Innovative research initiated by an international consortium of experts is moving forward with the development of a groundbreaking patient-reported outcome (PRO) instrument dedicated to measuring the health-related quality of life (HRQOL) for children and adolescents with lower limb deformities. This recent initiative is expected to fill a critical gap identified in a systematic review that highlighted the absence of a PRO instrument that captures the HRQOL in this patient population.

The team, led by Harpreet H. Chhina from BC Children’s Hospital in Vancouver, Canada, and comprising researchers from various countries including Ethiopia, India, and the United States, aims to create a scientifically robust and clinically relevant tool that can be applied internationally. Below details the study protocol, published in BMJ Open with DOI: 10.1136/bmjopen-2018-027079, which sets out the multi-phase process to develop and validate the PRO instrument.

The Protocol: A Multi-Phase Mixed Methods Study

Phase 1: Conceptual Framework and Preliminary Scale Development

The research begins with a comprehensive development of a conceptual framework for HRQOL based on systematic review findings, followed by qualitative interviews across five research sites. These interviews with young patients will guide the creation of an item pool, destined to form the basis of preliminary scales. Input from participating children and expert opinions from clinicians across the study sites will further refine the items, ensuring they resonate with the experiences and perceptions of the target population.

Phase 2: International Field-Test Study

Following the initial phase, a large-scale international field-test study will ensue, seeking to refine the scales meticulously. This phase’s focus on testing across diverse populations aims to guarantee that the instrument’s psychometric properties are consistent and reliable.

Phase 3: Reliability, Validity, and Responsiveness

The final phase involves rigorous testing of reliability, validity, and responsiveness to determine the PRO instrument’s efficacy. Tests of translations and cultural adaptations are also an integral part of this phase, anchoring the tool in intercultural competence.

The Expected Outcome: An Internationally Applicable PRO Instrument

The outcomes of this elaborate research are anticipated to be revelatory. An internationally applicable PRO instrument specifically designed for children and adolescents with lower limb deformities promises significant improvements in assessing how treatments affect patients’ quality of life.

Peer-reviewed Publications and International Presentations

As the study progresses, the findings and developments will be submitted to peer-reviewed journals and presented at national and international conferences. This wide dissemination ensures that healthcare practitioners worldwide receive the insights needed to adopt and apply the new PRO instrument comprehensively.

Knowledge Translation and Community Engagement

The research deployment mirrors an integrated knowledge translation approach, recognizing the value of engaging patients, families, and clinicians from the study’s inception to its conclusion. The research team aims to relay findings back to these stakeholders, ensuring continuous feedback and application of the instrument in clinical settings.

The Impact and Future Implications

The development of this PRO instrument stands to markedly advance the field of pediatric orthopedics and rehabilitative medicine. It has the potential to transform the evaluation of treatments and interventions designed for children and adolescents with lower limb deformities by grounding outcomes in patient-reported experiences.

Ethical Approval and Open Access

In line with ethical research practices, the study has secured approval from the Research Ethics Boards at each participating site. True to the spirit of open science, the authors and their employers have allowed re-use of the published work under a CC BY-NC license, with no commercial re-use.

A Collaborative International Effort

This endeavor to develop a PRO instrument underscores the power of collaborative international research. The team harnesses the expertise from multiple countries, ensuring that the tool is comprehensive, culturally sensitive, and adaptable to global use.

References

1. Chhina, H. H., Klassen, A. A., Kopec, J. A., Oliffe, J. J., & Cooper, A. A. (2019). International multiphase mixed methods study protocol to develop a patient-reported outcome instrument for children and adolescents with lower limb deformities. BMJ Open, 9(5), e027079. doi: 10.1136/bmjopen-2018-027079

2. Reeve, B. B., Wyrwich, K. W., Wu, A. W., et al. (2013). ISOQOL recommends minimum standards for patient-reported outcome measures used in patient-centered outcomes and comparative effectiveness research. Quality of Life Research, 22(8), 1889-1905. doi: 10.1007/s11136-012-0344-y

3. Wild, D., Grove, A., Martin, M., et al. (2005). Principles of Good Practice for the Translation and Cultural Adaptation Process for Patient-Reported Outcomes (PRO) Measures: report of the ISPOR Task Force for Translation and Cultural Adaptation. Value in Health, 8(2), 94-104. doi: 10.1111/j.1524-4733.2005.04054.x

4. Matza, L. S., Patrick, D. L., Riley, A. W., et al. (2013). Pediatric patient-reported outcome instruments for research to support medical product labeling: report of the ISPOR PRO good research practices for the assessment of children and adolescents task force. Value in Health, 16(4), 461-479. doi: 10.1016/j.jval.2013.04.004

5. Patrick, D. L., Burke, L. B., Gwaltney, C. J., et al. (2011). Content Validity—Establishing and Reporting the Evidence in Newly Developed Patient-Reported Outcomes (PRO) Instruments for Medical Product Evaluation: ISPOR PRO Good Research Practices Task Force Report: Part 1—Eliciting Concepts for a New PRO Instrument. Value in Health, 14(8), 967-977. doi: 10.1016/j.jval.2011.06.014

Keywords

1. Patient-Reported Outcome Instrument
2. Children Lower Limb Deformities
3. Health-Related Quality of Life
4. Pediatric Orthopedic Research
5. Multinational Healthcare Study

The advanced multi-phased research methodology promises to yield an invaluable tool—a patient-reported outcome (PRO) instrument—poised to revolutionize the measurement of health-related quality of life for young patients affected by lower limb deformities. With its international scope and commitment to patient-centric evaluation, this research is setting a new standard in pediatric healthcare and orthopedic research.