Author: Mesquita Camelo, Ricardo ; Gonçalves de Oliveira, Andrea ; Lemos Jardim, Letícia ; Dias, Maíse Moreira ; Rezende, Suely Meireles ; Callado, Fábia Michelle Rodrigues de Araújo ; Karla Brognoli Franco, Vivian ; Ribeiro, Rosângela de Albuquerque ; Lorenzato, Cláudia Santos ; de Magalhães, Laura Peixoto

01 Nov 2023·Current Neurology and Neuroscience Reports

Neurological Complications Associated with Hereditary Bleeding Disorders

Review

Author: Bhatti, Muhammad Qasim ; Di Napoli, Mario ; Divani, Afshin A ; Bhatia, Kunal ; Hinduja, Archana ; Gonzalez-Fernandez, Ezekiel ; Datta, Yvonne H

PURPOSE OF REVIEWHereditary bleeding disorders may have a wide variety of clinical presentations ranging from mild mucosal and joint bleeding to severe central nervous system (CNS) bleeding, of which intracranial hemorrhage (ICH) is the most dreaded complication. In this review, we will discuss the pathophysiology of specific hereditary bleeding disorders, namely, hemophilia A, hemophilia B, and von Willebrand disease (vWD); their clinical manifestations with a particular emphasis on neurological complications; a brief overview of management strategies pertaining to neurological complications; and a review of literature guiding treatment strategies.RECENT FINDINGSICH is the most significant cause of morbidity and mortality in patients with hemophilia. Adequate control of bleeding with the administration of specific factors or blood products, identification of risk factors for bleeding, and maintaining optimal coagulant activity are essential for appropriately managing CNS bleeding complications in these patients. The administration of specific recombinant factors is tailored to a patient's pharmacokinetics and steady-state levels. During acute bleeding episodes, initial factor activity should be maintained between 80 and 100%. Availability of monoclonal antibody Emicizumab has revolutionized prophylactic therapies in patients with hemophilia. Management of ICH in patients with vWD involves using plasma-derived factor concentrates, recombinant von Willebrand factor, and supportive antifibrinolytic agents individualized to the type and severity of vWD. Hemophilia and vWD are the most common hereditary bleeding disorders that can predispose patients to life-threatening CNS complications-intracranial bleeds, intraspinal bleeding, and peripheral nerve syndromes. Early care coordination with a hematologist can help develop an effective prophylactic regimen to avoid life-threatening bleeding complications in these patients. Further research is needed to evaluate using emicizumab as an on-demand treatment option for acute bleeding episodes in patients with hemophilia.

01 Nov 2023·Research and Practice in Thrombosis and Haemostasis

Inhibitor development according to concentrate in severe hemophilia: reporting on 1392 Previously Untreated Patients from Europe and Canada

Article

Author: Peyvandi, Flora ; Kaczmarek, Radek ; Lassila, Riitta ; Fischer, Kathelijn ; Bettle, Amanda ; Hollingsworth, Rob ; Iorio, Alfonso ; Lambert, Thierry ; Makris, Mike ; Rivard, Georges-Étienne ; Gatt, Alexander ; Carcao, Manuel ; Samji, Nasrin

BackgroundClotting factor concentrates have been the mainstay of severe hemophilia treatment over the last 50 years. Differences in risk of neutralizing antibody (inhibitor) formation according to concentrate used remain clinically relevant.ObjectivesTo assess inhibitor development according to type of clotting factor concentrate in previously untreated patients (PUPs) with severe hemophilia A and B.MethodsThe European Haemophilia Safety Surveillance (EUHASS) and Canadian Bleeding Disorders Registry (CBDR) have been monitoring adverse events overall and according to concentrate for 11 and 8 years, respectively. Inhibitors were reported quarterly, and PUPs completed 50 exposure days without inhibitor development annually. Cumulative inhibitor incidences and 95% confidence intervals (CIs) were compared without adjustment for other risk factors.ResultsFifty-six European and 23 Canadian centers reported inhibitor development in 312 of 1219 (26%; CI, 23%-28%) PUPs with severe hemophilia A and 14 of 173 (8%; CI, 5%-13%) PUPs with severe hemophilia B. Inhibitor development was lower on plasma-derived factor (F)VIII (pdFVIII, 20%; CI, 14%-26%) than on standard half-life recombinant FVIII (SHL-rFVIII, 27%; CI, 24%-30% and odds ratio, 0.67; CI, 0.45%-0.98%; P = .04). Extended half-life recombinant FVIII (EHL-rFVIII, 22%; CI, 12%-36%) showed an intermediate inhibitor rate, while inhibitor rates for Advate (26%; CI, 22%-31%) and Kogenate/Helixate (30%; CI, 24%-36%) overlapped. For other SHL-rFVIII concentrates, inhibitor rates varied from 3% to 43%. Inhibitor development was similar for pdFIX (11%; CI, 3%-25%), SHL-rFIX (8%; CI, 3%-15%), and EHL-rFIX (7%; CI, 1%-22%).ConclusionWhile confirming expected rates of inhibitors in PUPs, inhibitor development was lower in pdFVIII than in SHL-rFVIII. Preliminary data suggest variation in inhibitor development among different SHL-rFVIII and EHL-rFVIII concentrates.

100 Deals associated with Plasma-derived factor VIII (Octapharma AG)

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Indication	Country/Location	Organization	Date
Hemophilia A	DK	Octapharma AG	-
Hemophilia A	NL	Octapharma AG	-
Hemophilia A	LV	Octapharma AG	-
Hemophilia A	CZ	Octapharma AG	-
Hemophilia A	SK	Octapharma AG	-

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ISTH2021	Not Applicable	Hemophilia A	283	Third-generation Recombinant Factor VIII	ziwxmfqjiw(iwennchmvo) = dauajtxouw wkmlnvbpqb (vsphuwaows ) View more	-	17 Jul 2021
				Plasma-derived Factor VIII	ziwxmfqjiw(iwennchmvo) = kifsctjvvu wkmlnvbpqb (vsphuwaows ) View more
NIS-Previq (ISTH2019)	Not Applicable	Hemophilia A	34	simoctocog alfa	aqqzljxqkl(hnpvywqgkt) = cozsiseohf fckxxcncyt (rvnvymzlxb ) View more	-	10 Jun 2019
				plasma-derived factor VIII concentrates	aqqzljxqkl(hnpvywqgkt) = ygopznknmm fckxxcncyt (rvnvymzlxb ) View more

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