Evolution of congenital haemophilia care in Taiwan

Chen, Yeu‐Chin; Chang, Ching-Yu; Cheng, Shin‐Nan; Pan, Ru-Yu; Shih, Yu‐Lueng; Li, Tsung-Ying; Wang, Shenghao

doi:10.1016/j.jfma.2021.07.017

Cited by 6 publications

(3 citation statements)

References 45 publications

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“…We found that the proportion of the so defined severe patients was approximately 55% among all PWHA (data not shown), which was consistent with a previous report from Taiwan. 23 …”

Section: Methodsmentioning

confidence: 99%

Effectiveness of Prophylactic Coagulation Factor Replacement Therapy in Patients with Severe Hemophilia A in Taiwan – A Population-Based Study

Hsieh¹,

Chiou²,

Liao³

et al. 2022

CLEP

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Purpose Taiwan launched reimbursement of prophylactic coagulation factor replacement therapy (CFRT) for patients with severe hemophilia type A (severe PWHA) in 2014. However, since then, the effectiveness of prophylactic CFRT in real-world practice has not been evaluated thoroughly. This study aimed to evaluate the effectiveness of prophylactic CFRT in severe PWHA cases on the outcome of bleeding risks. Patients and Methods We included male, severe PWHA cases from a nationwide, population-based database in Taiwan. Given that the database lacked details of the dosing regimen for prophylactic CFRT, we applied group-based trajectory modeling using the proportion of days covered (PDC) by CFRT from 2014 to 2015 in order to classify patients. A high PDC level corresponded to a greater proportion of time under CFRT, thus implying that the patient was probably receiving prophylactic therapy. We followed up patients from January 01, 2016 until occurrence of any bleeding events, death or December 31st 2017. Results We identified a total of 420 severe PWHA and classified them into high- (n = 88), medium- (n = 181) and low- (n = 151) PDC groups. The mean (±SD) PDC values of the three groups were 0.78 (±0.1), 0.40 (±0.1) and 0.12 (±0.1), respectively. Using Cox regression models with propensity score adjustment, we found patients with medium- (hazard ratio: 0.69; 95% CI: 0.56–0.89) or high-PDC (0.45; 0.36–0.68) under CFRT had reduced risks of any bleeding, compared to the low PDC group. Conclusion The findings demonstrated the effectiveness of prophylactic CFRT in the prevention of bleeding events in real-life severe PWHA.

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“…We found that the proportion of the so defined severe patients was approximately 55% among all PWHA (data not shown), which was consistent with a previous report from Taiwan. 23 …”

Section: Methodsmentioning

confidence: 99%

Effectiveness of Prophylactic Coagulation Factor Replacement Therapy in Patients with Severe Hemophilia A in Taiwan – A Population-Based Study

Hsieh¹,

Chiou²,

Liao³

et al. 2022

CLEP

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“…15 A registry of PWH by several centres in Taiwan revealed incidence of inhibitors to be 22.1% in haemophilia A patients and 10.2% in haemophilia B patients. 16 The prevalence of children who developed FVIII inhibitors was 9.6% in Indonesia. 17 Emicizumab is approved in Thailand, Vietnam, Indonesia, Malaysia, and India to prevent bleeding or reduce the frequency of bleeding episodes in adults and children with haemophilia A with or without factor VIII inhibitors.…”

Section: Neutralizing Antibodiesmentioning

confidence: 99%

“…In Hong Kong, the cumulative proportion of patients with severe‐to‐moderate deficiency with inhibitors was 8.5% 15 . A registry of PWH by several centres in Taiwan revealed incidence of inhibitors to be 22.1% in haemophilia A patients and 10.2% in haemophilia B patients 16 . The prevalence of children who developed FVIII inhibitors was 9.6% in Indonesia 17 .…”

Section: Management Of People With Haemophiliamentioning

confidence: 99%

Haemophilia care in Asia: Learning from clinical practice in some Asian countries

Angchaisuksiri,

Amurao‐Abiera,

Chou

et al. 2024

Haemophilia

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BackgroundThe healthcare systems in Asia vary greatly due to the socio‐economic and cultural diversities which impact haemophilia management.MethodsAn advisory board meeting was conducted with experts in haemophilia care from Asia to understand the heterogeneity in clinical practices and care provision in the region.FindingsThe overall prevalence of haemophilia in Asia ranges between 3 and 8.58/100,000 patients. Haemophilia A was more prevalent as compared to haemophilia B with a ratio of around 5:1. There is under‐diagnosis in the region due to lack of diagnosis, registries and/or lack of appropriate facilities in suburban areas. Most patients are referred to the haematologists by their families or primary care physicians, while some are identified during bleeding episodes. Genetic testing faces obstacles like resource constraints, services available at limited centres and unwillingness of patients to participate. Prophylaxis is offered for people with haemophilia (PWH) with a severe bleeding phenotype. Recombinant factors are approved in most countries across the region and are the preferred therapy. The challenges highlighted for not receiving a high standard of care include patients’ reluctance to use an intravenous treatment, poor patient compliance due to frequency of infusions, budget constraints and lack of funding, insurance, availability and accessibility of factor concentrates. Prevalence of neutralizing antibodies ranged from 5% to 20% in the region. Use of immune tolerance induction and bypassing agents to treat inhibitors depends on their cost and availability.ConclusionHaemophilia care in Asia has evolved to a great extent. However, some challenges remain for which a strategic approach along with multi‐stakeholder involvement are needed.

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Real-world bleeding outcomes and product utilization in people with severe-type hemophilia A before and after switching to extended half-life rFVIIIFc prophylaxis therapy

et al. 2022

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Evolution of congenital haemophilia care in Taiwan

Cited by 6 publications

References 45 publications

Effectiveness of Prophylactic Coagulation Factor Replacement Therapy in Patients with Severe Hemophilia A in Taiwan – A Population-Based Study

Effectiveness of Prophylactic Coagulation Factor Replacement Therapy in Patients with Severe Hemophilia A in Taiwan – A Population-Based Study

Haemophilia care in Asia: Learning from clinical practice in some Asian countries

Real-world bleeding outcomes and product utilization in people with severe-type hemophilia A before and after switching to extended half-life rFVIIIFc prophylaxis therapy

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