{"id":"https://openalex.org/W3213601753","doi":"https://doi.org/10.1182/blood-2021-145902","title":"Trametinib for Refractory Chylous Effusions in Children with Noonan Syndrome","display_name":"Trametinib for Refractory Chylous Effusions in Children with Noonan Syndrome","publication_year":2021,"publication_date":"2021-11-05","ids":{"openalex":"https://openalex.org/W3213601753","doi":"https://doi.org/10.1182/blood-2021-145902","mag":"3213601753"},"language":"en","primary_location":{"id":"doi:10.1182/blood-2021-145902","doi":"https://doi.org/10.1182/blood-2021-145902","is_oa":false,"landing_page_url":"https://doi.org/10.1182/blood-2021-145902","pdf_url":null,"source":{"id":"https://openalex.org/S200071133","display_name":"Blood","issn_l":"0006-4971","issn":["0006-4971","1528-0020"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-a","doyens","jufo-3","ki-jl-3","medline","norway-2","poland-200","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Blood","raw_type":"journal-article"},"volume":null,"type":"conference-abstract","indexed_in":["crossref"],"open_access":{"is_oa":false,"oa_status":"closed","oa_url":null,"any_repository_has_fulltext":false},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5023281720","display_name":"Alexander W. Rankin","orcid":"https://orcid.org/0000-0002-6089-9279","observed_orcids":["https://orcid.org/0000-0002-6089-9279"]},"institutions":[{"id":"https://openalex.org/I4210096275","display_name":"Children's Hospital Colorado","ror":"https://ror.org/00mj9k629","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210096275"]},{"id":"https://openalex.org/I51713134","display_name":"University of Colorado Anschutz","ror":"https://ror.org/03wmf1y16","country_code":"US","type":"education","lineage":["https://openalex.org/I51713134"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Alexander W. Rankin","raw_affiliation_strings":["Center for Cancer and Blood Disorders, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Center for Cancer and Blood Disorders, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO","institution_ids":["https://openalex.org/I4210096275","https://openalex.org/I51713134"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5010311294","display_name":"Aparna Annam","orcid":"https://orcid.org/0000-0002-3709-2805","observed_orcids":["https://orcid.org/0000-0002-3709-2805"]},"institutions":[{"id":"https://openalex.org/I4210096275","display_name":"Children's Hospital Colorado","ror":"https://ror.org/00mj9k629","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210096275"]},{"id":"https://openalex.org/I51713134","display_name":"University of Colorado Anschutz","ror":"https://ror.org/03wmf1y16","country_code":"US","type":"education","lineage":["https://openalex.org/I51713134"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Aparna Annam","raw_affiliation_strings":["Department of Pediatric Radiology, Children's Hospital Colorado, Aurora, CO","Vascular Anomalies Center, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pediatric Radiology, Children's Hospital Colorado, Aurora, CO","institution_ids":["https://openalex.org/I4210096275"]},{"raw_affiliation_string":"Vascular Anomalies Center, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO","institution_ids":["https://openalex.org/I4210096275","https://openalex.org/I51713134"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5088867670","display_name":"Kathryn C. Chatfield","orcid":"https://orcid.org/0000-0003-0021-1097","observed_orcids":["https://orcid.org/0000-0003-0021-1097"]},"institutions":[{"id":"https://openalex.org/I4210096275","display_name":"Children's Hospital Colorado","ror":"https://ror.org/00mj9k629","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210096275"]},{"id":"https://openalex.org/I51713134","display_name":"University of Colorado Anschutz","ror":"https://ror.org/03wmf1y16","country_code":"US","type":"education","lineage":["https://openalex.org/I51713134"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Kathryn Chatfield","raw_affiliation_strings":["Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO","Vascular Anomalies Center, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO","institution_ids":["https://openalex.org/I51713134"]},{"raw_affiliation_string":"Vascular Anomalies