GAS-Hēm was feasible and tapped constructs not captured by ABR or QoL measures.
Polyethylene glycol (PEG) is an inert, water soluble polymer, used for decades in pharmaceuticals. Although PEG is considered safe, concerns persist about the potential adverse effects of long-term exposure to PEG-containing therapies, specifically in children, following the introduction of PEGylated recombinant factor products used for the treatment of hemophilia. Given the absence of long-term surveillance data, and to evaluate the potential risk, we estimated PEG exposure in the pediatric population receiving PEGylated therapies with pediatric indications administered intravenously or intramuscularly. We used a range of pediatric weights and doses based on prescribing information (PI) or treatment guidelines. PIs and reporting websites were searched for information about adverse events (AEs). For a child weighing 50 kg on the highest prophylactic dose of a FVIII product, the range of total PEG exposure was 40–21,840 mg/year; for factor IX (FIX) products, the range was 13–1342 mg/year; and for other products, the range was 383–26,743 mg/year, primarily as a derivative excipient. No AE patterns attributable to PEG were found for any of these products, including potential renal, neurological, or hepatic AEs. Our analyses suggest the pediatric population has had substantial exposure to PEG for several decades, with no evidence of adverse consequences.
Background: Goal Attainment Scaling for Hemophilia (GOAL-Hēm) is a novel, hemophilia-specific, validated patient engagement tool and patient-reported outcome instrument.Objective:We evaluated the degree to which the language of GOAL-Hēm was patientcentric and the content valuable and relevant for people with hemophilia (PWH) and/ or their caregivers.Patients/Methods: Patients and caregivers participated in one of three investigations: an online survey, one-on-one patient interviews, or a focus group. The survey and interviews assessed the clarity and relevance of the GOAL-Hēm menu items.Interviews were semistructured, audio recorded, and transcribed verbatim. Feedback from interviews was coded as "clear," "unclear," "remove," or "add." The focus group explored participants' experience of GOAL-Hēm and elicited recommendations for implementation. Quotations from focus group and interview transcripts were indexed and charted to emergent themes for analysis.Results:Participants comprised 19 adults with hemophilia and 19 caregivers of children with hemophilia (survey, n = 20; interview, n = 12; focus group, n = 6). After their feedback, 32% (15/48) of goals were retained unchanged. Further feedback resulted in the removal of 45% (286/635) of the goal descriptors, and 30% (193/635) of the retained descriptors were modified. Three new (total = 38) goals and 42 descriptors (total = 368) were added to the menu. Thematic analysis indicated that participants were enthusiastic about patient-centric language, empowered through the goal-setting process, and recognized GOAL-Hēm could measure clinically meaningful change. Conclusion:By listening closely to patients and caregivers, we refined GOAL-Hēm to better capture the experiences of PWH, enhance content validity, and augment implementation strategies. Incorporating the patient voice is integral to developing patient-centered outcome measures.
Purpose Guidelines for the use of goal attainment scaling (GAS) recommend that the patient specify at least three goals. Even so, this may not always be feasible or align with patient preferences. Investigations into the psychometric properties of GAS using three or more goals largely support its reliability, validity, and responsiveness compared with standard measures. As evaluations of responsiveness rely on variability estimates, this metric may be impacted when GAS is based on fewer than three goals. For this reason, we investigated the responsiveness of one- and two-goal GAS. Methods Secondary analyses were conducted on data from a mixed sample of pediatric, adolescent and adult subjects with hemophilia A. The standardized response mean (SRM) and its 95% confidence intervals (CI) were used to assess responsiveness of one- and two-goal GAS at six and twelve weeks. Results Both one-goal and two-goal GAS demonstrated similar responsiveness to change at 6-week (Patient-Rated GAS: one-goal SRM [95% CI] = 0.70 [0.45–1.08], two-goal = 0.96 [0.68–1.30]; Clinician-Rated GAS: one-goal = 1.26 [0.81–1.77], two-goal = 1.01 [0.73–1.32]) and 12-week follow-up (Patient-Rated GAS: one-goal SRM [95% CI] = 1.14 [0.53–1.71], two-goal = 1.35 [0.92–1.82]; Clinician-Rated GAS: one-goal = 1.71 [1.12–2.30], two-goal = 1.48 [1.02–2.02]). Larger SRMs were observed for clinician-rated GAS, but all were within the rubric of a large effect size. Conclusions One-goal GAS is responsive to change in a clinical population. Further research is recommended in a larger sample where responsiveness of one- and multiple-goal GAS can be compared
Introduction: GOAL-H ēm is a novel, haemophilia-specific, patient-centred outcome measure (PCOM) based on goal attainment scaling, allowing people with haemophilia (PwH) to set and monitor the attainment of individualized goals for treatment. Aim:To provide a thorough overview of the creation, validation, and development of GOAL-H ēm.Methods: Clinician workshops were held to develop a haemophilia-specific goal menu.Qualitative data from semistructured interviews with PwH and their caregivers guided further revisions to the goal menu (i.e., goal domains and descriptors). A feasibility study was performed including a 12-week, prospective, noninterventional evaluation involving clinicians and PwH at four US haemophilia treatment centres. Finally, the Patient Voice Study gathered feedback from PwH and their caregivers via an online survey, interviews, and a focus group. Results:The feasibility study validated GOAL-H ēm with successful outcomes in construct/content validity and responsiveness, including a large effect in patient-and clinician-rated goal attainments. The Patient Voice Study led to significant refinement of GOAL-H ēm goals and descriptors, resulting in a more straightforward and relatable menu for PwH and their caregivers. Overall, GOAL-H ēm captured qualitative data in areas important to PwH and employed quantitative methods to evaluate meaningful changes in those areas. The individualized tool was well equipped to handle the complex and chronic nature of haemophilia and was endorsed by PwH, their caregivers, and clinicians. Conclusion:The GOAL-H ēm development journey may serve as a roadmap for other PCOMs in a variety of settings, including clinical studies, haemophilia treatment centres for care planning, and as a tool to gather real-world evidence.
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