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1.
Indian J Tuberc ; 71(2): 185-194, 2024 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-38589123

RESUMO

Tuberculous pericarditis (TBP) is a relatively uncommon but potentially fatal extrapulmonary manifestation of tuberculosis. Despite its severity, there is no universally accepted gold standard diagnostic test for TBP currently. The objective of this study is to compare the diagnostic accuracy of the most commonly used tests in terms of specificity, sensitivity, negative predictive value (NPV), and positive predictive value (PPV), and provide a summary of their diagnostic accuracies. A comprehensive literature review was performed using Scopus, MEDLINE, and Cochrane central register of controlled trials, encompassing studies published from start to April 2022. Studies that compared Interferon Gamma Release Assay (IGRA), Xpert MTB/RIF, Adenosine Deaminase levels (ADA), and Smear Microscopy (SM) were included in the analysis. Bayesian random-effects model was used for statistical analysis and mean and standard deviation (SD) with 95% confidence intervals were calculated using the absolute risk (AR) and odds ratio (OR). Rank probability and heterogeneity were determined using risk difference and Cochran Q test, respectively. Sensitivity and specificity were evaluated using true negative, true positive, false positive, and false negative rates. Area under the receiver operating characteristic (AUROC) was calculated for mean and standard error. A total of seven studies comprising 16 arms and 618 patients were included in the analysis. IGRA exhibited the highest mean (SD) sensitivity of 0.934 (0.049), with a high rank probability of 87.5% for being the best diagnostic test, and the AUROC was found to be 94.8 (0.36). On the other hand, SM demonstrated the highest mean (SD) specificity of 0.999 (0.011), with a rank probability of 99.5%, but a leave-one-out analysis excluding SM studies revealed that Xpert MTB/RIF ranked highest for specificity, with a mean (SD) of 0.962 (0.064). The diagnostic tests compared in our study exhibited similar high NPV, while ADA was found to have the lowest PPV among the evaluated methods. Further research, including comparative studies, should be conducted using a standardized cutoff value for both ADA levels and IGRA to mitigate the risk of threshold effect and minimize bias and heterogeneity in data analysis.


Assuntos
Mycobacterium tuberculosis , Pericardite Tuberculosa , Tuberculose , Humanos , Pericardite Tuberculosa/diagnóstico , Metanálise em Rede , Teorema de Bayes , Tuberculose/diagnóstico , Sensibilidade e Especificidade
2.
Cureus ; 15(9): e45680, 2023 Sep.
Artigo em Inglês | MEDLINE | ID: mdl-37868481

