scholarly journals Dextroamphetamine Treatment for Children With Hypothalamic Obesity

2021 ◽  
Vol 5 (Supplement_1) ◽  
pp. A62-A63
Author(s):  
Jiska van Schaik ◽  
Mila Sofie Welling ◽  
Corjan De Groot ◽  
Ozair Abawi ◽  
M Burghard ◽  
...  
Keyword(s):  
Weight Loss ◽  
Side Effects ◽  
Children And Adolescents ◽  
Resting Energy Expenditure ◽  
Inhibitory Effect ◽  
Activity Level ◽  
Hypothalamic Obesity ◽  
Stimulant Effect ◽  
Genetic Obesity

Abstract Introduction: Hypothalamic obesity (HO) in children can be either genetic or acquired, as a result of a suprasellar tumor or its treatment. HO, resulting from hyperphagia and/or a decreased resting energy expenditure (REE), may have devastating consequences for the child and its family. Currently, no effective drug treatment is yet available for HO. Amphetamines – commonly used in children with attention-deficit/hyperactivity disorder – are known for their stimulant effect on REE and inhibitory effect on appetite. We here present our experiences of dextroamphetamine treatment in children and adolescents with acquired or genetic HO. Methods: A retrospective cohort evaluation was performed of patients (n = 18) treated with dextroamphetamine at 2 endocrine pediatric clinics. Off-label use of dextroamphetamine was initiated in patients with progressive therapy resistant acquired HO (n = 13) and in patients with genetic obesity (n = 5). Initial treatment dosing was once or twice daily 5mg. This dose was weekly increased with 5 mg/day depending on the patient’ wellbeing and the presence of side effects, to a maximum of 0.5 mg/kg/day. Anthropometrics and REE at start and during follow-up, changes in (hyperphagic) behavior, and side effects were assessed. Results: At start of treatment, mean age was 12.8 years ± 3.4 [range 7.1–17.9] and mean REE was 69.5%± 18.5 (n = 15). At follow-up, mean treatment duration was 18.3 months ± 14.7. Ten out of eighteen children (55.6%) showed clinically relevant weight loss. In 10/13 patients with acquired HO, weight loss was observed (mean ΔBMI SDS -1.09 ± 1.00), in one patient BMI stabilization (ΔBMI SDS +0.03), and in two patients an increase in BMI SDS was seen (mean ΔBMI SDS +0.32 ± 0.05). Of nine children with acquired HO and measurement of REE before and during treatment, a mean REE increase of +15.3% ± 10.5 was observed. In three out of five patients with genetic obesity, initially weight loss was observed resulting in BMI stabilization at end of follow-up due to weight regain (mean ΔBMI SDS -0.08 ± 0.19). In these patients, no difference in REE before and during treatment was observed. In two patients an increase in BMI SDS was seen (mean ΔBMI SDS +0.29 ± 0.25). However, one patient discontinued treatment after one month, due to hypertension. Thirteen out of 18 children (72.2%) reported improvement of either their hyperphagia, energy level, and/or behavior. No serious side effects were reported. Conclusion: In children and adolescents with acquired HO, treatment with dextroamphetamine may significantly lower BMI, reduce hyperphagia and improve activity level. In genetic HO, these effects were less pronounced. Future studies in a larger cohort and with randomized controlled designs are needed to support these results.

scholarly journals Effects of Glucagon-Like-Peptide-1 Analogue Treatment in Genetic Obesity

2021 ◽  
Vol 5 (Supplement_1) ◽  
pp. A33-A34
Author(s):  
Mila Sofie Welling ◽  
Cornelis J de Groot ◽  
Lotte Kleinendorst ◽  
Bibian van der Voorn ◽  
Jan Steven Burgerhart ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Weight Loss ◽  
Side Effects ◽  
Metabolic Parameters ◽  
Deletion Syndrome ◽  
Genetic Obesity ◽  
Lifestyle Treatment ◽  
Glucagon Like Peptide ◽  
Glucagon Like Peptide 1

