Psychological Burden Among Patients With Inherited Bleeding Disorders in Madinah Province, Saudi Arabia.

Introduction Hereditary bleeding disorders, such as hemophilia and von Willebrand disease (VWD), result from specific deficiencies or malformations in the coagulation cascade proteins. These disorders can significantly impact both physical and psychological health. Complications such as depression, anxiety, and stress (DAS) can further exacerbate these impacts. Despite their significance, detailed prevalence data remain limited, especially for regions such as Madinah province in Saudi Arabia. This study aimed to determine the prevalence of DAS and their associated risk factors among patients with hereditary bleeding disorders in Madinah province, Saudi Arabia. Methods We conducted a cross-sectional study using telephonic interviews involving patients diagnosed with severe hemophilia A or B or VWD attending a hematology clinic in Madinah. Patients over 10 were included, and the study excluded those with central nervous system insults and platelet count concerns. The validated and reliable Depression Anxiety Stress Scale-21-item questionnaire and Statistical Product and Service Solutions (SPSS), version 26.0 (IBM SPSS Statistics for Windows, Armonk, NY), facilitated data collection and analysis, respectively. Results Of the 44 patients studied, 25% exhibited symptoms of depression, 45.5% showed signs of anxiety, and 29.5% had stress symptoms. Regarding symptom severity, 9.1% of patients experienced extremely severe depression, 15.9% had moderate anxiety, and 13.6% reported moderate stress. The prevalence of these psychological issues varied with patients' age and economic status. Notably, a significantly higher rate of depression was observed in patients over 15 years (42.9% vs. 8.7%; p=0.009). Additionally, while not statistically significant, patients with a high economic status reported increased rates of DAS. Conclusions Patients with inherited bleeding disorders, particularly those older than 15, manifest significant psychological distress. There is a pressing need for enhanced awareness, specialized screenings, and tailored counseling services to improve treatment adherence and overall quality of life. Given the findings, a comprehensive national study in Saudi Arabia is highly recommended, alongside the integration of specialized psychological services.

The Clinical Advances of Oncolytic Viruses in Cancer Immunotherapy.

A promising future for oncology treatment has been brought about by the emergence of a novel approach utilizing oncolytic viruses in cancer immunotherapy. Oncolytic viruses are viruses that have been exploited genetically to assault malignant cells and activate a robust immune response. Several techniques have been developed to endow viruses with an oncolytic activity through genetic engineering. For instance, redirection capsid modification, stimulation of anti-neoplastic immune response, and genetically arming viruses with cytokines such as IL-12. Oncolytic viral clinical outcomes are sought after, particularly in more advanced cancers. The effectiveness and safety profile of the oncolytic virus in clinical studies with or without the combination of standard treatment (chemotherapy, radiotherapy, or primary excision) has been assessed using response evaluation criteria in solid tumors (RECIST). This review will comprehensively outline the most recent clinical applications and provide the results from various phases of clinical trials in a variety of cancers in the latest published literature.

A Child With Hereditary Spherocytosis Associated With Von Willebrand's Disease: A Case Report From Saudi Arabia.

Von Willebrand disease (VWD) is an autosomal inherited hemostasis disorder caused by a deficiency or defect in the blood protein known as von Willebrand factor, which is necessary for platelets to adhere to damaged vessel walls. The main symptoms of the condition include spontaneous bleeding from mucosal membranes, excessive wound bleeding, and menorrhagia in girls. On the other hand, hereditary spherocytosis (HS) is a heterogeneous group of diseases that damage red blood cells, with clinical manifestations depending on the different membrane protein-encoding gene mutations, their different functional consequences, and the mechanism of inheritance. It is typically characterized by the presence of jaundice, anemia, and splenomegaly. Here, we report a novel pathogenic mutation in a child with HS that led to hemolytic anemia since the age of two years associated with recently discovered type 1 VWD, as we were unable to find any cases that have been previously reported to have HS associated with VWD. According to our analysis of the literature, there is no definitive link between the two hematological disorders.

