DAAs Efficacious, Tolerable in HCV Patients with Inherited Blood Disorders


Patients with hematological disorders like sickle cell disease or β-thalassemia are at high risk for HCV infection as a result of frequent transfusions.

Isaac Ruiz, MD, PhD

Isaac Ruiz, MD, PhD

Direct-acting antiviral (DAA) drugs were deemed well-tolerated and efficacious in patients with chronic hepatitis C virus (HCV) infection and inherited blood disorders, according to findings from a new study.

Patients with inherited blood disorders, especially those with sickle cell disease or β-thalassemia, face high risk of HCV infection and associated morbidity. Many of these patients become infected from the multiple transfusions required to address the complications with their disease.

However, few clinical trials have reported the impact and effect of DAAs within this high-risk population.

Therefore, a team led by Isaac Ruiz, MD, PhD, Department of Hepatology, Henri Mondor University Hospital, France, conducted a real-world prospective cohort study that assessed the efficacy and safety of DAA-based regimens in this often difficult-to-treat patient population.

They evaluated a total of 27 patients—25 with sickle cell disease, 1 with β-thalassemia, and 1 with D-punjab—who were treated for HCV between 2013-2017. Additionally, 3 had cirrhosis.

Of this cohort, 7 were male, and the mean age was 49 years old.

Patients were treated with sofosbuvir in combination with ribavirin, daclatasvir, ledipasvir, or velpatasvir or with grazoprevir/ebasvir for 8 or 12 weeks. If treatment failed, then the investigators proceeded with in-vitro assessment of resistance-associated substitutions (RASs) and full-length genome sequence analysis through deep sequencing.

Data was collected at baseline, and weeks 4, 8, and 12 of therapy, as well as weeks 4 and 12 post-treatment.

The primary endpoint sought by the investigators was undetectable HCV RNA (<12 IU/mL) at the end of their treatment reigmen.

Thus, results showed that 26 out of the 27 patients were HCV undetectable following treatment course, and 25 achieved sustained virological response at week 12 of post-treatment.

“One 59 years-old female patient infected with HCV genotype 2 receiving sofosbuvir and daclatasvir stopped treatment at 18 days of administration due to adverse events that were not ascribed to HCV drugs,” Ruiz and colleagues noted.

Another 49-year old treatment-naïve patient without cirrhosis and diagnosed with HCV genotype 2 relapsed 4 weeks following a treatment regimen of sofobuvir plus daclatasvir. However, they were retreated with sofosbuvir/velpatasvir for 12 weeks and once more achieved sustained virological response.

Nonetheless, treatment was considered safe and well-tolerated. Of the total, 4 reported clinical adverse patients, which included headaches, asthenia, and myalgia.

Furthermore, there were no observed changes in hemoglobin levels during treatment. The only exception was a patient treated with 400 mg of ribavirin who experienced a drop 11.0 g/dL at baseline to 8.7 g/dL at end of treatment course, and 8.2 g/dL at sustained virological level.

The investigators reported no other biological abnormalities.

In terms of limitations, they acknowledged the small sample size of the population as well as the fact that treatment regimens were decided at the physician’s discretion over a long period of time. They noted the rapidly changing HCV treatment paradigms within this time frame.

Despite these limitations, they affirmed the high efficacy and safety of 8 or 12 week DAA regimens for these patients.

“HCV treatment should be prioritized in this thus far undertreated population, as universal treatment access is required to achieve the WHO’s goal to eliminate HCV infection as a public health threat,” Ruiz and his team concluded.

The study, “Real-world efficacy and safety of direct-acting antiviral drugs in patients with chronic hepatitis C and inherited blood disorders,” was published online in the European Journal of Gastroenterology and Hepatology.

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