The treatment of hyperphagia and obesity in Prader-Willi Syndrome
DOI:
https://doi.org/10.12775/QS.2024.17.52948Keywords
Prader-Willi syndrome, obesity, treatmentAbstract
Introduction: Prader-Willi Syndrome (PWS) is a genetic neurodevelopmental disorder, in which the central clinical characteristics are hyperphagia and obesity. Obesity leads to higher mortality rates in PWS patients and thus, effective and safe treatment of hyperphagia and obesity in PWS individuals is an important aspect of PWS therapy.
Aim of the study: The aim of the study was to research both the new and the more standard treatment options of hyperphagia and obesity in PWS patients.
Methods and Materials: An extensive literature search was conducted using PubMed database. Only articles from the past 5 years were taken into consideration. The keywords used during the search were: prader willi syndrome, obesity, treatment.
Results: Our research showed that in the treatment of hyperphagia and obesity in PWS patients, health professionals may consider the use of pharmacological treatment options, surgery and neuromodulatory options. Moreover, physical activity and environmental control are beneficial in limiting caloric intake and improving body mass index. Furthermore, early diagnosis and thus starting treatment in early childhood are beneficial in preventing the progression of obesity. However, more research is needed to determine which treatment options are best suited for specific patients.
References
Ahmed, S., Naz, A., & K, M. (2023). Weight Loss of Over 100 lbs in a Patient of Prader-Willi Syndrome Treated With Glucagon-Like Peptide-1 (GLP-1) Agonists. Cureus, 15(2). https://doi.org/10.7759/CUREUS.35102
Alfaro, D. L. P., Lemoine, P., Ehlinger, V., Molinas, C., Diene, G., Valette, M., Pinto, G., Coupaye, M., Poitou-Bernert, C., Thuilleaux, D., Arnaud, C., & Tauber, M. (2019). Causes of death in Prader-Willi syndrome: lessons from 11 years’ experience of a national reference center. Orphanet Journal of Rare Diseases, 14(1). https://doi.org/10.1186/S13023-019-1214-2
Alves, C., & Franco, R. R. (2020). Prader-Willi syndrome: endocrine manifestations and management. Archives of Endocrinology and Metabolism, 64(3), 223. https://doi.org/10.20945/2359-3997000000248
Barrea, L., Vetrani, C., Fintini, D., Giulia De Alteriis, •, Filippo, •, Panfili, M., Bocchini, S., Ludovica Verde, •, Colao, A., Savastano, S., & Muscogiuri, G. (n.d.). Prader-Willi Syndrome in Adults: An Update On Nutritional Treatment and Pharmacological Approach. Current Obesity Reports, 1, 3. https://doi.org/10.1007/s13679-022-00478-w
Bellis, S. A., Kuhn, I., Adams, S., Mullarkey, L., & Holland, A. (2022). The consequences of hyperphagia in people with Prader-Willi Syndrome: A systematic review of studies of morbidity and mortality. European Journal of Medical Genetics, 65(1), 104379. https://doi.org/10.1016/J.EJMG.2021.104379
Ben-Cnaan, E., Tam, J., Permyakova, A., Azar, S., Hirsch, S., Baraghithy, S., & Hinden, L. (2022). The Metabolic Efficacy of a Cannabidiolic Acid (CBDA) Derivative in Treating Diet- and Genetic-Induced Obesity. International Journal of Molecular Sciences, 23(10), 5610. https://doi.org/10.3390/IJMS23105610
Calcaterra, V., Magenes, V. C., Destro, F., Baldassarre, P., Silvestro, G. S., Tricella, C., Visioli, A., Verduci, E., Pelizzo, G., & Zuccotti, G. (2023). Prader–Willi Syndrome and Weight Gain Control: From Prevention to Surgery—A Narrative Review. Children, 10(3). https://doi.org/10.3390/CHILDREN10030564
