Naomi A Mckoy

Cystic Fibrosis Pulmonary Guidelines: Treatment of Pulmonary Exacerbations

The natural history of cystic fibrosis lung disease is one of chronic progression with intermittent episodes of acute worsening of symptoms frequently called acute pulmonary exacerbations These exacerbations typically warrant medical intervention. It is important that appropriate therapies are recommended on the basis of available evidence of efficacy and safety. The Cystic Fibrosis Foundation therefore established a committee to define the key questions related to pulmonary exacerbations, review the clinical evidence using an evidence-based methodology, and provide recommendations to clinicians. It is hoped that these guidelines will be helpful to clinicians in the treatment of individuals with cystic fibrosis.

Development of a framework to identify research gaps from systematic reviews.

OBJECTIVE Our objective was to develop a framework to identify research gaps from systematic reviews. STUDY DESIGN AND SETTING We reviewed the practices of (1) evidence-based practice centers (EPCs), and (2) other organizations that conduct evidence syntheses. We developed and pilot tested a framework for identifying research gaps. RESULTS Four (33%) EPCs and three (8%) other organizations reported using an explicit framework to determine research gaps. Variations of the PICO (population, intervention, comparison, outcomes) framework were most common. We developed a framework incorporating both the characterization of the gap using PICOS elements (also including setting) and the identification of the reason(s) why the gap exists as (1) insufficient or imprecise information, (2) biased information, (3) inconsistency or unknown consistency, and (4) not the right information. We mapped each of these reasons to concepts from three common evidence-grading systems. CONCLUSION Our framework determines from systematic reviews where the current evidence falls short and why or how the evidence falls short. This explicit identification of research gaps will allow systematic reviews to maximally inform the types of questions that need to be addressed and the types of studies needed to address the research gaps.

Management of Infants with Cystic Fibrosis: A Summary of the Evidence for the Cystic Fibrosis Foundation Working Group on Care of Infants with Cystic Fibrosis

OBJECTIVE To inform the development of Cystic Fibrosis (CF) Foundation guidelines on the care of infants with CF, we systematically reviewed the evidence for diagnosis and assessment of pancreatic and pulmonary disorders; management of pancreatic and pulmonary function; management of nutrition and nutritional disorders; and prevention and control of infections. STUDY DESIGN In May-June 2008, we searched The Cochrane Library for existing reviews; and MEDLINE, the National Guideline Clearinghouse, the CF Foundation Clinical Practice Guidelines and Consensus Statements, and the UK CF Trust for existing guidelines. MEDLINE, Cumulative Index to Nursing and Allied Health Literature (CINAHL) and the Excerpta Medica Database (EMBASE) were searched for primary studies in January 2008. Bibliographies of eligible articles were searched and expert input was sought. We selected English-language articles of any study design that provided original data on any of our questions on infants up to 2 years of age. RESULTS We identified 14 relevant guidelines and 3 Cochrane reviews. Fifty-nine articles (55 primary studies) were included. Only four of these were randomized controlled trials. Sample sizes of infants ranged from 2 to 768 study participants; the median sample size was 24. Of our 21 review topics, 5 topics had only one study while for 5 we identified no relevant studies. We identified one or no primary studies for 20 of 32 review questions. CONCLUSIONS There is a paucity of evidence on the care of infants diagnosed with CF. For several of the review questions no guidelines or primary studies were identified, but for other questions, studies limited by weak design and small sample sizes were the only studies identified. With increasing numbers of infants with CF being diagnosed by newborn screening there is an opportunity to study the management of infants diagnosed with CF.

Identification of research gaps from evidence-based guidelines: A pilot study in cystic fibrosis

OBJECTIVES Evidence-based guideline committees are multidisciplinary and explicitly consider the existing evidence. They are thus in an ideal position to identify research gaps. However, gaps have not been systematically identified through guidelines. We pilot tested a method to systematically identify and classify gaps from evidence-based guidelines. METHODS We reviewed all evidence-based guidelines published by the Cystic Fibrosis Foundation. We identified research gaps as topics for which there was insufficient evidence (recommendations were not made or consensus recommendations were made) and topics specified as needing further research. We characterized gaps using a standard framework and classified them by type of management issue, specificity of target population, and age of target population. RESULTS We identified sixty-two research gaps in five guidelines (mean = 12.4/guidelines document). While thirteen gaps were topics specified as needing further research, most (n = 49) were topics with insufficient evidence. Of these forty-nine, recommendations were not made for twenty-two topics while consensus recommendations were made for twenty-seven topics. Most gaps were issues of comparative effectiveness (44/62), addressed the general cystic fibrosis population (40/62), and were specific to infants (33/62). Relevant comparisons and outcomes were explicitly stated for only 7 percent and 16 percent of gaps respectively. CONCLUSIONS Almost 80 percent of the gaps were not topics identified as future research needs in the guidelines documents but rather were topics with insufficient evidence for making recommendations. Although we used cystic fibrosis in the United States as an example, the method we developed could be applied in other settings, including other countries and for different diseases.