Request for further standardization issues for future studies in paediatric haemophilia patients - Rebuttal to 'Including the life-time cumulative number of joint bleeds in the definition of primary prophylaxis'

摘要

With great interest we read the careful comments of Ingrid den Uijl and Kathelijn Fischer (1) with respect to our article "effects of primary and secondary prophylaxis on the clinical expression of joint damage in children with severe haemophilia A (HA) -Results of a multicenter non-concurrent cohort study" (2). The authors comment on i) the classifications used for primary and secondary prophylaxis, ii) the inclusion of inhibitor patients in the present database study, and iii) the possible calculation of life time cumulative number of joint bleeds.Ad i (definition of terms used in the present database study): In the present non-concurrent cohort study, primary prophylaxis was defined as factor infusions given to prevent bleeding before the third but usually starting after the first bleed (3). In addition, patients who did not suffer more than one symptomatic joint bleed into the same joint within a six-month period before the start of long-term continuous treatment were classified as primary prophylaxis patients (modification to ). Secondary prophylaxis was defined as long-term continuous factor replacement therapy not fulfilling the modified criteria for primary prophylaxis.