RESUMO

Background Patients on hemodialysis (HD) are most likely to contract hepatitis C (HCV) infection, which is associated with significant morbidity and disease progression. Direct-acting antivirals (DAAs) are safe and tolerable in chronic kidney disease (CKD) with a 90-100% cure rate, and limited data exist regarding their efficacy in end-stage renal disease (ESRD), particularly for HD patients in South Asia. The study aimed to assess the outcome of a 12-week sofosbuvir (SOF) and velpatasvir (VEL) treatment regimen on ESRD patients with chronic HCV infection undergoing HD in the Pakistani Asian population. Methodology This prospective cohort study was conducted between January 2022 and January 2023 at the outpatient nephrology and gastroenterology clinic of Sheikh Zayed Medical College and Hospital, Rahim Yar Khan, Pakistan. This study included a total of 220 ESRD patients fulfilling the inclusion criteria, aged 20-55 years, who had been undergoing weekly HD sessions for at least two years, with acquired HCV infection. Data on demographic and clinical characteristics were collected through patient interviews. Laboratory and dialysis profiling was executed to assess ESRD and discover the underlying cause by ultrasound abdomen, blood pressure measurement by sphygmomanometer, random blood sugar for diabetes, and taking note of the duration and frequency of dialysis. HCV RNA PCR was done at selected intervals to evaluate the virological response to treatment. Sustained virological response (SVR), liver cirrhosis status, and number of weekly HD sessions were compared at one year of SOF/VEL regimen. Results The mean age of patients with ESRD was 41.8 with a standard deviation (SD) of 9.3 years, and HCV diagnosis was 1.3 years with SD of 0.4 years; 52.7% (n=116) were males, 47.3% (n=104) were females, 75% (n=165) were urban dwellers, and 93.6% (n=206) were married. CKD that requires dialysis was caused mainly by hypertension (78, 35%), diabetes mellitus type 2 (52, 24%), bilateral small kidney disease (40, 18%), and others (34, 16%). One hundred and six (48.2%) received dialysis thrice weekly, 83 (37.7%) twice, and 31 (14.1%) once weekly. The study monitored the rapid virological response (RVR) at four weeks of SOF/VEL regimen in 89.5% of ESRD patients, observed end-of-treatment response (ETR) at 12 weeks in 93.2%, and noted 91.4% SVR response at one year. Only four (1.8%) relapses were observed in the study, which was statistically insignificant. The status of liver cirrhosis showed a 50% improvement, decreasing from 40% to 20%. The frequency of weekly HD sessions decreased from thrice to twice-thrice a week. Conclusion The prevalence of contracting HCV is high among CKD and dialysis ESRD patients. All-oral DAA therapy has revolutionized HCV treatment with co-morbidities. Renal functions improved after the SOF/VEL regimen for chronic HCV infection in ESRD patients undergoing HD, with the number of weekly dialysis sessions reduced and SVR reaching 91.4%. Thus, a single-tablet, pan-genotypic DAA regimen of SOF/VEL for 12 weeks is safe, effective, and tolerable regardless of the underlying etiology of ESRD, complications of cirrhosis, HCV genotype, or previous treatment exposure. The successful treatment of HCV and achieving SVR lowers the risk of ESRD complications, improves extra-hepatic manifestations, and greatly enhances survival. Further studies are warranted after the availability of other DAAs to confirm findings with no limitations.

3.
Cureus ; 15(8): e43190, 2023 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-37692611

RESUMO

Background Microalbuminuria (MA) is an important clinical marker for the early detection of kidney damage in patients with type 2 diabetes (T2DM). Urine albumin-to-creatinine ratio (ACR), also known as urine microalbumin, is a sign of diabetic nephropathy (DN), which is a prevalent complication of diabetes and can result in end-stage renal disease (ESRD) if not managed. The prevalence of MA in T2DM has been steadily increasing worldwide, making it a significant public health concern. The goal of this study was to estimate the prevalence of MA and its relationship to hypertension and other diabetic complications among people with T2DM. Methodology This descriptive cross-sectional study was conducted from February 5, 2022, to February 10, 2023, to analyse data from T2DM patients who visited the outpatient diabetic clinic of Sheikh Zayed Medical College and Hospital, Rahim Yar Khan, Pakistan. This study included a total of 640 patients, aged 35-60 years, who had been diagnosed with T2DM for at least five years and fulfilled the inclusion criteria. Data on demographic and clinical characteristics, blood pressure (BP) measurements, and laboratory investigations were collected. MA was assessed based on the ACR in a spot urine sample of more than 30 mg/l. Blood pressure greater than 140/90 or already taking anti-hypertensives was taken to constitute hypertension. Factors associated with MA like hypertension, gender, mode of diabetes treatment, duration of diabetes, glycosylated haemoglobin (HbA1c), dyslipidemia, and other diabetic complications such as retinopathy and neuropathy were also recorded. Results The prevalence of MA in this study of T2DM patients study was 39.1%. The mean age of the participants with MA was 53.9 with a standard deviation (SD) of 6.1 years, and the mean duration of diabetes was 10.1 years (SD 6.2 years); 101 (33.4%) males (n=302) and 103 (30.5%) females (n=338) had MA.  There was a statistically significant correlation between MA > 30mg/d and hypertension (p = <0.001), diabetes duration since diagnosis (p=0.04), HbA1C level (p = <0.001), dyslipidemia (p=0.001), therapy type (p = <0.001), triglyceridemia (p = 0.03), history of diabetes retinopathy (p= <0.002), and peripheral neuropathy (p= <0.001). However, there was no statistically significant correlation between MA and age (p = 0.56), female gender (p = 0.08), low- and high-density lipids, or statin use (p = 0.06). Conclusion The prevalence of microalbuminuria among T2DM patients is significantly high (39.1%) and is positively correlated with various factors such as male gender, hypertension, suboptimal control of diabetes mellitus, high HbA1c levels, longer disease duration, dyslipidemia with high triglycerides, treatment modalities of T2DM, and other diabetic complications like neuropathy and retinopathy. As diabetes is very prevalent in our country, the number of patients with diabetic kidney disease will rise significantly in the near future, leading to ESRD and other diabetic complications, and immediate intervention is needed to prevent this. Further research is warranted to explore potential interventions and evaluate their impact on patient outcomes.

