Patient perspective on living with mild hemophilia in Germany: results from a nationwide survey
IntroductionThe disease burden and bleeding risk of patients with mild hemophilia may be underestimated. Their health-related quality of life (QoL) may be negatively impacted by insufficient treatment and bleed-related joint damage connected to a potentially delayed diagnosis.AimThis study aims to g...
Ausführliche Beschreibung
Autor*in: |
Rosa Sonja Alesci [verfasserIn] Georg Goldmann [verfasserIn] Susan Halimeh [verfasserIn] Katharina Holstein [verfasserIn] Christoph Königs [verfasserIn] Wolfgang Miesbach [verfasserIn] Christian Pfrepper [verfasserIn] Martin Olivieri [verfasserIn] |
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E-Artikel |
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Sprache: |
Englisch |
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2024 |
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Übergeordnetes Werk: |
In: Frontiers in Medicine - Frontiers Media S.A., 2014, 11(2024) |
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Übergeordnetes Werk: |
volume:11 ; year:2024 |
Links: |
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DOI / URN: |
10.3389/fmed.2024.1347024 |
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Katalog-ID: |
DOAJ094875707 |
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520 | |a IntroductionThe disease burden and bleeding risk of patients with mild hemophilia may be underestimated. Their health-related quality of life (QoL) may be negatively impacted by insufficient treatment and bleed-related joint damage connected to a potentially delayed diagnosis.AimThis study aims to gain information on the care reality and QoL of patients aged ≥12 years with mild hemophilia in Germany.MethodsAn anonymous cross-sectional patient survey using standardized questionnaires was conducted in a validated electronic patient-reported outcome system. Medical specialists, hemophilia centers, patient organizations, and support groups across Germany invited the patients.ResultsA total of 43 patients (35 patients with hemophilia A, 5 patients with hemophilia B, and 3 patients for whom the information was missing) with a median age of 33 years were analyzed. The median age at diagnosis was 6.0 years (interquartile range [IQR] 2.0–15.0), and the median factor activity was 14.0% (IQR 12.0–25.0). Nearly 85% of the patients received factor concentrates in the past, and the most common reasons for the treatment were surgery or joint bleeding (each 65.6%). Half of the patients who provided feedback experienced complications during bleeding episodes. Prophylactic treatment with factor concentrates was rare (10.3%). The patients had minor problems regarding their health status.ConclusionBleeding complications and joint bleeding, in particular, may be highly underestimated in patients with mild hemophilia, highlighting a medical need in this population. Patients with a potential benefit from prophylaxis need to be identified. Mild hemophilia has a negative impact on patients’ QoL. Hemophilia centers satisfied the patients’ needs. Further research is needed to address the current lack of awareness and improve adequate treatment in the future. | ||
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10.3389/fmed.2024.1347024 doi (DE-627)DOAJ094875707 (DE-599)DOAJ19f8d462bcaa46e585eedb6913bcf9bd DE-627 ger DE-627 rakwb eng R5-920 Rosa Sonja Alesci verfasserin aut Patient perspective on living with mild hemophilia in Germany: results from a nationwide survey 2024 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier IntroductionThe disease burden and bleeding risk of patients with mild hemophilia may be underestimated. Their health-related quality of life (QoL) may be negatively impacted by insufficient treatment and bleed-related joint damage connected to a potentially delayed diagnosis.AimThis study aims to gain information on the care reality and QoL of patients aged ≥12 years with mild hemophilia in Germany.MethodsAn anonymous cross-sectional patient survey using standardized questionnaires was conducted in a validated electronic patient-reported outcome system. Medical specialists, hemophilia centers, patient organizations, and support groups across Germany invited the patients.ResultsA total of 43 patients (35 patients with hemophilia A, 5 patients with hemophilia B, and 3 patients for whom the information was missing) with a median age of 33 years