Factors that influence the bleeding phenotype in severe hemophilic patients

Rendo, Pablo; Shafer, Frank; Korth‐Bradley, Joan M.; Sivamurthy, Krupa M.; Korín, Jorge

doi:10.1097/mbc.0b013e3283614210

Cited by 20 publications

(16 citation statements)

References 59 publications

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“…4,16,17 It is possible that some of these other factors played a role in the dogs reported here and accounted for the apparent lack of a relationship between FVIII:C and bleeding characteristics. In doing so, it was difficult to include detailed questions regarding the frequency of bleeding events and the treatment required for each event.…”

Section: Discussionmentioning

confidence: 90%

Clinical outcome after diagnosis of hemophilia A in dogs

Aslanian¹,

Sharp²,

Rozanski³

et al. 2014

javma

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Section: Discussionmentioning

confidence: 90%

Clinical outcome after diagnosis of hemophilia A in dogs

Aslanian¹,

Sharp²,

Rozanski³

et al. 2014

javma

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“…Although FVIII levels and PK parameters are associated with bleeding risk , many other factors are also involved . Phenotype‐guided dosing is based on the overall bleeding pattern in each individual patient.…”

Section: Discussionmentioning

confidence: 99%

Safety and efficacy of BAY 94‐9027, a prolonged‐half‐life factor VIII

Reding

Poulsen

et al. 2017

Journal of Thrombosis and Haemostasis

104

177

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To cite this article: Reding MT, Ng HJ, Poulsen LH, Eyster ME, Pabinger I, Shin H-J, Walsch R, Lederman M, Wang M, Hardtke M, Michaels LA. Safety and efficacy of BAY 94-9027, a prolonged-half-life factor VIII. J Thromb Haemost 2017; 15: 411-9. Essentials• Recombinant factor VIII BAY 94-9027 conjugates in a site-specific manner with polyethylene glycol.• BAY 94-9027 was given to patients with severe hemophilia A as prophylaxis and to treat bleeds.• BAY 94-9027 prevented bleeds at dose intervals up to every 7 days and effectively treated bleeds.• BAY 94-9027 treatment was mainly well tolerated and no patient developed factor VIII inhibitors. tional weeks until randomization arms were filled. Patients who were eligible but not randomized continued twiceweekly prophylaxis. The primary efficacy outcome was annualized bleeding rate (ABR). Results: The intent-totreat population included 132 patients (prophylaxis, n = 112; on demand, n = 20). Median ABR (quartile [Q1; Q3]) for patients treated two times per week who were not eligible for randomization (n = 13) improved after dose increase (17.4 [14.3; 26.0] to 4.1 [2.0; 10.6]). Median ABR for patients randomized to every-5-days treatment (n = 43) was 1.9 (0; 4.2), similar to patients eligible for randomization but who continued treatment two times per week (n = 11). Median ABR for 32/43 patients (74%) who continued every-7-days prophylaxis until study end was 0.96 (0.0; 4.3). Six hundred and thirty-six of 702 bleeds (90.6%) were controlled with ≤ 2 infusions. No patient developed a FVIII inhibitor. Conclusions: BAY 94-9027 prevented bleeding across three individually tailored dose regimens and was effective for treatment of bleeds.

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“…An individual's bleeding phenotype depends on his factor level, quality of the vascular structures and function of the platelets . All three components are genetically determined, but no study of haemophilia phenotype has comprised all.…”

Section: Discussionmentioning

confidence: 99%

Similar bleeding phenotype in young children with haemophilia A or B: a cohort study

et al. 2014

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The bleeding phenotype has been suggested to differ between haemophilia A and B. More knowledge on the bleeding phenotype at initiation of treatment is important to optimize patient care. The aim of this study was to investigate the severity of the bleeding phenotype and the variation in bleeding in children with severe or moderate haemophilia A and B. Consecutive, previously untreated patients with severe or moderate haemophilia A and B (factor VIII or IX activity <0.01 or 0.01-0.05 IU mL(-1) respectively) born between January 1st 2000 and January 1st 2010 were included. Primary outcome was severity of bleeding tendency. Secondary outcome was variation in bleeding pattern. A total of 582 patients with severe haemophilia A and 76 with severe haemophilia B did not differ in age at first exposure to clotting factor (0.81 vs. 0.88 years, P = 0.20), age at first bleed (0.82 vs. 0.88 years, P = 0.36), and age at first joint bleed (1.18 vs. 1.20 years, P = 0.59). Patients with moderate haemophilia were older compared to patients with severe haemophilia. In patients with moderate haemophilia there were no clear differences between haemophilia A and B. Severity and variation in bleeding phenotype are similar during the early stage of treatment in patients with severe and moderate haemophilia A and B respectively. The findings imply that children with haemophilia B should be observed and treated as vigilantly as those with haemophilia A.

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Factors that influence the bleeding phenotype in severe hemophilic patients

Cited by 20 publications

References 59 publications

Clinical outcome after diagnosis of hemophilia A in dogs

Clinical outcome after diagnosis of hemophilia A in dogs

Safety and efficacy of BAY 94‐9027, a prolonged‐half‐life factor VIII

Similar bleeding phenotype in young children with haemophilia A or B: a cohort study

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