The Patient Voice in Neovascular Age-Related Macular Degeneration: Findings from a Qualitative Study.

Introduction: Visual impairment resulting from diseases such as neovascular age-related macular degeneration (nAMD) may cause behavioural, environmental, psychological, and logistical challenges that could act as barriers to effective uptake and sustainability of treatment with anti-vascular endothelial growth factor agents (anti-VEGFs). Understanding emotions and experiences of patients with nAMD may help inform the determinants of adherence, and could contribute to improvements in ophthalmic outcomes and quality of life.

Methods: Seventeen patients with nAMD receiving anti-VEGF injections were enrolled from three clinics: one each in France (n = 5), Germany (n = 6), and the UK (n = 6).

Real-world evidence on Kovaltry (81-8973) in children with moderate or severe hemophilia A in Europe: a nested cohort analysis.

Background: Untreated hemophilia A patients may experience recurrent bleeding events leading to debilitating joint damages. While RCT and pharmacokinetic data support the value of Kovaltry [an unmodified full-length recombinant factor VIII (FVIII) product], real world evidence in children is lacking. This report describes a descriptive and multivariate analysis of the effectiveness of Kovaltry in children with hemophilia A in the real-world setting, using data from medical chart abstraction and cross-sectional surveys of physicians, patients, and caregivers.

Nonadherence or Nonpersistence to Intravitreal Injection Therapy for Neovascular Age-Related Macular Degeneration: A Mixed-Methods Systematic Review.

Topic: Systematic review of risk factors for nonadherence and nonpersistence to intravitreal anti-vascular endothelial growth factor (VEGF) injection therapy for neovascular age-related macular degeneration (nAMD).

Clinical Relevance: Lack of adherence (nonadherence) or undertreatment (nonpersistence) with respect to evidence from clinical trials remains a significant barrier to optimizing real-world outcomes for patients with nAMD. Contributing factors and strategies to address this are poorly understood.

Evaluating the Safety of Medication Exposures During Pregnancy: A Case Study of Study Designs and Data Sources in Multiple Sclerosis.

Background: Regulatory agencies often request prospective, product-specific post-authorization pregnancy exposure registries to monitor safety during pregnancy, even though studies using existing health databases could also be employed.

Objectives: Using multiple sclerosis (MS) as a case study, we evaluated various study designs and data sources previously used to study medication exposure in pregnancy.

Methods: We examined (1) strengths and limitations of study designs used for pregnancy safety studies in women exposed to MS-specific medications during pregnancy and (2) existing data sources used to conduct such studies in other disease areas.

Selecting Patients with Ovarian Cancer for Germline BRCA Mutation Testing: Findings from Guidelines and a Systematic Literature Review.

Introduction: One of the most significant risk factors for the development of ovarian cancer (OC) is a genetic mutation in BRCA1 (breast cancer gene 1) or BRCA2. Here we describe the impact of previous and current guidance on BRCA testing practices and provide evidence about which characteristics best identify patients with OC and an underlying germline BRCA mutation.

Methods: A search was conducted for guidelines recommending genetic testing to identify constitutional pathogenic mutations in the BRCA genes.

Prophylactic antibiotics for preventing pneumococcal infection in children with sickle cell disease.

Background This is an update of a Cochrane Review first published in 2002, and previously updated in 2012. People with sickle cell disease are particularly susceptible to infection. Infants and very young children are especially vulnerable, and the 'Co-operative Study of Sickle Cell Disease' observed an incidence rate of 10 per 100 patient years of pneumococcal septicaemia in children under the age of three.

Splenectomy versus conservative management for acute sequestration crises in people with sickle cell disease.

Background: Acute splenic sequestration crises are a complication of sickle cell disease, with high mortality rates and frequent recurrence in survivors of first attacks. Splenectomy and blood transfusion, with their consequences, are the mainstay of long-term management used in different parts of the world.

Objectives: To assess whether splenectomy (total or partial), to prevent acute splenic sequestration crises in people with sickle cell disease, improved survival and decreased morbidity in people with sickle cell disease, as compared with regular blood transfusions.

Prophylactic antibiotics for preventing pneumococcal infection in children with sickle cell disease.

Background: People with sickle cell disease are particularly susceptible to infection. Infants and very young children are especially vulnerable, and the 'Co-operative Study of Sickle Cell Disease' observed an incidence rate of 10 per 100 patient years of pneumococcal septicaemia in children under the age of three. Vaccines, including customary pneumococcal vaccines, may be of limited use in this age group.

Preoperative blood transfusions for sickle cell disease.

Background: Sickle cell disease is one of the most common inherited diseases in the world, and can cause haemolytic anaemia, vaso-occlusive crises and dysfunction in virtually any organ system in the body. Surgical procedures are often required. Blood transfusion regimens can be used preoperatively in an attempt to increase transport of oxygen around the body and dilute the sickled red blood cells, thus reducing the risk of vaso-occlusion.

Use of long-acting beta-agonists and inhaled steroids in asthma: meta-analysis of observational studies.

Background: Current asthma guidelines recommend the use of long-acting beta-agonists (LABAs) in combination with inhaled corticosteroids (ICSs) for long-term control and prevention of symptoms in persistent asthma. Data on the risk of asthma exacerbations of LABAs in combination with ICSs, as prescribed in typical clinical practice, are very scarce.

Methods: The authors conducted a systematic literature review and meta-analysis of observational studies to examine the risk of asthma exacerbations, measured as asthma-related hospitalization and/or asthma-related emergency room (ER) visits, in adults receiving LABAs plus ICSs in a fixed-dose combination compared with patients receiving ICSs alone.

A call for international harmonization in therapeutic risk management.

Therapeutic risk management is required to ensure that the benefits of a particular drug outweigh the risks in general practice. Current risk management strategies and handling of risk management and pharmacovigilance issues differ across borders. Differences in key regulatory decisions on the same product around the world, including the handling of safety issues with cisapride, dofetilide, and isotretinoin, bring into question the robustness of these decisions and the procedures currently in place to manage the risks to the public of products with potentially unfavorable risk-benefit balances.