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

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 adheren...

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Autores principales: Dover, Saunya, Blanchette, Victor S., Wrathall, Darius, Pullenayegum, Eleanor, Kazandjian, Daniel, Song, Byron, Hawes, Sue Ann, Cloutier, Stéphanie, Rivard, Geroges E., Klaassen, Robert J., Paradis, Elizabeth, Laferriere, Nicole, Stain, Ann Marie, Chan, Anthony K., Israels, Sara J., Sinha, Roona, Steele, MacGregor, Wu, John K. M., Feldman, Brian M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7040543/
https://www.ncbi.nlm.nih.gov/pubmed/32110763
http://dx.doi.org/10.1002/rth2.12301
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author Dover, Saunya
Blanchette, Victor S.
Wrathall, Darius
Pullenayegum, Eleanor
Kazandjian, Daniel
Song, Byron
Hawes, Sue Ann
Cloutier, Stéphanie
Rivard, Geroges E.
Klaassen, Robert J.
Paradis, Elizabeth
Laferriere, Nicole
Stain, Ann Marie
Chan, Anthony K.
Israels, Sara J.
Sinha, Roona
Steele, MacGregor
Wu, John K. M.
Feldman, Brian M.
author_facet Dover, Saunya
Blanchette, Victor S.
Wrathall, Darius
Pullenayegum, Eleanor
Kazandjian, Daniel
Song, Byron
Hawes, Sue Ann
Cloutier, Stéphanie
Rivard, Geroges E.
Klaassen, Robert J.
Paradis, Elizabeth
Laferriere, Nicole
Stain, Ann Marie
Chan, Anthony K.
Israels, Sara J.
Sinha, Roona
Steele, MacGregor
Wu, John K. M.
Feldman, Brian M.
author_sort Dover, Saunya
collection PubMed
description 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.
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spelling pubmed-70405432020-02-27 Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis Study Dover, Saunya Blanchette, Victor S. Wrathall, Darius Pullenayegum, Eleanor Kazandjian, Daniel Song, Byron Hawes, Sue Ann Cloutier, Stéphanie Rivard, Geroges E. Klaassen, Robert J. Paradis, Elizabeth Laferriere, Nicole Stain, Ann Marie Chan, Anthony K. Israels, Sara J. Sinha, Roona Steele, MacGregor Wu, John K. M. Feldman, Brian M. Res Pract Thromb Haemost Original Articles: Hemostasis 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. John Wiley and Sons Inc. 2020-01-29 /pmc/articles/PMC7040543/ /pubmed/32110763 http://dx.doi.org/10.1002/rth2.12301 Text en © 2020 The Authors. Research and Practice in Thrombosis and Haemostasis published by Wiley Periodicals, Inc on behalf of International Society on Thrombosis and Haemostasis. This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.
spellingShingle Original Articles: Hemostasis
Dover, Saunya
Blanchette, Victor S.
Wrathall, Darius
Pullenayegum, Eleanor
Kazandjian, Daniel
Song, Byron
Hawes, Sue Ann
Cloutier, Stéphanie
Rivard, Geroges E.
Klaassen, Robert J.
Paradis, Elizabeth
Laferriere, Nicole
Stain, Ann Marie
Chan, Anthony K.
Israels, Sara J.
Sinha, Roona
Steele, MacGregor
Wu, John K. M.
Feldman, Brian M.
Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis Study
title 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_short 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
topic Original Articles: Hemostasis
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7040543/
https://www.ncbi.nlm.nih.gov/pubmed/32110763
http://dx.doi.org/10.1002/rth2.12301
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