Background
The worldwide pandemic spread of SARS‐CoV‐2 can lead to either respiratory infection or containment‐associated isolation with possible higher impact on chronic diseases such as inherited ...bleeding disorders (IBD). The aim of the study was to evaluate the impact of COVID‐19 on patients and caregivers of IBD patients regarding their concerns and worries related to own health, access to treatment and availability of factor concentrates and their experiences related to medical care.
Methods
Multicentre, cross‐sectional study evaluating the impact of COVID‐19 on mental health of IBD patients. An ad hoc questionnaire was developed and sent to 586 patients/caregivers with haemophilia A, haemophilia B and VWD type III. The survey included information on demographic and clinical data, needs, concerns and experiences regarding medical care during COVID‐19 pandemic.
Results
In total, 355 of the IBD‐Group (200 patients, 155 caregivers) completed the survey (61.7% response rate). Most patients suffered from haemophilia A (73.8%) and were severely affected (64.7%). Eleven patients were in quarantine due to suspected COVID‐19; none had symptoms. One quarter worried (very) strongly about getting the coronavirus, 71.3% asked themselves what will happen to them when they will get COVID‐19, 40.1% felt unchanged, and 18.9% worried about delivery difficulties of their IBD treatment product. In 52.8%, medical appointments were postponed. Significant differences between caregivers and patients were found in most aspects.
Discussion
The IBD patients affected by a chronic disorder have particular thoughts and worries regarding COVID‐19. Haemophilia specialists should be committed to address these concerns and guarantee treatment despite containment strategies.
Introduction
The use of virtual interventions is of interest to patients with chronic disease and healthcare professionals. This study aimed to determine the effect of virtual child disease ...management programme on burden and social adjustment of caregivers of children with coagulation factor deficiencies. Moreover, the effect of this intervention on children's acute pain and bleeds was assessed.
Methods
This clinical trial was conducted on 80 caregivers of children with coagulation factor deficiencies. The subjects were randomly assigned into the intervention and control groups. A comprehensive virtual child disease management programme was conducted for 8 weeks and caregiver’ burden and their social adjustment were assessed with the HEMOCAB and social adjustment subscale of Bell Adjustment Inventory, respectively. Data were analysed using ANCOVA and Wilcoxon test.
Results
Before the intervention, both groups were similar regarding the caregivers’ burden and social adjustment and children's acute pain and bleeds. However, a significant difference was observed between groups in concern to caregivers’ burden (P < .001), women's and men's social adjustment (P = .001, P = .03), and children's acute pain and bleeds (P < .001) after the virtual disease management programme.
Conclusion
This study showed that using a virtual child disease management programme reduced burden and improved social adjustment of caregivers of children with coagulation factor deficiencies. This intervention decreased frequencies of acute pain and bleeds in children. Therefore, using this effective intervention in clinical practice is warranted to decrease the caregivers’ burden as well as acute pain and bleeds in children.
Introduction
The Haem‐A‐QoL is frequently utilized in haemophilia clinical trials and captures relevant aspects of disease impact. Thresholds for some domains ‘Physical Health’ (PH), ‘Sports & ...Leisure’ (S&L) and ‘Total Score’ (TS) have previously been identified to benchmark the amount of change that is meaningful to patients, but not been independently confirmed.
Aim
The objective of this analysis was to determine the clinically important responder (CIR) thresholds for these three domains.
Methods
CIR thresholds in adult persons with haemophilia A (PwHA) enrolled in HAVEN 1, 3 and 4 studies were determined for improvements from baseline to 24 weeks of emicizumab prophylaxis using anchor‐based methodology with the EQ‐5D‐5L as the validated anchor, cumulative distribution functions (CDF) and distribution‐based methodology. The results were compared with previously published thresholds.
Results
At baseline and after 24 weeks of emicizumab prophylaxis, Haem‐A‐QoL data from 241/258 patients were available. Concordance was observed between the Haem‐A‐QoL and EQ‐5D‐5L in patterns of improvement, deterioration or lack of change. CDF estimates of the Haem‐A‐QoL PH and TS grouped by response categories on the Mobility, Pain/Discomfort and Usual Activities EQ‐5D‐5L domains demonstrated the same pattern of responses to each scale; distribution‐based estimates were 11.9 for PH, 13.9 for S&L, and 8.3 for TS.
