Category: Statistics

Acta Orthop Belg. 2024 Mar;90(1):11-15. doi: 10.52628/90.1.12345.

ABSTRACT

The utilization of local infiltration analgesia (LIA) is a common practice in total hip arthroplasty (THA) procedures to mitigate postoperative pain and diminish the necessity for opioids. However, contemporary literature reports conflicting results. Our working hypothesis was that LIA renders better postoperative VAS-scores and reduces the need for oral analgetics. We performed a randomized, double-blind, placebo-controlled trial aimed at examining the effectiveness of LIA in THA. A total of 90 patients were included for statistical analysis. Our primary endpoint was the Visual Analogue Scale, VAS, (0: no pain, 10: unbearable pain) preoperatively, at the 1st, 2nd, 3rd, 4th and 12th hour postoperative intervals and at discharge. Our secondary endpoints included the postoperative opioid consumption, as well as patient satisfaction at 2 and 6 weeks postoperatively, measured using the Numeric Rating Scale, NRS. LIA has a tendency for superior results regarding VAS- Scores at 3 and 4 hours postoperatively. There were no notable statistical distinctions observed in terms of patients necessitating rescue opioid consumption. Patient satisfaction using the NRS at both the 2-week and 6-week postoperatively did not differ significantly between both groups. The administration of LIA could offer advantages during the initial stages of postoperative recovery, which could be particularly valuable in rapid recovery programs.

PMID:38669643 | DOI:10.52628/90.1.12345

Acta Orthop Belg. 2024 Mar;90(1):5-10. doi: 10.52628/90.1.11289.

ABSTRACT

The aim of the study is to determine the revision ratio after implantation of the femoral neck system (FNS) for the treatment of femoral neck fractures. A retrospective single center cohort analysis with a total of 71 patients who underwent the implantation of the FNS between December 2019 and December 2021, was performed. 31 males and 40 females were included. There was no exclusion based on BMI, ASA score, Garden classification or Pauwels classification. Primary outcome was the revision rate after FNS implantation. Secondary outcomes comprise the reason for revision surgery as well as the time toward revision surgery and the 30-day mortality. The revision ratio was 11 out of 71 patients (15.5%) with an average time to revision surgery of 10 months. Most common reason for revision was avascular necrosis (AVN) in 45.5%. Other reasons for revision surgery were implant failure due to a secondary fall on to the hip with the FNS implant in place, cut-out, cut-through and malunion in respectively 27.3%, 9%, 9% and 9% of the revision patients. The one- hole plate was used in 72% of the patients. Mean follow-up was 18.07 months (range 6-30 months). Full weight bearing instruction was given to 85.9% of the patients. Partial weight bearing in 14.1% of the patients. In conclusion, the FNS has similar revision ratio when used for femoral neck fractures compared to cannulated screw fixation in literature. The predominant reason for revision is AVN and implant failure with no difference between the use of the one- or-two-hole plate in this study.

PMID:38669642 | DOI:10.52628/90.1.11289

Blood. 2024 Apr 26:blood.2024023944. doi: 10.1182/blood.2024023944. Online ahead of print.

ABSTRACT

Obinutuzumab (O) and Rituximab (R) are two CD antibodies that have never been compared in a prospective randomised trial in mantle cell lymphoma (MCL). Herein, we report the long-term outcome of the LYMA-101 (NCT02896582) trial, in which newly diagnosed MCL patients were treated with chemotherapy plus O before transplantation followed by O maintenance (O group). We then compared these patients to those treated with the same treatment design with Rituximab instead of O (R group) (NCT00921414). A propensity score matching (PSM) was used to compare the two populations (O vs R groups) in terms of MRD at the end of induction (EOI), PFS and OS. In LYMA-101, the estimated five-year PFS and OS since inclusion (n=85) were 83.4% (95%CI: 73.5-89.8%) and 86.9% (95%CI: 77.6-92.5%), respectively. At EOI, patients treated in the O group had more frequent bone marrow MRD negativity than those treated in the R group (83.1% vs 63.4% Chi2 p=0.007). The PSM resulted in 2 sets of 82 patients with comparable characteristics at inclusion. From treatment initiation, the O group had a longer estimated five-year PFS (p=0.029; 82.8% versus 66.6%, HR 1.99, IC95 1.05-3.76) and OS (p=0.039; 86.4% versus 71.4% (HR 2.08, IC95 1.01-4.16) compared to the R group. Causes of death were comparable in the 2 groups, the most common cause being lymphoma. Obinutuzumab prior to transplantation and in maintenance provides better disease control and enhances PFS and OS, as compared to Rituximab in transplant-eligible MCL patients.

