Quality of life in children and adolescents with blood coagulation disorders and hemoglobinopathies.

The aim of this study was to evaluate the impact of oral conditions and health-related quality of life (HRQoL) on oral health-related quality of life (OHRQoL) in children and adolescents with blood coagulation disorders and hemoglobinopathies (BCDH). The study was cross-sectional and included 61 individuals aged 2 to 18 years with BCDH. Exams for dental caries (dmft/DMFT index), oral hygiene (simplified oral hygiene index - OHI-S), and gingival health (modified gingival index - MGI) were performed. The pediatric quality of life inventory™ (PedsQL™) generic core scale and oral health scale were used to measure HRQoL and OHRQoL. Spearman's correlation coefficient (ρ) and the Mann-Whitney test (α = 0.05) were conducted to assess the relationship between covariates and the PedsQL™ oral health scale. The mean PedsQL™ oral health scale score was 76.66 (SD = 21.36). Worse OHRQoL was correlated with poor oral hygiene (ρ = -0.383; p: 0.004), poor gingival health (ρ = -0.327; p = 0.014), and better HRQoL (ρ = 0.488; p < 0.001). Greater untreated dental caries experience was associated with worse OHRQoL (p = 0.009). Worse oral health status in children and adolescents with BCDH negatively impacts OHRQoL, and OHRQoL and quality of life analyzed from a generic perspective are positively correlated constructs in this population.

Improvements in Communication and Coordination of Care in a Hemophilia Treatment Center.

INTRODUCTION: In response to the increasing complexity of care for patients with bleeding disorders, we established new clinical teams for our hemophilia treatment center (HTC). AIMS: We undertook a quality improvement project to improve the coordination and communication with our patients by establishing primary assignments of clinical staff to individual patients (primary teams). METHODS: A quality improvement project group was formed that established the goals and assignment of primary teams. Patients were surveyed for their knowledge of their primary teams as well as their ability to schedule and contact their primary providers. As a measure of the effects on clinical staff, a balancing survey was also conducted among providers impacted by the clinical assignment of teams. RESULTS: Our results demonstrate improvements across both coordination and communication as reported by patients. Additionally, the assignment of primary teams was met with high satisfaction and improvement in coordination and communication as reported by the clinical staff members of the HTC. CONCLUSIONS: Initiation of a quality improvement project and the creation of a primary team system were feasible at a large HTC and resulted in improvements in both patient-reported and staff-reported outcomes of coordination and communication of care.

Cross-Sectional and Longitudinal Construct Validity of the Generic KINDL-A(dult)B(rief) Questionnaire in Adults with Thrombophilia or with Hereditary and Acquired Bleeding Disorders.

BACKGROUND/AIMS: The newly adapted generic KINDL-A(dult)B(rief) questionnaire showed satisfactory cross-sectional psychometric properties in adults with bleeding disorders or thrombophilia. This investigation aimed to evaluate its cross-sectional and longitudinal construct validity. METHODS: After ethical committee approval and written informed consent, 335 patients (mean age 51.8 ± 16.6 years, 60% women) with either predominant thrombophilia (n = 260) or predominant bleeding disorders (n = 75) participated. At baseline, patients answered the KINDL-AB, the MOS 36-item Short-Form Health Survey (SF-36), and the EQ-5D-3L. A subgroup of 117 patients repeated the questionnaire after a median follow-up of 2.6 years (range: 0.4-3.5). A priori hypotheses were evaluated regarding convergent correlations between KINDL-AB overall well-being and specific subscales, EQ-5D-3L index values (EQ-IV), EQ-5D visual analog scale (EQ-VAS), and SF-36 subscales. RESULTS: Contrary to hypothesis, baseline correlations between the KINDL-AB and EQ-IV/EQ-VAS were all moderate while, as hypothesized, several KINDL-AB subscales and SF-36 subscales correlated strongly. At follow-up, no significant changes in all three instruments occurred. Correlations between instruments over the follow-up were mostly moderate and partially strong. Contrary to hypothesis but consistent with no significant changes in health-related quality of life, convergent correlations between changes in KINDL-AB overall well-being, physical and psychological well-being, and EQ-IV/EQ-VAS were all weak. CONCLUSIONS: While repeated measures of KINDL-AB showed moderate to strong correlations, changes in KINDL-AB overall well-being and subscales correlated more weakly than expected with changes involving two established instruments of generic health status.

