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Advances in HCV Treatment Volume 20 Issue 1 April/May  2012


Antivir Med. 2012;20(1):5-10 ©2012, IAS–USA

 Perspective

Advances in HCV Treatment Volume 20 Issue 1 April/May 2012 
Since 2007, the annual age-adjusted mortality rate in HIV disease in the United 
States has been surpassed by that of hepatitis C virus (HCV) disease,
reflecting  the continuing decline in HIV-related mortality and the continuing
increase in  HCV-related mortality.1 The prevalence of HCV-related cirrhosis is
projected to  continue to increase until it reaches a peak around 2020,
reflecting what is  commonly a 20- to 40-year period between HCV acquisition and
the later-stage  manifestations of cirrhosis, end-stage liver disease, and liver
cancer. These  projections assumed no changes in our ability to treat HCV
infection. The rate  of sustained virologic response (SVR; ie, absence of HCV
RNA in blood for 6  months after the end of treatment) with what has been the
standard treatment of  peginterferon alfa plus ribavirin is approximately 40% in
patients with HCV  genotype 1 Dr Thomas is Professor of Medicine and Director of
the Division of  Infectious Diseases at The Johns Hopkins University in
Baltimore, MD. Since  2007, the annual age-adjusted mortality rate in hepatitis
C virus (HCV)  infection in the United States has been greater than that in HIV
disease,  reflecting the continuing decline in HIV-related mortality and the
continuing  increase in HCV-related mortality. The approval of 2 new
direct-acting  antivirals within the past year, as well as the promise offered
by numerous  other direct-acting agents in development, provides hope that we
will be able to  markedly improve our ability to cure HCV disease. The addition
of a protease  inhibitor (PI) to what has been the standard HCV therapy of
peginterferon alfa  and ribavirin dramatically improves sustained virologic
response rates in  treatment-naive patients with genotype 1 infection. Similar
results have been  observed in some treatment-experienced patients in whom prior
peginterferon  alfa/ribavirin therapy has failed. The use of these new agents
has also  permitted response-guided therapy, wherein early sustained virologic
response to  treatment allows for a shortened treatment duration. However, these
new PIs add  cost and adverse effects to HCV therapy. Boceprevir is associated
with increased  risk of anemia and dysgeusia, and telaprevir is associated with
increased risk  of anemia and skin and gastrointestinal adverse effects. Early
studies indicate  that the addition of PIs results in high response rates in
patients with HCV/HIV  coinfection. Other studies suggest that combinations of
PIs and other  direct-acting antivirals may ultimately permit cure when used in
interferon  sparing regimens. This article summarizes a presentation by David L.
Thomas, MD,  MPH, at the IAS–USA live continuing medical education course held
in New York  City in October 2011.


Since 2007, the annual age-adjusted mortality rate in HIV 
disease in the United States has been surpassed by that of hepatitis C virus 
(HCV) disease, reflecting the continuing decline in HIV-related mortality and 
the continuing increase in HCV-related mortality.1 The prevalence of
HCV-related  cirrhosis is projected to continue to increase until it reaches a
peak around  2020, reflecting what is commonly a 20- to 40-year period between
HCV  acquisition and the later-stage manifestations of cirrhosis, end-stage
liver  disease, and liver cancer. These projections assumed no changes in our
ability  to treat HCV infection.

The rate of sustained virologic response (SVR; ie, absence 
of HCV RNA in blood for 6 months after the end of treatment) with what has been 
the standard treatment of peginterferon alfa plus ribavirin is approximately
40%  in patients with HCV genotype 1 infection, the predominant type of
infection in  the United States. The rate is less than 30% in HIV/HCV-coinfected
patients with  HCV genotype 1. However, the past year has brought the approval
of 2 new drugs  for treating HCV infection—the HCV protease inhibitors
boceprevir and  telaprevir— and numerous new drugs are in advanced stages of
development. It is  hoped that these new weapons will allow us to improve the
projections for HCV  disease outcomes. 

