Temporal trends and outcome of splenectomy in adults with immune thrombocytopenia in the USA.

Splenectomy is one of the treatments of immune thrombocytopenia (ITP) with a high response rate. However, it is an irreversible procedure that can be associated with morbidity in this setting. Our aim was to study the trends of splenectomy in adults with ITP, and the factors associated with splenectomy and resource utilization during these hospitalizations. We used the National (Nationwide) Inpatient Sample (NIS) to identify hospitalizations for adult patients with a principal diagnosis of ITP between 2007 and 2017. The primary outcome was the splenectomy trend. Secondary outcomes were (1) incidence of ITP trend, (2) in-hospital mortality, length of stay, and total hospitalization costs after splenectomy trend, and (3) independent predictors of splenectomy, length of stay, and total hospitalization costs. A total of 36,141 hospitalizations for ITP were included in the study. The splenectomy rate declined over time (16% in 2007 to 8% in 2017, trend p < 0.01) and so did the in-hospital mortality after splenectomy. Of the independent predictors of splenectomy, the strongest was elective admissions (adjusted odds ratio [aOR]: 22.1, 95% confidence interval [CI]:17.8-27.3, P < 0.01), while recent hospitalization year, older age, and Black (compared to Caucasian) race were associated with lower odds of splenectomy. Splenectomy tends to occur during elective admissions in urban medical centers for patients with private insurance. Despite a stable ITP hospitalization rate over the past decade and despite listing splenectomy as a second-line option for management of ITP in major guidelines, splenectomy rates consistently declined over time.

Assessment of Lebanese healthcare professionals' awareness on acquired haemophilia: a cross-sectional study.

BACKGROUND: Acquired haemophilia A (AHA) is a rare immune-mediated disorder characterised by the development of autoantibodies against factor VIII. Morbidity and mortality are in general high due to multiple factors including the age of the patient, underlying diseases, toxic effects of available treatments and bleeding itself. OBJECTIVE: To assess the awareness about AHA among healthcare professionals (HCPs) in Lebanon where patients can present to non-haematologists with life-threatening bleeding disorders. METHODS: A cross-sectional survey was conducted in September and October 2017 all over Lebanon among HCPs. The survey covered: (i) the geographic area of practice and specialty; (ii) bleeding disorders encountered within the last 2 years; (iii) assessment of knowledge on AHA; and (iv) importance of increasing awareness on AHA among HCPs. RESULTS: A total of 362 participants completed and returned the questionnaire (response rate 100%). The majority of the HCPs were practicing in Beirut (n=164; 45.3%) and were internists (n=106; 29.3%). 332 (93%) HCPs have encountered patients with bleeding problems within the last 2 years all over Lebanon. 327 (92.1%) HCPs agreed that increasing awareness on AHA among health care professionals is important. HCPs gave an average of 75.9% of correct answers on the survey. CONCLUSIONS: Appropriate treatment and diagnosis are essential when dealing with AHA. Knowledge gaps exist in the clinical practice when dealing with patients who have AHA. This study shows the need for increasing awareness about AHA among HCPs in Lebanon regarding the diagnosis and treatment of this disorder.

Overview of risk assessment models for venous thromboembolism in ambulatory patients with cancer.

A B S T R A C T Important progress has been made in the development of risk assessment models (RAM) for the identification of outpatients on anticancer treatment at risk of venous thromboembolism (VTE). Since the breakthrough publication of the original Khorana risk score (KRS) more than 10 years ago, a new generation of KRS-based scores have been developed, including the Vienna Cancer and Thrombosis Study, PROTECHT, CONKO, ONCOTEV, TicOnco and the CATS/MICA score. Among these the CATS/MICA score showed that a simplified score composed of only two calibrated predictors, the type of cancer and the D-dimer levels, offers a user-friendly tool for the evaluation of cancer-associated thrombosis (CAT) risk. The COMPASS-CAT score is the first that introduced a more synthetic approach of risk evaluation by combining cancer-related predictors with patient comorbidity in a score which is designed for the types of cancer frequently seen in the community (i.e. breast, lung colon or ovarian cancers) and has been externally validated in independent studies. The Throly score is registered as part of the same group as it has a similar structure to the COMPASS-CAT score and is applicable in patients with lymphoma. The incorporation of specific biomarkers of hypercoagulability to the RAM for CAT offers the possibility to perform a precision medicine approach in the prevention of CAT. The improvement of RAM for CAT with artificial intelligence methodologies and deep learning techniques is the challenge in the near future.

