[HTML][HTML] Hemophilic arthropathy: current knowledge and future perspectives

R Gualtierotti, LP Solimeno, F Peyvandi - Journal of Thrombosis and …, 2021 - Elsevier
Hemophilia A and B are rare X‐linked inherited bleeding disorders caused by complete or
partial deficiency in or the absence of coagulation factors VIII and IX. Recurrent joint …

Scales to assess the quality of randomized controlled trials: a systematic review

SA Olivo, LG Macedo, IC Gadotti, J Fuentes… - Physical …, 2008 - academic.oup.com
Background and Purpose The methodological quality of randomized controlled trials (RCTs)
is commonly evaluated in order to assess the risk of biased estimates of treatment effects …

Gene therapy with Fidanacogene Elaparvovec in adults with hemophilia B

A Cuker, K Kavakli, L Frenzel, JD Wang… - … England Journal of …, 2024 - Mass Medical Soc
Background Fidanacogene elaparvovec, an adeno-associated virus (AAV) gene-therapy
vector for hemophilia B containing a high-activity human factor IX variant (FIX-R338L/FIX …

[HTML][HTML] Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia

MJ Manco-Johnson, TC Abshire… - … England Journal of …, 2007 - Mass Medical Soc
Background Effective ways to prevent arthropathy in severe hemophilia are unknown.
Methods We randomly assigned young boys with severe hemophilia A to regular infusions …

[HTML][HTML] A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study)

A Gringeri, B Lundin, S Von Mackensen… - Journal of Thrombosis …, 2011 - Elsevier
Background: Prevention of arthropathy is a major goal of hemophilia treatment. While
studies in adults have demonstrated an impact of prophylaxis on the incidence of joint …

[HTML][HTML] A randomized comparison of two prophylaxis regimens and a paired comparison of on‐demand and prophylaxis treatments in hemophilia A management

LA Valentino, V Mamonov, A Hellmann… - Journal of thrombosis …, 2012 - Elsevier
Background: Prophylaxis with factor (F) VIII is considered the optimal treatment for managing
hemophilia A patients without inhibitors. Objectives: To compare the efficacy of two …

Intermediate-dose versus high-dose prophylaxis for severe hemophilia: comparing outcome and costs since the 1970s

K Fischer, K Steen Carlsson, P Petrini… - Blood, The Journal …, 2013 - ashpublications.org
Prophylactic treatment in severe hemophilia is very effective but is limited by cost issues.
The implementation of 2 different prophylactic regimens in The Netherlands and Sweden …

[图书][B] Proteins: biochemistry and biotechnology

G Walsh - 2014 - books.google.com
Proteins Biochemistry and Biotechnology 2e is a definitive source of information for all those
interested in protein science, and particularly the commercial production and isolation of …

Treatment adherence in hemophilia

CD Thornburg, NA Duncan - Patient preference and adherence, 2017 - Taylor & Francis
Prophylactic clotting-factor regimens reduce the occurrence of bleeding episodes and
maintain joint health in individuals with moderate and severe hemophilia. However, these …

Hemophilia A in the third millennium

M Franchini, PM Mannucci - Blood reviews, 2013 - Elsevier
Hemophilia A is an X-linked hereditary bleeding disorder due to the deficiency of
coagulation factor VIII (FVIII). According to the degree of FVIII deficiency, mild, moderate or …