Consoli, A., Çabal Berthoumieu, S., Raffin, M., Thuilleaux, D., Poitou, C., Coupaye, M., Pinto, G., Lebbah, S., Zahr, N., Tauber, M., Cohen, D., & Bonnot, O. (2019). Effect of topiramate on eating behaviours in Prader-Willi syndrome: TOPRADER double-blind randomised placebo-controlled study. Translational Psychiatry, 9(1). https://doi.org/10.1038/S41398-019-0597-0
Contreras López, W. O., Navarro, P. A., & Crispín, S. (2022). Effectiveness of Deep Brain Stimulation in Reducing Body Mass Index and Weight: A Systematic Review. Stereotactic and Functional Neurosurgery, 100(2), 75–85. https://doi.org/10.1159/000519158
Couto-Rosende, Y., Garcia-Tirado, D., Palacio-Marco, M., Caixàs, A., & Corripio, R. (2023). A Personalized Approach to Determining the Caloric Needs of Children with Prader–Willi Syndrome Treated with Growth Hormone. Journal of Clinical Medicine, 12(12), 3967. https://doi.org/10.3390/JCM12123967
Cowen, N., & Bhatnagar, A. (2020). The Potential Role of Activating the ATP-Sensitive Potassium Channel in the Treatment of Hyperphagic Obesity. Genes, 11(4). https://doi.org/10.3390/GENES11040450
Damen, L., Damen, L., Donze, S. H., Donze, S. H., Kuppens, R. J., Kuppens, R. J., Bakker, N. E., Bakker, N. E., De Graaff, L. C. G., Van Der Velden, J. A. E. M., Hokken-Koelega, A. C. S., & Hokken-Koelega, A. C. S. (2020). Three years of growth hormone treatment in young adults with Prader-Willi syndrome: sustained positive effects on body composition. Orphanet Journal of Rare Diseases, 15(1). https://doi.org/10.1186/S13023-020-01440-6
Damen, L., Grootjen, L. N., Juriaans, A. F., Donze, S. H., Huisman, T. M., Visser, J. A., Delhanty, P. J. D., & Hokken-Koelega, A. C. S. (2021). Oxytocin in young children with Prader‐Willi syndrome: Results of a randomized, double‐blind, placebo‐controlled, crossover trial investigating 3 months of oxytocin. Clinical Endocrinology, 94(5), 774. https://doi.org/10.1111/CEN.14387
Diene, G., Angulo, M., Hale, P. M., Jepsen, C. H., Hofman, P. L., Hokken-Koelega, A., Ramesh, C., Turan, S., & Tauber, M. (2023). Liraglutide for Weight Management in Children and Adolescents With Prader–Willi Syndrome and Obesity. The Journal of Clinical Endocrinology and Metabolism, 108(1), 4. https://doi.org/10.1210/CLINEM/DGAC549
Drabik, M., Lewiński, A., & Stawerska, R. (2022). Management of Prader-Labhart-Willi syndrome in children and in adults, with particular emphasis on the treatment with recombinant human growth hormone. Pediatric Endocrinology, Diabetes, and Metabolism, 28(1), 64. https://doi.org/10.5114/PEDM.2022.112861
Erhardt, É., & Molnár, D. (2022). Prader–Willi Syndrome: Possibilities of Weight Gain Prevention and Treatment. Nutrients, 14(9). https://doi.org/10.3390/NU14091950
Faccioli, N., Poitou, C., Clément, K., & Dubern, B. (2023). Current Treatments for Patients with Genetic Obesity. Journal of Clinical Research in Pediatric Endocrinology, 15(2), 108. https://doi.org/10.4274/JCRPE.GALENOS.2023.2023-3-2
Felix, G., Felix, G., Kossoff, E., Barron, B., Krekel, C., Testa, E. G., Scheimann, A., & Scheimann, A. (2020). The modified Atkins diet in children with Prader-Willi syndrome. Orphanet Journal of Rare Diseases, 15(1). https://doi.org/10.1186/S13023-020-01412-W
