scholarly journals Preventive use of nitisinone in alkaptonuria

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Bruce H. R. Wolffenbuttel ◽  
M. Rebecca Heiner-Fokkema ◽  
Francjan J. van Spronsen
Keyword(s):  
Enzyme Activity ◽  
Side Effects ◽  
Intervention Studies ◽  
Homogentisic Acid ◽  
Beneficial Effects ◽  
Rare Congenital Disorder ◽  
Fourth Decade ◽  
Long Term Consequences ◽  
Plasma Tyrosine

AbstractAlkaptonuria (AKU, OMIM 203500) is a rare congenital disorder caused by a deficiency of the enzyme homogentisate-1,2,-dioxygenase. The long-term consequences of AKU are joint problems, cardiac valve abnormalities and renal problems. Landmark intervention studies with nitisinone 10 mg daily, suppressing an upstream enzyme activity, demonstrated its beneficial effects in AKU patients with established complications, which usually start to develop in the fourth decade. Lower dose of nitisinone in the range of 0.2–2 mg daily will already reduce urinary homogentisic acid (uHGA) excretion by > 90%, which may prevent AKU-related complications earlier in the course of the disease while limiting the possibility of side-effects related to the increase of plasma tyrosine levels caused by nitisinone. Future preventive studies should establish the lowest possible dose for an individual patient, the best age to start treatment and also collect evidence to which level uHGA excretion should be reduced to prevent complications.

Long-Term Inhalative Sedation in Children With Pulmonary Diseases

2020 ◽  
pp. 088506662095339
Author(s):  
Jochen Meyburg ◽  
David Frommhold ◽  
Johann Motsch ◽  
Navina Kuss
Keyword(s):  
Side Effects ◽  
Lung Function ◽  
Medical Center ◽  
Academic Medical Center ◽  
Tertiary Care ◽  
Prospective Trial ◽  
Pediatric Intensive Care ◽  
Normal Lung ◽  
Beneficial Effects

Objectives: To describe safety and feasibility of long-term inhalative sedation (LTIS) in children with severe respiratory diseases compared to patients with normal lung function with respect to recent studies that showed beneficial effects in adult patients with acute respiratory distress syndrome (ARDS). Design: Single-center retrospective study. Setting: 12-bed pediatric intensive care unit (PICU) in a tertiary-care academic medical center in Germany. Patients: All patients treated in our PICU with LTIS using the AnaConDa® device between July 2011 and July 2019. Measurements and Main Results: Thirty-seven courses of LTIS in 29 patients were analyzed. LTIS was feasible in both groups, but concomitant intravenous sedatives could be reduced more rapidly in children with lung diseases. Cardiocirculatory depression requiring vasopressors was observed in all patients. However, severe side effects only rarely occured. Conclusions: In this largest cohort of children treated with LTIS reported so far, LTIS was feasible even in children with severely impaired lung function. From our data, a prospective trial on the use of LTIS in children with ARDS seems justified. However, a thorough monitoring of cardiocirculatory side effects is mandatory.

Stimulants and Hyperkinesis: Drug Use or Abuse?

1975 ◽  
Vol 5 (4) ◽  
pp. 371-379 ◽  
Author(s):  
Sherwood O. Cole ◽  
Samuel F. Moore
Keyword(s):  
Side Effects ◽  
Drug Use ◽  
Primary Concern ◽  
Welfare Of The Child ◽  
Long Term Consequences ◽  
Total Welfare

The use of stimulants in the treatment of hyperkinesis is discussed in terms of: 1) the side effects and long-term consequences of drugs; 2) drugs as easy solutions to problems; and 3) the potential for abuse and addiction. While the effectiveness of stimulants in controlling hyperactivity is clearly recognized, the total welfare of the child should be our primary concern. Accordingly, the present paper stresses the need for a more intelligent and restrained use of stimulants than has heretofore been practiced.

