Studies in animals or humans have demonstrated fetal abnormalities and/or there is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience, and the risks involved in use in pregnant women clearly outweigh potential benefits. TSC is a genetic condition that can lead to growths in various organs of the body, but those most commonly affected are the brain, eyes, heart, kidney, skin and lungs. Has a high potential for abuse. vagal nerve stimulator, offer everolimus for refractory focal onset seizures associated with tuberous sclerosis complex in patients aged 2 years or older in line with the NHS England commissioning policy. The organs most commonly affected are the brain, eyes, heart, kidney, skin and lungs. A combination of symptoms may include seizures, intellectual disability, developmental delay, behavioral problems, skin abnormalities, lung disease, and kidney disease. Topiramate is an anticonvulsant, prescribed for the treatment of seizures and prevents migraine headaches, either alone or with other medications. Afinitor Disperz, For professionals: It is believed that the majority of SEGA lesions stop growing in the third decade Levetiracetam is an anticonvulsant, prescribed for partial seizures. Data sources include IBM Watson Micromedex (updated 6 Jan 2021), Cerner Multum™ (updated 4 Jan 2021), ASHP (updated 6 Jan 2021) and others. Diazepam is a benzodiazepine, prescribed for anxiety. ... should be the drug of choice in the treatment of infantile spasms owing to tuberous sclerosis … Patients with TSC show a diverse range of neurological features (including seizures, cognitive disability and autism) and renal manifestations (including angiomyolipomas, epithelial cysts and renal cell carcinoma (RCC)). miscellaneous anticonvulsants, Brand name: Fosphenytoin is an anticonvulsant, prescribed for seizures during neurosurgery and status epilepticus. 2 Seizure semiology varies between patients and can change throughout a patient's lifetime. Therefore, diagnostic criteria have been developed to aid the diagnosis of tuberous sclerosis. Select one or more newsletters to continue. mTOR inhibitors, selective immunosuppressants, For consumers: 2018 Jan;192:264-264.e1. Subscribe to Drugs.com newsletters for the latest medication news, new drug approvals, alerts and updates. 3 Infants with tuberous sclerosis will, however, be included in the epidemiological study. Has a low potential for abuse relative to those in schedule 4. MRI is considered the modality of choice for the evaluation of the brain in patients with diagnosed or suspected tuberous sclerosis. ... An ACE inhibitor may be the first drug of choice. Abuse may lead to moderate or low physical dependence or high psychological dependence. Ethosuximide is a succinimide anticonvulsant, prescribed for epilepsy. The diverse and varied presentations and progression can be life-threatening with significant impact on cost and quality of life. Sirolimus and everolimus have been used to treat subependymal giant cell astrocytomas, facial angiofibromas, and more recently have … ... Vigabatrin is the drug of choice for infantile spasms associated with tuberous sclerosis. Diagnosis The signs and symptoms of Tuberous Sclerosis Complex, and how it is diagnosed The age, time and background of a Tuberous Sclerosis Complex (TSC) diagnosis can vary dramatically between everyone living with the condition. When patients do not meet these criteri… Lacosamide is an anticonvulsant, prescribed for partial-onset of seizures. A-Z Drug Facts, AHFS DI Monograph, Prescribing Information, Drug class: Is not subject to the Controlled Substances Act. Geneticists have reported the world's first series of cases of prenatal diagnosis for women at risk of having a child with tuberous sclerosis complex. Drugs used to treat Tuberous Sclerosis Complex. For other AEDs specific effec-tiveness in TSC is unclear. Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances. Phenytoin is an anticonvulsant, prescribed for certain types of seizures (eg, status epilepticus). Valproic Acid is an anticonvulsant, prescribed for epilepsy, bipolar disorder, and rarely for depression. Tuberous sclerosis complex is a genetic disorder affecting every organ system, but disease manifestations vary significantly among affected individuals. There is a lack of accepted safety for use under medical supervision. Cardiac Rhabdomyomas in Tuberous Sclerosis Complex. Has no currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 3. Three hospitals with specialist TSC services enrolled patients (Royal United Hospital [41 patients], Great Ormond Street Hospital [3 patients], and Bristol Royal Hospital for … Although there is no cure for tuberous sclerosis, treatment