THE EFFECTS OF RG-CSF ON HEALTH-RELATED QUALITY-OF-LIFE IN CHILDREN WITH CONGENITAL AGRANULOCYTOSIS

A multicentre Phase II clinical trial was recently undertaken to docum ent the value of treatment with recombinant human granulocyte colony-s timulating factor (rG-CSF) in children with congenital agranulocytosis . To assess the impact of such therapy on health-related quality of li fe (HRQOL), we developed a questionnaire that was administered to the parents of study patients, twice prior to the initiation of treatment, and then monthly thereafter for six months. The questionnaire focused on several aspects of HRQOL that we thought were important in this pa tient population, including functional status, general health percepti ons, activity limitation, disease symptoms, and discomfort associated with therapy. In this paper, we describe the questionnaire that we dev eloped and the process by which it was translated into several languag es. We also report on the impact of rG-CSF therapy on HRQOL. A total o f 130 questionnaire were administered to the parents of 19 study patie nts between the ages of four and one-half months and 18 years in 11 st udy centres in four countries. Although our sample size is small, our findings suggest that treatment with rG-CSF may result in significant improvements in general health perceptions, limitations of daily activ ities, and symptoms of the disease.