Beneficial effects of a probiotic blend on gastrointestinal side effects induced by leflunomide and amlodipine in a rat model

2017 ◽  
Vol 8 (5) ◽  
pp. 801-808 ◽  
Author(s):  
S.-C. Song ◽  
Y.-M. An ◽  
J.-H. Shin ◽  
M.-J. Chung ◽  
J.-G. Seo ◽  
...  
Keyword(s):  
Side Effects ◽  
Gut Microbiota ◽  
Large Intestine ◽  
Rat Model ◽  
Beneficial Effects ◽  
Oral Medications ◽  
Tnf Α ◽  
Gastrointestinal Side Effects ◽  
Total Composition

Patients with metabolic disorders frequently suffer from side effects induced by long-term oral medications. The present study using a rat model system indicated that leflunomide (LF) and amlodipine (AMD), the active ingredients contained in the medications for rheumatoid arthritis and hypertension, respectively, appeared to induce various bowel problems including constipation and inflammation. In the small and large intestine, LF increased the expression of tumour necrosis factor (TNF)-α, interleukin (IL)-1β, and IL-6 compared to the null control and AMD increased the expression of both TNF-α and IL-1β, although its effect on IL-6 was only increased in the large intestine. It is noteworthy that the probiotic blend tested was found to alleviate intestinal complications caused by LF and AMD. Analysis of the gut microbiota revealed that AMD induced compositional changes in the gut microbiota. Namely, members of the phylum Bacteroidetes, which constituted only about 0.3% of the microbiota in the null control, made up more than 10% of the total composition in the AMD-administered rats. Interestingly, the probiotic blend was also found to normaliSe the gut microbiota.

scholarly journals The Incoherence of Institutional Reform: Decentralization as a Structural Solution to Immediate Political Needs

2021 ◽  
Author(s):  
Jean-Paul Faguet ◽  
Mahvish Shami
Keyword(s):  
Side Effects ◽  
Local Government ◽  
Structural Changes ◽  
Institutional Reform ◽  
Short Term ◽  
Institutional Reforms ◽  
Long Term Consequences ◽  
Main Effects ◽  
Structural Solution

AbstractInstitutional reforms are structural changes in the rules and norms of authority, with effects that are long-term and unpredictable on government, politics, and society. But leaders may undertake them to solve unrelated, discrete, short-term political problems. Understanding the latter is key to understanding the characteristics of many real reforms, and hence their fate. We introduce the concept of instrumental incoherence and use it to construct a theory of decentralization where reform is motivated by orthogonal objectives. We show that reformers’ incentives map onto the specifics of reform design via their side effects, not their main effects, which in turn lead to the medium- and long-term consequences eventually realized. We characterize downwardly accountable decentralization, which ties the hands of the center to empower local voters, vs. upwardly accountable decentralization, which ties the hands of local government to empower the center. We use these ideas to explain highly divergent outcomes in two extreme cases, Bolivia and Pakistan, using detailed, original evidence. Our analysis likely extends to a broader class of reforms where the incentives of agents pursuing a change, and the effects of that change, are highly asymmetric in time and dimension.

Chemotherapy and Radiation Therapy

2017 ◽  
Author(s):  
Mary R. Welch ◽  
Craig Nolan
Keyword(s):  
Side Effects ◽  
Radiation Toxicity ◽  
Close Attention ◽  
Profound Impact ◽  
Wide Range ◽  
Long Term Consequences ◽  
Effects Of Radiation ◽  
Oncology Practice

Neurotoxicity is a common problem in oncology practice and neurologists who care for cancer patients encounter a wide range of symptoms attributable to the side effects of radiation and/or chemotherapy. Complications involving the nervous system may be debilitating. Though generally improved by dose reduction or cessation of an offending agent, such symptoms can be irreversible and frequently have a profound impact on quality of life. The appropriate balance between therapeutic efficacy and drug or radiation toxicity requires close attention to the patient’s complaints as well as a thorough understanding of long-term consequences of both the disease and a given treatment’s side effects.

scholarly journals Should Dopamine Agonists be Given Early or Late in the Treatment of Parkinson’s Disease?

1984 ◽  
Vol 11 (S1) ◽  
pp. 229-232 ◽  
Author(s):  
A. Rascol ◽  
J.L. Montastruc ◽  
O. Rascol
Keyword(s):  
Parkinson’S Disease ◽  
Parkinson's Disease ◽  
Side Effects ◽  
Clinical State ◽  
Partial Loss ◽  
Number Of Patients ◽  
Advanced Stages ◽  
Long Term Consequences ◽  
Late Side

