{"id":"https://openalex.org/W7204495702","doi":"https://doi.org/10.3389/fphar.2026.1906711","title":"Case Report: Inotersen reintroduction after thrombocytopenia in hereditary transthyretin amyloidosis: a case series","display_name":"Case Report: Inotersen reintroduction after thrombocytopenia in hereditary transthyretin amyloidosis: a case series","publication_year":2026,"publication_date":"2026-08-28","ids":{"openalex":"https://openalex.org/W7204495702","doi":"https://doi.org/10.3389/fphar.2026.1906711","pmid":"https://pubmed.ncbi.nlm.nih.gov/42729459","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/13562058"},"language":"en","primary_location":{"id":"doi:10.3389/fphar.2026.1906711","doi":"https://doi.org/10.3389/fphar.2026.1906711","is_oa":true,"landing_page_url":"https://doi.org/10.3389/fphar.2026.1906711","pdf_url":"https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2026.1906711/pdf","source":{"id":"https://openalex.org/S132108250","display_name":"Frontiers in Pharmacology","issn_l":"1663-9812","issn":["1663-9812"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","ki-jl-1","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320527","host_organization_name":"Frontiers Media","host_organization_lineage":["https://openalex.org/P4310320527"],"host_organization_lineage_names":["Frontiers Media"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Frontiers in Pharmacology","raw_type":"journal-article"},"volume":null,"type":"article","is_traditional":true,"indexed_in":["crossref","doaj","pubmed"],"open_access":{"is_oa":true,"oa_status":"gold","oa_url":"https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2026.1906711/pdf","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5113255368","display_name":"C\u00e9sar A. Crespi","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210100657","display_name":"Hospital San Juan de Dios","ror":"https://ror.org/0166mge61","country_code":"VE","type":"healthcare","lineage":["https://openalex.org/I4210100657"]}],"countries":["VE"],"is_corresponding":true,"raw_author_name":"Cesar A. Crespi","raw_affiliation_strings":["Centro de Referencia en Enfermedades Raras y de Dificultoso Diagn\u00f3stico (CERyD), Hospital Interzonal Especializado de Agudos y Cr\u00f3nicos San Juan de Dios"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Centro de Referencia en Enfermedades Raras y de Dificultoso Diagn\u00f3stico (CERyD), Hospital Interzonal Especializado de Agudos y Cr\u00f3nicos San Juan de Dios","institution_ids":["https://openalex.org/I4210100657"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5059692404","display_name":"Gisela Zanga","orcid":"https://orcid.org/0000-0002-2730-6734","observed_orcids":["https://orcid.org/0000-0002-2730-6734"]},"institutions":[{"id":"https://openalex.org/I4210163650","display_name":"Hospital C\u00e9sar Milstein","ror":"https://ror.org/047krn550","country_code":"AR","type":"healthcare","lineage":["https://openalex.org/I4210163650"]}],"countries":["AR"],"is_corresponding":false,"raw_author_name":"Gisela Zanga","raw_affiliation_strings":["Servicio de Neurolog\u00eda, Hospital Cesar Milstein"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Servicio de Neurolog\u00eda, Hospital Cesar Milstein","institution_ids":["https://openalex.org/I4210163650"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5083569463","display_name":"Mar\u00eda Florencia Ardanaz","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210100657","display_name":"Hospital San Juan de Dios","ror":"https://ror.org/0166mge61","country_code":"VE","type":"healthcare","lineage":["https://openalex.org/I4210100657"]}],"countries":["VE"],"is_corresponding":false,"raw_author_name":"Maria Florencia Ardanaz","raw_affiliation_strings":["Servicio de Alergia, Hospital Interzonal Especializado de Agudos y Cr\u00f3nicos San Juan de Dios"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Servicio de Alergia, Hospital Interzonal Especializado de Agudos y Cr\u00f3nicos San Juan de Dios","institution_ids":["https://openalex.org/I4210100657"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5092373327","display_name":"Marcela Chinigo","orcid":"https://orcid.org/0009-0005-9990-8602","observed_orcids":["https://orcid.org/0009-0005-9990-8602"]},"institutions":[{"id":"https://openalex.org/I4210100657","display_name":"Hospital San Juan de Dios","ror":"https://ror.org/0166mge61","country_code":"VE","type":"healthcare","lineage":["https://openalex.org/I4210100657"]}],"countries":["VE"],"is_corresponding":false,"raw_author_name":"Marcela