UNASSIGNED: Knowledge of prevalence and epidemiology of cystic fibrosis (CF) and healthcare resource use among Finnish people with CF is incomplete.
UNASSIGNED: We conducted a population-based matched cohort study using retrospective real-world data from linked Finnish national registries. Electronic healthcare data and drug purchases of 102 people with CF were analyzed between January 2015 and December 2019 (follow-up). A 5-fold control population was matched by sex, age, and place of residence. Comorbidities and medication use that occurred at any time during follow-up were assessed; annual rates of hospital service use were adjusted for follow-up.
UNASSIGNED: The prevalence of CF in Finland was 1.85 per 100,000. Median age at diagnosis was 1 year, with 60 % diagnosed at age <2 years and 80 % at age <10 years. Median age at death in people with CF was 31.4 years (n < 5); no controls died. The most common comorbidities included chronic sinusitis (39.2 %), pneumonia (38.2 %), diabetes (20.6 %), and nasal polyps (18.6 %). The most purchased medications were antibiotics (99.0 %) and pancreatic enzymes (84.3 %). The annualized rate of hospital visits was higher in people with CF vs controls (outpatient: mean [SD], 17.4 [14.5] vs 0.9 [3.3]; median, 13.6 vs 0.4, respectively; inpatient: mean [SD], 1.0 [1.66] vs 0.03 [0.14]; median, 0.34 vs 0, respectively).
UNASSIGNED: The prevalence of CF in Finland is remarkably low, likely reflecting unique population characteristics and, in part, delayed diagnosis. Antibiotic use is frequent among Finnish people with CF. Inpatient hospital visits are >30 times higher in people with CF than matched controls.

BACKGROUND: Findings from studies assessing Long Covid in children and young people (CYP) need to be assessed in light of their methodological limitations. For example, if non-response and/or attrition over time systematically differ by sub-groups of CYP, findings could be biased and any generalisation limited. The present study aimed to (i) construct survey weights for the Children and young people with Long Covid (CLoCk) study, and (ii) apply them to published CLoCk findings showing the prevalence of shortness of breath and tiredness increased over time from baseline to 12-months post-baseline in both SARS-CoV-2 Positive and Negative CYP.
METHODS: Logistic regression models were fitted to compute the probability of (i) Responding given envisioned to take part, (ii) Responding timely given responded, and (iii) (Re)infection given timely response. Response, timely response and (re)infection weights were generated as the reciprocal of the corresponding probability, with an overall \'envisioned population\' survey weight derived as the product of these weights. Survey weights were trimmed, and an interactive tool developed to re-calibrate target population survey weights to the general population using data from the 2021 UK Census.
RESULTS: Flexible survey weights for the CLoCk study were successfully developed. In the illustrative example, re-weighted results (when accounting for selection in response, attrition, and (re)infection) were consistent with published findings.
CONCLUSIONS: Flexible survey weights to address potential bias and selection issues were created for and used in the CLoCk study. Previously reported prospective findings from CLoCk are generalisable to the wider population of CYP in England. This study highlights the importance of considering selection into a sample and attrition over time when considering generalisability of findings.

UNASSIGNED: Traumatic brain injury (TBI) is associated with health problems across multiple domains and TBI patients are reported to have high rates of medication use. However, prior evidence is thin due to methodological limitations. Our aim was thus to examine the use of a wide spectrum of medications prescribed to address pain and somatic conditions in a population-based cohort of TBI patients, and to compare this to a sex- and age-matched cohort. We also examined how patient factors such as sex, age, and TBI severity were associated with medication use.
UNASSIGNED: We assessed Swedish nationwide registers to include all individuals treated for TBI in hospitals or specialist outpatient care between 2006 and 2012. We examined dispensed prescriptions for eight different non-psychotropic medication classes for the 12 months before, and 12 months after, the TBI. We applied a fixed-effects model to compare TBI patients with the matched population cohort. We also stratified TBI patients by sex, age, TBI severity and carried out comparisons using a generalized linear model.
UNASSIGNED: We identified 239,425 individuals with an incident TBI and 239,425 matched individuals. TBI patients were more likely to use any medication [Odds ratio (OR) = 2.03, 95% Confidence Interval (CI) = 2.00-2.05], to present with polypharmacy (OR = 1.96, 95% CI = 1.90-2.02), and to use each of the eight medication classes before their TBI, as compared to the matched population cohort. Following the TBI, TBI patients were more likely to use any medication (OR = 1.83, 95% CI = 1.80-1.86), to present with polypharmacy (OR = 1.74, 95% CI = 1.67-1.80), and to use all medication classes, although differences were attenuated. However, differences increased for antibiotics/antivirals (OR = 2.02, 95% CI = 1.99-2.05) and NSAIDs/antirheumatics (OR = 1.62, 95% CI = 1.59-1.65) post-TBI. We also found that females and older patients were more likely to use medications after their TBI than males and younger patients, respectively. Patients with more severe TBIs demonstrated increased use of antibiotics/ antivirals and NSAIDs/antirheumatics than those with less severe TBIs.
UNASSIGNED: Taken together, our results point to poor overall health in TBI patients, suggesting that medical follow-up should be routine, particularly in females with TBI, and include a review of medication use to address potential polypharmacy.

