Developing an International Registry for Uterus Transplantation (IRUTx): Promises and Challenges.

Uterus Transplantation (UTx) is an experimental vascular composite allograft designed to provide women with absolute uterine factor infertility with the opportunity to gestate and give birth to their future offspring. The number of UTx procedures performed worldwide now stands at ≥70 and as the number of cases performed increases so too does the volume of potential data that may be gathered to inform the development, practice and regulation of UTx. Given the value of this data, and the challenges associated with keeping track of cases and outcomes where data remains unpublished and/or scattered, scientists and academics conducting research into UTx have increasingly called for the swift creation, implementation and management of an international registry for Uterus Transplantation (IRUTx). This article explores and provides practical guidance regarding the potential benefits the IRUTx may provide to stakeholders, as well as the legal and ethical challenges that its creation may pose in terms of dataset design, consent, privacy, researcher compliance and governance.

The building of an institutional liver transplant registry: opportunities and challenges.

To improve current data systems for institutional decision-making, the Adult Liver Transplant Registry was established at the Hospital Italiano de Buenos Aires, Argentina. This article describes its design and implementation and reports on the outcomes for patients transplanted since its January 2020 launch. A multidisciplinary team designed the registry by identifying key variables from a literature review while considering balance between data depth and feasibility. Rigorous quality control measures were enforced, including monthly audits and staff training. Benchmark indicators for post-transplant outcomes were established. As of November 2023, the registry included 136 transplants. Its implementation and maintenance were straightforward, with no significant difficulties encountered. Cirrhosis was the predominant indication (77%) for transplant. Only one living donor transplantation was performed. Post-transplant results generally aligned with benchmarks, but rates of biliary complications slightly exceeded the recommended thresholds. The one-year post-transplant survival rate was 87%. The successful registry implementation provides a robust framework for research, treatment management, and patient care enhancement within a liver transplant unit.

Smidt Heart Institute Takotsubo Registry - Study design and baseline characteristics.

Background: Takotsubo syndrome (TTS) is an acute form of transient systolic heart failure that occurs predominantly among women and in association with emotional or physical stressors. The Smidt Heart Institute Takotsubo Registry aims to establish a database through an online patient-advocate registry for deep phenotyping of this syndrome. Methods: The Takotsubo Registry is a retrospective and prospective observational registry of individuals with a prior history of TTS. Participants are sourced through physician referrals, medical records review, peer- and self-referrals using social media. Research Electronic Data Capture (REDCap) and Mitra® microsamplers are used to collect questionnaire data and blood samples to facilitate completely remote study enrollment and participation for most participants. Results: From January 2019 to May 2021, 125 participants (99% female, mean age: 61.5 ± 9.9 years) enrolled in the registry across 25 US states and 3 international countries, with reported first TTS event a median of 2 years prior to enrollment. Psychosocial characteristics determined by standardized questionnaires at baseline include relatively high anxiety trait (44%), moderate to severe depression severity (19%), moderate to high severity of posttraumatic stress disorder symptoms (58%) and a history of childhood trauma/abuse (50%). Conclusions: The Smidt Heart Institute Takotsubo Registry will contribute to advancing the management of TTS by deep phenotyping to understand its pathophysiology, and identify treatment targets in a participant base for future clinical trials.

Exploring outcomes and characteristics of myasthenia gravis: Rationale, aims and design of registry - The EXPLORE-MG registry.

OBJECTIVES: Though much information exists about the diagnosis, treatment, and epidemiology of myasthenia gravis (MG), a comprehensive data registry and biorepository is critical to better understand disease mechanisms, treatment outcomes, and the impact of treatment strategies. We aimed to design and implement the "Exploring Outcomes and Characteristics of Myasthenia Gravis (EXPLORE-MG) Registry" to address these knowledge gaps. METHODS: A web-based, non-interventional, longitudinal, observational disease and outcomes registry was developed; incorporating NIH recommended common data elements for the study of MG. Individuals diagnosed with MG based on prespecified criteria were eligible to participate. The registry was further strengthened by a complementary biorepository. An interim analysis was completed on registry data collected through data-lock in 2017. RESULTS: A total of 232 MG patients, followed at the Yale MG Clinic from 2011 to 2017, were enrolled, which included 2142 total visit entries. Of the 232 MG patients (mean age 60 years, range 17-99; female:male, 1.04:1), 165 were acetylcholine receptor antibody-positive, 20 were muscle-specific kinase antibody-positive, and 47 were seronegative. This cohort consisted of 64 patients with ocular disease, 168 patients with generalized disease, and 65 patients post-thymectomy, including 20 with thymoma-associated MG. CONCLUSIONS: Identification of key clinical features that may predict treatment responsiveness or provide insight into patient outcomes is essential to improve patient care. As current research focuses on the development of patient-tailored, targeted-treatment regimens, this registry can help provide important clinical and epidemiological data from a large contemporary patient cohort with long-term follow-up. REGISTRATION: ClinicalTrials.gov Identifier: NCT03792659.

Creating an effective clinical registry for rare diseases.

The exposure of clinicians to patients with rare gastrointestinal diseases is limited. This hurts clinical studies, which impedes accumulation of scientific knowledge on the natural disease course, treatment outcomes and prognosis in these patients. An excellent method to detect patterns on an aggregate level that would not be possible to discover in individual cases, is a registry study. This paper aims to describe a template to create a successful international registry for rare diseases. We focus mainly on rare hepatic diseases, but lessons from this paper serve other fields in medicine, as well.