Exploring changes in distress among individuals with bleeding disorders: What is linked to improvements in distress?

Adults with chronic bleeding disorders report high distress, but it is unclear how distress varies over time. Patients rated their distress on a 10-point scale at two clinic visits. Of 83 patients, roughly one-quarter reported consistent no/low distress (29%), one-quarter reported consistent distress (22%), and half (49%) reported a change in distress of at least two points. Overall activity levels, depressive symptoms, and non-White race were significantly associated with worsening and consistent distress in adjusted analyses while improvements in activity levels and depressive symptoms during the study period were associated with distress improvement. Our results suggest that distress is modifiable.

Psychometric properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire.

OBJECTIVE: To assess the psychometric properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire. METHODS: This study was a cross-sectional, multinational study. Participants were enrolled if they were more than 10 years old and people with haemophilia A or B or people without a bleeding disorder. Participants were invited through non-governmental patient organisations in 21 countries between 01/27/2016 and 02/23/2017. The following psychometric properties: missing data, floor and ceiling effects, exploratory factor analysis and internal consistency reliability were examined. A PROBE Score was derived and assessed for its convergent and known groups validity. RESULTS: The study analysed the data on 916 participants with median age of 37.0 (IQR 27.0 to 48.0) years, 74.8% male. In the domain assessing patient-reported outcomes (PROs), more than 15% of participants presented a ceiling effect for all items but two, and a floor effect for one item. Factor analysis identified three factors explaining the majority of the variance. Cronbach's alpha coefficient indicated good internal consistency reliability (0.84). PROBE items showed moderate to strong correlations with corresponding EuroQol five dimension 5-level instrument (EQ-5D-5L) domains. The PROBE Score has a strong correlation (r=0.67) with EQ-5D-5L utility index score. The PROBE Score has a known groups validity among various groups. CONCLUSIONS: The results of this study suggest that PROBE is a valid questionnaire for evaluating PROs in people with haemophilia as well as control population. The known-group property of PROBE will allow its use in future clinical trials, longitudinal studies, health technology assessment studies, routine clinical care or registries. Additional studies are needed to test responsiveness and sensitivity to change. TRIAL REGISTRATION NUMBER: NCT02439710; Results.

Psychometric Properties of a Modified KINDL-R Questionnaire for Adolescents and Adults, and Construction of a Brief Version, the KINDL-A(dult)B(rief) Questionnaire, KINDL-AB.

BACKGROUND/AIMS: The generic quality of life KINDL-R -questionnaire is validated for use in children/adolescents ≤16 years. The aim of this cross-sectional investigation was to modify the KINDL-R questionnaire for use in adults and to validate its psychometric properties. METHODS: Five items of the KINDL-R questionnaire were adapted and the newly developed KINDL-A(dult) questionnaire administered to 255 patients with hereditary and acquired bleeding disorders (mean age 53 years). Its internal consistency and convergent and divergent construct validity were investigated and confirmatory factor analysis was used to evaluate the latent factor structure. RESULTS: The KINDL-A questionnaire showed satisfactory reliability, varying construct validity, but inconclusive factor structure. The KINDL-AB(rief) was developed by removing half of the items and combining 2 sub-axes. This led to factor loadings between 0.62 and 0.91 and increased overall fit (Goodness of fit > 0.8 and Root Mean Square Error of Approximation, RMSEA, < 0.08). Results were validated in 966 healthy blood donors (mean age 38 years). In this group, the KINDL-AB questionnaire showed factor loadings between 0.43 and 0.77, Goodness of fit > 0.95 and RMSEA < 0.05. CONCLUSIONS: The new KINDL-AB suggests sufficient to good psychometric properties in adult patients with hereditary and acquired bleeding disorders.

