MRI predicts 5-year joint bleeding and development of arthropathy on radiographs in hemophilia

Foppen, Wouter; Schaaf, Irene C. van der; Beek, F. J. A.; Mali, W. P. T. M.; Fischer, Kathelijn

doi:10.1182/bloodadvances.2019001238

Cited by 35 publications

(54 citation statements)

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“…Despite receiving FIX prophylaxis, patients with severe hemophilia B continue to experience breakthrough bleeding and may eventually develop hemophilic arthropathy [ 31 ]. We observed target joints in approximately 20% of patients, and a mean ABR of 1.73 that was consistent with other recent real-world studies [ 27 , 32 ].…”

Section: Discussionmentioning

confidence: 99%

Clinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US+ population surveys

Burke

Asghar

O’Hara

et al. 2021

Orphanet J Rare Dis

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Background Hemophilia B is a rare congenital bleeding disorder that has a significant negative impact on patients’ functionality and health-related quality of life. The standard of care for severe hemophilia B in the United States is prophylactic factor IX replacement therapy, which incurs substantial costs for this lifelong condition. Accurate estimates of the burden of hemophilia B are important for population health management and policy decisions, but have only recently accounted for current management strategies. The ‘Cost of Severe Hemophilia across the US: a Socioeconomic Survey’ (CHESS US) is a cross-sectional database of medical record abstractions and physician-reported information, completed by hematologists and care providers. CHESS US+ is a complementary database of completed questionnaires from patients with hemophilia. Together, CHESS US and CHESS US+ provide contemporary, comprehensive information on the burden of severe hemophilia from the provider and patient perspectives. We used the CHESS US and CHESS US+ data to analyze the clinical, humanistic, and economic burden of hemophilia B for patients treated with factor IX prophylaxis between 2017 and 2019 in the US. Results We conducted analysis to assess clinical burden and direct medical costs from 44 patient records in CHESS US, and of direct non-medical costs, indirect costs, and humanistic burden (using the EQ-5D-5L) from 57 patients in CHESS US+. The mean annual bleed rate was 1.73 (standard deviation, 1.39); approximately 9% of patients experienced a bleed-related hospitalization during the 12-month study period. Nearly all patients (85%) reported chronic pain, and the mean EQ-5D-5L utility value was 0.76 (0.24). The mean annual direct medical cost was $614,886, driven by factor IX treatment (mean annual cost, $611,971). Subgroup analyses showed mean annual costs of $397,491 and $788,491 for standard and extended half-life factor IX treatment, respectively. The mean annual non-medical direct costs and indirect costs of hemophilia B were $2,371 and $6,931. Conclusions This analysis of patient records and patient-reported outcomes from CHESS US and CHESS US+ provides updated information on the considerable clinical, humanistic, and economic burden of hemophilia B in the US. Substantial unmet needs remain to improve patient care with sustainable population health strategies.

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Section: Discussionmentioning

confidence: 99%

Clinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US+ population surveys

Burke

Asghar

O’Hara

et al. 2021

Orphanet J Rare Dis

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“…Subclinical synovitis occurs long before the appearance of clinical HA. 4 Magnetic resonance imaging (MRI) is the most sensitive radiological modality for detection of HA. It can detect early synovial and cartilage changes, unlike plain radiography, which is less sensitive in detecting subclinical joint changes.…”

Section: Backg Rou N Dmentioning

confidence: 99%

“…Subclinical synovitis occurs long before the appearance of clinical HA 4 . Magnetic resonance imaging (MRI) is the most sensitive radiological modality for detection of HA.…”

Section: Introductionmentioning

confidence: 99%

Tissue‐inhibitors of metalloproteinase‐1 and vascular‐endothelial growth‐factor in severe haemophilia A children on low dose prophylactic recombinant factor VIII: Relation to subclinical arthropathy

