scholarly journals Natural History of Wild-Type Transthyretin Cardiac Amyloidosis and Risk Stratification Using a Novel Staging System

2016 ◽  
Vol 68 (10) ◽  
pp. 1014-1020 ◽  
Author(s):  
Martha Grogan ◽  
Christopher G. Scott ◽  
Robert A. Kyle ◽  
Steven R. Zeldenrust ◽  
Morie A. Gertz ◽  
...  
Keyword(s):  
Natural History ◽  
Risk Stratification ◽  
Cardiac Amyloidosis ◽  
Staging System ◽  
Wild Type ◽  
History Of

Cardiac transthyretin wild-type amyloidosis (ATTRwt): a prospective study of 400 patients followed at the Italian referral center

2020 ◽  
Vol 41 (Supplement_2) ◽  
Author(s):  
P Milani ◽  
L Obici ◽  
R Mussinelli ◽  
M Basset ◽  
G Manfrinato ◽  
...  
Keyword(s):  
Natural History ◽  
Median Survival ◽  
Troponin I ◽  
Stage I ◽  
Stage Ii ◽  
Stage Iii ◽  
Wild Type ◽  
Staging Systems ◽  
Significant Difference ◽  
History Of

Abstract Background Cardiac wild type transthyretin (ATTRwt) amyloidosis, formerly known as senile systemic amyloidosis, is an increasingly recognized, progressive, and fatal cardiomyopathy. Two biomarkers staging systems were proposed based on NT-proBNP (in both cases) and troponin or estimated glomerular filtration rate, that are able to predict survival in this population. The availability of novel effective treatments requires large studies to describe the natural history of the disease in different populations. Objective To describe the natural history of the disease in a large, prospective, national series. Methods Starting in 2007, we protocolized data collection in all the patients diagnosed at our center (n=400 up to 7/2019). Results The referrals to our center increased over time: 5 cases (1%) between 2007–2009, 33 (9%) in 2010–2012, 90 (22%) in 2013–2015 and 272 (68%) in 2016–2019. Median age was 76 years [interquartile range (IQR): 71–80 years] and 372 patients (93%) were males. One hundred and seventy-three (43%) had atrial fibrillation, 63 (15%) had a history of ischemic cardiomyopathy and 64 (15%) underwent pacemaker or ICD implantation. NYHA class was I in 58 subjects (16%), II in 225 (63%) and III in 74 (21%). Median NT-proBNP was 3064 ng/L (IQR: 1817–5579 ng/L), troponin I 0.096 ng/mL (IQR: 0.063–0.158 ng/mL), eGFR 62 mL/min (IQR: 50–78 mL/min). Median IVS was 17 mm (IQR: 15–19 mm), PW 16 mm (IQR: 14–18 mm) and EF 53% (IQR: 45–57%). One-hundred and forty-eight subjects (37%) had a concomitant monoclonal component in serum and/or urine and/or an abnormal free light chain ratio. In these patients, the diagnosis was confirmed by immunoelectron microscopy or mass spectrometry. In 252 (63%) the diagnosis was based on bone scintigraphy. DNA analysis for amyloidogenic mutations in transthyretin and apolipoprotein A-I genes was negative in all subjects. The median survival of the whole cohort was 59 months. The Mayo Clinic staging based on NT-proBNP (cutoff: 3000 ng/L) and troponin I (cutoff: 0.1 ng/mL) discriminated 3 different groups [stage I: 131 (35%), stage II: 123 (32%) and stage III: 127 (33%)] with different survival between stage I and II (median 86 vs. 81 months, P=0.04) and between stage II and III (median 81 vs. 62 months, P<0.001). The UK staging system (NT-proBNP 3000 ng/L and eGFR 45 mL/min), discriminated three groups [stage I: 170 (45%), stage II: 165 (43%) and stage III: 45 (12%)] with a significant difference in survival: between stage I and stage II (86 vs. 52 months, P<0.001) and between stage II and stage III (median survival 52 vs. 33 months, P=0.045). Conclusions This is one of the largest series of patients with cardiac ATTRwt reported so far. Referrals and diagnoses increased exponentially in recent years, One-third of patients has a concomitant monoclonal gammopathy and needed tissue typing. Both the current staging systems offered good discrimination of staging and were validated in our independent cohort. Funding Acknowledgement Type of funding source: None

A rational clinical staging system for childhood rhabdomyosarcoma.