4.
J Orthop ; 44: 5-11, 2023 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-37637498

RESUMO

Purpose: Supracondylar humeral fractures (SCHFs) rank among the frequently observed fractures in children. Nonetheless, there exists a dearth of consensus regarding the optimal surgical approach. This meta-analysis aims to thoroughly evaluate and compare two distinct pinning techniques (cross pinning versus lateral pinning) for SCHFs, using data from Randomized controlled trials (RCTs). Methods: Literature review was done using PubMed, CINAHL, Scopus, and The Cochrane Library for RCTs comparing the two pinning methods and providing information on at least one of the following: Loss of Baumann's angle, loss of carrying angle, elbow function assessed based on Flynn criteria, pin tract infection, and iatrogenic ulnar nerve injury. Random effect model was used to calculate standardized mean difference or Odds Ratio (OR) for the outcomes. Review Manager 5.4.1. was used to perform quality assessment and statistical analysis. Results: A total of 22 RCTs were included. 20 studies reported data for iatrogenic ulnar nerve injury, the OR was calculated to be 3.76 (95% CI 1.75-8.06), showing a significantly lower risk of surgical ulnar nerve injury with the lateral technique. However, no significant difference was found between the pinning techniques in regard to the other outcomes. Conclusion: In comparison to lateral pinning, the utilization of cross pinning technique exposes the patient to a heightened susceptibility of iatrogenic nerve injury. Therefore, it is recommended that surgeons prioritize the implementation of the lateral pinning technique whenever feasible, as it offers greater protection against iatrogenic ulnar nerve injury. For the other intraoperative and postoperative outcomes, both surgical techniques yield comparable results.

5.
Cureus ; 15(5): e39076, 2023 May.
Artigo em Inglês | MEDLINE | ID: mdl-37323312

RESUMO

Background The chronic macro and microvascular complications of diabetes mellitus pose serious health challenges. Metabolic syndrome (MetSy) is characterized by central obesity, glucose intolerance, hyperinsulinemia, low high-density lipoproteins (HDLs), high triglycerides (TGs), and hypertension. MetSy precedes or accompanies diabetes, and it has been linked to an increased risk of cardiovascular disease and premature death. This study aimed to estimate prevalence, identify risk factors, and evaluate associated microvascular complications among MetSy patients with type 2 diabetes mellitus (T2DM). Methodology Over the period of March 20, 2022, to March 31, 2023, a prospective cohort study was conducted at the Outdoor Clinic and Medicine Department of Sheikh Zayed Hospital, Rahim Yar Khan. Based on the International Diabetes Federation MetSy criteria, a total of 160 patients fulfilling the inclusion criteria were selected. A special proforma was used to obtain sociodemographic, clinical, and laboratory variables of MetSy in diabetic participants. Blood pressure and anthropometric measurements such as waist circumference (WC) and body mass index (BMI) were measured. Fasting venous blood was collected to analyze biochemical variables such as fasting blood sugar (FBS), TG, and high-density lipoprotein-cholesterol (HDL-C). The microvascular complications of T2DM were established using fundus ophthalmoscopy and neurological and kidney function assessments with the help of laboratory tests. These variables were matched between MetSy and no MetSy groups along with the presence or absence of diabetes microvascular complications. This information was analyzed based on these assessments and patient interviews. Results Of the 160 T2DM patients, the mean age was 52 years with a predominance of females (51.8%) in the 50-59-year age group (56.8%). The average BMI for females was 29.38 ± 0.54 kg/m², and 32 (20%) had obesity. Females exhibited a large WC of 93.52 ± 1.58 cm, and 48 of 83 females had reported diabetes microvascular complications. A significant p-value was observed for hypertension, high TG, low HDL-C, large WC, obesity, BMI, age, and female gender on comparing diabetics with metabolic syndrome (MetSy+) and those without metabolic syndrome (MetSy-). The prevalence of microvascular complications in T2DM patients with MetSy+ was 52.5% compared with 47.5% in MetSy-. The prevalence of diabetic retinopathy was 24.9% (95% confidence interval (CI) = 20.3%-29.6%), nephropathy was 16.8% (95% CI = 12.8%-20.7%), and neuropathy was 10.8% (95% CI = 7.4%-13.3%). Conclusions The prevalence of MetSy observed among T2DM patients was 65%, with married obese females in the 50-59-year age group being more likely to be affected than males. Hypertension, poor glycemic control, high TG, low HDL-C, and greater anthropometric waist measurements and BMI were additional risk factors that tended to increase the MetSy burden in T2DM. Diabetic retinopathy, nephropathy, and neuropathy were the most prevalent microvascular complications of diabetes, and immediate attention is needed to stop their detrimental effects. Longer uncontrolled diabetes, increasing age, and hypertension were independent predictors of microvascular complications. To further reduce the risks of complications that threaten healthy aging and prognosis for these patients, MetSy screening, health education, and better diabetic management are crucial.

