title: Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized.
abstract: The frequency of joint bleeds and orthopaedic joint scores were evaluated in 121 patients with severe haemophilia who had started prophylactic treatment with clotting factor concentrates at least once weekly before the age of 10. 75 of the patients started before the age of 3, 31 at the age of 3-5 and 15 at the age of 6-9. Each subgroup was evaluated separately. In addition, a regimen of one infusion weekly was compared with that of two (haemophilia B) or three (haemophilia A) infusions weekly in each patient. A significant decrease in the overall number of joint bleeds per year was found after shortening the infusion interval (P<0.005), but the individual bleeding pattern varied. In survival analysis of the first pathologic joint score event, those who started prophylaxis before the age of 3 had a better outcome overall than those starting at later ages (P=0.001). However, in subgroup analysis, no significant difference was seen in the annual number of joint bleeds and the development of arthropathy between those starting with, or shifting to, the more intensive regimen before the age of 3 and those that were put on this regimen at the age of 3-5. Age at start of prophylaxis was found to be an independent predictor for the development of arthropathy (P=0.0002), whereas dose and infusion interval at start were not. Our data emphasize the importance of starting replacement therapy during the first years of life. However, it seems that when beginning the regimen it can be individualized and adjusted according to the bleeding pattern. In this way, the need for a venous access system may be assessed on an individual basis.
authors.affiliation: Department for Coagulation Disorders, University of Lund, University Hospital, Malmö.
authors.forename: J
authors.initials: J
authors.lastname: Astermark
authors.forename: P
authors.initials: P
authors.lastname: Petrini
authors.forename: L
authors.initials: L
authors.lastname: Tengborn
authors.forename: S
authors.initials: S
authors.lastname: Schulman
authors.forename: R
authors.initials: R
authors.lastname: Ljung
authors.forename: E
authors.initials: E
authors.lastname: Berntorp
mesh_terms: D000367:Age Factors; D001779:Blood Coagulation Factors / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D002985:Clinical Protocols; D006467:Hemophilia A / Q000517:prevention & control*; D002836:Hemophilia B / Q000517:prevention & control*; D006470:Hemorrhage / Q000209:etiology; D006801:Humans; D007592:Joint Diseases / Q000209:etiology; D008297:Male
chemical_list: D001779:Blood Coagulation Factors