Examining Risk Factors of Health-Related Quality of Life Impairments Among Adolescents with Inflammatory Bowel Disease.

Inflammatory Bowel Disease (IBD) is a chronic, costly, and burdensome disease that is typically diagnosed during adolescence. Despite the use of effective treatments, rates of relapse and intestinal inflammation remain high and put patients at risk for long term physical and psychosocial health complications. Given the costs associated with IBD, it is critical to examine potential risk factors of poor health-related quality of life (HRQoL) among patients for the enhancement and further development of interventions. As such, the aim of the current study was to examine how sociodemographic and disease characteristics, psychosocial problems, and adherence behaviors impact HRQoL among a sample of youth with IBD. 107 adolescents with IBD and their caregiver completed self- and parent-report measures as part of a psychosocial screening service. Medical records were reviewed to obtain information regarding diagnosis, insurance, medication use, illness severity, and disease activity. Results revealed lower HRQoL scores among adolescents with more psychosocial problems (Est. = -3.08; p < .001), greater disease severity (Est. = -.40; p = .001), and those who identified as Black (Est. = -.38; p < .05). Greater disease severity (Est. = .13 p = .004), use of nonpublic insurance (Est. = .32 p = .004), and fewer psychosocial problems (Est. = -.13 p = .04) were associated with greater adherence behaviors. These findings suggest that implementing individually tailored, evidence-based psychological interventions focused on coping with psychosocial problems and symptoms may be important in enhancing adherence behaviors and HRQoL among adolescents with IBD.

Response of Blue Rubber Bleb Nevus Syndrome to Sirolimus Treatment.

BACKGROUND: Blue rubber bleb nevus syndrome (BRBNS) is a rare multifocal venous malformation syndrome involving predominantly the skin and gastrointestinal tract. Traditional treatment modalities include corticosteroids, interferon-α, sclerotherapy, and aggressive surgical resection. Sirolimus has been used in several single case reports. PROCEDURE: We performed a single-institution retrospective review of four children with BRBNS, who received sirolimus as part of their treatment regimens. A diagnosis of BRBNS was based on clinical, radiologic, and pathologic criteria. RESULTS: Median age was 6.5 years (range: 2-16 years). Pathologic evaluations revealed a combined malformation with venous and lymphatic components. The novel finding of a lymphatic component was confirmed with PROX-1 immunostaining. Patients received oral sirolimus with target drug levels between 10 and 13 ng/ml. Responses to treatment were defined as stabilization/decrease in size of lesions; resolution of transfusion requirements; reduction in pain, and improvement in quality of life (QOL). Median time to response was 1.5 months (SD ± 0.96 month, range: 1-3 months). Median follow-up was 21 months (range: 18-26 months). Lesion size and characteristics improved in all patients. All patients reported decrease in pain and improvement in QOL. All three patients requiring transfusions became transfusion-independent. One patient had resolution of coagulopathy. Adverse effects of sirolimus consisted of mucositis in three patients and neutropenia in one patient. CONCLUSIONS: Sirolimus is safe and efficient for the treatment of BRBNS. Further prospective studies are needed to evaluate the long-term effectiveness of this drug. This is the first report that identifies a lymphatic component as part of BRBNS.

Pulmonary vascular complications in asymptomatic children with portal hypertension.

OBJECTIVES: : To determine the prevalence of portopulmonary hypertension, hepatopulmonary syndrome (HPS), and intrapulmonary vascular shunting (IPVS) in children with clinically stable portal hypertension and to assess the value of vasoactive peptide levels, biochemical tests and clinical signs or symptoms to predict these conditions. PATIENTS AND METHODS: : A prospective, cross-sectional analysis was conducted on 33 children, ages 4 to 17 years, with stable cirrhosis (n = 28) or extrahepatic portal hypertension (n = 5). The children were screened for IPVS and hypoxia with contrast-enhanced echocardiography (cECHO) and pulse oximetry, and screened for pulmonary hypertension with Doppler echocardiography. Chemistries, radiographs, physical examinations, and levels of vasoactive peptides were compared between subjects with IPVS and those with normal cECHO. RESULTS: : No subject had pulmonary hypertension. Six (19%) had IPVS, all of which had intrahepatic causes of portal hypertension, and 1 of whom had HPS. Compared with subjects with normal cECHO, those with IPVS had biochemical evidence of more advanced liver disease and higher b-type natriuretic peptide levels. CONCLUSIONS: : Prevalence of portopulmonary hypertension and HPS appear to be rare in clinically stable children with portal hypertension. Intrapulmonary vascular shunting was present in 19% of these patients. A novel finding of this study is the elevation of b-type natriuretic peptide in children with IPVS.

Graduation Day: Healthcare Transition From Pediatric to Adult.

Because more patients with pediatric-onset chronic conditions are surviving into adulthood, they are graduating from pediatric healthcare to self-management and adult healthcare. This transfer of care needs to be a process of transitioning medical and nutrition care. Despite having position statements from professional organizations and several proposed models, issues in the transition process have been well described, and gaps in transition care persist. Healthcare providers need to be aware of special needs of emerging adults related to education on chronic condition and self-management skills, emotional support before and after transition, and legal rights for both the patient and the parent if the emerging adult is not developmentally appropriate to make his or her own healthcare decisions. Both pediatric and adult providers need to be in active communication with each other and the patient to develop trusting relationships and actively support the transition of care. This review of literature describes several models for transitioning, measureable outcomes, insurance provider issues, and legal issues pertaining to healthcare transition.

Portopulmonary hypertension in pediatric patients.

OBJECTIVES: To investigate the clinical presentation, manifestations, and response to therapy of portopulmonary hypertension (PPHTN) in pediatric patients. STUDY DESIGN: This study was a retrospective chart review describing the evaluation and course of 7 patients with PPHTN. RESULTS: Causes of portal hypertension (HTN) included biliary atresia (3 cases), cavernous transformation of the portal vein (2 cases), and primary sclerosing cholangitis and cryptogenic cirrhosis (1 case each). The median interval from the diagnosis of portal HTN to PPHTN was 12.1 years. Four patients presented with a new heart murmur, 4 presented with syncope, and 3 presented with dyspnea. Although electrocardiograms (ECGs) and chest x-rays were normal in 3 and 2 patients, respectively, echocardiograms diagnosed pulmonary HTN in all 7 patients. Five patients had cardiac catheterizations; the average mean pulmonary artery pressure was 65 +/- 20 mm Hg. Response to therapy was variable, and 4 patients died. Postmortem lung tissue examination revealed plexiform lesions and pulmonary arteriopathy. CONCLUSIONS: Because symptoms are subtle and may be overlooked, pediatric patients with portal HTN who develop a new heart murmur, dyspnea, syncope, or who are being evaluated for liver transplantation require evaluation for PPHTN. ECG and chest x-ray are insensitive screens for PPHTN. An echocardiogram and cardiology evaluation is essential for the diagnosis.