scholarly journals Kezdeti tapasztalataink spinalis muscularis atrophiában szenvedő gyermekek intravénás génterápiájával

2020 ◽  
Vol 161 (42) ◽  
pp. 1806-1816
Author(s):  
Borbála Mikos ◽  
Anita Gergely ◽  
Réka Balázsfi ◽  
Edina Bányász ◽  
Beáta Gyömörei ◽  
...  
Keyword(s):  
Side Effects ◽  
Troponin I ◽  
Muscular Atrophy ◽  
Safety Data ◽  
Clinical Results ◽  
Genetic Diagnostics ◽  
Number Of Patients ◽  
Movement Performance ◽  
Term Basis

Összefoglaló. A veleszületett gerincvelői izomsorvadás ritka, progresszív neurodegeneratív betegség, a gyermekkori halál egyik legjelentősebb genetikai oka. Az orvostudomány lehetőségei a XXI. század előtt a progresszió megfékezésére, a szövődmények késleltetésére és ellátására korlátozódtak. A legsúlyosabb génhibában szenvedő gyermekeket általában kétéves kor előtt elveszítettük. A genetikai diagnosztika fejlődése lehetővé teszi a korai diagnózist, a súlyosság és a progresszió előrejelzését. A 2018-ban hazánkban engedélyezett intrathecalis nuszinerszennel a nagy betegszámon alapuló klinikai eredmények meggyőzőek. A 2019-től elérhető új, intravénás génpótló terápiával (onaszemnogén abeparvovek) kapcsolatos tapasztalatok még kisebb betegszámon alapulnak. Hazánkban öt betegnél került sor az alkalmazására a Bethesda Gyermekkórházban, szigorú szakmai kritériumok és előkészületek alapján. Közülük annak a három gyermeknek a kezeléséről számolunk be dolgozatunkban, akiknél már rendelkezünk utánkövetési tapasztalatokkal. Vizsgálatunk szerint a készítmény elősegíti a mozgásteljesítmény javulását. A mellékhatások elsősorban reverzibilis májenzim-emelkedésben, thrombocytopeniában, granulocytopeniában és a szívizom-nekroenzim emelkedésében nyilvánultak meg. Ezért fontosnak tartjuk a betegek szoros és tartós követését, a mellékhatások korai észlelése és elhárítása érdekében. Orv Hetil. 2020; 161(42): 1806–1816. Summary. Congenital spinal muscular atrophy is a rare, progressive neurodegenerative disease, one of the major genetic causes of childhood death. The possibilities of medicine to curb its progression and to delay and treat the disease’s complications were limited before the 21st century. Therefore, children with the most severe genetic defects were usually lost before their second birthday. Advances in genetic diagnostics allow for early diagnosis, prediction of severity and expected progression. Using intrathecal nusinersen (available in Hungary since 2018), clinical results based on a large number of patients are convincing. Experience with the new intravenous gene therapy (onasemnogene abeparvovec) available from 2019 is still based on a less number of patients. It was used in Hungary in Bethesda Hospital in five children based on strict professional criteria and preparations. Our paper summarizes the most important efficacy and safety data of the first three consecutive patients. According to our experiences, the product helps to improve movement performance. Side effects are mainly reversible elevations of liver enzymes and serum troponin-I levels, thrombocytopenia and granulocytopenia. Therefore, we found that it is important to monitor patients closely on a long-term basis in order to detect and eliminate side effects early. Orv Hetil. 2020; 161(42): 1806–1816.

scholarly journals ES-1 Clinical results of tumor treating fields in patients with glioblastoma in Japan, compared with global surveillance

2020 ◽  
Vol 2 (Supplement_3) ◽  
pp. ii3-ii3
Author(s):  
Yoshihiro Muragaki ◽  
Masayuki Nitta ◽  
Taiichi Saito ◽  
Shunichi Tutsuki ◽  
Atsushi Fukui ◽  
...  
Keyword(s):  
Side Effects ◽  
Insurance Coverage ◽  
Intermediate Frequency ◽  
Clinical Results ◽  
Tumor Treating Fields ◽  
Post Marketing Surveillance ◽  
Post Marketing ◽  
Us Fda ◽  
Medical University

