scholarly journals Efficacy of steroid therapy for Fukuyama congenital muscular dystrophy

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Terumi Murakami ◽  
Takatoshi Sato ◽  
Michiru Adachi ◽  
Kumiko Ishiguro ◽  
Minobu Shichiji ◽  
...  

AbstractAlthough there is only symptomatic treatment for Fukuyama congenital muscular dystrophy (FCMD), several reports have suggested that steroid therapy could be effective for FCMD; however, no independent intervention studies have been conducted. This study aimed to evaluate the efficacy of steroid therapy for restoring motor functions in FCMD patients. This study involved 3-to-10-year-old FCMD patients who exhibited a decline in motor functions, requested steroid therapy. Patients with consent started oral administration of 0.5-mg/kg prednisolone every alternate day, which was increased to 1.0 mg/kg if the response was inadequate. We used the Gross Motor Function Measure (GMFM) to evaluate and compare the motor functions of all patients. Wilcoxon signed-rank test (significance level, P ≤ 0.05) was used for statistical analysis. At the onset of steroid therapy, 8.10 years (SD, 2.14 years) was the mean age of FCMD patients. The mean GMFM difference between before and after the steroid therapy was + 1.23 (SD, 1.10), and a P value of 0.015 represented significant improvement in GMFM. Our results indicate that steroid therapy may contribute to the maintenance and improvement of the motor functions of advanced-stage FCMD patients.Clinical Trial Registration Registration Number: UMIN000020715, Registration Date: Feb 1st, 2016 (01/02/2016).

2021 ◽  
Author(s):  
Terumi Murakami ◽  
Takatoshi Sato ◽  
Michiru Adachi ◽  
Kumiko Ishiguro ◽  
Minobu Shichiji ◽  
...  

Abstract Background: Although a fundamental treatment for Fukuyama congenital muscular dystrophy (FCMD) is not developed yet, several reports have suggested that steroid therapy could be effective for FCMD; however, no independent intervention studies have been conducted.Objective: This study aimed to evaluate the efficacy of steroid therapy for restoring motor functions in FCMD patients.Subjects: This study involved 3-to-10-year-old FCMD patients who exhibited a decline in motor functions, requested steroid therapy.Methods: The dose regimen started with oral administration of prednisolone at 0.5 mg/kg every other day and was increased to 1.0 mg/kg if the response was insufficient. The motor functions of the patients were evaluated and compared using the Gross Motor Function Measure (GMFM). Statistical analysis was performed using the Wilcoxon signed-rank test (significance level, P ≤ 0.05)Results: The mean age of FCMD patients at the onset of steroid therapy was 8.10 years (SD, 2.14 years). The mean GMFM difference between before and after the steroid therapy was +1.23 (SD, 1.10), with a P value of 0.015 representing a significant improvement in the GMFM.Conclusions: Our results indicate that steroid therapy may contribute to the maintenance and improvement of the motor functions of advanced-stage FCMD patients.Clinical Trial Registration: Registration Number: UMIN000020715, Registration Date: 01/02/2016.


2021 ◽  
Vol 1 (2) ◽  
pp. 22
Author(s):  
Hidayatul Hamdiah ◽  
Nik Mohd Rahimi Nik Yusoff

This article focused on the effectiveness of Number Head Together in teaching writing in the EFL Classroom. The aim of the study was to improve EFL students’ writing. This study was quantitative research and used an experimental design. The population was 30 students and the sample was 15 students. Purposive sampling was used in determining the sample of the study. The writing test was the instrument used to collect the data. The data were analyzed by using descriptive statistics and paired sample t-test to test the hypothesis. The students' instrument was an essay-informed descriptive text. Furthermore, the researchers used pre and post to compare scores before and after implied Number Head Together. The mean score of the pre-test was 49.06 at p = 1.364 and the post-test was 78.40 at p = 1.576 which was higher than 0.05 (as the standard significance level in SPSS). This value showed the differences before and after implementing Number Head Together. Hence, it can be concluded that Number Head Together was significantly effective in teaching writing in the tenth graders of Public Vocational High School (SMKN) 1 Janapria, Indonesia. However, The student's writing improves because in grouping the students able to help each other. Keywords:  Number Head Together (NHT), EFL Classroom,  Teaching Writing


Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 27-28
Author(s):  
Artem A. Gusak ◽  
Kirill V. Lepik ◽  
Natalia B. Mikhailova ◽  
Elena Kondakova ◽  
Yuri R. Zalaylov ◽  
...  