1984 ◽  
Vol 2 (2) ◽  
pp. 135-139 ◽  
Author(s):  
S S Donaldson ◽  
J A Belli
Keyword(s):  
Natural History ◽  
Therapeutic Strategy ◽  
Tumor Volume ◽  
Staging System ◽  
Clinical Staging ◽  
Tumor Node Metastasis ◽  
Node Metastasis ◽  
History Of ◽  
Clinical Staging System ◽  
Childhood Rhabdomyosarcoma

There is a need for a rational and useful staging system for childhood rhabdomyosarcoma that predicts prognosis based on the biology and natural history of the disease. Important factors to consider are age, histology, tumor volume, extent of invasion, and lymphatic involvement. A clinical staging system based on a classical tumor/node/metastasis status, and which accounts for natural history as a function of site of primary, local invasion, and lymphatic or hematogenous metastases is presented. This staging system eliminates the requirement that stage is assigned following an initial therapeutic strategy and removing the implication that prognosis is determined by that strategy.

scholarly journals NIMG-43. RADIOLOGICAL CHARACTERISTICS AND NATURAL HISTORY OF ADULT IDH WILD-TYPE ASTROCYTOMAS WITH TERT PROMOTER MUTATIONS

2018 ◽  
Vol 20 (suppl_6) ◽  
pp. vi185-vi186
Author(s):  
Cristina Izquierdo ◽  
Marc Barritault ◽  
Delphine Poncet ◽  
Stéphanie Cartalat ◽  
Bastien Joubert ◽  
...  
Keyword(s):  
Natural History ◽  
Wild Type ◽  
Tert Promoter ◽  
Tert Promoter Mutations ◽  
History Of ◽  
Promoter Mutations

scholarly journals OS3.3 Radiological characteristics and natural history of adult IDH wild type astrocytomas with TERT promoter mutations

2018 ◽  
Vol 20 (suppl_3) ◽  
pp. iii221-iii221
Author(s):  
C Izquierdo ◽  
M Barritault ◽  
D Poncet ◽  
S Cartalat ◽  
B Joubert ◽  
...  
Keyword(s):  
Natural History ◽  
Wild Type ◽  
Tert Promoter ◽  
Tert Promoter Mutations ◽  
History Of ◽  
Promoter Mutations

scholarly journals B-PO02-149 INCIDENCE, PREDICTORS, AND NATURAL HISTORY OF LEFT ATRIAL APPENDAGE THROMBI IN TRANSTHYRETIN CARDIAC AMYLOIDOSIS

Heart Rhythm ◽  
2021 ◽  
Vol 18 (8) ◽  
pp. S158
Author(s):  
Eoin Donnellan ◽  
Oussama M. Wazni ◽  
Walid I. Saliba ◽  
Divyang Rajesh Patel ◽  
Jeffrey Hedley ◽  
...  
Keyword(s):  
Natural History ◽  
Left Atrial ◽  
Cardiac Amyloidosis ◽  
Left Atrial Appendage ◽  
Atrial Appendage ◽  
History Of

Chronic Lymphocytic Leukemia

Hematology ◽  
2004 ◽  
Vol 2004 (1) ◽  
pp. 163-183 ◽  
Author(s):  
John C. Byrd ◽  
Stephan Stilgenbauer ◽  
Ian W. Flinn
Keyword(s):  
Monoclonal Antibodies ◽  
High Risk ◽  
Chronic Lymphocytic Leukemia ◽  
Natural History ◽  
Risk Stratification ◽  
Nucleoside Analogs ◽  
Response To Therapy ◽  
Lymphocytic Leukemia ◽  
Rapid Progression ◽  
History Of

Abstract Chronic lymphocytic leukemia (CLL) is one of the most commonly diagnosed leukemias managed by practicing hematologists. For many years patients with CLL have been viewed as similar, with a long natural history and only marginally effective therapies that rarely yielded complete responses. Recently, several important observations related to the biologic significance of VH mutational status and associated ZAP-70 overexpression, disrupted p53 function, and chromosomal aberrations have led to the ability to identify patients at high risk for early disease progression and inferior survival. Concurrent with these investigations, several treatments including the nucleoside analogues, monoclonal antibodies rituximab and alemtuzumab have been introduced. Combination of these therapies in clinical trials has led to high complete and overall response rates when applied as initial therapy for symptomatic CLL. Thus, the complexity of initial risk stratification of CLL and treatment has increased significantly. Furthermore, when these initial therapies do not work, approach of the CLL patient with fludarabine-refractory disease can be quite challenging. This session will describe the natural history of a CLL patient with emphasis on important decision junctures at different time points in the disease. In Section I, Dr. Stephan Stilgenbauer focuses on the discussion that occurs with CLL patients at their initial evaluation. This includes a review of the diagnostic criteria for CLL and prognostic factors utilized to predict the natural history of the disease. The later discussion of risk stratification focuses on molecular and genomic aberrations that predict rapid progression, poor response to therapy, and inferior survival. Ongoing and future efforts examining early intervention strategies in high risk CLL are reviewed. In Section II, Drs. Ian Flinn and Jesus G. Berdeja focus on the discussion of CLL patients when symptomatic disease has developed. This includes an updated review of monotherapy trials with nucleoside analogs and recent trials that have combined these with monoclonal antibodies and/or alternative chemotherapy agents. Appropriate application of more aggressive therapies such as autologous and allogeneic immunotherapy and less aggressive treatments for appropriate CLL patient candidates are discussed. In Section III, Dr. John Byrd focuses on the discussion that occurs with CLL patients whose disease is refractory to fludarabine. The application of genetic risk stratification in choosing therapy for this subset of patients is reviewed. Available data with conventional combination based therapies and monoclonal antibodies are discussed. Finally, alternative promising investigational therapies including new antibodies, kinase inhibitors (CDK, PDK1/AKT, PKC) and alternative targeted therapies (DNA methyltransferase inhibitors, histone deacetylase inhibitors, etc.) are reviewed with an emphasis on the most promising agents for this patient population.