were analyzed. The median age at diagnosis was 6.0 years (interquartile range [IQR] 2.0–15.0), and the median factor activity was 14.0% (IQR 12.0–25.0). Nearly 85% of the patients received factor concentrates in the past, and the most common reasons for the treatment were surgery or joint bleeding (each 65.6%). Half of the patients who provided feedback experienced complications during bleeding episodes. Prophylactic treatment with factor concentrates was rare (10.3%). The patients had minor problems regarding their health status.ConclusionBleeding complications and joint bleeding, in particular, may be highly underestimated in patients with mild hemophilia, highlighting a medical need in this population. Patients with a potential benefit from prophylaxis need to be identified. Mild hemophilia has a negative impact on patients’ QoL. Hemophilia centers satisfied the patients’ needs. Further research is needed to address the current lack of awareness and improve adequate treatment in the future. mild hemophilia hemophilia A hemophilia B care reality joint bleeding quality of life Medicine (General) Georg Goldmann verfasserin aut Susan Halimeh verfasserin aut Katharina Holstein verfasserin aut Christoph Königs verfasserin aut Wolfgang Miesbach verfasserin aut Christian Pfrepper verfasserin aut Martin Olivieri verfasserin aut In Frontiers in Medicine Frontiers Media S.A., 2014 11(2024) (DE-627)789482991 (DE-600)2775999-4 2296858X nnns volume:11 year:2024 https://doi.org/10.3389/fmed.2024.1347024 kostenfrei https://doaj.org/article/19f8d462bcaa46e585eedb6913bcf9bd kostenfrei https://www.frontiersin.org/articles/10.3389/fmed.2024.1347024/full kostenfrei https://doaj.org/toc/2296-858X Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 11 2024 |
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10.3389/fmed.2024.1347024 doi (DE-627)DOAJ094875707 (DE-599)DOAJ19f8d462bcaa46e585eedb6913bcf9bd DE-627 ger DE-627 rakwb eng R5-920 Rosa Sonja Alesci verfasserin aut Patient perspective on living with mild hemophilia in Germany: results from a nationwide survey 2024 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier IntroductionThe disease burden and bleeding risk of patients with mild hemophilia may be underestimated. Their health-related quality of life (QoL) may be negatively impacted by insufficient treatment and bleed-related joint damage connected to a potentially delayed diagnosis.AimThis study aims to gain information on the care reality and QoL of patients aged ≥12 years with mild hemophilia in Germany.MethodsAn anonymous cross-sectional patient survey using standardized questionnaires was conducted in a validated electronic patient-reported outcome system. Medical specialists, hemophilia centers, patient organizations, and support groups across Germany invited the patients.ResultsA total of 43 patients (35 patients with hemophilia A, 5 patients with hemophilia B, and 3 patients for whom the information was missing) with a median age of 33 years were analyzed. The median age at diagnosis was 6.0 years (interquartile range [IQR] 2.0–15.0), and the median factor activity was 14.0% (IQR 12.0–25.0). Nearly 85% of the patients received factor concentrates in the past, and the most common reasons for the treatment were surgery or joint bleeding (each 65.6%). Half of the patients who provided feedback experienced complications during bleeding episodes. Prophylactic treatment with factor concentrates was rare (10.3%). The patients had minor problems regarding their health status.ConclusionBleeding complications and joint bleeding, in particular, may be highly underestimated in patients with mild hemophilia, highlighting a medical need in this population. Patients with a potential benefit from prophylaxis need to be identified. Mild hemophilia has a negative impact on patients’ QoL. Hemophilia centers satisfied the patients’ needs. Further research is needed to address the current lack of awareness and improve adequate treatment in the future. mild hemophilia hemophilia A hemophilia B care reality joint bleeding quality of life Medicine (General) Georg Goldmann verfasserin aut Susan Halimeh verfasserin aut Katharina Holstein verfasserin aut Christoph Königs verfasserin aut Wolfgang Miesbach verfasserin aut Christian Pfrepper verfasserin aut Martin Olivieri verfasserin aut In Frontiers in Medicine Frontiers Media S.A., 2014 11(2024) (DE-627)789482991 (DE-600)2775999-4 2296858X nnns volume:11 year:2024 https://doi.org/10.3389/fmed.2024.1347024 kostenfrei https://doaj.org/article/19f8d462bcaa46e585eedb6913bcf9bd kostenfrei https://www.frontiersin.org/articles/10.3389/fmed.2024.1347024/full kostenfrei https://doaj.org/toc/2296-858X Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 11 2024 |
