The main goal of clinical registries is to collect information about patients to better understand disease conditions and treatment options and to improve quality of care. At least three registries in otolaryngology have played important roles in steering treatment decisions and bolstering outcomes. ENTtoday spoke with three founders of these registries to discuss their journeys, successes, and barriers.
Explore This IssueNovember 2019
Recurrent Respiratory Papillomatosis
One of the longest running registries in otolaryngology has been the recurrent respiratory papillomatosis (RRP) registry, which was established in 1995.
Because RRP is a rare disorder—affecting four out of 100,000 children, with approximately 1,000 new pediatric cases in the United States back in 1995—it has been designated as an orphan disease, making it a good candidate for a registry, according to Craig Derkay, MD, Fine Endowed Professor and vice chairman in the department of otolaryngology at Eastern Virginia Medical School in Norfolk.
With orphan diseases, clinicians often have experience with only a small number of patients, which may not provide enough information to change the way the disorder is managed. “But if you aggregate everybody’s small experience together, then you may have enough information to really learn enough to change the treatment,” Dr. Derkay told ENTtoday.
With the help of the Centers for Disease Control and Prevention (CDC), Dr. Derkay created the national registry of approximately 700 patients with RRP. Based on the information gathered, Dr. Derkay and his colleagues were able to confirm that the majority of the children were firstborn, that their mothers were young and had a prolonged labor, and that the mothers had previously had multiple sexual partners and abnormal Pap smears or warts in their genital region. “We were able to use the registry to much better describe the disease than what we had previously been able to do just based on everybody taking care of five or 10 of these children,” he added.
Once the registry had this information, Dr. Derkay and his colleagues were able to design clinical trials to evaluate treatment options. They conducted trials looking at a heat shock protein (HSPE7), and gathered expert opinions on the use of cidofovir. Based on the findings, “we were able to come up with clinical practice guidelines for use of cidofovir, addressing which children are most likely to benefit, along with ideal dosing and intervals to use, as well as what side effects warranted monitoring.”
“Within the papilloma community, we were able to get both the US and international experts to agree upon these guidelines, and we all signed off on them as part of an organized effort to try and help our fellow otolaryngologists. The guidelines are widely quoted and widely utilized,” Dr. Derkay said (Laryngoscope. 2013;123:705–712).
Since the introduction of the human papillomavirus vaccine (Gardasil, Merck) in 2006, the RRP registry was revived to look at the effect of the vaccine on the number of new cases of RRP in the United States. “We’ve now gone back to most of the same 30 academic centers used in the original registry. We now have a second registry built out of 22 sites that have contributed their retrospective data and 26 sites that have prospective data,” Dr. Derkay said. “What we are seeing is a dramatic decrease in new cases, which mirrors the Canadian registry experience led by Paolo Campisi, MD, from Hospital for Sick Children at the University of Toronto.”