Metatropic dysplasia: Clinical and radiographic findings in 11 patients demonstrating long-term natural history

2007 ◽  
Vol 143A (21) ◽  
pp. 2512-2522 ◽  
Author(s):  
Peter Kannu ◽  
Salim Aftimos ◽  
Val Mayne ◽  
Leo Donnan ◽  
Ravi Savarirayan
Keyword(s):  
Natural History ◽  
Radiographic Findings ◽  
Metatropic Dysplasia

Reduction of major amputations in the diabetic ischaemic foot: A strategy to "take control" with conservative care as well as revascularisation

VASA ◽  
2001 ◽  
Vol 30 (Supplement 58) ◽  
pp. 6-14 ◽  
Author(s):  
Edmonds ◽  
Foster
Keyword(s):  
Natural History ◽  
Treatment Plan ◽  
Staging System ◽  
Vascular Control ◽  
New Approach ◽  
Conservative Care ◽  
Arterial Bypass ◽  
Therapeutic Footwear ◽  
Frequent Problem

The diabetic ischaemic foot has become an increasingly frequent problem over the last decade. However, we report a new approach consisting of a basic classification, a simple staging system of the natural history and a treatment plan for each stage, within a multi-disciplinary framework. This approach of "taking control" consists of two parts: 1. long-term conservative care including debridement of ulcers (to obtain wound control), eradication of sepsis (micribiological control), and provision of therapeutic footwear (mechanical control), and 2. revascularisation by angioplasty and arterial bypass (vascular control). This approach has led to a 50% reduction in the rate of major amputations in patients attending with ischaemic ulceration and absent foot pulses from 1989 to 1999 (from 4.6% to 2.3% per year). Patients who underwent angioplasty increased from 6% to 13%. Arterial bypass similarly increased from 3% to 7% of cases. However, even with an increased rate of revascularisation, 80% of patients responded to conservative care alone. This,we conclude, is an essential part of the management of all patients with ischaemic feet.

The natural history of pituitary apoplexy: long term follow-up study

2019 ◽  
Author(s):  
Ayesha Shaikh ◽  
Natasha Shrikrishnapalasuriyar ◽  
Giselle Sharaf ◽  
David Price ◽  
Maneesh Udiawar ◽  
...  
Keyword(s):  
Natural History ◽  
Pituitary Apoplexy ◽  
Follow Up Study ◽  
Long Term Follow Up ◽  
History Of

scholarly journals Spontaneous improvement of carbohydrate-deficient transferrin in PMM2-CDG without mannose observed in CDG natural history study

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Peter Witters ◽  
Andrew C. Edmondson ◽  
Christina Lam ◽  
Christin Johnsen ◽  
Marc C. Patterson ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Natural History ◽  
Randomized Study ◽  
Short Term ◽  
Double Blind ◽  
Natural History Study ◽  
Natural History Studies ◽  
Carbohydrate Deficient Transferrin ◽  
Spontaneous Improvement

AbstractA recent report on long-term dietary mannose supplementation in phosphomannomutase 2 deficiency (PMM2-CDG) claimed improved glycosylation and called for double-blind randomized study of the dietary supplement in PMM2-CDG patients. A lack of efficacy of short-term mannose supplementation in multiple prior reports challenge this study’s conclusions. Additionally, some CDG types have previously been reported to demonstrate spontaneous improvement in glycosylated biomarkers, including transferrin. We have likewise observed improvements in transferrin glycosylation without mannose supplementation. This observation questions the reliability of transferrin as a therapeutic outcome measure in clinical trials for PMM2-CDG. We are concerned that renewed focus on mannose therapy in PMM2-CDG will detract from clinical trials of more promising therapies. Approaches to increase efficiency of clinical trials and ultimately improve patients’ lives requires prospective natural history studies and identification of reliable biomarkers linked to clinical outcomes in CDG. Collaborations with patients and families are essential to identifying meaningful study outcomes.

scholarly journals Treatments and outcomes of untreated cerebral cavernous malformations in China: study protocol of a nationwide multicentre prospective cohort study

BMJ Open ◽  
2020 ◽  
Vol 10 (10) ◽  
pp. e037957
Author(s):  
Fuxin Lin ◽  
Qiu He ◽  
Zhuyu Gao ◽  
Lianghong Yu ◽  
Dengliang Wang ◽  
...  
Keyword(s):  
Informed Consent ◽  
Natural History ◽  
Study Protocol ◽  
Treated Group ◽  
Secondary Outcome ◽  
Long Term Outcomes ◽  
Cavernous Malformations ◽  
Electronic Data Capture System