Goldman, V. E., Naguib, M. N., & Vidmar, A. P. (2021). Anti-Obesity Medication Use in Children and Adolescents with Prader–Willi Syndrome: Case Review and Literature Search. Journal of Clinical Medicine, 10(19). https://doi.org/10.3390/JCM10194540
Grugni, G., Sartorio, A., Soranna, D., Zambon, A., Grugni, L., Zampino, G., & Crinò, A. (2023). Long-term effects of GH therapy in adult patients with Prader-Willi syndrome: a longitudinal study. Frontiers in Endocrinology, 14. https://doi.org/10.3389/FENDO.2023.1198616
Hirsch, H. J., & Gross-Tsur, V. (2021). Growth hormone treatment for adults with Prader-Willi syndrome: another point of view. Orphanet Journal of Rare Diseases, 16(1), 337. https://doi.org/10.1186/S13023-021-01952-9
Höybye, C., Holland, A. J., Driscoll, D. J., & Organisation, T. C. and S. A. B. of T. I. P.-W. S. (2021). Time for a general approval of growth hormone treatment in adults with Prader–Willi syndrome. Orphanet Journal of Rare Diseases, 16(1). https://doi.org/10.1186/S13023-020-01651-X
Hu, H., & Lei, L. (2024). Bariatric surgery for the adolescent with Prader–Willi syndrome: A literature review. Asian Journal of Surgery, 47(3), 1610–1611. https://doi.org/10.1016/J.ASJSUR.2023.12.033
Kalinderi, K., Goula, V., Sapountzi, E., Tsinopoulou, V. R., & Fidani, L. (2024). Syndromic and Monogenic Obesity: New Opportunities Due to Genetic-Based Pharmacological Treatment. Children, 11(2). https://doi.org/10.3390/CHILDREN11020153
Kim, S. J., Cho, S. Y., & Jin, D. K. (2021). Prader-Willi syndrome: an update on obesity and endocrine problems. Annals of Pediatric Endocrinology & Metabolism, 26(4), 227. https://doi.org/10.6065/APEM.2142164.082
Kim, Y. M., Lee, Y. J., Kim, S. Y., Cheon, C. K., & Lim, H. H. (2020). Successful rapid weight reduction and the use of liraglutide for morbid obesity in adolescent Prader-Willi syndrome. Annals of Pediatric Endocrinology & Metabolism, 25(1), 52. https://doi.org/10.6065/APEM.2020.25.1.52
Kimonis, V. E., Tamura, R., Gold, J. A., Patel, N., Surampalli, A., Manazir, J., Miller, J. L., Roof, E., Dykens, E., Butler, M. G., & Driscoll, D. J. (2019). Early Diagnosis in Prader–Willi Syndrome Reduces Obesity and Associated Co-Morbidities. Genes, 10(11). https://doi.org/10.3390/GENES10110898
Kimonis, V., Surampalli, A., Wencel, M., Gold, J. A., & Cowen, N. M. (2019). A randomized pilot efficacy and safety trial of diazoxide choline controlled-release in patients with Prader-Willi syndrome. PLoS ONE, 14(9). https://doi.org/10.1371/JOURNAL.PONE.0221615
Mahmoud, R., Kimonis, V., & Butler, M. G. (2023). Clinical Trials in Prader–Willi Syndrome: A Review. International Journal of Molecular Sciences, 24(3). https://doi.org/10.3390/IJMS24032150
Miller, J. L., Lacroix, A., Bird, L. M., Shoemaker, A. H., Haqq, A., Deal, C. L., Clark, K. A., Ames, M. H., Suico, J. G., De La Peña, A., & Fortier, C. (2022). The Efficacy, Safety, and Pharmacology of a Ghrelin O-Acyltransferase Inhibitor for the Treatment of Prader-Willi Syndrome. The Journal of Clinical Endocrinology and Metabolism, 107(6), e2373. https://doi.org/10.1210/CLINEM/DGAC105
Miller, J. L., & Tan, M. (2020). Dietary Management for Adolescents with Prader–Willi Syndrome. Adolescent Health, Medicine and Therapeutics, 11, 113. https://doi.org/10.2147/AHMT.S214893