PMID:38669626 | DOI:10.1182/blood.2024023944

J Osteopath Med. 2024 Apr 29. doi: 10.1515/jom-2024-0013. Online ahead of print.

ABSTRACT

CONTEXT: Children and adolescents young adults (AYAs) undergoing treatment for oncologic diagnoses are frequently hospitalized and experience unwanted therapy-induced side effects that diminish quality of life. Osteopathic manipulative treatment (OMT) is a medical intervention that utilizes manual techniques to diagnose and treat body structures. Few studies have investigated the implementation of OMT in the pediatric oncology outpatient setting. To date, no studies have investigated the safety and feasibility of OMT in the pediatric oncology inpatient setting.

OBJECTIVES: The objective of this study is to investigate the safety and feasibility of OMT in the pediatric oncology inpatient setting.

METHODS: This is a prospective, single-institution pilot study evaluating children and AYAs aged ≥2 years to ≤30 years with a diagnosis of cancer hospitalized at Riley Hospital for Children (RH) from September 2022 to July 2023. Approval was obtained from the Indiana University Institutional Review Board (IRB). Patients were evaluated daily with a history and physical examination as part of routine inpatient management. Patients who reported chemotherapy side effects commonly encountered and managed in the inpatient setting, such as pain, headache, neuropathy, constipation, or nausea, were offered OMT. Patients provided written informed consent/assent prior to receiving OMT. OMT was provided by trained osteopathic medical students under the supervision of a board-certified osteopathic physician and included techniques commonly taught in first- and second-year osteopathic medical school curricula. Safety was assessed by a validated pain (FACES) scale immediately pre/post-OMT and by adverse event grading per Common Terminology Criteria for Adverse Events (CTCAE) 24 h post-OMT. All data were summarized utilizing descriptive statistics.

RESULTS: A total of 11 patients were screened for eligibility. All patients met the eligibility criteria and were enrolled in the study. The majority of patients were male (n=7, 63.6 %) with a median age of 18.2 years at time of enrollment (range, 10.2-29.8 years). Patients had a variety of hematologic malignancies including B-cell acute lymphoblastic leukemia (ALL) (n=5, 45.5 %), T-cell ALL (n=1, 9.1 %), acute myeloid leukemia (AML) (n=2, 18.2 %), non-Hodgkin’s lymphoma (n=2, 18.2 %), and Hodgkin’s lymphoma (n=1, 9.1 %). All patients were actively undergoing cancer-directed therapy at the time of enrollment. There were 40 unique reasons for OMT reported and treated across 37 encounters, including musculoskeletal pain (n=23, 57.5 %), edema (n=7, 17.5 %), headache (n=5, 12.5 %), peripheral neuropathy (n=2, 5.0 %), constipation (n=2, 5.0 %), and epigastric pain not otherwise specified (n=1, 2.5 %). Validated FACES pain scores were reported in 27 encounters. Of the 10 encounters for which FACES pain scores were not reported, 8 encounters addressed lower extremity edema, 1 encounter addressed peripheral neuropathy, and 1 encounter addressed constipation. The total time of OMT was documented for 33 of the 37 encounters and averaged 9.8 min (range, 3-20 min).

CONCLUSIONS: Hospitalized children and AYAs with cancer received OMT safely with decreased pain in their reported somatic dysfunction(s). These findings support further investigation into the safety, feasibility, and efficacy of implementing OMT in the pediatric oncology inpatient setting and to a broader inpatient pediatric oncology population.

PMID:38669608 | DOI:10.1515/jom-2024-0013

Biostatistics. 2024 Apr 26:kxae009. doi: 10.1093/biostatistics/kxae009. Online ahead of print.

