Real‐world comparative analysis of bleeding complications and health‐related quality of life in patients with haemophilia A and haemophilia B

Booth, J.; Oladapo, Abiola; Walsh, Shaun; O’Hara, Jamie; Carroll, Liz; Diego, Daniel-Anibal Garcia; O’Mahony, Brian

doi:10.1111/hae.13596

Cited by 11 publications

(14 citation statements)

References 24 publications

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“…The majority of patients reported mild or moderate pain (76%) and impact of haemophilia on daily life (63%), consistent with published reports, particularly for patients with moderate to severe disease [ 4 , 24 ]. The overall mean EQ-5D score among CHESS II patients with severe disease (mean 0.67) was slightly lower than that reported from severe haemophilia B patients in CHESS I (mean 0.76) [ 25 ]. This may be reflective of a persistent unmet need over time.…”

Section: Discussionmentioning

confidence: 81%

Clinical, humanistic, and economic burden of severe haemophilia B in adults receiving factor IX prophylaxis: findings from the CHESS II real-world burden of illness study in Europe

Burke

Asghar

O’Hara

et al. 2021

Orphanet J Rare Dis

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Background Real-world studies of the burden of severe haemophilia B in the context of recent therapeutic advances such as extended half-life (EHL) factor IX (FIX) products are limited. We analysed data from the recent CHESS II study to better understand the clinical, humanistic, and economic burden of severe haemophilia B in Europe. Data from male adults with severe haemophilia B receiving prophylaxis were analysed from the retrospective cross-sectional CHESS II study conducted in Germany, France, Italy, Spain and the United Kingdom. Inhibitors were exclusionary. Patients and physicians completed questionnaires on bleeding, joint status, quality of life, and haemophilia-related direct and indirect costs (2019–2020). All outcomes were summarised using descriptive statistics. Results A total of 75 CHESS II patients were eligible and included; 40 patients (53%) provided self-reported outcomes. Mean age was 36.2 years. Approximately half the patients were receiving EHL versus standard half-life (SHL) prophylaxis (44% vs 56%). Most patients reported mild or moderate chronic pain (76%) and had ≥ 2 bleeding events per year (70%), with a mean annualised bleed rate of 2.4. Mean annual total haemophilia-related direct medical cost per patient was €235,723, driven by FIX costs (€232,328 overall, n = 40; €186,528 for SHL, €290,620 for EHL). Mean annual indirect costs (€8,973) were driven by early retirement or work stoppage due to haemophilia. Mean quality of life (EQ-5D) score was 0.67. Conclusions These data document a substantial, persistent real-world burden of severe haemophilia B in Europe. Unmet needs persist for these patients, their caregivers, and society.

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Section: Discussionmentioning

confidence: 81%

Clinical, humanistic, and economic burden of severe haemophilia B in adults receiving factor IX prophylaxis: findings from the CHESS II real-world burden of illness study in Europe

Burke

Asghar

O’Hara

et al. 2021

Orphanet J Rare Dis

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“…health-related quality of life (HRQoL) such as social functioning (eg interactions with others, family planning), role functioning (eg ability to attend work/school) and emotional functioning. [3][4][5][6][7][8] Understanding the impact of the disease on persons with HA (PwHA) is critical, and characterizing the improvement in physical function from treatments that significantly reduce bleeds is of high interest. Physical function and impact on HRQoL are frequently captured in clinical trials via patient-reported outcomes (PROs), including the Haemophilia-specific Health-related Quality of Life Questionnaire for Adults (Haemo-QoL-A), Hemofilia-QoL and Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL).…”

Section: Introductionmentioning

confidence: 99%

“…Haemophilia A (HA) is a congenital bleeding disorder characterized by spontaneous or traumatic bleeding particularly into joints and muscles, and potential resultant deterioration in physical function 1,2 . Moreover, HA and complications such as factor (F)VIII inhibitor development can impact aspects of patients’ health‐related quality of life (HRQoL) such as social functioning (eg interactions with others, family planning), role functioning (eg ability to attend work/school) and emotional functioning 3‐8 . Understanding the impact of the disease on persons with HA (PwHA) is critical, and characterizing the improvement in physical function from treatments that significantly reduce bleeds is of high interest.…”