Assessment of an Electronic Intervention in Young Women with Heavy Menstrual Bleeding.

STUDY OBJECTIVE, DESIGN, SETTING, PARTICIPANTS, INTERVENTIONS, AND MAIN OUTCOME MEASURES: Bleeding disorders (BD) occur in up to 50% of adolescents with heavy menstrual bleeding (HMB). This presents unique challenges to health care providers because of the complexity of treating the condition and such complexity can result in difficulty with patients understanding basic information about their condition, limit communication with medical providers, and patient compliance. The aim of the study was to use an electronic approach to enhance patient compliance with medications used to treat their HMB, and to provide educational access to adolescents with BD. This was a prospective cohort study involving patients in a Young Women's Bleeding Disorder Clinic at a single children's hospital. Subjects were given an iPod Touch (Apple Inc, Cupertino, CA) device (ITD), preloaded with the iPeriod (Winkpass Creations) application. Participants recorded information about their BD that they learned about on BD Web sites, and menses, and medications. Electronic and charted data were collected to monitor compliance with prescribed treatment regimens. All ITD allowed Wi-Fi access to allow teens to explore BD Web sites and knowledge was assessed. RESULTS: Twenty-three of 45 subjects completed the study. The mean age was 14.1 ± 1.9 years. Subjects who were compliant with the ITD (group 1), charted on baseline symptoms, menstrual flow (83.3%), cramps (100%, 23/23), breakthrough bleeding (95.6%, 22/23), mood (95.6%, 22/23), and medication use (91.7%) for a mean of 9.3 ± 3.1 months. Subjects who were nonusers (group 2) did not report on symptoms, their condition, or medication use in the device (n = 22). More than 75% (17/23) of subjects in group 1 used hormones alone or hormones with antifibrinolytic agents to control HMB. No subjects stopped or missed medications who were in group 1 intentionally, and also there were 9 enrollees within this same group who missed a medication related to awaiting the prescription to be filled from pharmacy. In group 2, 17 enrollees missed medications, resulting in 19% (4/22) of these enrollees being admitted to hospital for 1-2 days. In addition, enrollees in group 2 missed more medications on average compared with group 1. No subjects in group 1 required admission for HMB treatment failure during the study period, compared with those in group 2 (P = .006). All subjects in group 1 reported accessing Web sites using their ITD to learn about their BD. Groups 1 and 2 did not differ in the number of medications that were prescribed during the time frame (P = .77) or the number of follow-up clinic visits (P = .49). Furthermore, those in group 1 reported fewer breakthrough bleeding episodes than those in group 2 according to clinic notes (P = .03). Users of the ITD were given a set of knowledge questions. Group 2 subjects were not consistent users of the ITD use and did not complete the knowledge questions. Group 1 and 2 could not be compared with regard to knowledge as a result. CONCLUSION: ITD is an excellent tool for adolescents with HMB and BD to allow self-monitoring, provider monitoring, and improve educational access through engaging technology; compliance with device use was associated with several parameters suggestive of improved clinical outcomes.

Health-related quality of life in children, adolescents and adults with hereditary and acquired bleeding disorders.

BACKGROUND: To better understand self-reported health-related quality-of-life (HrQoL) in children and adults with chronic hemostatic conditions compared with healthy controls. METHODS/PATIENTS/RESULTS: Group 1 consisted of 74 children/adolescents aged 8-18years with hereditary bleeding disorders (H-BD), 12 siblings and 34 peers. Group 2 consisted of 82 adult patients with hereditary/acquired bleeding disorders (H/A-BD), and group 3 of 198 patients with deep venous thrombosis (DVT) on anticoagulant therapy. Adult patients were compared to 1011 healthy blood donors. HrQoL was assessed with a 'revised KINDer Lebensqualitaetsfragebogen' (KINDL-R)-questionnaire adapted to adolescents and adults. No differences were found in multivariate analyses of self-reported HrQoL in children with H-BD. In contrast, apart from family and school-/work-related wellbeing in female patients with DVT the adult patients showed significantly lower HrQoL sub-dimensions compared to heathy control subjects. Furthermore, adults with H/A-BD disorders reported better friend-related HrQoL compared to patients with DVT, mainly due to a decreased HrQoL subscale in women on anticoagulation. CONCLUSION: In children with H-BD, HrQoL was comparable to siblings and peers. In adults with H/A-BD HrQoL was comparable to patients with DVT while healthy blood donors showed better HrQoL. The friend-related HrQoL subscale was significantly reduced in female compared to male patients.