Conclusion
Our responder thresholds are mostly consistent with those proposed by Wyrwich et al (cut‐offs of −10 and −7 for PH and TS, respectively). The majority of responders to emicizumab prophylaxis had improvements greater than the previously reported 10‐point reduction in PH and 7‐point reduction in TS.
Introduction
Persons with haemophilia A (PwHA) with factor (F)VIII inhibitors, including children, have impaired health‐related quality of life (HRQoL). The HAVEN 2 study (NCT027955767) of paediatric ...PwHA with FVIII inhibitors demonstrated that subcutaneous emicizumab prophylaxis resulted in low annualizedbleed rates.
Aim
We assessed the impact of emicizumab prophylaxis on the HRQoL of children and their caregivers participating in HAVEN 2.
Methods
Children aged 8‐11 years self‐reported HRQoL using the Haemophilia‐Specific Quality of Life Assessment Instrument for Children and Adolescents Short Form (Haemo‐QoL SF II). Caregivers of children aged 0‐11 years completed the Adapted Inhibitor‐Specific Quality of Life Assessment with Aspects of Caregiver Burden. All scores were transformed to a 0‐100 scale, where lower scores reflect a better HRQoL. The number of missed days from school/day care and hospitalizations was also recorded.
Results
In HAVEN 2 (n = 88), the median age was 6.5 years (range: 1‐15 years); 85 participants were aged < 12 years and included in this analysis, and 34 participants were aged 8‐11 years, thereby eligible to complete the Haemo‐QoL SF II questionnaire. The mean (standard deviation, n) baseline Haemo‐QoL SF II ‘Total’ score was 30.2 (14.9, 30), indicating moderate impairment; with emicizumab, mean score decreased by −9.62 (7.73, 17) points to 23.0 (13.93, 20) by Week 49. The most improved domains were ‘Sports & School’ and ‘Physical Health’. Caregivers reported similar improvements.
Conclusion
Prophylactic emicizumab is accompanied by substantial and sustained improvements in HRQoL of paediatric PwHA with FVIII inhibitors and their caregivers.
Introduction
Severe haemophilia A (HA) has a major impact on health‐related quality of life (HRQoL).
Aim
Assess the impact of emicizumab on HRQoL in persons with severe HA (PwHA) without factor VIII ...(FVIII) inhibitors in the phase 3 HAVEN 3 and 4 studies.
Methods
This pooled analysis examines the HRQoL of PwHA aged ≥ 18 years treated with emicizumab prophylaxis via Haemophilia‐Specific Quality of Life Questionnaire for Adults (Haem‐A‐QoL) and EuroQoL 5‐Dimensions 5‐levels (EQ‐5D‐5L). In particular, changes from baseline in Haem‐A‐QoL ‘Physical Health’ (PH) domain and ‘Total Score’ (TS) are evaluated.
Results
Among 176 evaluable participants, 96 (55%) had received prior episodic treatment and 80 (45%) prophylaxis; 70% had ≥ 1 target joint and 51% had experienced ≥ 9 bleeds in the previous 24 weeks. Mean Haem‐A‐QoL PH and TS improved after emicizumab initiation. Mean (standard deviation) –12.0 (21.26)‐ and –8.6 (12.57)‐point improvements were observed in PH and TS from baseline to Week 73; Week 73 scores were 27.9 (24.54) and 22.0 (14.38), respectively. Fifty‐four percent of participants reported a clinically meaningful improvement in PH scores (≥ 10 points) by Week 73. Subgroups with poorer HRQoL prior to starting emicizumab (i.e. receiving episodic treatment, ≥ 9 bleeds, target joints) had the greatest improvements in PH scores, and corresponding reductions in missed workdays; change was not detected among those previously taking prophylaxis. No change over time was detected by the EQ‐5D‐5L questionnaire.
