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The bleeding phenotype in people with nonsevere hemophilia

Detailed information on the onset, frequency, and severity of bleeding in nonsevere hemophilia is limited. We aimed to assess the bleeding phenotype of persons with nonsevere hemophilia and to analyze the association between baseline factor VIII/IX (FVIII/IX) levels and the joint bleeding rate. In t...

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Autores principales: Kloosterman, Fabienne R., Zwagemaker, Anne-Fleur, Bagot, Catherine N., Beckers, Erik A. M., Castaman, Giancarlo, Cnossen, Marjon H., Collins, Peter W., Hay, Charles, Hof, Michel, Laros-van Gorkom, Britta, Leebeek, Frank W. G., Male, Christoph, Meijer, Karina, Pabinger, Ingrid, Shapiro, Susan, Coppens, Michiel, Fijnvandraat, Karin, Gouw,, Samantha C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Hematology 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9327532/
https://www.ncbi.nlm.nih.gov/pubmed/35533261
http://dx.doi.org/10.1182/bloodadvances.2022007620
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author Kloosterman, Fabienne R.
Zwagemaker, Anne-Fleur
Bagot, Catherine N.
Beckers, Erik A. M.
Castaman, Giancarlo
Cnossen, Marjon H.
Collins, Peter W.
Hay, Charles
Hof, Michel
Laros-van Gorkom, Britta
Leebeek, Frank W. G.
Male, Christoph
Meijer, Karina
Pabinger, Ingrid
Shapiro, Susan
Coppens, Michiel
Fijnvandraat, Karin
Gouw,, Samantha C.
author_facet Kloosterman, Fabienne R.
Zwagemaker, Anne-Fleur
Bagot, Catherine N.
Beckers, Erik A. M.
Castaman, Giancarlo
Cnossen, Marjon H.
Collins, Peter W.
Hay, Charles
Hof, Michel
Laros-van Gorkom, Britta
Leebeek, Frank W. G.
Male, Christoph
Meijer, Karina
Pabinger, Ingrid
Shapiro, Susan
Coppens, Michiel
Fijnvandraat, Karin
Gouw,, Samantha C.
author_sort Kloosterman, Fabienne R.
collection PubMed
description Detailed information on the onset, frequency, and severity of bleeding in nonsevere hemophilia is limited. We aimed to assess the bleeding phenotype of persons with nonsevere hemophilia and to analyze the association between baseline factor VIII/IX (FVIII/IX) levels and the joint bleeding rate. In the DYNAMO (Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B) study, an international multicenter cohort, we included males with nonsevere hemophilia (FVIII/IX, 0.02-0.35 IU/mL) aged 12 to 55 years. Information on age at first treated (joint) bleed, annual bleeding rates (ABRs), and annual joint bleeding rates (AJBRs) was collected from the medical files. The association between baseline FVIII/IX levels and the joint bleeding rate was assessed by using a frailty model for recurrent events. In total, 304 persons (70 with moderate hemophilia and 234 with mild hemophilia) were included. The median age was 38 years (interquartile range [IQR], 25-49 years), and the median baseline FVIII/IX level was 0.12 IU/mL (IQR, 0.05-0.21 IU/mL). In total, 245 (81%) persons had experienced at least 1 bleed, and 156 (51%) had experienced at least 1 joint bleed. The median age at first bleed and first joint bleed was 8 and 10 years, respectively. The median ABR and AJBR was 0.2 (IQR, 0.1-0.5) and 0.0 (IQR, 0.0-0.2). From baseline FVIII/IX levels 0.02 to 0.05 IU/mL to >0.25 IU/mL, the median ABR decreased from 0.6 (IQR, 0.2-1.4) to 0.1 (IQR, 0.0-0.2) and the AJBR from 0.2 (IQR, 0.0-0.4) to 0.0 (IQR, 0.0-0.0). Baseline FVIII/IX was inversely associated with the joint bleeding rate (P < .001). Low bleeding rates were observed in persons with nonsevere hemophilia. However, one-half of all adolescents and adults had experienced a joint bleed.