Muscogiuri, G., Barrea, • L, Faggiano, • F, Maiorino, • M I, Parrillo, • M, Pugliese, • G, Ruggeri, • R M, Scarano, • E, Savastano, • S, & Colao, • A. (2021). Obesity in Prader-Willi syndrome: physiopathological mechanisms, nutritional and pharmacological approaches on behalf of RESTARE. 44, 2057–2070. https://doi.org/10.1007/s40618-021-01574-9
Nolan, B. J., Proietto, J., & Sumithran, P. (2022). Intensive management of obesity in people with Prader-Willi syndrome. Endocrine, 77(1), 57. https://doi.org/10.1007/S12020-022-03064-1
Passone, C. D. G. B., Franco, R. R., Ito, S. S., Trindade, E., Polak, M., Damiani, D., & Bernardo, W. M. (2020). Growth hormone treatment in Prader-Willi syndrome patients: systematic review and meta-analysis. BMJ Paediatrics Open, 4(1), 630. https://doi.org/10.1136/BMJPO-2019-000630
Poje, A. B., Manzardo, A., Gustafson, K. M., Liao, K., Martin, L. E., & Butler, M. G. (2021). Effects of Transcranial Direct Current Stimulation (tDCS) on Go/NoGo Performance Using Food and Non-Food Stimuli in Patients with Prader–Willi Syndrome. Brain Sciences, 11(2), 1–11. https://doi.org/10.3390/BRAINSCI11020250
Qaddra, Q. F., Jufri, N. F., & Hamid, A. (2023). Hyperphagia in Prader-Willi syndrome with obesity: From development to pharmacological treatment. Intractable & Rare Diseases Research, 12(1), 5. https://doi.org/10.5582/IRDR.2022.01127
Qiu, L., Chang, A., Ma, R., Strong, T. V., Okun, M. S., Foote, K. D., Wexler, A., Gunduz, A., Miller, J. L., & Halpern, C. H. (2024). Neuromodulation for the treatment of Prader-Willi syndrome – A systematic review. Neurotherapeutics, 21(3). https://doi.org/10.1016/J.NEUROT.2024.E00339
Roof, E., Deal, C. L., McCandless, S. E., Cowan, R. L., Miller, J. L., Hamilton, J. K., Roeder, E. R., McCormack, S. E., Roshan Lal, T. R., Abdul-Latif, H. D., Haqq, A. M., Obrynba, K. S., Torchen, L. C., Vidmar, A. P., Viskochil, D. H., Chanoine, J. P., Lam, C. K. L., Pierce, M. J., Williams, L. L., … Ryman, D. C. (2023). Intranasal Carbetocin Reduces Hyperphagia, Anxiousness, and Distress in Prader-Willi Syndrome: CARE-PWS Phase 3 Trial. The Journal of Clinical Endocrinology and Metabolism, 108(7), 1696. https://doi.org/10.1210/CLINEM/DGAD015
Rosenberg, A. G. W., Passone, C. G. B., Pellikaan, K., Damiani, D., Van Der Lely, A. J., Polak, M., Bernardo, W. M., & De Graaff, L. C. G. (2021). Growth Hormone Treatment for Adults With Prader-Willi Syndrome: A Meta-Analysis. The Journal of Clinical Endocrinology and Metabolism, 106(10), 3068. https://doi.org/10.1210/CLINEM/DGAB406
Strong, T. V., Miller, J. L., McCandless, S. E., Gevers, E., Yanovski, J. A., Matesevac, L., Bohonowych, J., Ballal, S., Yen, K., Hirano, P., Cowen, N. M., & Bhatnagar, A. (2024). Behavioral changes in patients with Prader-Willi syndrome receiving diazoxide choline extended-release tablets compared to the PATH for PWS natural history study. Journal of Neurodevelopmental Disorders, 16(1). https://doi.org/10.1186/S11689-024-09536-X
Wolfe, G., Salehi, V., Browne, A., Riddle, R., Hall, E., Fam, J., Tichansky, D., & Myers, S. (2023). Metabolic and bariatric surgery for obesity in Prader Willi syndrome: systematic review and meta-analysis. Surgery for Obesity and Related Diseases, 19(8), 907–915. https://doi.org/10.1016/j.soard.2023.01.017
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Copyright (c) 2024 Ilona Jastrzębska, Paulina Cuper, Michał Andrzej Kozicz, Krzysztof Bilecki, Hubert Gugulski, Michał Bado, Patrycja Nowoświat
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