Giancarlo Castaman1,2, Angiola Rocino3, M Gabriella Mazzucconi4, Ezio Zanon5, Fabio Gagliano6, Angelo C Molinari7. 1. Centre for Bleeding Disorders, Careggi University Hospital, Florence, Italy. 2. Haemophilia and Thrombosis Centre, Department of Haematology, San Bortolo Hospital, Vicenza, Italy. 3. Haemophilia and Thrombosis Centre, San Giovanni Bosco Hospital, Naples, Italy. 4. Department of Cellular Biotechnology and Haematology, La Sapienza University, Rome, Italy. 5. Department of Cardiologic, Thoracic and Vascular Sciences, Haemophilia Center-University of Padua, Italy. 6. Reference Centre for Paediatric Haemostasis and Thrombosis, G. Di Cristina Hospital, Palermo, Italy. 7. Thrombosis and Haemostasis Unit, Haemophilia Treatment Centre, Giannina Gaslini Children's Hospital, Genoa, Italy.
Abstract
BACKGROUND: Although current treatment guidelines recommend prophylaxis in paediatric patients with haemophilia, specific indications for and barriers to the prescription of prophylaxis in the paediatric haemophiliac population have not been established. The aim of this web-based survey of clinicians at Haemophilia Treatment Centres in Italy was to identify factors for and against the initiation of prophylactic coagulation factor replacement therapy in paediatric patients with haemophilia. MATERIALS AND METHODS: A literature search was conducted to identify factors to include in the survey. Seventeen clinicians from Italian Haemophilia Centres were invited to complete the web-based survey and to rank factors in favour of and those that acted as barriers to prophylaxis in terms of "importance" and "influence" on a numerical scale (0=not important to 100=very important). Any factors for which there was a large discrepancy in results from the survey were further "ranked" by clinicians at an interactive question and answer session at a symposium. RESULTS: A total of 13 web surveys were returned; the most highly scored factors favouring prophylaxis were "bleeding frequency", "bleeding severity" and "presence of target joints", and the most highly scored barriers were "parents' acceptance", "venous access" and "compliance to therapy". Other important factors favouring prophylaxis were "severity of coagulation defect" and "orthopaedic score". DISCUSSION: This survey gives helpful clinician-derived information for people treating haemophiliacs in Italy, to help the treatment-providers orient themselves better regarding the prescription of prophylaxis for paediatric patients.
BACKGROUND: Although current treatment guidelines recommend prophylaxis in paediatric patients with haemophilia, specific indications for and barriers to the prescription of prophylaxis in the paediatric haemophiliac population have not been established. The aim of this web-based survey of clinicians at Haemophilia Treatment Centres in Italy was to identify factors for and against the initiation of prophylactic coagulation factor replacement therapy in paediatric patients with haemophilia. MATERIALS AND METHODS: A literature search was conducted to identify factors to include in the survey. Seventeen clinicians from Italian Haemophilia Centres were invited to complete the web-based survey and to rank factors in favour of and those that acted as barriers to prophylaxis in terms of "importance" and "influence" on a numerical scale (0=not important to 100=very important). Any factors for which there was a large discrepancy in results from the survey were further "ranked" by clinicians at an interactive question and answer session at a symposium. RESULTS: A total of 13 web surveys were returned; the most highly scored factors favouring prophylaxis were "bleeding frequency", "bleeding severity" and "presence of target joints", and the most highly scored barriers were "parents' acceptance", "venous access" and "compliance to therapy". Other important factors favouring prophylaxis were "severity of coagulation defect" and "orthopaedic score". DISCUSSION: This survey gives helpful clinician-derived information for people treating haemophiliacs in Italy, to help the treatment-providers orient themselves better regarding the prescription of prophylaxis for paediatric patients.
Authors: Ronald D Barr; Mahassen Saleh; William Furlong; John Horsman; Julia Sek; Mohan Pai; Irwin Walker Journal: Am J Hematol Date: 2002-11 Impact factor: 10.047
Authors: K Fischer; J G van der Bom; P Molho; C Negrier; E P Mauser-Bunschoten; G Roosendaal; P De Kleijn; D E Grobbee; H M van den Berg Journal: Haemophilia Date: 2002-11 Impact factor: 4.287
Authors: J Astermark; A Rocino; M Von Depka; H M Van Den Berg; A Gringeri; L G Mantovani; M Morado; R P Garrido; M Schiavoni; A Villar; J Windyga Journal: Haemophilia Date: 2007-01 Impact factor: 4.287
Authors: M Morfini; G Auerswald; R A Kobelt; G F Rivolta; J Rodriguez-Martorell; F A Scaraggi; C Altisent; J Blatny; A Borel-Derlon; V Rossi Journal: Haemophilia Date: 2007-09 Impact factor: 4.287
Authors: Marilyn J Manco-Johnson; Thomas C Abshire; Amy D Shapiro; Brenda Riske; Michele R Hacker; Ray Kilcoyne; J David Ingram; Michael L Manco-Johnson; Sharon Funk; Linda Jacobson; Leonard A Valentino; W Keith Hoots; George R Buchanan; Donna DiMichele; Michael Recht; Deborah Brown; Cindy Leissinger; Shirley Bleak; Alan Cohen; Prasad Mathew; Alison Matsunaga; Desiree Medeiros; Diane Nugent; Gregory A Thomas; Alexis A Thompson; Kevin McRedmond; J Michael Soucie; Harlan Austin; Bruce L Evatt Journal: N Engl J Med Date: 2007-08-09 Impact factor: 91.245
Authors: Sophie le Doré; Nathalie Grinda; Emmanuelle Ferré; Valerie Roussel-Robert; Birgit Frotscher; Pierre Chamouni; Sandrine Meunier; Sophie Bayart; Edita Dolimier; Francoise Truong-Berthoz; Emmanuelle de Raucourt Journal: J Blood Med Date: 2021-03-09