ABSTRACT

There is an increasing interest in the use of joint models for the analysis of longitudinal and survival data. While random effects models have been extensively studied, these models can be hard to implement and the fixed effect regression parameters must be interpreted conditional on the random effects. Copulas provide a useful alternative framework for joint modeling. One advantage of using copulas is that practitioners can directly specify marginal models for the outcomes of interest. We develop a joint model using a Gaussian copula to characterize the association between multivariate longitudinal and survival outcomes. Rather than using an unstructured correlation matrix in the copula model to characterize dependence structure as is common, we propose a novel decomposition that allows practitioners to impose structure (e.g., auto-regressive) which provides efficiency gains in small to moderate sample sizes and reduces computational complexity. We develop a Markov chain Monte Carlo model fitting procedure for estimation. We illustrate the method’s value using a simulation study and present a real data analysis of longitudinal quality of life and disease-free survival data from an International Breast Cancer Study Group trial.

PMID:38669589 | DOI:10.1093/biostatistics/kxae009

J Parkinsons Dis. 2024;14(3):545-556. doi: 10.3233/JPD-230285.

ABSTRACT

BACKGROUND: REM-sleep behavior disorder (RBD) and other non-motor symptoms such as hyposmia were proposed by the Movement Disorder Society as research criteria for prodromal Parkinson’s disease (P-PD). Global cognitive deficit was later added.

OBJECTIVE: To compare non-motor symptoms, focusing on cognition, between a P-PD group and a matched control group.

METHODS: In this cross-sectional, case-control study, in a first set of analyses, we performed extensive cognitive testing on people with (n = 76) and a control group without (n = 195) probable RBD and hyposmia. Furthermore, we assessed motor and non-motor symptoms related to Parkinson’s Disease (PD). After propensity score matching, we compared 62 P-PD with 62 age- and sex-matched controls. In addition, we performed regression analyses on the total sample (n = 271). In a second set of analyses, we used, a.o., the CUPRO to evaluate retrograde procedural memory and visuo-constructive functions.

RESULTS: People with P-PD showed significantly poorer performances in global cognition, visuo-constructive and executive functions, mainly in mental flexibility (p < 0.001; p = 0.004; p = 0.003), despite similar educational levels (p = 0.415). We observed significantly more motor and non-motor symptoms (p < 0.001; p = 0.004), higher scores for depression (p = 0.004) and apathy (p < 0.001) as well as lower quality of life (p < 0.001) in P-PD.

CONCLUSIONS: Our findings confirm that global cognitive, executive, and visuo-constructive deficits define the P-PD group. In addition, depression, apathy, and lower quality of life were more prevalent in P-PD. If replicated in other samples, executive and visuo-constructive deficits should be considered in non-motor P-PD. Determining specific patterns will support early recognition of PD, secondary prevention of complications and the development of neuroprotective treatments.

PMID:38669560 | DOI:10.3233/JPD-230285

JMIR Mhealth Uhealth. 2024 Apr 26;12:e51201. doi: 10.2196/51201.

ABSTRACT

BACKGROUND: Numerous smartphone apps are targeting physical activity (PA) and healthy eating (HE), but empirical evidence on their effectiveness for the initialization and maintenance of behavior change, especially in children and adolescents, is still limited. Social settings influence individual behavior; therefore, core settings such as the family need to be considered when designing mobile health (mHealth) apps.

OBJECTIVE: The purpose of this study was to evaluate the effectiveness of a theory- and evidence-based mHealth intervention (called SMARTFAMILY [SF]) targeting PA and HE in a collective family-based setting.

METHODS: A smartphone app based on behavior change theories and techniques was developed, implemented, and evaluated with a cluster randomized controlled trial in a collective family setting. Baseline (t₀) and postintervention (t₁) measurements included PA (self-reported and accelerometry) and HE measurements (self-reported fruit and vegetable intake) as primary outcomes. Secondary outcomes (self-reported) were intrinsic motivation, behavior-specific self-efficacy, and the family health climate. Between t₀ and t₁, families of the intervention group (IG) used the SF app individually and collaboratively for 3 consecutive weeks, whereas families in the control group (CG) received no treatment. Four weeks following t₁, a follow-up assessment (t₂) was completed by participants, consisting of all questionnaire items to assess the stability of the intervention effects. Multilevel analyses were implemented in R (R Foundation for Statistical Computing) to acknowledge the hierarchical structure of persons (level 1) clustered in families (level 2).