Pia Chinigo","raw_affiliation_strings":["Servicio de Alergia, Hospital Interzonal Especializado de Agudos y Cr\u00f3nicos San Juan de Dios"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Servicio de Alergia, Hospital Interzonal Especializado de Agudos y Cr\u00f3nicos San Juan de Dios","institution_ids":["https://openalex.org/I4210100657"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5148483014","display_name":"Graciana Bianco","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210100657","display_name":"Hospital San Juan de Dios","ror":"https://ror.org/0166mge61","country_code":"VE","type":"healthcare","lineage":["https://openalex.org/I4210100657"]}],"countries":["VE"],"is_corresponding":false,"raw_author_name":"Graciana Bianco","raw_affiliation_strings":["Servicio de Farmacia, Hospital Interzonal Especializado de Agudos y Cr\u00f3nicos San Juan de Dios"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Servicio de Farmacia, Hospital Interzonal Especializado de Agudos y Cr\u00f3nicos San Juan de Dios","institution_ids":["https://openalex.org/I4210100657"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5148531258","display_name":"Andrea Del Mar Iba\u00f1ez Aldecoa","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210100657","display_name":"Hospital San Juan de Dios","ror":"https://ror.org/0166mge61","country_code":"VE","type":"healthcare","lineage":["https://openalex.org/I4210100657"]}],"countries":["VE"],"is_corresponding":false,"raw_author_name":"Andrea Del Mar Iba\u00f1ez Aldecoa","raw_affiliation_strings":["Servicio de Farmacia, Hospital Interzonal Especializado de Agudos y Cr\u00f3nicos San Juan de Dios"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Servicio de Farmacia, Hospital Interzonal Especializado de Agudos y Cr\u00f3nicos San Juan de Dios","institution_ids":["https://openalex.org/I4210100657"]}]}],"institutions":[],"countries_distinct_count":2,"institutions_distinct_count":2,"corresponding_author_ids":["https://openalex.org/A5113255368"],"corresponding_institution_ids":["https://openalex.org/I4210100657"],"apc_list":{"value":3150,"currency":"CHF","value_usd":3801},"apc_paid":{"value":3150,"currency":"CHF","value_usd":3801},"fwci":0.0,"has_fulltext":true,"cited_by_count":0,"citation_normalized_percentile":{"value":0.0,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":null,"biblio":{"volume":"17","issue":null,"first_page":"1906711","last_page":"1906711"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T11305","display_name":"Amyloidosis: Diagnosis, Treatment, Outcomes","score":1.0,"subfield":{"id":"https://openalex.org/subfields/2724","display_name":"Internal Medicine"},"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/T11305","display_name":"Amyloidosis: Diagnosis, Treatment, Outcomes","score":1.0,"subfield":{"id":"https://openalex.org/subfields/2724","display_name":"Internal Medicine"},"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/T10086","display_name":"Alzheimer's disease research and treatments","score":0.0,"subfield":{"id":"https://openalex.org/subfields/2804","display_name":"Cellular and Molecular Neuroscience"},"field":{"id":"https://openalex.org/fields/28","display_name":"Neuroscience"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T10855","display_name":"Amyotrophic Lateral Sclerosis Research","score":0.0,"subfield":{"id":"https://openalex.org/subfields/2728","display_name":"Clinical Neurology"},"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/inotersen","display_name":"inotersen","score":0.9829999804496765},{"id":"https://openalex.org/keywords/familial-amyloid-polyneuropathy","display_name":"hereditary transthyretin amyloidosis","score":0.9390000104904175},{"id":"https://openalex.org/keywords/thrombocytopenia","display_name":"thrombocytopenia","score":0.9869999885559082},{"id":"https://openalex.org/keywords/drug-reintroduction","display_name":"drug reintroduction","score":0.8730000257492065},{"id":"https://openalex.org/keywords/cardiac-amyloidosis","display_name":"cardiac amyloidosis","score":0.7839999794960022},{"id":"https://openalex.org/keywords/adverse-drug-reactions","display_name":"adverse drug reactions","score":0.5640000104904175},{"id":"https://openalex.org/keywords/platelet-monitoring","display_name":"platelet monitoring","score":0.7559999823570251}],"concepts":[{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.9175999760627747},{"id":"https://openalex.org/C121157162","wikidata":"https://www.wikidata.org/wiki/Q419999","display_name":"Transthyretin","level":2,"score":0.7324000000953674},{"id":"https://openalex.org/C197934379","wikidata":"https://www.wikidata.org/wiki/Q2047938","display_name":"Adverse