BACKGROUND: Although some systemic infections are associated with Parkinson\'s disease (PD), the relationship between herpes zoster (HZ) and PD is unclear.
OBJECTIVE: The objective is to investigate whether HZ is associated with incident PD risk in a matched cohort study using data from the US Department of Veterans Affairs.
METHODS: We compared the risk of PD between individuals with incident HZ matched to up to five individuals without a history of HZ using Cox proportional hazards regression. In sensitivity analyses, we excluded early outcomes.
RESULTS: Among 198,099 individuals with HZ and 976,660 matched individuals without HZ (median age 67.0 years (interquartile range [IQR 61.4-75.7]); 94% male; median follow-up 4.2 years [IQR 1.9-6.6]), HZ was not associated with an increased risk of incident PD overall (adjusted HR 0.95, 95% CI 0.90-1.01) or in any sensitivity analyses.
CONCLUSIONS: We found no evidence that HZ was associated with increased risk of incident PD in this cohort. © 2024 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

OBJECTIVE: This study aims to describe cervical cancer during pregnancy (CCP) and investigate factors associated with survival outcomes.
METHODS: This retrospective matched study included CCP patients from May 2007 to August 2021 and matched non-pregnant cervical cancer patients (1:2) based on age (±5 years), year at diagnosis (±2 years), histological type and stage (2018 FIGO). The Kaplan-Meier method and multivariate Cox regression analyses were used to assess the impact of pregnancy and clinicopathologic factors on prognosis.
RESULTS: Thirty-eight CCP patients (stage IA to IIIC) and 76 non-pregnant patients were included. Most CCP patients were diagnosed in the first (31.6%) or second (47.4%) trimester. CCP patients had a longer waiting time than non-pregnant patients. Pregnancy continued in 42.1% (continuation of pregnancy [COP] group) and was terminated in 57.9% (termination of pregnancy [TOP] group) of patients. Survival analysis showed no significant differences in recurrence-free survival (RFS) or overall survival (OS) between pregnant and non-pregnant patients or between the COP and TOP groups. At the end of the follow-up period (range 12-178 months), 23 children born to CCP patients exhibited normal development.
CONCLUSIONS: Pregnancy does not impact cervical cancer prognosis. The oncologic outcomes of the TOP and COP groups were comparable. A pregnancy-preserving strategy could be considered for managing CCP patients.

BACKGROUND: Although 99% of children and young people have been exposed to SARS-CoV-2, the long-term prevalence of post-COVID-19 symptoms in young people is unclear. The aim of this study is to describe symptom profiles 12 months after SARS-CoV-2 testing.
METHODS: A matched cohort study of a national sample of 20,202 children and young people who took a SARS-CoV-2 PCR test between September 2020 and March 2021.
RESULTS: 12 months post-index-test, there was a difference in the number of symptoms reported by initial negatives who never tested positive (NN) compared to the other three groups who had at least one positive test (p < 0.001). Similarly, 10.2% of the NN group described five-plus symptoms at 12 months compared to 15.9-24.0% in the other three groups who had at least one positive test. The most common symptoms were tiredness, sleeping difficulties, shortness of breath, and headaches for all four groups. For all these symptoms, the initial test positives with subsequent reports of re-infection had higher prevalences than other positive groups (p < 0.001). Symptom profiles, mental health, well-being, fatigue, and quality of life did not vary by vaccination status.
CONCLUSIONS: Following the pandemic, many young people, particularly those that have had multiple SARS-CoV-2 positive tests, experience a range of symptoms that warrant consideration and potential investigation and intervention.

