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

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

Dataset for: Mixed-effects models for slope-based endpoints in clinical trials of chronic kidney disease

In March of 2018, the National Kidney Foundation, in collaboration with the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), sponsored a workshop in which surrogate endpoints other than currently established event-time endpoints for clinical trials in chronic kidney disease (CKD) were presented and discussed. One such endpoint is a slope-based parameter describing the rate of decline in the estimated glomerular filtration rate (eGFR) over time. There are a number of challenges that can complicate such slope-based analyses in CKD trials. These include the possibility of an early but short-term acute treatment effect on the slope, both within-subject and between-subject heteroscedasticity, and informative censoring resulting from patient dropout due to death or onset of end-stage kidney disease (ESKD). To address these issues, we first consider a class of mixed-effects models for eGFR that are linear in the parameters describing the mean eGFR trajectory but which are intrinsically nonlinear when a power-of-mean (POM) variance structure is used to model within-subject heteroscedasticity. We then combine the model for eGFR with a model for time to dropout to form a class of shared parameter (SP) models which, under the right specification of shared random effects, can minimize bias due to informative censoring. The models and methods of analysis are described and illustrated using data from two CKD studies one of which was one of 56 studies made available to the workshop analytical team. Lastly, methodology and accompanying software for prospectively determining sample size/power estimates are presented