6.
Cureus ; 15(3): e35787, 2023 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-37025723

RESUMO

Background The symptoms of gastroparesis, such as bloating, postprandial fullness, early satiety, nausea, and abdominal discomfort, progressively worsen the quality of life of the affected individuals. The diagnosis is established on the assessment of gastric function that confirms delayed gastric emptying in the absence of structural etiologies. This study aimed to detect gastroparesis-related clinical symptoms early in patients with type 2 diabetes mellitus (T2DM), investigate the concomitant risk factors, and evaluate the prevalence. Methodology This study was conducted at the Department of Medicine and Diabetes Outdoor Clinic of Sheikh Zayed Hospital, Rahim Yar Khan from February 13, 2022, to February 11, 2023. The study involved 175 patients with T2DM who reported gastroparesis-related symptoms. The demographic and clinical characteristics, symptom severity, complications, related risk factors, duration of disease, medications, body mass index (BMI), fasting plasma glucose, and glycated hemoglobin (HbA1C) levels were assessed. The severity of diabetic gastroparesis was established using the disease-specific Patient Assessment of Gastrointestinal Disorders-Symptom Severity Index (PAGI-SYM) and the Gastroparesis Cardinal Symptom Index (GCSI). The five-point scale of the PAGI-SYM and the four-degree severity scores of GCSI were assessed. Neuropathy disability scores and motor evacuation functions were analyzed. Data were analyzed from these questionnaires, special proforma, and patient interviews. Results The clinical features of diabetic gastroparesis were observed in 44% of T2DM patients with mild-grade gastroparesis in 38 (21.7%), moderate in 30 (17.1%), and severe-grade gastroparesis-related symptoms in nine (5.2%) patients. The main manifestations were early satiety (45.1%), stomach fullness (44.5%), bloating (38.3%), and nausea (33.1%). Diabetic gastroparesis symptoms were considerably linked to disease duration of more than 10 years (p = 0.02), high HbA1c (p = 0.001), increased fasting blood glucose (p = 0.003), polyneuropathy, cigarette smoking, and history of comorbid conditions (p = 0.009). Obesity and the female gender were the forecasters of the manifestation of at least one cardinal gastroparesis symptom. Conclusions Gastric emptying is significant in the pathogenesis of gastroparesis-related symptoms. Disease duration of more than 10 years, poor glycemic control with hyperglycemia, high HbA1C, polyneuropathy, and cigarette smoking must be considered as predictors for early detection and risk factors for the advancement of gastroparesis in T2DM. Gastroparesis-related common symptoms of early satiety, bloating, and stomach fullness were considerably linked to the additional risk factors of hypercholesteremia, chronic microvascular complications, concomitant cardiovascular diseases, and a positive family history of diabetes mellitus. There was no relationship between BMI, age, types of treatment, and the degree of gastroparesis severity. The prevalence and severity of gastroparesis symptoms were particularly high among obese females with poor glycemic control and longer disease duration.

