Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort

Feldman, Brian M.; Rivard, Georges E.; Babyn, Paul; Wu, John; Steele, MacGregor; Poon, Man‐Chiu; Card, Robert T.; Israels, Sara J.; Laferriere, Nicole; Gill, Kulwant; Chan, Anthony; Carção, Manuel; Klaassen, Robert J.; Cloutier, Stéphanie; Price, Victoria; Dover, Saunya; Blanchette, Victor S.

doi:10.1016/s2352-3026(18)30048-6

Cited by 33 publications

(60 citation statements)

References 37 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…This could explain to some extent why, at the individual level, some boys over time manifested clinically significant joint disease as measured by MRI. We previously reported that end‐of‐study imaging outcomes were good overall but that some individuals had evidence of significant joint damage with individual joint scores on the 17‐point IPSG MRI scale as high as 14 (on this scale, a score of 17 indicates maximal damage per index joint) …”

Section: Discussionmentioning

confidence: 84%

“…The CHPS was an inception cohort of 56 young boys with severe hemophilia A, defined by a circulating FVIII level of <2%. Detailed descriptions of the study design have been previously reported . For the current analysis, we assessed the factor infusion and bleeding logs of all 56 boys on study from 1997 to 2013.…”

Section: Methodsmentioning

confidence: 99%

“…Detailed descriptions of the study design have been previously reported. 13,14 For the current analysis, we assessed the factor infusion and bleeding logs of all 56 boys on study from 1997 to 2013.…”

Section: Methodsmentioning

confidence: 99%

“…11,12 The CHPS was a single-arm, multicenter prospective study designed to investigate the efficacy of a tailored, frequency-escalated, primary prophylaxis regimen. 13 This current substudy had 2 objectives: first, to describe the treatment adherence rates for CHPS participants and determine if the frequency-escalated approach resulted in better adherence than is reported in the literature and second, to examine the relationship between treatment adherence and bleeding episodes. Specifically, we sought to confirm the theorized strong relationship between adherence and bleeding.…”

Section: Introductionmentioning

confidence: 99%

See 3 more Smart Citations

Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis Study

Dover

Blanchette

Wrathall

et al. 2020

Research and Practice in Thrombosis and Haemostasis

Self Cite

View full text Add to dashboard Cite

Background Standard of care for persons with severe hemophilia A includes regular replacement of factor VIII (FVIII). Prophylaxis regimens using standard half‐life (SHL) FVIII concentrates, while effective, are costly and require frequent intravenous infusions. Aim This study evaluated the adherence of 56 boys with severe hemophilia A to tailored, frequency‐escalated prophylaxis with an SHL recombinant FVIII concentrate. Methods We reviewed the factor infusion and bleeding logs of study subjects. Adherence to the prescribed regimen was calculated on a weekly basis, and bleeding rates were determined from self/proxy‐reported bleeding logs. The primary outcome was adherence to the prescribed prophylaxis regimen. Results The median (range of values [ROV]) weekly adherence to prophylaxis was 85.7% (37.4%‐99.8%). The median (ROV) adherent weeks on steps 1 (weekly), 2 (twice weekly), and 3 (alternate‐day) were 92.9% (50%‐100%), 80.3 (32%‐96%), and 72.6% (14%‐98%); relative to step 1, subjects were less likely to be adherent on steps 2 and 3 (P < 0.00). On step 1, our cohort had higher adherence than previously reported rates. The median (ROV) adherence to the breakthrough bleeding protocol was 47.1% (0%‐100%). At any given time, bleeding risk was reduced by 15% for each 10% increase in adherence during the preceding 12 weeks (hazard ratio, 0.85; 95% confidence interval, 0.81‐0.90). Conclusion This cohort had high rates of adherence to the prescribed prophylaxis regimen. Initiating prophylaxis with once‐weekly infusions facilitated adherence to the prophylaxis regimen in this cohort of boys with severe hemophilia A started on primary prophylaxis at a very young age.

