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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...
Autores principales: | , , , , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Hematology
2022
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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. |
format | Online Article Text |
id | pubmed-9327532 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | American Society of Hematology |
record_format | MEDLINE/PubMed |
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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