High prevalence of heavy menstrual bleeding in women with rare bleeding disorders in the Netherlands:retrospective data from the RBiN study

Background: Heavy menstrual bleeding (HMB) is associated with a reduced quality of life and limitations in social and physical functioning. Data on HMB in women with rare bleeding disorders (RBDs), including coagulation factor deficiencies and fibrinolytic disorders, are scarce. Objectives: To analyze the prevalence, severity, and treatment of HMB in Dutch women with an RBD. Methods:The Rare Bleeding Disorders in the Netherlands (RBiN) study included 263 patients with an RBD from all 6 hemophilia treatment centers (October 2017-November 2019). In this analysis, data of 111 women aged ≥16 years were studied. According to the International Society on Thrombosis and Haemostasis bleeding assessment tool, HMB symptoms were scored from 0 (no/trivial) to 4 (severe symptoms requiring medical intervention). HMB was defined as a score ≥1. Age at RBD diagnosis was extracted from patient files. Results: HMB was reported by 80% of women (89/111) and was more prevalent in women with a fibrinolytic disorder (33/35; 94%) than in women with a coagulation factor deficiency (56/76; 74%) (P = .011). Of the 89 women with HMB, 82% (n = 73) ever required treatment. Multiple treatment modalities were frequently used, both in severe and mild deficiencies. Hormonal treatment was mostly used (n = 64; 88%), while antifibrinolytics were prescribed less frequently (n = 18; 25%). In women with HMB since menarche (n = 61; 69%), median age at RBD diagnosis was 28 years (IQR, 14-41).Conclusion: HMB is common in women with RBDs. Women with mild deficiencies also frequently reported HMB. Only a minority of women were treated with hemostatic agents. A significant diagnostic delay was observed after the onset of HMB symptoms.</p

Validation of PROMIS Profile-29 in adults with hemophilia in the Netherlands

Background The Patient-Reported Outcomes Measurement Information System (PROMIS) Profile-29 questionnaire is widely used worldwide, but it has not yet been validated in the Netherlands, nor in persons with hemophilia. Objective To validate the Dutch-Flemish version of the PROMIS-29 Profile v2.01 in adults with hemophilia. Methods Dutch males with hemophilia (all severities) completed questionnaires that contained sociodemographic and clinical characteristics, the PROMIS-29, RAND-36, and the Hemophilia Activities List (HAL). Structural validity of each subscale was assessed with confirmatory factor analysis (CFA). Internal consistency was calculated for each subscale with sufficient model fit in CFA. Construct validity was assessed by testing hypotheses about (1) correlations of each PROMIS-29 subscale with corresponding scales of RAND-36 and domains of HAL, and (2) mean differences in T-scores between subgroups with different hemophilia severities, self-reported joint impairment, and HIV infection status. We considered >= 75% of data in accordance with the hypotheses evidence for construct validity. Results In total, 770 persons with hemophilia participated in this cross-sectional study. CFA revealed sufficient structural validity for five subscales: Physical Function, Depression, Sleep Disturbance, Ability to Participate in Social Roles and Activities, and Pain Interference. Internal consistency was high and Cronbach's alpha ranged from 0.79 for Sleep Disturbance to 0.96 for Pain Interference. Differences between clinical subgroups were in the expected direction. Construct validity was confirmed for Physical Function, Anxiety, Depression, Fatigue, Sleep Disturbance, and Pain Intensity. Conclusion This study revealed sufficient evidence for structural validity, internal consistency, and construct validity for most PROMIS Profile-29 subscales among people with hemophilia in the Netherlands.Clinical epidemiolog

Methods to quantify soft tissue-based cranial growth and treatment outcomes in children: a systematic review

