Disease flares with baricitinib dose reductions and development of flare criteria in patients with CANDLE/PRAAS.

OBJECTIVES: Patients with chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature/proteasome-associated autoinflammatory syndrome (CANDLE/PRAAS) respond to the janus kinase inhibitor 1/2 inhibition with baricitinib at exposures higher than in rheumatoid arthritis. Baricitinib dose reductions to minimise exposure triggered disease flares which we used to develop 'flare criteria'. METHODS: Of 10 patients with CANDLE/PRAAS treated with baricitinib in an open-label expanded-access programme, baricitinib doses were reduced 14 times in 9 patients between April 2014 and December 2019. Retrospective data analysis of daily diary scores and laboratory markers collected before and after the dose reductions were used to develop 'clinical' and 'subclinical' flare criteria. Disease flare rates were compared among patients with <25% and >25% dose reductions and during study visits when patients received recommended 'optimized' baricitinib doses (high-dose visits) versus lower than recommended baricitinib doses (low-dose visits) using two-sided χ2 tests. RESULTS: In the 9/10 patients with CANDLE with dose reduction, 7/14 (50%) times the dose was reduced resulted in a disease flare. All four dose reductions of >25% triggered a disease flare (p <0.05). Assessment of clinical and laboratory changes during disease flares allowed the development of disease flare criteria that were assessed during visits when patients received high or low doses of baricitinib. Disease flare criteria were reached during 43.14% of low-dose visits compared with 12.75% of high-dose visits (p <0.0001). Addition of an interferon score as an additional flare criterion increased the sensitivity to detect disease flares. CONCLUSION: We observed disease flares and rebound inflammation with baricitinib dose reductions and proposed flare criteria that can assist in monitoring disease activity and in designing clinical studies in CANDLE/PRAAS.

Cost transparency in otolaryngology: Outpatient procedures cost information at New England hospitals.

PURPOSE: Healthcare costs have dramatically increased, resulting in barriers to care for many Americans. To address this, the Centers for Medicare & Medicaid Services implemented a price transparency mandate, requiring hospitals to provide cost-estimate tools. This study evaluates the accessibility and usability of these tools for common otolaryngology outpatient procedures. MATERIALS AND METHODS: Cost transparency was investigated using cost-estimate tools from websites of the seven New England hospitals ranked on the US News top 50 list. Ten common current procedural terminology codes were used to collect data on availability of cost information, cost comparison, and ease-of-use by six investigators acting as "patients" for each hospital and procedure. RESULTS: All investigated hospitals had cost-estimate tools, with a 35.7 % mean success rate of generating an estimate. The mean times to cost-estimate tools and generated estimates were 35.69 and 34.15 s, respectively. Pre-insurance costs varied by hospital and procedure; creation of eardrum had the largest range. Seven out of ten procedures resulted in lower post-insurance costs. The mean ease-of-use rating was 5.76 out of ten. CONCLUSION: All hospitals complied with the Centers for Medicare & Medicaid Services price transparency policy. The information available is sparse, difficult to access, and frequently lacks specific estimates for common otolaryngology procedures. Although hospitals are following new Centers for Medicare & Medicaid Services mandates, the estimators currently in existence are ineffective tools for financial decision-making.