Abstract Introduction: Obesity is highly prevalent, comes with serious health burden and is difficult to treat. In a minority, there is a genetic cause for the obesity. In these patients, therapy-resistant obesity is often observed despite intensive lifestyle treatment. Moreover, it is still unclear whether bariatric surgery is less successful in genetic obesity. Liraglutide is a Glucagon-Like-Peptide-1 (GLP-1) receptor agonist or GLP-1 analogue, showing positive effects on metabolic parameters, satiety and weight loss in lifestyle-induced obesity. We present our experiences of GLP-1 analogue treatment in patients with genetic obesity disorders. Methods: Adults with overweight or severe obesity and a molecularly proven genetic cause were treated with liraglutide 3,0 mg daily, in addition to ongoing intensive supportive lifestyle treatment. Anthropometrics, metabolic parameters, resting energy expenditure (REE), side effects, and subjectively reported satiety and quality of life were assessed. Results: Two patients with a heterozygous pathogenic melanocortin 4 recepter variant and two patients with 16p11.2 deletion syndrome, ranging in age between 21 and 32 years and in BMI between 28.1 and 55.7 kg/m2 at baseline, were treated. At end of follow-up, ranging between 33 weeks and 12 years, a mean change in BMI and waist circumference was observed of -5.7 ± 3.8 kg/m2 and -15.2 ± 21.1 cm, respectively. All patients reported better quality of life, three of them also reported improved satiety. Moreover, improvement of metabolic parameters was seen. No clear effect on REE was observed. Two patients experienced mild side effects, e.g. nausea and stomach pain, for a brief period. Conclusion: We here show beneficial effects of GLP-1 analogues on weight, metabolic parameters, and quality of life in four patients with genetic obesity. Satiety improved in three of the four patients. All patient achieved at least the clinically relevant 5–10% weight loss. Our findings suggest that GLP-1 analogue treatment might be an effective treatment option, in addition to a healthy lifestyle, for patients with genetic obesity.

scholarly journals Response to Front-Line Imatinib Treatment in Children and Adolescents with CML - Data from a Large Pediatric Cohort

Blood ◽  
2017 ◽  
Vol 130 (Suppl_1) ◽  
pp. 898-898
Author(s):  
Meinolf Suttorp ◽  
Philipp Schulze ◽  
Ingmar Glauche ◽  
Gudrun Göhring ◽  
Nils Von Neuhoff ◽  
...  
Keyword(s):  
Stem Cell ◽  
Side Effects ◽  
Stem Cell Transplantation ◽  
Children And Adolescents ◽  
Research Funding ◽  
Newly Diagnosed ◽  
First Line ◽  
Free Survival ◽  
Grade 3