The natural history of skeletal complications in achondroplasia (ACH) is well-described. However, it remains unclear how the rates of non-skeletal complications, surgical procedures, healthcare needs and mortality differ between individuals with ACH and the general population. This study aimed to contextualise the extent of these outcomes by comparing event rates across the lifespan, between those with ACH and matched controls in a United Kingdom (UK) population.
This retrospective, matched cohort study used data from national UK databases: the Clinical Practice Research Database (CPRD) GOLD from primary care, the secondary care Hospital Episode Statistics (HES) databases and the Office of National Statistics mortality records. ACH cases were identified using disorder-specific Read Codes or International Classification of Diseases 10th Revision codes. For each ACH case, up to four age- and sex-matched controls (defined as those without evidence of skeletal/growth disorders) were included. Event rates per 100 person-years were calculated for a pre-defined set of complications (informed by reviews of existing ACH literature and discussion with clinical authors), healthcare visits and mortality. Rate ratios (RRs) with 95% confidence intervals (CIs) were used to compare case and control cohorts.
541 ACH cases and 2052 controls were identified for the CPRD cohort; of these, 275 cases and 1064 matched controls had linkage to HES data. Approximately twice as many non-skeletal complications were reported among individuals with ACH versus controls (RR [95% CI] 1.80 [1.59-2.03]). Among ACH cases, a U-shaped distribution of complications was observed across age groups, whereby the highest complication rates occurred at < 11 and > 60 years of age. Individuals with ACH had greater needs for medication, GP referrals to specialist care, medical imaging, surgical procedures and healthcare visits versus controls, as well as a mortality rate of almost twice as high.
Patients with ACH experience high rates of a range of both skeletal and non-skeletal complications across their lifespan. To manage these complications, individuals with ACH have significantly increased healthcare needs compared to the general population. These results underscore the need for more coordinated and multidisciplinary management of people with ACH to improve health outcomes across the lifespan.

It is well-known that the additive hazards model is collapsible, in the sense that when omitting one covariate from a model with two independent covariates, the marginal model is still an additive hazards model with the same regression coefficient or function for the remaining covariate. In contrast, for the proportional hazards model under the same covariate assumption, the marginal model is no longer a proportional hazards model and is not collapsible. These results, however, relate to the model specification and not to the regression parameter estimators. We point out that if covariates in risk sets at all event times are independent then both Cox and Aalen regression estimators are collapsible, in the sense that the parameter estimators in the full and marginal models are consistent for the same value. Vice-versa, if this assumption fails, then the estimates will change systematically both for Cox and Aalen regression. In particular, if the data are generated by an Aalen model with censoring independent of covariates both Cox and Aalen regression is collapsible, but if generated by a proportional hazards model neither estimators are. We will also discuss settings where survival times are generated by proportional hazards models with censoring patterns providing uncorrelated covariates and hence collapsible Cox and Aalen regression estimates. Furthermore, possible consequences for instrumental variable analyses are discussed.

暂无摘要。

UNASSIGNED: The effect of immunomodulatory therapy with tocilizumab for coronavirus disease 2019 (COVID-19) in real-life clinical practice remains controversial.
UNASSIGNED: Single-center retrospective matched cohort analysis including 47 consecutive patients treated with intravenous tocilizumab for severe COVID-19 pneumonia (\"TCZ group\"), matched by age, comorbidities, time from symptoms onset and baseline SpO2/FiO2 ratio with 47 patients receiving standard of care alone (\"SoC group\").
UNASSIGNED: There were no significant differences between the TCZ and SoC groups in the rate of clinical improvement (hospital discharge and/or a decrease of ≥2 points on a six-point ordinal scale) by day 7 (51.1% [24/47] versus 48.9% [23/47]; P-value = 1.000). No differences were observed at day 14 in terms of clinical improvement (72.3% versus 76.6%; P-value = 0.791), all-cause mortality (10.6% versus 12.8%; P-value = 1.000), and the composite of invasive mechanical ventilation and/or death (25.5% versus 23.4%; P-value = 1.000) either. Patients in the TCZ group had a more rapid normalization of C-reactive protein levels.
UNASSIGNED: No apparent benefit was observed in patients with severe COVID-19 treated with tocilizumab as compared to a matched retrospective cohort.
UNASSIGNED: El efecto del tratamiento inmunomodulador con tocilizumab en la COVID-19 sigue siendo controvertido.
UNASSIGNED: Estudio unicéntrico de cohortes retrospectivas pareadas que incluyó a 47 pacientes con COVID-19 grave tratados con tocilizumab intravenoso («grupo TCZ»), emparejados por edad, comorbilidades mayores, evolución de síntomas y cociente SpO2/FiO2 basal con 47 pacientes que recibieron tratamiento estándar únicamente («grupo SoC»).
UNASSIGNED: No observamos diferencias significativas entre los grupos de TCZ y SoC en la tasa de mejoría clínica (alta hospitalaria y/o descenso de ≥ 2 puntos en una escala ordinal de 6 puntos) al día 7 (51,1% [24/47] vs. 48,9% [23/47]; P = 1,000). Tampoco hubo diferencias al día 14 en las tasas de mejoría clínica (72,3% vs. 76,6%; P = 0,791), mortalidad (10,6% vs. 12,8%; P = 1,000) o en el compuesto de ventilación mecánica invasiva y/o muerte (25,5% vs. 23,4%; P = 1,000). Los pacientes en el grupo de TCZ presentaron una normalización más rápida de la proteína C reactiva.
UNASSIGNED: Respecto a una cohorte retrospectiva pareada, no detectamos un beneficio asociado al tratamiento con tocilizumab en pacientes con neumonía por COVID-19.