effect","level":2,"score":0.558899998664856},{"id":"https://openalex.org/C2775892965","wikidata":"https://www.wikidata.org/wiki/Q1502312","display_name":"Polyneuropathy","level":2,"score":0.5435000061988831},{"id":"https://openalex.org/C2779951007","wikidata":"https://www.wikidata.org/wiki/Q816798","display_name":"Amyloidosis","level":2,"score":0.4887999892234802},{"id":"https://openalex.org/C187212893","wikidata":"https://www.wikidata.org/wiki/Q123028","display_name":"Pediatrics","level":1,"score":0.46630001068115234},{"id":"https://openalex.org/C89560881","wikidata":"https://www.wikidata.org/wiki/Q101026","display_name":"Platelet","level":2,"score":0.44780001044273376},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.4323999881744385},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.39399999380111694},{"id":"https://openalex.org/C141071460","wikidata":"https://www.wikidata.org/wiki/Q40821","display_name":"Surgery","level":1,"score":0.38370001316070557},{"id":"https://openalex.org/C2777601897","wikidata":"https://www.wikidata.org/wiki/Q3409113","display_name":"Presentation (obstetrics)","level":2,"score":0.3296999931335449},{"id":"https://openalex.org/C2779701055","wikidata":"https://www.wikidata.org/wiki/Q929833","display_name":"Rare disease","level":3,"score":0.313400000333786},{"id":"https://openalex.org/C3019796121","wikidata":"https://www.wikidata.org/wiki/Q5048356","display_name":"Case presentation","level":2,"score":0.29980000853538513},{"id":"https://openalex.org/C90924648","wikidata":"https://www.wikidata.org/wiki/Q120569","display_name":"Gastroenterology","level":1,"score":0.2687999904155731},{"id":"https://openalex.org/C2776689292","wikidata":"https://www.wikidata.org/wiki/Q23925170","display_name":"Platelet transfusion","level":3,"score":0.2646999955177307}],"mesh":[],"locations_count":4,"locations":[{"id":"doi:10.3389/fphar.2026.1906711","doi":"https://doi.org/10.3389/fphar.2026.1906711","is_oa":true,"landing_page_url":"https://doi.org/10.3389/fphar.2026.1906711","pdf_url":"https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2026.1906711/pdf","source":{"id":"https://openalex.org/S132108250","display_name":"Frontiers in Pharmacology","issn_l":"1663-9812","issn":["1663-9812"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","ki-jl-1","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320527","host_organization_name":"Frontiers Media","host_organization_lineage":["https://openalex.org/P4310320527"],"host_organization_lineage_names":["Frontiers Media"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Frontiers in Pharmacology","raw_type":"journal-article"},{"id":"pmh:oai:pubmedcentral.nih.gov:13562058","doi":null,"is_oa":true,"landing_page_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC13562058/","pdf_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC13562058/pdf/fphar-17-1906711.pdf","source":{"id":"https://openalex.org/S2764455111","display_name":"PubMed Central","issn_l":null,"issn":null,"is_oa":true,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Front Pharmacol","raw_type":"Text"},{"id":"pmid:42729459","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/42729459","pdf_url":null,"source":{"id":"https://openalex.org/S4306525036","display_name":"PubMed","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Frontiers in pharmacology","raw_type":"Case Reports"},{"id":"pmh:oai:doaj.org/article:174e1cf5e67349bfb5c9763adb36a14b","doi":null,"is_oa":false,"landing_page_url":"https://doaj.org/article/174e1cf5e67349bfb5c9763adb36a14b","pdf_url":null,"source":{"id":"https://openalex.org/S4306401280","display_name":"DOAJ (DOAJ: Directory of Open Access Journals)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Frontiers in Pharmacology, Vol 17 (2026)","raw_type":"article"}],"best_oa_location":{"id":"doi:10.3389/fphar.2026.1906711","doi":"https://doi.org/10.3389/fphar.2026.1906711","is_oa":true,"landing_page_url":"https://doi.org/10.3389/fphar.2026.1906711","pdf_url":"https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2026.1906711/pdf","source":{"id":"https://openalex.org/S132108250","display_name":"Frontiers in Pharmacology","issn_l":"1663-9812","issn":["1663-9812"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","ki-jl-1","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320527","host_organization_name":"Frontiers