An assessment of the global usability of quality-of-life questionnaires in children receiving recombinant growth hormone therapy.
Generic PROMs are generally more usable than condition-specific ones, with significant barriers like cost affecting their routine use in assessing quality of life for children receiving growth hormone therapy.
Where it sits
this study against the rest of the hgh (somatropin) corpusSummary and findings
This study evaluated the usability of quality-of-life questionnaires in children receiving recombinant growth hormone therapy. A total of 50 patient-reported outcome measures (PROMs) were identified, with 37 remaining after exclusions. Key findings included that 43% of the PROMs were available in multiple languages and 65% had a completion time of less than 15 minutes.
Abstract
<h4>Background</h4>In the field of childhood growth hormone therapy (rhGH), patient and parent/observer-reported outcome measures (PROMs) are rarely used routinely.<h4>Aim</h4>To identify barriers and facilitators for the global implementation of PROMs for evaluating QoL in children receiving rhGH.<h4>Methods</h4>PROMs used in childhood rhGH therapy that were identified through a narrative literature search and expert group consultation were evaluated on reporter's perspective, language availability, administration mode, cost, age range, and completion time.<h4>Results</h4>Of the 50 PROMs identified, 13 were excluded due to methodological limitations. Of the remaining 37, that included 26 generic and 11 condition-specific PROMs, 16 (43%) were available in self-report and parent-proxy formats in over 30 languages and 24 (65%) had a reported completion time less than 15 min. The median lower age limit was 7 yrs (range: 3.0, 12.7). Of the 37, 7 (19%) were free to use, 12 (32%) required a fee, and for 18 (49%), the cost was unclear or varied between clinical practice and research. The mostly commonly represented domains in the condition-specific PROMs included "physical functioning & symptoms" and "emotional & mental health". Fees for condition-specific PROMs were often not explicit, and none were unconditionally free of charge. Overall, generic PROMS tended to rank higher across the six usability parameters than condition-specific tools.<h4>Conclusion</h4>Cost and unclear fee structures represent barriers to the routine use of condition-specific PROMs for assessing rhGH therapy in real-world settings. Whilst both, generic and condition-specific PROMs play important complementary roles, the former are more likely to exhibit characteristics that support global implementation.
Background
This paper addresses the implementation of patient and parent-reported outcome measures (PROMs) in evaluating quality of life (QoL) for children undergoing growth hormone therapy. Previous research has indicated a lack of routine use of PROMs in this context, highlighting a gap in patient-centered care. Understanding the usability of these measures is essential for improving clinical practices and ensuring that patient perspectives are incorporated into treatment evaluations.
Methods
The study utilized a narrative literature search and expert group consultation to identify PROMs used in childhood rhGH therapy. A total of 50 PROMs were initially identified, with 37 remaining after excluding those with methodological limitations. The evaluation focused on various usability parameters, including reporter's perspective, language availability, administration mode, cost, age range, and completion time.
Results
Of the 37 PROMs evaluated, 16 (43%) were available in self-report and parent-proxy formats in over 30 languages. Additionally, 24 (65%) had a reported completion time of less than 15 minutes. The median lower age limit for these PROMs was 7 years, with a range from 3.0 to 12.7 years. Cost analysis revealed that 7 (19%) were free to use, while 12 (32%) required a fee, and for 18 (49%), the cost was unclear or varied.
Interpretation
The findings suggest that while many PROMs are available, significant barriers exist regarding cost and clarity of fee structures, particularly for condition-specific measures. The study indicates that generic PROMs may be more feasible for global implementation compared to condition-specific tools. However, the clinical significance of these findings is not directly assessed, and the usability parameters do not guarantee that the PROMs will effectively capture meaningful patient outcomes.
Key findings
- Of the 50 PROMs identified, 13 were excluded due to methodological limitations.
- Of the remaining 37, 16 (43%) were available in self-report and parent-proxy formats in over 30 languages.
- 24 (65%) had a reported completion time less than 15 min.
- The median lower age limit was 7 yrs (range: 3.0, 12.7).
- 7 (19%) were free to use, 12 (32%) required a fee, and for 18 (49%), the cost was unclear or varied.
Limitations
- The study relies on a narrative literature search and expert consultation, which may introduce bias.
- The findings are based on a limited selection of PROMs and do not assess their clinical impact directly.