropoietina used Neutropenia =growing factor used Asthenia Disguesya Diarrhea Pruritis 2 SYNCOPES NO

WHICH SENSATION? AFTER 186 interferon INJECTIONS Ribavirin and other PILLS AND MANY OTHER MEDICATIONS

PURCHASED THE RETURN TICKET FROM HELL

DAAs EARLY ACCESS with compassionate use may SAVE LIFES And patients are Back to life

Back to life: In a DISCO with my daughters

Each year, 5 – 7% of patients with compensated cirrhotics progress to decompensated cirrhosis (D’amico et al J Hepatol Jan;44(1):217-31).

In these patients, all drug regimes based on pegylated interferon, ribavirin and other DAAs cannot be used due to the severe side effects and contraindications. Inevitably those people will eventually face liver cancer, liver transplant or death, which dramatically increases the patients suffering as well as social and economical costs.

WARNING REGISTRATIVE TRIALS, CLINICAL TRIALS, EXPANDED ACESS PROGRAMS AND ALSO COMPASSIONATE USE HAVE SEVERAL EXCLUSION CRITERIA THAT MEAN CLOSE THE DOOR TO THE PATIENTS MOST IN URGENT NEED FOR THOSE PATIENTS THE QUESTION IS: WAIT FOR TOTAL SAFETY DATA OR OPEN A RISKLY WINDOW TO THE EARLY ACCESS ?

To remove from compassionate use protocols most of the exclusion criteria that may lead to exclude patients with HCV advanced disease. This regards the compassionate use and the named patient basis programs The right of the patients to have curative choices even taking some risks, with European regulatory agencies to issue opinions on the subject An european register for the results of curative DAAs combinations given by compassionate use a common legislation approach of Member States particularly on Compassionate use programmes for hcv patients WHAT WE ASK FOR IS….

Thank you!!