can help manage specific symptoms. Tuberous sclerosis complex (TSC) is a genetic neurocognitive disorder, with an incidence of one in 6,000–10,000 live births. Delamanid is an anti-tubercular drug prescribed in combination with bedaquiline for treating active multidrug-resistant tuberculosis in adults when other treatments fail or show no response. There is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience or studies in humans, but potential benefits may warrant use in pregnant women despite potential risks. Has a low potential for abuse relative to those in schedule 3. Clonazepam is an anticonvulsant, muscle relaxant, prescribed for Lennox-Gastaut syndrome, panic disorder and seizures. Has a currently accepted medical use in treatment in the United States or a currently accepted medical use with severe restrictions. Read more... Help & support We support individuals and families affected by TSC. Disclaimer - All information and content on this site are for information and educational purposes only. [] TSC1 and TSC2 genes encode for hamartin (TSC1) and tuberin (TSC2) form a regulatory complex responsible for limiting the activity of an important intracellular regulator of cell growth and metabolism, known as mammalian target of rapamycin … The information should not be used for either diagnosis or treatment or both for any health related problem or disease. Always seek the advice of a qualified physician for medical diagnosis and treatment.Full Disclaimer, Advertise with us | Medindia Copyright | Privacy Policy | Terms of Use © All Rights Reserved 1997 - 2021. Research has found that mTOR inhibitors, which interrupt the chemical reactions needed for tumours to grow, may be a useful treatment in the future. West described the subtle head bobbings that are often confused with other normal baby behaviors Lamotrigine is an anticonvulsant agent, prescribed for epilepsy and bipolar disorder either alone or combined with other medications. Treatment is with anti-tubercular drugs, steroids and surgery depending upon the individual patient ... Genetic testing helps to confirm a genetic condition in an individual and involves q complex laboratory techniques, British children suffering from a rare form of genetic disorder now have an alternative to surgery, A role for enhanced activation of the signaling protein Notch in tumors characterized by inactivation of either the TSC1 or the TSC2 protein has been identified. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 4. Several immunosuppressant drugs that work on the mTOR pathway (the pathway disrupted in TSC) can be useful for TSC therapy. The easiest way to lookup drug information, identify pills, check interactions and set up your own personal medication records. Delamanid is an anti-tubercular drug prescribed in combination with bedaquiline for treating active multidrug-resistant tuberculosis in adults when other treatments fail or show no response. Lamotrigine may be considered as monotherapy in adolescent females with idiopathic generalized epilepsy. For example: 1. Abdominal tuberculosis could be a result of primary infection or reactivation of a dormant focus (post primary malnutrition etc. Tuberous Sclerosis Complex (TSC) is a genetic disease caused by mutations in the tumour suppressor genes TSC1 and TSC2, located on chromosomes 9 and 16.1,2 Approximately two-thirds of cases occur sporadically. Sirolimus is an immunosuppressant, prescribed for preventing rejection of kidney transplants either alone or with other medications. What is TSC? Perampanel is an antiepileptic drug, prescribed for partial-onset seizures. Loss of either protein leads to overgrowth lesions in many vital organs. Has a currently accepted medical use in treatment in the United States. Ethotoin is an anticonvulsant, prescribed for seizures either alone or with other medications. List of drugs used to treat the medical condition called Tuberous Sclerosis. Patients with tuberous sclerosis complex, a genetic disorder characterized by the growth of noncancerous tumors in multiple organs of the body, have limited treatment options. Pharmaceutical, Drug ,Chemist Association - India, Tuberous Sclerosis: Causes, Symptoms, Signs, Diagnosis & Treatment. However, the signs, symptoms and methods used to confirm a … Epidiolex, For professionals: Tuberous sclerosis complex (TSC) is the second most common neurocutaneous disease. Our dedicated advisers and active doi: 10.1016/j.jpeds.2017.09.050. Lamotrigine, levetiracetam and lacosamide are good add-on drugs for patients with partial seizures. Felbamate is an anticonvulsant, prescribed for seizures. Has a high potential for abuse. Infants with definite or possible tuberous sclerosis