Harmonizing patient‐reported outcome measurements in inherited bleeding disorders with PROMIS

Hoorn, Evelien S van; Teela, Lorynn; Kuijlaars, Isolde A R; Fischer, Kathelijn; Gouw, Samantha C.; Cnossen, Marjon H.; Haverman, Lotte

doi:10.1111/hae.14694

Cited by 4 publications

(10 citation statements)

References 13 publications

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“…The recently developed ‘Item Response Theory’ (IRT) may however be key to overcome these issues. IRT scales items and persons on a single metric, comparing the results between the groups longitudinally in time 87 . In paediatric care, this is even more relevant as longitudinal follow‐up of a child into adulthood is of advisable and otherwise requires various age‐adjusted tools. HRQoL research should provide detailed descriptive statistics.Many studies solely used the mean and SD, or median and IQR as the single descriptive statistics for HRQoL‐results.…”

Section: Discussionmentioning

confidence: 99%

Knowledge gaps in health‐related quality of life research performed in children with bleeding disorders – A scoping review

Huisman,

Mussert,

Bai

et al. 2024

Haemophilia

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IntroductionBleeding disorders (BDs) may influence health‐related quality of life (HRQoL) in children and caregivers. Measuring HRQoL gives insight into domains requiring support and provides an opportunity to evaluate the effects of novel therapies.AimTo gain insight in the current body of literature on HRQoL in children with BDs in order to identify knowledge gaps for research and further development of this field.MethodsScoping review.ResultsWe included 53 articles, describing studies mainly performed in Europe and North–America (60.4%) and mostly within the last ten years. Only 32% studies included children <4 years. Almost all studies (47/53, 88.7%) were performed in boys with haemophilia, pooling haemophilia A and B (n = 21) and different disease severities (n = 20). Thirteen different generic and five disease‐specific HRQoL‐questionnaires were applied; all questionnaires were validated for haemophilia specifically. Six (11,3%) combined generic and disease‐specific questionnaires. Self‐reports were most frequently applied (40/53, 75.5%), sometimes combined with proxy and/or parent‐reports (17/53, 32.1%). Eleven studies used a reference group (20.8%). Statistical analyses mostly consisted of mean and SD (77.4%).ConclusionHRQoL‐research is mainly performed in school‐aged boys with haemophilia, treated in developed countries. Pitfalls encountered are the pooling of various BDs, subtypes and severities, as well as the application of multiple generic questionnaires prohibiting comparison of results. More attention is needed for broader study populations including other BDs, young children, feminine such as young children, feminine bleeding issues and platelet disorders, as well as the use of HRQoL as an effect‐measurement tool for medical interventions, and more thorough statistical analysis.

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Section: Discussionmentioning

confidence: 99%

Knowledge gaps in health‐related quality of life research performed in children with bleeding disorders – A scoping review

Huisman,

Mussert,

Bai

et al. 2024

Haemophilia

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“…26 Although the abovementioned outcome sets were designed for different purposes, the selected outcomes overlap substantially and include clinical outcomes such as bleeding episodes, side effects and complications and PROs, such as joint damage, pain, physical functioning, impact on daily life including school and work and mental health. 27…”

Section: Outcomes Relevant For Patientsmentioning

confidence: 99%

“…37 In the Netherlands, the National Symphony inherited BD consortium aims to make the PROMIS item banks available for all patients with bleeding disoders. 27…”

Section: Patient-reported Outcomes Measurement Information System (Pr...mentioning

confidence: 99%

“…For other congenital BDs than haemophilia, including von Willebrand disease, rare coagulation factor deficiencies and platelet function disorders, a set of patient‐relevant outcomes has been defined 26 . Although the abovementioned outcome sets were designed for different purposes, the selected outcomes overlap substantially and include clinical outcomes such as bleeding episodes, side effects and complications and PROs, such as joint damage, pain, physical functioning, impact on daily life including school and work and mental health 27 …”

Section: Qol Measures: Limitations and How To Improvementioning

confidence: 99%

“…In haemophilia, the PROMIS‐29 short form is being used in the ATHN 7 Haemophilia national history study 37 . In the Netherlands, the National Symphony inherited BD consortium aims to make the PROMIS item banks available for all patients with bleeding disoders 27 …”

Section: Qol Measures: Limitations and How To Improvementioning

confidence: 99%

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Outcomes and outcome measures

Castaman,

Jimenez‐Yuste,

Gouw

et al. 2024

Haemophilia

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IntroductionAdvances in haemophilia treatment have resulted in a near‐normal life expectancy, lower burden of bleeding and treatment, and improved quality of life in high‐income countries. Bleeding rate is approaching zero and novel parameters should be evaluated to assess the efficacy of treatment not only from the clinical point of view by using new methodologies (e.g. joint health assessment), but also from the patient's perspective (e.g. pain, quality of life, treatment satisfaction).Methods and resultsThis approach should be aimed at combining objective clinical methodologies and patient‐reported outcomes (PROs). However, some instruments used for assessing PROs are still suboptimal and not properly validated. Recent evidence suggests that these tools can take advantage from a more personalized designed approach and could be effectively improved and serve to facilitate the patient's self‐evaluation. For other congenital bleeding disorders (BDs), a set of patient‐relevant outcomes has been also defined that overlap substantially those of haemophilia, including bleeding, side effects and complications, and PROs, such as pain, physical functioning, impact on daily life including school and work and mental health. There is a growing focus on addressing women‐specific outcomes in BDs, reflecting an increased awareness of the unique challenges faced by women in this context. However, the development of tailored tools is imperative to further advance the progress in managing women with BDs, ensuring more accurate monitoring and personalized care.ConclusionsHow incorporating these outcome measures in the process of approval of novel treatments for these disorders by regulatory authorities remains to be established.

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Psychometrics of the patient-reported outcomes measurement information system measures in hemophilia: the applicability of the pediatric item banks

Teela,

Luijten,

Kuijlaars

et al. 2023

Research and Practice in Thrombosis and Haemostasis

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Harmonizing patient‐reported outcome measurements in inherited bleeding disorders with PROMIS

Cited by 4 publications

References 13 publications

Knowledge gaps in health‐related quality of life research performed in children with bleeding disorders – A scoping review

Knowledge gaps in health‐related quality of life research performed in children with bleeding disorders – A scoping review

Outcomes and outcome measures

Psychometrics of the patient-reported outcomes measurement information system measures in hemophilia: the applicability of the pediatric item banks

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