Joint disease and its medical and socio-economic impact on haemophilia care in Romania

Abstract

Hemophilia is a congenital, hereditary, rare bleeding disorder, which fortunately has nowadays an optimal therapeutical solution, namely a life-long replacement therapy (RT) with the missing coagulation factor VIII (hemophilia A) or IX (hemophilia B). Inappropriate treatment results in secondary morbidity, dominated by the chronic hem arthropathy, with high medical and socioeconomic burden. Objectives: As in our country, RT has been started only in 1997 and a real prophylactic replacement (PR) only in 2016/2017, we aimed at conducting a study of persons mainly with prophylactic RT and of those mainly with on-demand (OD) regimen for obtaining a comparative evaluation of the medical and socio-economic outcomes. Material and methods: This observational cross-sectional patient-reported outcome (PRO) survey included 122 patients with a severe form of the disease, willing to answer to a large questionnaire with 56 items focused on 4 domains: socio-demographic, medical, quality of health and life, and direct costs of care. Results: There have been assessed striking, statistically different results, concerning the markers which reveal clinical severity (annualized joint and non-joint bleeding rate, target joints, hem arthropathy), the need for chronic analgesic therapy and orthopedic interventions, frequency of deficiency, disability, handicap and social assistance in the group of persons with OD treatment vs. PR. The high costs related to surgical interventions and occurrence of inhibitors have been underlined, related to a secondary morbidity of hemophilia. Conclusions: The favorable effects of PR are evident also in our country; its high costs are counterbalanced by the deleterious health and quality of life results of OD treatment, and also by its high costs for the specific secondary morbidity, pleading in the favor of PR, in accordance with the principles of care in Europe.