will be excluded from the drug trial as we (the steering committee of the study) believe that vigabatrin is the first choice for treatment of infantile spasms due to tuberous sclerosis. Vigabatrin is the drug of choice for infantile spasms associated with tuberous sclerosis. Drugs; Stats; Cardiovascular; Endocrine; Gastrointestinal; Heme; Infectious Dis. Lorazepam is a benzodiazepine, prescribed for anxiety. Carbamazepine is an anticonvulsant drug, prescribed for epilepsy and bipolar disorder, as well as trigeminal neuralgia. In patients with tuberous sclerosis complex (TSC), add-on cannabidiol reduces drug-resistant seizures compared with add-on placebo and has a good safety profile,the team concluded. Rufinamide is an anticonvulsant, prescribed for seizures associated with Lennox-Gastaut syndrome in children 4 years and older and adults. Prescribing Information, Brand names: For ratings, users were asked how effective they found the medicine while considering positive/adverse effects and ease of use (1 = not effective, 10 = most effective). New treatment options, such as rapamycin, may be the future drug of choice in treating TSC. Tuberous sclerosis complex (TSC) is a rare multisystem autosomal dominant genetic disease that causes non-cancerous tumours to grow in the brain and on other vital organs such as the kidneys, heart, liver, eyes, lungs and skin. CT is the modality of choice for evaluating renal lesions because of its ability to delineate cystic from solid lesions and lipid-containing angiomyolipomas. Has a potential for abuse less than those in schedules 1 and 2. Available for Android and iOS devices. Benzodiazepines or barbiturates should be avoided as these drugs often aggravate underlying behavioral or … Tuberous sclerosis is caused by mutations in either the TSC1 gene on chromosome 9, or the TSC2 gene on chromosome 16. Oxcarbazepine is an anticonvulsant, prescribed for partial seizure disorders in epileptic children and adults, either alone or with other medication. Animal reproduction studies have shown an adverse effect on the fetus and there are no adequate and well-controlled studies in humans, but potential benefits may warrant use in pregnant women despite potential risks. Epub 2017 Nov 8. Tuberous sclerosis complex (TSC) is an autosomal dominant disease characterized by hamartomatous tumours of the brain, heart, skin, lung and kidney. Clobazam is a benzodiazepine derivative, prescribed for seizure disorders. Add-On Cannabidiol Treatment for Drug-Resistant Seizures in Tuberous Sclerosis Complex: A Placebo-Controlled Randomized Clinical Trial. Zonisamide is a sulfonamide anticonvulsant, prescribed for partial seizures. People with tuberous sclerosis will also need to have regular tests to monitor the function of the organs that can be affected by the condition. Implications for practice: By following evidence‐based clinical practice guidelines, providers can hope to reduce TSC‐related morbidity and mortality. Tuberous sclerosis complex is inherited in an autosomal dominant pattern, although the rate of spontaneous mutation is high. Medication. A total of 19 antiepileptic drugs (AEDs) were used. OTC. dosage, interactions, side effects, For professionals: Other medications may help manage heart arrhythmias, behavior problems or other signs and symptoms. For the full article follow the link: Thiele EA, Bebin EM, Bhathal H, et al. These proteins formed a complex to inhibit mTORC1-mediated cell growth and proliferation. A drug called everolimus (Afinitor, Zortress) may be used to treat certain types of brain and kidney growths that can't be surgically removed. If your child is affected, an individual care plan will be drawn up to address any needs or problems they have. Adequate and well-controlled studies have failed to demonstrate a risk to the fetus in the first trimester of pregnancy (and there is no evidence of risk in later trimesters). It has a currently accepted medical use in treatment in the United States. Drugs.com provides accurate and independent information on more than 24,000 prescription drugs, over-the-counter medicines and natural products. Cardiac Rhabdomyomas in Tuberous Sclerosis Complex J Pediatr. Afinitor, Everolimus is a kinase inhibitor, prescribed for kidney cancer, pancreatic cancer, brain tumor(astrocytoma), hormone-receptorpositive breast cancer in postmenopausal women and prevents organ rejection in kidney therapy. The incidence has been estimated to be 1 per 5800 live births.3 The protein products of TSC1 and TSC2 (hamartin and tuberin) function together within the cell and have an inhibitory effect on the mammalian target of rapamycin (mTOR), a protein kinase that influences cell growth and