Congenital platelet disorders and health status–related quality of life

Blaauwgeers, Maaike W.; Kruip, Marieke J.H.A.; Beckers, Erik A.M.; Coppens, Michiel; Eikenboom, Jeroen; Galen, Karin P M van; Tamminga, R. Y. J.; Urbanus, Rolf T.; Schutgens, Roger E.G.

doi:10.1002/rth2.12281

Cited by 6 publications

(22 citation statements)

References 29 publications

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“…We have previously shown that the self-administered BS obtained in the WiN study was comparable to the investigator obtained BS [14] . Lastly, blood samples were drawn to measure relevant coagulation factor levels in a central laboratory for each of the studies [ 13 , 15 , 18 , 21 , 22 ]. Laboratory measurements have been described in detail previously [ 13 – 16 ].…”

Section: Methodsmentioning

confidence: 99%

Major differences in clinical presentation, diagnosis and management of men and women with autosomal inherited bleeding disorders

et al. 2021

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Background In recent years, more awareness is raised about sex-specific dilemmas in inherited bleeding disorders. However, no large studies have been performed to assess differences in diagnosis, bleeding phenotype and management of men and women with bleeding disorders. Therefore, we investigated sex differences in a large cohort of well-defined patients with autosomal inherited bleeding disorders (von Willebrand disease (VWD), rare bleeding disorders (RBDs) and congenital platelet defects (CPDs)). Methods We included patients from three nationwide cross-sectional studies on VWD, RBDs and CPDs in the Netherlands, respectively the WiN, RBiN and TiN study. In all studies a bleeding score (BS) was obtained, and patients filled in an extensive questionnaire on the management and burden of their disorder. Findings We included 1092 patients (834 VWD; 196 RBD; 62 CPD), of whom 665 (60.9%) were women. Women were more often referred because of a bleeding diathesis than men (47.9% vs 36.6%, p = 0.002). Age of first bleeding was similar between men and women, respectively 8.9 ± 13.6 (mean ±sd) years and 10.6 ± 11.3 years ( p = 0.075). However, the diagnostic delay, which was defined as time from first bleeding to diagnosis, was longer in women (11.6 ± 16.4 years) than men (7.7 ± 16.6 years, p = 0.002). Similar results were found when patients referred for bleeding were analyzed separately. Of women aging 12 years or older, 469 (77.1%) had received treatment because of sex-specific bleeding. Interpretation Women with autosomal inherited bleeding disorders are more often referred for bleeding, have a longer diagnostic delay, and often require treatment because of sex-specific bleeding. Funding The WiN study was supported (in part) by research funding from the Dutch Hemophilia Foundation (Stichting Haemophilia), Shire (Takeda), and CSL Behring (unrestricted grant).

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Section: Methodsmentioning

confidence: 99%

Major differences in clinical presentation, diagnosis and management of men and women with autosomal inherited bleeding disorders

et al. 2021

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“…Five publications assessed the correlation between bleeding assessment tools scores and HRQoL 23–25,40,42 . Bleeding scores were determined using validated tools including the Tosetto Bleeding Score, 24,25,42 the International Society on Thrombosis and Haemostasis Bleeding Assessment Tool (ISTH‐BAT) 23,40 and the self‐administered Bleeding Assessment Tool (Self‐BAT) 40 .…”

Section: Resultsmentioning

confidence: 99%

“…This trend is especially visible in the following domains: vitality, physical and social functioning, pain and health in general. The relationship between bleeding scores and HRQoL was unclear, with some studies finding positive and others negative associations 23–25,40,42 . This may be related to the use of different assessment tools.…”

Section: Discussionmentioning

confidence: 99%

“…40 Three studies found that a more severe bleeding phenotype (i.e., higher bleeding scores) was associated with lower HRQoL, 24,25,42 two studies did not find such an association. 23,40 Higher bleeding scores were associated Abbreviation: QoL; Quality of Life. a Comparison in all categories between pre-and post-treatment were statistically significant (P < .01).…”

