New asthma drug helps kids, but the price is steep – Consumer Health News
THURSDAY, December 9, 2021 (HealthDay News) – Children with hard-to-control asthma may be relieved by adding an injectable antibody to their standard treatment, according to a clinical trial.
The drug, called dupilumab (Dupixent), has been available for several years to treat stubborn asthma in adults and adolescents. Based on the new findings, the U.S. Food and Drug Administration recently gave it the green light for children ages 6 to 11.
The drug, which is injected every two weeks, has been shown to be effective in reducing severe asthma attacks and improving lung function in children over one year.
It’s not a replacement for standard inhaler drugs, the researchers said, and it’s only for some children.
“This is for patients for whom standard treatment does not meet their needs,” said lead researcher Dr. Leonard Bacharier, professor and chair of pediatrics at Vanderbilt University Medical Center in Nashville, Tenn.
Childhood asthma often responds well to standard “controller” drugs, Bacharier said. But, he added, a “substantial portion” of children continue to have asthma attacks, miss school and even end up in hospital with severe symptoms.
Dupilumab is one of many effective – and very expensive – monoclonal antibodies approved in recent years for the management of these severe asthma cases. Drugs are antibodies designed in the lab that target specific substances in the immune system that are involved in causing asthma symptoms.
People with asthma all have similar symptoms, including shortness of breath, cough, and chest tightness. But the disease actually has many forms, based on the underlying biology, said Dr Michael Wechsler, an asthma specialist who was not involved in the trial.
On a general level, Wechsler said, asthma is divided into two groups: type 2 and non-type 2. Type 2 is marked by a systemic allergic response that triggers inflammation. It is estimated that two-thirds of people with asthma suffer from type 2 inflammation, according to Wechsler, who heads the Cohen Family Asthma Institute at National Jewish Health in Denver.
The children in the current trial had type 2 inflammation, which was assessed by measuring certain markers in their blood.
“Dupilumab is very effective in the subset of asthma patients with type 2 inflammation,” said Wechsler.
The study – published on December 9 in the New England Journal of Medicine – was funded by the makers of Dupixent, Sanofi and Regeneron Pharmaceuticals.
It included 408 children aged 6 to 11 whose asthma was not controlled despite standard inhaler medications to prevent symptomatic attacks.
The children were randomized to add either dupilumab or a placebo to their usual treatment. The drug injections were given every two weeks – first by a health care provider, then at home if families were comfortable with it, Bacharier said.
Over one year, children taking the drug showed an approximately 60% reduction in severe asthma attacks, compared to the placebo group. About 78% had no severe asthma attack, compared to 59% of children who received placebo.
The drug also improved children’s overall lung function and reduced their need for oral steroids, which can interfere with bone growth and development.
Wechsler said children with severe asthma are at risk for abnormal lung development, which later in life can increase their risk for lung diseases like emphysema. It is therefore particularly important to improve asthma control in children of this age, he said.
A big obstacle in the real world, however, is the cost. Monoclonal antibody drugs are expensive, and dupilumab can cost as much as $ 45,000 per year.
Bacharier said insurers typically make coverage decisions on a case-by-case basis. Families should therefore have their child thoroughly assessed by a specialist to see if dupilumab, or another monoclonal antibody, is appropriate.
One limitation of the current study, Wechsler said, is that most of the patients were white – a hallmark of many clinical trials in medicine. Yet in the United States, black children have particularly high rates of asthma, including severe asthma. And in his own research, Wechsler found that black children may respond differently to standard asthma medications, compared to white children.
He said it’s likely the current findings will extend to black children as well – but ideally trials with more children of color should be done.
Bacharier agreed. “We really need studies on these under-represented populations to understand the clinical effectiveness of these treatments,” he said.
The American Academy of Allergy, Asthma & Immunology has more information on monoclonal antibodies for asthma.
SOURCES: Leonard Bacharier, MD, president, pediatrics and professor, pediatrics, allergy / immunology / pulmonary medicine, Vanderbilt University Medical Center, Nashville, Tenn. ; Michael Wechsler, MD, director, Cohen Family Asthma Institute and professor of medicine, National Jewish Health, Denver; New England Journal of Medicine, December 9, 2021