Pih76 Itch Reported Outcome Tool for Caregivers of Pediatric Patients With Cholestatic Liver Disease: An Analysis of Validation and Scoring From the Iconic Maralixibat Study

Abstract

Objectives: Tobacco smoking is considered as one of the major global public health issues, especially in developing countries. Nowadays, pregnant women are suffering from the harm from second-hand smoke (SHS) and third-hand smoke (THS) exposure due to high rates of husband smoking, which are associated with several adverse clinical outcomes of pregnant women such as spontaneous abortion and postpartum depression. Methods: A national based cross-sectional study was conducted to examine the associations between different types of tobacco exposure and health-related quality of life (HRQoL) in pregnant women from different regions in China. A web-based questionnaire was asked during participation in prenatal examination. HRQoL was measured using the EuroQoL five-dimension-five-level (EQ-5D-5L) questionnaire and demographic data were collected. Results: 16,811 pregnant women were included and 51.8% of them were in different types of tobacco exposure. Significant difference in EQ-VAS scores were detected between non-tobacco exposure and tobacco exposure groups [First-hand smoke (FHS) (p=0.002), SHS (p,0.01) and THS (p,0.01)]. Moreover, increased amounts of SHS and THS exposure could result in lower HRQoL of pregnant women (p=0.039 and p=0.007 respectively). Conclusions: Approximately half of pregnant women were under the influence of different types of tobacco exposure. Compared to non-tobacco exposure, FHS, SHS and THS had negative impact on the HRQoL of pregnant women. Deleterious effect of tobacco exposure on HRQoL of pregnant women was in dose-response relationship. Tobacco exposure is common and severe health problem causing economic burden. Government need to make more relevant policies to limit not only FHS and SHS exposure but also THS exposure, which was ignored in the past.

Maralixibat for the treatment of PFIC: Long‐term, IBAT inhibition in an open‐label, Phase 2 study

“…Pruritus was measured using the ItchRO tool, which was specifically developed and validated to evaluate pruritus in children with cholestatic liver diseases, based on a 5‐point scale where 0 = “none” and 4 = “very severe.” [ 18 , 19 ] This tool has been shown to detect clinically relevant changes in cholestatic pruritus in children across all ages, with changes ≥ 1.0 point being clinically meaningful. [ 19 , 20 ] The ItchRO measure was completed twice daily via an eDiary. Parents/caregivers completed the ItchRO(Obs) assessment for all patients.…”

“…Patients with BSEP deficiency demonstrated predictably varied responses to treatment according to disease type, with patients with nt-BSEP (n = 19) showing the greatest response. During the study, sBA response (reduction in sBAs of > 75% from baseline or concentrations < 102.0 μmol/L) was achieved by a subset of 7 patients (37%) with nt-BSEP (hereafter referred to as "sBA responders").…”

“…[18,19] This tool has been shown to detect clinically relevant changes in cholestatic pruritus in children across all ages, with changes ≥ 1.0 point being clinically meaningful. [19,20] The ItchRO measure was completed twice daily via an eDiary. Parents/caregivers completed the ItchRO(Obs) assessment for all patients.…”

“…Changes in mean ItchRO(Obs) scores are found in Table 2. All 7 sBA responders experienced a > 1.0-point clinically meaningful reduction in ItchRO(Obs) scores after achieving sBA response (Figure S2A), and an additional 3 sBA nonresponders achieved clinically meaningful reductions in ItchRO(Obs) scores [19,20] (Figure S2B).…”