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Continued benefit demonstrated with BAY 81-8973 prophylaxis in previously treated children with severe haemophilia A : Interim analysis from the LEOPOLD Kids extension study

Kenet, Gili ; Ljung, Rolf LU orcid ; Rusen, Luminita ; Kerlin, Bryce A ; Blanchette, Victor ; Saulytė Trakymienė, Sonata ; Uscatescu, Valentina ; Beckmann, Horst ; Tseneklidou-Stoeter, Despina and Church, Nikki (2020) In Thrombosis Research 189. p.96-101
Abstract

INTRODUCTION: BAY 81-8973 (Kovaltry®), a recombinant factor VIII (rFVIII) product, was efficacious and well tolerated in paediatric previously treated patients (PTPs) with severe haemophilia A for ≥50 exposure days (EDs) in the LEOPOLD Kids study. Because long-term prophylaxis (≥100 EDs) can provide substantial patient benefits, FVIII products should demonstrate long-term safety and efficacy.

AIM: To demonstrate long-term (≥100 EDs) efficacy and safety of BAY 81-8973 in paediatric PTPs.

METHODS: PTPs aged ≤12 years with severe haemophilia A without inhibitors could continue in the ongoing open-label extension study after completing ≥50 EDs in the LEOPOLD Kids main study. Patients received BAY 81-8973 for prophylaxis (25-50... (More)

INTRODUCTION: BAY 81-8973 (Kovaltry®), a recombinant factor VIII (rFVIII) product, was efficacious and well tolerated in paediatric previously treated patients (PTPs) with severe haemophilia A for ≥50 exposure days (EDs) in the LEOPOLD Kids study. Because long-term prophylaxis (≥100 EDs) can provide substantial patient benefits, FVIII products should demonstrate long-term safety and efficacy.

AIM: To demonstrate long-term (≥100 EDs) efficacy and safety of BAY 81-8973 in paediatric PTPs.

METHODS: PTPs aged ≤12 years with severe haemophilia A without inhibitors could continue in the ongoing open-label extension study after completing ≥50 EDs in the LEOPOLD Kids main study. Patients received BAY 81-8973 for prophylaxis (25-50 IU/kg ≥2×/week), bleed treatment, and surgery. Bleeds were documented in electronic patient diaries. Inhibitor development was monitored every 6 months.

RESULTS: At the August 2017 interim data cutoff, 46 patients (median [range] age at enrolment, 6.0 [1.0-11.0] years) had spent a median (range) of 602.5 (148-1069) EDs and 4.6 (1.0-5.9) years in the main plus extension studies. Median (quartile [Q]1; Q3) annualised bleeding rate for bleeds within 48 h after a prophylaxis infusion and total bleeds was 1.0 (0.2; 1.9) and 2.0 (0.4; 3.6), respectively. Most (>94%) bleeds were mild or moderate; 71.8% were treated with ≤1 infusion. BAY 81-8973 was also well tolerated with only one treatment-related adverse event (transient, low-titre inhibitor which did not require treatment adjustment).

CONCLUSION: BAY 81-8973 was efficacious for prophylaxis and treatment of bleeds during >4.5 years in paediatric PTPs with severe haemophilia A.

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organization
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type
Contribution to journal
publication status
published
subject
in
Thrombosis Research
volume
189
pages
6 pages
publisher
Elsevier
external identifiers
  • pmid:32197139
  • scopus:85081672189
ISSN
1879-2472
DOI
10.1016/j.thromres.2020.03.005
language
English
LU publication?
yes
id
6c482f47-315b-4966-abbb-63959d831199
date added to LUP
2020-03-26 19:28:07
date last changed
2024-04-03 04:53:05
@article{6c482f47-315b-4966-abbb-63959d831199,
  abstract     = {{<p>INTRODUCTION: BAY 81-8973 (Kovaltry®), a recombinant factor VIII (rFVIII) product, was efficacious and well tolerated in paediatric previously treated patients (PTPs) with severe haemophilia A for ≥50 exposure days (EDs) in the LEOPOLD Kids study. Because long-term prophylaxis (≥100 EDs) can provide substantial patient benefits, FVIII products should demonstrate long-term safety and efficacy.</p><p>AIM: To demonstrate long-term (≥100 EDs) efficacy and safety of BAY 81-8973 in paediatric PTPs.</p><p>METHODS: PTPs aged ≤12 years with severe haemophilia A without inhibitors could continue in the ongoing open-label extension study after completing ≥50 EDs in the LEOPOLD Kids main study. Patients received BAY 81-8973 for prophylaxis (25-50 IU/kg ≥2×/week), bleed treatment, and surgery. Bleeds were documented in electronic patient diaries. Inhibitor development was monitored every 6 months.</p><p>RESULTS: At the August 2017 interim data cutoff, 46 patients (median [range] age at enrolment, 6.0 [1.0-11.0] years) had spent a median (range) of 602.5 (148-1069) EDs and 4.6 (1.0-5.9) years in the main plus extension studies. Median (quartile [Q]1; Q3) annualised bleeding rate for bleeds within 48 h after a prophylaxis infusion and total bleeds was 1.0 (0.2; 1.9) and 2.0 (0.4; 3.6), respectively. Most (&gt;94%) bleeds were mild or moderate; 71.8% were treated with ≤1 infusion. BAY 81-8973 was also well tolerated with only one treatment-related adverse event (transient, low-titre inhibitor which did not require treatment adjustment).</p><p>CONCLUSION: BAY 81-8973 was efficacious for prophylaxis and treatment of bleeds during &gt;4.5 years in paediatric PTPs with severe haemophilia A.</p>}},
  author       = {{Kenet, Gili and Ljung, Rolf and Rusen, Luminita and Kerlin, Bryce A and Blanchette, Victor and Saulytė Trakymienė, Sonata and Uscatescu, Valentina and Beckmann, Horst and Tseneklidou-Stoeter, Despina and Church, Nikki}},
  issn         = {{1879-2472}},
  language     = {{eng}},
  pages        = {{96--101}},
  publisher    = {{Elsevier}},
  series       = {{Thrombosis Research}},
  title        = {{Continued benefit demonstrated with BAY 81-8973 prophylaxis in previously treated children with severe haemophilia A : Interim analysis from the LEOPOLD Kids extension study}},
  url          = {{http://dx.doi.org/10.1016/j.thromres.2020.03.005}},
  doi          = {{10.1016/j.thromres.2020.03.005}},
  volume       = {{189}},
  year         = {{2020}},
}