International Retrospective Chart Review of Treatment Patterns in Severe Familial Mediterranean Fever, Tumor Necrosis Factor Receptor–Associated Periodic Syndrome, and Mevalonate Kinase Deficiency/Hyperimmunoglobulinemia D Syndrome

Objective: Periodic fever syndrome (PFS) conditions are characterized by recurrent attacks of fever and localized inflammation. This study examined the diagnostic pathway and treatments at tertiary centers for familial Mediterranean fever (FMF), tumor necrosis factor receptor–associated periodic syndrome (TRAPS), and mevalonate kinase deficiency (MKD)/hyperimmunoglobulinemia D syndrome (HIDS). Methods: PFS specialists at medical centers in the US, the European Union, and the eastern Mediterranean participated in a retrospective chart review, providing de‐identified data in an electronic case report form. Patients were treated between 2008 and 2012, with at least 1 year of followup; all had clinical and/or genetically proven disease and were on/eligible for biologic treatment. Results: A total of 134 patients were analyzed: FMF (n = 49), TRAPS (n = 47), and MKD/HIDS (n = 38). Fever was commonly reported as severe across all indications. Other frequently reported severe symptoms were serositis for FMF patients and elevated acute‐phase reactants and gastrointestinal upset for TRAPS and MKD/HIDS. A long delay from disease onset to diagnosis was seen within TRAPS and MKD/HIDS (5.8 and 7.1 years, respectively) compared to a 1.8‐year delay in FMF patients. An equal proportion of TRAPS patients first received anti–interleukin‐1 (anti‐IL‐1) and anti–tumor necrosis factor (anti‐TNF) biologic agents, whereas IL‐1 blockade was the main choice for FMF patients resistant to colchicine and MKD/HIDS patients. For TRAPS patients, treatment with anakinra versus anti‐TNF treatments as first biologic agent resulted in significantly higher clinical and biochemical responses (P = 0.03 and P < 0.01, respectively). No significant differences in responses were observed between biologic agents among other cohorts. Conclusion: Referral patterns and diagnostic delays highlight the need for greater awareness and improved diagnostics for PFS. This real‐world treatment assessment supports the need for further refinement of treatment practices

Africa’s response to the COVID-19 pandemic : A review of the nature of the virus, impacts and implications for preparedness

Background: COVID-19 continues to wreak havoc in different countries across the world, claiming thousands of lives, increasing morbidity and disrupting lifestyles. The global scientific community is in urgent need of relevant evidence, to understand the challenges and knowledge gaps, as well as the opportunities to contain the spread of the virus. Considering the unique socio-economic, demographic, political, ecological and climatic contexts in Africa, the responses which may prove to be successful in other regions may not be appropriate on the continent. This paper aims to provide insight for scientists, policy makers and international agencies to contain the virus and to mitigate its impact at all levels. Methods: The Affiliates of the African Academy of Sciences (AAS), came together to synthesize the current evidence, identify the challenges and opportunities to enhance the understanding of the disease. We assess the potential impact of this pandemic and the unique challenges of the disease on African nations. We examine the state of Africa’s preparedness and make recommendations for steps needed to win the war against this pandemic and combat potential resurgence. Results: We identified gaps and opportunities among cross-cutting issueswhich must be addressed or harnessed in this pandemic. Factors such as the nature of the virus and the opportunities for drug targeting, point of care diagnostics, health surveillance systems, food security, mental health, xenophobia and gender-based violence, shelter for the homeless, water and sanitation, telecommunications challenges, domestic regional coordination and financing. Conclusion: Based on our synthesis of the current evidence, while there are plans for preparedness in several African countries, there are significant limitations. A multi-sectoral efforts from the science, education, medical, technology, communication, business, and industry sectors, as well as local communities, must work collaboratively to assist countries in order to win this fight

Real-World Patient Characteristics, Treatment Patterns, and Survival among Locally-Advanced/Metastatic Alk+ Non-Small Cell Lung Cancer Patients in Latin America

Objectives To describe patient characteristics, treatment patterns, and survival among Latin American patients diagnosed with locally-advanced/metastatic ALK+ non-small cell lung cancer (NSCLC). Methods Mexican and Argentine oncologists (N=5) reviewed patient charts and reported characteristics, treatment patterns, and survival on their patients diagnosed with ALK+ locally-advanced/metastatic NSCLC. Treatment duration and overall survival (OS) were estimated using Kaplan-Meier analyses. Results Patients (N=25) averaged 57 years old when diagnosed with locally-advanced/metastatic NSCLC; 64% were female, 56% were Hispanic, 44% were Caucasian, 48% were uninsured, and 24% were unemployed or on sick leave. Smoking history varied (36% never-smokers, 20% light/moderate smokers, and 32% heavy smokers). At primary diagnosis, 72% had metastatic disease. Over the course of their disease (until end of follow-up), 48% of patients developed brain metastases, 20% bone, and 32% lung. In first-line therapy, 17 patients (68%) received chemotherapy and six patients (24%) received crizotinib. After first-line chemotherapy, six patients (24%) received crizotinib. 13 patients (52%) never received an ALK inhibitor; of these, nine patients (69%) did not receive crizotinib because they could not afford it or crizotinib was not covered by insurance. Out of the 12 patients who received crizotinib, three died, and eight discontinued by the end of follow-up (median duration of 127 days), with three patients switching to chemotherapy, one to afatinib, and four receiving no further antineoplastic therapy. After diagnosis of locally-advanced/metastatic NSCLC, the OS rate among all patients was 74% at 12 months. Conclusions Though the sample size is small, the study provides the first analysis of patient characteristics, treatment patterns, and survival among ALK+ NSCLC patients in Latin America. Many patients were women, uninsured, never received an ALK inhibitor, and their OS was low. These findings suggest that there could be an unmet need for access to effective treatments for ALK+ NSCLC patients in Latin America.Fil: Arrieta, O.. Instituto Nacional de Cancerologia; MacaoFil: Mascheroni, M. B.. Sanatorio Nosiglia; ArgentinaFil: Recondo, Gonzalo. Consejo Nacional de Investigaciones Científicas y Técnicas. Oficina de Coordinación Administrativa Parque Centenario. CEMIC-CONICET. Centro de Educaciones Médicas e Investigaciones Clínicas "Norberto Quirno". CEMIC-CONICET.; ArgentinaFil: Kaen, D. Centro Oncologico Riojano Integral; ArgentinaFil: Zhang, J.. Novartis Pharmaceuticals Corporation; Estados UnidosFil: Patel, D.. Navigant Consulting; Estados UnidosFil: Swallow, E.. Analysis Group; Estados UnidosFil: Balu, S. Novartis Pharmaceuticals Corporation; Estados UnidosFil: Camacho, O. C.. Novartis Farmaceutica; Estados UnidosFil: Ratto, B. Novartis Argentina S.A; ArgentinaFil: Kageleiry, A. Analysis Group; Estados UnidosFil: Stein, K. Novartis Pharmaceuticals Corporation; Estados UnidosFil: Degun, R. Navigant Consulting Inc.; Reino UnidoFil: Martin, C. Fleming Institute; Argentin