The prevalence of factor VIII inhibitor in patients with severe hemophilia-A and its clinical characteristics

Harijadi, Harijadi; Gatot, Djajadiman; Akib, Arwin Ap

doi:10.14238/pi45.4.2005.177-81

“…Haemophilia is an impairment of the blood-clotting process that results in repeated spontaneous bleeding in joints and muscles [ 22 ]. In Indonesia, haemophiliacs are treated only if bleeding occurs [ 23 ]. We included patients with severe/moderate levels who usually had 1 bleeding/month [ 24 ].…”

Section: Methodsmentioning

confidence: 99%

Comparing measurement properties of EQ-5D-Y-3L and EQ-5D-Y-5L in paediatric patients

Fitriana

¹

,

Purba

²

,

Rahmatika

³

et al. 2021

Health Qual Life Outcomes

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Background The adult versions EQ-5D-3L and EQ-5D-5L have been extensive compared. This is not the case for the EQ-5D youth versions. The study aim was to compare the measurement properties and responsiveness of EQ-5D-Y-3L and EQ-5D-Y-5L in paediatric patients. Methods A sample of patients 8–16 years old with different diseases and a wide range of disease severity was asked to complete EQ-5D-Y-3L, EQ-5D-Y-5L, PedsQL Generic Core Scale, and selected, appropriate disease-specific instruments, three times. EQ-5D-Y-3L and EQ-5D-Y-5L were compared in terms of: feasibility, (re-)distribution properties, discriminatory power, convergent validity, test–retest reliability, and responsiveness. Results 286 participating patients suffered from one of the following diseases: major beta-thalassemia, haemophilia, acute lymphoblastic leukaemia, acute illness. Missing responses were comparable between versions of the EQ-5D-Y, suggesting comparable feasibility. The number of patients in the best health state (level profile 11111) was equal in both EQ-5D-Y versions. The projection of EQ-5D-Y-3L scores onto EQ-5D-Y-5L for all dimensions showed that the two additional levels in EQ-5D-Y-5L slightly improved the accuracy of patients in reporting their problems, especially if severe. Convergent validity with PedsQL and disease-specific measures showed that the two EQ-5D-Y versions performed about equally. Test–retest reliability (EQ-5D-Y-3L 0.78 vs EQ-5D-Y-5L 0.84), and sensitivity for detecting health changes, were both better in EQ-5D-Y-5L. Conclusions Extending the number of levels did not give clear superiority to EQ-5D-Y-5L over EQ-5D-Y-3L based on the criteria assessed in this study. However, increasing the number of levels benefitted EQ-5D-Y performance in the measurement of moderate to severe problems and especially in longitudinal study designs.

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“…In this study, another frequent bleeding location that was a combination of hemarthrosis-and hematomas-developed inhibitor. A previous study stated that hemarthrosis is a typical bleeding manifestation found in severe hemophilia, especially in patients receiving no maintenance treatment or prophylactic FVIII treatment [25]. Other previous studies also demonstrated that there was an insignificant correlation between bleeding type or location and inhibitor development [5,25].…”

Section: Discussionmentioning

confidence: 96%

“…In severe hemophilia A, recurrent clinical and subclinical joint bleeding episodes might occur all the time, gradually leading to irreversible changes such as hemophilic arthropathy (HA) [1,25]. In spite of numerous studies, the pathophysiology of HA has not been fully elucidated, especially as regards immunopathological mechanisms which are associated with the subclinical and early stage of the disease and to be more precise, with chronic joint inflammation.…”

Section: Discussionmentioning

confidence: 99%

Serum TNF-α Level as a Possible Predictor of Inhibitor Levels in Severe Hemophilia A