Contains fulltext : 137438.pdf (publisher's version ) (Open Access)CONTEXT: Longitudinal assessment of cranial dimensions of growing children provides healthcare professionals with information about normal and deviating growth as well as treatment outcome. OBJECTIVE: To give an overview of soft tissue-based methods for quantitative longitudinal assessment of cranial dimensions in children until age 6 years and to assess the reliability of these methods in studies with good methodological quality. DATA SOURCE: PubMed, EMBASE, Cochrane Library, Web of Science, Scopus, and CINAHL were searched. A manual search was performed to check for additional relevant studies. STUDY SELECTION: Primary publications on facial growth and treatment outcomes in children younger than age 6 years were included. DATA EXTRACTION: Independent data extraction was performed by two observers. A quality assessment instrument was used to determine methodological quality. Methods used in studies with good methodological quality were assessed for reliability expressed as the magnitude of the measurement error and the correlation coefficient between repeated measurements. RESULTS: In total, 165 studies were included, forming three groups of methods: head circumference anthropometry, direct anthropometry, and 2D photography and 3D imaging techniques (surface laser scanning and stereophotogrammetry). In general, the measurement error was below 2 mm, and correlation coefficients were very good. CONCLUSION: Various methods for measuring cranial dimensions have shown to be reliable. Stereophotogrammetry is the most versatile method for quantitative longitudinal assessment of cranial dimensions and shapes in children. However, direct anthropometry continues to be the best method for routine clinical assessments of linear cranial dimensions in growing children until age 6 years

Total daily energy expenditure relative to resting energy expenditure in clinically stable patients with COPD

BACKGROUND: An elevated resting energy expenditure (REE) commonly occurs in patients with chronic obstructive pulmonary (COPD). The purpose of this study was to investigate the effect of an increased REE on total daily energy expenditure (TDE) in 20 patients with COPD (19 men) with mean (SD) forced expiratory volume in one second of 37 (14)% predicted. METHODS: TDE was measured over a two week interval using doubly labelled water. Fat-free mass (FFM) was calculated from total body water assessed by deuterium dilution. REE was measured by indirect calorimetry using a ventilated hood system. RESULTS: The patients (10 men) with a significantly higher REE than those with a normal REE (median difference 205 kcal/ 24 h, p < 0.05) had a comparable TDE (hypermetabolic at rest: median 2593; range 2127-3083 kcal/24 h, normometabolic at rest: median 2629; range 2032-3179 kcal/ 24 h). There was no difference in mean daily heart rate (HR) between the groups (hypermetabolic at rest: median 92 (range 82-98), normometabolic at rest: median 98 (range 75-116) beats/min) or in the variation in the heart rate during the day. By means of multiple regression analysis it was shown that REE did not correlate significantly with TDE when FEM was taken into account. CONCLUSIONS: This study shows that there is no significant difference in free living TDE between clinically stable patients with COPD with a normal REE and those with an increased REE. The variation in TDE in patients with COPD appears to reflect differences in energy expenditure for activities, but not differences in REE. 



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

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

Validation of PROMIS Profile-29 in adults with hemophilia in the Netherlands

BACKGROUND: The Patient-Reported Outcomes Measurement Information System (PROMIS) Profile-29 questionnaire is widely used worldwide, but it has not yet been validated in the Netherlands, nor in persons with hemophilia. OBJECTIVE: To validate the Dutch-Flemish version of the PROMIS-29 Profile v2.01 in adults with hemophilia. METHODS: Dutch males with hemophilia (all severities) completed questionnaires that contained sociodemographic and clinical characteristics, the PROMIS-29, RAND-36, and the Hemophilia Activities List (HAL). Structural validity of each subscale was assessed with confirmatory factor analysis (CFA). Internal consistency was calculated for each subscale with sufficient model fit in CFA. Construct validity was assessed by testing hypotheses about (1) correlations of each PROMIS-29 subscale with corresponding scales of RAND-36 and domains of HAL, and (2) mean differences in T-scores between subgroups with different hemophilia severities, self-reported joint impairment, and HIV infection status. We considered ≥75% of data in accordance with the hypotheses evidence for construct validity. RESULTS: In total, 770 persons with hemophilia participated in this cross-sectional study. CFA revealed sufficient structural validity for five subscales: Physical Function, Depression, Sleep Disturbance, Ability to Participate in Social Roles and Activities, and Pain Interference. Internal consistency was high and Cronbach's alpha ranged from 0.79 for Sleep Disturbance to 0.96 for Pain Interference. Differences between clinical subgroups were in the expected direction. Construct validity was confirmed for Physical Function, Anxiety, Depression, Fatigue, Sleep Disturbance, and Pain Intensity. CONCLUSION: This study revealed sufficient evidence for structural validity, internal consistency, and construct validity for most PROMIS Profile-29 subscales among people with hemophilia in the Netherlands