The prevalence of self-reported bleeding tendency symptoms among adolescents in Almadinah Almunawwarah, Kingdom of Saudi Arabia.

Bleeding tendency is a common medical problem that is usually caused by either coagulation factors abnormalities, platelets disorders, or vasculopathy. Detection of patients with high bleeding risk through history taking and accurate diagnosis followed by appropriate management will help decreasing chances of significant complications. METHODOLOGY: A cross-sectional survey of a random sample of adolescent students in the preparatory year at Taibah University in Almadinah Almunawwarah, Saudi Arabia were interviewed using a validated semi-structured condensed bleeding tendency questionnaire generated from Molecular and Clinical Markers for the Diagnosis and Management of (MCMDM-1). RESULTS: A total of 680 questionnaires were collected, the phase I data were analyzed to determine participants who gave a positive response to any related question while in Phase II respondents with at least one positive response of any bleeding symptom were interviewed. More than half 52.6% (358 out of 680) were positive for any bleeding symptoms. The incidence rose to 70.9% (482 out of 680) if "Mild" symptoms were also included. Epistaxis was positive in 21.8%, Cutaneous symptoms were reported by 22.65%, Bleeding from the oral cavity was reported in 39.6%, Gastrointestinal bleeding was observed by 5.9%, bleeding after the first surgery reported in 21% and Muscle hematomas/hemoarthrosis was observed by 3.2%, Among female papulation, 16.1% reported having 6-10 "Heavy Days" during their menstrual cycles. DISCUSSION: High prevalence of bleeding symptoms having the possibility of the presence of an Inherited Bleeding Disorders were observed in our study in comparison to other studies, this might be attributed to the high percentage of consanguinity marriages in our area. RECOMMENDATIONS: Raising awareness among family doctors and pediatricians about the possibility of presence of bleeding tendency utilizing good history taking followed by appropriate examination and laboratory confirmatory testing will help in early detection and managements.

Depression, Anxiety, and Stress Symptoms in Patients With Beta Thalassemia Major in Almadinah Almunawwarah, Saudi Arabia.

Introduction Beta thalassemia major (BTM) is a chronic hereditary blood disorder. Patients are dependent on blood transfusion and are prone to multiple comorbidities. Depression, anxiety, and stress (DAS) can complicate their condition. No reports from Saudi Arabia to measure DAS in BTM patients. We report the prevalence of DAS symptoms in our BTM patients using the DASS-21 (Depression, Anxiety, and Stress Scale - 21 Items) test. Methods A cross-sectional study including adolescents and adults aged 14 years and above with BTM treated in Almadinah Almunawwarah and excluding patients who had bone marrow transplant or with central nervous system insults were performed. Results A total of 31 male and 31 female patients were enrolled, with a mean age of 24.32 ± 7.05 years. Depression symptoms were detected in 60 % of patients, anxiety symptoms were detected in half of the studied group, and stress symptoms were detected in 38.7% of patients. We found a significant positive correlation between DAS and DASS total score. Age below or above 18, parent's employment, patient's educational level, and status of satisfaction about medical care were statistically significant in having positive effects on scores of DAS symptoms.  Conclusions BTM patients are prone to develop psychological disorders, which can affect the course of the disease. Our results are comparable to international and Arab population studies, which have the highest reported prevalence. It is important to not ignore the psychological evaluation of patients with BTM and to refer them for proper evaluation and management.

Oral Health Profiles and Related Quality of Life in Thalassemia Children in Relation to Iron Overload: A Cross-Sectional Study.

