Patient‐relevant health outcomes for hemophilia care: Development of an international standard outcomes set

Balen, Erna C. van; O’Mahony, Brian; Cnossen, Marjon H.; Dolan, Gerard; Blanchette, Victor S.; Fischer, Kathelijn; Gue, Deborah; O’Hara, Jamie; Iorio, Alfonso; Jackson, Shannon; Konkle, Barbara A.; Nugent, Diane J.; Coffin, Donna; Skinner, Mark W.; Smit, Cees; Srivastava, Alok; Eenennaam, F. van; Bom, Johanna G. van der; Gouw, Samantha C.

doi:10.1002/rth2.12488

Cited by 29 publications

(46 citation statements)

References 39 publications

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“…Recently, the International Standard Outcomes Set working group proposed a set of 10 PROMs, focusing on what increases value for patients and families receiving hemophilia care. 4 While we designed this study prior to its publication, as a proof of concept, we mapped our PROMs to the framework proposed by the working group (Table 1 ). We feel that inclusion of different dimensions of PROMs is critical, not only to assign value to meaningful patient outcomes (eg, productivity, physical activity) not traditionally included in trials, but also to better discern the benefits of prophylaxis with EHL compared with SHL‐FVIII/FIX.…”

Section: Discussionmentioning

confidence: 99%

“…A standard set of 10 patient‐relevant health outcomes was recently developed by an international working group to harmonize longitudinal data collection and improve value‐based health care for persons with hemophilia. 4 The panel also recommended suitable outcome measure instruments based on psychometric properties. We present our PROM instruments using the framework proposed by the International Standard Outcomes Set working group (Table 1 ).…”

Section: Methodsmentioning

confidence: 99%

“…We present our PROM instruments using the framework proposed by the International Standard Outcomes Set working group (Table 1 ). 4 …”

Section: Methodsmentioning

confidence: 99%

“…We selected age‐appropriate PROMs based on validity, relevance, and burden of administration. A standard set of 10 patient‐relevant health outcomes was recently developed by an international working group to harmonize longitudinal data collection and improve value‐based health care for persons with hemophilia 4 …”

Section: Methodsmentioning

confidence: 99%

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The impact of extended half‐life factor concentrates on patient reported health outcome measures in persons with hemophilia A and hemophilia B

Sun

Yang²,

Poon

et al. 2021

Research and Practice in Thrombosis and Haemostasis

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Background Recombinant factors VIII and IX Fc (rFVIIIFc/rFIXFc) were the only available extended half‐life (EHL) products in Canada during 2016 to 2018. Objectives To evaluate if patient‐reported outcome measures (PROMs) improved in Canadian persons with hemophilia who switched from standard half‐life (SHL) to EHL products (rFVIIIFc/rFIXFc). Patients/Methods This prospective cohort study enrolled persons with moderate or severe hemophilia aged ≥6 years who switched to rFVIIIFc/rFIXFc (2016‐2018) and those who remained on SHL. Health‐related quality of life (HRQoL) was assessed using the Haemophilia‐specific Quality of Life (Haem‐A‐QoL) and 36‐item Short‐Form Survey (SF‐36) at baseline, 3‐months, 12 months, and 24 months. Other PROMs included the Work Productivity and Impairment Questionnaire, chronic pain scale, partner/parent ratings of mood, International Physical Activity Questionnaire, and Treatment Satisfaction Questionnaire for Medication. We identified meaningful changes using minimally important difference for SF‐36 and responder definition for Haem‐A‐QoL. Results We enrolled 25 switchers (16 rFVIIIFc, 9 rFIXFc) and 33 nonswitchers. Those switched to rFVIIIFc/rFIXFc had improved overall HRQoL, and improved subscale physical activity, mental health, and social functioning at 3 months. The rFIXFc switchers had improved chronic pain and ability to engage in normal activities while the rFVIIIFc switchers had improved treatment satisfaction. There was no change in work impairment after the switch. Observed improvement disappeared by 24 months in most domains. Conclusion Switching from SHL to rFVIIIFc/rFIXFc resulted in short‐term meaningful improvement in overall HRQoL and other PROMs in a small proportion. Longitudinal changes on PROMs are affected by ceiling effects and response shift, warranting further studies in instrument optimization in the era of EHL and nonfactor products.

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Section: Discussionmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

“…We present our PROM instruments using the framework proposed by the International Standard Outcomes Set working group (Table 1 ). 4 …”

Section: Methodsmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

See 2 more Smart Citations

The impact of extended half‐life factor concentrates on patient reported health outcome measures in persons with hemophilia A and hemophilia B

Sun

Yang²,

Poon

et al. 2021

Research and Practice in Thrombosis and Haemostasis

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“…Regular assessment of the patients should be carried out through specialized hemophilia centers ( 18 ). However, the rarity of the disease does not allow for a high number of such centers ( 19 ). The patients must travel long distances to be assessed by dedicated specialists, which poses a problem compliancy-wise.…”

Section: Introductionmentioning

confidence: 99%

Mobile Health Technology for the Personalized Therapy of Hemophilia

et al. 2021

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The management of patients with hemophilia has evolved significantly since the first treatment attempts were made in the late 1930s. Since then, each new step in the treatment of patients with hemophilia has brought important advancements, as well as its unique set of challenges. Today, a patient-centered, individualized comprehensive approach is the new paradigm, moving away from the traditional “one size-fits-all” approach, to provide the best possible care for each patient with a bleeding disorder. As part of this complex task, mobile health applications might have the capacity to play an important role in reaching that goal. However, the use of new electronic technologies as part of a comprehensive treatment approach for patients with hemophilia simultaneously presents a new set of challenges that needs consideration. In the first section, currently available treatment of hemophilia patients will be revised, while in the second part the role of IT software in the treatment monitoring of hemophilia patients will be discussed.

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

Kuijlaars

Teela

Vulpen

et al. 2021

Research and Practice in Thrombosis and Haemostasis

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Patient‐relevant health outcomes for hemophilia care: Development of an international standard outcomes set

Abstract: This is an open access article under the terms of the Creative Commons Attribution-NonCommercial-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 29 publications

References 39 publications

The impact of extended half‐life factor concentrates on patient reported health outcome measures in persons with hemophilia A and hemophilia B

The impact of extended half‐life factor concentrates on patient reported health outcome measures in persons with hemophilia A and hemophilia B

Mobile Health Technology for the Personalized Therapy of Hemophilia

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

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