scholarly journals P48 Prescribing patterns of antiasthma medication in children and adolescents in primary care in france

2019 ◽  
Vol 104 (6) ◽  
pp. e37.1-e37
Author(s):  
I Naiim ◽  
F Tubach ◽  
S Guillo ◽  
A Ajrouche ◽  
V Houdouin ◽  
...  
Keyword(s):  
Primary Care ◽  
Children And Adolescents ◽  
Prescribing Patterns ◽  
First Year ◽  
Asthmatic Children ◽  
Healthcare Insurance ◽  
Short And Long Term ◽  
Second Year

BackgroundLittle is known about the short and long-term therapeutic management of asthmatic children. The aim of this study was to assess the prescribing patterns of antiasthma drugs in primary care.MethodsThis is a retrospective cohort study performed between January 2011 and December 2017 using the EGB (Echantillon Généraliste de Bénéficiaires) database, a 1/97th sample of the French national healthcare insurance system. Claims data for all individuals aged from 5 to 18 years‘ old who had received at least one antiasthma drug in the study period without any delivery in the previous 24 months and with 24 months of follow-up after first delivery, were analysed.ResultsA total of 7,680 children and adolescents (68.6% aged 5–11, 31.4% aged 12–18 years) were delivered at least one antiasthma drug (ATC code R03) during study period. The majority (66%) did not redeem another prescription in the following year (occasional users), when 18.4% redeemed prescriptions twice (low users) and 15.6% ≥3 times (high users). Most users (67%) were delivered only one class of R03 per dispensing in the first year and short-acting β2-agonists (SABAs) were the most frequently dispensed drugs. However, 33.4% of users were not prescribed SABAs. During the second year, only 27% of first-year users redeemed R03 prescriptions: 15.8% among occasional users, 35.5% of low users and 64.7% of high users. Among low and high first-year users who redeemed R03 drugs during the second year, 39.7% did not use inhaled corticoids alone or in association to LABAs.ConclusionsA high proportion of children and adolescents that used antiasthmatic drugs, even on a regular basis, were not prescribed these drugs in the long term. This finding may correspond either to the widespread use of antiasthmatic drugs in indications other than asthma or to an important undertreatment of asthmatic children and adolescents.Disclosure(s)Nothing to disclose

scholarly journals 5-Year Angiographic and Clinical Follow-up of Coil-Embolised Intradural Saccular Aneurysms

2002 ◽  
Vol 8 (4) ◽  
pp. 349-366 ◽  
Author(s):  
LL Batista ◽  
J. Mahadevan ◽  
M. Sachet ◽  
H. Alvarez ◽  
G. Rodesch ◽  
...  
Keyword(s):  
First Year ◽  
Coil Embolisation ◽  
Short Term ◽  
Detachable Coil ◽  
Complete Occlusion ◽  
Guglielmi Detachable Coil ◽  
Short And Long Term ◽  
Arterial Aneurysms

The purpose of the paper is the follow-up of embolised intradural saccular Arterial Aneurysms (AA), excluding giant, dissecting, inflammatory, fusiform or AA associated to BVAM. Since its introduction in 1991, the Guglielmi Detachable Coil has offered protection against aneurysmal rebleeding in the critical few days and months after SAH regardless of the grade. A number of questions remain: is complete angiographic obliteration necessary at first embolisation? What duration of clinical / angiographic follow-up (FU) is required to ensure the risk of haemorrhage has been eliminated? What is the long-term protection against rebleeding? One hundred and two patients with 160 intradural saccular AA embolised before april 1997 were selected for this study. They had at least 5-yrs clinical FU, of which 22 patients had a mid-term (3 years) and 45 patients had a 5-year or more angiographic FU (mean 67,7 months per patient). Twenty-eight embolised AAs with 100% occlusion at 1 year, remained unchanged on the 5-year angiograms. A further 14 patients with complete occlusion at 1 year showed persisting complete occlusion on angiogram at 3-years FU, which in our series means that complete occlusion after the first year post-embolisation implies that the aneurysm will remain completely occluded. All secondary spontaneous thromboses (27.6% of cases), occurred during the first year pos-embolisation. In six patients with subtotal or partial occlusion no change was seen for three consecutive years of FU; none showed later change at 5-year angiography. Below 80% occlusion our series does not provide enough information but we consider the situation instable. No mortality related to the procedure was observed in the unruptured AA group. No bleeding or re-bleeding has occurred since the beginning of our experience (1993) in saccular AA treated by GDC-Coil. Coil-embolisation of properly selected patients is effective in protecting against bleeding or re-bleeding at short and long-term with stable morphological results provided a strict follow-up control is established at short term.