Treatment Outcomes With Telaprevir and  Boceprevir Treatment-Naive Patients 

In the trial supporting approval of telaprevir, more than 
1000 treatment-naive patients with HCV genotype 1 infection were randomly 
assigned to receive telaprevir for 8 weeks or 12 weeks plus concurrent standard 
peginterferon alfa/ribavirin therapy for up to 48 weeks, or peginterferon 
alfa/ribavirin alone for 48 weeks.2 Patients receiving telaprevir who achieved
a  virologic response that was sustained between weeks 8 and 12 were further 
randomly assigned to stop peginterferon alfa/ribavirin after week 24 or
continue  for the full 48 weeks. Overall, cure (ie, SVR) was achieved in 69% of
patients  receiving 8 weeks of telaprevir and 75% of those receiving 12 weeks of 
telaprevir, compared with 44% of those receiving peginterferon alfa/ribavirin 
alone. In black patients, who are known to have lower rates of response to 
peginterferon alfa/ribavirin, SVR rates were 25% with standard therapy, versus 
58% and 62% with 8 weeks and 12 weeks of telaprevir, respectively. Among 
nonblack patients, SVR rates were 48% with peginterferon alfa/ribavirin, 
compared with 73% and 79% with the addition of telaprevir for 8 weeks and 12 
weeks, respectively.

Patients who stopped therapy at 24 weeks after an early 
response to telaprevir-containing therapy had outcomes similar to those who 
continued to receive peginterferon alfa/ribavirin for the full 48-week course. 
The 12-week course of telaprevir was approved in 2011 by the US Food and Drug 
Administration (FDA) for use in combination with peginterferon alfa/ ribavirin, 
 as was the shortened treatment duration in patients with early sustained  response to treatment.

With regard to the ability to abbreviate therapy based on 
early response to treatment, Sherman and colleagues performed a study in 
treatment-naive, genotype 1–infected patients.3 Patients who achieved early 
rapid virologic response (eRVR; defined as undetectable HCV RNA at week 4 and 
week 12) with telaprevir plus peginterferon alfa/ribavirin therapy were
randomly  assigned to continue receiving peginterferon alfa/ribavirin for the
full 48  weeks or to stop treatment after a total of 24 weeks. The overall SVR
rate was  72%, with 65% of the total of 540 patients achieving eRVR. SVR rates
were 92%  among those stopping treatment after 24 weeks and 88% among those
receiving 48  weeks of treatment. Among those who did not achieve eRVR, the SVR
rate was  64%.

In the pivotal boceprevir trial, approximately 1100 
treatment-naive patients with genotype 1 infection received a lead-in of 
peginterferon alfa/ ribavirin for 4 weeks.4 This was followed by either 
continuation of peginterferon alfa/ribavirin treatment for 44 weeks (total of
48  weeks); addition of boceprevir for 44 weeks (fixed-duration group); or
addition  of boceprevir for 24 weeks followed by treatment discontinuation if
virus was  undetectable from 8 weeks to 24 weeks or treatment continuation with 
peginterferon alfa/ribavirin alone for 20 weeks if virus was detectable 
(responseguided therapy group). Overall, SVR rates were 63% in the 
response-guided therapy boceprevir group and 66% in the fixed-duration 
boceprevir group, compared with 38% in the peginterferon alfa/ribavirin 
treatment group. SVR rates were improved with the addition of boceprevir in
black patients (42% in the response-guided therapy group and 53% in the 
fixed-duration group vs 23% in the standard treatment group) and nonblack 
patients (67% and 69% vs 41%, respectively). Boceprevir was approved by the FDA 
in 2011 for use in combination with peginterferon alfa/ribavirin, including a 
shortened response-guided course of therapy in treatment-naive patients.