Center, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO","institution_ids":["https://openalex.org/I4210096275","https://openalex.org/I51713134"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5009873031","display_name":"Lauren R. S. Hill","orcid":"https://orcid.org/0000-0002-0220-7262","observed_orcids":["https://orcid.org/0000-0002-0220-7262"]},"institutions":[{"id":"https://openalex.org/I4210096275","display_name":"Children's Hospital Colorado","ror":"https://ror.org/00mj9k629","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210096275"]},{"id":"https://openalex.org/I51713134","display_name":"University of Colorado Anschutz","ror":"https://ror.org/03wmf1y16","country_code":"US","type":"education","lineage":["https://openalex.org/I51713134"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Lauren R. Hill","raw_affiliation_strings":["Vascular Anomalies Center, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Vascular Anomalies Center, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO","institution_ids":["https://openalex.org/I4210096275","https://openalex.org/I51713134"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5080587020","display_name":"Ann Marie Kulungowski","orcid":"https://orcid.org/0009-0003-5563-107X","observed_orcids":["https://orcid.org/0009-0003-5563-107X"]},"institutions":[{"id":"https://openalex.org/I4210096275","display_name":"Children's Hospital Colorado","ror":"https://ror.org/00mj9k629","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210096275"]},{"id":"https://openalex.org/I51713134","display_name":"University of Colorado Anschutz","ror":"https://ror.org/03wmf1y16","country_code":"US","type":"education","lineage":["https://openalex.org/I51713134"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Ann Kulungowski","raw_affiliation_strings":["Department of Pediatric Surgery, Children's Hospital Colorado, Aurora, CO","Vascular Anomalies Center, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Pediatric Surgery, Children's Hospital Colorado, Aurora, CO","institution_ids":["https://openalex.org/I4210096275"]},{"raw_affiliation_string":"Vascular Anomalies Center, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO","institution_ids":["https://openalex.org/I4210096275","https://openalex.org/I51713134"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5056906558","display_name":"Leslie M. McCallen","orcid":"https://orcid.org/0000-0002-8358-5168","observed_orcids":["https://orcid.org/0000-0002-8358-5168"]},"institutions":[{"id":"https://openalex.org/I4210096275","display_name":"Children's Hospital Colorado","ror":"https://ror.org/00mj9k629","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210096275"]},{"id":"https://openalex.org/I51713134","display_name":"University of Colorado Anschutz","ror":"https://ror.org/03wmf1y16","country_code":"US","type":"education","lineage":["https://openalex.org/I51713134"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Leslie McCallen","raw_affiliation_strings":["Vascular Anomalies Center, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Vascular Anomalies Center, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO","institution_ids":["https://openalex.org/I4210096275","https://openalex.org/I51713134"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5065735225","display_name":"Taizo A. Nakano","orcid":"https://orcid.org/0000-0003-0389-2297","observed_orcids":["https://orcid.org/0000-0003-0389-2297"]},"institutions":[{"id":"https://openalex.org/I4210096275","display_name":"Children's Hospital Colorado","ror":"https://ror.org/00mj9k629","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210096275"]},{"id":"https://openalex.org/I51713134","display_name":"University of Colorado Anschutz","ror":"https://ror.org/03wmf1y16","country_code":"US","type":"education","lineage":["https://openalex.org/I51713134"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Taizo A. Nakano","raw_affiliation_strings":["Center for Cancer and Blood Disorders, Children's Hospital Colorado, Denver, CO","Vascular Anomalies Center, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Center for Cancer and Blood Disorders, Children's Hospital Colorado, Denver, CO","institution_ids":["https://openalex.org/I4210096275"]},{"raw_affiliation_string":"Vascular Anomalies