Intracranial haemorrhage in children with inherited bleeding disorders in the <scp>UK</scp> 2003‐2015: A national cohort study

Chalmers, Elizabeth; Alamelu, Jayanthi; Collins, Peter William; Mathias, Mary; Payne, Jeanette; Richards, Michael; Tunstall, Oliver; Williams, M.; Palmer, Ben; Mumford, Andrew D

doi:10.1111/hae.13461

Cited by 45 publications

(39 citation statements)

References 32 publications

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“…Patients with haemophilia suffer from recurrent bleeding episodes, mostly occurring in the joints, resulting in a permanent and crippling arthropathy. In addition, life‐threatening internal bleeding, including central nervous system bleeding, can occur and tends to occur most commonly in young children (Chalmers et al , ). While, historically, a fatal disease in the most severe forms, haemophilia currently carries an overall excellent prognosis in well‐resourced countries where access to effective therapies is not limited.…”

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confidence: 99%

How I treat children with haemophilia and inhibitors

Young

2019

Br J Haematol

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Haemophilia is a serious inherited bleeding disorder resulting from a deficiency of coagulation factor VIII (haemophilia A) or coagulation factor IX (haemophilia B). While effective therapies have been developed to replace the missing factor and restore normal coagulation, they can lead to the development of neutralizing anti-drug antibodies, termed inhibitors, which significantly worsen morbidity and quality of life. While advancements have been made to improve the management of patients, and particularly for this review, children with inhibitors, there remain significant unmet needs including inhibitor prevention, inhibitor eradication and, when those fail, more effective bleed prevention and a reduction in the treatment burden. This review will discuss current treatment options including the recently licensed bispecific antibody, emicizumab, and provide insights into how I treat children with inhibitors.

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confidence: 99%

How I treat children with haemophilia and inhibitors

Young

2019

Br J Haematol

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“…Intracranial hemorrhages in the pediatric HA population are often spontaneous 9 and more frequent in the first 2 years of life. 5,19 Management of ICH requires early recognition and immediate FVIII replacement.…”

Section: Neurosurgical Managementmentioning

confidence: 99%

“…31 Surgical intervention is performed in 33%-39% of cases. 9,32,39 In one retrospective study of 66 children with a primary coagulopathy and ICH, 22 underwent surgery, which consisted of craniotomy and evacuation in 16 cases and insertion of a VP shunt or drain in 6 cases. 9 However, these studies did not describe the management of FVIII levels for children who develop hydrocephalus and require surgery.…”

Section: Neurosurgical Managementmentioning

confidence: 99%

Management of hydrocephalus in infants with severe hemophilia A: report of 2 cases

Bergin

Dunn

Smith³

et al. 2019

Journal of Neurosurgery: Pediatrics

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The authors report on the clinical course of two infants with severe hemophilia A (HA) and concomitant progressive hydrocephalus that required management with a ventriculoperitoneal shunt. The first child, with known HA, presented with a spontaneous intracranial hemorrhage and acquired hydrocephalus. He underwent cerebrospinal fluid diversion with a temporary external ventricular drain, followed by placement of a ventriculoperitoneal shunt. The second child had hydrocephalus secondary to a Dandy-Walker malformation and was diagnosed with severe HA during preoperative evaluation. He underwent placement of a ventriculoperitoneal shunt after progression of the hydrocephalus. The authors also review the treatment of hydrocephalus in patients with HA and describe the perioperative protocols used in their two cases. Treatment of hydrocephalus in infants with HA requires unique perioperative management to avoid complications.

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“…There are ad hoc ED attendances and often multiple, emotionally traumatic attempts to administer CFC intravenously in a small child. There is an everpresent risk of intracranial hemorrhage in this first year, in the absence of prophylaxis [29]. The early treatment of these previously untreated patients (PUPs) requires a systematic surveillance for inhibitory antibody responses against the infused therapeutic CFC [30].…”

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confidence: 99%

Re-personalization and stratification of hemophilia care in an evolving treatment landscape

2019

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Hemophilia therapeutics are evolving rapidly. Comprehensive care must also evolve to embrace this change. Online tools and guidelines are widely available to optimize prophylaxis with conventional clotting factor concentrates using an individual's predicted pharmacokinetic profile. Novel hemostatic agents (e.g. biphenotypic antibody) are also becoming widely available, with other agents with differing mechanisms of action in final stages of trial. Contemporary issues including challenges of prophylaxis; bleed treatment; laboratory monitoring and inhibitor risk/surveillance are summarized in this narrative review, focusing on how a re-personalization of education and treatment will be necessary to meet these challenges of the rapidly changing therapeutic landscape.

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Intracranial haemorrhage in children with inherited bleeding disorders in the UK 2003‐2015: A national cohort study

Abstract: Intracranial haemorrhage occurs most frequently in children with severe IBDs, during the first 2 years of life and in children not receiving prophylaxis. Intracranial haemorrhage often occurs without documented trauma.

Cited by 45 publications

References 32 publications

How I treat children with haemophilia and inhibitors

How I treat children with haemophilia and inhibitors

Management of hydrocephalus in infants with severe hemophilia A: report of 2 cases

Re-personalization and stratification of hemophilia care in an evolving treatment landscape

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