 Treatment-Experienced  Patients 

Telaprevir and boceprevir have each been shown to achieve 
cure in a substantial proportion of HCV-infected patients in whom prior 
peginterferon alfa/ribavirin therapy had failed. In a study of more than 600 
treatmentexperienced patients, Zeuzem and colleagues found SVR rates of 64%
with  the combination of 12 weeks of telaprevir plus 48 weeks of peginterferon 
alfa/ribavirin; 66% with a 4-week lead-in regimen of peginterferon alfa/ 
ribavirin followed by 12 weeks of telaprevir and 44 weeks of peginterferon 
alfa/ribavirin; and 17% with retreatment with 48 weeks of peginterferon 
alfa/ribavirin.5 Among patients with relapse (ie, those who relapsed after 
having undetectable virus at the end of prior treatment) SVR rates were 83%, 
88%, and 24%, respectively. Among those who had shown a partial virologic 
response to prior treatment, SVR rates were 59%, 54%, and 15%, respectively.
For  those with no virologic response to prior treatment (null responders), SVR
rates  were 29%, 33%, and 5%, respectively.

In a trial in approximately 400 treatment-experienced 
patients conducted by Bacon and colleagues, overall SVR rates were 66% in 
patients receiving boceprevir and 48 weeks of peginterferon alfa/ribavirin, 59% 
in those receiving boceprevir with response-guided therapy, and 21% in those 
receiving standard peginterferon alfa/ribavirin.6 SVR rates were 75%, 69%, and 
29%, respectively, among patients who had relapsed after prior therapy and 52%, 
40%, and 7%, respectively, among those who had partial response to prior treatment.

Increased Toxic Effects With  Addition of Telaprevir or Boceprevir \

 Jacobson and colleagues reported that adverse events
  occurred more frequently in telaprevir-containing study arms than in the
  peginterferon alfa/ribavirin alone arm. Adverse effects included pruritus
  (45%-50% with telaprevir vs 36% with peginterferon alfa/ribavirin), nausea
  (40%-43% vs 31%), rash (35%-37% vs 24%), anemia (37%-39% vs 19%), and diarrhea
  (28%-32% vs 22%). In the boceprevir trial conducted by Poordad and colleagues,
  anemia (49% in the boceprevir group vs 29% in standard treatment group) and
  dysgeusia (37%-43% vs 18%, respectively) were more common in
  boceprevircontaining study arms.

Resistance to HCV Protease Inhibitors 

Because neither interferon alfa nor ribavirin is a 
direct-acting antiviral agent, viral resistance is a new phenomenon in HCV 
treatment. Resistance to the protease inhibitors (PIs) telaprevir and
boceprevir  is detected in approximately 50% of patients in whom therapy
containing these  agents fails.7,8 To date, there is no evidence that resistant
variants have  greater replicative fitness or pathogenicity than wild-type
virus. As has been  observed with HIV, there is a return to predominance of
wild-type virus  generally within 18 months of stopping HCV PI treatment.9
However, unlike HIV,  there is no biologic basis for archiving of PI-resistant
variants in the body. 

Table 1. Sustained
Virologic Response Rates According to Patient and  Disease Characteristics in
Treatment-Naive Patients Receiving Telaprevir or  Boceprevir plus Peginterferon
Alfa/Ribavirin Compared with Peginterferon  Alfa/Ribavirin Alone



Picture
TPV 12 indicates patients receiving  telaprevir for 12 weeks; Peg/RBV 24-48, peginterferon alfa and ribavirin for 24
  to 48 weeks; Peg/RBV 48, peginterferon alfa and ribavirin for 48 weeks; BOC 44, boceprevir for 44 weeks. Adapted from Jacobson et al2 and Poordad et  al.4



The long-term consequences of selecting for HCV PI resistance are unclear at this time.
Investigations are currently underway on whether emergence of resistance will result
in poorer response to subsequent treatment containing a PI.

There are no convincing data thus far that baseline 
 resistance to HCV PIs affects response to treatment. Thus, although there is a 
commercially available assay for testing for HCV resistance, for now there is
no  indication for testing to guide immediate treatment decisions. However, it
may  be prudent to document resistant variants in case the information becomes
useful  in the future.