Background.Tumor tissue in classical Hodgkin Lymphoma (cHL) contains 1-10% malignant Hodgkin/Reed-Sternberg cells and a significant number of immune cells in the tumor microenvironment that are characterized by expression of inhibitory molecules (PD-1, CTLA-4, LAG-3, TIM-3, TIGIT). Despite overall effectiveness of anti-PD-1 treatment many patients still have relapsed or refractory (r/r) disease, therefore the search for predictive/prognostic biomarkers in patients on immunotherapy is highly demanded. Materials and methods.The study included 39 primary tumor specimens from patients with r/r cHL obtained before starting the treatment with nivolumab (primary biopsies). Specimens from 11 patients were studied before and after treatment (sequential biopsies). Treatment response was evaluated by PET-CT according to LYRIC criteria. Immunohistochemical staining for CD68, CD163, PD-1, LAG-3, TIM-3, CTLA-4, TIGIT was performed with an automated staining system (Bond III; Leica Biosystems). The slides were scanned with Aperio ScanScope XT (AperioTechnologies Inc.) and were analyzed with ImageScope Analysis software (Aperio Technologies) и Qupath (https://qupath.github.io). We explored progression-free survival (PFS) depending on the proportion of cells positive for CD68, CD163, PD-1, LAG-3, TIM-3, CTLA-4, TIGIT in the tumor microenvironment and analyzed the changes of these parameters between primary and sequential biopsies after treatment with nivolumab. Statistical analysis was performed using SPSS software (v.23). Data on sequential biopsies were analyzed with the Wilcoxon signed-rank test. PFS was calculated with the Kaplan-Meier method. The significance level was p ≤ 0.05. Results.A significant correlation was found in primary biopsies group between the value of CD163 and CTLA-4 (correlation coefficient -0,62, p < 0.05). There was no significant association between PFS and proportion of cells positive for CD68, PD-1, TIM-3, CTLA-4, TIGIT, LAG-3 in primary biopsies group. ROC analysis allowed to establish a 9% cut-off value of CD163 expression, dividing these patients into subgroups of CD163high and CD163low. In the CD163low group, the two-year PFS was 19,1% (95% CI 6%-37,7%) with a median PFS of 8,8 months (95% CI 5,7-12) and in the CD163high group - 53,8% (95% CI 28,4%-73,7%) with a median of 24,8 months (95% CI 18,8 - 39,2). In sequential biopsies, a statistically significant increase in numbers of PD-1+ and TIGIT+ T-cells and depletion of CD68+ and CD163+ cells was observed compared to corresponding cell counts in primary biopsies (median PD-1 - 3% vs 10%; median TIGIT - 10% vs 14%; median CD68 - 10% vs 7%; median CD163- 8% vs 3,5%; р <0,05). Conclusion.A comprehensive analysis of expression of CD68, CD163, LAG-3, TIGIT, CTLA4, TIM-3, PD-1 was performed in patients with r/r cHL before and after treatment with nivolumab. Significant association was found between the expression of CD163 and CTLA4. The results of the study indicate inferior PFS among patients with low expression (<9%) of CD163 in lymph node samples before immunotherapy. Biopsies taken after treatment with nivolumab showed a statistically significant increase in the number of PD-1+ and TIGIT+ cells and a decrease in the number of CD68+ and CD163+ cells compared with data from primary biopsies. The results of the study may contribute to our knowledge regarding biology of classical Hodgkin lymphoma and the mechanisms of resistance to therapy with immune checkpoints inhibitors. This study was supported by BMS research grant CA209-8EG Disclosures Ionova: Takeda:Other: principal investigator of the observational studies sponsored by Takeda;BMS:Other: principal investigator of the observational studies sponsored by BMS.


