A Case of Asymptomatic Essential Thrombocythemia in a Child.

Various factors can be linked to an increase in platelet count. Yet thrombocytosis could be essential. Many genetic mutations have been associated with essential thrombocytosis, which also increases the possibility of myelofibrotic transformation. In pediatrics, essential thrombocytosis is not well-studied. In this article, we present a rare case of a 42-month-old male patient who presented with essential thrombocytosis associated with myeloproliferative leukemia (MPL) gene mutation.

Efficacy of Different Doses of Daprodustat for Anemic Non-dialysis Patients with Chronic Kidney Disease: A Systematic Review and Network Meta-Analysis.

(1) Background: Anemia affects about 40% of patients with chronic kidney disease (CKD). Daprodustat improves serum hemoglobin in anemic patients by inhibiting prolyl hydroxylase of hypoxia-inducible factor. We conducted a network meta-analysis to investigate the direct and indirect effects of different doses of daprodustat compared to each other and erythropoietin and placebo. (2) Methods: We searched PubMed, Cochrane Library, Web of Science, and Scopus, for randomized clinical trials (RCTs) reporting data about different doses of daprodustat for anemia in nondialysis of CKDs. (3) Results: We eventually included five RCTs with a total sample size of 4566 patients. We found that the higher the dose of daprodustat, the greater the change in serum total iron binding capacity (TIBC), hemoglobin, and ferritin from baseline. Compared to placebo, daprodustat 25-30 mg was associated with the highest significant increase in serum hemoglobin (MD = 3.27, 95% CI = [1.89; 4.65]), a decrease in serum ferritin (MD = -241.77, 95% CI = [-365.45; -118.09]) and increase in serum TIBC (MD = 18.52, 95% CI = [12.17; 24.87]). (4) Conclusion: Higher daprodustat doses were associated with a higher impact on efficacy outcomes as serum total iron-binding capacity (TIBC), hemoglobin, and ferritin. However, data about the safety profile of different doses of daprodustat is still missing.

Clinical features and therapeutic outcomes of patients with acromegaly in Saudi Arabia: a retrospective analysis.

BACKGROUND: Acromegaly is a rare disease resulting in clinical sequelae with significant morbidity and mortality due to the central tumor mass effect and prolonged growth hormone (GH) hypersecretion. OBJECTIVES: The goal is to describe the epidemiology, clinical features, presence of comorbidities, and treatment outcomes of acromegaly in Saudi Arabia. METHODS: Data was collected through a retrospective review of the charts of all patients diagnosed with acromegaly from nine major hospitals in Saudi Arabia over a period of more than 25 years. RESULTS: A total of 195 patients (116 males and 79 females), with a mean age at diagnosis of 43 ± 12 (males) and 46 ± 14 years (females), from nine major hospitals were identified and included in the analysis. All cases were caused by pituitary adenomas, of which 92.4% were macroadenomas. Headache, coarse facial features, acral growth, and sweating/oily skin were by far the most frequent presenting complaints. The most common comorbidities were diabetes mellitus (51.7%), followed by hypertension (50%) and visual field defect (30.5%). The vast majority (95%) of patients were treated surgically (98%). Twenty-four percent also received radiotherapy, and 74.4% received medical therapy. When stringent criteria were applied for assessment of outcomes of therapy, 28.7% of the patients were cured and 30.1% had their disease under control, while 28.7% were found to have active disease despite receiving multimodal therapy. CONCLUSIONS: Our findings highlight the need for a national acromegaly registry to enable early identification, evaluation, and selection of the best therapeutic approaches to improve the outcome and remission rate of the disease.

Addressing Cancer Treatment Shortages in Saudi Arabia: Results of a National Survey and Expert Panel Recommendations.

PURPOSE: Cancer treatment shortages are complex and a persistent problem worldwide. Patients with cancer are most vulnerable to drug shortages, which provides opportunities to examine the extent of the challenge(s) facing Saudi Arabia and to provide recommendations toward mitigating the impact of cancer treatment shortages on patient outcomes. MATERIALS AND METHODS: A qualitative methodologic approach was conducted in April 2019 using a validated questionnaire and structured panel discussion for data generation. RESULTS: Overall, 55 responses were received from practicing oncology health care professionals (26 pharmacists and 29 physicians). The annual average number of treated patients with cancer per institution was 640 (adults [n = 400] and pediatric [n = 240]). All respondents (100%) reported that cancer treatment shortages constitute a current problem in their center, with an average of 5 (range, 1-9) per month. The panelists recognized 2 fundamental points. First, the definition of cancer drug shortages should be standardized and recognized at the national level. Second, the current system must be improved to ensure proper and efficient use of the current resources. On that basis, the panelists developed 9 recommendations for action. CONCLUSION: Cancer drug shortage is a significant problem in all health centers in Saudi Arabia. This study presents challenges that should be addressed at the national level and essential consensus recommendations for a coordinated action developed by a panel of experts to tackle the current national problem of cancer treatment shortages. Implementing these recommendations will provide a blueprint for management of national drug shortages in general and cancer treatment shortages in particular.

Study on risk factors (predisposing factors) for poor diabetes control during Hajj (1436/2015) in people with diabetes.

BACKGROUND AND OBJECTIVE: Each year millions of Muslims perform pilgrimage to Makkah, Saudi Arabia. It is particularly stressful during the peak five days, when all rituals have to be performed at specific periods of time at different sites. Poor diabetes control in people with diabetes predisposes to morbidity and increases risk of acute complications. We wanted to see how well their blood glucose control was before coming to Hajj and whether they were aware, about self management of Diabetes and what were reasons for hospital admissions. METHOD: We performed an observational prospective study, based on questionnaire. Sixty one patients were enrolled after taking informed consent. Patients included in the study were known or newly diagnosed diabetics who were admitted to KAMC between 1st and 30th Zil'Hajj. RESULTS: Of the total 61 patients, 16 were newly diagnosed, (not known diabetic, before), while 45 were known diabetics. Among known diabetics, about 77% patients had poor diabetes control on admission, 72% did not bring glucometer, about 55% received diabetic education before coming to Makkah; 37% were doing SMBG occasionally and only 22% were aware that more frequent SMBG required during illness. CONCLUSIONS: Most people in our study population suffered from poor glycemic control before coming to Hajj. A significant number were unaware of their diagnosis. The most significant risk factor in our study was a lack of knowledge about self-management of diabetes and Hajj specific management.