Susanah

¹

,

Raspati

²

,

Sari

³

et al. 2021

BioMed Research International

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Background. The development of factor VIII (FVIII) inhibitor in patients with hemophilia A (PWHA) is a great challenge for hemophilia care. Both genetic and environmental factors led to complications in PWHA. The development of inhibitory antibodies is usually induced by the immune response. Tumor necrosis factor α (TNF-α), one of the cytokines, might contribute to its polymorphism. In this study, we investigated the clinical factors, level of serum TNF-α, and polymorphism of c . − 308 G > A TNF − α gene in inhibitor development in severe PWHA. Methods. A cross-sectional study was conducted among all PWHA in West Java province. The clinical parameters, FVIII, FVIII inhibitor, and serum TNF-α level were assessed. The genotyping of − 380 G > A TNF-α gene polymorphism was performed using polymerase chain reaction and Sanger sequencing. Results. Among the 258 PWHA, 216 (83.7%) were identified as severe PWHA. The FVIII inhibitor was identified in 90/216 (41.6%) of severe PWHA, consisting of 45 high-titer inhibitors (HTI) and 45 low-titer inhibitors (LTI). There was a significant correlation between serum TNF-α level and the development of HTI ( p = 0.043 ). The cutoff point of serum TNF-α level, which can be used to differentiate between HTI and LTI, was 11.45 pg/mL. The frequency of FVIII replacement therapy was significant only in HTI of severe PWHA regarding serum TNF-α level ( p = 0.028 ). There is no correlation between polymorphisms of − 380 G > A TNF-α gene and inhibitor development ( p = 0.645 ). Conclusions. The prevalence of FVIII inhibitor in severe PWHA in West Java, Indonesia, was 41.6%. The frequency of replacement therapy is a risk factor for inhibitor development. Serum TNF-α level might be used to differentiate between high and low inhibitor levels in severe hemophilia A, and this might support decision making regarding treatment options for inhibitor in severe hemophilia A.

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“…Prevalence of factor VIII inhibitors in hemophilia patients varies greatly, but overall ranges are from 3.6% to 52%. The prevalences of inhibitors in Taiwan, Jordan, and Saudi Arabia are 10.4%, 9%, 9.7%, respectively, whereas the prevalences in 1,[6][7][8] Indonesia vary from 35% to 37.5%. Inhibitors are problem in management of hemophilia because 2 treatment becomes difficult and expensive.…”

Section: Introductionmentioning

confidence: 99%

“…Hemophilia is a bleeding disorder due to hereditary coagulation abnormalities, inherited by [1][2][3] X-linked recessive.…”

Section: Introductionmentioning

confidence: 99%

Non-Genetic Risk Factors for The Formation of Factor VIII Inhibitors in Hemophilia A Patients in RSUD Dr. Soetomo

Fauzi¹,

Larasati²,

Ugrasena³

2019

Indonesian J. Clin. Pathol. Med. Lab.

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Background: Neutralizing alloantibodies (inhibitors) is one of the complications that affect the morbidity and quality of life of hemophilia patient and can be caused by multifactorials. Genetic and inhibitors examination are not routine and expensive. Therefore, identification of non-genetic factors is important to predict the formation of inhibitors. Objective: To analyze non-genetic risk factors for the formation of factor VIII inhibitors in children with Hemophilia A. Methods: A cross-sectional study of hemophilia children aged 1-18 years at the pediatric hematology oncology outpatient clinic of Dr. Soetomo hospital in March-April 2018. Factors analyzed included the severity of hemophilia, early age of diagnosis, initial age of therapy, type of replacement therapy, frequency of factor VIII administration, and severity of bleeding by bleeding score. Statistical analysis using Chi square, Fisher, ANOVA and logistic regression analysis. Results: A total of 29 children were evaluated, 7/29 mild, 15/29 moderate, and 7/29 severe hemophilia. Inhibitors were found in 11/29 subjects, 7/11 low (1-5 BU) and 4/11 high titer (> 5 BU). The initial age of diagnosis ≤ 1 year is associated with the formation of factor VIII inhibitors (OR 8.75; 95% CI = 1.5-50.2; p = 0.015). Severity of hemophilia, early age of therapy, type of replacement therapy, frequency of therapy, and bleeding score was not significantly associated with the formation of factor VIII inhibitors. Conclusion: The initial age of diagnosis less than 1 year is a significant risk factor for the formation of factor VIII inhibitors.

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The prevalence of factor VIII inhibitor in patients with severe hemophilia-A and its clinical characteristics

Cited by 6 publications

References 11 publications

Comparing measurement properties of EQ-5D-Y-3L and EQ-5D-Y-5L in paediatric patients

Comparing measurement properties of EQ-5D-Y-3L and EQ-5D-Y-5L in paediatric patients

Serum TNF-α Level as a Possible Predictor of Inhibitor Levels in Severe Hemophilia A

Non-Genetic Risk Factors for The Formation of Factor VIII Inhibitors in Hemophilia A Patients in RSUD Dr. Soetomo

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