Abstract Purpose Results of the prospective trial "CML-PAED-II" assessing treatment efficacy and side effects in children and adolescents with newly diagnosed chronic myeloid leukemia (CML) are reported. Patients and Methods 156 patients (age range 1.3-18.0 years, 91 male) with newly diagnosed CML (N= 146 chronic phase (CML-CP), N= 3 accelerated phase (CML-AP), N= 7 blastic phase (CML-BP)) received imatinib upfront (300 mg/m², 400 mg/m², 500 mg/m², respectively). Therapy response, progression-free survival, causes of treatment failure and proportion of patients undergoing stem cell transplantation were analyzed in 148 patients with complete data. Results Event-free survival rate at 18 months for pediatric patients diagnosed in CML-CP (median follow-up time 25 months, range: 0.1-120) was 97% (95% CI, 94.2%-99.9%). According to the 2006 ELN-criteria complete hematologic response at month 3, complete cytogenetic response (CCyR) at month 12, and molecular response (MR3.0) at month 18 were achieved in 98%, in 63%, and 59% of the patients, respectively. At month 36 on continuous first line imatinib or 2nd generation tyrosine kinase inhibitor treatment, 86% of the patients achieved CCyR and 74% achieved MR3.0. 66% of the patients experienced at least one side effect. Imatinib-related anemia was the most frequent toxicity observed if all grades were considered (N=98; 66%) while neutropenia was the most frequently reported grade 3/4 hematologic adverse effect (N=22; 15%). Among non-hematologic toxicities, all grades of gastrointestinal toxicity were observed most frequently (N=57, 38%), however, it occurred at lower grades 1/2 in all but one patient. Higher grade 3/4 musculoskeletal pain was also frequent (N=53, 36%). Twenty-seven patients (18%) had to discontinue treatment temporarily while nine patients permanently terminated imatinib due to non-tolerable side effects (neutropenia N= 4, muscle cramps N= 3, skin N= 1, liver N= 1). Thirty-eight patients (27%) experienced imatinib failure because of unsatisfactory response (N= 27) or intolerance (N= 9). 28/148 patients (19%) underwent stem cell transplantation (SCT). In the SCT sub-cohort 2/23 patients diagnosed in CML-CP, 0/1 in CML-AP, and 2/4 in CML-BP, respectively, died of relapse (N=3) or SCT-related complications (N=2). Conclusion This large pediatric trial provides evidence confirming that first line imatinib in children is highly effective. Observed adverse effects are acceptable and mainly comprise hematological side effects. Long term outcome and effects of a potentially life-long TKI treatment have to be registered in cooperation with adult hematologists in extended surveillance follow-up studies. Disclosures Suttorp: Novartis: Research Funding. Schrappe: JAZZ Pharma: Consultancy, Research Funding; Baxalta: Consultancy, Research Funding; Novartis: Consultancy, Research Funding; SigmaTau: Consultancy, Research Funding; Medac: Consultancy, Research Funding. Thiede: Novartis: Consultancy, Speakers Bureau; Bayer: Consultancy, Speakers Bureau; Roche: Consultancy; Agendix: Employment.

scholarly journals Hypothalamic obesity: prevalence, associations and longitudinal trends in weight in a specialist adult neuroendocrine clinic

2013 ◽  
Vol 168 (4) ◽  
pp. 501-507 ◽  
Author(s):  
Caroline A Steele ◽  
Daniel J Cuthbertson ◽  
Ian A MacFarlane ◽  
Mohsen Javadpour ◽  
Kumar S V Das ◽  
...  
Keyword(s):  
Weight Loss ◽  
Weight Gain ◽  
Clinic Visit ◽  
Morbidly Obese ◽  
Obesity Prevalence ◽  
Hypothalamic Obesity ◽  
Physical Activity Advice ◽  
The Impact ◽  
Hypothalamic Damage

ObjectiveObesity is highly prevalent among adults with acquired, structural hypothalamic damage. We aimed to determine hormonal and neuroanatomical variables associated with weight gain and obesity in patients following hypothalamic damage and to evaluate the impact of early instigation of weight loss measures to prevent or limit the severity of obesity in these patients.DesignRetrospective study of 110 adults with hypothalamic tumours attending a specialist neuroendocrine clinic. BMI was calculated at diagnosis and at last follow-up clinic visit. Endocrine data, procedures, treatments and weight loss measures were recorded and all available brain imaging reviewed.ResultsAt last follow-up, 82.7% of patients were overweight or heavier (BMI≥25 kg/m2), 57.2% were obese (BMI≥30 kg/m2) and 14.5% were morbidly obese (BMI≥40 kg/m2). Multivariate analysis revealed that use of desmopressin (odds ratio (OR)=3.5;P=0.026), GH (OR=2.7;P=0.031) and thyroxine (OR=3.0;P=0.03) was associated with development of new or worsened obesity. Neuroimaging features were not associated with weight gain. Despite proactive treatments offered in clinic in recent years (counselling, dietetic and physical activity advice, and anti-obesity medications), patients have continued to gain weight.ConclusionsDespite increased awareness, hypothalamic obesity is difficult to prevent and to treat. Improved understanding of the underlying pathophysiologies and multicentre collaboration to examine efficacy of novel obesity interventions are warranted.