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10.3389/fmed.2024.1347024 doi (DE-627)DOAJ094875707 (DE-599)DOAJ19f8d462bcaa46e585eedb6913bcf9bd DE-627 ger DE-627 rakwb eng R5-920 Rosa Sonja Alesci verfasserin aut Patient perspective on living with mild hemophilia in Germany: results from a nationwide survey 2024 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier IntroductionThe disease burden and bleeding risk of patients with mild hemophilia may be underestimated. Their health-related quality of life (QoL) may be negatively impacted by insufficient treatment and bleed-related joint damage connected to a potentially delayed diagnosis.AimThis study aims to gain information on the care reality and QoL of patients aged ≥12 years with mild hemophilia in Germany.MethodsAn anonymous cross-sectional patient survey using standardized questionnaires was conducted in a validated electronic patient-reported outcome system. Medical specialists, hemophilia centers, patient organizations, and support groups across Germany invited the patients.ResultsA total of 43 patients (35 patients with hemophilia A, 5 patients with hemophilia B, and 3 patients for whom the information was missing) with a median age of 33 years were analyzed. The median age at diagnosis was 6.0 years (interquartile range [IQR] 2.0–15.0), and the median factor activity was 14.0% (IQR 12.0–25.0). Nearly 85% of the patients received factor concentrates in the past, and the most common reasons for the treatment were surgery or joint bleeding (each 65.6%). Half of the patients who provided feedback experienced complications during bleeding episodes. Prophylactic treatment with factor concentrates was rare (10.3%). The patients had minor problems regarding their health status.ConclusionBleeding complications and joint bleeding, in particular, may be highly underestimated in patients with mild hemophilia, highlighting a medical need in this population. Patients with a potential benefit from prophylaxis need to be identified. Mild hemophilia has a negative impact on patients’ QoL. Hemophilia centers satisfied the patients’ needs. Further research is needed to address the current lack of awareness and improve adequate treatment in the future. mild hemophilia hemophilia A hemophilia B care reality joint bleeding quality of life Medicine (General) Georg Goldmann verfasserin aut Susan Halimeh verfasserin aut Katharina Holstein verfasserin aut Christoph Königs verfasserin aut Wolfgang Miesbach verfasserin aut Christian Pfrepper verfasserin aut Martin Olivieri verfasserin aut In Frontiers in Medicine Frontiers Media S.A., 2014 11(2024) (DE-627)789482991 (DE-600)2775999-4 2296858X nnns volume:11 year:2024 https://doi.org/10.3389/fmed.2024.1347024 kostenfrei https://doaj.org/article/19f8d462bcaa46e585eedb6913bcf9bd kostenfrei https://www.frontiersin.org/articles/10.3389/fmed.2024.1347024/full kostenfrei https://doaj.org/toc/2296-858X Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 11 2024 |
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10.3389/fmed.2024.1347024 doi (DE-627)DOAJ094875707 (DE-599)DOAJ19f8d462bcaa46e585eedb6913bcf9bd DE-627 ger DE-627 rakwb eng R5-920 Rosa Sonja Alesci verfasserin aut Patient perspective on living with mild hemophilia in Germany: results from a nationwide survey 2024 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier IntroductionThe disease burden and bleeding risk of patients with mild hemophilia may be underestimated. Their health-related quality of life (QoL) may be negatively impacted by insufficient treatment and bleed-related joint damage connected to a potentially delayed diagnosis.AimThis study aims to gain information on the care reality and QoL of patients aged ≥12 years with mild hemophilia in Germany.MethodsAn anonymous cross-sectional patient survey using standardized questionnaires was conducted in a validated electronic patient-reported outcome system. Medical specialists, hemophilia centers, patient organizations, and support groups across Germany invited the patients.ResultsA total of 43 patients (35 patients with hemophilia A, 5 patients with hemophilia B, and 3 patients for whom the