Clinical and hematological features among ß-thalassemia major patients in Jazan region: A hospital-based study.

CONTEXT: Beta-thalassemia is a prevalent hereditary blood disorder in Saudi Arabia. Various clinical manifestations and hematological abnormalities are common among these patients. AIMS: To evaluate the clinical manifestations and hematological parameters of ß-thalassemia patients in Jazan, Saudi Arabia. SETTINGS AND DESIGN: This is a cross-sectional study conducted in Jazan region, Saudi Arabia. METHODS AND MATERIALS: We included 36 ß-Thalassemia major patients who admitted to PMNH during the period from February 1, 2016 to December 31, 2017. Clinical features were obtained via physical examination. The hematological parameters were obtained from the patients' medical records. STATISTICAL ANALYSIS USED: Descriptive and inferential statistics were conducted using SPSS. Continuous data was evaluated using independent sample t-test and Chi-square was used to evaluate categorical variables. P < 0.05 indicated statistical significance. RESULTS: Mean age was 18.56 ± 6.89 years. Males were 23 (63.9%). About 41.4% of patients were underweight. Exposing of the upper teeth was the most prominent feature (n = 19, 59.4%). The majority of patients were splenectomized (62.1%). About two-thirds have moderate anemia. More than half of the patients (58.8%) had high WBCs counts and 35.3% had a high platelet count, especially among those who underwent splenectomy. Approximately, two-thirds had a ferritin level higher than 2500 ng/ml. O+ blood group was the most frequent blood group among the patients (n = 15, 46.9%). CONCLUSIONS: Deteriorated clinical and hematological parameters in our study justify the need for more efforts for regular evaluation and follow-up of the ßTM patients and reevaluation of the management protocols to be improved or modified.

A Novel Small-Molecule Inhibitor of MRCK Prevents Radiation-Driven Invasion in Glioblastoma.

Glioblastoma (GBM) is an aggressive and incurable primary brain tumor that causes severe neurologic, cognitive, and psychologic symptoms. Symptoms are caused and exacerbated by the infiltrative properties of GBM cells, which enable them to pervade the healthy brain and disrupt normal function. Recent research has indicated that although radiotherapy (RT) remains the most effective component of multimodality therapy for patients with GBM, it can provoke a more infiltrative phenotype in GBM cells that survive treatment. Here, we demonstrate an essential role of the actin-myosin regulatory kinase myotonic dystrophy kinase-related CDC42-binding kinase (MRCK) in mediating the proinvasive effects of radiation. MRCK-mediated invasion occurred via downstream signaling to effector molecules MYPT1 and MLC2. MRCK was activated by clinically relevant doses per fraction of radiation, and this activation was concomitant with an increase in GBM cell motility and invasion. Furthermore, ablation of MRCK activity either by RNAi or by inhibition with the novel small-molecule inhibitor BDP-9066 prevented radiation-driven increases in motility both in vitro and in a clinically relevant orthotopic xenograft model of GBM. Crucially, treatment with BDP-9066 in combination with RT significantly increased survival in this model and markedly reduced infiltration of the contralateral cerebral hemisphere.Significance: An effective new strategy for the treatment of glioblastoma uses a novel, anti-invasive chemotherapeutic to prevent infiltration of the normal brain by glioblastoma cells.Cancer Res; 78(22); 6509-22. ©2018 AACR.

Nasal allergies in the Middle Eastern population: results from the "Allergies in Middle East Survey".

