Generic PROMIS item banks in adults with hemophilia for patient‐reported outcome assessment: Feasibility, measurement properties, and relevance

Kuijlaars, Isolde A R; Teela, Lorynn; Vulpen, Lize F D van; Timmer, Merel A.; Coppens, Michiel; Gouw, Samantha C.; Peters, Marjolein; Kruip, Marieke J H A; Cnossen, Marjon H.; Muis, Jelmer J; Hoorn, Evelien S van; Haverman, Lotte; Fischer, Kathelijn

doi:10.1002/rth2.12621

Cited by 10 publications

(11 citation statements)

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“…When investigated, floor and ceiling effects were found in less than 2% of stroke patients. Only on the PROMIS pain interference short forms, 31-36% had the lowest score, similar to the 26% found in adults with hemophilia reported by Kuijlaars et al [29], but this does not have to indicate lack of validity because these patients may just have no pain. No studies were found that examined content validity, criterion validity and cross-cultural validity/measurement invariance in stroke populations.…”

Section: Discussionsupporting

confidence: 82%

Measurement properties and interpretability of the PROMIS item banks in stroke patients: a systematic review

Oosterveer¹,

Arwert

Terwee

et al. 2022

Qual Life Res

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Purpose Both the International Consortium for Health Outcomes Measurement and the National Institutes of Health recommend the use of Patient-Reported Outcomes Measurement Information System (PROMIS®) measures in clinical care and research for stroke patients. This study aimed to systematically review the literature on the measurement properties and interpretability of PROMIS measures in stroke patients. Methods Nine databases were searched from January 1st, 2007 till April 12th, 2021 for studies concerning the measurement properties and interpretability of PROMIS measures in stroke patients. The findings of these studies were analyzed according to the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) guideline for systematic reviews of Patient-Reported Outcome Measures (PROMs). ResultsTen studies were included. The PROMIS Global Health was studied the most: its two subscales had sufficient structural validity in one study of very good quality, sufficient construct validity with > 75% of hypotheses tested confirmed (high GRADE rating), sufficient internal consistency, i.e. α ≥ 0.70 in two studies (high GRADE rating), sufficient reliability, i.e. ICC ≥ 0.70 in one study of doubtful quality, and indeterminate responsiveness in one study of inadequate quality. For other PROMIS measures, the measurement properties and interpretability were limitedly studied. ConclusionThe PROMIS Global Health showed sufficient structural and construct validity and internal consistency in stroke patients. There is a need for further research on content validity, structural validity, and measurement invariance of PROMIS measures in stroke patients. Trial Registration Information: CRD42020203044 (PROSPERO).

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Section: Discussionsupporting

confidence: 82%

Measurement properties and interpretability of the PROMIS item banks in stroke patients: a systematic review

Oosterveer¹,

Arwert

Terwee

et al. 2022

Qual Life Res

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“…To solve issues like lengthy questionnaires, another development in the field of haemophilia is the introduction of generic Patient Reported Outcomes Measurement Information System (PROMIS) item banks. In adults, the PROMIS Computer Adaptive Test (CAT) ‘physical function’ was demonstrated to be a feasible, reliable and valid alternative to the HAL full for PWH, with a low number of items (mean number of items was 6.0) which is even lower than the 18 items of the HAL short 25 . However, before implementation of PROMIS CATs in day‐to‐day care and research several issues need to be addressed like good IT facilities for digital administration of CATs, clear visual feedback and cut‐off scores which helps in interpreting, monitoring and discussing individual items or scores, and budget for using CATs 26,27 .…”

Section: Discussionmentioning

confidence: 99%

Validation of the pedHAL_short and HAL_short in Dutch children and adults with haemophilia

et al. 2022

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Introduction The Haemophilia Activities List (HAL) and paediatric HAL assess self‐reported limitations in various daily activities. To reduce patient burden, shorter versions of the pedHAL (22 items) and HAL (18 items) have been developed. Aim This study aimed to determine the agreement between the pedHAL/HALfull and pedHAL/HALshort and construct validity and internal consistency of the pedHAL/HALshort in persons with haemophilia (PWH). Methods A cross‐sectional secondary analysis of the Hemophilia in the Netherlands‐6 national survey was performed. Adult and paediatric PWH completed the original pedHAL/HALfull, from which pedHAL/HALshort were derived. Score differences between the original and short versions were calculated. Construct validity was studied by testing hypotheses regarding the relationship of the pedHAL/HALshort with the pedHAL/HALfull, Haemophilia & Exercise Project Test‐Questionnaire (HEP‐Test‐Q), Canadian Haemophilia Outcomes‐Kids’ Life Assessment Tool (CHO‐KLAT) and RAND 36‐item Health Survey (RAND‐36) (convergent/discriminant validity) as well as its ability to discriminate between subgroups (known‐group validity). Internal consistency was assessed with Cronbach's α. Results We included 113 children (median 10y [range 4–17], 53% severe haemophilia) and 691 adults (median 51y [range 18–88], 35% severe). Scores of the pedHAL/HALfull and pedHAL/HALshort were similar with high correlations (>0.9). Construct validity was confirmed for the pedHAL/HALshort. The HALshort was able to discriminate between different disease severities and ages. Cronbach's α of the pedHAL/HALshort was 0.95–0.97. Conclusion This study confirmed the agreement between the pedHAL/HALfull and the pedHAL/HALshort and the construct validity of the pedHAL/HALshort. The next step is to study construct validity of the pedHAL/HALshort when administered as short forms.