2017 ◽  
Vol 35 (15_suppl) ◽  
pp. e13527-e13527
Author(s):  
Keeratikarn Boonyawan ◽  
Jie Yang ◽  
Lihong Long ◽  
Qianghu Wang ◽  
Ravesanker Ezhilarasan ◽  
...  

e13527 Background: Thrombocytosis is associated with poor survival in several malignancies and is both triggered by and promoting of tumor growth. We hypothesized that an increase in circulating platelets following standard temozolamide (TMZ) CRT and/or adjuvant TMZ would predict for decreased survival in patients with glioblastoma (GBM). Methods: We reviewed data of 122 patients with newly diagnosed, pathological-proven GBM from 2007 to 2016 with documented complete blood counts before the start of CRT, after CRT, before adjuvant TMZ, and after a minimum 2 cycles of adjuvant TMZ. The association between changes in blood count levels and patient survival was analyzed by log-rank test. Multivariate analysis was performed using known prognostic co-variates, including MGMT methylation, patient age, and performance status (PS). Results: The mean change in platelets before and after CRT was -46 k/µl (range -387 to 179 k/µl) and before and after adjuvant TMZ was -25 k/µl (range -408 to 143 k/µl). As a reference, the mean change in lymphocytes was -5.7 k/µl (range -0.7 to 1.3 k/µl) and -0.0 k/µl (range -1.6 to 0.9 k/µl) for CRT and adjuvant TMZ, respectively. Patients were dichotomized based on the relative change in platelets and lymphocytes from baseline (≤30% increase, “low”, n = 101 vs > 30% increase, “high”, n = 12). The median survival of low patients vs. high patients was 30 vs 13 months (p = 0.006). Potential confounders, such as infection or thrombosis, were not associated with the high group. However, no significant survival difference was observed between groups based on platelet changes during adjuvant TMZ. Similarly, changes in lymphocyte counts were not significantly prognostic. In multivariate analysis, extent of resection, MGMT status, PS and the increase in platelets after CRT were significantly associated with survival (HR for platelets 4.0, 95% CI 1.6-10.1). Conclusions: Increased platelet counts after CRT predict for poor survival in patients with GBM. The effect is platelet specific and does not reflect general bone marrow changes, as lymphocyte changes were not significantly prognostic. These results suggest a potential interaction between platelets and tumor aggressiveness.


2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Elias Shiferaw ◽  
Fadil Murad ◽  
Mitikie Tigabie ◽  
Mareye Abebaw ◽  
Tadele Alemu ◽  
...  

Abstract Background Visceral leshimaniasis is a parasitic disease characterized by systemic infection of phagocytic cells and an intense inflammatory response. The progression of the disease or treatment may have an effect on hematological parameters of these patients'. Thus, the current study sought to compare the hematological profiles of visceral leishmaniasis patients before and after treatment with anti-leishmaniasis drugs. Method An institutional-based retrospective cohort study was conducted among visceral leishmaniasis patients admitted to the University of Gondar comprehensive specialized referral hospital leishmaniasis research and treatment centre between September 2013 and August 2018. Hematological profiles were extracted from the laboratory registration book before and after treatment. Data were entered to Epi-info and exported to SPSS for analysis. Descriptive statistics were summarized using frequency and percentage to present with the table. The mean, standard deviation, median, and interquartile range were used to present the data. Furthermore, using the paired t-test and the Wilcoxon Signed rank test, the mean difference for normally and non-normally distributed data was compared. Spearman and Pearson correlation analysis were used to describe the relationship between hematological parameters and various variables. A P value of 0.05 was considered statistically significant. Result With the exception of the absolute neutrophil count, all post-treatment hematological parameters show a significant increase when compared to pre-treatment levels. Prior to treatment, the prevalence of anemia, leukopenia, and thrombocytopenia was 85.5, 83.4, and 75.8%, respectively, whereas it was 58.3, 38.2, and 19.2% following treatment. Furthermore, parasite load was found to have a statistically significant negative correlation with hematological profiles, specifically with white blood cell and red blood cell parameters. Conclusion According to our findings, patients with visceral leishmaniasis had improved hematological profiles after treatment. The effect of treatment on parasite proliferation and concentration within visceral organs, in which the parasite load could directly affect the patient's hematological profiles, may be associated with the change in hematological profiles.