Andrawes

Saker²,

Salah

et al. 2020

Haemophilia

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Background: Subclinical synovitis occur long before clinical haemophilic arthropathy (HA). New biomarkers are needed for early detection of HA. Aim: To compare the levels of tissue inhibitors of metalloproteinase-1 (TIMP-1) and vascular endothelial growth factor (VEGF)in severe haemophilia A boys on prophylaxis and on-demand therapy to healthy boys and correlate them with the haemophilia joint health score (HJHS) & the Denver magnetic resonance imaging (MRI) scale; hence, determine their values in early detection of HA. Methods: Haemophilia joint health score, serum TIMP-1, VEGF and Denver MRI score were assessed in 50 boys with severe haemophilia A (31 on prophylactic factor VIII therapy (62%) with a dose of 15 IU/kg/twice weekly) and 50 age-matched healthy boys. Results: Boys with severe haemophilia A had significantly higher TIMP-1 240 ng/mL, SD200-350 (P < .001) and VEGF 600 pg/mL, SD400-1100 (P < .001). Their mean HJHS was 4.5 ± 3.0 (0-11) and their mean Denver MRI score was 5.55 ± 1.6 (2.00-8.00). A significant positive correlation was found between TIMP-1 and VEGF (P < .001), BMI Z-score (P = .029), HJHS (P = .041)and total MRI score (<.001). Significant correlations were found between VEGF and age (P < .001), HJHS (P = .003) and total MRI score (P < .001). Boys with severe haemophilia A on prophylaxis therapy had significantly lower HJHS (P = .021), VEGF (P < .001), TIMP-1 (P = .002) and total MRI score (P = .021) than those on on-demand therapy. Receiver operating characteristic curve, defined a cutoff value of 160 ng/mL for TIMP-1 with a sensitivity of 90% and specificity of 60% and that of 350 pg/mL for VEGF with a sensitivity of 78% and specificity of 88% for discrimination between severe haemophilia A and healthy boys. Conclusion: Vascular endothelial growth factor and TIMP-1 can be used for early detection of HA. Further prospective studies should include larger study populations. In addition, studies should address the role of various anti-VEGFs as potential therapy for HA and their impact on prevention and treatment of HA.

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“…Eventually, joint degeneration can cause clinical symptoms and affect the quality of life. [ 20 ] After 5-year follow-up of patients with hemophilia, Foppen et al [ 21 ] found that all MRI changes except effusion were strong predictors for development of arthropathy on radiographs. MRI is inferior to CT in the display of osteoporosis.…”

Section: Discussionmentioning

confidence: 99%

Multislice spiral computed tomography imaging in evaluating hemophilic arthropathy

Zhang

Wei

et al. 2020

Chinese Medical Journal

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Background Certain hemophilia patients are unable to cooperate with or afford magnetic resonance imaging (MRI) examinations. The purpose of our study was to explore the value of multislice spiral computed tomography (MSCT) in evaluating hemophilic arthropathy (HA). Methods Thirty-eight patients with 73 joints of HA were consecutively selected from January 2016 to May 2018 for this prospective study. All 73 joints were examined by X-ray, CT, and MRI within 2 days. The MRI scores of the joints were determined by the International Prophylaxis Study Group (IPSG) standard. The CT findings were quantified according to the IPSG standard, except for cartilage injury, which was quantified by joint space narrowing using the X-ray Pettersson score. The CT and MRI scores were compared by the paired Wilcoxon signed-rank test. The correlations between the CT score of joint space narrowing and MRI score of cartilage injury and the total CT and MRI scores were analyzed by Spearman rank correlation. The kappa test was used to compare the consistency of CT and MRI scores. Results MRI was superior to CT based on the scores for small amount of effusion ( P < 0.05), synovial hypertrophy and hemosiderin deposition in the mild groups ( P < 0.05). The CT and MRI scores were not significantly different for moderate and massive effusion, synovial hypertrophy, and hemosiderin deposition in the moderate and severe groups, bone erosion or cystic changes ( P > 0.05), and there was a high degree of consistency between the two scores (kappa > 0.81). The consistency between the Pettersson scores of joint space narrowing on CT and the IPSG scores of cartilage injury on MRI was high (kappa = 0. 774, P < 0.05). Conclusion The image scores of MSCT are generally consistent with MRI except for mild synovitis, which can be used as an alternative for the evaluation of HA.

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MRI predicts 5-year joint bleeding and development of arthropathy on radiographs in hemophilia

Cited by 35 publications

References 36 publications

Clinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US+ population surveys

Clinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US+ population surveys

Tissue‐inhibitors of metalloproteinase‐1 and vascular‐endothelial growth‐factor in severe haemophilia A children on low dose prophylactic recombinant factor VIII: Relation to subclinical arthropathy

Multislice spiral computed tomography imaging in evaluating hemophilic arthropathy

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