Section: Introductionmentioning

confidence: 99%

Determining meaningful health‐related quality‐of‐life improvement in persons with haemophilia A using the Haemophilia Quality of Life Questionnaire for Adults (Haem‐A‐QoL)

et al. 2020

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“…Previous studies investigating predictors of ABR identified that endogenous factor levels, adherence, BMI, primary dosing regimen, inhibitor development, presence of arthropathy, and intensity of physical activity as variables that can influence bleeding rates. 1,4,[26][27][28][29][30] In the present analysis, the post-N8-GP 12-week prophylaxis model demonstrated that total bleed count up to 12 weeks postprophylaxis switch, baseline vWF level, and mean FVIII at 30 minutes postdose were the most important variables for predicting observed ABR with N8-GP prophylaxis. The mean absolute SHAP values demonstrated that the total bleed count was substantially more informative than all other baseline characteristics (vWF level) and PK measures (mean FVIII at 30 minutes and mean trough level), demonstrating the importance of clinical observation for predicting long-term outcomes.…”

Section: Discussionmentioning

confidence: 49%

Predictive Modeling Identifies Total Bleeds at 12-Weeks Postswitch to N8-GP Prophylaxis as a Predictor of Treatment Response

et al. 2021

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Background Predicting annualized bleeding rate (ABR) during factor VIII (FVIII) prophylaxis for severe hemophilia A (SHA) is important for long-term outcomes. This study used supervised machine learning-based predictive modeling to identify predictors of long-term ABR during prophylaxis with an extended half-life FVIII. Methods Data were from 166 SHA patients who received N8-GP prophylaxis (50 IU/kg every 4 days) in the pathfinder 2 study. Predictive models were developed to identify variables associated with an ABR of ≤1 versus >1 during the trial's main phase (median follow-up of 469 days). Model performance was assessed using area under the receiver operator characteristic curve (AUROC). Pre-N8-GP prophylaxis models learned from data collected at baseline; post-N8-GP prophylaxis models learned from data collected up to 12-weeks postswitch to N8-GP, and predicted ABR at the end of the outcome period (final year of treatment in the main phase). Results The predictive model using baseline variables had moderate performance (AUROC = 0.64) for predicting observed ABR. The most performant model used data collected at 12-weeks postswitch (AUROC = 0.79) with cumulative bleed count up to 12 weeks as the most informative variable, followed by baseline von Willebrand factor and mean FVIII at 30 minutes postdose. Univariate cumulative bleed count at 12 weeks performed equally well to the 12-weeks postswitch model (AUROC = 0.75). Pharmacokinetic measures were indicative, but not essential, to predict ABR. Conclusion Cumulative bleed count up to 12-weeks postswitch was as informative as the 12-week post-switch predictive model for predicting long-term ABR, supporting alterations in prophylaxis based on treatment response.

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Real‐world comparative analysis of bleeding complications and health‐related quality of life in patients with haemophilia A and haemophilia B

Abstract: Data suggest comparable ABR, incidence of target joints and HRQoL between patients with HB and HA indicating comparable clinical severity and disease impact on patient quality of life.

Cited by 11 publications

References 24 publications

Clinical, humanistic, and economic burden of severe haemophilia B in adults receiving factor IX prophylaxis: findings from the CHESS II real-world burden of illness study in Europe

Clinical, humanistic, and economic burden of severe haemophilia B in adults receiving factor IX prophylaxis: findings from the CHESS II real-world burden of illness study in Europe

Determining meaningful health‐related quality‐of‐life improvement in persons with haemophilia A using the Haemophilia Quality of Life Questionnaire for Adults (Haem‐A‐QoL)

Predictive Modeling Identifies Total Bleeds at 12-Weeks Postswitch to N8-GP Prophylaxis as a Predictor of Treatment Response

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