Abstract INTRODUCTION: The tumor treatment field induces apoptosis of tumor cells by providing a low intensity, intermediate frequency, alternating current electric field via a transducer array. TTFields is based on Phase 3 EF-11 and EF-14 trials for glioblastoma in the US FDA and Japan PMDA. Therefore, I will report the statistics of TTFields use in Japan along with recent papers. METHODS: 410 patients were treated with TTFields in Japan (December 2017-), of which 17 were at Tokyo Women’s Medical University. We also referred to papers about global post-marketing surveillance and recent studies. RESULTS: Of the 410 patients, 409 (99.8%) were diagnosed with ndGBM(male: female, 66.8%: 33.2%). As of June 2020, 222 patients (54.1%) were on treatment and 188 (45.9%) were discontinued. In 17 cases at TWMU, the average age was 46.3 years. The average treatment period was 218 days, with 6 patients (35%) continuing treatment, 6 patients (35%) discontinuing due to patient wishes, and 5 patients (30%) discontinuing treatment due to recurrence. Side effects were contact dermatitis under the array in 9 patients (57%) and mild malaise in 7 patients (43%). We experienced long-term progression-free cases with TTF use of 25 months (survival 30 months after surgery) with a glioma partially resected and 21 months (survival 27 months after surgery) with a biopsied glioma. In the biopsy case, bevacizumab was used in combination during the treatment. Conclusion: In global surveillance, use for rGBM accounts for 39%, but Japan is limited to use for ndGBM due to insurance coverage. In terms of side effects, it showed a good safety profile comparable to previous trials. Long-term progression-free cases have been observed, and it is necessary to examine the characteristics of patients who respond to treatment and the effect of concomitant use with bevacizumab by prospective studies

scholarly journals EP20 Long term safety and efficacy of denosumab and zoledronate

Rheumatology ◽  
2020 ◽  
Vol 59 (Supplement_2) ◽  
Author(s):  
Ziad Alkutobi ◽  
Deena Laila ◽  
Mohammad Tariq
Keyword(s):  
Side Effects ◽  
Neck Of Femur ◽  
Dxa Scan ◽  
Safety And Efficacy ◽  
Oral Bisphosphonate ◽  
Retrospective Audit ◽  
Number Of Patients ◽  
Gastrointestinal Side Effects ◽  
To Receive

Abstract Background Denosumab and zoledronate are increasingly prescribed for primary and secondary osteoporosis long-term management. Methods A retrospective audit was conducted at Basildon Hospital during 2012-2019 using the NICE guidelines standards to evaluate the long term safety and efficacy of denosumab and zoledronate. Number of patients was 84, diagnosed with osteoporosis or osteopenia, 34 of them received 10 or more denosumab injections and 50 patients planned to receive 5 zoledronate infusions (9 patients received 5 and 38 patients received 4). Results Forty percent of the patients were at their 8th decade followed by 28.57% and 21.42 % at their 9th and 7th decades respectively. More than 90% were Caucasian females. Primary prevention was in 39.28% and secondary prevention in 60.71%. The commonest sites of fracture were the wrist and vertebrae at 34.37% for each; followed by the neck of femur and humerous at 12.5% for each. Denosumab was the 1st, 2nd, 3rd or 4th line of treatment in 9.52%, 13.09%, 16.66% and 1.19% respectively; whereas zoledronate was the 1st, 2nd or 3rd line of treatment in 13.09%, 40.47% and 5.95% respectively. The commonest reason for choosing denosumab as the first line was chronic kidney disease, whereas the reason for choosing it as the 2nd or 3rd line was inefficacy of bisphosphonate in 69% or gastrointestinal side effects in 14%. Zoledronate was chosen in all cases because of intolerability to oral bisphosphonate or gastrointestinal side effects. Repeat DXA scan was performed after the 5th, 10th denosumab injections and the 3rd zoledronate infusions. After the 5th Denosumab, DXA scan showed improvement or stability in 100% and 85.29% at the spine and hip respectively whilst deterioration was seen in 14.7% at the hip. After the 10th denosumab, the rate of improvement or stability at the spine was reduced to 88.23% and decline was seen in 2.94%. At the hip area, 73.52% continued to show improvement or stability, whilst 17.66 % showed deterioration. After the 3rd zoledronate, 98% and 88% showed improvement or stability at the spine and hip respectively whilst deterioration seen in 2% and 12% at the spine and hip respectively. One patient on zoledronate experienced dental issues after the 4th injection and stopped treatment. There were no jaw osteonecrosis, no new fractures and no significant side effects with either denosumab or zoledronate. Ninety percent of patients who completed 10 denosumab injections were planned to continue for another 5 injections. Conclusion After the period of 3-5 years, denosumab and zoledronate were well tolerated and BMD was either stable or improved at both spinal and hip sites. There were few cases of deterioration mainly at the hip area with both zoledronate and denosumab. Future researches are needed to stratify guidelines on discontinuation of denosumab. Disclosures Z. Alkutobi None. D. Laila None. M. Tariq None. A. Nandagudi None.