scholarly journals 0923 Doxepin in Children and Adolescents With Symptoms of Insomnia: A Single Center Experience

SLEEP ◽  
2020 ◽  
Vol 43 (Supplement_1) ◽  
pp. A351-A351
Author(s):  
Y Shah ◽  
S Kothare
Keyword(s):  
Side Effects ◽  
Children And Adolescents ◽  
Developmental Disorders ◽  
Pediatric Population ◽  
Single Center ◽  
Single Center Experience ◽  
First Line Therapy ◽  
Psychophysiological Insomnia ◽  
Pediatric Insomnia

Abstract Introduction Pediatric insomnia is a widespread problem and especially difficult to manage in children with neurodevelopmental disorders. There are currently no FDA- approved medications for pediatric patients to use once first line therapy fails. Doxepin is FDA-approved at low doses for use in transient or chronic sleep maintenance insomnia in adults. The objective of this study is to determine the tolerability and efficacy of doxepin in the pediatric population. Methods This is a retrospective single center chart review of children and adolescents (2-17 years of age) whose sleep failed to improve with behavioral intervention and melatonin who were then trialed on doxepin. Treatment was initiated at a median starting dose of 2mg and slowly escalated to a median maintenance dose of 10mg. Improvement in sleep was recorded using a 4-point Likert scale reported by parents on follow up visits. Results Total of 29 patients were included in analysis. Mean follow-up duration was 6.5 months (±3.5). Out of 29 patients, 4 (13.8%) patients discontinued doxepin due to lack of efficacy or side effects. 8 (27.6%) patients showed significant improvement of their insomnia, 8 (27.6%) showed moderate, 10 (34.5%) showed mild and 3 (10.3%) showed minimal to no improvement on treatment with doxepin (P<0.05) Only two patients (6.8%) experienced adverse effects in the form of behavioral side effects (aggression) and enuresis. Conclusion Our data suggests that doxepin is effective, safe and well-tolerated in the treatment of sleep initiation and maintenance insomnia as well as psychophysiological insomnia in child and adolescents with Ausitsm specutrm disorder, other neuro-developmental disorders and attention deficit hyperactivity disorder. It is also an effective, safe, and well-tolerated alternative in children suffering from chronic persistent insomnia. The results of this study suggests a promising emerging therapy for the treatment of insomnia in the pediatric population. Support None

Relationship between volition, physical activity and weight loss maintenance: Study rationale, design, methods and baseline characteristics

2016 ◽  
Vol 45 (3) ◽  
pp. 299-304 ◽  
Author(s):  
Sune Dandanell ◽  
Anne-Marie Elbe ◽  
Gertrud Pfister ◽  
Peter Elsborg ◽  
Jørn W Helge
Keyword(s):  
Physical Activity ◽  
Weight Loss ◽  
Oxygen Uptake ◽  
Maximal Oxygen Uptake ◽  
Physical Activity Level ◽  
Mixed Model ◽  
Linear Mixed Model ◽  
Weight Loss Maintenance ◽  
Activity Level

Aims: To investigate the relationship between volition, physical activity and weight loss maintenance. Methods: We recruited 84 sedentary (maximal oxygen uptake: 25 ± 5 ml/min), overweight and obese (Body mass index (BMI) 38 ± 7 m/h2, fat 44 ± 7 %) women ( n = 55) and men ( n = 29) for an interdisciplinary prospective study with follow-up. The change in lifestyle and weight loss is promoted via a 3-month intensive lifestyle intervention at a private health school. The intervention consists of supervised training (1–3 hours/day), a healthy hypo-caloric diet (−500 to −700 kCal/day) and education in healthy lifestyle in classes/groups. The participants’ body weight and composition (Dual Energy X-ray absorptiometry), volitional skills (questionnaire), physical activity level (heart rate accelerometer/questionnaire) and maximal oxygen uptake (indirect calorimetry) are to be monitored before, after, and 3 and 12 months after the intervention. Results: At the 12-month follow-up, three different groups will be established: Clinical weight loss maintenance (> 10% weight loss from baseline), moderate weight loss maintenance (1–10% weight loss) and no weight loss (or weight regain). A linear mixed model analysis will be used to compare levels of volitional skills, physical activity and maximal oxygen uptake over time, between the three groups. Correlational analyses will be used to investigate possible associations between volition, maximal oxygen uptake, physical activity level and weight loss maintenance. Conclusions: If specific volitional skills are identified as predictors of adherence to physical activity and success in clinical weight loss maintenance, these can be trained in future intensive lifestyle interventions in order to optimize the success rate.

