Methodological Insights from Health Valuation Studies in Low- and Middle-Income Countries: A Scoping Review.

Objectives: Valuation studies generate utility values for health states using stated preference methods. These studies are complex and resource intensive, posing implementation challenges in low- and middle-income countries (LMICs). This review aimed to examine how valuation studies in LMICs have navigated these challenges.

Healthcare resource utilization and caregiver burden associated with rotavirus gastroenteritis hospitalizations in Taiwan.

Background: Rotavirus infection is a leading cause of acute gastroenteritis in children aged < 5 years. However, few studies assessed the spillover effect on caregivers' health-related quality of life (HRQoL) and out-of-pocket costs associated with rotavirus gastroenteritis (RGE) in Taiwan.

Methods: This is a prospective, observational, survey-based study conducted in Northern Taiwan between March 2021-September 2023.

Valuing health states for infants and toddlers: challenges and methodological considerations in EQ-TIPS preference elicitation.

Many health care interventions are aimed at very young children, including public health measures such as vaccines, and new, high-cost medicines for rare diseases. This has led to increasing interest in evidence on the effectiveness and cost effectiveness of treatments in this age group. EQ-TIPS has been developed as a concise, generic health outcomes measure in 0-3-year-olds.

Assessing the experimental EuroQol toddler and infant populations (EQ-TIPS) descriptive system: a protocol integrating discrete choice experiment (DCE) surveys in instrument development.

Introduction: The experimental EuroQol Toddler and Infant Populations (EQ-TIPS) instrument is currently under development as a health-related quality of life (HRQoL) measure for toddlers and infants aged 0-36 months. Using this protocol, researchers can conduct surveys with discrete choice experiments (DCEs) that examine the key properties of HRQoL instruments, specifically whether severity aligns with preferences across each attribute and the extent to which attributes influence choices. To demonstrate this protocol, we will conduct two waves of DCE surveys using different choice tasks and a common scenario (a 1-month health episode for a 1-year-old child).

Comparing the measurement properties of the EQ-5D-Y-3L, EQ-5D-Y-5L and CHU9D in children and adolescents: a measurement property study.

Background: The EQ-5D-Y-3L, EQ-5D-Y-5L and Child Health Utility 9-dimension (CHU9D) are instruments that measures health-related quality of life. These instruments are widely used in children and adolescents with health conditions, however the measurement properties of the three instruments have not been tested in Brazilian children.

Objective: To compare and test the measurement properties of the EQ-5D-Y-3L, EQ-5D-Y-5L and CHU9D in Brazilian children and adolescents with and without any self-reported musculoskeletal pain.

Real-world outcomes of generic elexacaftor/tezacaftor/ivacaftor (gETI) in South Africans (SA) with CF using standard versus clarithromycin-boosted gETI, modulator-sparing strategies to reduce cost.

Objective: Access to highly effective modulator therapies (HEMT) in resource-limited countries is limited by prohibitive cost and restrictive patents. We report the clinical outcomes of a cost-reduction strategy in South Africa (SA), where generic elexacaftor/tezacaftor/ivacaftor (gETI) was pharmacokinetically enhanced with clarithromycin (gETI/c) for people with CF (pwCF) eligible for HEMT.

Methods: A multi-center observational study from December 2021 to May 2024.

EuroQol Toddler and Infant Populations (EQ-TIPS): Age-Related Differences in Performance.

Objectives: The EQ-TIPS was developed to measure the Health-Related Quality of Life in infants/toddlers. Considering the rapid development in this period, this study aimed to investigate age-related variations in EQ-TIPS performance.

Methods: Data from 551 infants/toddlers living with a health condition were analysed.

Transitioning between the EQ-5D youth and adult descriptive systems in a group of adolescents.

Purpose: To investigate whether the same health state results in the same distribution of responses on the EQ-5D youth and adult descriptive systems.

Methods: Adolescents aged 13-18 years with a range of health conditions and from the general school going population were recruited in South Africa (ZA) and Ethiopia (ET). In ZA participants completed the English EQ-5D-3L, EQ-5D-Y-3L and EQ-5D-5L in parallel.

The reliability and validity of the isixhosa version of the euroqol toddler and infant populations (EQ-TIPS) health related quality of life instrument.

Background: Considering the high burden of disease in young children measurement of Health-Related Quality of Life is needed to evaluate the burden of morbidity. This study aims to report on the validity and reliability of the isiXhosa EuroQol Toddler and Infant Populations (EQ-TIPS) measure for South Africa.