Trastuzumab-Related Cardiac Dysfunction

2011 ◽  
Vol 9 (2) ◽  
pp. 243-249 ◽  
Author(s):  
Melinda L. Telli ◽  
Ronald M. Witteles
Keyword(s):  
Breast Cancer ◽  
Heart Failure ◽  
Cardiac Dysfunction ◽  
Cardiac Troponin ◽  
Troponin I ◽  
Safety Data ◽  
Cardiac Safety ◽  
Ongoing Controversy ◽  
Mechanistic Insight

The use of trastuzumab in the adjuvant and metastatic treatment of breast cancer is associated with both symptomatic and asymptomatic cardiotoxicity. The long-term significance of these events, isolating known cardiotoxic effects of anthracyclines from those of trastuzumab, and the appropriateness of referring to trastuzumab-related cardiotoxicity as reversible rather than responsive to trastuzumab withdrawal and heart failure medical therapy, are issues that continue to be debated. This article provides an overview of the available cardiac safety data from the major trastuzumab clinical trials in breast cancer, highlighting areas of ongoing controversy. Important recent data documenting the occurrence and prognostic use of cardiac troponin I elevations among patients treated with trastuzumab are placed into context with the mechanistic insight these data provide and the implications for clinical practice today.

Long Term Results of Stereotactic Thalamotomy for Cerebral Palsy

Neurosurgery ◽  
1983 ◽  
Vol 12 (2) ◽  
pp. 195-202 ◽  
Author(s):  
G. Broggi ◽  
L. Angelini ◽  
R. Bono ◽  
C. Giorgi ◽  
N. Nardocci ◽  
...  
Keyword(s):  
Cerebral Palsy ◽  
Side Effects ◽  
Clinical Picture ◽  
Clinical Results ◽  
Long Term Results ◽  
Abnormal Movements ◽  
Average Intelligence ◽  
Stereotactic Thalamotomy

Abstract A group of 33 patients (between 10 and 30 years old and with average intelligence) underwent stereotactic surgery for abnormal movements due to cerebral palsy. Neurological, neurofunctional, and neuropsychological examinations were performed pre- and postoperatively. The length of follow-up ranged between 1 and 4 years. The clinical results are reported and discussed in relation to the targets, the side of the lesion, and the clinical picture. Our data show that better results are obtained in patients with tremor and hyperkinesias; dystonia is improved to a lesser extent, whereas spasticity tends to recur. Operation is more effective for patients with unilateral signs than for patients with bilateral symptoms. The clinical results are stable in time, and the side effects fade away after a few months.

scholarly journals Prospective evaluation of multitarget treatment of pediatric patients with helical intensity-modulated radiotherapy

2020 ◽  
Vol 196 (12) ◽  
pp. 1103-1115
Author(s):  
Maria-Elena A. Salfelder ◽  
Kerstin A. Kessel ◽  
Uwe Thiel ◽  
Stefan Burdach ◽  
Severin Kampfer ◽  
...  
Keyword(s):  
Survival Rate ◽  
Side Effects ◽  
Survival Rates ◽  
Young Patients ◽  
Intensity Modulated Radiotherapy ◽  
High Survival ◽  
Single Target ◽  
Number Of Patients ◽  
Grade 3

