Removing a child’s tonsils and adenoids, or adenotonsillectomy, currently is the primary therapy for childhood obstructive sleep apnea syndrome (OSAS), but many parents remain wary about surgery and would welcome an alternative approach. Researchers from the Sleep Center at The Children’s Hospital of Philadelphia are set to launch the “Steroids for Pediatric Research in Kids (SPARK)” trial in September that will investigate the use of nasal corticosteroids as a possible treatment for OSAS.
OSAS is extremely common, according to Carole L. Marcus, MBBCh, director of CHOP’s Sleep Center. It is seen in 2 to 4 percent of children, but she maintains that it is greatly underdiagnosed. Usually, a blockage from enlarged tonsils or adenoids cause obstruction in breathing, called apneas and hypopneas, that repeatedly disrupt a child’s sleep. If OSAS is left untreated, it can result in significant complications, ranging from behavioral problems and sleepiness in mild-to-moderate cases, and cognitive abnormalities, high blood pressure, and growth disturbances in more severe cases.
One of the lessons that Dr. Marcus learned from being a co-investigator of the Childhood Adenotonsillectomy Study for Children With OSAS (CHAT Study) — a large, randomized, multicenter control study that compared the effectiveness of surgery vs. watchful waiting for OSAS — was that better treatments and more therapeutic choices for families are needed. While adenotonsillectomy is relatively safe, about 3 percent of children will have significant hemorrhage postoperatively, and other complications can occur.
“Most families were hoping to hold off on surgery because they were scared,” Dr. Marcus said.
Over the past few years, several small, short-term studies have reported on nasal steroids as an alternative treatment for pediatric OSAS. They showed overall benefit for mild OSAS cases but did not study more severe cases; in addition, the results demonstrated tremendous individual variability. The American Academy of Pediatrics clinical practice guidelines for the diagnosis and management of OSAS issued in 2012, which Dr. Marcus chaired, included nasal steroids as a treatment option, but there was not enough evidence to give a strong recommendation.
Dr. Marcus and colleagues are launching the SPARK study to increase knowledge about the effect of nasal steroids and to identify which subgroups of patients with OSAS are most likely to benefit. For example, the research team will determine if children with asthma and/or atopy respond better to nasal steroids. Another subgroup they will focus on is African-American children because strong data from CHAT and other studies have shown that they have more severe OSAS and do not respond as well to surgery. Sophisticated genetic tests included in the SPARK study will help the investigators to further characterize responders vs. non-responders.
The double-blind, randomized control trial also will assess nasal steroids’ duration of action and possible side effects, which for a small minority of children include growth problems, ocular abnormalities, and adrenal suppression.
“Does the disease recur if you stop nasal steroids?” Dr. Marcus asked. “Is it cured forever? Do you keep doing sleep studies? If kids are on the steroids long-term, are you likely to see complications?”
In order to answer those questions, the study is designed to randomize children to receive either nasal steroids or placebo for three months. After three months, those in the nasal steroid arm of the study will be re-randomized to either ongoing steroids or placebo for another nine months.
The multidisciplinary study team will include investigators from general pediatrics and several specialty areas including sleep medicine, endocrinology, ophthalmology, allergy, and otolaryngology. They aim to recruit about 300 patients, with a goal of randomizing 156 participants, from these diverse practice settings.
“I’m hoping we’ll find a nonsurgical alternative for some children but that it also will lead to more personalized medicine,” Dr. Marcus said. “One treatment does not fit all.”
The National Heart, Lung, and Blood Institute is funding the research project.
For more information on the SPARK study, contact research coordinator Ruth Bradford.