title: Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group.
abstract: Inhibitors in patients with hemophilia are a rare complication of a rare disease causing pain and disability in patients and impairment to the quality of their lives. Recent advances in treatment have brought improvements, but they have done so by absorbing larger amounts of financial resources. This study involved 52 Italian patients with hemophilia with high-responding inhibitors who were longitudinally observed for 18 months to evaluate concomitantly cost of care and quality of life. Overall, 0.6 bleeding episodes per patient per month were recorded. This frequency of events was lower than that reported in other cohorts of patients with hemophilia who were not taking inhibitors. The average monthly cost of care was, in euros, 18,000 (18,000 US dollars) per patient, mainly because of treatment products. Recombinant activated factor VII, mostly used for orthopedic surgery, represented 50% of the expenses. Quality of life, measured through validated questionnaires, was similar to that of patients with severe hemophilia without inhibitors. In particular, physical quality of life was similar to that in patients with diabetes and on dialysis, whereas mental quality of life was comparable to that in the general population. This study shows that hemophilia complicated by inhibitors, a prototype of rare disease, requires high amounts of resources for management that provides a satisfactory quality of life.
authors.affiliation: Angelo Bianchi Bonomi Hemphilia and Thrombosis Center, University of Milan, Italy.
authors.forename: Alessandro
authors.initials: A
authors.lastname: Gringeri
authors.forename: Lorenzo G
authors.initials: LG
authors.lastname: Mantovani
authors.forename: Luciana
authors.initials: L
authors.lastname: Scalone
authors.forename: Pier Mannuccio
authors.initials: PM
authors.lastname: Mannucci
mesh_terms: D000328:Adult; D000906:Antibodies; D017281:Cost of Illness; D005164:Factor IX / Q000191:economics / Q000276:immunology / Q000627:therapeutic use; D005169:Factor VIII / Q000191:economics / Q000276:immunology / Q000627:therapeutic use; D005382:Financing, Personal; D017048:Health Care Costs; D006467:Hemophilia A / Q000188:drug therapy / Q000191:economics* / Q000453:epidemiology; D002836:Hemophilia B / Q000188:drug therapy / Q000191:economics* / Q000453:epidemiology; D006801:Humans; D008137:Longitudinal Studies; D008875:Middle Aged; D015995:Prevalence; D011446:Prospective Studies; D011788:Quality of Life; D011795:Surveys and Questionnaires
chemical_list: D000906:Antibodies; D005169:Factor VIII; D005164:Factor IX