information was missing) with a median age of 33 years were analyzed. The median age at diagnosis was 6.0 years (interquartile range [IQR] 2.0–15.0), and the median factor activity was 14.0% (IQR 12.0–25.0). Nearly 85% of the patients received factor concentrates in the past, and the most common reasons for the treatment were surgery or joint bleeding (each 65.6%). Half of the patients who provided feedback experienced complications during bleeding episodes. Prophylactic treatment with factor concentrates was rare (10.3%). The patients had minor problems regarding their health status.ConclusionBleeding complications and joint bleeding, in particular, may be highly underestimated in patients with mild hemophilia, highlighting a medical need in this population. Patients with a potential benefit from prophylaxis need to be identified. Mild hemophilia has a negative impact on patients’ QoL. Hemophilia centers satisfied the patients’ needs. Further research is needed to address the current lack of awareness and improve adequate treatment in the future. mild hemophilia hemophilia A hemophilia B care reality joint bleeding quality of life Medicine (General) Georg Goldmann verfasserin aut Susan Halimeh verfasserin aut Katharina Holstein verfasserin aut Christoph Königs verfasserin aut Wolfgang Miesbach verfasserin aut Christian Pfrepper verfasserin aut Martin Olivieri verfasserin aut In Frontiers in Medicine Frontiers Media S.A., 2014 11(2024) (DE-627)789482991 (DE-600)2775999-4 2296858X nnns volume:11 year:2024 https://doi.org/10.3389/fmed.2024.1347024 kostenfrei https://doaj.org/article/19f8d462bcaa46e585eedb6913bcf9bd kostenfrei https://www.frontiersin.org/articles/10.3389/fmed.2024.1347024/full kostenfrei https://doaj.org/toc/2296-858X Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 11 2024 |
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10.3389/fmed.2024.1347024 doi (DE-627)DOAJ094875707 (DE-599)DOAJ19f8d462bcaa46e585eedb6913bcf9bd DE-627 ger DE-627 rakwb eng R5-920 Rosa Sonja Alesci verfasserin aut Patient perspective on living with mild hemophilia in Germany: results from a nationwide survey 2024 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier IntroductionThe disease burden and bleeding risk of patients with mild hemophilia may be underestimated. Their health-related quality of life (QoL) may be negatively impacted by insufficient treatment and bleed-related joint damage connected to a potentially delayed diagnosis.AimThis study aims to gain information on the care reality and QoL of patients aged ≥12 years with mild hemophilia in Germany.MethodsAn anonymous cross-sectional patient survey using standardized questionnaires was conducted in a validated electronic patient-reported outcome system. Medical specialists, hemophilia centers, patient organizations, and support groups across Germany invited the patients.ResultsA total of 43 patients (35 patients with hemophilia A, 5 patients with hemophilia B, and 3 patients for whom the information was missing) with a median age of 33 years were analyzed. The median age at diagnosis was 6.0 years (interquartile range [IQR] 2.0–15.0), and the median factor activity was 14.0% (IQR 12.0–25.0). Nearly 85% of the patients received factor concentrates in the past, and the most common reasons for the treatment were surgery or joint bleeding (each 65.6%). Half of the patients who provided feedback experienced complications during bleeding episodes. Prophylactic treatment with factor concentrates was rare (10.3%). The patients had minor problems regarding their health status.ConclusionBleeding complications and joint bleeding, in particular, may be highly underestimated in patients with mild hemophilia, highlighting a medical need in this population. Patients with a potential benefit from prophylaxis need to be identified. Mild hemophilia has a negative impact on patients’ QoL. Hemophilia centers satisfied the patients’ needs. Further research is needed to address the current lack of awareness and improve adequate treatment in the future. mild hemophilia hemophilia A hemophilia B care reality joint bleeding quality of life Medicine (General) Georg Goldmann verfasserin aut Susan Halimeh verfasserin aut Katharina Holstein verfasserin aut Christoph Königs verfasserin aut Wolfgang Miesbach verfasserin aut Christian Pfrepper verfasserin aut Martin Olivieri verfasserin aut In Frontiers in Medicine Frontiers Media S.A., 2014 11(2024) (DE-627)789482991 (DE-600)2775999-4 2296858X nnns volume:11 year:2024 https://doi.org/10.3389/fmed.2024.1347024 kostenfrei https://doaj.org/article/19f8d462bcaa46e585eedb6913bcf9bd kostenfrei https://www.frontiersin.org/articles/10.3389/fmed.2024.1347024/full kostenfrei https://doaj.org/toc/2296-858X Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 11 2024 |
