Worse PNH found for woman with COVID-19 despite Empaveli: Report
COVID-19 vaccine, virus trigger return of symptoms for patient, 29
A woman with paroxysmal nocturnal hemoglobinuria (PNH) that was being well-controlled with Empaveli (pegcetacoplan) saw her symptoms return after being vaccinated for COVID-19, and then again after contracting COVID-19 itself.
According to researchers, the patient experienced breakthrough hemolysis, or a reoccurrence of blood cell destruction, following a COVID-19 vaccination — and “significant exacerbations after her second COVID-19 vaccine and [subsequent] COVID-19 infection” — despite continuing on Empaveli treatment.
“Our case study suggests that the traditional [treatment with eculizumab, sold as Soliris, and ravulizumab, marketed as Ultomiris] and … [Empaveli] may leave PNH patients vulnerable to [breakthrough hemolysis] after COVID-19 vaccination and infection,” the team wrote.
Her case was described in the study, “Breakthrough Hemolysis Associated With COVID-19 Vaccination and Active COVID-19 Infection in a Patient With Paroxysmal Nocturnal Hemoglobinuria Maintained on Pegcetacoplan: A Case Report,” published in the journal Cureus.
Patient now needs blood transfusions despite Empaveli treatment
In PNH, abnormal activation of a part of the immune system called the complement system leads to the destruction of blood cells, called hemolysis, which drives disease symptoms.
There now are three approved treatments for PNH, all of which work by blocking complement activation: Soliris, Ultomiris, and Empaveli. These therapies can be effective for controlling symptoms; however, patients may still experience breakthrough hemolysis, where symptoms occur despite treatment.
In the last few years, there have been reports of PNH patients on Soliris or Ultomiris who experienced breakthrough hemolysis after being vaccinated for COVID-19. However, no such reports of breakthrough symptoms related to COVID-19 vaccination have been described for patients on Empaveli, which is a newer medication.
“Current literature supports a correlation between COVID-19 vaccination and hemolytic episodes in PNH patients treated with older therapies [Soliris and Ultomiris]. However, this is the first report of acute hemolysis associated with COVID-19 vaccinations and acute COVID-19 infection in a patient maintained on [Empaveli],” the scientists wrote.
The U.S.-based team detailed the case of a 29-year-old woman with PNH. She had first started to experience symptoms at age 20, and had been diagnosed with PNH about four years later, after initially being misdiagnosed with another disorder.
After getting the correct diagnosis, the patient started treatment with Soliris, which kept her symptoms under control for several years. However, in early 2021, her symptoms resurfaced despite treatment. She then started treatment with Empaveli. After that, her disease went into complete remission, with the woman showing no notable symptoms for several months.
In July 2021, she received her first dose of the Pfizer COVID-19 vaccine. Shortly after, she had a recurrence of PNH, with signs of active hemolysis, fatigue, and lethargy resurfacing. These symptoms were relatively short-lived and resolved about a week after she got her first vaccine dose.
The next month, she got her second dose of the vaccine, and again her symptoms recurred, with laboratory tests indicating blood cell destruction consistent with PNH disease activity. These symptoms eased, but did not disappear completely over the next months.
In early 2022, she tested positive for COVID-19. At the time, she was experiencing shortness of breath and chest pain, and had a substantial recurrence of PNH symptoms and related blood cell destruction. Although her symptoms related to COVID-19 infection have resolved, renewed PNH symptoms persisted after the infection.
As of mid-2022, the patient needed blood transfusions every two to three months, and was undergoing evaluation for a possible bone marrow transplant.
This is the first report of acute hemolysis associated with COVID-19 vaccinations and acute COVID-19 infection in a patient maintained on [Empaveli].
Her case shows that breakthrough hemolysis may occur in patients on any of the three available PNH treatments after a COVID-19 vaccination or infection, according to the researchers.
The team noted that it’s not entirely clear why vaccines or infection with COVID-19 might trigger a worsening of PNH symptoms — which, they say, highlights a need for more in-depth research to understand the connection and identify potential avenues for intervention.
“This data provides a basis for additional investigation to clarify the mechanism of interaction between the COVID-19 vaccine, the COVID-19 virus, and current PNH … therapies,” they wrote.
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