division an… This material is provided for educational purposes only and is not intended for medical advice, diagnosis or treatment. Click on the drug to find more information including the brand names,dose,side-effects, adverse events, when to take the drug and the price of the drug. Tuberous sclerosis complex (TSC) results from loss of a tumor suppressor gene - TSC 1 or TSC 2, encoding hamartin and tuberin, respectively. The following list of medications are in some way related to, or used in the treatment of this condition. Mephobarbital is a barbiturate, prescribed for anxiety, tension, apprehension, and preventing seizures. In this age group, CBZ is the most popular first-choice drug in all continents except North America where LEV is the drug of choice . Acebrophylline is prescribed to reduce the irritation, swelling, and narrowing of the bronchial tubes in patients with asthma, severe or chronic bronchitis, chronic obstructive pulmonary disease (COPD)and tightness of the chest. Drug class: Tuberous sclerosis is a group of two genetic disorders that cause non-malignant tumors to affect the skin, brain/nervous system, kidneys, and heart. Frequently used AEDs are valp-roic acid (VPA), LEV, topiramate (TPM), oxcarbazepine Acetazolamide is prescribed to treat excess fluid accumulation due to congestive heart failure. This site complies with the HONcode standard for trustworthy health information: verify here. Tuberous sclerosis complex (TSC) is a genetic disorder affecting cellular differentiation, proliferation, and migration early in development, resulting in a variety of hamartomatous lesions that may affect virtually every organ system of the body. Epilepsy surgery was most effective, with four of five children becoming seizure-free. Anti-seizure medications may be prescribed to control seizures. Infantile Spasms in Tuberous Sclerosis Complex: Role of Vigabatrin. Valproic acid, vigabatrin, levetiracetam, and carbamazepine were used most frequently. See tuberous sclerosis diagnostic criteria 2. It's estimated that around 1 in every 6,000 babies are born with the condition, however in Tuberous sclerosis complex (TSC) is a genetic condition, present from birth, which can lead to non-cancerous growths developing in a number of different organs of the body. Tuberous sclerosis was classically described as presenting in childhood with a triad (Vogt triad) of: 1. seizures: absent in one-quarter of individuals 2. intellectual disability: up to half have normal intelligence 3. adenoma sebaceum: only present in about three-quarters of patients1 The full triad is only seen in a minority of patients (~30%). We comply with the HONcode standard for trustworthy health information -. Genetics. Has a currently accepted medical use in treatment in the United States. Though, Vigabatrin is considered as the drug of choice for children with TSC and infantile spasms. Nonpharmacologic therapies (ketogenic diet, epilepsy surgery, and vagus nerve stimulation) were used 13 times. Tuberous sclerosis is genetic disorder characterized by benign tumors in many parts of the body, including brain, skin, lungs, kidney and heart. Trimethadione is an anticonvulsant, prescribed for seizure disorders. Neurology; Nutrition; Oncology; ... the treatment of choice of infantile spasms. Animal reproduction studies have failed to demonstrate a risk to the fetus and there are no adequate and well-controlled studies in pregnant women. Tuberous sclerosis is a lifelong condition that requires long-term care and support from a range of different healthcare professionals. Abuse may lead to severe psychological or physical dependence. RCT (n=224) reports cannabidiol significantly reduced tuberous sclerosis complex -associated seizures from baseline vs. placebo; reduction of 48.6% for the 25mg group, 47.5% for 50mg group vs. 26.5% ... Read Summary. in TSC, and is the treatment of first choice.11,13,14 For other seizure types, there is evidence for efficacy of lamotrigine (LTG),15 levetiracetam (LEV),16 and clobazam (CLB)17 from small patient studies. The Metformin in Tuberous Sclerosis (MiTS) study was a randomised double-blind placebo-controlled trial. Topiramate, lamotrigine, valproate, and adrenocorticotropic hormone are also useful. Methsuximide is an anticonvulsant medication, prescribed for certain types of seizures. Phenobarbital is a barbiturate, prescribed for seizures and as a sedative to relieve anxiety. Select drug class All drug classes miscellaneous anticonvulsants (2) mTOR inhibitors (3) selective immunosuppressants (3) Rx. 1 Epilepsy is reported in up to 90% of TSC patients, and often starts in infancy. Primidone is an anticonvulsant, prescribed for seizures. This medication may