AHP patients report fewer symptoms, better life with Givlaari
Interview study finds patients in US, Europe 'very satisfied' with therapy
Patients are very satisfied with Givlaari (givosiran), the only approved treatment that targets the cause of acute hepatic porphyria (AHP) to help reduce attacks, according to a new study based on interviews with more than 20 people with AHP in the U.S. and Europe.
Indeed, these AHP patients — who previously took part in clinical trials of the therapy and then continued receiving treatment — reported experiencing fewer symptoms and having a better quality of life.
“All participants reported being ‘very satisfied’ with [Givlaari] treatment,” the researchers wrote. “Participants described a return to normality, including increased ability to care for children and other family members … work, and attend school, as well as resumption of social activities … and physical activities … that had become difficult or impossible to perform.”
One patient reported that, after Givlaari, “I can swim and do all of the things that I did pre-porphyria.”
The study, “Patient experience with acute hepatic porphyria before and after long-term givosiran treatment in a qualitative interview study,” was published in the journal Molecular Genetics and Metabolism Reports. It was funded by Alnylam Pharmaceuticals, which markets Givlaari.
Nearly all patients had intense abdominal pain, fatigue before Givlaari
Porphyria occurs when the body is missing one of the enzymes needed to produce a molecule called heme. This enzyme deficiency causes heme intermediates to go unused and build up to toxic levels in the body. In AHP, they build up in the liver, causing a range of symptoms.
Given by a subcutatneous, or under-the-skin, injection once per month, Givlaari is a small interfering RNA that’s approved for the treatment of AHP in adults. It works by reducing the levels of an enzyme involved in an earlier step of the production of heme, preventing toxic building blocks from accumulating in the liver.
In this study, the team of researchers in 2022 interviewed 21 people with AHP who had been treated with Givlaari for about four to five years. All had previously participated in one of two clinical trials of the therapy and were continuing to receive treatment through an expanded access program or commercial drug use. The interviews explored how the treatment impacted the patients’ symptoms, and their lives overall.
Before starting treatment with Givlaari in the trials, nearly all patients (95%) said they had experienced intense abdominal pain and fatigue. These symptoms disrupted their daily lives, including work or school and relationships.
After starting treatment, all evaluable patients reported improvements in key symptoms, such as abdominal pain, fatigue, and nausea. All 21 patients also stated they were very satisfied with Givlaari, with the therapy’s benefits exceeding expectations in some cases.
“It’s been great for my life,” one patient said.
Another reported: “My hope was that it would significantly decrease my symptoms to the point that I would be able to function a little bit more. I had never expected to be able to get off of disability.”
Treatment led to lasting benefits for AHP patients, reports suggest
For another person on Givlaari, the treatment helped reduce hospitalization time.
“I don’t spend any time in the hospital anymore and I don’t spend weeks at a time in bed so that’s a huge improvement,” this patient said.
Treatment with Givlaari also reduced the need for opioids — often used by patients to manage pain. Nearly all of the individuals in the study (90%) used opioids before treatment. After starting Givlaari, 59% of those evaluable stopped using them completely, and 24% lowered their dose.
All evaluable patients reported having better relationships with family and loved ones, and 90% experienced improvements in work or school performance.
Participants reported meaningful improvements in AHP symptoms, increased [quality of life], and reduced opioid use with long-term monthly [Givlaari] treatment.
Some patients experienced complete relief from specific symptoms, including vomiting (73%), nausea (67%), and abdominal pain (42%). Younger patients, those recently diagnosed, or those with better health at the start of treatment were more likely to have complete relief, according to the researchers.
Those reports suggest that early treatment with Givlaari may lead to the best outcomes.
Patients who had been on preventive treatment with hemin before starting Givlaari often had more severe symptoms initially. However, these patients also experienced significant relief, with 75% reporting complete relief from abdominal pain compared with 18% of those who had not used hemin.
Overall, according to the researchers, these findings suggest that Givlaari offers lasting benefits for patients.
“Participants reported meaningful improvements in AHP symptoms, increased [quality of life], and reduced opioid use with long-term monthly [Givlaari] treatment,” the team wrote.
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