More Potent Therapy Reduces  Predictive Value of Some Risk Factors for Poor Response 

More potent anti-HCV therapy reduces the value of some of 
the traditional factors predictive of poor response to peginterferon 
alfa/ribavirin therapy. This is a good thing, however, because the loss of 
predictive value is the result of higher cure rates in subgroups of patients 
with traditionally greater risk of poor response. Most notable is the
diminished  effect of higher HCV viral load in predicting poorer treatment
outcome with  peginterferon alfa/ribavirin (see Table 1). For example, in the
pivotal  telaprevir trial, SVR rates were similar among telaprevir-receiving
patients  with baseline HCV RNA viral load 800,000 IU/mL or higher and those
with viral  load less than 800,000 IU/mL (74% and 78%, respectively).2 The SVR
rate in those  with elevated viral load receiving telaprevir represents a
striking improvement  over the response rate among patients with high viral load
receiving  peginterferon alfa/ ribavirin alone (36%). In the pivotal boceprevir
trial, the  SVR rate among boceprevir recipients with elevated baseline viral
load was 63%,  compared with 33% among patients with elevated baseline viral
load receiving  peginterferon alfa/ribavirin alone.4

As noted previously, black race is also a risk factor for 
poorer response to peginterferon alfa/ribavirin. The difference in the
frequency  of the unfavorable interleukin-28B genotype explains about half of
the  difference in treatment response between black and nonblack patients.
Although  there was still a difference in SVR rates between black patients and
white  patients receiving telaprevir (62% and 75%, respectively), the SVR rate
in black  patients represents a striking improvement over that achieved with
peginterferon  alfa/ribavirin alone (25%).2 Similarly, black patients receiving
boceprevir had  a lower SVR rate than white patients, but the high cure rate in
black patients  receiving boceprevir compared with those receiving peginterferon
alfa/ribavirin  alone is another striking improvement—53% versus 23%,
respectively.4 Some of the  differences observed between the telaprevir and
boceprevir studies, with regard  to response rates in patient subgroups, likely
reflect the fact that the post  hoc analyses were performed in different patient
populations.

Comparison of Telaprevir and Boceprevir-Containing  Regimens 

Table 2 provides an overview of characteristics of HCV 
treatment with telaprevir- and boceprevir-containing regimens. A 4-week lead-in 
period with peginterferon alfa/ribavirin is recommended before adding
boceprevir  and no lead-in is recommended for patients receiving telaprevir,8,10
reflecting  the way the drugs were developed in phase II and, especially, phase
III studies.  Boceprevir is administered for 24 weeks or 44 weeks in
treatment-naive patients  and for 32 weeks or 44 weeks in treatment-experienced
patients, depending on  early virologic response, whereas telaprevir is
administered for 12 weeks in  both treatment-naive and treatment–experienced
patients.

Table 2. Selected  Characteristics of Boceprevir and  Telaprevir

Picture
*RGT indicates response-guided therapy; PI, protease inhibitor; eRVR, early rapid virologic response. RGT is
  not recommended in patients with cirrhosis or HIV coinfection.

 Response-guided therapy is not recommended in patients 
with cirrhosis or in HIV-coinfected patients. Responseguided therapy in 
HIV-seronegative, noncirrhotic, treatment-naive patients is permitted based on 
an HCV RNAnegative response during weeks 8 to 24 with boceprevir treatment and 
at weeks 4 and 12 with telaprevir treatment. Based on clinical trial data, it
is  estimated that 44% of treatment-naive patients receiving boceprevir and 58%
to  65% of treatment-naive patients receiving telaprevir are eligible for 
response-guided therapy. The total duration of anti-HCV treatment in treatment- 
naive patients, depending on presence or absence of early virologic response,
is  28 weeks or 48 weeks for boceprevir, and 24 weeks or 48 weeks for 
telaprevir.

Response-guided therapy in treatment- experienced patients 
is not recommended for patients receiving boceprevir who were null responders
to  prior treatment or for patients receiving telaprevir who were partial or
null  responders. For treatment-experienced patients receiving boceprevir, total 
anti-HCV treatment duration is 36 weeks (for those with eRVR) or 48 weeks.
Total  treatment duration is 24 weeks or 48 weeks for patients receiving
telaprevir.  Anti-HCV therapy with boceprevir should be stopped due to futility
if HCV RNA  level is greater than 100 IU/mL at week 12 or if there is detectable
HCV RNA at  week 24. The recommended stopping rule for telaprevir- containing
therapy is a  viral load of greater than 1000 IU/mL at week 4 or 12, or
detectable virus at  week 24.