Health-Related Quality of Life in Children and Adolescents with Hereditary Bleeding Disorders and in Children and Adolescents with Stroke: Cross-Sectional Comparison to Siblings and Peers.

Objectives. To investigate self-reported health-related quality of life (HrQoL) in children and adolescents with chronic medical conditions compared with siblings/peers. Methods. Group 1 (6 treatment centers) consisted of 74 children/adolescents aged 8-16 years with hereditary bleeding disorders (HBD), 12 siblings, and 34 peers. Group 2 (one treatment center) consisted of 70 children/adolescents with stroke/transient ischemic attack, 14 siblings, and 72 peers. HrQoL was assessed with the "revised KINDer Lebensqualitätsfragebogen" (KINDL-R) questionnaire. Multivariate analyses within groups were done by one-way ANOVA and post hoc pairwise single comparisons by Student's t-tests. Adjusted pairwise comparisons were done by hierarchical linear regressions with individuals nested within treatment centers (group 1) and by linear regressions (group 2), respectively. Results. No differences were found in multivariate analyses of self-reported HrQoL in group 1, while in group 2 differences occurred in overall wellbeing and all subdimensions. These differences were due to differences between patients and peers. After adjusting for age, gender, number of siblings, and treatment center these differences persisted regarding self-worth (p = .0040) and friend-related wellbeing (p < .001). Conclusions. In children with HBD, HrQoL was comparable to siblings and peers. In children with stroke/TIA HrQoL was comparable to siblings while peers, independently of relevant confounder, showed better self-worth and friend-related wellbeing.

Menstrual management and reproductive concerns in adolescent and young adult women with underlying hematologic or oncologic disease.

PURPOSE OF REVIEW: Heavy menstrual bleeding is common among adolescent and young adult women, and can affect health-related quality of life. The cause of heavy menstrual bleeding is not uncommonly because of an underlying hematologic or oncologic disease process, which substantially influences the way patients are counseled and treated. RECENT FINDINGS: Options for menstrual management are more numerous today than ever before and range from minimizing monthly blood loss to suppressing the cycle altogether. However, an underlying bleeding disorder or malignancy can introduce many nuances and limits in individual patient care, which this review highlights. Additionally, because survival rates for adolescent and young adult cancers are improving, more of these patients are planning for lives after their disease, which may include starting or adding to a family. Options for fertility preservation during cancer therapy regimens are solidifying and both primary practitioners and subspecialists should be aware of the possibilities. SUMMARY: Patients with underlying hematologic or oncologic disease require management of menstrual bleeding, but also deserve a comprehensive evaluation and counseling regarding their individualized contraceptive needs and fertility preservation options during their reproductive years. This review employs the latest evidence from current literature to help guide clinicians caring for this unique demographic.

Providing young women with credible health information about bleeding disorders.

