Real‐world case series and summary of current literature of infants and toddlers with severe hemophilia A with inhibitor on prophylaxis with emicizumab

Garcia, Jéssica; Zia, Ayesha

doi:10.1002/pbc.28942

Cited by 13 publications

(14 citation statements)

References 5 publications

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“…All studies confirmed safety and efficacy of Emicizumab [3,[5][6][7]. Only few other studies have since investigated pediatric patients under prophylaxis with Emicizumab [8][9][10][11][12], especially data on PUPs and MTPs [13,14] are limited.…”

Section: Introductionmentioning

confidence: 73%

“…Data on efficacy and safety of Emicizumab in children, especially in young children < 3 years and PUPs is still scarce. Between 2019-2021 several publications have reported the use of Emicizumab in children, including about 40 children under the age of 3 years [8,9,[11][12][13]. All studies showed similar results regarding reduction of ABR, safety and laboratory results as adult cohorts.…”

Section: Discussionmentioning

confidence: 95%

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Emicizumab in children: bleeding episodes and outcome before and after transition to Emicizumab

et al. 2022

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Purpose Real-world data and study data regarding therapy with Emicizumab in pediatric cohorts with haemophilia A is scarce. Especially, data on previously untreated pediatric patients (PUPs) and minimally treated patients (MTPs) are missing. Methods Thirteen pediatric patients with haemophilia A and treatment with Emicizumab were retrospectively evaluated for Annual Bleeding Rates (ABR) pre-and post-Emicizumab treatment. Safety data and data on management of minor surgery as well as laboratory results were collected. Additionally, we describe the clinical features of two PUPs and one MTP that are included in our cohort. Results Median age at initiation of Emicizumab was 5.3 (range: 0.26–17.5) years, three patients were younger than one year at initiation of treatment with Emicizumab. Median follow-up time on Emicizumab was 23.8 (range: 0.7–40) months. Total ABR (p = 0.009) as well as spontaneous (p = 0.018), traumatic (p = 0.018), and joint (p = 0.027) ABR reduced significantly post-Emicizumab transition. Safety profile was favourable as only one local site reaction occurred; no cessation of treatment was necessary. Surgery was successfully performed in three patients receiving rFVlla pre- and post-surgery. Emicizumab trough levels showed a median of 43.2 μg/ml (range: 23.9–56.8) after three doses of 3 mg/kg and 51.9 μg/ml (range: 30.4–75) at first follow-up with 1.5 mg/kg. Conclusion Emicizumab is safe and efficient in pediatric patients with and without inhibitors. More data on larger multicenter cohorts and especially on PUPs/MTPs are still needed.

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Section: Introductionmentioning

confidence: 73%

Section: Discussionmentioning

confidence: 95%

Emicizumab in children: bleeding episodes and outcome before and after transition to Emicizumab

et al. 2022

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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][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.…”

Section: Emicizumab Rolloutmentioning

confidence: 99%

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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“…There is little information on the effectiveness of emicizumab administered to children, but factors that can contribute to the decrease in the efficacy of the treatment are knownt: immunologic naivety, lower production of vitamin K dependent proteins, and higher clearance of emicizumab 28 . But emicizumab administered prophylactically for 18 months protected a child with severe hemophilia A and high titer inhibitors from bleeding, after treating him with recombinant-activated factor VII for a spontaneously occurring spinal epidural hematoma 29 .…”

Section: Treatment With Emicizumabmentioning

confidence: 99%

From a bispecific monoclonal antibody to gene therapy: A new era in the treatment of hemophilia A

Mihăilă¹

2023

Biomed Pap Med Fac Univ Palacky Olomouc Czech Repub

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The treatment of hemophilia A has progressed amazingly in recent years. Emicizumab, a bispecific-humanized monoclonal antibody, is able to improve coagulation by bridging activated factor IX and factor X. Emicizumab is administered subcutaneously and much less often compared to factor VIII products. It has low immunogenicity, does not require dose adjustment, and can be administered regardless of the presence of factor VIII inhibitors. Thrombin generation assays but not factor VIII activity are indicated to guide and monitor the treatment. Emicizumab has enabled the conversion of patients with severe forms into patients with milder forms of hemophilia A. It has reduced the number of bleeding episodes compared to both on-demand and prophylactic substitution therapy and has an excellent safety profile. Gene therapy can elevate factor VIII plasma levels for many years after a single treatment course, could offer longterm protection from bleeding episodes, and minimize or eliminate the need for substitutive treatment with factor VIII concentrates. Gene therapy can provoke an immune response, manifested by an increase in common liver enzymes, that require immunotherapy. Long term monitoring is necessary to identify possible adverse effects. Future objectives are: the development of an ideal viral vector, the possibility of its re-administration, the use of gene therapy in hemophiliac children, and determining whether it can be successfully used to induce immune tolerance to factor VIII ceteri paribus. The future will determine the place of each type of treatment and group of patients for which it is indicated.

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Real‐world case series and summary of current literature of infants and toddlers with severe hemophilia A with inhibitor on prophylaxis with emicizumab

Cited by 13 publications

References 5 publications

Emicizumab in children: bleeding episodes and outcome before and after transition to Emicizumab

Emicizumab in children: bleeding episodes and outcome before and after transition to Emicizumab

Emicizumab state‐of‐the‐art update

From a bispecific monoclonal antibody to gene therapy: A new era in the treatment of hemophilia A

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