2019 ◽  
Vol 3 (4) ◽  
pp. 204
Author(s):  
I Ketut Labir ◽  
Ni Luh Kompyang Sulisnadewi ◽  
I Nyoman Ribek

Cases of sexual violence and abuse in Indonesia are increasingly common. Not only in adults, children are now victims. Some cases of victims or perpetrators involve underage children. Many parents feel taboo to talk about early childhood education with children. This causes children not to get information that is supposed to protect themselves from sexual violence. Need to do various efforts to provide education to children about knowledge to protect themselves from sexual violence. The purpose of this study was to determine the effect of sexual violence education on children on efforts to protect from sexual violence. This study uses pre-experimental design with approach pretest-posttest design. The number of samples is 195 children taken by purposive sampling technique. Analyze data using  different Wilcoxon Signed Rank test with 95% significance level, p ≤ (0.05). The results showed that the average age of the respondents was 10.2 years and most of the men were 52.8%. Before giving education, the average score of respondents' knowledge about efforts to protect themselves from sexual violence was 6.7 and increased to 7.79 after giving education. There is a difference in knowledge about efforts to protect themselves from sexual violence before and after being given education (p value ≤ 0.001; <α = 0.05). It is recommended that the provision of information about sexual violence be given regularly by teachers and parents.Keywords: Education - Elementary School Age Knowledge - Efforts to Protect Themselves from Sexual Violence


2019 ◽  
Vol 2 (2) ◽  
pp. 147-158
Author(s):  
Faisal Adnan Reza ◽  
Rahma Widyana

AbstractThis study aims to determine the effect of empathy training to improve forgiveness for female students who are victims of violence in dating relationships. Research subjects were 5 female students who were victims of violence in dating relationships. This study was only an experimental group, taken from the results of the pre-test on the empathy and forgiveness scale in the low and medium categories and interviews. The research design used was the experimental design of one group group pretest-posttest design. Data collection tools used in the form of empathy, forgiveness, observation and interviews. Analysis of the data used is the Wilcoxon Rank Test to see the difference in experimental group scores between before and after treatment. The Wilcoxon Rank Test found that there was a difference in the forgiveness score in the experimental group after giving empathy with a Z = -2.023 with a significance of 0.043 (p <0.05) with the mean pre-test (X test = 36.40) lower than the mean post-test (X ̅ = 63.40). The conclusion of this study is that there are differences in forgiveness scores between before and after being given empathy training.


2021 ◽  
Vol 9 (B) ◽  
pp. 1570-1574
Author(s):  
Imam Hafidh Zaini ◽  
Widyanti Soewoto ◽  
Ida Bagus Budhi

