MaineTrack 2022 Program Report for Tufts University School of Medicine & Maine Medical Center

The Tufts University School of Medicine – Maine Medical Center Maine Track Program was founded with three primary goals in mind: to address the shortage of doctors in Maine; to offer Maine’s brightest students the financial means to pursue a career in medicine; and to develop an innovative curriculum focused on community-based education.https://knowledgeconnection.mainehealth.org/annualreports/1020/thumbnail.jp

A Brief Report of Caregiver Needs and Resource Utilization During Pediatric Hematopoietic Stem Cell Transplantation

Hematopoietic stem cell transplantation (HSCT) is used to eradicate disease and restore normal hematopoietic, immunologic, and/or metabolic functioning. HSCT is a complex treatment that is physiologically and psychologically demanding on the recipient, caregiver, and family. The purpose of this study was to identify needs and resources of family caregivers of pediatric HSCT recipients during the first year after transplant. Parental caregivers (n = 161) completed an online survey. The most cited sources of information were the HSCT team (87.7%), books and other print materials (83.1%), and the Internet (81.5%). However, more than half of the respondents reported that finding resources and services was a problem. More than half identified managing the emotional and social impact of the transplant on their child, posttransplant and follow-up care, practical strategies for caregiving, maintaining the family, and taking care of themselves during this first year as important topics to address. Adequately and regularly assessing caregiver and family needs and providing resources to meet those needs, especially during transitions in care, are important components of transplant care

The role relationship between victim and perpetrator as a predictor of characteristics of intrafamilial sexual abuse

It is hypothesized that the closeness of the relationship between the perpetrator of sexual abuse and the victim will determine the number of instances of sexual abuse, the duration of the sexually abusive relationship, the level of coercion necessary to gain compliance, and how long it takes the victim to tell. Differences for cases where the perpetrator is the victim's father and married to the victim's mother, the victim's stepfather or victim's mother's live-in boyfriend, and the victim's noncustodial father are explored. It is argued that in the first case type, the relationship is the closest, the second case type falls in the middle, and in the third, the relationship is the most distant. Hypotheses regarding number of instances of sexual abuse, its duration, and the delay in telling are supported by the data.Peer Reviewedhttp://deepblue.lib.umich.edu/bitstream/2027.42/44245/1/10560_2004_Article_BF00755849.pd

Estimating individualized treatment effects from randomized controlled trials: a simulation study to compare risk-based approaches

Abstract Background Baseline outcome risk can be an important determinant of absolute treatment benefit and has been used in guidelines for “personalizing” medical decisions. We compared easily applicable risk-based methods for optimal prediction of individualized treatment effects. Methods We simulated RCT data using diverse assumptions for the average treatment effect, a baseline prognostic index of risk, the shape of its interaction with treatment (none, linear, quadratic or non-monotonic), and the magnitude of treatment-related harms (none or constant independent of the prognostic index). We predicted absolute benefit using: models with a constant relative treatment effect; stratification in quarters of the prognostic index; models including a linear interaction of treatment with the prognostic index; models including an interaction of treatment with a restricted cubic spline transformation of the prognostic index; an adaptive approach using Akaike’s Information Criterion. We evaluated predictive performance using root mean squared error and measures of discrimination and calibration for benefit. Results The linear-interaction model displayed optimal or close-to-optimal performance across many simulation scenarios with moderate sample size (N = 4,250; ~ 785 events). The restricted cubic splines model was optimal for strong non-linear deviations from a constant treatment effect, particularly when sample size was larger (N = 17,000). The adaptive approach also required larger sample sizes. These findings were illustrated in the GUSTO-I trial. Conclusions An interaction between baseline risk and treatment assignment should be considered to improve treatment effect predictions

Estimating individualized treatment effects from randomized controlled trials: a simulation study to compare risk-based approaches

Abstract Background Baseline outcome risk can be an important determinant of absolute treatment benefit and has been used in guidelines for “personalizing” medical decisions. We compared easily applicable risk-based methods for optimal prediction of individualized treatment effects. Methods We simulated RCT data using diverse assumptions for the average treatment effect, a baseline prognostic index of risk, the shape of its interaction with treatment (none, linear, quadratic or non-monotonic), and the magnitude of treatment-related harms (none or constant independent of the prognostic index). We predicted absolute benefit using: models with a constant relative treatment effect; stratification in quarters of the prognostic index; models including a linear interaction of treatment with the prognostic index; models including an interaction of treatment with a restricted cubic spline transformation of the prognostic index; an adaptive approach using Akaike’s Information Criterion. We evaluated predictive performance using root mean squared error and measures of discrimination and calibration for benefit. Results The linear-interaction model displayed optimal or close-to-optimal performance across many simulation scenarios with moderate sample size (N = 4,250; ~ 785 events). The restricted cubic splines model was optimal for strong non-linear deviations from a constant treatment effect, particularly when sample size was larger (N = 17,000). The adaptive approach also required larger sample sizes. These findings were illustrated in the GUSTO-I trial. Conclusions An interaction between baseline risk and treatment assignment should be considered to improve treatment effect predictions

PowerPoint Slides for: Rationale and Design of a Clinical Trial Investigating Tolvaptan Safety and Efficacy in Autosomal Dominant Polycystic Kidney Disease

Background: In TEMPO 3:4, the vasopressin V2-receptor antagonist tolvaptan slowed kidney growth and function decline in autosomal dominant polycystic kidney disease (ADPKD) patients with relatively preserved kidney function. Methods: Prospective, phase 3b, multi-center, randomized-withdrawal, placebo-controlled, double-blind trial of tolvaptan in ADPKD patients with late stage 2 to early stage 4 chronic kidney disease (CKD). The primary endpoint was estimated glomerular filtration rate (eGFR) change from pre-treatment baseline to post-treatment follow-up. Secondary endpoints included annualized eGFR slope, incidence of ADPKD complications, and overall and hepatic safety profiles. Participants were 18-55 year-old ADPKD patients with baseline eGFR ≥25 and ≤65 mL/min/1.73 m2 or 56-65 year-old with eGFR ≥25 and ≤44 mL/min/1.73 m2 and evidence of eGFR decline >2.0 mL/min/1.73 m2 per year. Daily split doses of tolvaptan were titrated to tolerance (30/15, 45/15, 60/30, or 90/30 mg) and maintained for 12 months, after an 8-week pre-randomization period to screen out subjects unable to tolerate at least 60/30 mg for 3 weeks. Results: Of 1,495 subjects who entered the tolvaptan titration period, 125 (8.4%) discontinued the study before randomization. One thousand three hundred seventy subjects (684 tolvaptan, 686 placebo) from 213 centers across 21 countries were randomized. Baseline demographics were well balanced across treatment arms. Information collected during the study included eGFR, survey scores (PKD history and outcome), adverse events, vital signs, hematology, urinalysis, and serum chemistry tests. Conclusion: Replicating Evidence of Preserved Renal Function: An Investigation of Tolvaptan Safety and Efficacy (REPRISE) determines whether tolvaptan administered over 1 year exhibits disease-modifying properties in ADPKD patients with late stage 2 to early stage 4 CKD, which provides an important therapeutic advancement for this difficult-to-treat disease