Abstract Background and purpose Radiotherapy (RT) is persistently gaining significance in the treatment of pediatric tumors. However, individual features of a growing body and multifocal stages complicate this approach. Tomotherapy offers advantages in the treatment of anatomically complex tumors with low risks of side effects. Here we report on toxicity incidence and outcome of tomotherapy with a focus on multitarget RT (mtRT). Materials and methods From 2008 to 2017, 38 children diagnosed with sarcoma were treated with tomotherapy. The median age was 15 years (6–19 years). Toxicity was graded according to the Common Terminology Criteria for Adverse Events v.4.03 and classified into symptoms during RT, acutely (0–6 months) and late (>6 months) after RT, and long-term sideeffects (>24 months). Results The main histologies were Ewing sarcoma (n = 23 [61%]) and alveolar rhabdomyosarcoma (n = 5 [13%]). RT was performed with a median total dose of 54 Gy (40.5–66.0 Gy) and a single dose of 2 Gy (1.80–2.27 Gy). Twenty patients (53%) received mtRT. Median follow-up was 29.7 months (95% confidence interval 15.3–48.2 months) with a 5-year survival of 55.2% (±9.5%). The 5‑year survival rate of patients with mtRT (n = 20) was 37.1 ± 13.2%, while patients who received single-target RT (n = 18) had a 5-year survival rate of 75 ± 10.8%. Severe toxicities (grade 3 and 4) emerged in 14 patients (70%) with mtRT and 7 patients (39%) with single-target RT. Two non-hematological grade 4 toxicities occurred during RT: one mucositis and one radiodermatitis. After mtRT 5 patients had grade 3 toxicities acute and after single-target RT 4 patients. One patient had acute non-hematological grade 4 toxicities (gastritis, pericarditis, and pericardial effusion) after mtRT. Severe late effects of RT occurred in 2 patients after mtRT and in none of the single-target RT patients. No severe long-term side effects appeared. Conclusion Our results showed acceptable levels of acute and late toxicities, considering the highly advanced diseases and multimodal treatment. Hence, tomotherapy is a feasible treatment method for young patients with anatomically complex tumors or multiple targets. Especially mtRT is a promising and innovative treatment approach for pediatric sarcomas, delivering unexpectedly high survival rates for patients with multifocal Ewing sarcomas in this study, whereby the limited number of patients should invariably be considered in the interpretation.

A Ten-Year Assessment of Methicillin-Associated Side Effects

PEDIATRICS ◽  
1976 ◽  
Vol 58 (3) ◽  
pp. 329-334
Author(s):  
Martha D. Yow ◽  
Larry H. Taber ◽  
Fred F. Barrett ◽  
A. Aaron Mintz ◽  
G. Richard Blankinship ◽  
...  
Keyword(s):  
Side Effects ◽  
Great Majority ◽  
Incidence Rates ◽  
Term Therapy ◽  
Average Dose ◽  
True Incidence ◽  
Number Of Patients ◽  
Chemical Irritation ◽  
Long Term Therapy

The exact incidence and importance of side effects associated with methicillin therapy in children is unknown. During the ten-year period from 1964 to 1974 approximately 3,000 Houston children who received methicillin were observed for side effects. The great majority of these patients received the drug for less than ten days; however, side effects were more common in patients receiving long-term therapy. In order to assess these side effects, experiences with 124 children who received methicillin for ten days or longer were reviewed in depth. The average dose of methicillin was 200 mg/kg/day and the average duration was 22 days. In this highly selected group there were no adverse side effects in 54.8% and only eosinophilia in an additional 13%. Leukopenia occurred in 16%, microscopic hematuria in 8%, gross hematuria in 4%, skin rash in 6%, and "drug fever" in 6%. In many instances several of these side effects occurred within a single patient so that the total number of patients with definite side effects was 39 of 124 (31.5%). The true incidence figure of overall side effects is much lower than 31.5%. Corrected incidence rates based on a conservative figure of 3,000 methicillin-treated children are less than 1.5% for all side effects. In some patients the mechanism producing the adverse reactions seemed to be chemical irritation or toxicity and in others hypersensitivity. In nine of the 39 patients follow-up studies were not optimal. In the other 30 patients all side effects were reversible.

Attitudes toward acupuncture in Hong Kong

2015 ◽  
Vol 9 (2) ◽  
pp. 158-174 ◽  
Author(s):  
Kara Chan ◽  
Lennon Tsang ◽  
Timothy K.F. Fung
Keyword(s):  
Hong Kong ◽  
Side Effects ◽  
Online Survey ◽  
Focus Group Study ◽  
Convenience Sample ◽  
Content Type ◽  
Term Basis ◽  
Quota Sampling ◽  
Practical Implications

Purpose – This paper aims at describing and exploring how consumers perceive acupuncture as a medical treatment in relation to biomedicine. Design/methodology/approach – Data were collected through an online survey using quota sampling. The attitudes of 879 Hong Kong residents aged 20 or above were surveyed. Questions were generated from a previous focus group study. Findings – Factor analysis found that attitude toward acupuncture consisted of five underlying dimensions, including trust in biomedicine, risks versus benefits, cure and effectiveness, qualification and skills of acupuncturists, side effects and costs and severe aftereffects. Lack of trust in acupuncturists and perceived inferiority of acupuncture to biomedicine were the major barriers of public acceptance of acupuncture. Perceived strengths of acupuncture identified were not involving taking medicine, fewer side effects and being good for preventive care. Research limitations/implications – This study used a convenience sample recruited through personal networks, so the findings cannot automatically be generalized to the rest of the population. Practical implications – There is a need to build trust in the therapy and the reputation of acupuncturists through better communication of the profession’s qualification and accreditation system, as well as dissemination of clinical evidence on a long-term basis. Consumers need to be better educated about the sensations to be expected during acupuncture and the criteria for selecting an acupuncturist. Originality/value – This has been the first quantitative study focusing on Hong Kong consumers’ perceptions of acupuncture.

