BMI changes in paediatric type 1 narcolepsy under sodium oxybate treatment

SLEEP ◽  
2021 ◽  
Author(s):  
Virginia Ponziani ◽  
Fabio Pizza ◽  
Corrado Zenesini ◽  
Luca Vignatelli ◽  
Andrea Pession ◽  
...  

Abstract Paediatric Type 1 Narcolepsy (NT1) is often associated with overweight and obesity. Sodium oxybate (SO), approved for the treatment of narcolepsy with cataplexy from the age of 7 years old in US, has been associated with weight loss, although longitudinal paediatric studies are lacking. We report a retrospective cohort of 129 consecutive patients with a 4 years follow-up, to analyse the impact of different pharmacological treatments on BMI z-score. At baseline the prevalence of obesity and overweight was 26.4 % (34/129) and 29.5% (38/129), respectively. Patients were divided in 3 groups: children treated with SO alone (group 1), with SO-combined therapy (group 2), and without SO (group 3). At the end of the first year of follow-up, group 1 and group 2 showed a significant BMI z-score reduction compared to baseline: from 1.2±1.1 to 0.4±1.4 for group 1 (p < 0.001), and from 1.4±1.1 to 1±1.3 for group 2 (p = 0.002), independently from baseline clinical features. In the second year only group 2 experienced a further and significant BMI z-score decrease (from 1.0±1.2 to 0.6 ± 1.2, p = 0.037). No further significant BMI z-score changes were observed in SO treated patients in the following years. Instead, children treated without SO developed a significant weight increase between the second and third year of therapy (BMI z-score from 0.3±0.9 to 0.5±0.9). In conclusion, SO treatment in paediatric NT1 is associated with a favourable weight reduction in the first year of treatment.

2021 ◽  
Vol 29 (1) ◽  
pp. 230949902199799
Author(s):  
Tianming Yu ◽  
Jichong Ying ◽  
Jianlei Liu ◽  
Dichao Huang ◽  
Hailin Yan ◽  
...  

Purpose: The study described a novel surgical treatment of Haraguchi type 1 posterior malleolar fracture in tri-malleolar fracture and patient outcomes at intermediate period follow-up. Methods: All patients from January 2015 to December 2017 with tri-malleolar fracture of which posterior malleolar fractures were Haraguchi type 1, were surgically treated in this prospective study. Lateral and medial malleolar fractures were managed by open reduction and internal fixation through dual incision approaches. 36 cases of Haraguchi type 1 posterior malleolar fractures were randomly performed by percutaneous posteroanterior screw fixation with the aid of medial exposure (group 1). And 40 cases were performed by percutaneous anteroposterior screw fixation (group 2). Clinical outcomes, radiographic outcomes and patient-reported outcomes were recorded. Results: Seventy-six patients with mean follow-up of 30 months were included. There were no significant differences in the mean operation time (81.0 ± 11.3 vs. 77.2 ± 12.4), ankle function at different periods of follow-up, range of motions and visual analog scale (VAS) at 24 months between the two groups ( p > 0.05). However, the rate of severe post-traumatic arthritis (Grade 2 and 3) and the rate of step-off rather than gap in radiological evaluation were lower in group 1 than that in group 2 ( p < 0.05). Conclusion: Using our surgical technique, more patients had good outcome with a lower rate of severe post-traumatic arthritis, compared with the group of percutaneous anteroposterior screw fixation. Percutaneous posteroanterior screw fixation can be a convenient and reliable alternative in treating Haraguchi type 1 posterior malleolar fracture.