As noted previously, there are added adverse effects with 
the addition of either of the PIs to peginterferon alfa/ ribavirin. There is an 
increased risk of anemia with boceprevir compared with peginterferon 
alfa/ribavirin therapy alone, and telaprevir is associated with increased risk 
of anemia and skin and gastrointestinal side effects. Pill burdens differ 
between the two treatments, with boceprevir requiring four 200 mg pills every 8 
hours and telaprevir requiring two 375 mg pills every 8 hours. There is also a 
difference in food requirements: boceprevir needs to be taken with some food, 
whereas each dose of telaprevir needs to be taken with a meal containing at 
least 20 g of fat.

The addition of a new agent to HCV treatment regimens 
increases cost as well as cure rates. A 48-week course of peginterferon 
alfa/ribavirin costs approximately $38,000. Full courses of telaprevir (12 
weeks) and boceprevir (up to 44 weeks) cost approximately $50,000.

Ongoing  Studies of HCV PIs 

Patients with HCV infection in whom PI treatment has yet 
to be fully evaluated are those with more advanced disease (eg, patients with 
decompensated cirrhosis and transplant patients), those with HBV coinfection, 
and those with HIV coinfection. In addition, safety and efficacy of these drugs 
have not been established in patients with HCV genotype 2 or 3 infection. 
Genotype 2 infection is responsive to peginterferon alfa/ribavirin in most 
patients, and there is some indication that cure rates are improved with the 
addition of a PI. Genotype 3 infection is more difficult to treat in many
cases,  and there is some evidence indicating that response rates are not
improved with  the addition of a PI.

 Studies in HIV Coinfection 

In a small study by Sulkowski and colleagues, patients 
with HCV/HIV coinfection received a full 48-week course of anti-HCV therapy
with  telaprevir plus peginterferon alfa/ribavirin or peginterferon
alfa/ribavirin  alone with or without antiretroviral therapy.11 The group
receiving  peginterferon alfa/ribavirin without antiretroviral therapy included
patients  with high CD4+ cell counts who did not meet current guidelines for
initiation of  antiretroviral therapy. Patients who received antiretroviral
therapy took  efavirenz/tenofovir/emtricitabine, or ritonavir-boosted atazanavir
with  tenofovir/emtricitabine or tenofovir/lamivudine. Patients who received the 
efavirenz-containing regimen received an additional telaprevir pill with each 
dose to compensate for lowered blood levels due to pharmacokinetic interaction 
with efavirenz. As shown in Figure 1, the telaprevir-containing regimen
markedly  improved week 4 and week 12 virologic responses in patients receiving
and not  receiving antiretroviral therapy. These promising findings need to be
confirmed  in larger studies.

Picture
Figure 1. Hepatitis C virus (HCV) virologic responses to  telaprevir-containing therapy at week 4
(left) and week 12 (right) in patients  with HCV/HIV coinfection, according to
antiretroviral regimen. Numerals in bars  show total number of patients in
treatment group. EFV indicates  efavirenz/tenofovir/emtricitabine; ATV/r,
ritonavir-boosted atazanavir with  tenofovir/emtricitabine or
tenofovir/lamivudine. Adapted from Sulkowski et  al.11


 A phase II trial of boceprevir with peginterferon 
alfa/ribavirin in HIV/ HCV-coinfected patients is ongoing. A total of 99 
coinfected patients with stable HIV disease are being treated with a lead-in of 
4 weeks of peginterferon alfa plus weight-based ribavirin, then randomly 
assigned to add boceprevir (800 mg every 7-9 hours) or placebo for an
additional  44 weeks. Subjects were allowed into the study if they were on
raltegravir or  ritonavirboosted PIs. Baseline HCV RNA level was above 800,000
IU/mL for 88% of  subjects; 82% were white, and 5% had cirrhosis.12

The proportion of patients with undetectable HCV RNA at 
week 8 was higher in the group receiving boceprevir (24 of 64 [37.5%] with 
undetectable HCV RNA) than in the group receiving placebo (5 of 34 [14.7%]). At 
week 24, HCV RNA was undetectable in 43 of 61 patients (70.5%) in the
boceprevir  arm and undetectable in 11 of 32 (34.4%) in the placebo arm.
Treatment was  discontinued in 3 (9%) and 9 (14%) of the patients in the placebo
and boceprevir  arms, respectively, because of adverse events.

