Pursuing a multivariable approach in a functionally independent older hemophiliac

Severe hemophilia A is a bleeding disorder indicating plasma factor levels of <1% and usually presents with episodes of spontaneous bleeding. Typically, a patient with severe untreated hemophilia A has at most 20 joint bleeds a year. In contrast, the patient of interest in this case report previously had up to 250 spontaneous bleeding episodes in a year, despite receiving prophylactic treatment. Here, we present a 65-year-old patient with an extreme phenotype of severe hemophilia A who, despite the excessive number of spontaneous bleeds, has demonstrated strong resilience and an uncommon achievement of significant milestones. The patient was managed with a multivariable approach, which included a thorough and consistent physical therapy regimen, prophylactic measures, and multiple different treatments, which most recently incorporated emicizumab (Hemlibra ® ). Because this patient is 65 years old, he has essentially grown up with the scientific advancements in his treatments. This case report suggests the possibility of a significant improvement of quality of life in older populations with severe hemophilia A by considering a multivariable approach.

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Publication Details

Journal
Journal of Hematology and Allied Sciences
Published
2025-03-13
DOI
https://doi.org/10.25259/jhas_62_2025
Primary Topic
Hemophilia Treatment and Research
Type
article
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article

Pursuing a multivariable approach in a functionally independent older hemophiliac

Samuel A. Acuña, Secili DeStefano, Danielle H. Oh
Journal of Hematology and Allied Sciences
Hemophilia Treatment and Research
article

Pursuing a multivariable approach in a functionally independent older hemophiliac

Samuel A. Acuña, Secili DeStefano, Danielle H. Oh
article en

Abstract

Severe hemophilia A is a bleeding disorder indicating plasma factor levels of <1% and usually presents with episodes of spontaneous bleeding. Typically, a patient with severe untreated hemophilia A has at most 20 joint bleeds a year. In contrast, the patient of interest in this case report previously had up to 250 spontaneous bleeding episodes in a year, despite receiving prophylactic treatment. Here, we present a 65-year-old patient with an extreme phenotype of severe hemophilia A who, despite the excessive number of spontaneous bleeds, has demonstrated strong resilience and an uncommon achievement of significant milestones. The patient was managed with a multivariable approach, which included a thorough and consistent physical therapy regimen, prophylactic measures, and multiple different treatments, which most recently incorporated emicizumab (Hemlibra ® ). Because this patient is 65 years old, he has essentially grown up with the scientific advancements in his treatments. This case report suggests the possibility of a significant improvement of quality of life in older populations with severe hemophilia A by considering a multivariable approach.

Journal of Hematology and Allied SciencesVol. 0
George Mason University (US)
Openalex Percentile: Top 20%
Hemophilia Treatment and Research
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