2021 ◽  
Author(s):  
Aylin Karalezli ◽  
Sema Kaderli ◽  
Ahmet Kaderli ◽  
Cansu Kaya ◽  
Sabahattin Sul

Abstract Purpose: To compare the effect of intravitreal ranibizumab (IVR) or intravitreal dexamethasone implants (IVD) on regression of hyperreflective dots (HRDs) on optical coherence tomography (OCT) B-scan in patients with branch retinal vein occlusion (BRVO). Methods: 37 eyes of 37 patients with cystoid macular edema who received IVR or IVD and followed up for at least 12 months were included in this study. The patients were divided into three groups according to intravitreal treatment. Group 1 consisted of 12 eyes who received only IVD, group 2 consisted of 10 eyes who received only IVR on a pro re nata and group 3 consisted of 15 eyes who received both IVD and IVR. OCT parameters (CMT, number of HRDs, status of external limiting membrane (ELM) and ellipsoid zone (EZ)) and best-corrected visual acuity (BCVA) were compared between the groups over the follow-up time. HRDs were categorized as HRD in inner retinal layers (from the internal limiting membrane to the inner nuclear layer) or HRD in outer retinal layers (from the outer plexiform layer to the outer border of the photoreceptor layer).Results: There was no significant difference between groups in terms of BCVA, CMT, HRDs in the inner and the outer retinal layers at baseline visit. (p˃0.05 for all) Comparing the baseline values in all groups, a significant decrease was observed in CMT in the first year. (For group 1; p=0.013, group 2; p=0.010; group 3, p<0.001) The BCVA was significantly increased after 1 year in all groups. (p=0.001, p=0.006, p<0.001) The mean number of HRDs in inner and outer retinal layers were significantly decreased in group 1 and group 3. (For group 1; p<0.001, p=0.001, for group 3; p<0.001, p<0.001) However, there was no significant difference in terms of the mean number of HRDs in inner and outer retinal layers for group 2. (p=0.134, p=0.477) At the first year, the number of HRDs in inner and outer retinal layers was significantly lower in group 1 and group 3 than group 2. (For inner HRDs; group 1 vs. group 2 p=0.007, group 2 vs. group 3 p<0.001. For outer HRDs group 1 vs. group 2 p<0.001, group 2 vs. group 3 p<0.001.) The BCVA was higher in group 3 than group 2 at 1year. (p=0.048). There was no significant difference in terms of post-treatment CMT and the number of HRDs between group 1 and group3 in posthoc tests (p=0.621, p=0.876, and p=0.632).Conclusion: The reduction in HRDs at 12 months and better BCVA after IVD intimates that the HRDs should be considered as inflammatory markers in the follow-up of CME in BRVO. Thus, IVD injection could be more appropriate for patients with higher HRDs after BRVO.


2020 ◽  
Author(s):  
Chan Ho Park ◽  
Jun-Il Yoo ◽  
Chang Hyun Choi ◽  
You-Sung Suh

Abstract Background: Switching the prescription from bone-forming medication to resorptive agents is reportedly effective for patients with severe osteoporosis. The objective of this study is to determine the impact of implementing short-term teriparatide (TPTD) intervention before denosumab (DMab) therapy compared with DMab therapy alone for 1 year after hip fracture.Methods: TPTD was administered to 24 patients for an average of 12.1 weeks after which the intervention was switched to DMab therapy for 12 months (group 1). DMab alone was administered to 16 patients for 12 months (group 2). Bone mineral density (BMD) was evaluated before and after treatment at the 1-year follow-up. The improvement of BMD and T-score in hip and spine was compared with the levels of bone turnover marker.Results: The difference of hip BMD after osteoporosis treatment was -0.0081±0.03 in group 1 and 0.0074±0.04 in group 2 (p=0.180). The difference of spine BMD was 0.0819±0.04 in group 1 and 0.0145±0.03 in group 2 (p<0.001). BMD and T-score of the spine improved significantly in groups 1 and 2 (p < 0.001). There was no statistical difference in C-terminal telopeptide and osteocalcin level. Conclusion: Short-term TPTD administration followed by DMab alone was effective only in improving spine BMD. Short-term treatment with TPTD caused mild improvement in femur neck BMD compared with DMab alone. However, further research with a longer duration of TPTD treatment is warranted, as our findings lack statistical significance.


