The hereditary angioedema burden of illness study in Europe (HAE-BOIS-Europe) : background and methodology

Background: Hereditary angioedema (HAE) is a rare but serious disease marked by swelling attacks in the extremities, face, trunk, airway, or abdominal areas that can be spontaneous or the result of trauma and other triggers. It can be life-threatening due to the risk of asphyxiation. While there have been major advancements in our understanding of the immunogenetics of HAE, there are significant gaps in the literature regarding understanding of the humanistic and economic impact of the disease, particularly in Europe. The purpose of the HAE Burden of Illness Study-Europe (HAE-BOIS-Europe), the development and methodology of which is described here, is to better understand the management and impact of HAE from the patient perspective in Europe. Methods/Design: This is a cross-sectional study in which retrospective data were also collected being conducted in Denmark, Germany and Spain. The study is open to patients ages 12 and older with a diagnosis of HAE-I or HAE-II. Data collection includes: (i) a survey on individuals’ health care resource use, direct and indirect medical costs, impact on work and school, treatment satisfaction, and emotional functioning (via the Hospital Anxiety and Depression Scale); and (ii) one-on-one interviews to collect detailed descriptive data and patient testimonials on the impact of HAE on patients’ health-related quality of life. Discussion: The present manuscript describes the development and plans for implementing a multi-country European study with the aim of characterizing the humanistic and economic burden of HAE from the patient perspective. This study will help raise awareness of HAE as a rare but debilitating condition with wide-ranging impacts

Chemotherapy Exposure and outcomes of Chronic Lymphoid Leukemia Patients

This study describes chemotherapy exposure, healthcare utilization, overall survival (OS) and progression-free survival (PFS) among patients diagnosed with chronic lymphoid leukemia (CLL). Newly diagnosed CLL patients who received chemotherapy were selected from the Eindhoven Cancer Registry between 1998-2011, linked on a patient-level to the PHARMO Database Network including data on in- and out-patient drug dispensings, hospitalizations and clinical laboratory measurements. Chemotherapy was classified in regimens of use based on chemotherapy combinations. OS and PFS were determined after diagnosis and after chemotherapy. Healthcare utilization was assessed in the year before diagnosis and in the year after chemotherapy. In total, 125 CLL patients received chemotherapy: 52 patients (42%) started chemotherapy within 6 months and 73 patients (58%) started chemotherapy ≥6 months after diagnosis. Mean (±SD) age was 67(±10) years and 68% was male. About 50% had one treatment line and about 25% two lines of treatment. Chlorambucil was the most common type of first line chemotherapy. Prior diagnosis, 44% were hospitalized for any cause and 94% had at least one drug dispensing. After chemotherapy, this was 43% and 98%, respectively. One-year survival rate after diagnosis was 94%. Median PFS after first treatment line was 17 months for patients starting within 6 months and 27 months for patients starting ≥6 months after diagnosis. In conclusion, most CLL patients receiving chemotherapy were treated with chlorambucil. One-year after initial diagnosis, 94% were still alive. Median PFS after first line chemotherapy ranged from 17 to 27 months, depending on the timing of chemotherapy