Ravulizumab is a drug that is an antibody treatment for PNH. It works in the same way as another PNH antibody drug, eculizumab, by binding to a key protein and preventing the immune system from breaking down red blood cells.
It was licenced as a treatment for adults with PNH, in Europe, in 2019, and in this 2022 study, Austin Kulasekararaj and colleagues assessed how patients taking the drug do in the long term. They combined the results from two large clinical trials, in which ravulizumab was pitted against a control treatment, in 600 PNH patients. The patients were followed over two years, and during that time they did well. Their levels of lactate dehydrogenase (LDH) became normal, indicating that their red blood cells were no longer being destroyed. Most of the patients (around 80%) no longer required red blood cell transfusions, there were very few serious side effects, and their levels of fatigue improved.
Ravulizumab (also known as Ultomiris) is called a second-generation treatment, because it came after eculizumab and was based on it. Both treatments are equally effective. The big difference between the two, however, is that ravulizumab has a longer half-life. This means it stays in the body for longer before being broken down. As a result, instead of being given once every two weeks, as happens with eculizumab, ravulizumab can be given intravenously once every eight weeks. Many patients prefer this, for obvious reasons, and now it’s up to patients and clinicians to decide which of the treatments to take.
Fitbits collect important information about life with PNH (2024)
Ravulizumab has been a game changer for people with PNH. It is given intravenously, once every two months. The antibody drug lessens the risk of blood clots, reduces fatigue, and improves the quality and length of life for those who take it, yet despite this, little is known about its effects on sleep, and physical and mental health.
In this 2024 study, 28 patients, all taking ravulizumab, were enrolled into a 32-week clinical trial called REVEAL. They completed weekly questionnaires about their experiences, and also wore a FitbitTM, which recorded their heart rate, steps and sleep patterns. When the trial was ended, their results were compared with the values for the US general population. There was very little difference between the two sets of measurements.
Elizabeth Griffiths and colleagues reported that levels of tiredness, sleep, and physical and mental health were similar between the PNH patients and the control group. This is good to know. The study also highlights the value of using smartwatches within clinical trials for PNH patients, where it can be used to help collect quality of life data.