Recurrent intracranial bleed in a child receiving prophylaxis with emicizumab

Teo, Hong; Wong, Wan Hui; Lam, Joyce Ching Mei

doi:10.1111/hae.14274

Cited by 3 publications

(4 citation statements)

References 10 publications

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“…The real‐world experience with emicizumab efficacy is still limited to date, with all published efficacy data showing similar results to those reported in the clinical trials 15–17,27–29 . However, treatment failure in the absence of anti‐emicizumab antibodies has now been reported in a child with recurrent intracranial bleeds 30 . This was not seen during the clinical trial conduct.…”

Section: Emicizumab Rolloutmentioning

confidence: 74%

“…[15][16][17][27][28][29] However, treatment failure in the absence of anti-emicizumab antibodies has now been reported in a child with recurrent intracranial bleeds. 30 This was not seen during the clinical trial conduct. In addition, recent real-world experience from the Israeli group indicates that long-term, the efficacy of emicizumab tends to reduce in adults with more breakthrough bleeds and need to go back to replacement in some cases.…”

Section: Emicizumab Rolloutmentioning

confidence: 99%

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Emicizumab state‐of‐the‐art update

2022

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Introduction:Emicizumab is a bispecific monoclonal antibody developed to address the unmet needs of clotting factor replacement therapy and has become the benchmark for optimal prophylaxis in managing patients with haemophilia A with inhibitors.We describe the emicizumab rollout and pharmacokinetic strategies and their use in paediatric patients. Methods:The evolving real-world experience in using emicizumab has confirmed its safety, efficacy and pharmacokinetic profile in paediatric, adolescent and adult patients receiving emicizumab at various prophylactic dosing regimens. The emicizumab current global rollout includes over 100 countries with 29 low to middle-income countries accessing emicizumab through the World Federation of Haemophilia (WFH) Humanitarian Aid Program. The diversity of emicizumab dosing and pharmacokinetic tools such as the Calibra® and the WAPPS-Hemo platforms make it possible to achieve prophylaxis goals in line with the WFH Haemophilia treatment guidelines recommendations, with minimal drug wastage. The emerging experience from long term clinical trials and long-term real-world follow-up confirm the safety, efficacy, and pharmacokinetic profile of emicizumab in paediatric haemophilia A patients. A few questions, including inhibitor recurrence, concurrent use of emicizumab with various replacement therapies and inhibitor eradication, are being addressed through multiple ongoing clinical studies. Conclusion:The current global rollout of emicizumab is remarkable, and versatile dosing regimens and evolving pharmacokinetic tools such as the Calibra® and WAPPS-Hemo platforms make it a treatment choice available also for pharmacokinetic guided personalised treatment. Data from paediatric studies are consistent with those seen in adolescent and adult Haemophilia A.

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Section: Emicizumab Rolloutmentioning

confidence: 74%

Section: Emicizumab Rolloutmentioning

confidence: 99%

Emicizumab state‐of‐the‐art update

2022

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“…However, some articles report the effect of emicizumab combined with FVIII replacement therapy. Two case reports showed that TG could be used to monitor treatment of HA patients with low‐titer inhibitors when emicizumab was used 58,59 . In a spiking study, HA plasma with emicizumab at a concentration of 50 ng/ml (the steady‐state concentration observed in the HAVEN‐1 study) was combined with a pdFVIII/von Willebrand factor (VWF) concentrate.…”

Section: Emicizumabmentioning

confidence: 99%

“…Two case reports showed that TG could be used to monitor treatment of HA patients with low‐titer inhibitors when emicizumab was used. 58 , 59 In a spiking study, HA plasma with emicizumab at a concentration of 50 ng/ml (the steady‐state concentration observed in the HAVEN‐1 study) was combined with a pdFVIII/von Willebrand factor (VWF) concentrate. As expected from previous studies, samples with emicizumab significantly showed improved TG profiles compared with FVIII‐deficient plasma.…”

Section: Emicizumabmentioning

confidence: 99%

Thrombin generation for monitoring hemostatic therapy in hemophilia A: A narrative review

Verhagen

Valke

Schols

2022

Journal of Thrombosis and Haemostasis

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Hemophilia A (HA) is characterized by an abnormal low coagulation factor VIII (FVIII) activity level. HA patients with severe (FVIII activity level <1%) disease have an increased risk of trauma-related and spontaneous bleeding, especially in joints and muscles. Prophylactic replacement therapy with FVIII concentrate is recommended for these patients to prevent bleeding and to preserve musculoskeletal health. 1 The prophylactic scheme is now based on patient body weight and individual pharmacokinetic (PK) studies. Remarkably, there is a large inter-individual variability in PK dosing based on this algorithm. 2 Interestingly, however, some patients still experience breakthrough bleeds despite adequate FVIII trough levels, whereas others do not bleed with trough levels below threshold. 3 In addition, large heterogeneity in bleeding phenotype between patients with the same baseline FVIII activity level is observed. [3][4][5]

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