Methods: A sample of 181 caregivers of children 0-36 months were recruited from a hospital in-patient (inpt) and outpatient (outpt) facility and crèches.

Fighting to Breathe and Fighting for Health-Related Quality of Life: Measuring the Impact of Being Dependent on Technology for Breathing on the Child and Their Caregiver.

Background And Objective: Medical advancement has enabled children to survive congenital airway anomalies, rare diseases and critical illnesses with medical technology including tracheostomies and long-term ventilation to support breathing. This study aimed to assess (1) the validity of the EQ-TIPS and EQ-5D-Y-3L in children dependent on technology and (2) the impact of caring for these children on the EQ-5D-5L and CarerQoL.

Methods: Caregivers of children aged 1 month to 18 years completed the EQ-TIPS or EQ-5D-Y-3L, Pediatric Quality of Life Inventory (PedsQL) and Paediatric Tracheostomy Health Status Instrument (PTHSI) to reflect the child's health.

Global disparities in cystic fibrosis outcomes prior to CFTR modulators: A CF registries cohort study in South Africa and Canada.

Background: Outcomes of cystic fibrosis (CF) differ between low-middle income and high-income countries, but comparative data are lacking. We compared South African (SA) and Canadian CF outcomes to explore what disparities existed prior to access of CFTR modulators in Canada.

Methods: A cross-sectional study of SA and Canadian CF registries data for period 1 January to 31 December 2018.

Clinical and Experimental Determination of Protection Afforded by BCG Vaccination against Infection with Non-Tuberculous Mycobacteria: A Role in Cystic Fibrosis?

is a nontuberculous mycobacterium (NTM) of particular concern in individuals with obstructive lung diseases such as cystic fibrosis (CF). Treatment requires multiple drugs and is characterised by high rates of relapse; thus, new strategies to limit infection are urgently required. This study sought to determine how Bacille Calmette-Guérin (BCG) vaccination may impact NTM infection, using a murine model of infection and observational data from a non-BCG vaccinated CF cohort in Sydney, Australia and a BCG-vaccinated CF cohort in Cape Town, South Africa.

Cross-Cultural Adaptation and Validation of the EuroQoL Toddler and Infant Populations Instrument Into Afrikaans for South Africa.

Objectives: The EuroQol Group is exploring the development of a health-related quality of life measure for toddlers and infant populations (EuroQoL Toddler and Infant Populations [EQ-TIPS]) aged 0 to 36 months. This study aimed to report on the cross-cultural adaptation and validity of the South African Afrikaans EQ-TIPS.

Methods: The development of the Afrikaans EQ-TIPS followed the EuroQol guidelines including forward-backward translation and cognitive interviews with 10 caregivers of children aged 0 to 36 months.

Psychometric performance of the Chichewa versions of the EQ-5D-Y-3L and EQ-5D-Y-5L among healthy and sick children and adolescents in Malawi.

Objectives: The EuroQol Group has developed an extended version of the EQ-5D-Y-3L with five response levels for each of its five dimensions (EQ-5D-Y-5L). The psychometric performance has been reported in several studies for the EQ-5D-Y-3L but not for the EQ-5D-Y-5L. This study aimed to psychometrically evaluate the EQ-5D-Y-3L and EQ-5D-Y-5L Chichewa (Malawi) versions.

The validity and reliability of the interviewer-administered EQ-5D-Y-3L version in young children.

Objectives: The aim of this study was to determine the validity and reliability of the EQ-5D-Y-3L interviewer-administered (IA) version in South African children aged 5-7-years compared to 8-10-years.

Methods: Children aged 5-10-years (n = 388) were recruited from healthcare facilities, schools for learners with special educational needs and mainstream schools across four known condition groups: chronic respiratory illnesses, functional disabilities, orthopaedic conditions and the general population. All children completed the EQ-5D-Y-3L IA, Moods and Feelings Questionnaire (MFQ), Faces Pain Scale-Revised (FPS-R) and a functional independence measure (WeeFIM) was completed by the researcher.

β-adrenergic sweat test in children with inconclusive cystic fibrosis diagnosis: Do we need new reference ranges?

