The socioeconomic burden of patients affected by hemophilia with inhibitors

D’Angiolella, Lucia Sara; Cortesi, P.A.; Rocino, Angiola; Coppola, Antonio; Hassan, Hamisa Jane; Giampaolo, Adele; Solimeno, Luigi Piero; Lafranconi, Alessandra; Micale, Mariangela; Mangano, Sveva; Crotti, Giacomo; Pagliarin, Federica; Cesana, Giancarlo; Mantovani, Lorenzo Giovanni

doi:10.1111/ejh.13108

Cited by 50 publications

(58 citation statements)

References 95 publications

(219 reference statements)

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“…Additionally, inhibitors can cause substantial increases in treatment costs and disease burden. [6][7][8] Diverse factors had been related to the development of inhibitors. These include type of pathogenic variants on the F8 gene, age of first exposure, intensity of treatment, type of product, dosage, exposure conditions, use of prophylaxis vs on-demand treatment, history of surgery, product changes, family history of inhibitors, differences in immune response genes, among others.…”

Section: Introductionmentioning

confidence: 99%

Prevalence of inhibitors and clinical characteristics in patients with haemophilia in a middle‐income Latin American country

et al. 2020

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Introduction Development of inhibitors is the most serious complication in patients with haemophilia (PWH). The prevalence of inhibitors in patients with severe haemophilia A (HA) is approximately 25%‐30%. Inhibitor prevalence differs among populations. Some studies report a prevalence of almost twice in Hispanic as compared to Caucasian patients. Most data available, on the prevalence of inhibitors and their predisposing factors, originate from centres in developed countries. Aim Establish the prevalence of inhibitors of FVIII and FIX in Mexico. Methods This was an observational, cross‐sectional and descriptive study. The records of all patients diagnosed with haemophilia A (HA) or B (HB), with and without inhibitors, were included. Clinical and demographical characteristics of patients with inhibitors were assessed. Statistical analysis was performed using IBM SPSS version 22. The Ethics Committees of the various participating institutions approved this study. Results A total of 1455 patients from the 20 participating centres were recruited, from which 1208 (83.02%) had HA and 247 (16.97%) were diagnosed with HB. The presence of inhibitors in severe HA was reported in 93/777(11.96%), and 10/162 (6.17%) in severe HB. Of them, 91.7% exhibited high titres in HA and 100% in HB. Conclusion In Mexico, the general prevalence of inhibitors varies considerably among centres. This study established a basis of comparison for future development and advances in the treatment and follow‐up of patients. These findings also augment our understanding of risk factors related to inhibitor development.

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Section: Introductionmentioning

confidence: 99%

Prevalence of inhibitors and clinical characteristics in patients with haemophilia in a middle‐income Latin American country

et al. 2020

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“…The economic and psychological impacts of having a child with haemophilia are well recognized . Moreover, treatment burden for the person with haemophilia has been documented . The burden in caring for a child is recognized for more common chronic diseases such as asthma, diabetes, cancer or cystic fibrosis .…”

Section: Introductionmentioning

confidence: 99%

“…13 Moreover, treatment burden for the person with haemophilia has been documented. 14,15 The burden in caring for a child is recognized for more common chronic diseases such as asthma, diabetes, cancer or cystic fibrosis. 16 However, there remains a paucity of data about caregiver burden in haemophilia.…”

Section: Introductionmentioning

confidence: 99%

The burden of bleeds and other clinical determinants on caregivers of children with haemophilia (the BBC Study)

et al. 2019

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Introduction Treatment burden for the people with haemophilia has been documented, as has the burden of caring for a child with a common chronic disease such as asthma or diabetes. However, there remains a paucity of data about caregiver burden in haemophilia. Aims The aim of this study was to evaluate the impact of bleeding on caregivers of children with haemophilia. Caregiver burden was stratified by the clinical status of their child. Methods A multinational, non‐interventional study of caregivers of children with severe or moderate haemophilia, using the HEMOCABquestionnaire to evaluate caregiver burden. Results A total of 144 caregivers from seven EU countries participated in the study. Differences in caregiver burden were identified based on the clinical situation of the child. Greater burden was seen in caregivers of children who experienced joint bleeding in the preceding 12 months, or had target joints or reduced range of motion in most domains of the HEMOCAB. Caring for a child with a current inhibitor also caused significantly higher burden for caregivers when compared to caring for a child with tolerized inhibitor or without inhibitor. Caregivers of children with chronic pain reported significantly higher burden in all domains of the HEMOCAB except for “interaction with the father.” Conclusion Caregiver burden can be affected by the child's haemophilia status, particularly if joint health is impacted (eg bleeds, decreased mobility) or if the child suffers from chronic pain which was moderately correlated with joint bleeds.

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“…Overall, the total cost of moderate and severe HA in Australia in 2018 was estimated to be AUD$111.080M. This equates to an average annual cost of AUD$119.956K per person which is very much in keeping with costs quoted overseas 11‐13 …”

Section: Discussionmentioning

confidence: 74%

The societal burden of haemophilia A. III – The potential impact of emicizumab on costs of haemophilia A in Australia

Brown

et al. 2020

Haemophilia

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IntroductionEmicizumab is a humanized monoclonal modified IgG4 antibody with bispecific antibody structure bridging Factor IXa and Factor X. Emicizumab has demonstrated efficacy and safety in adults, adolescents and paediatrics with HA, with or without inhibitors to Factor VIII (FVIII). There is currently no evidence that reports on the potential impact of the introduction of emicizumab on the societal costs of haemophilia A (HA). The purpose of this study was to explore the cost impact associated with the introduction of emicizumab on the current societal costs of people with HA (PwHA) in Australia.MethodsWe conducted an analysis of the impact of emicizumab on societal costs, based on changes in the direct and indirect costs incurred by PwHA. Potential impacts of emicizumab on outcomes in PwHA were modelled based on HAVEN 1, HAVEN 2 and HAVEN 3 studies. We assumed that eligible PwHA commenced use of emicizumab on 1 January 2018. The impact of emicizumab on costs of HA in Australia males was then estimated for the 12‐month period to 31 December 2018.ResultsOverall, uptake of emicizumab in its first year of use reduces annual costs associated with moderate/severe HA by AUD$69.197M (62.3%). This reflects 64.2% reduction in the cost of FVIII blood products and 92% reduction in cost of bypassing agents.ConclusionThe cost of emicizumab is likely to offset some or all of the projected reductions in treatment costs. However, we also found 30.7% reduction in non‐treatment direct costs (AUD$3.771M) and 19.1% reduction in indirect costs (AUD$2.732M).

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The socioeconomic burden of patients affected by hemophilia with inhibitors

Cited by 50 publications

References 95 publications

Prevalence of inhibitors and clinical characteristics in patients with haemophilia in a middle‐income Latin American country

Prevalence of inhibitors and clinical characteristics in patients with haemophilia in a middle‐income Latin American country

The burden of bleeds and other clinical determinants on caregivers of children with haemophilia (the BBC Study)

The societal burden of haemophilia A. III – The potential impact of emicizumab on costs of haemophilia A in Australia

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