scholarly journals An audit assessing the monitoring of SSRIS after initiation in children and adolescents

BJPsych Open ◽  
2021 ◽  
Vol 7 (S1) ◽  
pp. S41-S41
Author(s):  
Ella McGowan
Keyword(s):  
Adverse Effect ◽  
Side Effects ◽  
Adverse Effects ◽  
Children And Adolescents ◽  
Alternative Plan ◽  
Medication Dose ◽  
The Common ◽  
The One ◽  
Accurate Record

AimsTo identify children and adolescents started on SSRIs to see if they are being followed up in accordance to NICE and Maudsley guidelinesObjectivesHas the patient been followed up after a week to check for adverse effects or improvement in their mental state?Has the patient been re-evaluated every 4-6 weeks, if not is there an alternative plan?If there is no improvement has the dose been increased?If there is an adverse effect has the dose been lowered or the medication stopped?MethodPaper case notes including clinic letters and handwritten notes were reviewed on the 19/10/2020. The following data were collected anonymously.AgeGenderDate seen / Date medication startedName of medicationDate medication startedDate of Follow-upMonitoring of improvementMonitoring of adverse effectsOutcome of monitoringResultA total of 18 sets of cases were identified.Follow-up occurred in 17 of the 18 cases.The one case that had not been followed up had started the medication 8 weeks before the audit. The median follow-up time was 42 days (6 weeks). No cases were followed up within a week.Monitoring of improvement was recorded in 88% of case notes reviewed.Monitoring for adverse effects occurred in 36% of case notes and none of these patients had reported any side effects. 53% of cases did not have monitoring of adverse effects documented. There were two patients (11%) who did not take the medication as prescribed. One out of choice and one their parent had not collected it.The medication dose was increased in 22% of patients without clear documentation of monitoring for adverse effects.ConclusionAfter discussion with the clinical lead it was decided it is impractical to follow up patients a week after starting medication. However, patients and their carers should be informed of the side effects and advised to contact CAMHS if adverse effects occur.The area of practice that can be improved is the documentation of adverse effects at follow-up.Recommendations:All patients to be informed of the common side effects of the medication before it is initiated and advised to contact the CAMHS team if they have concernsAll CAMHS patients started on SSRIs should be followed up within 4-6 weeksAt follow-up any adverse events and clinical response should be discussedAn accurate record of the exchanges of the above information should be documented in the notesRe-audit

scholarly journals Multidisciplinary Approach for Hypothalamic Obesity in Children and Adolescents: A Preliminary Study

Children ◽  
2021 ◽  
Vol 8 (7) ◽  
pp. 531
Author(s):  
Daniele Tessaris ◽  
Patrizia Matarazzo ◽  
Gerdi Tuli ◽  
Antonella Tuscano ◽  
Ivana Rabbone ◽  
...  
Keyword(s):  
Weight Gain ◽  
Children And Adolescents ◽  
Psychological Assessment ◽  
Multidisciplinary Approach ◽  
Standard Deviation Score ◽  
Metabolic Parameters ◽  
Hypothalamic Obesity ◽  
Metabolic Outcome ◽  
Preliminary Study