7.
Cureus ; 14(9): e29133, 2022 Sep.
Artigo em Inglês | MEDLINE | ID: mdl-36258948

RESUMO

BACKGROUND: Systemic poisoning with paraphenylenediamine (PPD) also known as Kalapathar, is an emerging suicidal trend in developing south Asian and African countries. The clinical distinction of hair dye toxicity comprises severe angioedema of the face and neck, tongue swelling resulting in upper airway obstruction, acute liver injury, myocarditis, and rhabdomyolysis complicating to acute kidney injury. AIM: To raise awareness, document the characteristic clinical spectra, and prevent and predict the outcome of poisoning (suicidal or accidental) with PPD (hair-dye) based on clinical complications, early baseline laboratory results and creatine phosphokinase (CPK) levels monitoring. PLACE AND DURATION: Department of Medicine and Emergency, Sheikh Zayed Hospital, Rahim Yar Khan. One year of study from August 19, 2021 to August 17, 2022. METHODS: A total of 103 patients, with no comorbidities, who presented with acute PPD poisoning were included in this study. The demographic profile, clinical features, laboratory results, route and mode of intoxication were noted in a special proforma. Furthermore, clinical outcomes in the form of need for tracheostomy or mechanical ventilation, mortality and mean hospital stay were also documented. The percentages were calculated for categorical data like demographic profile, clinical features and clinical outcomes. Mean and standard deviation was calculated for continuous variables, i.e., laboratory parameters. RESULTS: Out of 103 patients, 88 (85.4%) were females who belonged to a low socioeconomic class (89%). The mean age of the patients was 26.39 ± 3.41 years. The majority of cases were suicidal self-poisoning (98%), and the route was oral (98%). In 82 (79.6%) of the patients, the characteristic cervicofacial angioedema, dysphagia, dysphonia, tongue swelling and stridor were noted. Clinical complications such as rhabdomyolysis (67.9%), chocolate-colored urine (82.5%), hepatitis (58.2%), and acute kidney injury (22.3%) were noted in the later clinical stages of PPD poisoning. Emergency tracheostomy was performed in 77.6% of patients. The mortality rate in this study stands at 12.6% and the mean hospital stay at 6.25 ±3.99 days. The mean and standard deviation of serum creatinine, CPK, alanine aminotransferase (ALT), aspartate aminotransferase (AST), total leukocyte count (TLC), and serum potassium were, respectively, noted at 2.431 ± 2.275 mg/dL, 1090.8 ± 218.6 IU/L, 476.8 ± 1038.8 IU/L, 639.1 ± 1006, 11100 ± 4124.1 cells/mm3, and 4.8 ± 1.061 mmol/L. CONCLUSION: PPD is emerging as the poison of choice in suicidal young female patients due to its easy, low-cost availability and higher mortality. The cervicofacial angioedema, tongue swelling and rhabdomyolysis impending acute kidney injury are hallmarks of PPD poisoning. The treatment is largely supportive with no specific antidote available. Early clinical diagnosis and supportive therapeutic management in the form of maintenance of airway patency, timely tracheostomy with post-operative tube care, intravenous medications (fluids, steroids, antihistamine), and renal dialysis can save lives and may lead to full recovery. In addition, strict legal measures should be endorsed to ban the sale and use of lethal PPD in hair dyes.