Preventing thalassemia in Lebanon: successes and challenges in a developing country.

Thalassemia continues to be a major health burden. The chronicity of the disease and the high cost of life-long treatment make prevention strategies crucial in the management of this disease. In this article, we revisit different successful prevention strategies, and underline the Lebanese model. The Chronic Care Center (CCC), Beirut, is the only specialized center in Lebanon for the treatment and prevention of thalassemia. The current number of patients registered up to August 2013 was 724, representing cases from all over Lebanon. In 1994, the center launched a national prevention program following the World Health Organization (WHO) recommendations. The major activities of the program include awareness campaigns, screening for thalassemia carriers in the general population and high risk groups, registry of new cases and follow-up on the mandatory premarital law (established at the same time). Screening programs showed a carrier rate of around 2.3% in the general population, and 4.0-41.0% in high risk groups. The major pitfall in the law is that only persons with a mean corpuscular volume (MCV) of >70.0 fL are asked to perform further hemoglobin (Hb) testing. A significant decrease in the number of new cases of thalassemia patients in Lebanon reflects the efforts deployed in the prevention of the disease. However, some limitations are faced in reaching a complete eradication of the disease, mainly due to the fact that abortion is illegal and due to pitfalls and incorrect implementation of the premarital law.

Pharmacogenomics variation in drug metabolizing enzymes and transporters in relation to docetaxel toxicity in Lebanese breast cancer patients: paving the way for OMICs in low and middle income countries.

We investigated the association of genetic polymorphisms in drug metabolizing enzymes (DMEs) and transporters in patients with docetaxel-induced febrile neutropenia, by a new high-throughput DMEs and transporters (DMETPlus) microarray platform, characterizing 1936 single nucleotide polymorphisms (SNPs) in 225 genes. We recruited 100 Lebanese breast cancer patients from a consecutive cohort of 277 patients who received docetaxel either alone, or in combination with trastuzumab. Out of 100 patients, 18 had developed febrile neutropenia (cases). They were age- and treatment- matched with 18 patients who did not develop febrile neutropenia on docetaxel (controls). We found that 12 SNPs in seven genes (ABCC6, ABCG1, ABCG2, CYP1A2, CYP2D6, FMO2, and FMO3) were significantly associated with febrile neutropenia after docetaxel treatment. Many of these SNPs have not been previously reported to be associated with toxicity due to docetaxel treatment. Interestingly, one SNP in the FMO3 gene (rs909530) was significantly associated with three clinical endpoints: febrile neutropenia, reduced absolute neutrophil count, and hemoglobin reduction. To the best of our knowledge, this is the first study that evaluated the effect of a large array of nearly 2000 polymorphisms in DMEs and transporters on docetaxel toxicity in breast cancer patients, and in a previously understudied population. Additionally, it attests to the feasibility of genomics research in low- and middle-income countries (LMICs). In light of the current global epidemic of noncommunicable diseases (NCDs) such as breast cancer impacting LMICs, we suggest pharmacogenomics is considered as an integral part of the global health research agenda for NCDs and personalized therapeutics.

Iron overload: consequences, assessment, and monitoring.

In patients suffering from transfusion-dependent anemia, excess iron secondary to regular transfusions cannot be physiologically excreted. This leads to a state of chronic iron overload with iron accumulating in the liver, heart, and endocrine organs, and ultimately results in significant morbidity and mortality. Historically, iron overload was assessed through measurement of serum ferritin or direct determination of liver iron concentration (LIC) by means of biopsy. Although both correlate well with iron overload severity, several limitations pertinent to both are of concern. This has led to the identification of novel noninvasive iron assessment measures, namely magnetic resonance imaging (MRI) R2 and T2*. Moreover, investigations of other potential indices like nontransferrin-bound iron (NTBI) and labile plasma iron (LPI) are yielding promising results. Optimal iron overload assessment and monitoring is a key element in the development of improved strategies of iron chelation therapy that can be tailored to meet the patient's specific needs.