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Nevin Manimala Statistics

Late presentation of chronic myeloid leukaemia patients in a low-income country: the prognostic implications and impact on treatment outcome

BMC Res Notes. 2024 Sep 3;17(1):245. doi: 10.1186/s13104-024-06910-9.

ABSTRACT

BACKGROUND: In Nigeria, since 2002, Imatinib mesylate (glivec®) has been available freely to chronic myeloid leukaemia (CML) patients but only at a tertiary health care centre in the southwestern part of the country. Despite this, it is not readily accessible to many patients due to the distance and other challenges including low socioeconomic status and political problems, preventing timely access to specialist care. This study evaluated the effect of the baseline characteristics on the prognostic implication and treatment outcome of CML patients in Nigeria.

METHOD: This study retrospectively evaluated the baseline characteristics, clinical presentations and treatment outcomes of 889 CML patients over 18 years (2002-2020). Of these, 576 (65%) patients had complete information with up-to-date BCR::ABL1 records. These 576 patients were categorized based on their responses to Imatinib therapy into three groups viz.; Optimal response (OR) defined as BCR::ABL1 ratio of < 0.1% or major molecular remission (≥ 3-log reduction of BCR::ABL1 mRNA or BCR::ABL1 ratio of < 0.1% on the International Scale), Suboptimal response (SR) with BCR::ABL ratio of 0.1-1%, and Treatment failure (TF) when MMR has not been achieved at 12 months. The variables were analyzed using descriptive and inferential statistics and a p-value < 0.05 was considered statistically significant.

RESULTS: The result revealed a median age of 37 years at diagnosis with a male-to-female ratio of 1.5:1. The majority (96.8%) of the patients presented with one or more symptoms at diagnosis with a mean symptom duration of 12 ± 10.6 months. The mean Sokal and EUTOS scores were 1.3 ± 0.8 and 73.90 ± 49.09 respectively. About half of the patients presented with high-risk Sokal (49%) and EUTOS (47%) scores. Interestingly, both the Sokal (r = 0.733, p = 0.011) and EUTOS (r = 0.102, p = 0.003) scores correlated positively and significantly with the duration of symptoms at presentation. Based on response categorization, 40.3% had OR while 27.1% and 32.6% had SR and TF respectively.

CONCLUSION: This study observed a low optimal response rate of 40.3% and treatment failure rate of 32.6% in our CML cohort while on first-line Imatinib therapy. This treatment response is strongly attributable to the long duration of symptoms of 12 months or more and high Sokal and EUTOS scores at presentation. We advocate prompt and improved access to specialist care with optimization of tyrosine kinase inhibitor therapy in Nigeria.

PMID:39227850 | DOI:10.1186/s13104-024-06910-9

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Association between systemic lupus erythematosus and autoimmune thyroid dysfunction in pediatric population: a single center experience

Ital J Pediatr. 2024 Sep 4;50(1):163. doi: 10.1186/s13052-024-01728-4.

ABSTRACT

BACKGROUND: Systemic Lupus Erythematosus (SLE) patients are more likely than the general population to suffer from thyroid illness. The major goal was to assess the thyroid dysfunctions due to immunological factors in Egyptian SLE children and how they are related to the course and severity of the illness.

METHODS: Fifty children and adolescents with SLE are included in this cross-sectional observational study. Every patient underwent a thorough physical examination and a comprehensive history taking. An enzyme-linked immunosorbent assay (ELISA) approach was used to evaluate the thyroid profile, anti-thyroglobulin (Anti-TG), and anti-thyroid peroxidase (anti-TPO) antibodies.

RESULTS: Of the 50 patients, the female: male ratio (F: M = 7:1) was 44 females and 6 males (12%). They were between the ages of 5 and 17. Out of the patients, thirty-two (64%) had thyroid dysfunctions, 19 (38%) had euthyroid sick syndrome, ten (20%) had overt hypothyroidism, three (6%) had subclinical hypothyroidism, and none had hyperthyroidism. Of the 50 patients, one (2%) had increased anti-TPO, whereas all other patients had normal anti-TG levels. A statistically significant negative correlation (p-value 0.007) was seen between the disease duration and free thyroxine (FT4). Furthermore, a significant negative correlation (p-values 0.015 and 0.028) was found when comparing the disease duration with thyroid antibodies (anti-TG and anti-TPO).

