As a reminder of what mattered most to our readers in 2019, here are the 10 most-read articles of last year, with a brief description of what made them relevant to those with angioedema and their loved ones.
A study published in the journal Archives of Rheumatology has found that people with systemic lupus erythematosus (SLE), the most common form of lupus, were approximately two times more likely to develop angioedema at some point in their lives, compared to those who did not have SLE. Researchers also reported that women, African-Americans, and younger people seemed to be particularly susceptible to the disease.
BioCryst Pharmaceuticals submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) in December, requesting the approval of berotralstat (BCX7353), a selective oral inhibitor of plasma kallikrein, as a preventive treatment for swelling attacks in people with hereditary angioedema (HAE).
The request was supported by data from two ongoing clinical trials: the APeX-2 Phase 3 trial (NCT03485911) and the long-term, open-label extension, APeX-S Phase 2/3 study (NCT03472040). Latest findings from both trials demonstrated that when administered at a daily oral dose of 150 mg, berotralstat reduced HAE attack rates from three to one or fewer per month. A joint analysis of the studies, incorporating data from 342 patients, also showed that treatment was safe and well-tolerated.
A pooled analysis of data from three clinical trials found that Firazyr (icatibant acetate), an approved treatment for HAE attacks marketed by Takeda, did not seem to be superior to a placebo or other types of therapies to treat bouts of swelling caused by angiotensin-converting enzyme (ACE) inhibitors. Findings were reported in a study published in the Journal of Clinical Pharmacy and Therapeutics.
Daily treatment with berotralstat was found to be safe, well-tolerated, and to reduce the frequency of swelling attacks in patients with HAE type 1 and 2. According to data from APeX-2, when administered at a dose of 110 mg and 150 mg, berotralstat lowered the rates of HAE attacks by 30% and 44%, respectively, compared to a placebo. The higher dose also led to a 70% or greater reduction in attack rates in half of the patients, compared to 15% of people given a placebo.
A study published in the The Journal of Allergy and Clinical Immunology: In Practice has found that people with HAE have a higher risk of developing non-alcoholic fatty liver disease — a condition where too much fat builds up in the liver — even before starting treatment with angioedema medications that are known to affect liver function, such as Danocrine (danazol).
Based on these observations, researchers recommended that those with HAE be closely monitored, so that fatty liver disease may be identified and treated in its early stages.
In another study published in the same journal, scientists described a new technique called allelic discrimination that is able to detect a specific mutation in the F12 gene (c.983C>A) that is relatively common among people with HAE.
According to investigators, this quick, accurate and cheap method might make HAE not caused by mutations in the SERPING1 gene easier to diagnose. They also proposed this technique should be used as a first-line genetic test for those suspected of having the disease.
A study described the case of a German family with four members who carried a rare genetic mutation in the plasminogen (PLG) gene (c.988A > G) that has recently been associated with HAE. All those carrying the mutation only started showing disease symptoms — which included swelling of the face, tongue, and larynx (vocal cords) — in adulthood, and all of them responded well to treatment with Firazyr.
Our third most-read story of the year reported that short-term use of Ruconest (conestat alfa), a recombinant human C1 inhibitor marketed by Pharming Group, as a preventive treatment can reduce the chances of swelling attacks triggered by medical and dental procedures or by stress in people with HAE.
When Ruconest was administered to 51 patients before a medical procedure or a stressful life situation, 88.6% of them remained attack-free for the following week. Ruconest is approved in the U.S. and EU to treat HAE attacks in adults and adolescents.
An approved inhibitor of plasma kallikrein, Takhzyro (lanadelumab) was found to reduce the number of swelling attacks in patients with HAE, even in early phases of treatment. An analysis of the HELP Phase 3 trial (NCT02586805) showed 300 mg of Takhzyro every two weeks reduced the mean number of monthly HAE attacks by 80%, reduced the frequency of severe HAE attacks, and increased the number of patients who remained attack-free for the first 69 days of treatment compared to a placebo.
Takhzyro, developed by Shire (now part of Takeda), is approved for routine prevention of recurrent swelling attacks in HAE patients in the U.S., Canada, and Europe, and has a recommended starting dose of 300 mg.
Our most-read article of 2019 reported data from an open-label, Phase 2 clinical trial (NCT01359969), where a single dose of Ruconest safely stopped almost all acute attacks (95.9%) in a group of 20 children with HAE. Its effect was also rapid, with children starting to feel relief within a median of one hour after treatment.
Ruconest was found to be safe and well-tolerated. No serious side effects or hypersensitivity reactions to the medication were reported, and none of the children discontinued treatment due to adverse events.
Angioedema News hopes that these news articles, along with our continued reporting throughout 2020, will help to educate, inform, and improve the lives of those living with angioedema, their family members, and caregivers.
We wish all our readers a happy 2020.
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