8.
J Ayub Med Coll Abbottabad ; 34(2): 360-363, 2022.
Artigo em Inglês | MEDLINE | ID: mdl-35576303

RESUMO

The workup of corona virus disease (COVID-19) involves analyzing samples for acute or past presence of SARS-CoV-2 (virus). A detection of 2019 novel Corona virus (2019-nCov) by real-time reverse transcriptase polymerase chain reaction (RT-PCR) indicates current infection and positive IgG antibody level implies a prior infection. Imaging techniques like high resolution computed tomography (HRCT) chest and Xray chest helps in diagnosing and monitoring the disease. Most cases of 2019-nCov are mild and range from asymptomatic carriers to critical illness leading to acute respiratory distress, septic shock and multiorgan failure. We report two cases of COVID-19 who manifested with high grade fever, myalgias, cough and shortness of breath on minimal exertion. All baseline laboratory findings were normal. Initial RT-PCR was negative for oropharyngeal and nasopharyngeal swabs. CT Chest showing typical peripheral patchy and ground glass opacities bilaterally, other markers of infectivity followed by antibody titer confirms the disease.


Assuntos
COVID-19 , COVID-19/diagnóstico , Diagnóstico Precoce , Humanos , Reação em Cadeia da Polimerase Via Transcriptase Reversa , SARS-CoV-2 , Tomografia Computadorizada por Raios X/métodos
9.
Orphanet J Rare Dis ; 16(1): 521, 2021 12 20.
Artigo em Inglês | MEDLINE | ID: mdl-34930388

RESUMO

BACKGROUND: Real-world studies of the burden of severe haemophilia B in the context of recent therapeutic advances such as extended half-life (EHL) factor IX (FIX) products are limited. We analysed data from the recent CHESS II study to better understand the clinical, humanistic, and economic burden of severe haemophilia B in Europe. Data from male adults with severe haemophilia B receiving prophylaxis were analysed from the retrospective cross-sectional CHESS II study conducted in Germany, France, Italy, Spain and the United Kingdom. Inhibitors were exclusionary. Patients and physicians completed questionnaires on bleeding, joint status, quality of life, and haemophilia-related direct and indirect costs (2019-2020). All outcomes were summarised using descriptive statistics. RESULTS: A total of 75 CHESS II patients were eligible and included; 40 patients (53%) provided self-reported outcomes. Mean age was 36.2 years. Approximately half the patients were receiving EHL versus standard half-life (SHL) prophylaxis (44% vs 56%). Most patients reported mild or moderate chronic pain (76%) and had ≥ 2 bleeding events per year (70%), with a mean annualised bleed rate of 2.4. Mean annual total haemophilia-related direct medical cost per patient was €235,723, driven by FIX costs (€232,328 overall, n = 40; €186,528 for SHL, €290,620 for EHL). Mean annual indirect costs (€8,973) were driven by early retirement or work stoppage due to haemophilia. Mean quality of life (EQ-5D) score was 0.67. CONCLUSIONS: These data document a substantial, persistent real-world burden of severe haemophilia B in Europe. Unmet needs persist for these patients, their caregivers, and society.


Assuntos
Fator IX , Hemofilia B , Adulto , Efeitos Psicossociais da Doença , Estudos Transversais , Europa (Continente) , Fator IX/uso terapêutico , Estresse Financeiro , Hemofilia B/prevenção & controle , Humanos , Masculino , Qualidade de Vida , Estudos Retrospectivos
10.
Artigo em Inglês | MEDLINE | ID: mdl-34234912

RESUMO

Bernard Soulier Syndrome is a genetically inherited platelet disorder that commonly presents with symptoms of impaired blood coagulation, such as epistaxis, menorrhagia, and petechiae formation. Here we present a case of Bernard Soulier Syndrome in which the individual has presented with melena, which is the appearance of black tarry stools due to bleeding from the upper gastrointestinal tract. This presentation is rare and should be discussed so that appearance of the less common symptoms can be caught early, leading to an early diagnosis and consequently earlier and more effective management options.