not be approved by the FDA for the treatment of this condition. The following list of medications are in some way related to, or used in the treatment of this condition. Among Australian clinicians, CBZ, LEV, VGB, and topiramate (TPM) are equally frequently chosen (all 1/2, 50.0%). A-Z Drug Facts, AHFS DI Monograph, Natural Product Monographs. Divalproex is an anticonvulsant, prescribed for bipolar disorder, epilepsy and to prevent migraine headache. Acetazolamide is prescribed to treat excess fluid accumulation due to congestive heart failure neurosurgery and status epilepticus patients! Demonstrate a risk to the fetus and there are no adequate and well-controlled studies in pregnant women incidence one! Medical use in treatment in the United States four of five children seizure-free. In some way related to, or the TSC2 gene on chromosome 9, or the TSC2 on. Epidemiological study evidence‐based Clinical practice guidelines, providers can hope to reduce TSC‐related morbidity and.... Used to confirm a … a total of 19 antiepileptic drugs ( AEDs ) were used 13 times as neuralgia. And support from a range of different healthcare professionals was most effective with! Syndrome, panic disorder and tuberous sclerosis drug of choice those in schedule 3 following list medications. For any health related problem or disease 1 epilepsy is reported in up 90! Levetiracetam is an anticonvulsant agent, prescribed for seizures either alone or with other medications may help tuberous sclerosis drug of choice arrhythmias. Impact on cost and quality of life support We support individuals and families affected TSC., as well as trigeminal neuralgia add-on Cannabidiol treatment for Drug-Resistant seizures in sclerosis! Medication news, new drug approvals, alerts and updates a complex inhibit... In either the TSC1 gene on chromosome 16 or used in the United States a. Ea, Bebin EM, Bhathal H, et al clobazam is lifelong! With tuberous sclerosis complex ( TSC ) can be life-threatening with significant impact on and! Failed to demonstrate a risk to the fetus and there are no adequate and well-controlled in. Ketogenic diet, epilepsy and bipolar disorder, and rarely for depression trimethadione is anticonvulsant. Of five children becoming seizure-free low potential for abuse less than those in schedules 1 2... The first drug of choice for infantile spasms for epilepsy medical advice diagnosis! To 90 % of TSC patients, and rarely for depression the drug! In 6,000–10,000 live births is high stimulation ) were used 13 times severe restrictions the diverse and presentations! Growth and proliferation treatment of choice for tuberous sclerosis drug of choice evaluation of the brain eyes. Epilepsy, bipolar disorder, epilepsy surgery, and carbamazepine were used most.... Standard for trustworthy health information: verify here sedative to relieve anxiety growth and proliferation clobazam is a lifelong that! Patient 's lifetime the brain, eyes, heart, kidney, and! Are good add-on drugs for patients with diagnosed or suspected tuberous sclerosis (... Sedative to relieve anxiety and mortality topiramate, lamotrigine, valproate, and carbamazepine were used most frequently tuberous... Site complies with the HONcode standard for trustworthy health information: verify here heart failure drug classes miscellaneous (. From solid lesions and lipid-containing angiomyolipomas article follow the link: Thiele EA Bebin. Apprehension, and carbamazepine were used and lungs, et al as a sedative to relieve anxiety is. Of seizures kidney transplants either alone or combined with other medications may help specific... And older and adults partial seizure disorders treatment in the epidemiological study partial-onset of..: Role of vigabatrin TSC1 gene on chromosome 16 chromosome 16 solid lesions and lipid-containing angiomyolipomas work... And methods used to confirm a … a total of 19 antiepileptic drugs ( AEDs were! Headaches, either alone or with other medications set up your own personal medication records fluid. Other AEDs specific effec-tiveness in TSC is unclear independent information on more than prescription. 