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“…Joint status will be measured by physical examination (Haemophilia Joint Health Score),26–28 ultrasound (if available, according to the Haemophilia Early Arthropathy Detection with Ultrasound (HEAD US) score) 29. HR-QoL will be assessed with EQ5D(Y)-3L,30–32 and PROMIS (Patient-Reported Outcomes Measurement Information System) instruments (Physical Function/mobility and Pain Interference short forms) 33–35. Assessment of pain during emicizumab administration will be scored with the Visual Analogue Scale.…”

Section: Methods and Analysismentioning

confidence: 99%

DosEmi study protocol: a phase IV, multicentre, open-label, crossover study to evaluate non-inferiority of pharmacokinetic-guided reduced dosing compared with conventional dosing of emicizumab in people with haemophilia A

et al. 2023

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IntroductionEmicizumab effectively prevents bleeding in people with haemophilia A (PwHA), but is a burden for national healthcare budgets and consequently may limit access. According to the drug label, dosing of emicizumab is based on body weight with fixed intervals of 7, 14 or 28 days, which leads to mean plasma concentrations of 55 µg/mL (SD 15 µg/mL). However, a moderate variability of concentrations and a minimal effective concentration of 30 µg/mL have been suggested in studies. Therefore, a dose of emicizumab that targets a trough concentration of 30 µg/mL is hypothesised to be equally effective as conventional dosing in the prevention of bleeding.Methods and analysisWe designed a phase IV, multicentre, open-label, crossover study to evaluate non-inferiority of bleed control of ≥6 months on conventional dosing in comparison to ≥6 months on dose intervention. This dose intervention consists of reducing the dose of emicizumab to target a trough concentrations of 30 µg/mL using individual pharmacokinetic (PK) parameters. Ninety-five PwHA aged >1 years who received conventional dosing of emicizumab for ≥12 months with good bleeding control during the last 6 months will be recruited from all Dutch haemophilia treatment centres. The study is powered to detect a clinically relevant decrease (risk difference) of 15% in the proportion of patients without treated bleeds during follow-up. Secondary endpoints are spontaneous joint or muscle bleeds, and annualised treated bleeding rates (using negative binomial regression). Cost-effectivity between conventional dosing and individualised PK-guided dosing of emicizumab will be compared.Ethics and disseminationThe DosEmi study was approved by the Medical Ethics Review Committee NedMec of the University Medical Center of Utrecht, The Netherlands. Study results will be communicated through publications in international scientific journals and presentations at (inter)national conferences.Trial registration numberEUCTR2021-004039-10-NL athttps://trialsearch.who.int.Protocol versionV.4.1 on 28 October 2022 (DosEmi protocol_V4.1; NL81112.041.22).

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Generic PROMIS item banks in adults with hemophilia for patient‐reported outcome assessment: Feasibility, measurement properties, and relevance

Abstract: This is an open access article under the terms of the Creat ive Commo ns Attri butio n-NonCo mmerc ial-NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made.

Cited by 10 publications

References 50 publications

Measurement properties and interpretability of the PROMIS item banks in stroke patients: a systematic review

Measurement properties and interpretability of the PROMIS item banks in stroke patients: a systematic review

Validation of the pedHAL_short and HAL_short in Dutch children and adults with haemophilia

DosEmi study protocol: a phase IV, multicentre, open-label, crossover study to evaluate non-inferiority of pharmacokinetic-guided reduced dosing compared with conventional dosing of emicizumab in people with haemophilia A

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Generic PROMIS item banks in adults with hemophilia for patient‐reported outcome assessment: Feasibility, measurement properties, and relevance

Abstract: This is an open access article under the terms of the Creat ive Commo ns Attri butio n-NonCo mmerc ial-NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made.

Cited by 10 publications

References 50 publications

Measurement properties and interpretability of the PROMIS item banks in stroke patients: a systematic review

Measurement properties and interpretability of the PROMIS item banks in stroke patients: a systematic review

Validation of the pedHALshort and HALshort in Dutch children and adults with haemophilia

DosEmi study protocol: a phase IV, multicentre, open-label, crossover study to evaluate non-inferiority of pharmacokinetic-guided reduced dosing compared with conventional dosing of emicizumab in people with haemophilia A

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Validation of the pedHAL_short and HAL_short in Dutch children and adults with haemophilia