CONCLUSION: In Juvenile Systemic Lupus Erythematosus (JSLE), thyroid dysfunctions can be identified. The disease duration but not its activity was significantly correlated with thyroid antibodies. For children with JSLE, thyroid function testing should be done on a regular basis. It is preferable to carry out additional thyroid antibody tests when necessary.

PMID:39227847 | DOI:10.1186/s13052-024-01728-4

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Factors Associated With Successful Withdrawal of Biologic Agents in Children With Colchicine-Resistant Familial Mediterranean Fever

J Clin Rheumatol. 2024 Sep 3. doi: 10.1097/RHU.0000000000002118. Online ahead of print.

ABSTRACT

BACKGROUND: Familial Mediterranean fever (FMF) is a monogenic autoinflammatory disease, and colchicine is the mainstay of treatment. Approximately 5%-10% of patients may respond inadequately to colchicine, and anti-interleukin-1 (anti-IL-1) agents are important treatment options in these patients. The aim of this study was to see whether there is any factor associated with the withdrawal of these anti-IL-1 agents and to investigate the characteristics of colchicine-resistant FMF patients who needed biological therapy.

METHODS: Demographic, clinical characteristics, and disease severity of patients, at 2 referral centers, between 2012 and 2022, in whom anti-IL-1 treatment was continued and discontinued, were compared in this study. The international severity scoring system for FMF (ISSF) was used for disease severity assessment.

RESULTS: In 64 colchicine-resistant FMF patients, the median (interquartile range) duration of biological treatment was 39 (45) months. Treatment of 26 patients (40.6%) was started with anakinra and 38 (59.4%) with canakinumab. During follow-up, anti-IL-1 treatment was discontinued in 23 patients (35.9%). High ISSF scores before biological treatment, presence of exertional leg pain, subclinical inflammation, and comorbidity were found to be statistically more frequent in the group whose biological therapy could not be discontinued (p = 0.009, p = 0.006, p = 0.026, p = 0.001, respectively).

CONCLUSIONS: Low ISSF scores before biological treatment with no accompanying exertional leg pain, subclinical inflammation, and comorbidities may be stated as an associated factors in terms of the discontinuation of biological agents in colchicine-resistant pediatric FMF patients.

PMID:39226611 | DOI:10.1097/RHU.0000000000002118

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Clinical prediction rules for surgical site infection after minor surgery in general practice

Aust J Gen Pract. 2024 Sep;53(9):640-646. doi: 10.31128/AJGP-08-23-6925.

ABSTRACT

BACKGROUND AND OBJECTIVES: Surgical site infection (SSI) after dermatological surgery is associated with poor outcomes. Developing clinical prediction rules based on the predicted probability of infection might encourage guided prophylaxis and judicious prescribing. The purpose of this study was to develop a clinical prediction rule based on identified risk factors for SSI in a large general practice patient cohort.

METHOD: We examined a large, pooled dataset from four randomised controlled trials performed in a regional centre of North Queensland, Australia. Multivariable logistic regression identified a prediction model. Bootstrapping was used for internal validation. A scoring system was based on predicted probabilities of infection.

RESULTS: The final prediction rule included age >55 years and the anatomical site, histology and complexity of the excision. The area under the curve was 0.704.

DISCUSSION: Our prediction rule encourages judicious use of prophylaxis in clinical practice.

PMID:39226598 | DOI:10.31128/AJGP-08-23-6925

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Melanoma overdiagnosis: What do we know and what do we do?

Aust J Gen Pract. 2024 Sep;53(9):612-616. doi: 10.31128/AJGP-02-24-7176.

ABSTRACT

BACKGROUND: Melanoma overdiagnosis occurs when melanomas, not destined to cause morbidity or death in a patient’s lifetime, are identified and treated.

OBJECTIVE: This study considers the causes and magnitude of melanoma overdiagnosis in Australia. We also speculate about a possible benefit of overdiagnosis in Australia; namely, a reduction in excess deaths in the geographical areas where melanoma is diagnosed most frequently.

DISCUSSION: Overdiagnosis can arguably be mitigated by factors that reduce the number of lesions treated for each melanoma identified. Data from the Australian Cancer Atlas show that there is a reduction in excess deaths from melanoma in geographical areas where diagnostic rates are higher (Pearson correlation coefficient r=-0.5978, 95% CI: -0.6243 to -0.5699, P<0.0001); this being the strongest inverse correlation observed among the 20 cancer types in the Atlas. Is early diagnosis of actual life-threatening melanomas in these geographical regions impacting survival? Further research is planned.