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Nearly 85% of the patients received factor concentrates in the past, and the most common reasons for the treatment were surgery or joint bleeding (each 65.6%). Half of the patients who provided feedback experienced complications during bleeding episodes. Prophylactic treatment with factor concentrates was rare (10.3%). The patients had minor problems regarding their health status.ConclusionBleeding complications and joint bleeding, in particular, may be highly underestimated in patients with mild hemophilia, highlighting a medical need in this population. Patients with a potential benefit from prophylaxis need to be identified. Mild hemophilia has a negative impact on patients’ QoL. Hemophilia centers satisfied the patients’ needs. 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Patient perspective on living with mild hemophilia in Germany: results from a nationwide survey |
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IntroductionThe disease burden and bleeding risk of patients with mild hemophilia may be underestimated. Their health-related quality of life (QoL) may be negatively impacted by insufficient treatment and bleed-related joint damage connected to a potentially delayed diagnosis.AimThis study aims to gain information on the care reality and QoL of patients aged ≥12 years with mild hemophilia in Germany.MethodsAn anonymous cross-sectional patient survey using standardized questionnaires was conducted in a validated electronic patient-reported outcome system. Medical specialists, hemophilia centers, patient organizations, and support groups across Germany invited the patients.ResultsA total of 43 patients (35 patients with hemophilia A, 5 patients with hemophilia B, and 3 patients for whom the information was missing) with a median age of 33 years were analyzed. The median age at diagnosis was 6.0 years (interquartile range [IQR] 2.0–15.0), and the median factor activity was 14.0% (IQR 12.0–25.0). Nearly 85% of the patients received factor concentrates in the past, and the most common reasons for the treatment were surgery or joint bleeding (each 65.6%). Half of the patients who provided feedback experienced complications during bleeding episodes. Prophylactic treatment with factor concentrates was rare (10.3%). The patients had minor problems regarding their health status.ConclusionBleeding complications and joint bleeding, in particular, may be highly underestimated in patients with mild hemophilia, highlighting a medical need in this population. Patients with a potential benefit from prophylaxis need to be identified. Mild hemophilia has a negative impact on patients’ QoL. Hemophilia centers satisfied the patients’ needs. Further research is needed to address the current lack of awareness and improve adequate treatment in the future. |
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IntroductionThe disease burden and bleeding risk of patients with mild hemophilia may be underestimated. Their health-related quality of life (QoL) may be negatively impacted by insufficient treatment and bleed-related joint damage connected to a potentially delayed diagnosis.AimThis study aims to gain information on the care reality and QoL of patients aged ≥12 years with mild hemophilia in Germany.MethodsAn anonymous cross-sectional patient survey using standardized questionnaires was conducted in a validated electronic patient-reported outcome system. Medical specialists, hemophilia centers, patient organizations, and support groups across Germany invited the patients.ResultsA total of 43 patients (35 patients with hemophilia A, 5 patients with hemophilia B, and 3 patients for whom the information was missing) with a median age of 33 years were analyzed. The median age at diagnosis was 6.0 years (interquartile range [IQR] 2.0–15.0), and the median factor activity was 14.0% (IQR 12.0–25.0). Nearly 85% of the patients received factor concentrates in the past, and the most common reasons for the treatment were surgery or joint bleeding (each 65.6%). Half of the patients who provided feedback experienced complications during bleeding episodes. Prophylactic treatment with factor concentrates was rare (10.3%). The patients had minor problems regarding their health status.ConclusionBleeding complications and joint bleeding, in particular, may be highly underestimated in patients with mild hemophilia, highlighting a medical need in this population. Patients with a potential benefit from prophylaxis need to be identified. Mild hemophilia has a negative impact on patients’ QoL. Hemophilia centers satisfied the patients’ needs. Further research is needed to address the current lack of awareness and improve adequate treatment in the future. |
