Emergencies in Hemophilia

Woods, Gary M.; Dunn, Max S.; Dunn, Amy L.

doi:10.1016/j.cpem.2018.05.004

Cited by 5 publications

(5 citation statements)

References 89 publications

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“…The PEGylated glyco-protein drug, also known as Rebinyn R (Novo Nordisk, Bagsvaerd, Denmark), received its FDA approval in 2017 [283]. It is used for patients suffering from factor IX (FIX) deficiency also known as hemophilia [284]. It consists of 40 kDa PEG attached to recombinant DNA-derived coagulation FIX concentrate [285,286].…”

Section: Rebinyn Rmentioning

confidence: 99%

Nanopharmaceuticals and Nanomedicines Currently on the Market: Challenges and Opportunities

Farjadian

Ghasemi

Gohari

et al. 2018

Nanomedicine (Lond.)

407

213

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There has been a revolution in nanotechnology and nanomedicine. Since 1980, there has been a remarkable increase in approved nano-based pharmaceutical products. These novel nano-based systems can either be therapeutic agents themselves, or else act as vehicles to carry different active pharmaceutical agents into specific parts of the body. Currently marketed nanostructures include nanocrystals, liposomes and lipid nanoparticles, PEGylated polymeric nanodrugs, other polymers, protein-based nanoparticles and metal-based nanoparticles. A range of issues must be addressed in the development of these nanostructures. Ethics, market size, possibility of market failure, costs and commercial development, are some topics which are on the table to be discussed. After passing all the ethical and biological assessments, and satisfying the investors as to future profitability, only a handful of these nanoformulations, successfully obtained marketing approval. We survey the range of nanomedicines that have received regulatory approval and are marketed. We discuss ethics, costs, commercial development and possible market failure. We estimate the global nanomedicine market size and future growth. Our goal is to summarize the different approved nanoformulations on the market, and briefly cover the challenges and future outlook.

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Section: Rebinyn Rmentioning

confidence: 99%

Nanopharmaceuticals and Nanomedicines Currently on the Market: Challenges and Opportunities

Farjadian

Ghasemi

Gohari

et al. 2018

Nanomedicine (Lond.)

407

213

View full text Add to dashboard Cite

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“…Similarly, another study (17) indicated a highly significant leukocytosis in hemophilic patients with arthropathy. In the same vein, it was found that hemophilic arthropathy could be considered as the most critical complication of hemophilia, especially in its severe form, so as the WBCs count elevated due to the inflammation of the joint (4) . In addition, hemophilic patients are prone to frequent infections due to their frequent hospitalization as well as the transfusion of plasma and cryoprecipitate.…”

Section: Discussionmentioning

confidence: 99%

“…Factor VIII deficit (Hemophilia A) accounts for 85% of instances, while Factor IX deficiency (Hemophilia B) accounts for 15% of cases (4,5) . Of note, in case a child inherits a factor deficiency from only one parent, the child will be a carrier of the disease, even if he or she does not show symptoms (3,6) .…”

Section: Introductionmentioning

confidence: 99%

Effect of Inherited Bleeding Disorder on Some Physiological Parameters for Patients In Karbala City of Iraq

2022

The Egyptian Journal of Hospital Medicine

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Background: Severe inherited bleeding disorders are most common during early childhood. Hereditary bleeding disorder refers to a group of medical conditions that cause poor blood clotting and uncontrollable bleeding.Objective: This study aimed to report the hematological changes that may occur in patients diseased with hemophilia to help the medical team know how the body responds to treatment, and consequently decrease complications and deaths. Methods: The current study was conducted in Maternity and Pediatrics Hospital, Karbala, Iraq. The study included 100 samples of female patients infected with hemophilia and 100 samples of healthy female children within the age range of 1-18 years. Results: The current study indicated total red blood cells count, hemoglobin, and packed cell volume that were significantly decreased in patients with hemophilia (p ≤ 0.05) compared to healthy children. In contrast, total white blood cell count and erythrocyte sedimentation rate were increased significantly in patients with hemophilia compared to healthy children (p ≤ 0.05). Conlcusion: Changes in hematological parameters for patients infected with hemophilia occur as a result of frequent bleeding, which is the primary sign of hemophilia.

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“…SSEH is a rare and severe complication in patients with hemophilia with high morbidity and mortality rates and neurological sequelae [4]. The most common affected level is the cervicothoracic spine [6], with the subdural and cerebellar spaces being most affected frequently [7]. The clinical symptomatology and ensuing complication derive from a mass effect of the hematoma on the spinal cord.…”

Section: Discussionmentioning

confidence: 99%

“…To this day, the management, treatment, and prevention of life-threatening bleeding in patients with hemophilia continue to be challenging [11]. Management should include rapid factor administration, even before a definitive confirmatory diagnosis with MRI is established [7]. Different doses of rFVIIa have been used depending on the severity and bleeding site, ranging from 90 to 270 mg/ kg.…”

Section: Discussionmentioning

confidence: 99%

Spinal epidural hematoma in a child with hemophilia A with high titer inhibitors and follow-up with prophylactic emicizumab: case report and literature review

Villarreal‐Martínez

Sepúlveda-Orozco

García-Viera³

et al. 2021

Blood Coagulation &Amp; Fibrinolysis

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Hemorrhage in the central nervous system is the most severe and debilitating manifestation affecting patients with hemophilia A. The spinal epidural space is the most unusual and clinically challenging site of central nervous system hemorrhage in hemophilia A. These patients often show insidious neurological signs and symptoms that delay diagnosis and treatment. We share our experience treating a 4-year-old male patient with severe hemophilia A and high titer inhibitors with a spontaneous spinal epidural hematoma. The patient presented initially with intense headache and neck pain. After blood tests and imaging studies, bypassing agent therapy with recombinant-activated factor VII was used until discharge; this was later replaced with emicizumab. After 18 months, the patient is without neurological sequelae and has not experienced subsequent bleeding episodes. We review the available literature and discuss the relevance of emicizumab compared with standard therapies in the context of spontaneous spinal epidural hematoma.

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Emergencies in Hemophilia

Cited by 5 publications

References 89 publications

Nanopharmaceuticals and Nanomedicines Currently on the Market: Challenges and Opportunities

Nanopharmaceuticals and Nanomedicines Currently on the Market: Challenges and Opportunities

Effect of Inherited Bleeding Disorder on Some Physiological Parameters for Patients In Karbala City of Iraq

Spinal epidural hematoma in a child with hemophilia A with high titer inhibitors and follow-up with prophylactic emicizumab: case report and literature review

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