scholarly journals Two Siblings with Familial Chylomicronemia Syndrome: Disease Course and Effectiveness of Early Treatment

2010 ◽  
Vol 2010 ◽  
pp. 1-5 ◽  
Author(s):  
Hanan AL Azkawi ◽  
Ibrahim AlAlwan

There are no adequate data that evaluate the safety and effectiveness of lowering triglyceride levels in very young children. The authors report a family with two male siblings, 7 and 4 years old, affected by familial hyperchylomicronemia. The oldest was diagnosed at birth during evaluation of jaundice, and the youngest showed asymptomatic hypertriglyceridemia by 6 months of age. Due to high triglyceride levels, Gemfibrozil (a fibric acid derivative) was started at diagnosis. Close clinical followup and laboratory monitoring of these children showed no side effects from the drug, and the risk of acute pancreatitis was significantly reduced.

2005 ◽  
Vol 28 (10) ◽  
pp. 1018-1024 ◽  
Author(s):  
G. Giannini ◽  
M. Valbonesi ◽  
F. Morelli ◽  
P. Carlier ◽  
M.C. De Luigi ◽  
...  

Patients with extremely high triglyceride levels and associated lipemia are at high risk for acute pancreatitis. Two factors can increase triglyceride-rich lipoproteins; one is overproduction and other is a defect in clearance. Either mechanism can cause hypertriglyceridemia and both may exist simultaneously. Causes can be either primary or secondary. Plasmapheresis is efficacious for severe hypertryceridemia in patients who have not responded to previous therapies. We have treated 15 cases of hypertrygliceridemia complicating the course of patients receiving Cyclosporin A after bone marrow transplantation. Five patients were treated with plasmapheresis, the other ten with cascade filtration. The removal rate for triglycerides was 58.0% for patients treated by cascade filtration and 63.5% for patients treated by plasmapheresis. The removal rates for triglycerides were low possibly as a consequence of early saturation of the filter.


Pancreas ◽  
2018 ◽  
Vol 47 (7) ◽  
pp. e40-e41 ◽  
Author(s):  
Daniel Lew ◽  
Bechien U. Wu ◽  
Stephen J. Pandol ◽  
Catherine A. Sugar ◽  
Damla Senturk ◽  
...  

2017 ◽  
Vol 36 (2) ◽  
pp. 151-158 ◽  
Author(s):  
Onur Taydas ◽  
Emre Unal ◽  
Ali Devrim Karaosmanoglu ◽  
Mehmet Ruhi Onur ◽  
Erhan Akpinar

2016 ◽  
Vol 175 (1) ◽  
pp. 37-41 ◽  
Author(s):  
S. V. Avakimyan ◽  
V. A. Avakimyan ◽  
M. T. Didigov ◽  
E. S. Babenko

2021 ◽  
Vol 8 (3) ◽  
pp. 590
Author(s):  
Rajesh K. Srinivas ◽  
Ashok Gupta ◽  
Priyanshu Mathur ◽  
Kamlesh Agarwal ◽  
Anil Sharma

Familial hypertriglyceridemia is rare in infancy. Diagnosis in infancy is very difficult and is usually diagnosed when acute pancreatitis sets in. Early diagnosis is important as it can prevent the complications associated with acute pancreatitis and pancreatic necrosis. Here is a case familial hypertriglyceridemia in an infant who presented to us with failure to thrive but was diagnosed early due to presence of highly viscous and milky blood. This holds importance as early treatment can reduce the complications and morbidity associated with familial hypertriglyceridemia.


2022 ◽  
Vol 20 (1) ◽  
Author(s):  
Luigi Barrea ◽  
Ludovica Verde ◽  
Claudia Vetrani ◽  
Francesca Marino ◽  
Sara Aprano ◽  
...  

Abstract Background Very Low-Calorie Ketogenic Diet (VLCKD) is currently a promising approach for the treatment of obesity. However, little is known about the side effects since most of the studies reporting them were carried out in normal weight subjects following Ketogenic Diet for other purposes than obesity. Thus, the aims of the study were: (1) to investigate the safety of VLCKD in subjects with obesity; (2) if VLCKD-related side effects could have an impact on its efficacy. Methods In this prospective study we consecutively enrolled 106 subjects with obesity (12 males and 94 females, BMI 34.98 ± 5.43 kg/m2) that underwent to VLCKD. In all subjects we recorded side effects at the end of ketogenic phase and assessed anthropometric parameters at the baseline and at the end of ketogenic phase. In a subgroup of 25 subjects, we also assessed biochemical parameters. Results No serious side effects occurred in our population and those that did occur were clinically mild and did not lead to discontinuation of the dietary protocol as they could be easily managed by healthcare professionals or often resolved spontaneously. Nine (8.5%) subjects stopped VLCKD before the end of the protocol for the following reasons: 2 (1.9%) due to palatability and 7 (6.1%) due to excessive costs. Finally, there were no differences in terms of weight loss percentage (13.5 ± 10.9% vs 18.2 ± 8.9%; p = 0.318) in subjects that developed side effects and subjects that did not developed side effects. Conclusion Our study demonstrated that VLCKD is a promising, safe and effective therapeutic tool for people with obesity. Despite common misgivings, side effects are mild, transient and can be prevented and managed by adhering to the appropriate indications and contraindications for VLCKD, following well-organized and standardized protocols and performing adequate clinical and laboratory monitoring.


2020 ◽  
Vol 7 (4) ◽  
pp. e719 ◽  
Author(s):  
Erik Ellwardt ◽  
Leoni Rolfes ◽  
Julia Klein ◽  
Katrin Pape ◽  
Tobias Ruck ◽  
...  

ObjectiveTo provide first real-world experience on patients with MS treated with the B cell–depleting antibody ocrelizumab.MethodsWe retrospectively collected data of patients who had received at least 1 treatment cycle (2 infusions) of ocrelizumab at 3 large neurology centers. Patients' characteristics including premedication, clinical disease course, and documented side effects were analyzed.ResultsWe could identify 210 patients (125 women, mean age ± SD, 42.1 ± 11.4 years) who had received ocrelizumab with a mean disease duration of 7.3 years and a median Expanded Disability Status Scale score of 3.75 (interquartile range 2.5–5.5; range 0–8). Twenty-six percent of these patients had a primary progressive MS (PPMS), whereas 74% had a relapsing-remitting (RRMS) or active secondary progressive (aSPMS) disease course. Twenty-four percent of all patients were treatment naive, whereas 76% had received immune therapies before. After ocrelizumab initiation (median follow-up was 200 days, range 30–1,674 days), 13% of patients with RRMS/aSPMS experienced a relapse (accounting for an annualized relapse rate of 0.17, 95% CI 0.10–0.24), and 5% of all patients with MS experienced a 12-week confirmed disability progression. Treatment was generally well tolerated, albeit only short-term side effects were recorded, including direct infusion-related reactions and mild infections.ConclusionsWe provide class IV evidence that treatment with ocrelizumab can stabilize naive and pretreated patients, indicating that ocrelizumab is an option following potent MS drugs such as natalizumab and fingolimod. Further studies are warranted to confirm these findings and to reveal safety concerns in the longer-term follow-up.Classification of evidenceThis study provides Class IV evidence that for patients with MS, ocrelizumab can stabilize both treatment-naive and previously treated patients.


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