BACKGROUND: Approximately 1% of U.S. women may have an undiagnosed bleeding disorder, which can diminish quality of life and lead to life-threatening complications during menstruation, childbirth, and surgery. PURPOSE: To understand young women's knowledge, attitudes, and perceptions about bleeding disorders and determine the preferred messaging strategy (e.g., gain- versus loss-framed messages) for presenting information. METHODS: In September 2010, a web-assisted personal interview of women aged 18-25 years was conducted. Preliminary analyses were conducted in 2011 with final analyses in 2013. In total, 1,243 women participated. Knowledge of blood disorders was tabulated for these respondents. Menstrual experiences of women at risk for a bleeding disorder were compared with those not at risk using chi-square analyses. Perceived influence of gain- versus loss-framed messages also was compared. RESULTS: Participants knew that a bleeding disorder is a condition in which bleeding takes a long time to stop (77%) or blood does not clot (66%). Of the women, 57% incorrectly thought that a bleeding disorder is characterized by thin blood; many were unsure if bleeding disorders involve blood types, not getting a period, or mother and fetus having a different blood type. Women at risk for a bleeding disorder were significantly more likely to report that menstruation interfered with daily activities (36% vs 9%); physical or sports activities (46% vs 21%); social activities (29% vs 7%); and school or work activities (20% vs 9%) than women not at risk. Gain-framed messages were significantly more likely to influence women's decisions to seek medical care than parallel loss-framed messages. Findings suggest that the most influential messages focus on knowing effective treatment is available (86% gain-framed vs 77% loss-framed); preventing pregnancy complications (79% gain- vs 71% loss-framed); and maintaining typical daily activities during menstrual periods. CONCLUSIONS: Lack of information about bleeding disorders is a serious public health concern. Health communications focused on gain-framed statements might encourage symptomatic young women to seek diagnosis and treatment. These findings and corresponding recommendations align with Healthy People 2020 and with CDC's goal of working to promote the health, safety, and quality of life of women at every life stage.

Posttraumatic stress disorder and cardiometabolic disease.

The need for addressing posttraumatic stress disorder (PTSD) among combat veterans returning from Afghanistan and Iraq is a growing public health concern. Current PTSD management addresses psychiatric parameters of this condition. However, PTSD is not simply a psychiatric disorder. Traumatic stress increases the risk for inflammation-related somatic diseases and early mortality. The metabolic syndrome reflects the increased health risk associated with combat stress and PTSD. Obesity, dyslipidemia, hypertension, diabetes mellitus, and cardiovascular disease are prevalent among PTSD patients. However, there has been little appreciation for the need to address these somatic PTSD comorbidities. Medical professionals treating this vulnerable population should screen patients for cardiometabolic risk factors and avail themselves of existing preventive diet, exercise, and pharmacologic modalities that will reduce such risk factors and improve overall long-term health outcomes and quality of life. There is the promise that cardiometabolic preventive therapy complementing psychiatric intervention may, in turn, help improve the posttraumatic stress system dysregulation and favorably impact psychiatric and neurologic function. © 2013 S. Karger AG, Basel.

Can activation of coagulation and impairment of fibrinolysis in patients with anxiety and depression be reversed after improvement of psychiatric symptoms? Results of a pilot study.

Anxiety and depression are associated with an activation of coagulation and impairment of fibrinolysis. This study addresses the question whether these findings are reversed after psychotherapy and improvement of psychiatric symptoms. Three factors of coagulation and fibrinolysis as well as level of anxiety and depression were reassessed in 12 patients 1 to 3 years after intensive inpatient psychotherapy. The patients showed a substantial improvement of their severe anxiety disorder and comorbid depressive disorder. Simultaneously, we found a significant decrease in factor VII and plasminogen activator inhibitor. We conclude that reduction of severe anxiety and depression may be associated with a reversal of the procoagulant effect (activation of coagulation and impairment of fibrinolysis) of these psychological states. Because of the small sample size of this pilot study, further research is needed.

Health-related quality of life and patient satisfaction after global endometrial ablation for menorrhagia in women with bleeding disorders: a follow-up survey and systematic review.

OBJECTIVE: The purpose of this study was to describe health-related quality of life and satisfaction after global endometrial ablation in women with bleeding disorders and a systematic review of the literature. STUDY DESIGN: A follow-up survey was mailed to 36 patients with bleeding disorders and 110 reference patients (no coagulopathies) who underwent global endometrial ablation for menorrhagia. The survey included a generic (SF-12) and menorrhagia multi-attribute utility scale questionnaires. RESULTS: Ninety-six women (66%) responded. The total menorrhagia multiattribute utility scale score increased from 35-100 in bleeding disorder cohort (P = .03) and from 48-100 in the reference cohort (P < .001). Although postablation SF-12 mental domain scores were comparable in both cohorts (55 vs 55; P = .67), physical domain scores were lower in the bleeding disorder cohort (50 vs 56; P < .001). High satisfaction was reported by both cohorts (95% vs 84%; P = .60). CONCLUSION: Global endometrial ablation improved health-related quality of life for women with bleeding disorders and had high satisfaction rates.

