Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis Study
Abstract 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...
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doaj-abd4dde1d5e44b079b95eefa7c063dbe2020-11-25T02:11:44ZengWileyResearch and Practice in Thrombosis and Haemostasis2475-03792020-02-014231832510.1002/rth2.12301Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis StudySaunya Dover0Victor S. Blanchette1Darius Wrathall2Eleanor Pullenayegum3Daniel Kazandjian4Byron Song5Sue Ann Hawes6Stéphanie Cloutier7Geroges E. Rivard8Robert J. Klaassen9Elizabeth Paradis10Nicole Laferriere11Ann Marie Stain12Anthony K. Chan13Sara J. Israels14Roona Sinha15MacGregor Steele16John K. M. Wu17Brian M. Feldman18Child Health Evaluative Sciences The Hospital for Sick Children Toronto ON CanadaChild Health Evaluative Sciences The Hospital for Sick Children Toronto ON CanadaChild Health Evaluative Sciences The Hospital for Sick Children Toronto ON CanadaChild Health Evaluative Sciences The Hospital for Sick Children Toronto ON CanadaChild Health Evaluative Sciences The Hospital for Sick Children Toronto ON CanadaChild Health Evaluative Sciences The Hospital for Sick Children Toronto ON CanadaDivision of Pediatric Hematology/Oncology IWK Health Centre Halifax NS CanadaCentre de l'hémophilie de l'est du Québec Hôpital de l’Enfant‐JésusUniversité Laval Québec QC CanadaDivision of Hematology/Oncology Department of Pediatrics CHU Sainte‐Justine Montreal QC CanadaDivision of Pediatric Hematology/Oncology Children’s Hospital of Eastern Ontario University of Ottawa Ottawa ON CanadaHealth Sciences North Sudbury ON CanadaDivision of Hematology/Oncology Thunder Bay Regional Cancer Care Thunder Bay ON CanadaDivision of Hematology/Oncology The Hospital for Sick Children Toronto ON CanadaDepartment of Pediatrics McMaster Children’s Hospital McMaster University Hamilton ON CanadaDepartment of Pediatrics and Child Health University of Manitoba Winnipeg MB CanadaSaskatchewan Bleeding Disorders Program Royal University Hospital Saskatoon SK CanadaSection of Pediatric Hematology Department of Pediatrics University of Calgary and Alberta Children’s Hospital Calgary AB CanadaDivision of Hematology/Oncology/BMT Department of Pediatrics UBC & BC Children’s Hospital Vancouver BC CanadaChild Health Evaluative Sciences The Hospital for Sick Children Toronto ON CanadaAbstract 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.https://doi.org/10.1002/rth2.12301bleedingfactor VIIIhemophilia Aprophylaxistreatment adherence and compliance |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Saunya Dover Victor S. Blanchette Darius Wrathall Eleanor Pullenayegum Daniel Kazandjian Byron Song Sue Ann Hawes Stéphanie Cloutier Geroges E. Rivard Robert J. Klaassen Elizabeth Paradis Nicole Laferriere Ann Marie Stain Anthony K. Chan Sara J. Israels Roona Sinha MacGregor Steele John K. M. Wu Brian M. Feldman |
spellingShingle |
Saunya Dover Victor S. Blanchette Darius Wrathall Eleanor Pullenayegum Daniel Kazandjian Byron Song Sue Ann Hawes Stéphanie Cloutier Geroges E. Rivard Robert J. Klaassen Elizabeth Paradis Nicole Laferriere Ann Marie Stain Anthony K. Chan Sara J. Israels Roona Sinha MacGregor Steele John K. M. Wu Brian M. Feldman Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis Study Research and Practice in Thrombosis and Haemostasis bleeding factor VIII hemophilia A prophylaxis treatment adherence and compliance |
author_facet |
Saunya Dover Victor S. Blanchette Darius Wrathall Eleanor Pullenayegum Daniel Kazandjian Byron Song Sue Ann Hawes Stéphanie Cloutier Geroges E. Rivard Robert J. Klaassen Elizabeth Paradis Nicole Laferriere Ann Marie Stain Anthony K. Chan Sara J. Israels Roona Sinha MacGregor Steele John K. M. Wu Brian M. Feldman |
author_sort |
Saunya Dover |
title |
Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis Study |
title_short |
Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis Study |
title_full |
Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis Study |
title_fullStr |
Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis Study |
title_full_unstemmed |
Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis Study |
title_sort |
hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: the canadian hemophilia primary prophylaxis study |
publisher |
Wiley |
series |
Research and Practice in Thrombosis and Haemostasis |
issn |
2475-0379 |
publishDate |
2020-02-01 |
description |
Abstract 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. |
topic |
bleeding factor VIII hemophilia A prophylaxis treatment adherence and compliance |
url |
https://doi.org/10.1002/rth2.12301 |
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