11.
Orphanet J Rare Dis ; 16(1): 143, 2021 03 20.
Artigo em Inglês | MEDLINE | ID: mdl-33743752

RESUMO

BACKGROUND: Hemophilia B is a rare congenital bleeding disorder that has a significant negative impact on patients' functionality and health-related quality of life. The standard of care for severe hemophilia B in the United States is prophylactic factor IX replacement therapy, which incurs substantial costs for this lifelong condition. Accurate estimates of the burden of hemophilia B are important for population health management and policy decisions, but have only recently accounted for current management strategies. The 'Cost of Severe Hemophilia across the US: a Socioeconomic Survey' (CHESS US) is a cross-sectional database of medical record abstractions and physician-reported information, completed by hematologists and care providers. CHESS US+ is a complementary database of completed questionnaires from patients with hemophilia. Together, CHESS US and CHESS US+ provide contemporary, comprehensive information on the burden of severe hemophilia from the provider and patient perspectives. We used the CHESS US and CHESS US+ data to analyze the clinical, humanistic, and economic burden of hemophilia B for patients treated with factor IX prophylaxis between 2017 and 2019 in the US. RESULTS: We conducted analysis to assess clinical burden and direct medical costs from 44 patient records in CHESS US, and of direct non-medical costs, indirect costs, and humanistic burden (using the EQ-5D-5L) from 57 patients in CHESS US+. The mean annual bleed rate was 1.73 (standard deviation, 1.39); approximately 9% of patients experienced a bleed-related hospitalization during the 12-month study period. Nearly all patients (85%) reported chronic pain, and the mean EQ-5D-5L utility value was 0.76 (0.24). The mean annual direct medical cost was $614,886, driven by factor IX treatment (mean annual cost, $611,971). Subgroup analyses showed mean annual costs of $397,491 and $788,491 for standard and extended half-life factor IX treatment, respectively. The mean annual non-medical direct costs and indirect costs of hemophilia B were $2,371 and $6,931. CONCLUSIONS: This analysis of patient records and patient-reported outcomes from CHESS US and CHESS US+ provides updated information on the considerable clinical, humanistic, and economic burden of hemophilia B in the US. Substantial unmet needs remain to improve patient care with sustainable population health strategies.


Assuntos
Hemofilia A , Hemofilia B , Efeitos Psicossociais da Doença , Estudos Transversais , Custos de Cuidados de Saúde , Humanos , Qualidade de Vida , Estados Unidos
12.
Haemophilia ; 26(4): 711-717, 2020 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-32268007

RESUMO

INTRODUCTION: The benefits of physical activity (PA) for people with haemophilia (PWH) may include improvements in joint, bone and muscle health. However, the factor VIII activity level required to avoid a bleeding episode associated with PA is unknown. AIM: To elicit the opinion of clinical experts on the minimum level and ideal factor VIII activity ('level') required to avoid a bleeding episode during participation in different types of PA for PWH. METHODS: Based on the 2017 National Hemophilia Foundation PA descriptions, clinical experts estimated a minimally acceptable and an ideal factor level at which a bleed could be avoided. The uncertainty around estimates was quantified using an approach to construct a probability distribution to represent expert opinion. RESULTS: Minimum and ideal factor level increased with higher risk PA, whether or not joint morbidity was present, as did the experts' uncertainty in their estimates (ie the range between lowest and highest estimates for minimum and ideal levels). Mean minimum levels ranged from 4% to 48% for low to high risk for people without joint morbidity, and from 7% to 47% for those with joint morbidity. For ideal factor levels, corresponding figures were 9%-52% and 12%-64%, respectively. CONCLUSION: To support a patient-centric outcome, expert opinion indicates that the clinical norm of 0.01 IU/mL (1%) trough level is insufficient. It is anticipated that introducing a more targeted approach to meet the needs of patients who are increasingly physically active will benefit patients further in addition to recent treatment advances.


Assuntos
Exercício Físico/fisiologia , Hemartrose/prevenção & controle , Hemofilia A/terapia , Hemorragia/prevenção & controle , Adolescente , Adulto , Idoso , Criança , Pré-Escolar , Conferências de Consenso como Assunto , Fator VIII/análise , Hemartrose/diagnóstico , Hemartrose/etiologia , Hemofilia A/sangue , Hemofilia A/complicações , Hemorragia/etiologia , Humanos , Lactente , Artropatias/sangue , Artropatias/diagnóstico , Artropatias/patologia , Pessoa de Meia-Idade , Medição de Risco , Adulto Jovem
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