Evaluation of serum VIP and aCGRP during pulmonary exacerbation in cystic fibrosis: A longitudinal pilot study of patients undergoing antibiotic therapy.

BACKGROUND: Objective monitoring of improvement during treatment of pulmonary exacerbation can be difficulty in children when pulmonary function testing cannot be obtained. Thus, the identification of predictive biomarkers to determine the efficacy of drug treatments is of high priority. The major aim of the current study was to investigate the serum levels of vasoactive intestinal peptide (VIP) and alpha calcitonin gene related peptide (aCGRP) of cystic fibrosis pediatric patients during pulmonary exacerbation and post-antibiotic therapy, and possible associations of their levels with different clinicopathological parameters. METHODS: 21 patients with cystic fibrosis were recruited at onset of pulmonary exacerbation. Serum was collected at time of admission, three days post-antibiotic therapy, and two weeks post-antibiotic therapy (end of antibiotic therapy). Serum VIP and aCGRP levels were measured using ELISA. RESULTS: Overall least square means of serum aCGRP level but not VIP changed from time of exacerbation to completion of antibiotic therapy (p = 0.005). Serum VIP was significantly associated with the presence of diabetes mellitus (p = 0.026) and other comorbidities (p = 0.013), and with type of antibiotic therapy (p = 0.019). Serum aCGRP level was significantly associated with type of antibiotic therapy (p = 0.012) and positive Staphylococcus aureus microbiology test (p = 0.046). CONCLUSION: This study could only show significant changes in serum aCGRP levels following treatment of pulmonary exacerbations. Future studies with larger sample size are required to investigate the clinical importance of VIP and aCGRP in cystic fibrosis patients.

Pseudo-bartter syndrome, pattern and correlation with other cystic fibrosis features.

Pseudo-Bartter Syndrome (PBS), although quite common in patients with cystic fibrosis (CF), is often missed as simple dehydration or Bartter syndrome. This study was performed in patients with PBS to compare the pattern and course of the disease with those with CF not manifesting with this syndrome. All patients with CF who attended the respiratory clinic at Queen Rania Al-Abdallah Hospital from January 2000 to April 2010 were included in this retrospective case-control study. A specially formulated data sheet was used and those with PBS and those not having the syndrome were identified. A total of 110 patients (51% female) with CF with a median age of seven years were followed-up. Eighteen (16.3%) of them had one or more episodes of PBS. The median follow-up period was 6.2 years. All the episodes occurred during summer and in infancy. Median age of the initial episode of PBS was three months. One-third of them were initially followed at the nephrology clinic. Three patterns of PBS were identified: single episode in three (16.6%) patients, recurrent in 12 (66.6%) patients and chronic in three (16.6%) patients. Early colonization of Pseudomonas spp before 1 st birthday was seen in 44% patients with PBS compared with 12% in other CF patients (P-value = 0.0075). The total number of colonized patients and other CF features at the time of the study did not differ significantly among patients, although the mean Shwachman-Kulczycki score is significantly lower in those with recurrent PBS (69 compared with 85 in other CF patients). Gene mutation was identified in only 30% of the entire cohort. PBS is common in patients with CF, and it should be kept in mind in any patient with hypotonic dehydration and metabolic alkalosis. Recurrent pattern is associated with earlier Pseudomonas colonization.

Multicentric epithelioid hemangioendothelioma involving the lungs, trachea, liver and skeletal muscles.

Epithelioid hemangioendothelioma (EH) is a rare benign vascular tumor, which typically present as multinodular lesions that can involve one organ or more. We report a 12 years old female who presented with one-year history of progressive intolerance to physical activity and 3 months history of dry cough and weight loss. Physical examination was positive for diminished breath sounds and crackles of right hemithorax, and small mass in abdominal wall. CT of chest and abdomen revealed multiple nodular lesions in both lungs, liver, and right abdominal rectal muscle. Bronchoscopy showed multiple small tracheal lesions. Immunhistochemical staining of biopsy specimens obtained from the trachea, liver and muscle was consistent with EH.

Bronchoscopy findings in children with recurrent and chronic stridor.

BACKGROUND: To describe the bronchoscopic findings and treatment decision of children with chronic or recurrent stridor referred to the pediatric respiratory clinic at Queen Rania Hospital for Children. METHODS: All children who underwent flexible bronchoscopy at the pediatric bronchoscopy unit for chronic stridor from January 2009 to January 2010 were included. Stridor was divided into 3 groups: inspiratory, expiratory, or biphasic. All patients from 2 weeks of age till 14 years were included. Files of these patients were retrospectively reviewed. A specially formulated data sheet including clinical history and physical findings, type of stridor, bronchoscopic findings, and management decision was used. Radiologic investigation results were included when relevant. Flexible bronchoscopy was performed under sedation and topical anesthesia. RESULTS: A total of 64 children [35 (54.7%) male and 29 (45.3%) female] were included. Twenty-four patients had inspiratory stridor. There were 33 patients with biphasic stridor (subglottic stenosis in 9, paradoxical vocal cord movement in 6, vascular ring in 5, subglottic hemangioma in 3, vocal cord paralyses in 4, foreign body in 2, laryngeal web in 2, and absent vocal cord and tracheal bronchus in 1 each). Seven children had expiratory stridor. Of patients with laryngomalacia, 50% had associated tracheomalacia and 70% of those with laryngomalacia and all patients with tracheomalacia had associated gastroesophageal reflux. Two patients with paradoxical vocal cord movement were found to have Arnold-Chiari malformation. All patients with vascular rings underwent surgery. CONCLUSION: Flexible bronchoscopy should be performed in all patients with chronic or recurrent stridor to assess the airway and guide further investigations and management.

Clinical profile of cystic fibrosis. Atypical presentation.

OBJECTIVE: To describe the unusual presentation among patients with confirmed cystic fibrosis. METHODS: A retrospective review was carried out on all children (n=90) with the diagnosis of classical cystic fibrosis who attended the Respiratory Pediatric Clinic at King Hussein Medical Center, Amman, Jordan from January 2002--December 2008. All children from one day old to 14 years of age were included. Files of those with unusual presentation were reviewed. Age at presentation and diagnosis, clinical presentation, and family history were collected. Relevant laboratory results, sweat chloride readings, and radiological features were also reviewed. RESULTS: Ninety children (males 51 [57%] and females 39 [43%]) with classic cystic fibrosis were included. The most common initial classical presenting manifestation was recurrent wheezy chest (24%). The least common presentation was direct hyperbirubinemia (3%). Seven cases (8%) had unusual clinical presentations: early pulmonary hypertension, non-obstructive left hydronephrosis with metabolic alkalosis, single isolated episode of metabolic alkalosis, severe iron deficiency anemia with short stature, and the finding of ichthyotic skin lesions. Three of these patients had a positive family history of cystic fibrosis. Two patients with pulmonary hypertension died. The overall mortality rate was 4%. CONCLUSION: The wide variability of clinical presentations reflects the diversity of clinical picture of cystic fibrosis as a disease. Neonatal screening programs at a national level can decrease the burden of the disease.