The aim was to assess the oral health of children with ß-thalassemia major (BTM) and their oral health-related quality of life (OHRQoL) in relation to the serum ferritin level (SFL). Thirty-nine children with BTM underwent an interview, salivary sampling and an oral clinical examination. The Early Childhood Oral Health Impact Scale (ECOHIS) was used to assess their OHRQoL. The mean age of the participants was 9 ± 3 years, with 62% females. The body mass index and salivary secretion rate were within normal ranges. The mean plaque index, gingival bleeding index and number of decayed, missing and filled tooth surfaces were 70 ± 29, 38 ± 25 and 3.2 ± 4, respectively, with no significant differences between individuals with SFL below or above 2000 ng/mL (p > 0.05). No significant differences were observed between the two groups in any of the ECOHIS questions (p > 0.05). The mean ECOHIS score was 4.2 ± 4. Individuals with SFL ≥2000 ng/mL had a significantly higher mean score in the family domain "Parent Distress" than those with lower SFL (p ≤ 0.05). Within the study limits, children with ß-thalassemia major generally had high dental caries experience and gingival inflammation, yet an acceptable OHRQoL. Those with high SFL had less favorable scores in the domain "Parent Distress".

Are we giving proper feedback to medical students? Experience from a Saudi Medical College.

OBJECTIVES: Providing feedback to medical students improves the quality of medical education. However, there is still a paucity of published data on feedback, particularly in the Gulf region. The aim of this study is to assess the feedback given to medical students and examine the obstacles and predictor factors preventing medical staff from providing their students with appropriate feedback. METHODS: This cross-sectional study was conducted at the College of Medicine, Taibah University, KSA. All teaching staff were invited to fill in a predesigned structured questionnaire about socio-demographic characteristics, data related to feedback given to students the previous academic year, and obstacles preventing them from providing appropriate feedback. RESULTS: Of the 110 invitees, 95 faculty members responded (response rate of 86.4%), and 65.3% reported giving feedback (95% CI = 55.8%-74.8%). There were no significant differences in the characteristics of the given feedback when analysed according to staff gender, department, degree, and academic rank. The mean number and duration of the given feedback were significantly higher among professors, followed by assistant then associate professors respectively. Self-confidence, being judgmental, no time to give appropriate feedback (female and clinical staff), teachers' inadequate skills in giving feedback (basic sciences staff), and absence of students' advisorship (male staff) were the most important obstacles to providing feedback. CONCLUSIONS: This study showed that a considerably high proportion of medical staff provide student feedback. There is a need to improve obstacles that potentially prevent giving appropriate feedback to medical students and to provide remediable solutions.

Age-related changes in biochemical bone profile in thalassemic children.

BACKGROUND: Osteopathy is an important cause of morbidity in ß-thalassemia major (TM). Although many of the etiopathological factors implicated in thalassemic osteoporosis commence in early disease phases during childhood, limited information exists on bone turnover in children with TM. This study was conducted with the objective to compare bone turnover markers (BTMs) in thalassemic children at different ages. METHODS: In a cross sectional case control study, 47 children (age range, 1.5-18 years) with TM were recruited. BTMs were compared to eighteen age- and sex-matched healthy controls and to 16 adults (age range, 19.67-31.08 years) with TM. RESULTS: Thalassemic children displayed unbalanced bone turnover with an increased bone resorption (shown by high levels of tartrate-resistant acid phosphatase 5b (TRACP5), receptor activator of nuclear factor-kappa B ligand (sRANKL) and sRANKL/osteoprotegerin (OPG) ratio) and a decreased bone neoformation (shown by low levels of osteocalcin (OC)) when compared to healthy children. TRACP5b was the only BTMs studied that showed a significant correlation with age in thalassemic children. For the whole thalassemic children group, regression analyses showed an influence of sex hormones replacement therapy on TRACP5b; pretransfusion hemoglobin and splenectomy on sRANKL; pretransfusion hemoglobin on sRANKL/OPG; and pretransfusion hemoglobin and serum ferritin on OC. CONCLUSION: The present study confirms that TM has profound effects on bone metabolism starting from early childhood. The early onset of bone turnover disturbances in TM indicates the need to investigate possible option to intervene early.

Pilot trial of a novel two-step therapy protocol using nebulized tranexamic acid and recombinant factor VIIa in children with intractable diffuse alveolar hemorrhage.

