&lt;p&gt;Clinical Evidence and Safety Profile of Emicizumab for the Management of Children with Hemophilia A&lt;/p&gt;

Quellec, Sandra Le

doi:10.2147/dddt.s167731

Cited by 13 publications

(15 citation statements)

References 77 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…35,36 Notably, all four patients were children receiving Q4W prophylaxis. 37 Two of the reported children (6 and 13 years old with high-titer inhibitors, respectively) had a discontinued emicizumab treatment due to lack of efficacy. Both children showed a significantly elevated bleeding tendency as represented by a prolonged aPTT after approximately 2 to 3 months of emicizumab therapy.…”

Section: Safetymentioning

confidence: 99%

“…In addition, emicizumab should be started in comprehensive care centers with sufficient clinical and laboratory experience. 1,37,38 The treatment of newborn or infants should be a case-by-case decision. 1,48 Particularly in the context of preventing ICH, Guy Young strongly recommends to discuss the option of an early emicizumab prophylaxis with parents of all newly diagnosed severe HA patients.…”

Section: Surgerymentioning

confidence: 99%

“…What about the functions of FVIII outside of coagulation, for instance, with regard to bone health? 37,48…”

Section: Older Patients/previously Treated Patientsmentioning

confidence: 99%

See 2 more Smart Citations

Emicizumab for All Pediatric Patients with Severe Hemophilia A

Wieland

2022

Hamostaseologie

View full text Add to dashboard Cite

Emicizumab is the first approved nonreplacement therapy for bleeding prophylaxis in hemophilia A (HA) patients. In 2018, it was licensed for HA patients with inhibitors, subsequently followed by an “European Medicines Agency (EMA)” approval for patients with severe HA in the absence of inhibitors in 2019. This is immediately raising the question whether emicizumab is suitable as a first-line treatment for all pediatric patients with severe HA. In this review, we want to discuss what we have, what we know, and what we would like to know. Severe HA is characterized by severe spontaneous and traumatic bleedings, particularly into muscles and joints leading to chronic joint damage. Standard of care is the regular, prophylactic replacement of factor VIII to prevent bleedings. Due to approval of emicizumab—the first nonreplacement therapy for bleeding prophylaxis—in HA patients with inhibitors, and severe HA patients without inhibitors, it is of pivotal interest whether emicizumab could be the first-line treatment in all pediatric patients with severe HA. Clinical trials and real-world observational studies could demonstrate a good efficacy and safety for bleeding prevention during emicizumab treatment in HA patients with and without inhibitors. This clearly indicates that emicizumab could improve HA treatment. However, some crucial and critical questions are remaining with regard to the use of emicizumab. Some of this missing information is already under investigation in the context of clinical trials. Until getting finalized data to shed insights into the points that are currently being discussed, there is a variety of expert and expert group recommendations, which are tackling questions concerning the treatment of HA patients. This review will address major information that is already available, but will also focus on important points that remain to be elucidated in the context of HA treatment.

show abstract

Section: Safetymentioning

confidence: 99%

Section: Surgerymentioning

confidence: 99%

See 1 more Smart Citation

Emicizumab for All Pediatric Patients with Severe Hemophilia A

Wieland

2022

Hamostaseologie

View full text Add to dashboard Cite

show abstract

“…[6][7][8] It bridges activated factor IX and X to restore missing activated factor VIII. 9 Patients treated with emicizumab (Hemlibra, Roche, Bazel, Switzerland) bleed less than patients treated with bypassing agent (BPA), 10,11 but acute bleeding requires BPA. 12 Surgeries for patients treated with emicizumab require BPA for bleeding risk, but the appropriate dose of BPA is unknown.…”

Section: Introductionmentioning

confidence: 99%

Surgery with emicizumab prophylaxis for two paediatric patients with severe haemophilia A with inhibitors

Lockhart

Tardy

Montmartin

et al. 2021

Pediatric Blood & Cancer

View full text Add to dashboard Cite

Emicizumab is a prophylaxis for patients with severe haemophilia A with and without inhibitor. Despite weekly administration of emicizumab, coagulation states stay below normal value and cannot be assessed by standard haemostasis tests. In our two patients, we used the thrombin-generation assay (endogenous thrombin potential and Peak) to monitor the patient's clotting status. Under emicizumab, it is necessary to add a bypassing agent (BPA) such as rFVIIa (Novoseven) to avoid bleeding before surgery. The BPA dosage was based on a thrombin-generation assay and collegial consultation.

show abstract

“…It bridges activated factor IX and X to restore missing activated factor VIII [8]. Patients under emicizumab (Hemlibra® , Roche, Bazel, Switzerland) bleed less than patients under bypassing agent (BPA) [9] , [10]. They still have acute bleeding needed BPA, mainly FVIIa (Novoseven® ,rVIIa, Novonordisk, Bagsvaerd, Denmark) [11].…”

Section: Main Text Introductionmentioning

confidence: 99%