Updates on the trials described above were presented at 
the 19th Conference on Retroviruses and Opportunistic Infections in March 2012. 
In the telaprevir trial in HIV/HCV-coinfected patients, 28 of 38 patients (74%) 
receiving telaprevir plus peginterferon alfa/ribavirin had undetectable levels 
of HCV RNA at week 24 (end of treatment), compared with 12 of 22 patients (55%) 
in the peginterferon alfa/ribavirin–only control group.13 Twelve weeks after 
stopping therapy, all 28 of the 38 (74%) who had undetectable levels of HCV RNA 
at the end of telaprevir treatment had sustained virologic response. In the 
control group, 10 of 22 patients (45%) had sustained virologic response.

In the boceprevir trial, 39 of 61 coinfected patients 
(63.9%) receiving boceprevir plus peginterferon alfa/ribavirin had undetectable 
HCV RNA at week 48 (end of treatment), compared with 10 of 34 (29.4%) receiving 
peginterferon/ alfa alone.14 Twelve weeks after stopping therapy, 37 of 61 
patients (60.7%) who had received boceprevir had sustained virologic response, 
compared with 9 of 34 (26.5%) in the peginterferon alfa/ribavirin–only group. 
 
These results in coinfected patients are notable because 
 in both studies, virologic response was substantially better than with 
 interferon alfa/ribavirin alone. Virologic response rates were also nearly as 
high as those in monoinfected patients.

 Potential  for Cure Without Interferon Alfa

Peginterferon alfa therapy is associated with considerable 
toxicity, and there is intense interest in developing treatments that would 
spare patients from the rigors of such therapy. An example of studies assessing 
this possibility was reported by Lok and colleagues.15 Patients who were prior 
null responders to peginterferon alfa/ribavirin therapy received a combination 
of an HCV PI and an HCV nonstructural protein 5A (NS5A) inhibitor (which is 
active at different steps of the viral replication process than PIs), with or 
without peginterferon alfa/ribavirin. 

Four of 11 patients receiving the PI and NS5A inhibitors 
without peginterferon alfa/ribavirin had viral loads that fell below the limit 
of quantitation at week 12 and remained undetectable after stopping therapy, 
showing in principle that cure is achievable without interferon alfa therapy. 
Six of the 11 patients exhibited viral breakthrough. It is also noteworthy that 
all 10 patients receiving the 2 direct-acting antivirals in combination with 
peginterferon alfa/ribavirin had undetectable virus at week 12, a remarkable 
outcome of treatment in prior null responders. There is considerable excitement 
over what might be achieved with multidrug combinations of the numerous 
investigational direct-acting agents.

Although formal guidelines for treatment of 
HIV/HCV-coinfected persons are being planned, at this time treatment should be 
prioritized for those with advanced liver fibrosis (cirrhosis and bridging 
fibrosis). When possible, coinfected patients should be enrolled in clinical 
trials to expand the available information on optimal HCV treatments in that  setting.

Summary 
The current era in HCV treatment is reminiscent of the 
transformation of HIV treatment that occurred in the mid-1990s. With the new
HCV  treatments, cure and complications occur more frequently. We can make smart 
applications of the treatments available to us right now in some patients, and 
we await tomorrow’s treatments for other patients. As with the first wave of
HIV  medications in the potent antiretroviral era, the new HCV drugs offer huge 
advantages but also present substantial challenges. 

Presented by Dr Thomas in October  2011. First draft prepared from transcripts by Matthew Stenger.
Reviewed and edited by Dr Thomas in February 2012.

 Dr Thomas has received grants and  research support from Gilead Sciences, Inc,
and Merck & Co, Inc. He has served as a consultant to Merck & Co, Inc.



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