Conclusions
Emicizumab prophylaxis in PwHA without FVIII inhibitors resulted in persistent and meaningful improvements in Haem‐A‐QoL PH and less work disruption than previous treatment.
Introduction
Patient‐reported outcomes (PROs) in people living with haemophilia A (PLWHA) are often under‐reported. Investigating PROs from a single study with a diverse population of PLWHA is ...valuable, irrespective of FVIII product or regimen.
Aim
To report available data from the Expanding Communications on Haemophilia A Outcomes (ECHO) registry investigating the associations of patient, treatment and disease characteristics with PROs and clinical outcomes in PLWHA.
Methods
ECHO (NCT02396862), a prospective, multinational, observational registry, enrolled participants aged ≥16 years with moderate or severe haemophilia A using any product or treatment regimen. Data collection, including a variety of PRO questionnaires, was planned at baseline and annually for ≥2 years. Associations between PRO scores and patient, treatment and disease characteristics were determined by statistical analyses.
Results
ECHO was terminated early owing to logistical constraints. Baseline data were available from 269 PLWHA from Europe, the United States and Japan. Most participants received prophylactic treatment (76.2%), with those using extended‐half‐life products (10.0%) reporting higher treatment satisfaction. Older age and body weight >30 kg/m2 (>BMI) were associated with poorer joint health. Older age was associated with poorer physical functioning and work productivity. Health‐related quality of life and pain interference also deteriorated with age and >BMI; >BMI also increased pain severity scores.
Conclusion
ECHO captured a variety of disease characteristics, treatment patterns, PROs and clinical outcomes obtained in real‐world practice with ≤1 year's follow‐up. Older age, poorer joint health and >BMI adversely affected multiple aspects of participant well‐being.
Introduction
Real‐world data on health‐related outcomes in persons with haemophilia A (PwHA) can provide useful information for improving patient care. The global, non‐interventional study (NIS; ...NCT02476942) prospectively collected high‐quality data in PwHA, including those without factor VIII (FVIII) inhibitors treated according to local routine clinical practice.
Aim
To report health‐related quality of life (HRQoL) and health status of adult/adolescent PwHA without FVIII inhibitors.
Methods
Participants were PwHA without FVIII inhibitors age ≥12 years; they remained on existing episodic treatment or prophylaxis. HRQoL was assessed by Haemophilia Quality of Life Questionnaire for Adults (Haem‐A‐QoL) or Haemophilia‐Specific Quality of Life Assessment for Children and Adolescents Short Form (Haemo‐QoL‐SF II). Health status was assessed through EuroQol 5‐Dimensions 5‐Levels (EQ‐5D‐5L) index utility score and visual analogue scale (EQ‐VAS).
Results
Ninety‐four participants enrolled; median age was 34.0 years (range 12–76). Forty‐five received episodic treatment and 49 received prophylaxis for a median time of 27.7 weeks and 30.4 weeks, respectively. Mean (standard deviation) baseline Haem‐A‐QoL total scores were 40.1 (17.0) for the episodic group and 26.6 (14.6) for the prophylaxis group, indicating impairments in HRQoL, which remained consistent over time. Mean EQ‐5D‐5L IUS scores were similar between treatment regimens (0.8 episodic; 0.9 prophylaxis) and consistent over time. The mean EQ‐VAS scores were similar between treatment regimens, and lower on days when bleeding occurred (79.0 vs 85.0 for episodic treatment; 77.0 vs 82.0 for prophylaxis, respectively).
Conclusions
Adult and adolescent PwHA without FVIII inhibitors had HRQoL impairments regardless of whether they were treated with episodic or prophylactic standard care with FVIII.
Introduction
Health‐related quality (HRQoL) evaluations are considered essential outcomes in the assessment of people with haemophilia. In developing countries, reliable HRQoL data are even more ...critical whilst enabling government agencies to develop national haemophilia care programmes. However, validated tools are not yet available in sub‐Saharan African countries.
Aims
This study sought to perform a cultural adaptation and validation of the Haemophilia Quality of Life Questionnaire for Adults (Haem‐A‐QoL) in Côte d'Ivoire.