show abstract

Section: Discussionmentioning

confidence: 84%

Section: Methodsmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis Study

Dover

Blanchette

Wrathall

et al. 2020

Research and Practice in Thrombosis and Haemostasis

Self Cite

View full text Add to dashboard Cite

show abstract

“…I typically initiate the discussion soon after diagnosis and recommend initiation prior to the first or second joint bleed. I discuss options of frequency‐escalated prophylaxis, starting once weekly via peripheral vein infusion, versus prophylaxis starting at 2–4 times week after central venous catheter placement. Once prophylaxis is initiated, the choice of factor product may be driven by individual pharmacokinetics, target trough level, and infusion frequency needed to achieve the target.…”

Section: Shared Decision‐making For Treatmentmentioning

confidence: 99%

How I approach: Previously untreated patients with severe congenital hemophilia A

Thornburg

2018

Pediatric Blood & Cancer

View full text Add to dashboard Cite

Previously untreated patients with severe hemophilia A are a vulnerable population at risk for severe bleeding which is currently managed with exogenous clotting factor replacement. The primary burden of current treatment is high‐titer inhibitor development. Evolving data on current treatment products as well as emerging therapeutics may inform treatment decisions to prevent bleeding and inhibitor formation. Considerations for diagnosis, education, and shared decision‐making related to product choice and treatment regimen are discussed.

show abstract

Understanding Early Hemophilic Arthropathy in Children and Adolescents Through MRI T₂ Mapping

Majeed

Ahmed

Sussman

et al. 2020

Magnetic Resonance Imaging

View full text Add to dashboard Cite

Background Persons with hemophilia experience hemarthrosis, which can lead to cartilage degeneration, causing physical impairment. MRI T2 mapping has the potential to be used as a tool to evaluate early arthropathic changes and cartilage degeneration in patients with hemophilia. Purpose To assess the value of MRI‐T2 mapping as a tool for investigating the cartilage status of children and adolescents with hemophilic arthropathy. Study Type Prospective, cross‐sectional. Subjects Twenty‐eight boys with hemophilia (aged 5–17 years) and 23 healthy boys (aged 7–17 years). Field Strength/Sequences A multiecho spin‐echo T2‐weighted gradient echo sequence was used on a 3.0T magnet. Assessment MRI‐T2 maps of ankle (tibia‐talus) (n = 19) or knee (femur‐tibia) (n = 9) cartilage were assessed in hemophilia and healthy groups. An anatomically‐based MRI score was also assigned to each ankle/knee. Statistical Tests Pearson's correlation coefficient (r), linear regression, intraclass correlation coefficient (ICC), and analysis of variance (ANOVA) test. Results Negative associations between age and ankle/knee cartilage T2 relaxation times were found in hemophilia (r = –0.72 [P = 0.03] to –0.55 [P = 0.01]) and healthy (r = –0.84 [P < 0.001] to –0.55 [P = 0.20]) groups. There were nonsignificant associations between ankle cartilage T2 relaxation times and MRI scores (r = –0.15 [P = 0.54] to 0.31 [P = 0.19]). Data Conclusion Results of this clinical investigation emphasize the potential importance of MRI‐T2 maps as a tool to understand the functional status of cartilage in children and adolescents with hemophilic arthropathy, while holding promise for the detection of early cartilage degeneration prior to macroscopic characterization by conventional MRI. MRI‐T2 mapping may provide novel information that is not reflected in the anatomically‐based MRI scoring system. Level of Evidence 3 Technical Efficacy Stage 2

show abstract

Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort

Cited by 33 publications

References 37 publications

Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis Study

Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis Study

How I approach: Previously untreated patients with severe congenital hemophilia A

Understanding Early Hemophilic Arthropathy in Children and Adolescents Through MRI T₂ Mapping

Contact Info

Product

Resources

About

Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort

Cited by 33 publications

References 37 publications

Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis Study

Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis Study

How I approach: Previously untreated patients with severe congenital hemophilia A

Understanding Early Hemophilic Arthropathy in Children and Adolescents Through MRI T2 Mapping

Contact Info

Product

Resources

About

Understanding Early Hemophilic Arthropathy in Children and Adolescents Through MRI T₂ Mapping