Media","host_organization_lineage":["https://openalex.org/P4310320527"],"host_organization_lineage_names":["Frontiers Media"],"type":"journal"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Frontiers in Pharmacology","raw_type":"journal-article"},"sustainable_development_goals":[{"score":1.0,"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being"}],"study_designs":[{"id":"https://openalex.org/study-designs/case-report","display_name":"Case Report"}],"awards":[],"funders":[],"has_content":{"grobid_xml":true,"pdf":true},"content_urls":{"pdf":"https://content.openalex.org/works/W7204495702.pdf","grobid_xml":"https://content.openalex.org/works/W7204495702.grobid-xml"},"referenced_works_count":9,"referenced_works":["https://openalex.org/W1874346921","https://openalex.org/W2026544302","https://openalex.org/W2054162904","https://openalex.org/W2084369625","https://openalex.org/W2264641932","https://openalex.org/W2769073740","https://openalex.org/W2809989268","https://openalex.org/W4380046654","https://openalex.org/W4412879635"],"related_works":["https://openalex.org/W4220795474","https://openalex.org/W3006387795","https://openalex.org/W4220752763","https://openalex.org/W3018892538","https://openalex.org/W2966060984","https://openalex.org/W2955523092","https://openalex.org/W2809063562","https://openalex.org/W7214902074","https://openalex.org/W2912011902","https://openalex.org/W2809989268"],"abstract_inverted_index":{"Background:":[0],"ATTRv":[1,66,92,124,222],"amyloidosis":[2],"is":[3,18,34,54],"a":[4,35,83,108,118,169,227,258],"progressive":[5],"multisystem":[6],"disease":[7],"caused":[8],"by":[9],"pathogenic":[10],"variants":[11],"in":[12,25,71,220],"the":[13,21],"transthyretin":[14,29,206],"(TTR)":[15],"gene.":[16],"Inotersen":[17],"approved":[19],"for":[20,249],"treatment":[22,43],"of":[23,50,105,111,161,164,205],"polyneuropathy":[24],"adults":[26],"with":[27,63,86,107,121,137,226],"hereditary":[28],"mediated":[30],"amyloidosis,":[31],"but":[32,235],"thrombocytopenia":[33,53,76,101,133,147,217,251],"clinically":[36],"relevant":[37],"adverse":[38],"event":[39],"that":[40],"may":[41],"require":[42],"interruption":[44],"or":[45],"discontinuation.":[46],"Evidence":[47],"guiding":[48],"reintroduction":[49,151,174,215,244],"inotersen":[51,78,183,214],"after":[52,102,134,216],"limited.":[55],"Case":[56],"Presentation:":[57],"We":[58],"retrospectively":[59],"reviewed":[60],"two":[61,69,221],"men":[62],"genetically":[64],"confirmed":[65],"treated":[67],"at":[68,186],"centers":[70],"Argentina":[72],"who":[73,97,129],"developed":[74,98,130,196],"treatment-emergent":[75],"during":[77,148],"therapy.":[79],"Patient":[80,115],"1":[81],"was":[82,117,184,218],"71-year-old":[84],"man":[85,120],"late-onset":[87],"mixed":[88],"neurologic":[89],"and":[90,144,154,159,200,231,240,252],"cardiac":[91],"due":[93,125],"to":[94,126],"TTR":[95,127],"p.Val50Met":[96,128],"grade":[99,131],"2":[100,116,190],"22":[103],"weeks":[104],"treatment,":[106],"platelet":[109,138,157,233],"nadir":[110],"58":[112],"\u00d7":[113,142],"10^9/L.":[114],"52-year-old":[119],"early-stage":[122],"neuropathic":[123],"4":[132],"65":[135],"weeks,":[136],"counts":[139],"below":[140],"20":[141],"10^9/L,":[143],"had":[145],"recurrent":[146,197],"an":[149],"initial":[150],"attempt.":[152],"Intervention":[153],"Outcomes:":[155],"After":[156],"recovery":[158],"exclusion":[160],"alternative":[162],"causes":[163],"thrombocytopenia,":[165,199],"both":[166,201],"patients":[167,223],"underwent":[168],"structured":[170,228],"7-day":[171],"stepwise":[172,229],"short-term":[173],"protocol":[175,181,230],"under":[176],"close":[177,232],"hematologic":[178],"monitoring.":[179],"Following":[180],"completion,":[182],"resumed":[185],"284":[187],"mg":[188],"every":[189],"weeks.":[191],"During":[192],"follow-up,":[193],"neither":[194],"patient":[195],"severe":[198,250],"maintained":[202],"biochemical":[203],"evidence":[204],"suppression.":[207],"Conclusion:":[208],"In":[209],"this":[210],"small":[211],"case":[212],"series,":[213],"feasible":[219],"when":[224],"implemented":[225],"monitoring,":[234],"these":[236],"findings":[237],"are":[238],"hypothesis-generating":[239],"not":[241,254],"generalizable.":[242],"One":[243],"occurred":[245],"outside":[246],"prescribing":[247],"recommendations":[248],"should":[253],"be":[255],"interpreted":[256],"as":[257],"recommended":[259],"strategy.":[260]},"counts_by_year":[],"updated_date":"2026-11-10T11:39:43.021295","created_date":"2026-08-28T00:00:00"}