scholarly journals Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 2059-2059 ◽  
Author(s):  
Wolfgang Miesbach ◽  
Karina Meijer ◽  
Michiel Coppens ◽  
Peter Kampmann ◽  
Dr. Klamroth ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Research Funding ◽  
Factor Ix ◽  
First Year ◽  
Severe Hemophilia ◽  
Advisory Committees ◽  
The Mean ◽  
Second Year

Background: The aim of gene therapy is to provide long-term therapeutic effect from a single administration, yet information on response durability is currently limited. AMT-060 is an adeno-associated virus serotype 5 (AAV5) vector with a codon-optimized wildtype human factor IX (FIX) gene and liver-specific promoter. AMT-060 is being analyzed in an ongoing study of 10 participants with severe/moderate-severe hemophilia B (Phase 1/2 study, NCT02396342). Aim: To describe efficacy and safety outcomes from a planned interim analysis at up to 4-years post-AMT-060. Methods: Adult males with FIX activity ≤2% and a severe bleeding phenotype received a single intravenous infusion of AMT-060 (5x1012gc/kg, Cohort 1, n=5) or (2×1013 gc/kg, Cohort 2, n=5). Assessments included FIX activity, FIX replacement use, annualized bleeding rate (ABR), treatment-related adverse events (TRAE), immunological and inflammatory biomarkers up to 4 years (Cohort 1) and 3.5 years (Cohort 2). Results: As of 8 May 2019, for Cohort 1 the mean yearly FIX activity (annualized to 4 years) was 6.0 as compared to 4.4% in the first year, 6.8% in the second year and 7.3% in the third year. Mean yearly FIX activity for Cohort 2 at 3 years was 7.9% as compared to 7.1% in the first year and 8.4% in the second year. Factor IX activity for each patient over the length of follow up is shown in Figure 1. Eight of 9 participants using prophylaxis at baseline were able to discontinue use. During the last 12 months of observation, the mean annualized bleed rate (ABR) was 1.7 for Cohort 1 and 0.7 for Cohort 2. Respectively, these represent a reduction in mean ABR to the year prior to treatment of 88% and 83%. During this same period the consumption of FIX replacement therapy declined 93% and 96% relative to pre-treatment respectively for Cohort 1 and Cohort 2. No participants developed FIX inhibitors or signs of sustained AAV5 capsid-specific T-cell activation. TRAE were mainly reported in the first 3.5-months after treatment, including three participants who experienced transient mild elevations in alanine aminotransferase as previously described. One new TRAE (joint swelling post-exercise) was observed during the last 12 months of observation post-treatment. Updated data, up to 4-years of observation, will be presented for the first time. Conclusions: Long-term stable endogenous FIX activity and reductions in ABR and FIX replacement use were observed following a single treatment with AMT-060. There were no additional safety concerns with longer term follow-up. These findings support the ongoing Phase III study of the enhanced construct, AMT-061, which encodes the highly active Padua FIX variant. Figure 1 Disclosures Miesbach: Bayer, BioMarin, CSL Behring, Chugai, Freeline, Novo Nordisk, Octapharma, Pfizer, Roche, Takeda/Shire, UniQure: Consultancy; Bayer, Novo Nordisk, Octapharma, Pfizer, Takeda/Shire: Research Funding; Bayer, Chugai, Novo Nordisk, Octapharma, Pfizer, Takeda/Shire, UniQure: Speakers Bureau. Meijer:Pfizer, Sanquin, Uniqure: Research Funding; Uniqure, BMS, Aspen, Boehringer Ingelheim, Sanquin, Bayer: Consultancy, Honoraria; Sanquin: Research Funding; Bayer: Research Funding. Coppens:Pfizer: Honoraria; Portola Pharmaceuticals, Inc: Honoraria; Daiichi Sankyo: Honoraria, Research Funding; Uniqure: Research Funding; Boehringer Ingelheim: Research Funding; Sanquin Blood Supply: Research Funding; Bayer: Honoraria, Research Funding; CSL Behring: Honoraria, Research Funding; Bristol-Myers Squibb: Honoraria. Kampmann:Uniqure BV: Research Funding. Klamroth:Bayer, Biomarin, CSL Behring, Novo Nordisk, Octapharma, Pfizer, Roche, SOBI, Takeda: Consultancy; Bayer, Novo Nordisk, SOBI: Research Funding. Schutgens:Baxalta Shire, Novo Nordisk, Bayer, CSL Behring, Pfizer, UniQure BV: Research Funding. Castaman:Bayer: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Novo Nordisk: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Sobi: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; CSL Behring: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Takeda (SHIRE): Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Kedrion: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Roche: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Werfen: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Pfizer: Research Funding; Uniqure: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Sanofi: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Seifried:Medac: Other: BSD owns IP and is contract manufacturer; Uniqure BV: Research Funding. Schwaeble:Uniqure BV: Research Funding. Bönig:Celgene, Novartis, Sandoz Hexal: Consultancy; Kiadis Pharma: Other: Contract manufacturing of ATIR101; Sandoz Hexal, Uniqure: Research Funding; Miletenyi: Speakers Bureau. Sawyer:Uniqure BV: Employment. Leebeek:CSL Behring: Research Funding; UniQure: Consultancy; Shire/Takeda: Research Funding; Novo Nordisk: Consultancy; Sobi: Other: Travel grant; Shire/Takeda: Consultancy.

