Determinants of successful immune tolerance induction in hemophilia A: systematic review and meta-analysis

Oomen, Ilja; Camelo, Ricardo Mesquita; Rezende, Suely Meireles; Voorberg, Jan; Mancuso, Maria Elisa; Oldenburg, J.; Carção, Manuel; Matino, Davide; Lillicrap, David; Fischer, Kathelijn; Fijnvandraat, Karin; Gouw, Samantha C.

doi:10.1016/j.rpth.2022.100020

Cited by 3 publications

(4 citation statements)

References 65 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…31 Another important factor related to QoL in hemophilia patients that was evaluated in this study was annual bleeding rate. The median and IQR 20 (2-55) which was determined for annual bleeding rate in our adult hemophilia population is much higher than that reported by the world federation of hemophilia in 2018 data reports of world bleeding disorders registry 6 (2)(3)(4)(5)(6)(7)(8)(9)(10)(11)(12)(13)(14)(15)(16) hemophilia patients. 38 This issue emphasizes paying more attention to close monitoring of hemophilia patients and the use of primary prophylaxis in patients with higher risk of bleeding.…”

Section: Discussionmentioning

confidence: 57%

“…8 Also, the optimal therapeutic strategy to eradicate inhibitors is immune tolerance induction, defined as repeated administration of FVIII concentrate. 9 However, due to high treatment costs, hemophilia patients with inhibitors are still far from achieving standard care and management, which leads to increased physical and emotional burden and reduced QoL. 10,11 Health-related quality of life (HRQoL), as a social measurement, has found its way into clinical studies since 1958; the World Health Organization (WHO) recognized QoL as an important component of health, in defining health as "not merely the absence of disease or infirmity, but a state of complete physical, mental and social wellbeing."…”

Section: The Impact Of Inhibitors On the Quality Of Life In Patients ...mentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

“…overall and 6(2)(3)(4)(5)(6)(7)(8)(9)(10)(11)(12)(13)(14) among severeIn the multiple linear regression model, disease severity as a categorical variable was converted into dummy variable and the scores of moderate and severe hemophilia were compared to mild hemophilia. Also, the value of scores in the positive inhibitor group was compared with the negative inhibitor group.…”

mentioning

confidence: 99%

See 3 more Smart Citations

The impact of inhibitors on the quality of life in patients with hemophilia

et al. 2023

View full text Add to dashboard Cite

Objective: To investigate the association of health-related quality of life in hemophilia patients with inhibitor and clinical and demographic characteristics. Methods: In this multi-center cross-sectional study, 41 male patients with hemophilia A were investigated from May to October 2021. All patients were registered at the Hemophilia Clinic affiliated with Shiraz and Zahedan Universities of Medical Sciences in Iran. Health-related quality of life of the patients was evaluated by the Short Form-36 questionnaire. Results: The patients’ mean ± SD of age was 36.9 ± 13.2 (range: 18–76) years. Eleven patients (26.8%) were inhibitor positive. In univariate analysis, physical function, mental health dimension, and total Short Form-36 scores were significantly lower in the inhibitor-positive patients ( p < 0.001, p = 0.045, and p = 0.035, respectively). Moreover, patients with severe disease showed significantly lower scores in physical function ( p < 0.001), physical health dimension ( p = 0.018), and total Short Form-36 ( p = 0.031) than those with mild and moderate hemophilia. Also, blood-borne infections showed a significant association with lower score in physical health dimension ( p = 0.038). In addition, annual bleeding rate showed significant negative correlations with physical health dimension ( rs = −0.609, p < 0.001), mental health dimension ( r = −0.317, p = 0.044), and total Short Form-36 ( r = −0.455, p = 0.003) scores. In multiple linear regression analysis, disease severity revealed a significant negative relationship with scores in physical function ( p = 0.001), role physical (RP) ( p = 0.015), general health (GH) ( p = 0.006), physical health dimension ( p = 0.006), and marginally in total Short Form-36 score ( p = 0.054). Also, age of the patients showed a significant negative association with physical function and GH scores ( p < 0.001 and p = 0.015, respectively). Conclusion: Disease severity and age were shown as independent factors affecting health-related quality of life, but inhibitor alone was not an independent influencing factor. Reduced health-related quality of life was also observed in hemophilia patients with higher annual bleeding rate and blood-borne infections. Therefore, it is necessary to pay more attention to these subgroups. Further studies with larger sample size are needed for more accurate results.

show abstract

Section: Discussionmentioning

confidence: 57%

Section: The Impact Of Inhibitors On the Quality Of Life In Patients ...mentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

mentioning

confidence: 99%

See 2 more Smart Citations

The impact of inhibitors on the quality of life in patients with hemophilia

et al. 2023

View full text Add to dashboard Cite

show abstract

Emerging approaches to induce immune tolerance to therapeutic proteins

Noel,

Lagassé,

Golding

et al. 2023

Trends in Pharmacological Sciences

View full text Add to dashboard Cite

Race and ethnicity and the success of immune tolerance induction among people with severe haemophilia A in the United States

Fedewa,

Kempton

2024

Haemophilia

View full text Add to dashboard Cite

IntroductionImmune tolerance induction (ITI) is the only treatment to eradicate inhibitors in people with severe haemophilia A with inhibitors. Since the risk of inhibitor development is greater among Black and Hispanic persons, it has been hypothesized that race and ethnicity may influence ITI success. Limited studies have evaluated this hypothesis.AimTo examine the success of ITI according to race and ethnicity.MethodsParticipants who entered the Community Counts (CC) Registry between 2013 and 2017, were aged ≥3 years at study entry, and received ITI were included (n = 559). The proportion of participants with successful ITI was examined with adjusted prevalence ratios (aPRs) and corresponding 95% confidence intervals (95% CIs).ResultsAmong 559 participants, 56.9%, 19.1%, 18.1% and 4.3% were Non‐Hispanic (NH) White, NH Black, Hispanic and Asian, respectively, and 1.7% were coded as other or missing. Approximately 80% of Hispanic, NH Black and NH White participants had good/very good prognosis, defined as having a pre‐ITI peak inhibitor of < 200 Bethesda Units per millilitre. Nearly 60% of participants (59.7%) achieved successful ITI, 20.7% and 19.5% experienced partially successful or failed ITI, respectively. Successful ITI was non‐significantly lower in NH Black (54.2%; aPR = 0.95, 95% CI 0.62–1.44) and Hispanic (55.4%; aPR = 0.89, 95% CI 0.71–1.13) relative to NH White participants (62.6%).ConclusionIn this study, 60% of participants in the CC Registry had successful ITI, consistent with previous studies. The proportion with successful ITI was generally comparable across racial and ethnic groups with similar prognosis. These findings do not support the hypothesis that ITI response varies according to race or ethnicity.

show abstract

Determinants of successful immune tolerance induction in hemophilia A: systematic review and meta-analysis

Cited by 3 publications

References 65 publications

The impact of inhibitors on the quality of life in patients with hemophilia

The impact of inhibitors on the quality of life in patients with hemophilia

Emerging approaches to induce immune tolerance to therapeutic proteins

Race and ethnicity and the success of immune tolerance induction among people with severe haemophilia A in the United States

Contact Info

Product

Resources

About