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EASL 2013
Ivan Gardini
MY PERSONAL VIETNAM
MY PERSONAL VIETNAM
20
YEARS OF CHRONIC HEPATITS
5
TREATMENTS FAIELD
3
2
CIRRHOSIS
LIVER TRANSPLANT
A virus that has stolen
20 years of my life
Kidnapping mind, body
and spirit
First transplant, cirrhosis due HCV:
December 1997, 34 year old
Second transplant, due to HCV recurrency:
July 2009, 46 years old
Post OLT diagnosis beginning 2011
CHOLESTATIC AND AGGRESSIV HCV RECURRENCY
NO RE-TRANSPLANT=
24-36 MONTHS LEAVING
CLINICAL PROFILE beginning 2011
 PARTIAL RESPONDER
 IL28 CT
 WIDESPREAD CIRRHOSIS
 93.000 PLATELET (Limits for compassionate use: 90.000)
 DIABETIC
 IMMUNOSOPPRESSIVE TREATMENT
MAINTENANCE THERAPY INF PEG + RIBA
Copie/mL
INF PEG alfa + RBV
February 2011
INF PEG alfa + RBV
Lead in: 18 weeks
Copie/mL
boceprevir
Febbraio 11
Maggio 11
INF PEG alfa + RBV + BOCEPREVIR
Week 2
4 log decrease
Copie/mL
boceprevir
boceprevir
February 11
May 11
INF PEG alfa
INF+ALFA
RBV + BOCEPREVIR
RBV SOC + BOC
Week 4
5 log decrease
Copie/mL
boceprevir
boceprevir
Febbraio 11
Maggio 11
INF PEG alfa
INF+ALFA
RBV + BOCEPREVIR
RBV SOC + BOC
Week 6
HCV RNA negative
Copie/mL
boceprevir
boceprevir
February 11
May 11
INF PEG alfa
INF+ALFA
RBV + BOCEPREVIR
RBV SOC + BOC
– 48 WEEKS
May 11
May 12
LAST HCV RNA
(FEBRUARY 2013)
SVR 36 !
WAS IT EASY? NO
Side effects
2 SYNCOPES
Anemia = Erithropoietina used
Neutropenia =growing factor used
Asthenia
Disguesya
Diarrhea
Pruritis
WHICH SENSATION?
AFTER
186 interferon INJECTIONS
6.714
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. 2006 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
?
WHAT WE ASK FOR IS….
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