scholarly journals Lung function variability in children and adolescents (LUV study): protocol for a prospective interventional trial in healthy individuals and patients with asthma

2020 ◽  
Author(s):  
Eirini Sofia Frima ◽  
Ilias Theodorakopoulos ◽  
Dimos Gidaris ◽  
Nikolaos Karantaglis ◽  
Grigorios Chatziparasidis ◽  
...  
Keyword(s):  
Lung Function ◽  
Children And Adolescents ◽  
Forced Expiratory Volume ◽  
Healthy Children ◽  
Daily Fluctuation ◽  
Asthmatic Children ◽  
Short And Long Term ◽  
Central Database ◽  
Detrended Fluctuation

Abstract Background Variability analysis of peak expiratory flow (PEF) and forced expiratory volume at 1 s (FEV1) has been used in research to predict exacerbations in adult individuals with asthma. However, there is a paucity of data regarding PEF and FEV 1 variability in healthy or asthmatic children and adolescents. The objective of the present study is the assessment of PEF and FEV 1 variability in: a) healthy children and adolescents, to define the normal daily fluctuation of PEF and FEV 1 and the parameters that may influence it, and b) children and adolescents with asthma, to explore the differences from healthy subjects and reveal any specific variability changes prior to exacerbation.Methods The study will include 100 healthy children and adolescents aged 6 to 18 years (assessment of normal PEF and FEV 1 variability) and 100 children and adolescents of the same age with diagnosed asthma (assessment of PEF and FEV 1 variability in asthmatics). PEF and FEV1 measurements will be performed using an ultra-portable spirometer (MIR Spirobank Smart) capable to smartphone connection. Measurements will be performed twice a day between 07:00-09:00 and 19:00-21:00 hours and will be dispatched via email to a central database for a period of 3 months. PEF and FEV1 variability will be assessed by detrended fluctuation and sample entropy analysis, aiming to define the normal pattern (healthy controls) and to detect and quantify any deviations (asthmatics). The anticipated duration of the study is 24 months.Discussion Healthy children and adolescents may present normal short- and long-term fluctuations in lung function; the pattern of this variability may be influenced by age, sex and environmental conditions. Significant lung function variability may also be present in children and adolescents with asthma, but the patterns may differ from those observed in healthy children and adolescents. Such data would improve our understanding regarding the chronobiology of asthma and permit the development of integrated tools for assessing the level of control and risk of future exacerbations.Trial registration ClinicalTrials.gov, NCT04163146. Registered on 14 November 2019

Short- and Long-Term Results of Total vs Subtotal Thyroid- ectomies in the Surgical Treatment of Graves' Disease

Swiss Surgery ◽  
2001 ◽  
Vol 7 (1) ◽  
pp. 20-24 ◽  
Author(s):  
Robert ◽  
Mariéthoz ◽  
Pache ◽  
Bertin ◽  
Caulfield ◽  
...  
Keyword(s):  
Recurrent Laryngeal Nerve ◽  
Nerve Palsy ◽  
Recurrent Laryngeal Nerve Palsy ◽  
Laryngeal Nerve ◽  
Long Term Results ◽  
Graves Disease ◽  
Laryngeal Nerve Palsy ◽  
Short And Long Term