2021 ◽  
Vol 104 (6) ◽  
pp. 895-901

Background: Pulmonary arterial hypertension (PAH) is a common complication of congenital heart disease (CHD) with uncorrected left-to-right shunts. Currently, no consensus guideline exists on the management of PAH-CHD in children, especially those who do not meet operability criteria. Objective: To compare survival between three groups of high-risk PAH-CHD, group 1: total correction including both surgical and percutaneous intervention, group 2: palliative treatment, and group 3: conservative with medical treatment group. Materials and Methods: All pediatric patients with PAH-CHD that underwent cardiac catheterization between January 1, 2008 and December 31, 2017 were retrospectively reviewed. Inclusion criteria were high risk PAH-CHD patients who had pulmonary vascular resistance (PVR) greater than 6 Wood unit·m² and PVR-to-SVR ratio greater than 0.3 evaluated in room air. Exclusion criteria were younger than three months of age, severe left side heart disease with pulmonary capillary wedge pressure greater than 15 mmHg, obstructive total pulmonary venous return, and single ventricle physiology. The Kaplan-Meier analysis was performed from the date of PAH diagnosis to the date of all-cause mortality or to censored date at last follow-up. Results: Seventy-six patients with a median age at diagnosis of 27.5 months (IQR 14.5 to 69.0 months) were included in this study. The patients were divided into three subgroups and included 38 patients (50.0%) in group 1, six patients (7.9%) in group 2, and 32 patients (42.1%) in group 3. The median follow-up time was 554 days (IQR 103 to 2,133 days). The overall mortality was 21.7%. One-year survival in patients with simple lesion in group 1 and 3 were 79.5% and 87.5% and patients with complex lesions in group 1, 2, and 3 were 93.8%, 83.3%, and 73.1%, respectively. The results showed that most mortalities occurred in the first year. There were no statistically significant differences in survival among difference types of treatment (log rank test, p=0.522). Conclusion: The mortality of high-risk PAH-CHD patients were not different among those who underwent corrective surgery, palliative, or conservative treatment. The mortality was high in the first year after PAH diagnosis and remain stable afterward. Management decision for an individual with high-risk PAH-CHD patients requires comprehensive clinical assessment to balance the risks and benefits before making individualized clinical judgment. Keywords: Pulmonary hypertension; Congenital heart disease; High-risk patients


Author(s):  
Fatih Akova

Objective: The aim of this study is to present the frenotomy technique in cases of ankyloglossia (tongue-tie), which is diagnosed and treated in the first year of life. Patients were operated by a single surgeon. Method: Cases of ankyloglossia operated at Biruni University Faculty of Medicine Hospital Pediatric Surgery Clinic Between 2016-2020 were evaluated retrospectively in terms of clinical complaints, age, type of ankyloglossia, surgical technique, indications and results of surgery. Results: Frenotomy was performed in 56 patients including 47 boys (84%) and 9 girls (16%), between the ages of 0-1. Average age of the patients was 93 days (1-360). Patients were divided into two groups as Group 1 (n: 40: 0-90 days old) and Group 2 (n: 16: 90-360 days old). Patients were admitted with complaints of having difficulty in sucking the mother’s breast, not being able to take their tongue out, feeding with a bottle, pain at the nipple and not being able to grasp the breast. No additional intervention was required for bleeding in Group 1, and in 12 (75%) patients in Group 2 bleeding control was achieved using bipolar cautery. During follow-up, significant improvement was obtained in all patients who had difficulty in sucking and gripping the nipple. Improvement was observed in 15 of 25 patients with nipple pain. Conclusion: Frenotomy is an easily applied surgical procedure with minimal complications. Additional application may be required for bleeding control in infants older than 3 months. It should be considered that the probability of recurrence may depend on the type, intervention used and thickness of the frenulum, and phrenotomy may not be sufficient. The improvement in breast feeding function of Frenotomy may provide a significant improvement in the complaints of nipple pain, and may contribute to the emotional attachment between the mother and her baby. Randomized controlled trials are required to determine the effects of phrenotomy.