Background: Investigating inconclusive cystic fibrosis (CF) diagnosis in children is difficult without advanced cystic fibrosis transmembrane conductance regulator (CFTR) function tests. This study investigated the utility of beta (β)-adrenergic sweat test to exclude CF in participants with inconclusive diagnosis (CF suspects) in South Africa.

Methods: β-adrenergic sweat test and sweat chloride tests (SCT) were performed simultaneously in CF suspects and adult controls (healthy, CFTR heterozygotes and CF).

The performance of the EQ-5D-Y-5L compared to the EQ-5D-Y-3L in children and adolescents with cerebral palsy (CP).

Objectives: The aim of this study is to compare the performance and validity of the EQ-5D-Y-3L (Y-3L) and EQ-5D-Y-5L (Y-5L) in South African children and adolescents with cerebral palsy (CP).

Methods: Children/adolescents with CP and those from the general population completed the Y-5L, Y-3L, and PedsQL. Physiotherapists at the school classified participants' functional ability on the Gross Motor Functioning Classification System (GMFCS).

Comparison of the EQ-5D-Y-5L, EQ-5D-Y-3L and PedsQL in children and adolescents.

Background: There is an increased use of Patient-Reported Outcome Measures (PROMs) in children and adolescents. The aim of this study was to compare the feasibility, concurrent validity and known-group validity of the EQ-5D-Y-3L (Y-3L), EQ-5D-Y-5L (Y-5L) and PedsQL self-report PROMs.

Methods: Five hundred and fifty children and adolescents, aged 8-15-years, with acute and chronic health conditions and a general population sample were recruited from schools and hospitals in Cape Town South Africa.

Comparing Measurement Properties of the English EQ-5D-Y 3-Level Version With the 5-Level Version in South Africa.

Objectives: This study aimed to compare the performance of the EQ-5D-Y 3-level version (Y-3L) and the expanded 5-level version, the EQ-5D-Y 5-level version (Y-5L).

Methods: Children or adolescents with an acute or chronic health condition and from the general population completed the Y-5L and Y-3L self-report questionnaires. The performance of the Y-5L and Y-3L was determined by comparing feasibility, redistribution of dimension responses, discriminatory power, validity, and test-retest reliability.

Measurement properties and responsiveness of the EQ-5D-Y-5L compared to the EQ-5D-Y-3L in children and adolescents receiving acute orthopaedic care.

Objective: The aim of this study is a head-to-head comparison of the instrument performance and responsiveness of the EQ-5D-Y-3L and the expanded English version of the EQ-5D-Y-5L in children/adolescents receiving acute orthopaedic management in South Africa.

Methods: Children/adolescents aged 8-15 years completed the EQ-5D-Y-5L, EQ-5D-Y-3L, self-rated health (SRH) question and PedsQL at baseline. The EQ-5D-Y-5L, EQ-5D-Y-3L and SRH question were repeated after 24 and 48 h.

Why Do Adults Value EQ-5D-Y-3L Health States Differently for Themselves Than for Children and Adolescents: A Think-Aloud Study.

Objectives: Following protocol, adults value EQ-5D-Y-3L health states from the perspective of a 10-year-old child. It remains unclear why adults value health states differently for themselves than for a 10-year-old child and whether the latter perspective is representative of adults' preferences for the 8 to 15 years age range of the EQ-5D-Y-3L. This study examines the reasons underlying (potential) differences in adults' health-state preferences for themselves, a 10-year-old child, and 15-year-old adolescent.

Performance of the EQ-5D-Y Interviewer Administered Version in Young Children.

(1) Background: An estimated 78% of South African children aged 9-10 years have not mastered basic reading, therefore potentially excluding them from self-reporting on health-related outcome measures. Thus, the aim of this study was to compare the performance of the EQ-5D-Y-3L self-complete to the newly developed interviewer-administered version in children 8-10 years. (2) Methods: Children ( = 207) with chronic respiratory illnesses, functional disabilities, orthopaedic conditions and from the general population completed the EQ-5D-Y-3L self-complete and interviewer-administered versions, Moods and Feelings Questionnaire (MFQ) and Faces Pain Scale-Revised (FPS-R).

Trends in cystic fibrosis survival over 40 years in South Africa: An observational cohort study.

Introduction: Temporal trends in cystic fibrosis (CF) survival from low-middle-income settings is poorly reported. We describe changes in CF survival after diagnosis over 40 years from a South African (SA) CF center.

Methods: An observational cohort study of people diagnosed with CF from 1974 to 2019.