Background And Aims: Cardiac amyloidosis (CA) is usually characterised by a poor outcome in the short-term; clinical and instrumental features are heterogeneous and could characterise subgroups with different prognoses. The aim of our study was to describe a subgroup of patients with CA showing an impressive favourable long-term survival.
Methods: Out of 50 patients (males 65%, 63 ± 11 years) with an echocardiographic and bioptic diagnosis of CA observed from 1991 to 2009, we selected a subgroup of patients surviving more than 50 months from diagnosis (group 1). We described their features at enrolment and during follow-up, comparing them with patients surviving less than 12 months (group 2).
Results: We found seven patients (14%) belonging to group 1 and 26 (52%) to group 2. Four out of seven long term survivors suffered from AL amyloidosis, in one case the underlying aetiology was a chronic inflammatory disease, while in two cases remained unknown. At enrolment, group 1 patients showed higher systolic blood pressure with respect to group 2 (140 ± 25 vs. 112 ± 18 mmHg, respectively, p=0.011), and a less thick interventricular septum (IVS) (IVS thickness > 15 mm in 29% vs. 69% of patients, p = 0.049). No patient of group 1 presented left ventricular restrictive filling pattern (0 vs. 31% in group 1 and 2 respectively, p = 0.035), atrial fibrillation (0 vs. 35%, p = 0.024), or progression towards a more severe disease during follow-up.
Conclusions: A not negligible proportion of patients with CA can have a long-term survival. They showed a less severe disease at diagnosis, with substantial stability over time. Further studies on larger populations are necessary to understand the mechanisms underlying this more favourable natural history of the disease.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1016/j.hlc.2013.01.010 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Pivotal trial characteristics and types of endpoints used to support Food and Drug Administration rare disease drug approvals between 2013 and 2022.
Clin Trials
January 2025
Rare Diseases Team, Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD, USA.
Background/aims: Rare disease drug development faces unique challenges, such as genotypic and phenotypic heterogeneity within small patient populations and a lack of established outcome measures for conditions without previously successful drug development programs. These challenges complicate the process of selecting the appropriate trial endpoints and conducting clinical trials in rare diseases. In this descriptive study, we examined novel drug approvals for non-oncologic rare diseases by the U.
View Article and Find Full Text PDFIn the context of Chinese clinical texts, this paper aims to propose a deep learning algorithm based on Bidirectional Encoder Representation from Transformers (BERT) to identify privacy information and to verify the feasibility of our method for privacy protection in the Chinese clinical context. We collected and double-annotated 33,017 discharge summaries from 151 medical institutions on a municipal regional health information platform, developed a BERT-based Bidirectional Long Short-Term Memory Model (BiLSTM) and Conditional Random Field (CRF) model, and tested the performance of privacy identification on the dataset. To explore the performance of different substructures of the neural network, we created five additional baseline models and evaluated the impact of different models on performance.
View Article and Find Full Text PDFElevated serum vitamin B12 concentrations in a patient with pancreatic exocrine insufficiency.
Biomark Med
January 2025
The Nutristasis Unit, Synnovis, St. Thomas' Hospital, London, UK.
This case describes a patient with pancreatic exocrine insufficiency and persistently elevated serum vitamin B12 concentrations that were not due to supplementation or associated with hepatic or hematological pathology. Laboratory investigations suggested the presence of macro-B12 as the cause of this patient's raised serum vitamin B12. Macro-B12 is often formed when vitamin B12-vitamin binding proteins (transcobalamin and haptocorrin) complex with immunoglobulins.
View Article and Find Full Text PDFAdaptive promising zone design for sequential parallel comparison design with continuous outcomes.
Clin Trials
January 2025
Department of Biostatistics, University of Florida, Gainesville, FL, USA.
Introduction: The sequential parallel comparison design has emerged as a valuable tool in clinical trials with high placebo response rates. To further enhance its efficiency and effectiveness, adaptive strategies, such as sample size adjustment and allocation ratio modification can be employed.
Methods: We compared the performance of Jennison and Turnbull's method and the Promising Zone approach for sample size adjustment in a two-phase sequential parallel comparison design study.
Bilateral Choroidal Metastases from Primary Cutaneous Melanoma Following Nivolumab Discontinuation: A Case Report.
Ocul Immunol Inflamm
January 2025
Ocular Oncology Service, Institute of Oncology, Tecnologico de Monterrey, Monterrey, Mexico.
Purpose: To present the case of a young patient with BRAF V600E-mutant cutaneous melanoma who developed bilateral choroidal metastases complicated by neovascular glaucoma (NVG) in both eyes following the interruption of nivolumab therapy.
Methods: A 28-year-old female with primary cutaneous melanoma of the left hand underwent surgical resection and adjuvant nivolumab. Immunotherapy was discontinued due to immune-related acute interstitial nephritis.
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!