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spelling pubmed-93275322022-08-01 The bleeding phenotype in people with nonsevere hemophilia Kloosterman, Fabienne R. Zwagemaker, Anne-Fleur Bagot, Catherine N. Beckers, Erik A. M. Castaman, Giancarlo Cnossen, Marjon H. Collins, Peter W. Hay, Charles Hof, Michel Laros-van Gorkom, Britta Leebeek, Frank W. G. Male, Christoph Meijer, Karina Pabinger, Ingrid Shapiro, Susan Coppens, Michiel Fijnvandraat, Karin Gouw,, Samantha C. Blood Adv Thrombosis and Hemostasis Detailed information on the onset, frequency, and severity of bleeding in nonsevere hemophilia is limited. We aimed to assess the bleeding phenotype of persons with nonsevere hemophilia and to analyze the association between baseline factor VIII/IX (FVIII/IX) levels and the joint bleeding rate. In the DYNAMO (Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B) study, an international multicenter cohort, we included males with nonsevere hemophilia (FVIII/IX, 0.02-0.35 IU/mL) aged 12 to 55 years. Information on age at first treated (joint) bleed, annual bleeding rates (ABRs), and annual joint bleeding rates (AJBRs) was collected from the medical files. The association between baseline FVIII/IX levels and the joint bleeding rate was assessed by using a frailty model for recurrent events. In total, 304 persons (70 with moderate hemophilia and 234 with mild hemophilia) were included. The median age was 38 years (interquartile range [IQR], 25-49 years), and the median baseline FVIII/IX level was 0.12 IU/mL (IQR, 0.05-0.21 IU/mL). In total, 245 (81%) persons had experienced at least 1 bleed, and 156 (51%) had experienced at least 1 joint bleed. The median age at first bleed and first joint bleed was 8 and 10 years, respectively. The median ABR and AJBR was 0.2 (IQR, 0.1-0.5) and 0.0 (IQR, 0.0-0.2). From baseline FVIII/IX levels 0.02 to 0.05 IU/mL to >0.25 IU/mL, the median ABR decreased from 0.6 (IQR, 0.2-1.4) to 0.1 (IQR, 0.0-0.2) and the AJBR from 0.2 (IQR, 0.0-0.4) to 0.0 (IQR, 0.0-0.0). Baseline FVIII/IX was inversely associated with the joint bleeding rate (P < .001). Low bleeding rates were observed in persons with nonsevere hemophilia. However, one-half of all adolescents and adults had experienced a joint bleed. American Society of Hematology 2022-07-21 /pmc/articles/PMC9327532/ /pubmed/35533261 http://dx.doi.org/10.1182/bloodadvances.2022007620 Text en © 2022 by The American Society of Hematology. Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved.
spellingShingle Thrombosis and Hemostasis
Kloosterman, Fabienne R.
Zwagemaker, Anne-Fleur
Bagot, Catherine N.
Beckers, Erik A. M.
Castaman, Giancarlo
Cnossen, Marjon H.
Collins, Peter W.
Hay, Charles
Hof, Michel
Laros-van Gorkom, Britta
Leebeek, Frank W. G.
Male, Christoph
Meijer, Karina
Pabinger, Ingrid
Shapiro, Susan
Coppens, Michiel
Fijnvandraat, Karin
Gouw,, Samantha C.
The bleeding phenotype in people with nonsevere hemophilia
title The bleeding phenotype in people with nonsevere hemophilia
title_full The bleeding phenotype in people with nonsevere hemophilia
title_fullStr The bleeding phenotype in people with nonsevere hemophilia
title_full_unstemmed The bleeding phenotype in people with nonsevere hemophilia
title_short The bleeding phenotype in people with nonsevere hemophilia
title_sort bleeding phenotype in people with nonsevere hemophilia
topic Thrombosis and Hemostasis
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9327532/
https://www.ncbi.nlm.nih.gov/pubmed/35533261
http://dx.doi.org/10.1182/bloodadvances.2022007620
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