Natural history of patients (pts) with advanced cholangiocarcinoma (CCA) with FGFR2 gene fusion/rearrangement or wild-type (WT) FGFR2.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 4089-4089
Author(s):  
Rachna T. Shroff ◽  
Jessica Rearden ◽  
Ai Li ◽  
Susan Moran ◽  
Stacie Peacock Shepherd ◽  
...  
Keyword(s):  
Natural History ◽  
Advanced Disease ◽  
Late Onset ◽  
Stage Iv ◽  
Primary Objective ◽  
Initial Diagnosis ◽  
Wild Type ◽  
Real World Data ◽  
Comprehensive Genomic Profiling ◽  
History Of

4089 Background: CCA is a rare, heterogeneous malignancy with poor prognosis due to late onset of symptoms and relative resistance to available therapies. FGFR2 fusions are common, occurring in 10–16% of intrahepatic CCA (iCCA). There is increasing awareness of the importance of molecular profiling to inform treatment choices, although real-world data (RWD) are lacking on the natural history of pts with CCA and FGFR2 fusions/rearrangements receiving therapies for advanced disease. This retrospective, observational, natural history study used a nationwide (US-based) de-identified clinico-genomic database (CGDB) to compare overall survival (OS) in pts with advanced CCA and FGFR2 fusions/rearrangements vs. those with wild-type (WT) FGFR2. Methods: This study used the nationwide (US-based) de-identified Flatiron Health-Foundation Medicine CCA CGDB (FH-FMI-CGDB). The data originated from approximately 280 US cancer clinics (̃800 sites of care). Pts were ≥18y of age, had chart-confirmed advanced CCA, comprehensive genomic profiling, and ≥2 visits within the Flatiron Health network since Jan 1, 2011. Primary objective: evaluate OS in pts with FGFR2 fusions/rearrangements and WT FGFR2 from the index date (date of diagnosis of advanced CCA) to date of death from any cause. A key secondary objective was to evaluate the influence of FGFR status on OS after adjusting for potential prognostic variables. Risk-set adjustment was used to account for left truncation for all time-to-event analyses. Results: As of May 2020, 571 pts from the CCA FH-FMI-CGDB met the inclusion criteria; 75 pts with FGFR2 fusions/rearrangements (median age 63y; 64% female; 95% iCCA; 68% stage IV at initial diagnosis), and 496 pts FGFR2 WT (median age 65y; 48% female; 74% iCCA; 55% stage IV at initial diagnosis). Median OS was numerically higher, but not statistically different, for pts with FGFR2 fusions/rearrangements vs FGFR2 WT (12.1m [95% CI 8.5−17.1] vs 7.1m [95% CI 5.7−8.8]; log rank p = 0.184). Median OS was also numerically higher, but not statistically different, for FGFR2 fusions/rearrangements vs FGFR WT in the subset of 437 pts with iCCA (12.1m [95% CI 8.4−17.1] vs 7.8m [95% CI 6.1−10.0]; log rank p = 0.375). FGFR2 status was not a significant factor contributing to OS in univariate, bivariate, or multivariate models after adjusting for potential prognostic covariates. Conclusions: This analysis of RWD did not demonstrate a clear survival advantage for pts with FGFR2 fusions/rearrangements vs FGFR2 WT CCA receiving therapies for advanced disease, although a non-significant trend towards longer OS was observed in pts with FGFR2 fusions/rearrangements. FGFR2 status was not a significant predictor of OS after adjusting for potential prognostic covariates. An additional sub-analysis is ongoing to determine OS from time of initiation of second-line therapy in pts with FGFR2 fusions/rearrangements.

scholarly journals Clinical characteristics and natural history of wild‐type transthyretin amyloid cardiomyopathy in Japan

2020 ◽  
Vol 7 (5) ◽  
pp. 2829-2837
Author(s):  
Toshihiro Yamada ◽  
Seiji Takashio ◽  
Yuichiro Arima ◽  
Masato Nishi ◽  
Mami Morioka ◽  
...  
Keyword(s):  
Natural History ◽  
Clinical Characteristics ◽  
Wild Type ◽  
History Of ◽  
Amyloid Cardiomyopathy

scholarly journals 8 Natural history of Wolff-Parkinson-White syndrome. Insights for risk stratification and management

EP Europace ◽  
2005 ◽  
Vol 7 (Supplement_1) ◽  
pp. 1-2
Author(s):  
V. Santinelli ◽  
I. Greiss ◽  
G. Augello ◽  
F. Manguso ◽  
C. Pappone
Keyword(s):  
Natural History ◽  
Risk Stratification ◽  
White Syndrome ◽  
History Of ◽  
Wolff Parkinson White Syndrome
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