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BAY 81-8973 Demonstrates Long-Term Safety and Efficacy in Children With Severe Haemophilia A : Results From the LEOPOLD Kids Extension Study

Ljung, Rolf LU orcid ; Chan, Anthony K C ; Ahuja, Sanjay P ; Mancuso, Maria Elisa ; Marquez, Jose Francisco Cabre ; Volk, Florian ; Blanchette, Victor ; Kerlin, Bryce A ; Trakymiene, Sonata Saulyte and Glosli, Heidi , et al. (2024) In European Journal of Haematology
Abstract

OBJECTIVES: To report the long-term safety and efficacy of BAY 81-8973 in the LEOPOLD Kids extension phase.

METHODS: Patients received BAY 81-8973 (25-50 IU/kg) at least twice weekly. The primary endpoint was safety, assessed in all patients who entered the extension phase (n = 82). Efficacy endpoints were assessed in patients without high-titre inhibitors/immune tolerance induction (n = 67).

RESULTS: Children (n = 82) received BAY 81-8973 for a median of 3.1 years per patient and a median of 405 exposure days per patient. Long-term BAY 81-8973 treatment was well tolerated, with no cases of de novo inhibitor development in the extension phase. Annualised bleeding rates (ABRs) within 48 h of prophylaxis were low for all... (More)

OBJECTIVES: To report the long-term safety and efficacy of BAY 81-8973 in the LEOPOLD Kids extension phase.

METHODS: Patients received BAY 81-8973 (25-50 IU/kg) at least twice weekly. The primary endpoint was safety, assessed in all patients who entered the extension phase (n = 82). Efficacy endpoints were assessed in patients without high-titre inhibitors/immune tolerance induction (n = 67).

RESULTS: Children (n = 82) received BAY 81-8973 for a median of 3.1 years per patient and a median of 405 exposure days per patient. Long-term BAY 81-8973 treatment was well tolerated, with no cases of de novo inhibitor development in the extension phase. Annualised bleeding rates (ABRs) within 48 h of prophylaxis were low for all bleeds (median [IQR], 0.7 [0-1.9]; mean, 1.4 [SD, 2.1]) and for joint bleeds (median [IQR], 0 [0-0.7]; mean, 0.5 [SD, 1.1]) (n = 67). Twenty-one of 67 patients (31.3%) had zero bleeds within 48 h of prophylaxis; the treatment response was 'good'/'excellent' in 87.9% of bleeds, and most bleeds resolved with ≤ 2 BAY 81-8973 infusions (83.5%).

CONCLUSION: Long-term BAY 81-8973 treatment is well tolerated and maintains low ABRs for all bleeds and joint bleeds in children with severe haemophilia A.

TRIAL REGISTRATION: ClinicalTrials.gov identifier: NCT01311648.

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Contribution to journal
publication status
epub
subject
in
European Journal of Haematology
publisher
Wiley-Blackwell
external identifiers
  • scopus:85211823601
  • pmid:39667975
ISSN
1600-0609
DOI
10.1111/ejh.14362
language
English
LU publication?
yes
id
730e9b1a-06f4-43de-95ea-e93c93c3a2af
date added to LUP
2024-12-13 10:04:02
date last changed
2025-07-05 02:41:42
@article{730e9b1a-06f4-43de-95ea-e93c93c3a2af,
  abstract     = {{<p>OBJECTIVES: To report the long-term safety and efficacy of BAY 81-8973 in the LEOPOLD Kids extension phase.</p><p>METHODS: Patients received BAY 81-8973 (25-50 IU/kg) at least twice weekly. The primary endpoint was safety, assessed in all patients who entered the extension phase (n = 82). Efficacy endpoints were assessed in patients without high-titre inhibitors/immune tolerance induction (n = 67).</p><p>RESULTS: Children (n = 82) received BAY 81-8973 for a median of 3.1 years per patient and a median of 405 exposure days per patient. Long-term BAY 81-8973 treatment was well tolerated, with no cases of de novo inhibitor development in the extension phase. Annualised bleeding rates (ABRs) within 48 h of prophylaxis were low for all bleeds (median [IQR], 0.7 [0-1.9]; mean, 1.4 [SD, 2.1]) and for joint bleeds (median [IQR], 0 [0-0.7]; mean, 0.5 [SD, 1.1]) (n = 67). Twenty-one of 67 patients (31.3%) had zero bleeds within 48 h of prophylaxis; the treatment response was 'good'/'excellent' in 87.9% of bleeds, and most bleeds resolved with ≤ 2 BAY 81-8973 infusions (83.5%).</p><p>CONCLUSION: Long-term BAY 81-8973 treatment is well tolerated and maintains low ABRs for all bleeds and joint bleeds in children with severe haemophilia A.</p><p>TRIAL REGISTRATION: ClinicalTrials.gov identifier: NCT01311648.</p>}},
  author       = {{Ljung, Rolf and Chan, Anthony K C and Ahuja, Sanjay P and Mancuso, Maria Elisa and Marquez, Jose Francisco Cabre and Volk, Florian and Blanchette, Victor and Kerlin, Bryce A and Trakymiene, Sonata Saulyte and Glosli, Heidi and Kenet, Gili}},
  issn         = {{1600-0609}},
  language     = {{eng}},
  month        = {{12}},
  publisher    = {{Wiley-Blackwell}},
  series       = {{European Journal of Haematology}},
  title        = {{BAY 81-8973 Demonstrates Long-Term Safety and Efficacy in Children With Severe Haemophilia A : Results From the LEOPOLD Kids Extension Study}},
  url          = {{http://dx.doi.org/10.1111/ejh.14362}},
  doi          = {{10.1111/ejh.14362}},
  year         = {{2024}},
}