Donna Beth Willey-Courand

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.

The Effect of Opening Wedge Thoracostomy on Thoracic Insufficiency Syndrome Associated with Fused Ribs and Congenital Scoliosis

BACKGROUND Thoracic insufficiency syndrome is the inability of the thorax to support normal respiration or lung growth and is seen in patients who have severe congenital scoliosis with fused ribs. Traditional spinal surgery does not directly address this syndrome. METHODS Twenty-seven patients with congenital scoliosis associated with fused ribs of the concave hemithorax had an opening wedge thoracostomy with primary longitudinal lengthening with use of a chest-wall distractor known as a vertical, expandable prosthetic titanium rib. Repeat lengthenings of the prosthesis were performed at intervals of four to six months. Radiographs were analyzed with respect to correction of the spinal deformity, as indicated by a change in the Cobb angle, and lateral deviation of the spine, as indicated by the interpedicular line ratio. Spinal growth was assessed by measuring the change in the length of the spine. Correction of the thoracic deformity and thoracic growth were assessed on the basis of the increase in the height of the concave hemithorax compared with the height of the convex hemithorax (the space available for the lung), the increase in the thoracic spinal height, and the increase in the thoracic depth and width. The thoracic deformity in the transverse plane was measured with computed tomography, and the scans were analyzed for spinal rotation, thoracic rotation, and the posterior hemithoracic symmetry ratio. Clinically, the patients were assessed on the basis of the relative heights of the shoulders and of head and thorax compensation. Pulmonary status was evaluated on the basis of the respiratory rate, capillary blood gas levels, and pulmonary function studies. RESULTS The mean age at the time of the surgery was 3.2 years (range, 0.6 to 12.5 years), and the mean duration of follow-up was 5.7 years. All patients had progressive congenital scoliosis, with a mean increase of 15 degrees /yr before the operation. The scoliosis decreased from a mean of 74 degrees preoperatively to a mean of 49 degrees at the time of the last follow-up. Both the mean interpedicular line ratio and the space available for the lung ratio improved significantly. The height of the thoracic spine increased by a mean of 0.71 cm/yr. At the time of the last follow-up, the mean percentage of the predicted normal vital capacity was 58% for patients younger than two years of age at the time of the surgery, 44% for those older than two years of age (p < 0.001), and 36% for those older than two years of age who had had prior spine surgery. In a group of patients who had sequential testing, all increases in the volume of vital capacity were significant (p < 0.0001), but the changes in the percentages of the predicted normal vital capacity were not. There was a total of fifty-two complications in twenty-two patients, with the most common being asymptomatic proximal migration of the device through the ribs in seven patients. CONCLUSIONS Opening wedge thoracostomy with use of a chest-wall distractor directly treats segmental hypoplasia of the hemithorax resulting from fused ribs associated with congenital scoliosis. The operation addresses thoracic insufficiency syndrome by lengthening and expanding the constricted hemithorax and allowing growth of the thoracic spine and the rib cage. The procedure corrects most components of chest-wall deformity and indirectly corrects congenital scoliosis, without the need for spine fusion. The technique requires special training and should be performed by a multispecialty team.

Cystic fibrosis foundation pulmonary guidelines: Use of cystic fibrosis transmembrane conductance regulator modulator therapy in patients with cystic fibrosis

Rationale: Cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulators are a new class of medications targeting the underlying defect in CF. Ivacaftor (IVA) and IVA combined with lumacaftor (LUM; IVA/LUM) have been approved by the U.S. Food and Drug Administration (FDA) for use in patients with CF. However, the FDA label for these medications encompasses patient groups that were not studied as part of the drug approval process. CF clinicians, patients, and their families have recognized a need for recommendations to guide the use of these medications. Objective: Develop evidence‐based guidelines for CFTR modulator therapy in patients with CF. Methods: A multidisciplinary committee of CF caregivers and patient representatives was assembled. A methodologist, an epidemiologist, a medical librarian, and a biostatistician were recruited to assist with the literature search, evidence grading, and generation of recommendations. The committee developed clinical questions using the Patient‐Intervention‐Comparison‐Outcome format. A systematic review was conducted to find relevant publications. The evidence was then evaluated using the GRADE (Grading of Recommendations, Assessment, Development, and Evaluation) approach, and recommendations were made based on this analysis. Results: For adults and children aged 6 years and older with CF due to gating mutations other than G551D or R117H, the guideline panel made a conditional recommendation for treatment with IVA. For those with the R117H mutation, the guideline panel made a conditional recommendation for treatment with IVA for 1) adults aged 18 years or older, and 2) children aged 6‐17 years with a forced expiratory volume in 1 second (FEV1) less than 90% predicted. For those with the R117H mutation, the guideline panel made a conditional recommendation against treatment with IVA for 1) children aged 12‐17 years with an FEV1 greater than 90% predicted, and 2) children less than 6 years of age. Among those with two copies of F508del, the guideline panel made a strong recommendation for treatment with IVA/LUM for adults and children aged 12 years and older with an FEV1 less than 90% predicted; and made a conditional recommendation for treatment with IVA/LUM for 1) adults and children aged 12 years or older with an FEV1 greater than 90% predicted, and 2) children aged 6‐11 years. Conclusions: Using the GRADE approach, we have made recommendations for the use of CFTR modulators in patients with CF. These recommendations will be of help to CF clinicians, patients, and their families in guiding decisions regarding use of these medications.

Improved screening for cystic fibrosis-related diabetes by an integrated care team using an algorithm.

To determine whether implementation of a standardized, clinic‐based algorithm improves compliance with cystic fibrosis‐related diabetes (CFRD) screening guidelines.

Non‐physician providers as clinical providers in cystic fibrosis: Survey of US programs

Non‐physician providers (NPPs) including nurse practitioners (NPs) and physician assistants (PAs) are important members of CF care teams, but limited data exist about the extent NPPs are involved in CF care. A subcommittee was established by the CF Foundation to gather information about current involvement of NPPs. Surveys were sent to adult, pediatric and affiliate CF program directors (PDs) and NPPs working in US CF programs.