Center, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO","institution_ids":["https://openalex.org/I4210096275","https://openalex.org/I51713134"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":2,"corresponding_author_ids":[],"corresponding_institution_ids":[],"apc_list":{"value":5500,"currency":"USD","value_usd":5500},"apc_paid":null,"fwci":null,"has_fulltext":false,"cited_by_count":5,"citation_normalized_percentile":null,"cited_by_percentile_year":null,"biblio":{"volume":"138","issue":"Supplement 1","first_page":"3140","last_page":"3140"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T12426","display_name":"Lymphatic Disorders and Treatments","score":0.9955999851226807,"subfield":{"id":"https://openalex.org/subfields/2746","display_name":"Surgery"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},"topics":[{"id":"https://openalex.org/T12426","display_name":"Lymphatic Disorders and Treatments","score":0.9955999851226807,"subfield":{"id":"https://openalex.org/subfields/2746","display_name":"Surgery"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},{"id":"https://openalex.org/T12104","display_name":"Protein Tyrosine Phosphatases","score":0.0008999999845400453,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T13430","display_name":"Lipid metabolism and disorders","score":0.00039999998989515007,"subfield":{"id":"https://openalex.org/subfields/2705","display_name":"Cardiology and Cardiovascular Medicine"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/noonan-syndrome","display_name":"Noonan syndrome","score":0.9660000205039978},{"id":"https://openalex.org/keywords/chylous-effusion","display_name":"chylous effusion","score":0.8339999914169312},{"id":"https://openalex.org/keywords/lymphatic-malformation","display_name":"lymphangioma","score":0.9359999895095825},{"id":"https://openalex.org/keywords/rasopathies","display_name":"RASopathies","score":0.9049999713897705},{"id":"https://openalex.org/keywords/mek-inhibition","display_name":"MEK inhibition","score":0.9160000085830688},{"id":"https://openalex.org/keywords/sos1-mutations","display_name":"SOS1 mutations","score":0.9959999918937683},{"id":"https://openalex.org/keywords/ptpn11-mutation","display_name":"PTPN11 mutation","score":0.9670000076293945},{"id":"https://openalex.org/keywords/chylous-pleural-effusion","display_name":"chylous pleural effusion","score":0.8659999966621399},{"id":"https://openalex.org/keywords/chylous-ascites","display_name":"chylous ascites","score":0.906000018119812},{"id":"https://openalex.org/keywords/hypertrophic-cardiomyopathy","display_name":"hypertrophic cardiomyopathy","score":0.7400000095367432},{"id":"https://openalex.org/keywords/pulmonary-valve-stenosis","display_name":"pulmonary valve stenosis","score":0.7749999761581421}],"concepts":[{"id":"https://openalex.org/C2776149932","wikidata":"https://www.wikidata.org/wiki/Q1543446","display_name":"Noonan syndrome","level":2,"score":0.9092857837677002},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.8654665946960449},{"id":"https://openalex.org/C2778472372","wikidata":"https://www.wikidata.org/wiki/Q7833138","display_name":"Trametinib","level":4,"score":0.6066215634346008},{"id":"https://openalex.org/C2779944767","wikidata":"https://www.wikidata.org/wiki/Q1136492","display_name":"Costello syndrome","level":5,"score":0.5527639389038086},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.496537983417511},{"id":"https://openalex.org/C90924648","wikidata":"https://www.wikidata.org/wiki/Q120569","display_name":"Gastroenterology","level":1,"score":0.3535487651824951},{"id":"https://openalex.org/C141071460","wikidata":"https://www.wikidata.org/wiki/Q40821","display_name":"Surgery","level":1,"score":0.327163428068161},{"id":"https://openalex.org/C121608353","wikidata":"https://www.wikidata.org/wiki/Q12078","display_name":"Cancer","level":2,"score":0.21160098910331726},{"id":"https://openalex.org/C2781187634","wikidata":"https://www.wikidata.org/wiki/Q20969980","display_name":"KRAS","level":4,"score":0.12120142579078674},{"id":"https://openalex.org/C526805850","wikidata":"https://www.wikidata.org/wiki/Q188874","display_name":"Colorectal cancer","level":3,"score":0.11178457736968994},{"id":"https://openalex.org/C95444343","wikidata":"https://www.wikidata.org/wiki/Q7141","display_name":"Cell