Improving screening for cystic fibrosis-related diabetes at a pediatric cystic fibrosis program.

Objective: Despite guidelines recommending an annual oral glucose tolerance test (OGTT) for all patients with cystic fibrosis (CF) aged ≥ 10 years, screening rates for cystic fibrosis-related diabetes (CFRD) remained low at our center. The aim of this project was to implement an outpatient system to provide effective, evidence-based screening for CFRD at a pediatric CF program.

Methods: Development of a system to improve outpatient screening for CFRD included structured education, communication with families, and processes for scheduling laboratory appointments.

Pulmonary function tests in idiopathic inflammatory myopathy: association with clinical parameters in children.

Objective: To determine the association of decreased lung function in children with idiopathic inflammatory myopathies (IIMs) with specific clinical parameters.

Methods: This study of 38 children ages 6-23 years diagnosed with definite/probable IIM evaluated the association of myositis-specific/-associated antibodies (MSAs/MAAs), duration of untreated disease at diagnosis, Disease Activity Score for muscle (DAS-M), muscle-derived enzymes (aldolase, lactate dehydrogenase [LDH], aspartate transaminase, and creatine phosphokinase [CPK]), neopterin and von Willebrand factor antigen, and the Childhood Myositis Assessment Scale (CMAS) scores with data from pulmonary function testing (PFT).

Results: Impaired PFTs were defined as total lung capacity (TLC) or diffusing capacity for carbon monoxide (DLCO) of <80% predicted.

Successful use of plasmapheresis for granulomatosis with polyangiitis presenting as diffuse alveolar hemorrhage.

Diffuse alveolar hemorrhage (DAH) is uncommon in pediatric patients and is a rare presenting sign of granulomatosis with polyangiitis (GPA). We present the case a 14-year-old girl who presented with respiratory failure secondary to DAH as the initial presenting sign of GPA. Her clinical course improved after initiation of plasmapheresis therapy and she is now in clinical remission.

Successful prolonged use of recombinant human insulin-like growth factor-1 in a child with cystic fibrosis.

Growth failure is a common and complicated process in children with cystic fibrosis (CF). Growth hormone, which is becoming a more commonly used agent in such patients, has demonstrated beneficial effects aside from increased growth velocity. Recently, insulin-like growth factor-1 has gained significant attention in the understanding of growth failure in children with CF.

Functional capacity in children and young adults with sickle cell disease undergoing evaluation for cardiopulmonary disease.

Although cardiopulmonary disease is associated with decreased functional capacity among adults with sickle cell disease (SCD), its impact on functional capacity in children with SCD is unknown. We evaluated 6-min walk (6MW) distance in 77 children and young adults with SCD undergoing screening for cardiopulmonary disease. Of 30 subjects who also underwent cardiopulmonary exercise testing, we found evidence for decreased exercise capacity in a significant proportion.

Tricuspid regurgitant jet velocity elevation and its relationship to lung function in pediatric sickle cell disease.

Concerns about the morbidity and mortality associated with tricuspid regurgitant jet velocity (TRJV) elevation, which may indicate pulmonary hypertension (PHT), in adults with sickle cell disease (SCD) have prompted growing interest in screening the pediatric sickle cell population. The goals of our study were to estimate the prevalence of TRJV elevation and determine its relationship to pulmonary function in children and young adults with SCD at baseline. Seventy-eight subjects (10-24 years old) with SCD underwent prospective screening by Doppler echocardiogram (ECHO), complete lung function evaluation, and laboratory testing as part of standard care at steady state.

EGF receptor tyrosine kinase inhibitors diminish transforming growth factor-alpha-induced pulmonary fibrosis.

Transforming growth factor-alpha (TGF-alpha) is a ligand for the EGF receptor (EGFR). EGFR activation is associated with fibroproliferative processes in human lung disease and animal models of pulmonary fibrosis. We determined the effects of EGFR tyrosine kinase inhibitors gefitinib (Iressa) and erlotinib (Tarceva) on the development and progression of TGF-alpha-induced pulmonary fibrosis.

Genomic profile of matrix and vasculature remodeling in TGF-alpha induced pulmonary fibrosis.

Expression of transforming growth factor alpha (TGF-alpha) in the respiratory epithelium of transgenic mice caused pulmonary fibrosis, cachexia, pulmonary hypertension, and altered lung function. To identify genes and molecular pathways mediating lung remodeling, mRNA microarray analysis was performed at multiple times after TGF-alpha expression and revealed changes consistent with a role for TGF-alpha in the regulation of extracellular matrix and vasculogenesis. Transcripts for extracellular matrix proteins were augmented along with transcripts for genes previously identified to have roles in pulmonary fibrosis, including tenascin C, osteopontin, and serine (or cysteine) peptidase inhibitor, clade F, member 1.