Effectiveness and Safety of Ibrutinib for Chronic Lymphocytic Leukemia in Routine Clinical Practice: 3-Year Follow-up of the Belgian Ibrutinib Real-World Data (BiRD) Study.

The multicenter observational BiRD study investigated the real-world effectiveness and safety of ibrutinib in patients with chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL) and Waldenström's macroglobulinemia (WM) in Belgium. This interim analysis reports results for patients with CLL, with a median follow-up of 34 months. Overall, patients had predominantly relapsed/refractory disease (73%) and were elderly (median age 72 years) with high-risk features such as del17p and/or TP53 mutations (59%). Patients were included either prospectively or retrospectively, and the total patient population effectiveness results were adjusted with left truncation. In the effectiveness population (N = 221: prospective, n = 71; retrospective, n = 150), the overall response rate was 90.0%. Median progression-free survival was 38.3 months (prospective, not estimable; retrospective, 51.5 months) and median overall survival was not yet estimable in the total, prospective and retrospective groups. Treatment-emergent adverse events (TEAEs) for the prospective and retrospective groups are reported separately. Any-grade TEAEs of interest in the prospective/retrospective groups included infections (67.1%/60.1%), diarrhea (20.5%/10.5%), hypertension (16.4%/9.8%) and atrial fibrillation (12.3%/7.2%). Major bleeding was reported in 5.5%/3.3% of prospective/retrospective patients, with little difference observed between those receiving versus not receiving antithrombotic treatment. Discontinuations due to toxicity were reported in 10.5% of patients. Results from this interim analysis show treatment with ibrutinib to be effective and tolerable, with no new safety signals observed. Future analyses will report on longer-term follow-up.

Information needs of physicians regarding the diagnosis of rare diseases: a questionnaire-based study in Belgium.

BACKGROUND: Late and misdiagnoses of rare disease patients are common and often result in medical, physical and mental burden for the patient, and financial and emotional burden for the patient's family. Low rare disease awareness among physicians is believed to be one of the reasons for these late and misdiagnoses of rare disease patients. The aim of this study was to investigate how information and education could be tailored to the needs and preferences of physicians in Belgium to increase their rare disease awareness and support them in diagnosing patients with a rare disorder. Nine exploratory interviews with Belgian rare disease experts were performed in December 2016 to help the development of a questionnaire on information needs of physicians and their consulted information sources in rare disease awareness and diagnosis. This online questionnaire was then completed by Belgian physicians (n = 295), including general practitioners (GPs), pediatricians and other specialists (i.e. neurologists, pediatric neurologists, endocrinologists and pediatric endocrinologists) during January and February 2017. RESULTS: Rare disease knowledge and awareness were the lowest among GPs and the highest among specialists. Interviewed experts indicated that physicians' academic and continuous medical education should be focused more on "red flags" to increase rare disease attentiveness in daily clinical practice. GPs scored their academic education on rare diseases as insufficient but pediatricians and other specialists scored it significantly better (p < 0.001). Even though GPs declared to only need information on rare diseases when having a rare disease patient in their practice, specialists indicated to need more rare disease information in general. Most physicians confirmed that they had specific information needs regarding rare diseases. Unlike specialists, the majority of GPs were unaware of information sources such as Orphanet. CONCLUSION: In order to effectively support physicians in Belgium to diagnose rare diseases early, the academic medical education on rare diseases should be revised. Teaching methods should be focused more on casuistry and "red flags". An Orphanet-like digital platform about rare disease symptoms, diagnostic tests and reference centers might be ideal to support correct and timely diagnosis.

Perception of Originator Biologics and Biosimilars: A Survey Among Belgian Rheumatoid Arthritis Patients and Rheumatologists.

BACKGROUND: Among patients and rheumatologists, current knowledge and perception of biosimilars in comparison with originator biologics is unknown. OBJECTIVES: The aim of this study was to investigate this knowledge and perception in Belgian rheumatologists and rheumatoid arthritis (RA) patients. METHODS: Anonymous web surveys were conducted in Belgian RA patients (n = 121) and rheumatologists (n = 41) during the period January-March 2016. The surveys covered topics on knowledge, similarity, price, preference, interchangeability, extrapolation and switching. Descriptive and statistical analyses of responses were performed. RESULTS: Familiarity with biosimilars was reported by 49% of patients, of whom 77% knew what biosimilars were. RA patients equally questioned the proven efficacy of originators and biosimilars in RA, as well as their side effects and suitability. Furthermore, RA patients questioned the safety of biosimilars more often than that of originators (35 vs. 20%, respectively; p = 0.0094). Rheumatologists, more so than patients, expressed concerns that there might be differences between originators and biosimilars in terms of quality, safety, and price (p = 0.0292, p < 0.0001, p = 0.0129, respectively). The opinions of rheumatologists on interchangeability and extrapolation of indications varied. The price of an originator contributed substantially to the medicine preference of rheumatologists (p = 0.0002), but not patients. CONCLUSION: Our study showed that rheumatologists, more so than patients, were convinced that there can be differences between originators and biosimilars. Despite safety being the major concern of patients, patients trusted their physician's decision to start on or switch to a biosimilar. The evolution of the uptake of biosimilars in Belgium might thus depend mainly on the perception of physicians.

Gastrointestinal risk assessment in patients requiring non-steroidal anti-inflammatory drugs for osteoarthritis: The GIRANO study.

OBJECTIVE: To evaluate gastrointestinal risk profiles in patients with osteoarthritis who are currently being treated, or who are candidates for treatment, with a non-steroidal anti-inflammatory drug. METHODS: Patients with osteoarthritis treated by primary care physicians or physical and rehabilitation medicine (PRM) specialists in Belgium and Luxembourg were scored for gastrointestinal risk profile, low, moderate or high, based on the presence of gastrointestinal risk factors. These included advanced age, gastrointestinal history, comorbidities, medication use, smoking, and alcohol consumption. Use of gastro-protective agents (proton pump inhibitors) was also assessed. RESULTS: A total of 190 primary care physicians and PRM specialists provided data on 885 patients. A large majority of patients were rated high-risk gastrointestinal (77.8%), with fewer moderate (19.4%) or low (2.8%) risk. The proportion of high-risk patients treated by PRM specialists was significantly lower than that treated by primary care physicians (64.7% vs 79.9%; p < 0.0001). Only 37.0% of high-risk patients received proton pump inhibitors co-prescription with a non-steroidal anti-inflammatory drug. CONCLUSION: A high prevalence of elevated gastrointestinal risk was found in this survey of patients with osteoarthritis who were current or candidate users of non-steroidal anti-inflammatory drugs. This appears to be insufficiently recognized, as preventative gastro-protective co-treatment was infrequently prescribed. Careful assessment of gastrointestinal risk factors should be made before prescribing non-steroidal anti-inflammatory drugs, with treatment tailored to the patient's gastrointestinal risk profile.