PMID:39226593 | DOI:10.31128/AJGP-02-24-7176

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Strongyloides stercoralis in Low-Income Immigrants from Portuguese Speaking African Countries in Lisbon, Portugal

Acta Med Port. 2024 Sep 2;37(9):668-669. doi: 10.20344/amp.21782. Epub 2024 Sep 2.

NO ABSTRACT

PMID:39226557 | DOI:10.20344/amp.21782

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Promoting Collaborative Scholarship During the COVID-19 Pandemic Through an Innovative COVID-19 Data Explorer and Repository at Yale School of Medicine: Development and Usability Study

JMIR Form Res. 2024 Sep 3;8:e52120. doi: 10.2196/52120.

ABSTRACT

BACKGROUND: The COVID-19 pandemic sparked a surge of research publications spanning epidemiology, basic science, and clinical science. Thanks to the digital revolution, large data sets are now accessible, which also enables real-time epidemic tracking. However, despite this, academic faculty and their trainees have been struggling to access comprehensive clinical data. To tackle this issue, we have devised a clinical data repository that streamlines research processes and promotes interdisciplinary collaboration.

OBJECTIVE: This study aimed to present an easily accessible up-to-date database that promotes access to local COVID-19 clinical data, thereby increasing efficiency, streamlining, and democratizing the research enterprise. By providing a robust database, a broad range of researchers (faculty and trainees) and clinicians from different areas of medicine are encouraged to explore and collaborate on novel clinically relevant research questions.

METHODS: A research platform, called the Yale Department of Medicine COVID-19 Explorer and Repository (DOM-CovX), was constructed to house cleaned, highly granular, deidentified, and continually updated data from over 18,000 patients hospitalized with COVID-19 from January 2020 to January 2023, across the Yale New Haven Health System. Data across several key domains were extracted including demographics, past medical history, laboratory values during hospitalization, vital signs, medications, imaging, procedures, and outcomes. Given the time-varying nature of several data domains, summary statistics were constructed to limit the computational size of the database and provide a reasonable data file that the broader research community could use for basic statistical analyses. The initiative also included a front-end user interface, the DOM-CovX Explorer, for simple data visualization of aggregate data. The detailed clinical data sets were made available for researchers after a review board process.

RESULTS: As of January 2023, the DOM-CovX Explorer has received 38 requests from different groups of scientists at Yale and the repository has expanded research capability to a diverse group of stakeholders including clinical and research-based faculty and trainees within 15 different surgical and nonsurgical specialties. A dedicated DOM-CovX team guides access and use of the database, which has enhanced interdepartmental collaborations, resulting in the publication of 16 peer-reviewed papers, 2 projects available in preprint servers, and 8 presentations in scientific conferences. Currently, the DOM-CovX Explorer continues to expand and improve its interface. The repository includes up to 3997 variables across 7 different clinical domains, with continued growth in response to researchers’ requests and data availability.

CONCLUSIONS: The DOM-CovX Data Explorer and Repository is a user-friendly tool for analyzing data and accessing a consistently updated, standardized, and large-scale database. Its innovative approach fosters collaboration, diversity of scholarly pursuits, and expands medical education. In addition, it can be applied to other diseases beyond COVID-19.

PMID:39226547 | DOI:10.2196/52120

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Evaluation of a Digital Previsit Tool for Identifying Stroke-Related Health Problems Before a Follow-Up Visit (Part 1): Survey Study

JMIR Hum Factors. 2024 Sep 3;11:e55852. doi: 10.2196/55852.

ABSTRACT

BACKGROUND: Stroke may lead to various disabilities, and a structured follow-up visit is strongly recommended within a few months after an event. To facilitate this visit, the digital previsit tool “Strokehealth” was developed for patients to fill out in advance. The concept Strokehälsa (or Strokehealth) was initially developed in-house as a Windows application, later incorporated in 1177.se.

OBJECTIVE: The study’s primary objective was to use a patient satisfaction survey to evaluate the digital previsit tool Strokehealth when used before a follow-up visit, with a focus on feasibility and relevance from the perspective of people with stroke. Our secondary objective was to explore the extent to which the previsit tool identified stroke-related health problems.