biology","level":1,"score":0.0},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.0},{"id":"https://openalex.org/C184235292","wikidata":"https://www.wikidata.org/wiki/Q421851","display_name":"Kinase","level":2,"score":0.0},{"id":"https://openalex.org/C57074206","wikidata":"https://www.wikidata.org/wiki/Q14197552","display_name":"MAPK/ERK pathway","level":3,"score":0.0}],"mesh":[],"locations_count":1,"locations":[{"id":"doi:10.1182/blood-2021-145902","doi":"https://doi.org/10.1182/blood-2021-145902","is_oa":false,"landing_page_url":"https://doi.org/10.1182/blood-2021-145902","pdf_url":null,"source":{"id":"https://openalex.org/S200071133","display_name":"Blood","issn_l":"0006-4971","issn":["0006-4971","1528-0020"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-a","doyens","jufo-3","ki-jl-3","medline","norway-2","poland-200","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Blood","raw_type":"journal-article"}],"best_oa_location":null,"sustainable_development_goals":[{"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being","score":1.0}],"study_designs":[],"awards":[],"funders":[],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":0,"referenced_works":[],"related_works":["https://openalex.org/W4280636445","https://openalex.org/W4366368403","https://openalex.org/W7140204388","https://openalex.org/W4391053177","https://openalex.org/W4403967367","https://openalex.org/W4407716003","https://openalex.org/W4366414695","https://openalex.org/W3100601967","https://openalex.org/W4411670579","https://openalex.org/W4293168873"],"abstract_inverted_index":{"Abstract":[0],"BACKGROUND":[1],"Noonan":[2,298,639],"syndrome":[3],"(NS)":[4],"is":[5,89,618,645],"one":[6,153,375,439],"of":[7,18,49,67,74,91,101,155,182,229,274,343,377,441,482,487,504,527,542,561,577,592,601,651],"several":[8],"autosomal":[9],"dominant":[10],"multisystem":[11],"disorders":[12],"known":[13],"as":[14,402,415,466,468,540],"RASopathies.":[15,502],"Common":[16],"manifestations":[17],"NS":[19,43,102,175,222,268,321,499],"include":[20],"congenital":[21],"heart":[22],"defects":[23],"and":[24,28,44,51,116,141,196,214,240,251,259,290,322,357,372,393,423,431,448,500,571,595],"cardiomyopathy,":[25,192,280,566],"lymphatic":[26,38,346,351,403,564,643],"malformations,":[27,565],"predisposition":[29],"to":[30,37,55,84,113,121,149,177,209,224,256,270,297,307,388,478,533,588,596],"myeloproliferative":[31,300],"disorders.":[32],"Chylous":[33],"fluid":[34],"accumulation":[35,352],"secondary":[36],"malformations":[39],"are":[40,45,103,366,494,586],"seen":[41],"in":[42,64,71,295,317,350,368,412,420,436,445,462,496,507,558,579,604,629,636,655],"a":[46,98,132,158,171,178,218,225,264,271,418,559,619],"major":[47],"cause":[48],"morbidity":[50],"mortality":[52],"often":[53],"refractory":[54,112,208,255,306],"conventional":[56,114],"medical":[57,327],"management.":[58,309],"There":[59],"has":[60,623],"been":[61,305,624],"increasing":[62],"interest":[63],"the":[65,72,139,163,183,230,358,479,485,505,515,537,543,574,590,598,627,648,652],"use":[66,125,603,628,635],"pharmacologic":[68],"MEK":[69,86,311,488,593],"inhibition":[70,312,489,594],"management":[73,115],"these":[75,656],"patients":[76,96,146,319,334,384,497,506,545],"given":[77],"that":[78,88,206,253,303,348,457,493,622],"activating":[79],"RAS":[80],"pathway":[81,654],"mutations":[82],"lead":[83],"downstream":[85],"activation":[87],"causative":[90],"this":[92,528,580,605],"pathology.":[93],"DESIGN/METHODS":[94],"Three":[95],"with":[97,123,174,189,221,236,267,278,286,313,320,325,443,455,459,498,638,641],"confirmed":[99],"diagnosis":[100],"described.":[104],"Each":[105],"patient":[106,134,364,519,581],"developed":[107,201,246,522],"complications":[108,324],"from":[109,128,138,390,514,518],"chylous":[110,203,248,292],"effusions":[111,205,250,294],"were":[117,147,207,254],"subsequently":[118],"enrolled":[119],"on-study":[120],"treat":[122],"compassionate":[124],"oral":[126,380],"trametinib":[127,314,379,578],"Novartis":[129],"Pharmaceuticals":[130],"on":[131,490,536,647],"single":[133],"Investigational":[135],"New":[136],"Drug":[137,142],"Food":[140],"Administration":[143],"(FDA).":[144],"All":[145,332],"consented":[148],"be":[150,534],"monitored":[151],"for":[152,363,626],"year":[154,440],"therapy":[156,529],"following":[157],"local":[159],"protocol":[160],"approved":[161,625],"by":[162,417,427],"Colorado":[164],"Institutional