Methods
The process comprised several steps, such as linguistic adaptation, cognitive debriefing interviews with adult haemophilia patients and psychometric testing, including reliability (internal consistency, test‐retest reliability) and validity assessments (convergent with EQ‐5D‐5L, criterion with HJHS 2.1, known‐groups).
Results
The final Ivoirian Haem‐A‐QoL version was obtained in December 2017 following linguistic adaptation and cognitive debriefings with six participants. The validation process included 25 patients, mainly haemophilia A patients (88%) with severe forms (80%). All participants received on‐demand treatment, with joint impairment observed in 92%. Internal consistency and test‐retest reliability of the Ivoirian Haem‐A‐QoL were very good. A Pearson correlation analysis revealed a moderate negative correlation between EQ‐VAS and total Haem‐A‐QoL scores and a moderate positive correlation between HJHS 2.1 and total Haem‐A‐QoL scores.
Conclusions
A cross‐culturally adapted and validated Haem‐A‐QoL version in Côte d'Ivoire is now available, enabling measurement of intervention outcomes in the targeted population and Ivorian participation to multisite international trials. However, further work is needed to ensure optimal understanding of HRQoL questionnaires, previously developed in culturally distinct countries, with almost unlimited access to different treatment regimens.
Objectives
To report the final results of the 2‐year TAURUS study, assessing weekly prophylaxis dosing regimens of octocog alfa (Kovaltry®/BAY 81–8973) used in standard clinical practice in patients ...with moderate‐to‐severe haemophilia A.
Methods
TAURUS (NCT02830477) is a phase 4, multinational, prospective, non‐interventional, single‐arm study in patients of any age with moderate or severe haemophilia A (≤5% factor FVIII activity). TAURUS was designed to primarily investigate weekly prophylaxis dosing regimens used in standard clinical practice. Annualised bleeding rates (ABRs), treatment satisfaction and adherence, and safety were also assessed.
Results
Of 302 patients included in the full analysis set, 84.4% (n = 255) maintained their octocog alfa prophylaxis baseline regimen throughout the study, with a majority of patients (76.5%, n = 231) on two times or three times weekly regimens at the end of the observation period (≥1–≤2 years). ABRs, treatment satisfaction, and adherence remained stable during the observation period. Octocog alfa was well tolerated and there were no new or unexpected adverse events.
Conclusions
These data show that a smooth transition is observed when switching to octocog alfa from a previous FVIII treatment, with no safety issues and stable bleeding rates in a real‐world setting of patients with moderate‐to‐severe haemophilia A.
Aims and objectives
To describe the perceived burden on parents of children with severe or moderate haemophilia and the impact of sociodemographic aspects and the child's medical condition on this.
...Background
Parents of children with haemophilia face a multitude of demands. The child needs frequent intravenous injections, hospital visits, extra supervision and care. The child's illness and related management might have psychosocial effects on the parents.
Design
A multicentre, cross‐sectional study.
Methods
Caregiver burden was evaluated in 102 parents of children with haemophilia, using the HEMOCAB questionnaire which consists of 54 items divided into 13 domains which are to be answered on a 5‐point Likert scale. To assess the impact of sociodemographic aspects and clinical data on parental burden, linear regression analyses were undertaken. The study followed the STROBE checklist throughout the research process.
Results
Greater burden was seen in parents of children with past or present inhibitors, in parents of younger children, if a family member administered the clotting factor and in parents of children with overweight/obesity. No significant differences in burden were observed for type of haemophilia, if the child had bleeding in the past 12 months, if the child self‐infused, had another family member with haemophilia or if the parent had more children.
Conclusions
Parental perceived burden can be negatively affected by the child's medical condition; our results underline that healthcare professionals need to be aware of increased parental burden if the child is young, has or had inhibitors and has overweight/obesity.
Relevance to clinical practice
More psychosocial support from the healthcare professionals needs to be directed towards parents of younger children and particularly the parents of young children with inhibitors, thus decreasing the parental burden. Parental burden may be reduced if healthcare professionals more actively treat overweight and refer children to appropriate specialists.
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