Comparison of Total Joint Replacement Rate Between Patients With Hemophilia A and Patients With Hemophilia B: A Population-Based and Retrospective Cohort Study

Lin, Wen-Ya; Wang, Jiaan-Der; Tsan, Yu‐Tse; Chan, Wei-Cheng; Tong, Kwok-Man; Chang, Shin-Tsu; Cheng, Yuan-Yang

doi:10.1177/1076029618794294

Cited by 4 publications

(2 citation statements)

References 34 publications

(44 reference statements)

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“…7 In contrast, our previous study showed no increased risk of total joint replacement in 782 patients with HA versus 153 patients with HB (HR: 0.92, 95% CI: 0.54-1.58) after adjusting for various confounders including age, pyogenic arthritis, and HIV infection. 27 While this national population-based study had a number of strengths, there were also some limitations. The NHI research database lacks records of laboratory data such as baseline FVIII/FIX and inhibitor level.…”

Section: Discussionmentioning

confidence: 99%

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Differences in Major Bleeding Events Between Patients With Severe Hemophilia A and Hemophilia B: A Nationwide, Population-Based Cohort Study

Shih

Wang

Yin

et al. 2019

Clin Appl Thromb Hemost

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There has been an ongoing debate as to whether hemophilia A (HA) is more severe than hemophilia B (HB), and there are studies supporting each side of the argument. The study aimed to investigate whether any differences in major bleeding events exist between patients with severe HA and HB. A nationwide, population-based retrospective cohort study using the National Health Insurance Research Database was conducted. We compared 658 patients with severe HA and 137 patients with severe HB without inhibitors from 1997 to 2013, during the period when adult patients older than 18 years old were treated with the on-demand therapy since birth. There was no significant difference between patients with severe HA and HB in the rate of major bleeding events, with an adjusted relative ratio of 0.79 (95% confidence interval [CI]: 0.36-1.71, P = .548). There was also no significant difference in the incidence rate of major bleeding events between adult patients with HA and HB with the on-demand therapy, and an adjusted hazard ratio (HR) of 0.82 (95% CI: 0.65-1.02). However, patients with HA had a lower incidence rate of intracranial hemorrhage, with an adjusted HR of 0.44 (95% CI: 0.25-0.79). In addition, no significant difference in the frequency of major bleeding events requiring hospitalization between patients with HA and HB was found, P > .05. In conclusion, the study demonstrated that patients with severe HB encountered a similar rate of major bleeding events to those with severe HA.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Differences in Major Bleeding Events Between Patients With Severe Hemophilia A and Hemophilia B: A Nationwide, Population-Based Cohort Study

Shih

Wang

Yin

et al. 2019

Clin Appl Thromb Hemost

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Evolution of congenital haemophilia care in Taiwan

Chen

Chang

Cheng

et al. 2022

Journal of the Formosan Medical Association

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The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective

Bolous¹,

Chen²,

Wang³

et al. 2021

Blood

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Adeno-associated virus (AAV)-mediated gene therapy is a novel treatment promising to reduce morbidity associated with hemophilia. While multiple clinical trials continue to evaluate efficacy and safety, limited cost-effectiveness data have been published. This study compared the potential cost-effectiveness of AAV-mediated factor IX(FIX)-Padua gene therapy for severe hemophilia B patients in the United States (US) to on-demand FIX replacement and primary FIX prophylaxis, using either standard or extended half-life FIX products. A microsimulation Markov model was constructed and transition probabilities between health states and utilities were informed by published data. Costs were aggregated using a micro-costing approach. An 18-years-old till death time-horizon from the perspective of a third-party payer in the US was conducted. Gene therapy was more cost-effective than both alternatives considering a $150,000/QALY threshold. The price for gene therapy was assumed $2,000,000 in the base-case scenario, yet one of the one-way sensitivity analyses was conducted using observed manufacturing, administration and five-year follow-up cost of $87,198 for AAV-mediated gene therapy vector as derived from the manufacturing facility and clinical practice at St. Jude Children's Research Hospital. One-way sensitivity analyses showed 10/102 scenarios in which gene therapy was not cost-effective compared to alternative treatments. Notably, gene therapy remained cost-effective in a hypothetical scenario in which we estimated that the discounted factor concentrate price was 20% of the wholesale acquisition cost in the US. Probabilistic sensitivity analysis estimated gene therapy cost-effective at 92% of simulations considering $150,000/QALY threshold. In conclusion, based on detailed simulation inputs and assumptions, gene therapy was more cost-effective than on-demand treatment and prophylaxis for patients with severe hemophilia B.

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Comparison of Total Joint Replacement Rate Between Patients With Hemophilia A and Patients With Hemophilia B: A Population-Based and Retrospective Cohort Study

Cited by 4 publications

References 34 publications

Differences in Major Bleeding Events Between Patients With Severe Hemophilia A and Hemophilia B: A Nationwide, Population-Based Cohort Study

Differences in Major Bleeding Events Between Patients With Severe Hemophilia A and Hemophilia B: A Nationwide, Population-Based Cohort Study

Evolution of congenital haemophilia care in Taiwan

The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective

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