ABSTRACTThe long term consequences of the use of a dopamine agonist, bromocriptine, in the treatment of Parkinson’s disease are reported. In a first study in 82 patients showing late side effects of levodopa, bromocriptine permitted a significant decrease of the gastro-intestinal adverse effects. In contrast, no significant improvement of end of dose deterioration from levodopa was noted. In cases where levodopa had ceased to be active, bromocriptine produced an improvement in the clinical state. The drug was ineffective in the very advanced stages of the disease or in the cases of dyskinesias without “on-off” effects. Bromocriptine did not significantly improve freezing or “on-off” effects, but reduced other side effects of levodopa, in particular dystonia. In a second group of 29 patients who had never received levodopa treatment, bromocriptine was shown to be very effective as a first treatment of the disease. The most important finding was the absence of long term side effects similar to those usually observed under levodopa: in this group and in comparison with 38 patients taking levodopa, dyskinesia, dystonia, oscillations in performance and especially “on-off” effects were not noted. However, a partial loss of efficacy of bromocriptine was observed in 27% of cases. In a third group of 10 patients, bromocriptine introduced according to a low and slow protocol was found to be active in a limited number of patients only.

Appearance of contraceptive steroids in human milk: effects on the child

1977 ◽  
Vol 9 (S4) ◽  
pp. 165-179 ◽  
Author(s):  
J. K. Harfouche
Keyword(s):  
Side Effects ◽  
Human Milk ◽  
Breast Feeding ◽  
Post Partum ◽  
First Trimester ◽  
Hormonal Contraceptives ◽  
Lactating Women ◽  
Systemic Side Effects ◽  
Long Term Consequences

SummaryThe realization that steroidal contraceptives administered immediately after delivery to lactating women may be excreted into milk, and adversely affect the neonate, is a cause for concern. Most investigations on hormonal contraceptives have dealt with the efficacy of ovulation suppression, and systemic side effects, but little is known about the appearance of steroids or their metabolites in milk. Apart from the immediate side effects on the neonate, unforeseen long-term consequences require special consideration. Until research provides us with more information, women should use breast-feeding and IUDs instead of steroidal contraceptives during the first trimester post-partum.

scholarly journals Dietary fibre and prebiotics in infant formulas

2003 ◽  
Vol 62 (1) ◽  
pp. 183-185 ◽  
Author(s):  
Jacques Ghisolfi
Keyword(s):  
Intestinal Microflora ◽  
Intervention Studies ◽  
Well Being ◽  
Infant Formulas ◽  
Long Term Effects ◽  
Bacterial Host ◽  
Host Interactions ◽  
Beneficial Effects ◽  
Health And Well Being

Prebiotics have the potential to promote immediate and long-term effects on the health and well-being of infants. They have been added to infant formulas in Japan for 20 years and have only recently been used in Europe. The objective is to change the intestinal microflora in order to mimic the bacteriological effect and, thus, the functional effects of human milk. There is, however, a potential risk of long-term effects of the use of these infant formulas. The consequences of using prebiotics in infants, during the first months of life, on the composition and development of the intestinal microflora, and on the resulting bacterial–bacterial and bacterial–host interactions are not known. Attempts have been made to improve infant and follow-on formulas by prebiotic supplementation, but intervention studies are needed to evaluate their immediate and long-term beneficial effects and demonstrate the absence of harmful consequences from their use.

5 facts on loperamid utility

2020 ◽  
Vol 13 (4) ◽  
pp. 504-507
Author(s):  
Dorota Waśko-Czopnik
Keyword(s):  
Chronic Disease ◽  
Side Effects ◽  
Combination Therapy ◽  
Infectious Complication ◽  
Chronic Diarrhea ◽  
Specific Symptom ◽  
Disease Symptoms ◽  
Beneficial Effects ◽  
Control Disease

Diarrhea is a very common problem that can only be a non-specific symptom of the disease, but can also be an infectious complication or a harbinger of a chronic disease. For years, loperamide has found its place in the treatment of acute and chronic diarrhea. The safety profile and efficacy of action allow the patient to self-use during the first disease period until symptoms disappear. The drug has beneficial effects in patients with urgency and after ileal surgery, by thickening and shaping the stool and influencing the tone of the rectal muscles. In addition, it works well in traveler’s diarrhea, alone or in combination therapy with a dedicated drug. It does not cause long-term side effects, it works quickly to control disease symptoms and prevent complications that worsen the course of the disease, such as dehydration. Despite other alternatives on the market, it is still at the forefront of effective and safe anti-diarrheal drugs.

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