Takhzyro safely prevents HAE attacks in real world in patients in Puerto Rico
New data show treatment benefits among people with Hispanic, Latino ethnicity
Written by |
For people in Puerto Rico with hereditary angioedema (HAE), long-term treatment with Takhzyro (lanadelumab) is safe and effective for preventing swelling attacks, new real-world data show.
Specifically, more than two years of treatment with the approved injection medication safely and effectively maintained low HAE attack rates among people of Hispanic or Latino ethnicity already on the therapy. Takhzyro use also was found to pronouncedly reduce monthly swelling attacks in newly treated HAE patients.
That’s according to data from a small number of patients enrolled at the Puerto Rico site of the Phase 4 EMPOWER clinical trial (NCT03845400), which evaluated the real-world long-term safety and efficacy of Takhzyro in more than 160 people with HAE in North America.
“Findings from the present analysis support the clinical benefits and safety of [Takhzyro] long-term [preventive] treatment in patients with HAE in Puerto Rico,” the researchers wrote.
The study, “Lanadelumab’s Enduring Effectiveness and Safety in the Management of Hereditary Angioedema for Patients from Puerto Rico: Data from EMPOWER,” was published in International Archives of Allergy and Immunology. It was sponsored by Takeda, which markets Takhzyro. One of the seven authors works for Takeda Pharmaceuticals USA, while four others are employed by Takeda Development Center Americas.
A chronic genetic disease, HAE is characterized by recurrent swelling attacks that can affect several parts of the body, including the abdomen, face, hands, and airways. These attacks are generally triggered by increased activity of the kallikrein enzyme and consequent excessive production of the bradykinin molecule.
This results in more permeable blood vessels, which allows fluid to leak into tissues and cause swelling.
Little real-world data on Takhzyro outcomes among Hispanic patients
Takhzyro is an antibody-based therapy designed to block kallikrein, thereby reducing bradykinin production and the frequency of swelling attacks. Given as a subcutaneous, or under-the-skin, injection, it is approved in the U.S., including Puerto Rico, and other regions for preventing swelling attacks in children and adults with HAE.
Clinical trials leading to the therapy’s approvals demonstrated that Takhzyro was similarly safe and effective among all patients, including those of Hispanic/Latino ethnicity; that is the largest ethnic group in Puerto Rico.
Nonetheless, “there is a paucity of real-world data on treatment outcomes in these patients,” the researchers wrote.
“Despite being a [U.S.] territory, residents of Puerto Rico face significant healthcare disparities compared with those on the mainland,” the researchers wrote. The team added that a previous study suggested “that patients with HAE in Puerto Rico report lower quality of life in both physical and mental domains when compared with their counterparts [on] the mainland.”
To learn more, the research team analyzed outcomes among nine people with HAE who enrolled in the EMPOWER study through its site in Puerto Rico. The patients’ median age was 55 — ranging from 36 to 73 years — and most (78%) were women. Slightly more than half (56%) had HAE type 1, the most common form of the disease.
Each of the participants was of Hispanic or Latino ethnicity; two-thirds were white individuals. Almost half (44%) had a family history of the disease.
All but one were already on Takhzyro. These eight participants had received at least four treatment doses before enrollment. They were on the therapy for a mean of 950.5 days, or about 2.6 years, in EMPOWER. For the remaining participant, who started the therapy in the trial, the mean treatment duration was 837 days, or about 2.3 years.
At the end of the study, eight participants, all already on Takhzyro, remained on the treatment.
1 newly treated patient saw 76% drop in monthly attack rates
Data showed that the individuals already on Takhzyro maintained a mean of 0.26 attacks per month throughout the study and experienced an average of eight attacks each. Most attacks (83%) were mild or moderate. Two of these participants were attack-free while on Takhzyro, and one was able to extend the dosing interval from every two weeks to every four weeks, the data showed.
The newly treated patient saw a 76% drop in monthly attack rates, from a mean of 1.99 before treatment to 0.47 during the study. This participant experienced 12 attacks overall, with 75% classified as mild or moderate.
Although this patient discontinued the treatment during the study due to insurance-related treatment access issues, the researchers noted that no HAE attacks were recorded between the last treatment dose and the end of data collection in the study.
[These findings] support the clinical benefits and safety of [Takhzyro] treatment [for preventing HAE swelling attacks in patients in Puerto Rico].
About three-quarters of attacks were treated with on-demand medication, including icatibant (sold as Firazyr, with generics available) and Ruconest (recombinant C1 esterase inhibitor, a protein that blocks kallikrein).
Overall, five adverse events were reported in four patients, including mild fatigue in one participant, which was considered related to the treatment. Other events were not treatment-related and included urinary tract infection, moderate back pain, and severe respiratory infection. Glaucoma, an eye condition, was also reported. None were deemed serious.
According to the researchers, these findings “support the clinical benefits and safety of [Takhzyro] treatment” for preventing HAE swelling attacks in patients in Puerto Rico.
The team noted that the number of included participants was small, and that a single patient from Puerto Rico was newly treated with Takhzyro. That may limit the generalizability of these findings, per the scientists.
However, these data “[represent] a considerable proportion (8.4%) of the known HAE population in Puerto Rico,” the researchers wrote.