Objective: Approximately one out of five patients with Graves' disease (GD) undergoes a thyroidectomy after a mean period of 18 months of medical treatment. This retrospective and non-randomized study from a teaching hospital compares short- and long-term results of total (TT) and subtotal thyroidectomies (ST) for this disease. Methods: From 1987 to 1997, 94 patients were operated for GD. Thirty-three patients underwent a TT (mostly since 1993) and 61 a ST (keeping 4 to 8 grams of thyroid tissue - mean 6 g). All patients had received propylthiouracil and/or neo-mercazole and were in a euthyroid state at the time of surgery; they also took potassium iodide (lugol) for ten days before surgery. Results: There were no deaths. Transient hypocalcemia (< 3 months) occurred in 32 patients (15 TT and 17 ST) and persistent hypocalcemia in 8 having had TT. Two patients developed transient recurrent laryngeal nerve palsy after ST (< 3 months). After a median follow-up period of seven years (1-15) with five patients lost to follow-up, 41 patients having had a ST are in a hypothyroid state (73%), thirteen are euthyroid (23%), and two suffered recurrent hyperthyroidism, requiring completion of thyroidectomy. All 33 patients having had TT - with follow-ups averaging two years (0.5-8) - are receiving thyroxin substitution. Conclusions: There were no instances of persistent recurrent laryngeal nerve palsy in either group, but persistent hypoparathyroidism occurred more frequently after TT. Long after ST, hypothyroidism developed in nearly three of four cases, whereas euthyroidy was maintained in only one-fourth; recurrent hyperthyroidy was rare.

Muenke syndrome: long-term outcome of a syndrome-specific treatment protocol

2019 ◽  
Vol 24 (4) ◽  
pp. 415-422 ◽  
Author(s):  
Bianca K. den Ottelander ◽  
Robbin de Goederen ◽  
Marie-Lise C. van Veelen ◽  
Stephanie D. C. van de Beeten ◽  
Maarten H. Lequin ◽  
...  
Keyword(s):  
Treatment Protocol ◽  
First Year ◽  
Ventricular Size ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Skull Growth ◽  
Muenke Syndrome ◽  
First Year Of Life

OBJECTIVEThe authors evaluated the long-term outcome of their treatment protocol for Muenke syndrome, which includes a single craniofacial procedure.METHODSThis was a prospective observational cohort study of Muenke syndrome patients who underwent surgery for craniosynostosis within the first year of life. Symptoms and determinants of intracranial hypertension were evaluated by longitudinal monitoring of the presence of papilledema (fundoscopy), obstructive sleep apnea (OSA; with polysomnography), cerebellar tonsillar herniation (MRI studies), ventricular size (MRI and CT studies), and skull growth (occipital frontal head circumference [OFC]). Other evaluated factors included hearing, speech, and ophthalmological outcomes.RESULTSThe study included 38 patients; 36 patients underwent fronto-supraorbital advancement. The median age at last follow-up was 13.2 years (range 1.3–24.4 years). Three patients had papilledema, which was related to ophthalmological disorders in 2 patients. Three patients had mild OSA. Three patients had a Chiari I malformation, and tonsillar descent < 5 mm was present in 6 patients. Tonsillar position was unrelated to papilledema, ventricular size, or restricted skull growth. Ten patients had ventriculomegaly, and the OFC growth curve deflected in 3 patients. Twenty-two patients had hearing loss. Refraction anomalies were diagnosed in 14/15 patients measured at ≥ 8 years of age.CONCLUSIONSPatients with Muenke syndrome treated with a single fronto-supraorbital advancement in their first year of life rarely develop signs of intracranial hypertension, in accordance with the very low prevalence of its causative factors (OSA, hydrocephalus, and restricted skull growth). This illustrates that there is no need for a routine second craniofacial procedure. Patient follow-up should focus on visual assessment and speech and hearing outcomes.

Long-Term Follow-Up of Early Cochlear Implant Device Activation

2021 ◽  
pp. 1-11
Author(s):  
Stefanie Bruschke ◽  
Uwe Baumann ◽  
Timo Stöver
Keyword(s):  
Speech Recognition ◽  
Side Effects ◽  
Cochlear Implant ◽  
Surgical Techniques ◽  
Control Group ◽  
First Year ◽  
Safe Procedure ◽  
Sound Processor