METHODS: Between November 2020 and June 2021, a web-based survey was sent to patients who were scheduled for a follow-up visit after discharge from a stroke unit and had recently filled in the previsit tool. The survey covered demographic characteristics, internet habits, and satisfaction rated using 5 response options. Descriptive statistics were used to present data from both the previsit tool and the survey. We also compared the characteristics of those who completed the previsit tool and those who did not, using nonparametric statistics. Free-text responses were thematically analyzed.

RESULTS: All patients filling out the previsit tool (80/171; age: median 67, range 32-91 years) were community-dwelling. Most had experienced a mild stroke and reported a median of 2 stroke-related health problems (range 0-8), and they were significantly younger than nonresponders (P<.001). The survey evaluating the previsit tool was completed by 73% (58/80; 39 men). The majority (48/58, 83%) reported using the internet daily. Most respondents (56/58, 97%) were either satisfied (n=15) or very satisfied (n=41) with how well the previsit tool captured their health problems. The highest level of dissatisfaction was related to the response options in Strokehealth (n=5). Based on the free-text answers to the survey, we developed 4 themes. First, Strokehealth was perceived to provide a structure that ensured that issues would be emphasized and considered. Second, user-friendliness and accessibility were viewed as acceptable, although respondents suggested improvements. Third, participants raised awareness about being approached digitally for communication and highlighted the importance of how to be approached. Fourth, their experiences with Strokehealth were influenced by their perceptions of the explanatory texts, the response options, and the possibility of elaborating on their answers in free text.

CONCLUSIONS: People with stroke considered the freely available previsit tool Strokehealth feasible for preparing in advance for a follow-up visit. Despite high satisfaction with how well the tool captured their health problems, participants indicated that additional free-text responses and revised information could enhance usability. Improvements need to be considered in parallel with qualitative data to ensure that the tool meets patient needs.

TRIAL REGISTRATION: Researchweb 275135; https://www.researchweb.org/is/vgr/project/275135.

PMID:39226546 | DOI:10.2196/55852

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Do 3-dimensional Spinopelvic Characteristics Normalize After THA? A Prospective, Comparative Study Using Motion Capture Analysis

Clin Orthop Relat Res. 2024 Sep 1;482(9):1642-1655. doi: 10.1097/CORR.0000000000003106. Epub 2024 May 14.

ABSTRACT

BACKGROUND: Spinopelvic stiffness (primarily in the sagittal plane) has been identified as a factor associated with inferior patient-reported outcomes (PROs) and increased dislocation risk after THA. Incorporating preoperative spinopelvic characteristics into surgical planning has been suggested to determine a patient-specific cup orientation that minimizes dislocation risk. Sagittal plane radiographic analysis of static postures indicates that patients exhibit a degree of normalization in their spinopelvic characteristics after THA. It is not yet known whether normalization is also evident during dynamic movement patterns, nor whether it occurs in the coronal and axial planes as well.

QUESTIONS/PURPOSES: (1) Does motion capture analysis of sagittal spinopelvic motion provide evidence of normalization after THA? (2) Do changes in coronal and axial plane motion accompany those in the sagittal plane?

METHODS: Between April 2019 and February 2020, 25 patients agreed to undergo motion capture movement analysis before THA for the treatment of hip osteoarthritis (OA). Of those, 20 underwent the same assessment between 8 and 31 months after THA. Five patients were excluded because of revision surgery (n = 1), contralateral hip OA (n = 1), and technical issues with a force plate during post-THA assessment (n = 3), leaving a cohort total of 15 (median age [IQR] 65 years [10]; seven male and eight female patients). A convenience sample of nine asymptomatic volunteers, who were free of hip and spinal pathology, was also assessed (median age 51 years [34]; four male and five female patients). Although the patients in the control group were younger than those in the patient group, this set a high bar for our threshold of spinopelvic normalization, reducing the possibility of false positive results. Three-dimensional motion capture was performed to measure spinal, pelvic, and hip motion while participants completed three tasks: seated bend and reach, seated trunk rotation, and gait on a level surface. ROM during each task was assessed and compared between pre- and post-THA conditions and between patients and controls. Statistical parametric mapping (SPM) was used to assess the timing of differences in motion during gait, and spatiotemporal gait parameters were also measured.