AIM: This study aims to evaluate the effect of adjuvant chemotherapy on estradiol levels in patients with HER 2-overexpression breast cancer in a developing country. METHODS: This comparative study with pre- and post-design model observation approach, involving patients with HER 2-overexpression breast cancer who had undergone surgery and had never received chemotherapy or hormonal therapy before, who were then given adjuvant chemotherapy. Estradiol levels were measured before and after chemotherapy. The study was carried out in the surgical oncology division of RSUD Dr. Moewardi (RSDM) Surakarta from January 2020-December 2020. Descriptive data are presented in a frequency table based on age, menstrual status, parity status, breastfeeding status, contraception, contraception duration, family history, stage, and histological grade. Before and after chemotherapy in patients with breast cancer, the estradiol levels employed the paired sample t-test of the Wilcoxon rank test because the data did not meet the normality assumption. RESULTS: From the total data of 21 patients, 15 patients experienced a decrease in estradiol levels after chemotherapy, while six patients underwent an increase. The mean estradiol level before chemotherapy was 89.41 pg/ml, whereas the mean estradiol level after chemotherapy was 55.90 pg/ml. It indicates a difference in the decrease in estradiol levels of 33.51 pg/ml. The statistical test results also obtained a p-value of = 0.033 (p < 0.05), which signifies a significant difference between estradiol levels before and after chemotherapy. Thus, chemotherapy is effective in lowering estradiol levels in patients with breast cancer. CONCLUSION: Chemotherapy affects decreasing estradiol levels in patients with HER2 overexpression breast cancer.


2014 ◽  
Vol 40 (5) ◽  
pp. 528-534 ◽  
Author(s):  
Tanyse Bahia Carvalho Marques ◽  
Juliana de Carvalho Neves ◽  
Leslie Andrews Portes ◽  
João Marcos Salge ◽  
Edmar Zanoteli ◽  
...  

OBJECTIVE: Respiratory complications are the main causes of morbidity and mortality in patients with neuromuscular disease (NMD). The objectives of this study were to determine the effects that routine daily home air-stacking maneuvers have on pulmonary function in patients with spinal muscular atrophy (SMA) and in patients with congenital muscular dystrophy (CMD), as well as to identify associations between spinal deformities and the effects of the maneuvers. METHODS: Eighteen NMD patients (ten with CMD and eight with SMA) were submitted to routine daily air-stacking maneuvers at home with manual resuscitators for four to six months, undergoing pulmonary function tests before and after that period. The pulmonary function tests included measurements of FVC; PEF; maximum insufflation capacity (MIC); and assisted and unassisted peak cough flow (APCF and UPCF, respectively) with insufflations. RESULTS: After the use of home air-stacking maneuvers, there were improvements in the APCF and UPCF. In the patients without scoliosis, there was also a significant increase in FVC. When comparing patients with and without scoliosis, the increases in APCF and UPCF were more pronounced in those without scoliosis. CONCLUSIONS: Routine daily air-stacking maneuvers with a manual resuscitator appear to increase UPCF and APCF in patients with NMD, especially in those without scoliosis.


2020 ◽  
Vol 14 (2) ◽  
pp. 139
Author(s):  
Ihda Mauliyah ◽  
Ratih Indah Kartikasari

Breastfeeding Self Efficacy (BSE) is a mother's belief regarding her ability to breastfeed her baby and decide whether the mother chooses to breastfeed or not. Aisyiyah through the Health Council has a program to increase the level of public health, especially women, infants, and children based on health services and community based on the spirit of al-Ma'un by increasing efforts to reduce maternal mortality through various activities and increasing efforts to reduce the numbers of infant and toddler deaths with one of priority which is exclusive breastfeeding through qoryah Thoyyibah program held by Balai Sakinah Aisyiyah. The design this study used Pre Experimental design with One Group Pretest-Posttest approach. Samples were pregnant women trimester III until the period of the puerperium (6 weeks) in the sub-district now Lamongan pregency of 20 postpartum mothers. Initial assessment was conducted to assess Early Breasfeeding Self Efficacy in pregnant women. The intervention was carried out by providing health care education by holding Mubalighot health motivators so that they were able to educate postpartum mothers. Furthermore, an Early Breasfeeding Self Efficacy assessment was reconducted. Data were analyzed by Wilcoxon Sign Rank test using SPSS.25. The results showed that the significance level (p) = 0.046 <α = 0.05 Ho was rejected which means that there was a significant influence between Breasfeeding Self Efficacy before and after conducting Mubalighot health motivator. This shows that the role of Mubalighot health motivator for Aisyiyah


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