RESULTS: Overall, 48 families (CG: n=22, 46%, with 68 participants and IG: n=26, 54%, with 88 participants) were recruited for the study. Two families (CG: n=1, 2%, with 4 participants and IG: n=1, 2%, with 4 participants) chose to drop out of the study owing to personal reasons before t₀. Overall, no evidence for meaningful and statistically significant increases in PA and HE levels of the intervention were observed in our physically active study participants (all P>.30).

CONCLUSIONS: Despite incorporating behavior change techniques rooted in family life and psychological theories, the SF intervention did not yield significant increases in PA and HE levels among the participants. The results of the study were mainly limited by the physically active participants and the large age range of children and adolescents. Enhancing intervention effectiveness may involve incorporating health literacy, just-in-time adaptive interventions, and more advanced features in future app development. Further research is needed to better understand intervention engagement and tailor mHealth interventions to individuals for enhanced effectiveness in primary prevention efforts.

TRIAL REGISTRATION: German Clinical Trials Register DRKS00010415; https://drks.de/search/en/trial/DRKS00010415.

INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): RR2-10.2196/20534.

PMID:38669071 | DOI:10.2196/51201

J Child Health Care. 2024 Apr 26:13674935241249770. doi: 10.1177/13674935241249770. Online ahead of print.

ABSTRACT

This pilot and acceptability study sought to report provider acceptability and developmental concerns expressed by caregivers of children with prenatal opioid exposure using the Shared Decision-Making Tool (SDMT), an instrument created by study authors. Data were collected from five health care and early intervention providers and 83 caregivers from a medical clinic and early intervention service center. Descriptive statistics were used to identify frequency, mean level, and prioritization of developmental concerns using the SDMT, and to summarize provider acceptability about integrating the SDMT into their workflow. Communication was the most frequently cited concern in four consecutive age categories, followed by Inattention/impulsivity and Problem behavior. All providers “strongly agreed” or “agreed” with all statements on the provider feedback survey, except two instances. Results of this study support the SDMT as a potential tool to help engage caregivers and providers of children with prenatal opioid exposure in the shared decision-making process by standardizing communication related to areas of developmental concern and caregivers’ priority needs. Findings from this pilot study will inform modifications to the SDMT and administration instructions before our next study, which will examine psychometric properties and caregiver acceptability of the scale.

PMID:38669063 | DOI:10.1177/13674935241249770

JMIR Form Res. 2024 Apr 26;8:e52687. doi: 10.2196/52687.

ABSTRACT

BACKGROUND: Type 2 diabetes disproportionately affects South Asian subgroups. Lifestyle prevention programs help prevent and manage diabetes; however, there is a need to tailor these programs for mobile health (mHealth).

OBJECTIVE: This study examined technology access, current use, and preferences for health communication among South Asian immigrants diagnosed with or at risk for diabetes, overall and by sex. We examined factors associated with interest in receiving diabetes information by (1) text message, (2) online (videos, voice notes, online forums), and (3) none or skipped, adjusting for sociodemographic characteristics and technology access.

METHODS: We used baseline data collected in 2019-2021 from two clinical trials among South Asian immigrants in New York City (NYC), with one trial focused on diabetes prevention and the other focused on diabetes management. Descriptive statistics were used to examine overall and sex-stratified impacts of sociodemographics on technology use. Overall logistic regression was used to examine the preference for diabetes information by text message, online (videos, voice notes, or forums), and no interest/skipped response.