2005 ◽  
Vol 71 (11) ◽  
pp. 920-930 ◽  
Author(s):  
M.L. Hawkins ◽  
F.D. Lewis ◽  
R.S. Medeiros

The purpose of this study was to compare the functional outcomes of two groups of patients with traumatic brain injury (TBI) with attention to the impact of reduced length of stay (LOS) in the trauma center (TC) and rehabilitation hospital (RH). From 1991 to 1994, 55 patients, Group 1, with serious TBI (Abbreviated Injury Scale score ≥3) were admitted to a level 1 TC and subsequently transferred to a comprehensive inpatient RH. These results have been previously published. From 1996 to 2002, 64 similarly injured patients, Group 2, received inpatient care at the same TC and RH. These patients had a marked decrease in length of stay. Functional Independence Measures (FIM) were obtained at admission (Adm), discharge (D/C), and at 1 year follow-up for both groups. The average length of stay at the TC dropped from 36 days in Group 1 to 26 days in Group 2. In addition, the average length of stay at the RH dropped from 46 days (Group 1) to 25 days (Group 2); overall, an average reduction of 31 days of inpatient care. Group 2 had significantly lower FIM scores at the time of RH discharge for self-care, locomotion, and mobility compared to Group 1. At the 1 year follow-up, however, there were no significant differences between Groups 1 and 2 in these FIM scores. FIM scores at 1 year were higher in Group 2 for communication (90% vs 71%) and social cognition (77% vs 49%) compared to Group 1. Over one-fourth of each group returned to work by the 1 year follow-up. Socially disruptive behavior occurred at least weekly in 28 per cent (Group 1) and 23 per cent (Group 2) of patients. The outcome for serious TBI is better than generally perceived. Reduction of inpatient LOS did not adversely affect the ultimate functional outcome. The decreased LOS placed a greater demand on outpatient rehabilitative services as well as a greater burden on the family of the brain-injured patient


Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 1223-1223
Author(s):  
Alessandro Corso ◽  
Silvia Mangiacavalli ◽  
Luciana Barbarano ◽  
Annalisa Citro ◽  
Paola Brasca ◽  
...  

