Numerous challenges confront adult hemophilia patients with inhibitors, including difficulty in controlling bleeding episodes, deterioration of joints, arthritic pain, physical disability, emotional turmoil, and social issues. High-intensity treatment regimens often used in the treatment of patients with inhibitors also impose significant scheduling, economic, and emotional demands on patients and their families or primary caregivers. A comprehensive multidisciplinary assessment of the physical, emotional, and social status of adult hemophilia patients with inhibitors is essential for the development of treatment strategies that can be individualized to address the complex needs of these patients.

Non-adherence to home based treatment even 10% of the time in hemophilia care may portend a poor outcome and long-range quality of life issues, despite a still enormous financial cost. This study was a rigorous effort to quantify adherence and then compare it to QofL. 79 subjects with hemophilia (48 >18-years and 31<18-years) participated in the study and completed the SF-36 or Child Health Questionnaire (validated QofL forms). Subjects were either using on-demand (OD) treatment regimens or on high intensity (HI) treatment strategies i.e. prophylaxis or immune tolerance therapy. Physician’s specific treatment order for dose and frequency of clotting factor concentrate (CFC) were collected. Factor infusion logs kept by patients were reviewed to determine how factor use matched treatment recommendations. Infusion logs were maintained by 47 of 79 subjects (59%). The amount of CFC used was entered into a database along with the date of the infusion and the reason for the infusion. Due to vial size variability doses that were within 80% to 150% of the prescribed dose for children and 80% to 120% for adults were considered adherent. A scoring system was developed for both OD and HI users based on documented usage patterns. A given subject’s adherence score could range from 0 (representing total non-adherence) to 100% (representing perfect adherence). A subject’s adherence percentile was then ranked accordingly: 0% to 33% = Low Adherence, 34% to 66% = Moderate Adherence, 67% to 100% = High Adherence. Chi Square was used to determine the relationship between adherence and treatment regimen and a two-way analysis of variance was used to compare QofL in subjects with treatment logs to adherence and treatment regimen. Subjects on HI had lower adherence patterns than those on OD (p=. 018). Significant findings related to QofL included that body pain in children was higher with OD compared with HI (p=. 036), however this was not demonstrated in adults. Adults with low adherence patterns had higher mean scores in mental health then high (p=. 021) and moderate (p=. 068) adhering adults. Quantifying adherence and attempting to understand the relationship between adherence and QofL is an important component in the management of hemophilia.