RESULTS: After THA, patients demonstrated improvements in sagittal spinal (median [IQR] 32° [18°] versus 41° [14°]; difference of medians 9°; p = 0.004), pelvis (25° [21°] versus 30° [8°]; difference of medians 5°; p = 0.02), and hip ROM (21° [18°] versus 27° [10°]; difference of medians 6°; p = 0.02) during seated bend and reach as well in sagittal hip ROM during gait (30° [11°] versus 44° [7°]; difference of medians 14°; p < 0.001) compared with their pre-THA results, and they showed a high degree of normalization overall. These sagittal plane changes were accompanied by post-THA increases in coronal hip ROM (12° [9°] versus 18° [8°]; difference of medians 6°; p = 0.01) during seated trunk rotation, by both coronal (6° [4°] versus 9° [3°]; difference of medians 3°; p = 0.01) and axial (10° [8°] versus 16° [7°]; difference of medians 6°; p = 0.003) spinal ROM, as well as coronal (8° [3°] versus 13° [4°]; difference of medians 5°; p < 0.001) and axial hip ROM (21° [11°] versus 34° [24°]; difference of medians 13°; p = 0.01) during gait compared with before THA. The SPM analysis showed these improvements occurred during the late swing and early stance phases of gait.

CONCLUSION: When restricted preoperatively, spinopelvic characteristics during daily tasks show normalization after THA, concurring with previous radiographic findings in the sagittal plane. Thus, spinopelvic characteristics change dynamically, and incorporating them into surgical planning would require predictive models on post-THA improvements to be of use.

LEVEL OF EVIDENCE: Level II, prognostic study.

PMID:39226524 | DOI:10.1097/CORR.0000000000003106

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Predictors of Care Home Admission and Survival Rate in Patients With Syndromes Associated With Frontotemporal Lobar Degeneration in Europe

Neurology. 2024 Oct 8;103(7):e209793. doi: 10.1212/WNL.0000000000209793. Epub 2024 Sep 3.

ABSTRACT

BACKGROUND AND OBJECTIVES: Data on care home admission and survival rates of patients with syndromes associated with frontotemporal lobar degeneration (FTLD) are limited. However, their estimation is essential to plan trials and assess the efficacy of intervention. Population-based registers provide unique samples for this estimate. The aim of this study was to assess care home admission rate, survival rate, and their predictors in incident patients with FTLD-associated syndromes from the European FRONTIERS register-based study.

METHODS: We conducted a prospective longitudinal multinational observational registry study, considering incident patients with FTLD-associated syndromes diagnosed between June 1, 2018, and May 31, 2019, and followed for up to 5 years till May 31, 2023. We enrolled patients fulfilling diagnosis of the behavioral variant frontotemporal dementia (bvFTD), primary progressive aphasia (PPA), progressive supranuclear palsy (PSP) or corticobasal syndrome (CBS), and FTD with motor neuron disease (FTD-MND). Kaplan-Meier analysis and Cox multivariable regression models were used to assess care home admission and survival rates. The survival probability score (SPS) was computed based on independent predictors of survivorship.

RESULTS: A total of 266 incident patients with FTLD were included (mean age ± SD = 66.7 ± 9.0; female = 41.4%). The median care home admission rate was 97 months (95% CIs 86-98) from disease onset and 57 months (95% CIs 56-58) from diagnosis. The median survival was 90 months (95% CIs 77-97) from disease onset and 49 months (95% CIs 44-58) from diagnosis. Survival from diagnosis was shorter in FTD-MND (hazard ratio [HR] 4.59, 95% CIs 2.49-8.76, p < 0.001) and PSP/CBS (HR 1.56, 95% CIs 1.01-2.42, p = 0.044) compared with bvFTD; no differences between PPA and bvFTD were found. The SPS proved high accuracy in predicting 1-year survival probability (area under the receiver operating characteristic curve = 0.789, 95% CIs 0.69-0.87), when defined by age, European area of residency, extrapyramidal symptoms, and MND at diagnosis.

DISCUSSION: In FTLD-associated syndromes, survival rates differ according to clinical features and geography. The SPS was able to predict prognosis at individual patient level with an accuracy of ∼80% and may help to improve patient stratification in clinical trials. Future confirmatory studies considering different populations are needed.

PMID:39226519 | DOI:10.1212/WNL.0000000000209793