Hemophilia: The Past, the Present, and the Future

Matuk-Villazon, Omar; Roberts, Jonathan C.; Corrales-Medina, Fernando F.

doi:10.1542/pir.2020-004143

Cited by 8 publications

(11 citation statements)

References 74 publications

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“…Bleeding events may occur spontaneously, or during minor procedures or major surgery. [1][2][3][4] Approximately 70% of people with haemophilia (PWH) in the US receive integrated care at federally supported haemophilia Treatment Centers (HTCs), which provide specialty clinical services from a multidisciplinary team. 5 HTC clinicians recommend prophylaxis to prevent bleeding, and rapid treatment of bleeding events to significantly reduce morbidity and mortality.…”

Section: Introductionmentioning

confidence: 99%

“…Haemophilia is a rare genetic bleeding disorder with serious and potentially fatal complications, as well as high economic costs for appropriate clinical management. Bleeding events may occur spontaneously, or during minor procedures or major surgery 1–4 …”

Section: Introductionmentioning

confidence: 99%

“…these surveys was also the development of breakthrough innovation in haemophilia therapies including EHLs and emicizumab. The development of EHLs lead many clinicians to consider factor 'peak' , 'trough' , and 'area under the curve' pharmacokinetic (PK) parameters; hence targeting high factor levels for improved bleed protection based upon a patient's individual PK 4,20. As the U.S. bleeding disorders patient and clinicians move towards the goal of a 'life without bleeds' , it is likely that this changing expectation ushered in increased dosing prescription practices as highlighted in this study.Of all prescribing characteristics, prescribing prophylaxis 'All of the time' showed the most consistent rise over the 22-year period.…”

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confidence: 99%

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Trends in prescribing practices for management of haemophilia: 1999–2021

Curtis¹,

Roberts

Crook

et al. 2023

Haemophilia

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Introduction:People with haemophilia rely on specialists for their care, yet the specific dosing regimens of treatments prescribed by these specialists have not been widely studied. Aim:The objective of this study is to describe trends in clinician prescribing practices for the management of haemophilia in the United States (US). Methods:We administered surveys to members of the Hemostasis and Thrombosis Research Society via paper surveys at its in-person annual symposia in 1999 and 2015, and an online survey in 2021. The surveys collected information on haemophilia treatments including factor dosing, inhibitor therapy and gene therapy.Results: Clinicians treating haemophilia for more than 50% of their practice time have increased from 37.5% of respondents in 1999 to 46.3% in 2021. Clinicians prescribing factor concentrates at >40 units/kg for routine bleeding events increased from 0% in 1999 to 29.3% in 2021 in haemophilia A (HA) and from 22.5% to 87.8% in haemophilia B (HB). In 2021, the clinicians reported prescribing emicizumab to treat HA patients (>89.5% paediatric, >85.7% adult) with or without inhibitors at least some of the time.Approximately 78.0% of respondents reported that they expected to recommend gene therapy at least some of time. Conclusion:These data indicate changing trends in prescribing practices among US haemophilia specialists during the past 22 years. Preference for high doses of factor (>40 units/kg) has increased during this period. Emicizumab prophylaxis has been prescribed for patients with and without HA inhibitors. Clinicians expect gene therapy to have value for some haemophilia patients.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

mentioning

confidence: 99%

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Trends in prescribing practices for management of haemophilia: 1999–2021

Curtis¹,

Roberts

Crook

et al. 2023

Haemophilia

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“…The incidence of congenital heart disease (CHD) is approximately 8 in 1000 births while the incidence of hemophilia A is around 1 in 5000 male birth and hemophilia B is around 1 in 30,000 male births. 1,2 An association of CHD and hereditary bleeding disorders like hemophilia is uncommon, the incidence of CHD in hemophilic patients is almost the same as the incidence of CHD in general population. 3 Although few pediatric cases have been reported with this unusual combination have successfully undergone heart surgery.…”

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Heart surgery in a pediatric patient with congenital heart disease and hemophilia B: A case report from Sudan

Mohmmedahmed,

Eltahir,

Mohammedali

et al. 2023

Clinical Case Reports

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Key Clinical MessageA multidisciplinary team approach, careful hemostasis, and factor replacement therapy are important in the perioperative management of hemophiliac patients undergoing pediatric cardiac surgery.AbstractThe combination of congenital heart diseases (CHD) and hemophilia is rare; furthermore, heart surgery and perioperative management of such cases is challenging. This report illustrates the challenges of pediatric cardiac surgery in an infant with both hemophilia B and CHD. Multidisciplinary team approach, careful hemostasis, and factor replacement therapy were key to success without hemorrhagic complications before, during and after surgery.

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