Abstract Abstract 1223 Poster Board I-245 Introduction This study aimed at evaluating the impact of three different pre-transplant therapies on the outcome of patients (pts) eligible for high-dose therapy. Methods two-hundred sixty eight newly diagnosed MM pts aged £65 years, Durie-Salmon stage III, II, or I in progression, were consecutively enrolled from 2000 to 2007 in three different protocols, with three different pre-transplant therapy: Group 1: (145 pts) 3 pulse-VAD cycles; Group 2: (67 pts) 3 pulse-VAD cycles plus 3 Thal-Dex cycles (thalidomide at the dose of 100 mg/day orally at bedtime, continuously for 3 months, oral dexamethasone at the dose of 20 mg on days 1-4 and 14-17 every 28 days); Group 3: (57pts) 4 Vel-Dex courses (Bortezomib 1.3 mg/m2 i.v. on days 1, 4, 8, 11; oral Dexamethasone 40 mg on days 1-4 and 8-11 every 3 weeks). After induction all pts received two DCEP-short cycles as mobilization (oral Dexamethasone 40 mg/day on days 1-4 + Cyclophosphamide 700 mg/m2/day i.v., Etoposide 100 mg/ m2/day i.v., cisPlatin 25 mg/m2/day for 2 days) with peripheral blood stem-cell (PBSC) collection prompted by G-CSF followed by one or two transplants (Tx) with melphalan 200 mg/m2 as conditioning regimen. Response was defined according to IMWG uniform criteria. Pts were considered responsive when obtaining at least a PR. Results pts in the three group were similar for age, gender, Ig type, ISS stage. A significant higher percentage of Durie and Salmon stages III was found in group 3 (83% vs 68% in group 1 and 67% in group 2, p=0.0002). The median follow-up was 46 (1-150) months for group 1, 43 (1-68) months for group 2, and 29.7 (1-79) months for group 3. At the time of this analysis in the three groups 51%, 65%, 90% of transplanted pts respectively were still alive, and progression after transplant was registered in 84%, 80%, 50% respectively. Patient flow before Tx was similar (p=0.45): 19% in group 1, 27% in group 2, 23% in group 3. In group 1, 2% of pts went off-study after VAD, and 17% after mobilization phase. In group 2, patient flow was equally distributed: 7% after pulse VAD, 10% after thal-dex, 9% after DCEP. In group 3, 12% of the pts went off-study after Vel-Dex, 11% after DCEP. Table 1 summarized responses. In group 3 (Vel-Dex) response was better along all protocol phases with respect to group 1 or 2 (p<0.00001). The number of responsive pts progressively increased from 87% after Vel-Dex (CR 31%), to 96% after transplant (CR 38%). Response rates of group 1 and 2 patients were not significantly different either after induction (p=0.6), after DCEP (p=0.5), and after Tx (p=0.65). On intention to treat basis, vel-dex induction produced a better, although not significant, PFS (34.6 months vs 29 in group 1 and 26.8 in group 2, p=0.56). OS were not statistically different among the three groups, event though the different follow-up could affect the analysis (median OS 110 in group 1, 66 months in group 2, and not reached in group 3, p=0.37). In multivariate analysis PFS was improved only by the achievement of CR (p=0.001). No significant difference was observed between VGPR or PR (p=0.43). Conclusion In this study, only CR not VGPR impacts on the outcome. Vel-Dex producing a significant high CR rate after TX (38%), seems to improve survival of MM patients candidate to high-dose therapy with respect to conventional pre-transplant strategies. Disclosures Morra: Roche:.


2021 ◽  
Vol 1 ◽  
pp. 14-19
Author(s):  
Archana Madu ◽  
Hemchand Krishna Prasad ◽  
Anand Thiagarajan ◽  
Kannan Narayanasamy ◽  
Nedunchelian Krishnamoorthy

Objectives: There is a paucity of data on impact of therapy of Hashimoto’s thyroiditis (HT) on catch-up growth. The objective of the study was to determine whether delayed diagnosis of HT and overt primary hypothyroidism has an impact on the catch-up of children and adolescents. Material and Methods: We conducted a prospective observational study over 3 years, in the thyroid clinic of a referral unit in South India. We assessed chronological age (CA), auxological parameters, clinical presentation, bone age (BA), and predicted adult height (PAH) in subjects with Hashimoto’s thyroiditis and overt primary hypothyroidism. Management and follow-up done as per standard protocols and study parameters reassessed after 1 year of therapy. Results: We recruited 38 subjects and divided them into two groups – Group 1 with BA within 2 standard deviations (SD) of CA (n = 20) and Group 2 beyond 2 SD (n = 18). During 1 year follow-up, height Z-scores were −0.1 ± 1.4 (baseline) and −0.1 ± 1.1 (endpoint) and −1.7 ± 1.7 (baseline) and −1.3 ± 1.3 (endpoint) in Groups 1 and 2, respectively. BA: CA ratio changed from 1.0 ± 0.1 to 1.0 ± 0.1 in Group 1 (P > 0.05) versus 0.7 ± 0.2 to 0.9 ± 0.1 in Group 2 (P < 0.05). The number of children who were pre-pubertal: pubertal changed from 15:5 to 11:9 in Group 1 and 14:4 to 7:11 in Group 2. For Group 1, baseline PAH Z score was −0.5 ± 1.7 and endpoint PAH Z score was −0.7 ± 1.6 versus a target height Z score of −1.1 ± 1.1 (P > 0.05); Group 2, the baseline PAH Z score −1.1 ± 1.6 and endpoint PAH Z score −2.2 ± 1.4 versus target height Z-score of −0.4 ± 1.7. Conclusion: Delayed diagnosis and treatment of juvenile autoimmune hypothyroidism results in permanent loss of height potential.


