AIMS: To determine immunogenicity, pharmacokinetics (PK), efficacy, safety and quality of life of prophylaxis with a polyethylene glycol (peg)-ylated FVIII (BAX 855) based on full-length recombinant FVIII (ADVATE) in paediatric previously treated patients (PTPs) with severe haemophilia A.
METHODS: PTPs <12 years without history of FVIII inhibitors received twice-weekly infusions of 50 ± 10 IU kg(-1) BAX 855 for ≥50 exposure days. Prophylactic dose increases to ≤80 IU kg(-1) were allowed under predefined conditions. PK was evaluated after single infusions of 60 ± 5 IU kg(-1) .
RESULTS: T1/2 and mean residence time were extended 1.3- to 1.5-fold compared to ADVATE (n = 31), depending on the analysis used. The point estimate for the mean annualized bleeding rate in 66 subjects receiving a median of 1.9 weekly infusions of 51.3 IU kg(-1) of BAX 855 each was 3.04 (median 2.0); 1.10 (median 0) for joint and 1.16 (median 0) for spontaneous bleeds. Overall, 38% of subjects had zero bleeds. No bleeds were severe. Haemostatic efficacy was rated excellent or good for 90% of bleeds; 91% were treated with one or two infusions. In 8/14 subjects all target joints resolved. No subject developed FVIII inhibitors or persistent binding antibodies that affected safety or efficacy. No adverse reactions occurred.
CONCLUSION: Twice-weekly prophylaxis with BAX 855 was safe and efficacious in paediatric PTPs with severe haemophilia A.
OBJECTIVE: To investigate the impact of customized CMI (C-CMI) on health-related quality of life (HRQoL) among type 2 diabetes mellitus (T2DM) patients in Qatar.
METHODS: This was a randomized controlled intervention study, in which the intervention group patients received C-CMI and the control group patients received usual care. HRQoL was measured using the EQ-5D-5L questionnaire and EQ visual analog scale (EQ-VAS) at three intervals [i.e. baseline, after 3 months and 6 months].
RESULTS: The EQ-5D-5L index value for the intervention group exhibited sustained improvement from baseline to the third visit. There was a statistically significant difference between groups in the HRQoL utility value (represented as EQ index) at 6 months (0.939 vs. 0.796; p = 0.019). Similarly, the intervention group compared with the control group had significantly greater EQ-VAS at 6 months (90% vs. 80%; p = 0.003).
CONCLUSIONS: The impact of C-CMI on health outcomes of T2DM patients in Qatar reported improvement in HRQoL indicators among the intervention patients. The study built a platform for health policymakers and regulatory agencies to consider the provision of C-CMI in multiple languages.
METHODS: This was a cross sectional study design. A total of 347 respondents from low household income groups, including persons with disability and Orang Asli were recruited from E-kasih. A semi-guided self-administered questionnaire was used. QOL measured by EQ. 5D utility value and health status measured by visual analogue score (VAS). Descriptive statistic, bivariate Chi-square analysis and binary logistic regression were conducted to determine factors influencing low QOL and poor health status.
RESULTS: Majority of the respondents were Malay, female (61%), 63% were married, 60% were employed and 46% with total household income of less than 1 thousand Ringgit Malaysia. 70% of them were not having any chronic medical problems. Factors that associated with low QOL were male, single, low household income, and present chronic medical illness, while poor health status associated with female, lower education level and present chronic medical illness. Logistic regression analysis has showed that determinants of low QOL was present chronic illness [AOR 4.15 95%CI (2.42, 7.13)], while determinants for poor health status were; female [AOR 1.94 95%CI (1.09,3.44)], lower education [AOR 3.07 95%CI (1.28,7.34)] and present chronic illness [AOR 2.53 95%CI (1.39,4.61)].
CONCLUSION: Low socioeconomic population defined as low total household income in this study. Low QOL of this population determined by present chronic illness, while poor health status determined by gender, education level and chronic medical illness.
METHODS/DESIGN: This is a randomized, single-blind, two-arm, controlled trial in patients with rheumatoid arthritis visiting outpatient rheumatology clinics in Karachi, Pakistan. We will enroll patients with established diagnosis of rheumatoid arthritis over 3 months. The patients would be randomized through a computer-generated list into the control group, i.e., usual care or into the intervention group, i.e., pharmaceutical care, in a ratio of 1:1, after providing signed written consent. The study will take place in two patient-visits over the course of 3 months. Patients in the intervention group would receive intervention from the pharmacist while those in the control group will receive usual care. Primary outcomes include change in mean score from baseline (week 0) and at follow up (week 12) in disease knowledge, adherence to medications and rehabilitation/physical therapy. The secondary outcomes include change in the mean direct cost of treatment, HRQoL and patient satisfaction with pharmacist counselling.
DISCUSSION: This is a novel study that evaluates the role of the pharmacist in improving treatment outcomes in patients with rheumatoid arthritis. The results of this trial could set the foundation for future delivery of care for this patient population in Pakistan. The results of this trial would be published in a peer-reviewed journal.
TRIAL REGISTRATION: ClinicalTrials.gov, NCT03827148 . Registered on February 2019.
OBJECTIVES: To determine a CE threshold for health care interventions in Malaysia.
METHODS: A cross-sectional, contingent valuation study was conducted using a stratified multistage cluster random sampling technique in four states in Malaysia. One thousand thirteen respondents were interviewed in person for their socioeconomic background, quality of life, and WTP for a hypothetical scenario.
RESULTS: The CE thresholds established using the nonparametric Turnbull method ranged from MYR12,810 to MYR22,840 (~US $4,000-US $7,000), whereas those estimated with the parametric interval regression model were between MYR19,929 and MYR28,470 (~US $6,200-US $8,900). Key factors that affected the CE thresholds were education level, estimated monthly household income, and the description of health state scenarios.
CONCLUSIONS: These findings suggest that there is no single WTP value for a quality-adjusted life-year. The CE threshold estimated for Malaysia was found to be lower than the threshold value recommended by the World Health Organization.