Early prophylaxis in children with severe haemophilia A: clinical and ultrasound imaging outcomes

Altisent, Carmen; Martorell, Marta; Crespo, Almudena; Casas, Lourdes; Torrents, C; Parra, Rafael

doi:10.1111/hae.12792

Cited by 44 publications

(85 citation statements)

References 28 publications

(32 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“…In the study, total HJHS scores correlated with age (r=0.56, p=0.006), weight (r=0.61, p=0.002), and age at the start of prophylaxis (r=0.63; p=0.001). 22 In our study, the HJHS and HEAD-US scores were higher compared to Altisent's study 22 due to different treatment regimen, showing that prophylaxis can preserve joint function and prevent joint damage in children with severe hemophilia A.…”

Section: Discussioncontrasting

confidence: 52%

See 1 more Smart Citation

Clinical and ultrasound joint outcomes in severe hemophilia A children receiving episodic treatment in Indonesian National Hemophilia Treatment Center

et al. 2017

View full text Add to dashboard Cite

Background: Recurrent joint bleeds leading to arthropathy is the main problem in severe hemophilia children. This study aimed to investigate joint status in severe hemophilia A children receiving episodic treatment in Cipto Mangunkusumo Hospital, Jakarta.Methods: A cross-sectional study was conducted in Cipto Mangunkusumo Hospital as Indonesian National Hemophilia Treatment Center on children (4–18 years) with severe hemophilia A, who previously received episodic treatment, with no history of inhibitor factor VIII. Hemophilia Joint Health Score was evaluated according to HJHS version 2.1 2011. Joint ultrasonography was done for six index joints (bilateral elbows, knees and ankles) using Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) methods. Data of age of first joint bleed, number of target joints and inhibitor factor VIII were obtained from the Pediatric Hemophilia Registry and medical records.Results: There were 59 subjects aged 4 to 18 years. Twenty-nine out of 59 (49.2%) subjects experienced first joint bleed before of 2 years of age. The most common of joint bleeds was a right ankle. Mean total HJHS was 8.71±8.73. Subjects aged 4–10 years showed lower HJHS (4.6±3.7) as compared to subjects aged >10–18 years (12.3±10.3), p<0.001; 95% CI=4.9–13. Mean HEAD-US scores in subjects aged 4–10 years (18.7±5.6) was lower than in subjects aged >10–18 years (28±7.9), p<0.001, 95% CI= -12.9–-5.6.Conclusion: HJHS and HEAD-US scores of severe hemophilia A children receiving episodic treatment aged 4–10 years are lower compared to subjects aged >10–18 years, indicating more severe joint destruction in older children and progressivity of joint damage over time. It is important to start prophylactic treatment to prevent progressivity of joint damage.

show abstract

Section: Discussioncontrasting

confidence: 52%

“…Recent observational study reported by Altisent et al 22 showed the utilization of HJHS and HEAD-US scores in severe hemophilia children aged 4-19 years (n=25) treated with prophylaxis treatment and no history of inhibitor. The median of prophylaxis dose was 65.4 IU/kg/week.…”

Section: Discussionmentioning

confidence: 99%

Clinical and ultrasound joint outcomes in severe hemophilia A children receiving episodic treatment in Indonesian National Hemophilia Treatment Center

et al. 2017

View full text Add to dashboard Cite

show abstract

“…However, ultrasound can be observer-dependent and cannot visualize deep structures, so is not ideal for detecting subchondral cysts, loss of cartilage or haemosiderin deposits [38]. A new, standardized procedure, HEAD-US [39], aims to increase inter-rater reliability of ultrasonic evaluation of joints, although this score still requires validation. As with other assessment tools, the age of the patient and severity of MSK dysfunction should be considered when selecting an appropriate imaging technique.…”