BACKGROUND AND OBJECTIVES: Diffuse alveolar hemorrhage (DAH) is a life threatening condition with very limited, often unsuccessful, therapeutic options. This study aimed at exploring the feasibility and efficacy of nebulized tranexamic acid TXA (n-TXA) and nebulized recombinant factor VIIa (n-rFVIIa) when used in a two-step therapy protocol in children with intractable DAH in a pediatric intensive care unit. METHODS: In a prospective trial, n-TXA (250 mg/dose for children < 25 kg and 500 mg/dose for children > 25 kg) was administered to 18 children (median age [interquartile range]; 24.0 months [11.3, 58.5]) with intractable DAH. N-rFVIIa (35 micro g/kg/dose for children < 25 kg, and 50 micro g/kg/dose for children > 25 kg) was added if no or minimal response was seen after 3 to 4 doses (18 to 24 hours) of n-TXA. RESULTS: DAH was stopped in 10 (55.6%) children with n-TXA alone within 24 hours of therapy. Documented concomitant respiratory infection showed a significant negative association with response to n-TXA in a step.wise regression analysis (OR=0.06; 95% CI=0.01-0.74). In the other 8 (44.4%) children, n-rFVIIa was added due to n-TXA failure. Six (75.0%) showed complete cessation of DAH, while two children failed to respond with the addition of n-rFVIIa (25.0%). None of the children who responded to therapy showed recurrence of DAH after therapy termination. No complications related to therapy were recorded. CONCLUSIONS: n-TXA and n-rFVIIa were effective and safe when used in a two-step-therapy protocol to control intractable DAH in pediatric patients in intensive care settings. This therapy modality warrants further exploration through larger multicenter clinical trials.

Comprehensive patient care improves quality of life in transfusion dependent patients with ß-thalassemia.

OBJECTIVES: To investigate levels of quality of life (QOL) among thalassemia patients at the Hereditary Blood Disorders Center in Al-Madinah Al-Munawarah, Kingdom of Saudi Arabia. METHODS: A cross-sectional study was performed on 43 transfusion dependent thalassemia patients compared with 43 normal subjects, as a control, using the World Health Organization Quality Of Life - Brief questionnaire between May 2012 and September 2012 at the Hereditary Blood Disorders Center, Maternity and Children Hospital in Al-Madinah Al-Munawarah, Kingdom of Saudi Arabia. RESULTS: Forty-three thalassemia patients were examined, 23 males and 20 females, and compared with 43 peers (control group), 24 males and 19 females. There was no statistical difference between patients and controls for psychological domains (53.4 versus 56.9, p=0.059) and environmental domains (56.6 versus 57.0, p=0.884). Patients had better social QOL than the control group (39.3 versus 31.7, p=0.003), while the control group had better physical QOL (55.4 versus 61.9, p=0.047). Among patients, there was no statistical difference in QOL domains for variables of age, desferroxamine use, serum ferritin level, disease severity, presence of complications; splenectomy status, hepatitis C virus status, or family history. CONCLUSION: Quality of life in thalassemia patients is similar to the control group particularly social life, though physical health is less. Improvement of patients care from all aspects will improve their QOL. More studies in this field are needed with a bigger sample size.

Histamine H1 antagonists and clinical characteristics of febrile seizures.

BACKGROUND: The purpose of this study was to determine whether seizure susceptibility due to antihistamines is provoked in patients with febrile seizures. METHODS: The current descriptive study was carried out from April 2009 to February 2011 in 250 infants and children who visited the Madinah Maternity and Children's Hospital as a result of febrile convulsions. They were divided into two groups according to administration of antihistamines at the onset of fever. RESULTS: Detailed clinical manifestations were compared between patients with and without administration of antihistamines. The time from fever detection to seizure onset was significantly shorter in the antihistamine group than that in the nonantihistamine group, and the duration of seizures was significantly longer in the antihistamine group than in the nonantihistamine group. No significant difference was found in time from fever detection to seizure onset or seizure duration between patients who received a first-generation antihistamine and those who received a second-generation antihistamine. CONCLUSION: Due to their central nervous system effects, H1 antagonists should not be administered to patients with febrile seizures and epilepsy. Caution should be exercised regarding the use of histamine H1 antagonists in young infants, because these drugs could potentially disturb the anticonvulsive central histaminergic system.