RESULTS: The overall sample (N=816) had a mean age of 51.8 years (SD 11.0), and was mostly female (462/816, 56.6%), married (756/816, 92.6%), with below high school education (476/816, 58.3%) and limited English proficiency (731/816, 89.6%). Most participants had a smartphone (611/816, 74.9%) and reported interest in receiving diabetes information via text message (609/816, 74.6%). Compared to male participants, female participants were significantly less likely to own smartphones (317/462, 68.6% vs 294/354, 83.1%) or use social media apps (Viber: 102/462, 22.1% vs 111/354, 31.4%; WhatsApp: 279/462, 60.4% vs 255/354, 72.0%; Facebook: Messenger 72/462, 15.6% vs 150/354, 42.4%). A preference for receiving diabetes information via text messaging was associated with male sex (adjusted odds ratio [AOR] 1.63, 95% CI 1.01-2.55; P=.04), current unemployment (AOR 1.62, 95% CI 1.03-2.53; P=.04), above high school education (AOR 2.17, 95% CI 1.41-3.32; P<.001), and owning a smart device (AOR 3.35, 95% CI 2.17-5.18; P<.001). A preference for videos, voice notes, or online forums was associated with male sex (AOR 2.38, 95% CI 1.59-3.57; P<.001) and ownership of a smart device (AOR 5.19, 95% CI 2.83-9.51; P<.001). No interest/skipping the question was associated with female sex (AOR 2.66, 95% CI 1.55-4.56; P<.001), high school education or below (AOR 2.02, 95% CI 1.22-3.36; P=.01), not being married (AOR 2.26, 95% CI 1.13-4.52; P=.02), current employment (AOR 1.96, 95% CI 1.18-3.29; P=.01), and not owning a smart device (AOR 2.06, 95% CI 2.06-5.44; P<.001).

CONCLUSIONS: Technology access and social media usage were moderately high in primarily low-income South Asian immigrants in NYC with prediabetes or diabetes. Sex, education, marital status, and employment were associated with interest in mHealth interventions. Additional support to South Asian women may be required when designing and developing mHealth interventions.

TRIAL REGISTRATION: ClinicalTrials.gov NCT03333044; https://classic.clinicaltrials.gov/ct2/show/NCT03333044, ClinicalTrials.gov NCT03188094; https://classic.clinicaltrials.gov/ct2/show/NCT03188094.

INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): RR2-10.1186/s13063-019-3711-y.

PMID:38669062 | DOI:10.2196/52687

JMIR Res Protoc. 2024 Apr 26;13:e54026. doi: 10.2196/54026.

ABSTRACT

BACKGROUND: Preeclampsia (PE) is one of the most common hypertensive diseases, affecting 2%-8% of all pregnancies. The high maternal and fetal mortality rates of PE are due to a lack of early identification of affected pregnant women that would have led to closer monitoring and care. Recent data suggest that misfolded proteins might be a promising biomarker for PE prediction, which can be detected in urine samples of pregnant women according to their congophilia (aggregated) characteristic.

OBJECTIVE: The main purpose of this trial is to evaluate the value of the urine congophilia-based detection of misfolded proteins for the imminent prediction of PE in women presenting with suspected PE. The secondary objectives are to demonstrate that the presence of urine misfolded proteins correlates with PE-related maternal or neonatal adverse outcomes, and to establish an accurate PE prediction model by combining misfolded proteins with multiple indicators.

METHODS: At least 300 pregnant women with clinical suspicion of PE will be enrolled in this prospective cohort study. Participants should meet the following inclusion criteria in addition to a suspicion of PE: ≥18 years old, gestational week between 20+0 and 33+6, and single pregnancy. Consecutive urine samples will be collected, blinded, and tested for misfolded proteins and other PE-related biomarkers at enrollment and at 4 follow-up visits. Clinical assessments of PE status and related complications for all participants will be performed at regular intervals using strict diagnostic criteria. Investigators and participants will remain blinded to the results. Follow-up will be performed until 42 days postpartum. Data from medical records, including maternal and fetal outcomes, will be collected. The performance of urine misfolded proteins alone and combined with other biomarkers or clinical variables for the prediction of PE will be statistically analyzed.

RESULTS: Enrollment started in July 2023 and was still open upon manuscript submission. As of March 2024, a total of 251 eligible women have been enrolled in the study and enrollment is expected to continue until August 2024. Results analysis is scheduled to start after all participants reach the follow-up endpoint and complete clinical data are collected.

CONCLUSIONS: Upon completion of the study, we expect to derive an accurate PE prediction model, which will allow for proactive management of pregnant women with clinical suspicion of PE and possibly reduce the associated adverse pregnancy outcomes. The additional prognostic value of misfolded proteins is also expected to be confirmed.

TRIAL REGISTRATION: Chinese Clinical Trials Registry ChiCTR2300074878; https://www.chictr.org.cn/showproj.html?proj=202096.

INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): PRR1-10.2196/54026.

PMID:38669061 | DOI:10.2196/54026