scholarly journals A Brief Review on Breast cancer treatment and current challenges

2021 ◽  
pp. 27-31
Author(s):  
Isha Shah ◽  
Nensi Raytthatha
Keyword(s):  
Breast Cancer ◽  
Side Effects ◽  
Cancer Treatment ◽  
Effective Treatment ◽  
Breast Cancer Survivors ◽  
Early Stage ◽  
Breast Cancer Treatment ◽  
Good News ◽  
Number Of Patients

Cancer is a global disease, so rational and effective treatment is needed. Breast cancer is one of the most common cancers in a woman and now the number of patients is increasing day by day. Therefore, development and research are underway for the effective treatment of breast cancer. Breast cancer treatment depends on the stage of cancer and the risk, based on this medical agents should be employed on patients to prevent breast cancer. In addition, breast cancer survival rates are rising which is good news for science but on the other hand the side effects of treatment present new challenges. An early-stage cancer diagnosis can save a patient's active or healthy life due to long-term and varied treatments that can be used for cancer otherwise breast cancer is a life-threatening disease. Breast cancer survivors not only have negative side effects of cancer treatment but also, have many other issues of previous treatment so it is a challenge for researchers. As a result, this review article deals with the effective treatment of breast cancer and its side effects. This review will help researchers better understand the long-term medical implications for breast cancer.

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.

scholarly journals Insights into Effects/Risks of Chronic Hypergastrinemia and Lifelong PPI Treatment in Man Based on Studies of Patients with Zollinger–Ellison Syndrome

2019 ◽  
Vol 20 (20) ◽  
pp. 5128 ◽  
Author(s):  
Lingaku Lee ◽  
Irene Ramos-Alvarez ◽  
Tetsuhide Ito ◽  
Robert T. Jensen
Keyword(s):  
Side Effects ◽  
Gastroesophageal Reflux Disease ◽  
Gastroesophageal Reflux ◽  
Reflux Disease ◽  
The United States ◽  
Long Term Effects ◽  
Ulcer Disease ◽  
Zollinger Ellison Syndrome ◽  
Number Of Patients

The use of proton pump inhibitors (PPIs) over the last 30 years has rapidly increased both in the United States and worldwide. PPIs are not only very widely used both for approved indications (peptic ulcer disease, gastroesophageal reflux disease (GERD), Helicobacter pylori eradication regimens, stress ulcer prevention), but are also one of the most frequently off-label used drugs (25–70% of total). An increasing number of patients with moderate to advanced gastroesophageal reflux disease are remaining on PPI indefinitely. Whereas numerous studies show PPIs remain effective and safe, most of these studies are <5 years of duration and little data exist for >10 years of treatment. Recently, based primarily on observational/epidemiological studies, there have been an increasing number of reports raising issues about safety and side-effects with very long-term chronic treatment. Some of these safety issues are related to the possible long-term effects of chronic hypergastrinemia, which occurs in all patients taking chronic PPIs, others are related to the hypo-/achlorhydria that frequently occurs with chronic PPI treatment, and in others the mechanisms are unclear. These issues have raised considerable controversy in large part because of lack of long-term PPI treatment data (>10–20 years). Zollinger–Ellison syndrome (ZES) is caused by ectopic secretion of gastrin from a neuroendocrine tumor resulting in severe acid hypersecretion requiring life-long antisecretory treatment with PPIs, which are the drugs of choice. Because in <30% of patients with ZES, a long-term cure is not possible, these patients have life-long hypergastrinemia and require life-long treatment with PPIs. Therefore, ZES patients have been proposed as a good model of the long-term effects of hypergastrinemia in man as well as the effects/side-effects of very long-term PPI treatment. In this article, the insights from studies on ZES into these controversial issues with pertinence to chronic PPI use in non-ZES patients is reviewed, primarily concentrating on data from the prospective long-term studies of ZES patients at NIH.

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