IntroductionThe treatment decision and long-term outcomes of previously untreated cerebral cavernous malformation (U-CCM) are still controversial. Therefore, we are conducting a nationwide multicentre prospective registry study in China to determine the natural history and effect of surgical treatment on long-term outcomes in Chinese people with U-CCM.Methods and analysisThis study was started on 1 January 2018 and is currently ongoing. It is a cohort follow-up study across a 5-year period. Patients will be followed up for at least 3 years after inception. Patients with U-CCM will be enrolled from 24 Grade III, level A hospitals distributed all over China. The cohort size is estimated to be 1200 patients. Patients are registered in surgically treated group and conservatively treated group. Clinical characteristics, radiology information and laboratory data are prospectively collected using an electronic case report form through an electronic data capture system. The primary outcome of this study is poor clinical outcome at the last follow-up (modified Rankin Scale score >2 lasting at least 1 year). The secondary outcome includes symptomatic haemorrhage, drug refractory epilepsy, focal neurological deficits, morbidity and all-cause mortality during follow-up. Univariate and multivariate regression analysis will be performed to determine the risk factors for poor outcomes in all patients, and to estimate the effect of surgery. Life tables, Kaplan-Meier estimates, log-rank test and proportional hazards Cox regression will be used to analyse the follow-up data of conservatively treated patients to determine the natural history of U-CCM. Initial presentation and location of U-CCM are prespecified subgroup factors.Ethics and disseminationThe study protocol and informed consent form have been reviewed and approved by the Research Ethical Committee of First Affiliated Hospital of Fujian Medical University (FAHFMU-2018-003).Written informed consent will be obtained from each adult participant or from the guardian of each paediatric participant. The final results will be published in peer-reviewed journals.Trial registration numberNCT03467295.

scholarly journals Long-term Natural History of Dry Eye Disease from the Patient's Perspective

Ophthalmology ◽  
2016 ◽  
Vol 123 (2) ◽  
pp. 425-433 ◽  
Author(s):  
Jeffrey P. Lienert ◽  
Laura Tarko ◽  
Miki Uchino ◽  
William G. Christen ◽  
Debra A. Schaumberg
Keyword(s):  
Natural History ◽  
Dry Eye ◽  
Dry Eye Disease ◽  
Eye Disease ◽  
Patient’S Perspective ◽  
History Of

The Natural History of Long-Term Cardiac Pacing

1977 ◽  
Vol 23 (6) ◽  
pp. 550-554 ◽  
Author(s):  
William S. Stoney ◽  
Frederick E. Finger ◽  
William C. Alford ◽  
George R. Burrus ◽  
Robert A. Frist ◽  
...  
Keyword(s):  
Natural History ◽  
Cardiac Pacing ◽  
History Of

Effects of long-term chloroquine administration on the natural history of aortic aneurysms in mice

2015 ◽  
Vol 93 (8) ◽  
pp. 641-648 ◽  
Author(s):  
Azza Ramadan ◽  
Mark D. Wheatcroft ◽  
Adrian Quan ◽  
Krishna K. Singh ◽  
Fina Lovren ◽  
...  
Keyword(s):  
Natural History ◽  
Thrombus Formation ◽  
Aortic Aneurysms ◽  
Ang Ii ◽  
Abdominal Aortic ◽  
Suprarenal Aorta ◽  
History Of ◽  
Categorical Distribution

Autophagy regulates cellular homeostasis and integrates the cellular pro-survival machinery. We investigated the role of autophagy in the natural history of murine abdominal aortic aneurysms (AAA). ApoE−/− mice were implanted with saline- or angiotensin II (Ang-II)-filled miniosmotic pumps then treated with either the autophagy inhibitor chloroquine (CQ; 50 mg·(kg body mass)–1·day–1, by intraperitoneal injection) or saline. Ang-II-elicited aneurysmal expansion of the suprarenal aorta coupled with thrombus formation were apparent 8 weeks later. CQ had no impact on the incidence (50% for Ang-II compared with 46.2% for Ang-II + CQ; P = NS) and categorical distribution of aneurysms. The markedly reduced survival rate observed with Ang-II (57.1% for Ang-II compared with 100% for saline; P < 0.05) was unaffected by CQ (61.5% for Ang-II + CQ; P = NS compared with Ang-II). CQ did not affect the mean maximum suprarenal aortic diameter (1.91 ± 0.19 mm for Ang-II compared with 1.97 ± 0.21 mm for Ang-II + CQ; P = NS). Elastin fragmentation, collagen accumulation, and smooth muscle attrition, which were higher in Ang-II-treated mice, were unaffected by CQ treatment. Long-term CQ administration does not affect the natural history and prognosis of experimental AAA, suggesting that global loss of autophagy is unlikely to be a causal factor in the development of aortic aneurysms. Manipulation of autophagy as a mechanism to reduce AAA may need re-evaluation.

The natural history of glycogen storage disease types VI and IX: Long-term outcome from the largest metabolic center in Canada

2014 ◽  
Vol 113 (3) ◽  
pp. 171-176 ◽  
Author(s):  
Anne Roscher ◽  
Jaina Patel ◽  
Stacy Hewson ◽  
Laura Nagy ◽  
Annette Feigenbaum ◽  
...  
Keyword(s):  
Natural History ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage ◽  
Term Outcome ◽  
Long Term Outcome ◽  
History Of
Sign in / Sign up

Export Citation Format

Share Document