Cost-effectiveness of an Evidence-Based Childhood Asthma Intervention in Real-World Primary Care Settings.

We present an incremental cost-effectiveness analysis of an evidence-based childhood asthma intervention (Community Healthcare for Asthma Management and Prevention of Symptoms [CHAMPS]) to usual management of childhood asthma in community health centers. Data used in the analysis include household surveys, Medicaid insurance claims, and community health center expenditure reports. We combined our incremental cost-effectiveness analysis with a difference-in-differences multivariate regression framework. We found that CHAMPS reduced symptom days by 29.75 days per child-year and was cost-effective (incremental cost-effectiveness ratio: $28.76 per symptom-free days). Most of the benefits were due to reductions in direct medical costs. Indirect benefits from increased household productivity were relatively small.

Hepatorenal findings in obligate heterozygotes for autosomal recessive polycystic kidney disease.

Autosomal recessive polycystic kidney disease (ARPKD), characterized by progressive cystic degeneration of the kidneys and congenital hepatic fibrosis (CHF), is the most common childhood onset ciliopathy, with an estimated frequency of 1 in 20,000 births. It is caused by mutations in PKHD1. The carrier frequency for ARPKD in the general population is estimated at 1 in 70. Given the recessive inheritance pattern, individuals who are heterozygous for PKHD1 mutations are not expected to have clinical findings. We performed ultrasound (USG) evaluations on 110 parents from 64 independent ARPKD families and identified increased medullary echogenicity in 6 (5.5%) and multiple small liver cysts in 10 parents (9%). All ARPKD parents with these abnormal imaging findings were asymptomatic; kidney and liver function tests were unremarkable. Complete sequencing of PKHD1 in the 16 ARPKD parents with abnormal imaging confirmed the mutation transmitted to the proband, but did not reveal any other pathogenic variants. Our data suggest that carrier status for ARPKD is a predisposition to polycystic liver disease and renal involvement associated with increased medullary echogenicity on USG. Whether some of these individuals become symptomatic as they age remains to be determined.