2018 ◽  
Vol 4 (Supplement 2) ◽  
pp. 114s-114s
Author(s):  
I. Sallam ◽  
G. Amira ◽  
A. Youssri

Background: According to the cultural and socioeconomic factors, breast cancer patients (BCP) experienced a relatively low health-related quality of life (Qol) during the journey of breast cancer treatment, that influence patient adaptation to the situation from diagnosis to after treatment. And the further effect on either starting the treatment or its continuation. This merited conducting a qualitative study to explore the importance of the psychooncological approach to BCP and the impact on commencing and continuation of treatment and follow-up. Aim: To describe the impact and importance of the psychooncology team in the cancer center and the effect of their approach on the BCP's Qol. Methods: The study involved 114 interviewed participants, excluding patients with wide variety of chronic illnesses, only 91 patients are the focused study group. Of which 11 patients group (1) refused to join the psychooncological approach due to cultural, and socioeconomic issues, and 80 patients group (2) joined and followed up by the team. Both groups are regularly surveyed. Qualitative and quantitative measurements were used. Data were collected as follows, at time of diagnosis, after surgery and after chemo-radiation therapies from group (1) and for group (2) after the psychooncology team management and care. Developing the psychooncology team for the cancer center. 2 psychooncologists, 5 psychoeducating nurses and a group therapy sessions at a world standard levels of care with ethics committee approvals, and caring for patients' privacy. Close follow-up and evaluation of the performance and Qol of our BCP, raising the awareness about psychoeducation and psychological approach importance for BCP that would help them cope with daily life challenges to improve Qol. Results: Category Group N. Diagnosis related depression N. Surgery related depression N. Treatment related depression Free of depression Group (1) No= 11 3 (27%) 2 (18%) 5 (45%) 1 (10%) Group (2) No= 80 12 (15%) 9 (11.3%) 23 (28.7%) 36 (45%) Conclusion: Results indicate that the most critical depressing points is at time of diagnosis and chemo-radiation therapy. Group (2) has positive indication and alternation on the level of Qol and a significant improvement on level of depression when compared with group (1). This study highlighted the importance of psychooncology team in BCPs' survivorship. It also brings to attention the important role of the government, health policy makers and health plans toward enriching all cancer centers with the psychooncology team. To maximize health and health care for BCP.


VASA ◽  
2011 ◽  
Vol 40 (3) ◽  
pp. 199-204 ◽  
Author(s):  
Xia ◽  
Yang ◽  
Qu ◽  
Cheng ◽  
Wang

Background: This study was designed to investigate the impact of carotid artery stenting (CAS) on plasma levels of P-selectin, von Willebrand (vWF) and endothelin-1. Patients and methods: Sixty-seven patients who received CAS were divided into group 1 (one stent for a simple lesion, n = 38) and group 2 (two stents for complex lesions, n = 29). The levels of P-selectin, vWF and endothelin-1 were measured before CAS, 1 h, 6h, 24 h and 2 weeks after the stenting. Results: Sixty-one patients completed one-year follow up. Restenosis was noted in 14 (23 %) patients, among these three (4.8 %) had a restenosis of > 50 % of the vascular lumen. In all patients, the levels of P-selectin, vWF and endothelin-1 increased immediately after CAS (P < 0.05 or < 0.01). The levels of vWF and endothelin-1 in group 2 were higher than in group 1 (P < 0.05 or 0.01). There was no significant difference in P-selectin and endothelin-1 between the restenosis and non-restenosis group (P > 0.05). The 24 h vWF in patients with restenosis were higher than in non-restenosis group (P < 0.05). Conclusions: CAS results in a significant increase in plasma P-selectin, vWF and endothelin-1. The post-CAS levels of P-selectin, vWF and endothelin-1 are related to the extent of endothelial injury. Whether they are associated with restenosis 12 months after the treatment requires further investigation.


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