Hypothalamic obesity (HO) is delineated by an inexorable weight gain in subjects with hypothalamic disorder (congenital or acquired). The aim of the present study was to evaluate the effect of a multidisciplinary approach on weight trend and metabolic outcome in children and adolescents with hypothalamic disease who were overweight or obese. Thirteen patients (aged 8.1–16.1 years) received a personalized diet, accelerometer-based activity monitoring, and psychological assessment. Height, weight, body mass index (BMI), and serum metabolic parameters were assessed at baseline (T0) and after six months (T1). Metformin was introduced at T1 in four subjects who were then re-evaluated after six months (T2). At T1, weight gain was significantly reduced compared with T0 (0.29 ± 0.79 kg/month vs. 0.84 ± 0.55 kg/month, p = 0.03), and weight standard deviation score (SDS) and BMI SDS did not change significantly, as serum metabolic parameters. The four subjects treated with metformin showed a reduction of weight SDS and BMI SDS at T2. In conclusion, patients treated with our multidisciplinary approach showed, after 6 months, favorable results characterized by decreased weight gain and stabilization of weight SDS and BMI SDS in a condition usually characterized by inexorable weight gain. However, further analysis, larger cohorts, and longer follow-up are needed to confirm these preliminary data.

scholarly journals MON-LB102 Metformin-Induced Weight Loss in Patients With or Without Type 2 Diabetes/Prediabetes

2020 ◽  
Vol 4 (Supplement_1) ◽  
Author(s):  
Tariq Chukir ◽  
Lindsay Mandel ◽  
Nada Al-Mulla ◽  
Rekha Babu Kumar ◽  
Leon I Igel ◽  
...  
Keyword(s):  
Type 2 Diabetes ◽  
Weight Loss ◽  
Side Effects ◽  
Office Visits ◽  
Weight Changes ◽  
Entire Cohort ◽  
Weight Losses ◽  
The Mean

Abstract Background: Metformin is the first-line pharmacologic treatment for type 2 diabetes (T2DM). Its use has been associated with significant weight loss in patients with obesity with or without T2DM. However, it is unknown whether weight loss outcomes differ with metformin monotherapy in patients with excess weight and euglycemia compared to patients with T2DM/prediabetes (PreDM). Methods: This is a retrospective study of new patients with overweight/obesity seen at an academic weight management center between 4/1/14-4/1/16. Patients who received metformin as a sole pharmacotherapy were identified, and data pertaining to demographics, medications, comorbidities, and weight changes during 1-year follow-up were obtained from their electronic medical records. Mean and categorical weight losses were compared between patients with and without T2DM/PreDM. We also assessed the rate of metformin discontinuation due to side effects, lack of efficacy or other reasons in the entire cohort. Results: Of 1056 patients who were prescribed metformin for weight loss over the 2-year study period and had at least 2 office visits, 99 (9.38%) discontinued the medication due to the following reasons: side effects 59 (60%), lack of efficacy 15(15%) or other reasons 25(25%). A total of 254 patients received metformin as a sole pharmacotherapy for weight loss and had 6 and/or 12-month follow-up visits. In this cohort, the mean age was 53 ± 14 years, 66% were women. The mean BMI was 35 ± 7 kg/m2. The average percent weight loss at 6 and 12 months were similar in patients with euglycemia compared to patients with T2DM/PreDM (6.12% ± 5.68 vs 6.36% ± 6.52 p=0.73 at 6 months; 7.13% ± 6.42 vs 7.23% ± 7.74 p=0.93 at 12 months). The proportion of patients who experienced ≥5% weight loss was similar in both groups at 6-month (54.04 vs 54.55%, p=0.94) and 12-month follow-up visits (63.95 vs 55.42%, p=0.26). The proportion of patients who experienced ≥10% weight loss was also similar in both groups at 6-month (25.81 vs 27.72%, p=0.81) and 12-month follow-up visits (33.72 vs 30.12%, p=0.62). Discussion: Among patients with obesity, metformin as a sole pharmacotherapy for weight loss achieved significant and comparable weight reduction in patients with or without T2DM/PreDM. Further studies are needed to evaluate the long-term weight loss in patients with euglycemia.

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