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IntroductionThe disease burden and bleeding risk of patients with mild hemophilia may be underestimated. Their health-related quality of life (QoL) may be negatively impacted by insufficient treatment and bleed-related joint damage connected to a potentially delayed diagnosis.AimThis study aims to gain information on the care reality and QoL of patients aged ≥12 years with mild hemophilia in Germany.MethodsAn anonymous cross-sectional patient survey using standardized questionnaires was conducted in a validated electronic patient-reported outcome system. Medical specialists, hemophilia centers, patient organizations, and support groups across Germany invited the patients.ResultsA total of 43 patients (35 patients with hemophilia A, 5 patients with hemophilia B, and 3 patients for whom the information was missing) with a median age of 33 years were analyzed. The median age at diagnosis was 6.0 years (interquartile range [IQR] 2.0–15.0), and the median factor activity was 14.0% (IQR 12.0–25.0). Nearly 85% of the patients received factor concentrates in the past, and the most common reasons for the treatment were surgery or joint bleeding (each 65.6%). Half of the patients who provided feedback experienced complications during bleeding episodes. Prophylactic treatment with factor concentrates was rare (10.3%). The patients had minor problems regarding their health status.ConclusionBleeding complications and joint bleeding, in particular, may be highly underestimated in patients with mild hemophilia, highlighting a medical need in this population. Patients with a potential benefit from prophylaxis need to be identified. Mild hemophilia has a negative impact on patients’ QoL. Hemophilia centers satisfied the patients’ needs. Further research is needed to address the current lack of awareness and improve adequate treatment in the future. |
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Patient perspective on living with mild hemophilia in Germany: results from a nationwide survey |
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Their health-related quality of life (QoL) may be negatively impacted by insufficient treatment and bleed-related joint damage connected to a potentially delayed diagnosis.AimThis study aims to gain information on the care reality and QoL of patients aged ≥12 years with mild hemophilia in Germany.MethodsAn anonymous cross-sectional patient survey using standardized questionnaires was conducted in a validated electronic patient-reported outcome system. Medical specialists, hemophilia centers, patient organizations, and support groups across Germany invited the patients.ResultsA total of 43 patients (35 patients with hemophilia A, 5 patients with hemophilia B, and 3 patients for whom the information was missing) with a median age of 33 years were analyzed. The median age at diagnosis was 6.0 years (interquartile range [IQR] 2.0–15.0), and the median factor activity was 14.0% (IQR 12.0–25.0). Nearly 85% of the patients received factor concentrates in the past, and the most common reasons for the treatment were surgery or joint bleeding (each 65.6%). Half of the patients who provided feedback experienced complications during bleeding episodes. Prophylactic treatment with factor concentrates was rare (10.3%). The patients had minor problems regarding their health status.ConclusionBleeding complications and joint bleeding, in particular, may be highly underestimated in patients with mild hemophilia, highlighting a medical need in this population. Patients with a potential benefit from prophylaxis need to be identified. Mild hemophilia has a negative impact on patients’ QoL. Hemophilia centers satisfied the patients’ needs. 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