Patterns of joint damage in severe haemophilia A treated with prophylaxis

The primary objective of this study was to assess whether there are different patterns (classes) of joint health in young boys with severe haemophilia A (SHA) prescribed primary tailored prophylaxis. We also assessed whether age at first index joint bleed, blood group, FVIII gene abnormality variant, factor VIII trough level, first‐year bleeding rate and adherence to the prescribed prophylaxis regimen significantly predicted joint damage trajectory, and thus class membership.

[1]  S. Israels,et al.  Hemophilia prophylaxis adherence and bleeding using a tailored, frequency‐escalated approach: The Canadian Hemophilia Primary Prophylaxis Study , 2020, Research and practice in thrombosis and haemostasis.

[2]  M. Recht,et al.  Early Prophylaxis Provides Continued Joint Protection in Severe Hemophilia A: Results of the Joint Outcome Continuation Study , 2018, Blood.

[3]  P. Babyn,et al.  Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort. , 2018, The Lancet. Haematology.

[4]  M. Morfini The History of Clotting Factor Concentrates Pharmacokinetics , 2017, Journal of clinical medicine.

[5]  J. Oldenburg Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens. , 2015, Blood.

[6]  Benoit Liquet,et al.  Estimation of extended mixed models using latent classes and latent processes: the R package lcmm , 2015, 1503.00890.

[7]  A. Bianchi,et al.  Correlation between phenotype and genotype in a large unselected cohort of children with severe hemophilia A. , 2013, Blood.

[8]  E. Berntorp,et al.  Haemophilia and joint disease: pathophysiology, evaluation, and management , 2011, Journal of comorbidity.

[9]  P. Helders,et al.  Joint health and functional ability in children with haemophilia who receive intensive replacement therapy , 2011, Haemophilia : the official journal of the World Federation of Hemophilia.

[10]  E. Pullenayegum,et al.  Age at first joint bleed and bleeding severity in boys with severe hemophilia A: Canadian Hemophilia Primary Prophylaxis Study , 2011, Journal of thrombosis and haemostasis : JTH.

[11]  L. Mantovani,et al.  A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study) , 2011, Journal of thrombosis and haemostasis : JTH.

[12]  L. Valentino,et al.  Blood‐induced joint disease: the pathophysiology of hemophilic arthropathy , 2010, Journal of thrombosis and haemostasis : JTH.

[13]  G. Dolan,et al.  Changing patterns of bleeding in patients with severe haemophilia A , 2009, Haemophilia : the official journal of the World Federation of Hemophilia.

[14]  P. Lenting,et al.  Models for Prediction of Factor VIII Half-Life in Severe Haemophiliacs: Distinct Approaches for Blood Group O and Non-O Patients , 2009, PloS one.

[15]  M. Margaglione,et al.  The Italian AICE-Genetics hemophilia A database: results and correlation with clinical phenotype , 2008, Haematologica.

[16]  Chih-Chien Yang,et al.  Separating Latent Classes by Information Criteria , 2007, J. Classif..

[17]  Alan R. Cohen,et al.  Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. , 2007, The New England journal of medicine.

[18]  M. Manco‐Johnson,et al.  The Colorado Haemophilia Paediatric Joint Physical Examination Scale: normal values and interrater reliability , 2007, Haemophilia : the official journal of the World Federation of Hemophilia.

[19]  V. Blanchette,et al.  Pharmacokinetics of recombinant factor VIII (Kogenate‐FS®) in children and causes of inter‐patient pharmacokinetic variability , 2006 .

[20]  B. Feldman,et al.  Hemophilia joint health score reliability study , 2006, Haemophilia : the official journal of the World Federation of Hemophilia.

[21]  P. Babyn,et al.  Tailored prophylaxis in severe hemophilia A: interim results from the first 5 years of the Canadian Hemophilia Primary Prophylaxis Study , 2006, Journal of thrombosis and haemostasis : JTH.

[22]  D. Grobbee,et al.  Variability in clinical phenotype of severe haemophilia: the role of the first joint bleed , 2005, Haemophilia : the official journal of the World Federation of Hemophilia.

[23]  D. Grobbee,et al.  Factor VIII half-life and clinical phenotype of severe hemophilia A. , 2005, Haematologica.

[24]  M. Baiget,et al.  The prothrombin 20210A allele influences clinical manifestations of hemophilia A in patients with intron 22 inversion and without inhibitors. , 2002, Haematologica.

[25]  M. Manco‐Johnson,et al.  Joint evaluation instruments for children and adults with haemophilia , 2000, Haemophilia : the official journal of the World Federation of Hemophilia.

[26]  J. Sixma,et al.  The Half-life of Infused Factor VIII Is Shorter in Hemophiliac Patients with Blood Group 0 than in those with Blood Group A , 2000, Thrombosis and Haemostasis.

[27]  H. Pettersson,et al.  A longitudinal study of orthopaedic outcomes for severe factor‐VIII‐deficient haemophiliacs , 1994, Journal of internal medicine.

[28]  In Colorado. , 1906 .