":[165],"Review":[166],"Board":[167],"(COMIRB).":[168],"Patient":[169,216,262,408,451],"1:":[170],"4-year-old":[172],"female":[173,220],"due":[176,223,269],"pathogenic":[179,226],"germline":[180,227],"mutation":[181,228,273],"RIT1":[184],"gene":[185,232],"[c.246T>G,":[186],"p.Phe82Leu]":[187],"born":[188,235],"severe":[190],"hypertrophic":[191,279,413],"mitral":[193],"valve":[194,198,243,282,568],"dysplasia,":[195],"pulmonary":[197,242,281,288,567],"stenosis.":[199,244],"She":[200,245],"bilateral":[202,247],"pleural":[204,249,293],"dietary":[210,257],"modification,":[211],"diuretics,":[212],"octreotide,":[213],"sirolimus.":[215],"2:":[217],"3-month-old":[219],"SOS1":[231],"[c.1322G>A;":[233],"p.Cys441Tyr]":[234],"esophageal":[237],"atresia/tracheoesophageal":[238],"fistula":[239],"moderate":[241],"ascites":[252],"modification":[258],"octreotide":[260],"therapy.":[261],"3:":[263],"4-month-old":[265],"male":[266],"gain-of-function":[272],"PTPN11":[275],"[c.854T>C;":[276],"p.Phe285Ser]":[277],"stenosis,":[283,569],"respiratory":[284],"insufficiency":[285],"suspected":[287],"lymphangiectasia,":[289],"persistent":[291],"addition":[296],"syndrome-associated":[299],"disorder":[301],"(NS-MPD)":[302],"had":[304,335,548],"traditional":[308],"RESULTS":[310],"was":[315],"used":[316],"three":[318,333,383,544],"life-threatening":[323],"no":[326],"or":[328],"surgical":[329],"treatment":[330],"options.":[331],"dynamic":[336],"contrast":[337],"magnetic":[338],"resonance":[339],"lymphangiography":[340],"(DCMRL)":[341],"evidence":[342,483],"primary,":[344],"central":[345],"dysplasia":[347],"manifested":[349],"affecting":[353],"cardiorespiratory":[354],"function,":[355],"nutrition,":[356],"immune":[359],"system.":[360],"DCMRL":[361],"imaging":[362],"2":[365,432,520],"highlighted":[367],"Figure":[369,607,609],"1":[370,430,608],"A":[371],"B.":[373],"Within":[374],"month":[376],"initiating":[378],"therapy,":[381,442],"all":[382],"demonstrated":[385,433],"response":[386],"adequate":[387],"wean":[389],"mechanical":[391],"ventilation":[392],"other":[394,501],"supportive":[395],"care":[396],"modalities.":[397],"Serum":[398],"albumin":[399],"levels":[400],"improved":[401],"leak":[404],"resolved":[405],"(Figure":[406,472],"1C).":[407],"3":[409,452],"showed":[410],"improvement":[411],"cardiomyopathy":[414],"evidenced":[416],"decrease":[419],"both":[421,446],"NT-proBNP":[422],"left":[424],"ventricular":[425],"mass":[426],"echocardiogram.":[428],"Patients":[429],"notable":[434],"improvements":[435,461,557],"growth":[437],"after":[438],"increase":[444],"weight":[447],"height":[449],"percentiles.":[450],"also":[453],"presented":[454],"NS-MPD":[456,572],"responded":[458],"marked":[460],"total":[463],"WBC":[464],"count":[465,471],"well":[467],"absolute":[469],"monocyte":[470],"1D).":[473],"DISCUSSION":[474],"Our":[475],"experience":[476],"adds":[477],"growing":[480],"body":[481],"demonstrating":[484],"effectiveness":[486],"disease":[491],"processes":[492],"common":[495],"None":[503],"our":[508],"series":[509],"experienced":[510],"significant":[511,642],"adverse":[512],"effects":[513],"medication":[516],"aside":[517],"who":[521],"mild":[523],"dermatitis.":[524],"The":[525],"efficacy":[526,591],"does":[530],"not":[531],"appear":[532],"based":[535,646],"underlying":[538,550],"genotype,":[539],"each":[541],"we":[546],"describe":[547],"different":[549],"molecular":[551],"alterations":[552],"(SOS1,":[553],"RIT1,":[554],"PTPN11).":[555],"Substantial":[556],"variety":[560],"parameters":[562],"including":[563],"growth,":[570],"highlight":[573],"potential":[575],"utility":[576],"population.":[582,606],"Larger,":[583],"prospective":[584],"studies":[585],"necessary":[587],"confirm":[589],"assess":[597],"long-term":[599],"safety":[600],"its":[602],"1.":[610],"Disclosures":[611],"Nakano:":[612],"Novartis:":[613],"Consultancy.":[614],"OffLabel":[615],"Disclosure:":[616],"Trametinib":[617],"MEK1/2":[620],"inhibitor":[621],"certain":[630],"malignancies.":[631],"Its":[632],"off":[633],"label":[634],"children":[637],"Syndrome":[640],"anomalies":[644],"up":[649],"regulation":[650],"MAPK":[653],"patients.":[657]},"counts_by_year":[{"year":2024,"cited_by_count":3},{"year":2023,"cited_by_count":1},{"year":2022,"cited_by_count":1}],"updated_date":"2026-09-22T06:50:58.402377","created_date":"2021-11-22T00:00:00"}