Background: The cochlear implant (CI) is a standard procedure for the treatment of patients with severe to profound hearing loss. In the past, a standard healing period of 3–6 weeks occurred after CI surgery before the sound processor was initially activated. Advancements of surgical techniques and instruments allow an earlier initial activation of the processor within 14 days after surgery. Objective: Evaluation of the early CI device activation after CI surgery within 14 days, comparison to the first activation after 4–6 weeks, and assessment of the feasibility and safety of the early fitting over a 12 month observation period were the objectives of this study. Method: In a prospective study, 127 patients scheduled for CI surgery were divided into early fitting group (EF, n = 67) and control group (CG, n = 60). Individual questionnaires were used to evaluate medical and technical outcomes of the EF. Medical side effects, speech recognition, and follow-up effort were compared with the CG within the first year after CI surgery. Results: The early fitting was feasible in 97% of the EF patients. In the EF, the processor was activated 25 days earlier than in the CG. No major complications were observed in either group. At the follow-up appointments, side effects such as pain and balance problems occurred with comparable frequency in both groups. At initial fitting, the EF showed a significantly higher incidence of medical minor complications (p < 0.05). When developing speech recognition within the first year of CI use, no difference was observed. Furthermore, the follow-up effort within the first year after CI surgery was comparable in both groups. Conclusions: Early fitting of the sound processor is a feasible and safe procedure with comparable follow-up effort. Although more early minor complications were observed in the EF, there were no long-term wound healing problems caused by the early fitting. Regular inspection of the magnet strength is recommended as part of the CI follow-up since postoperative wound swelling must be expected. The early fitting procedure enabled a clear reduction in the waiting time between CI surgery and initial sound processor activation.

Syncope in relation to pulmonary arterial obstructive burden, hemodynamic status and short- and long-term outcome in patients with acute pulmonary embolism

2020 ◽  
Vol 41 (Supplement_2) ◽  
Author(s):  
B Keskin ◽  
H.C Tokgoz ◽  
O.Y Akbal ◽  
A Hakgor ◽  
S Tanyeri ◽  
...  
Keyword(s):  
Pulmonary Embolism ◽  
Acute Pulmonary Embolism ◽  
Right Atrial ◽  
Term Outcome ◽  
Risk Status ◽  
Long Term Outcome ◽  
Short And Long Term ◽  
Severity Indexes

Abstract Background and aims Although syncope (S) has been reported as one of the presenting findings in patients (pts) with acute pulmonary embolism (APE), its clinical and haemodynamic correlates and impacts on the long-term outcome in this setting remains to be determined. In this single-centre study we evaluated the clinical and haemodynamic significance of S in APE in initial asessment, and during short- and long-term follow-up period. Methods Our study was based on the retrospective and prospective analysis of the overall 641 pts (age 65 (51–74 IQR) yrs, 56.2% female) with diagnosis of documented APE who underwent anticoagulant (n=207), thrombolytic (n=164), utrasound-facilitated thrombolysis (UFT) (n=218) or rheolytic thrombectomy (RT) (n=52). The systematic work- up including multidetector computed tomography (MDCT), Echo, biomarkers, and PE severity indexes were performed in all pts, and Qanadli score (QS) was used as the measure of the thrombotic burden in the pulmonary arteries (PA). Results The S as the presenting symptom In 30.2% of pts with APE. At baseline assessment, S(+) vs S(−) APE subgroups had a significantly shorter symptom-diagnosis interval, a higher risk status according to the significant elevations in troponin T, D-dimer, the higher PE severity indexes, a more deteriorated right ventricle/left ventricle ratio (RV/LV r), right atrial/left atrial ratio (LA/RAr) and RV longitudinal function indexes including tricuspid annular planary excursion (TAPSE) and tissue velocity (St), a significantly higher PA obstructive burden as assessed by QS and PA pressures. Thrombolytic therapy (36.2% vs 21%, p&lt;0.001) and RT (11.9% vs 6.47%, p=0.037) were more frequently utilized S(+) as compared to S(−) group. However, all these differences between two subgroups were found to disappear after evidence-based APE treatments. In-hospital mortality (IHM) (12.95% vs 6%, p=0.007) and minor bleeding (10.36% vs 2.9%, p&lt;0.001) were significantly higher in S(+) pts as compared to those in S(−) subgroup. Binominal logistic regression analysis revealed that PESI score and RV/LVr independently associated with S while IHM was only predicted by age and heart rate. The COX proportional hazard method showed that RV/LVr at discharge and malignancy were independently associated with cumulative mortality during follow-up duration of 620 (200–1170 IQ) days. Conclusions The presence of S in pts with APE was found to be asociated with a higher PA obstructive burden, a more deteriorated RV function and haemodynamics and higher risk status which may need more agressive reperfusion treatments. However, in the presence of the optimal treatments, S did not predict neither in-hospital outcome, nor long-term mortality. Funding Acknowledgement Type of funding source: None

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