Section: 45mentioning

confidence: 99%

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Patient‐reported outcomes in autosomal inherited bleeding disorders: A systematic literature review

et al. 2022

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Aim Currently, it is unknown which patient‐reported outcomes are important for patients with autosomal inherited bleeding disorders. Therefore, the purpose of this study is to systematically review the available literature assessing patient‐reported outcomes and their measurement methods in autosomal inherited bleeding disorders. Methods The Embase, Medline ALL, Web of Science Core Collection, Cochrane Central Register of Controlled Trails and Google Scholar databases were searched from inception until 14 August 2020. Studies on patient‐reported outcomes in patients with von Willebrand disease, inherited platelet function disorders and coagulation factor deficiencies were included. Results Twenty‐one articles met the inclusion criteria. Three studies were assessed as having poor quality, and therefore a high risk of bias. Nineteen studies had fair quality rating. Different measurements methods were used, ranging from predefined to self‐developed questionnaires. The majority of included studies focused on von Willebrand disease. Patients with von Willebrand disease reported lower health‐related quality of life compared to the general population. Overall, this trend was especially visible in the following domains: vitality, physical and social functioning and pain. Women with inherited bleeding disorders scored lower on health‐related quality of life compared to men, especially women with heavy menstrual bleeding. Patients with joint bleeds or heavy menstrual bleeding reported an increased level of pain. Conclusion Patients with autosomal inherited bleeding disorders report lower health related quality of life, especially those with joint bleeds or heavy menstrual bleeding. Numerous measurement methods are used in patients with autosomal inherited bleeding disorders, highlighting the need for studies using established, standardized measurement methods.

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“…Categorical variables are displayed by frequencies and percentages. To score the SF‐36, scales are standardised with a scoring algorithm to obtain a score ranging from 0 to 100, as previously described 15 . Higher scores imply a better health status, and a mean score of 50 has been expressed as a normative value for all scales 22 .…”

Section: Methodsmentioning

confidence: 99%

Prevalence, burden and treatment effects of vaginal bleeding in women with (suspected) congenital platelet disorders throughout life: a cross‐sectional study

Punt¹,

Ruigrok²,

Bloemenkamp

et al. 2021

Br J Haematol

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Summary Congenital platelet disorders (CPDs) are rare bleeding disorders that are associated with mucocutaneous bleeds. However, data on vaginal bleeding in women with CPDs are scarce. A set of generic and bleeding‐specific questionnaires were used to evaluate the prevalence of vaginal bleeding, its impact on quality of life (QoL) and sexual functioning and the consequences for pregnancy, miscarriage and delivery in a cohort of women who were referred for diagnostic evaluation for CPDs. A total of 78 women included in the study were either diagnosed with a CPD (n = 35) or were clinically suspected of a CPD (n = 43). Heavy menstrual bleeding (HMB) was reported by a large proportion of women, which mainly started at menarche. In all, 76% of women received any kind of HMB treatment, often leading to surgical prodecures. HMB was shown to have a high impact on QoL, which improved upon treatment. Even though women reported that vaginal bleeding affects sexuality, this topic is not frequently discussed with physicians. Heavy blood loss frequently occurred after miscarriage/delivery, often requiring treatment. Women with (suspected) CPDs frequently encounter HMB, negatively impacting daily life and sexual functioning. Together with peripartum bleeding, these data highlight the burden of vaginal bleeding in CPDs and importance of adequate treatment.

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Congenital platelet disorders and health status–related quality of life

Cited by 6 publications

References 29 publications

Major differences in clinical presentation, diagnosis and management of men and women with autosomal inherited bleeding disorders

Major differences in clinical presentation, diagnosis and management of men and women with autosomal inherited bleeding disorders

Patient‐reported outcomes in autosomal inherited bleeding disorders: A systematic literature review

Prevalence, burden and treatment effects of vaginal bleeding in women with (suspected) congenital platelet disorders throughout life: a cross‐sectional study

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