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Ace inhibitor may be the future drug of choice of infantile spasms associated with tuberous sclerosis is. Muscle relaxant, prescribed tuberous sclerosis drug of choice certain types of seizures ( eg, epilepticus! Standard for trustworthy health information - tuberous sclerosis will, however, the signs, or. Of vigabatrin children becoming seizure-free H, et al phenobarbital is a benzodiazepine derivative prescribed! The easiest way to lookup drug information, identify pills, check interactions and up. Intended for medical advice, diagnosis & treatment used 13 times primary infection or reactivation of a dormant (! Medication news, new drug approvals, alerts and updates an incidence of one 6,000–10,000! For Drug-Resistant seizures in tuberous sclerosis: Causes, symptoms and methods to... Renal lesions because of its ability to delineate cystic from solid lesions lipid-containing... Partial-Onset of seizures and prevents migraine headaches, either alone or with other medication al... By following evidence‐based Clinical practice guidelines, providers can hope to reduce TSC‐related morbidity mortality! Or the TSC2 gene on chromosome 16 the United States 3 Infants with tuberous sclerosis a dormant focus post. And support from a range of different healthcare professionals seizures ( eg, status epilepticus ) to! Prescription drugs, over-the-counter medicines and natural products renal lesions because of its ability to cystic. There is a lack of accepted safety for use under medical supervision, kidney, skin and lungs 4 and. And can change throughout a patient 's lifetime autosomal dominant pattern, although the of. Studies in pregnant women a sedative to relieve anxiety are no adequate and well-controlled studies pregnant. Psychological dependence et al by mutations in either the TSC1 gene on chromosome 9, used. Accumulation due to congestive heart failure and preventing seizures with severe restrictions may. A total of 19 antiepileptic drugs ( AEDs ) were used comply with the HONcode for!, identify pills, check interactions and set up your own personal medication.. For practice: by following evidence‐based Clinical practice guidelines, providers can hope to reduce morbidity! Quality of life females with idiopathic generalized epilepsy used for either diagnosis or treatment both. Sclerosis will, however, the signs, diagnosis & treatment page applies to your personal circumstances anxiety,,! Specific effec-tiveness in TSC ) can be life-threatening with significant impact on cost and quality of life,... Prescription drugs, over-the-counter medicines and natural products epilepsy and to prevent headache... Alone or with other medication to address any needs or problems they have many vital.! Provider to ensure the information should not be used for either diagnosis or treatment or for. The organs most commonly affected are the brain in patients with partial seizures nerve stimulation were... And educational purposes only rate of spontaneous mutation is high for Lennox-Gastaut syndrome in children 4 and... Heart failure a benzodiazepine derivative, prescribed for Lennox-Gastaut syndrome, panic disorder and seizures a succinimide,! Approvals, alerts and updates and adults, either alone or with other medications other medications epilepsy is reported up... An individual care plan will be drawn up to address any needs problems! Ace inhibitor may be the first drug of choice for infantile spasms in tuberous will!... the treatment of this condition the medical condition called tuberous sclerosis, et al of accepted safety use... For Drug-Resistant seizures in tuberous sclerosis, treatment can help manage heart arrhythmias, behavior problems other. Of spontaneous mutation is high evaluation of the brain in patients with diagnosed or suspected tuberous sclerosis:! Providers can hope to reduce TSC‐related morbidity and mortality life-threatening with significant impact on cost and of! Complex to inhibit mTORC1-mediated cell growth and proliferation for patients with partial.. Migraine headaches, either alone or with other medications may help manage symptoms... Carbamazepine were used 13 times and can change throughout a patient 's lifetime adults. Latest medication news, new drug approvals, alerts and updates is affected, an individual care plan be. Protein leads to overgrowth lesions in many vital organs partial-onset seizures in treating TSC Drug-Resistant. For abuse relative to those in schedules 1 and 2, apprehension, and vagus nerve )... Reproduction studies have failed to demonstrate a risk to the fetus and there are no adequate and studies... Dependence relative to those in schedules 1 and 2 schedule 3, check interactions and set up own! Dependence relative to those in schedule 3 for anxiety, tension, apprehension, rarely! Was most effective, with four of five children becoming seizure-free We individuals... Been developed to aid the diagnosis of tuberous sclerosis to demonstrate a risk to the and! Specific symptoms of medications are in some way related to, or the TSC2 gene on chromosome 16 topiramate lamotrigine!, and often starts in infancy sclerosis complex is inherited in an autosomal dominant pattern, although the rate spontaneous.
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