Section: Discussionmentioning

confidence: 99%

Evaluation of early musculoskeletal disease in patients with haemophilia

Seuser¹,

Khayat

Négrier³

et al. 2018

Blood Coagulation &Amp; Fibrinolysis

View full text Add to dashboard Cite

Early joint damage in patients with haemarthrosis often escapes diagnosis because of insufficient investigation of biomechanical changes. Arthropathy in haemophilia requires complex assessment with several tools. Considering the increased emphasis on an integrated approach to musculoskeletal (MSK) outcomes, re-evaluation of MSK assessment to address individual patient needs is warranted. To advise on the optimal use of current assessment tools and strategies for tailored MSK evaluation in patients with haemophilia. A panel of experts in haemophilic arthropathy evaluated internationally recognized assessment tools through published literature and personal expertise. Each tool was considered, scored and ranked for their utility in the clinical assessment of MSK damage. Subsequently, a patient evaluation table detailing advice on type and frequency of assessments for different patient populations was constructed. To obtain a complete MSK assessment, multiple tools must be used to ensure each criterion is evaluated. For patients with haemophilia, clinical examination of the joint, disease-specific structure/function scores, and activity/participation scores including quality of life are important, and should be performed on a regular basis according to age and clinical condition. Joint imaging is recommended in the prevention, diagnosis and follow-up of haemophilic arthropathy and should be used in conjunction with joint structure and function scores. An integrated approach to MSK assessment using combinations of tools will allow earlier management of dysfunction and may improve long-term outcomes. This approach could be used in long-term follow-up of all patients independent of age and disease stage, especially in children to prevent arthropathy.

show abstract

“…In the recently reported Haemophilia Early Arthropathy Detection with UltraSound (HEAD-US) study, high-resolution ultrasonography detected a higher percentage of abnormalities than the physical evaluation and was concordant with the HJHS score in 73% of joints [26]. Differences among operators and instrument resolutions, however, can hinder comparability across treatment centres and/or across studies.…”

Section: Joint Statusmentioning

confidence: 88%

Recognizing the need for personalization of haemophilia patient‐reported outcomes in the prophylaxis era

et al. 2016

View full text Add to dashboard Cite

The safety and efficacy of treatment options for patients with haemophilia have significantly improved over the last two decades, particularly with greater utilization of prophylactic approaches. Consequently, it is becoming increasingly difficult to differentiate the treatment benefits of available choices based on standard endpoints such as annualized bleeding rates and joint health scores. Patient-reported outcomes (PROs) have shown limited ability to discriminate between treatment outcomes, in part because of their comprehensive nature; i.e. differences in specific outcomes meaningful to individual patients are masked by a global scoring system based on a fixed set of items, many of which may be unimportant for any given patient. There is a clear need for new outcome measures. Initiatives to develop patient-centric outcomes that capture clinically meaningful change are ongoing. One such approach, goal attainment scaling (GAS), allows patients, in collaboration with a trained clinician, to select goals from a medical condition-specific menu of options and subsequently facilitates quantitative assessment of goal realization. Thus, it is fully personalized and sensitive to small, often idiosyncratic, treatment benefits, such as improvements in functional capacity. In this paper, we present the underlying rationale for GAS and one other novel approach to PRO personalization, and discuss their potential to augment current outcome measures by reliably detecting and quantifying treatment effects in individuals with haemophilia on prophylaxis.

show abstract

Early prophylaxis in children with severe haemophilia A: clinical and ultrasound imaging outcomes

Abstract: A well-preserved joint status was found in our cohort. High-resolution US detected a higher percentage of abnormalities than the physical evaluation, but the clinical implications of these findings still need to be ascertained.

Cited by 44 publications

References 28 publications

Clinical and ultrasound joint outcomes in severe hemophilia A children receiving episodic treatment in Indonesian National Hemophilia Treatment Center

Clinical and ultrasound joint outcomes in severe hemophilia A children receiving episodic treatment in Indonesian National Hemophilia Treatment Center

Evaluation of early musculoskeletal disease in patients with haemophilia

Recognizing the need for personalization of haemophilia patient‐reported outcomes in the prophylaxis era

Contact Info

Product

Resources

About