Information and self-management needs of people living with bleeding disorders: a survey.

The aim of the study was to conduct a survey of information and self-management needs among members of the UK Haemophilia Society (HS). Of 1082 members mailed, 307 took part in the survey, the mean age was 52.4 years (SD 14.8), 85% were male, 96% white and 66% were living with haemophilia A. 90% felt that there is a lack of understanding amongst the public regarding bleeding disorders, 76% felt that health professionals understood their health and information needs and 32% had experienced problems managing anxiety and or depression. Results suggest that there was a 'stigma' attached to having a bleeding disorder and almost half are very careful about informing others that they have a bleeding disorder. Self-efficacy was relatively high within the sample. Many respondents wanted to be kept informed about current research and the opportunity to participate in research. Women experienced a greater need for more information than men, particularly in the area of the 'medical management' and 'emotional' topics. Men appeared to need more information and or training on 'physical' aspects such as physiotherapy and joint replacement. The study provides an insight into the educational and self-management needs of those affected by bleeding disorders and highlights the importance of providing accessible information.

Pegylated interferon and ribavirin combination therapy for chronic hepatitis C in patients with congenital bleeding disorders: a single-centre experience.

Chronic hepatitis C is a major comorbidity in patients with haemophilia. Although the current state-of-the-art therapy consists of pegylated interferon (PegIFN) and ribavirin, there are no reports of the efficacy of this combination in the haemophilia population. The aim of this study was to assess the response and side-effects of PegIFN and ribavirin in patients with inherited bleeding disorders. Patients with chronic hepatitis C were treated with PegIFN alpha-2b (1.5 microg kg(-1) week(-1)) and ribavirin (800-1200 mg day(-1)) for 24 (genotype 2 and 3) or 48 weeks (genotype 1) and followed for an additional 24 weeks. In total, 56 patients were enrolled: 31 (55%) had genotype 1, 12 (21%) genotype 2, and 13 (23%) genotype 3. Ten patients (18%) were HIV co-infected and seven (13%) had been previously treated with IFN-alpha with or without ribavirin. The overall response was 55%. In HIV-negative and treatment-naïve patients, the sustained virological response was 70%. Successful treatment was associated with genotypes 2 and 3, absence of HIV, absence of previous IFN treatment, and decrease of hepatitis C virus load at weeks 4 and 12. Although many side-effects occurred, only a minority (11%) discontinued therapy for this reason. Dose reduction of PegIFN was required in 28% and of ribavirin in 35% of patients. Overall, 22% of patients developed a depression requiring antidepressant drugs and one patient developed psychosis. In conclusion, PegIFN and ribavirin is effective in patients with inherited bleeding disorders. Treatment is safe, but severe side-effects may occur and warrant close monitoring during therapy.

Health-related quality of life in chronic coagulation disorders.

Measurement of health-related quality of life (HR-QoL) is used in patients with haemophilia as a way of assessing the effectiveness of health care, especially as cure is not possible. We report the first such study on patients with chronic coagulation disorders in Australia, using The RAND 36-item Health Survey 1.0 (SF-36), a standardized validated questionnaire combined with a semistructured interview. The mean scores for the eight domains of the SF-36 ranged from 52.5 +/- 42.1 for physical role to 80.0 +/- 20.0 for social functioning. Comparison with normative data obtained from the Australian Bureau of Statistics (ABS) demonstrated a reduction in all domains in this population with statistically significant reductions in general health, physical role limitation and vitality. Comparison with other studies indicates that the HR-QoL of patients with haemophilia and von Willebrand's disorder in Australia is comparable with other Western countries, reflecting the overall similar quality of care available to these patients. This study also provides a good cross-sectional view of the psychosocial factors of life in patients in Australia and recognizes the positive family support available to this population, while raising relevant shortcomings in schools and at the workplace that need to be studied further in a controlled manner.