BACKGROUND: Chronic respiratory diseases such as asthma and allergic rhinitis (AR) are a major public health problem in developing countries including those in the Middle East. However, to date, there is a paucity of information related to physician-diagnosed AR in this region. The Allergies in Middle East Survey was undertaken to help clarify and broaden the understanding of physician-diagnosed AR across Egypt, Iran, Lebanon, Saudi Arabia, and the United Arab Emirates. The survey explores the frequency of physician-diagnosed AR, prevalence and types of associated symptoms, the impact on quality of life (QOL), current treatment practices, and therapy expectations. METHODS: In total, 7411 households in five countries (Egypt, Lebanon, Saudi Arabia, Iran and the United Arab Emirates) were screened to identify individuals that were ≥4 years old with a physician diagnosis of AR and either symptoms and/or treatment in the past 12 months. A total of 501 respondents from the five countries completed the survey. Standardized questionnaires were used to make comparisons across the regions; however, the data collection procedures were tailored for each country. The sample was probability based to ensure valid statistical inference to the population. RESULTS: Ten percent of the Middle East population surveyed had a physician diagnosis of AR, with 65% of respondents stating that their allergies were intermittent in nature. An otolaryngologist or allergist diagnosed the majority of the individuals surveyed. Runny nose, nasal and throat itching, postnasal drip, and nasal congestion or stuffed up nose were the most common and bothersome symptoms of AR. The majority of survey participants (58% of the overall survey population) with AR reported that the condition had an impact on their daily private and professional life. Seventy-two percent of adults reported that their AR symptoms limited their work/school activities and 35% reported that their AR interfered with and caused them to miss work or school within the past 12 months. One factor, in addition to the outward AR symptoms, that could have contributed to these function impairments may have been sleep disturbances. Although a secondary symptom to AR, sleep disturbances (difficulty getting to sleep, waking up during the night or lack of a good night's sleep) were shown in this survey to be extremely troubling in ∼15% of AR sufferers. In the past year >90% of patients reported taking a medication of any type for their AR, with nearly a 4:1 ratio of patients taking a prescription medication versus an over-the-counter (OTC) medication in the past 4 weeks. Over 75% of survey respondents reported taking an intranasal corticosteroid (INCS) in the last 4 weeks and the satisfaction rate of INCS medications was similar to that reported for OTC medications. The most common reasons cited for dissatisfaction with INCS medications were inadequate effectiveness, bothersome side effects (e.g., unpleasant taste and retrograde drainage into the pharynx), decreased effectiveness with chronic use, and failure to provide 24-hour relief. CONCLUSION: These data show that AR is common in the Middle East region as elsewhere in the world. Many patients with AR in Middle East region suffer from their symptoms (e.g., runny nose, nasal itching, nasal congestion, postnasal drip, and other symptoms) on all or most days during the times of the year that their allergies are worst. These symptoms have been shown to reduce QOL and performance at work/school to a significant degree. Additionally, the survey data underscore a considerable treatment gap with current therapies for AR and that many AR patients still have not found adequate effectiveness with currently available medications. Thus, through identification of disease impact on the Middle East population and highlighting treatment gaps, clinicians in the Middle East may better understand and treat AR, leading to improvements in overall patient satisfaction and QOL.

Nasal allergies in the Middle Eastern population: Results from the "Allergies in Middle East Survey".

BACKGROUND: Chronic respiratory diseases such as asthma and allergic rhinitis (AR) are a major public health problem in developing countries including those in the Middle East. However, to date, there is a paucity of information related to physician-diagnosed AR in this region. The Allergies in Middle East Survey was undertaken to help clarify and broaden the understanding of physician-diagnosed AR across Egypt, Iran, Lebanon, Saudi Arabia, and the United Arab Emirates. The survey explores the frequency of physician-diagnosed AR, prevalence and types of associated symptoms, the impact on quality of life (QOL), current treatment practices, and therapy expectations. METHODS: In total, 7411 households in five countries (Egypt, Lebanon, Saudi Arabia, Iran and the United Arab Emirates) were screened to identify individuals that were ≥4 years old with a physician diagnosis of AR and either symptoms and/or treatment in the past 12 months. A total of 501 respondents from the five countries completed the survey. Standardized questionnaires were used to make comparisons across the regions; however, the data collection procedures were tailored for each country. The sample was probability based to ensure valid statistical inference to the population. RESULTS: Ten percent of the Middle East population surveyed had a physician diagnosis of AR, with 65% of respondents stating that their allergies were intermittent in nature. An otolaryngologist or allergist diagnosed the majority of the individuals surveyed. Runny nose, nasal and throat itching, postnasal drip, and nasal congestion or stuffed up nose were the most common and bothersome symptoms of AR. The majority of survey participants (58% of the overall survey population) with AR reported that the condition had an impact on their daily private and professional life. Seventy-two percent of adults reported that their AR symptoms limited their work/school activities and 35% reported that their AR interfered with and caused them to miss work or school within the past 12 months. One factor, in addition to the outward AR symptoms, that could have contributed to these function impairments may have been sleep disturbances. Although a secondary symptom to AR, sleep disturbances (difficulty getting to sleep, waking up during the night or lack of a good night's sleep) were shown in this survey to be extremely troubling in ∼15% of AR sufferers. In the past year >90% of patients reported taking a medication of any type for their AR, with nearly a 4:1 ratio of patients taking a prescription medication versus an over-the-counter (OTC) medication in the past 4 weeks. Over 75% of survey respondents reported taking an intranasal corticosteroid (INCS) in the last 4 weeks and the satisfaction rate of INCS medications was similar to that reported for OTC medications. The most common reasons cited for dissatisfaction with INCS medications were inadequate effectiveness, bothersome side effects (e.g., unpleasant taste and retrograde drainage into the pharynx), decreased effectiveness with chronic use, and failure to provide 24-hour relief. CONCLUSION: These data show that AR is common in the Middle East region as elsewhere in the world. Many patients with AR in Middle East region suffer from their symptoms (e.g., runny nose, nasal itching, nasal congestion, postnasal drip, and other symptoms) on all or most days during the times of the year that their allergies are worst. These symptoms have been shown to reduce QOL and performance at work/school to a significant degree. Additionally, the survey data underscore a considerable treatment gap with current therapies for AR and that many AR patients still have not found adequate effectiveness with currently available medications. Thus, through identification of disease impact on the Middle East population and highlighting treatment gaps, clinicians in the Middle East may better understand and treat AR, leading to improvements in overall patient satisfaction and QOL.