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Long-term safety was consistent with the double-blind phase and contrasted with earlier SUPPORT study findings in higher-risk patients, suggesting that eltrombopag may have a role in treating thrombocytopenia in patients with low-/intermediate-risk myelodysplastic syndrome. </jats:p>","journal":"Acta Haematologica","year":2023,"id":642768,"datarank":0.24141568686511508,"base_score":1.6094379124341003,"endowment":1.6094379124341003,"self_citation_contribution":0.24141568686511508,"citation_network_contribution":0.0,"self_endowment_contribution":0.24141568686511508,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":4,"citer_count":4,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":6,"is_dataset":false,"is_dataset_confidence":null,"is_data_producer":false,"deposit_databanks":null,"is_oa":false,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":null,"fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":284265,"name":"Uwe Platzbecker","orcid":"0000-0003-1863-3239","position":1,"is_corresponding":false},{"id":1363125,"name":"Sebastian Grosicki","orcid":"0000-0003-2644-1050","position":2,"is_corresponding":false},{"id":1672080,"name":"Tomasz Lawniczek","orcid":null,"position":3,"is_corresponding":false},{"id":1672081,"name":"Zewen Zhu","orcid":null,"position":4,"is_corresponding":false},{"id":1672082,"name":"Dominik Selleslag","orcid":null,"position":5,"is_corresponding":false},{"id":1620873,"name":"Moshe Mittelman","orcid":null,"position":0,"is_corresponding":false}],"reference_count":0,"raw_metadata":{"has_enrichment":true,"resolved":true,"title":"Long-Term Safety and Efficacy of Eltrombopag for Advanced Myelodysplastic Syndromes or Acute Myeloid Leukemia and Severe Thrombocytopenia: Results of the ASPIRE Extension Study","abstract":"<jats:p>ASPIRE, a three-part, international, phase 2 trial (ClinicalTrials.gov identifier: NCT01440374), investigated eltrombopag efficacy and safety in patients with advanced myelodysplastic syndrome or acute myeloid leukemia and grade 4 thrombocytopenia (&amp;lt;25 × 109 platelets/L). Approximately 30–65% of patients in this open-label extension phase experienced clinically relevant thrombocytopenic events; no conclusions could be made regarding long-term efficacy (non-randomized design, no placebo control), and survival rates may simply reflect advanced disease. Long-term safety was consistent with the double-blind phase and contrasted with earlier SUPPORT study findings in higher-risk patients, suggesting that eltrombopag may have a role in treating thrombocytopenia in patients with low-/intermediate-risk myelodysplastic syndrome. </jats:p>","is_dataset_classified":null,"base_score":1.6094379124341003,"endowment":1.6094379124341003,"datacite_reuse_total":6,"file_count":0,"downloads":0,"views":0,"has_version_chain":false,"is_dataset":false,"is_oa":false,"pmid":"37231838","pmcid":"PMC10614231","openalex_id":"https://openalex.org/W4378348187","authors":[],"funders":[],"total_grants":0,"fwci":0.7345,"citation_percentile":0.70152629,"influential_citations":0,"citation_trend":[{"year":2025,"count":2},{"year":2026,"count":2}],"oa_status":"green","license":"cc-by-nc","oa_locations":[{"url":"https://www.ncbi.nlm.nih.gov/pmc/articles/10614231","host_type":"repository"},{"url":"https://www.ncbi.nlm.nih.gov/pmc/articles/10614231","host_type":"repository"},{"url":"https://karger.com/aha/article-pdf/146/5/373/4014283/000531146.pdf","host_type":"publisher"},{"url":"https://doi.org/10.1159/000531146","host_type":"journal"},{"url":"https://pubmed.ncbi.nlm.nih.gov/37231838","host_type":"repository"},{"url":"https://ul.qucosa.de/id/qucosa%3A103271","host_type":"repository"},{"url":"https://europepmc.org/articles/PMC10614231","host_type":"Europe_PMC"},{"url":"https://europepmc.org/articles/PMC10614231?pdf=render","host_type":"Europe_PMC"}],"fields_of_study":["Acute Myeloid Leukemia Research","Platelet Disorders and Treatments","Myeloproliferative Neoplasms: Diagnosis and Treatment","Humans","Benzoates","Hydrazines","Leukemia, Myeloid, Acute","Leukopenia","Myelodysplastic Syndromes","Thrombocytopenia","Treatment Outcome","Pyrazoles"],"mesh_terms":["Benzoates","Humans","Hydrazines","Leukopenia","Myelodysplastic Syndromes","Pyrazoles","Thrombocytopenia","Leukemia, Myeloid, Acute","Treatment Outcome"],"keywords":["Eltrombopag","Medicine","Myelodysplastic syndromes","Myeloid leukemia","Internal medicine","Placebo","Randomized controlled trial","Acute leukemia","Oncology","Leukemia","Platelet","Bone marrow","Pathology","Acute myeloid leukemia","Thrombocytopenia","Advanced Myelodysplastic Syndrome"],"sdg_mappings":[{"sdg_number":0,"sdg_label":"Good health and well-being"}],"linked_datasets":[{"doi":"10.6084/m9.figshare.22954532","title":"Supplementary Material for: Long-term Safety and Efficacy of Eltrombopag for Advanced Myelodysplastic Syndromes or Acute Myeloid Leukemia and Severe Thrombocytopenia: Results of the ASPIRE Extension Study","publisher":"Karger Publishers","resource_type":"Dataset"},{"doi":"10.6084/m9.figshare.22954532.v1","title":"Supplementary Material for: Long-term Safety and Efficacy of Eltrombopag for Advanced Myelodysplastic Syndromes or Acute Myeloid Leukemia and Severe Thrombocytopenia: Results of the ASPIRE Extension Study","publisher":"Karger Publishers","resource_type":"Dataset"},{"doi":"10.6084/m9.figshare.22954019.v1","title":"Supplementary Material for: Long-term Safety and Efficacy of Eltrombopag for Advanced Myelodysplastic Syndromes or Acute Myeloid Leukemia and Severe Thrombocytopenia: Results of the ASPIRE Extension Study","publisher":"Karger Publishers","resource_type":"Dataset"},{"doi":"10.6084/m9.figshare.22954019","title":"Supplementary Material for: Long-term Safety and Efficacy of Eltrombopag for Advanced Myelodysplastic Syndromes or Acute Myeloid Leukemia and Severe Thrombocytopenia: Results of the ASPIRE Extension Study","publisher":"Karger Publishers","resource_type":"Dataset"},{"doi":"10.6084/m9.figshare.22953908","title":"Supplementary Material for: Long-term Safety and Efficacy of Eltrombopag for Advanced Myelodysplastic Syndromes or Acute Myeloid Leukemia and Severe Thrombocytopenia: Results of the ASPIRE Extension Study","publisher":"Karger Publishers","resource_type":"Dataset"},{"doi":"10.6084/m9.figshare.22953908.v1","title":"Supplementary Material for: Long-term Safety and Efficacy of Eltrombopag for Advanced Myelodysplastic Syndromes or Acute Myeloid Leukemia and Severe Thrombocytopenia: Results of the ASPIRE Extension Study","publisher":"Karger Publishers","resource_type":"Dataset"}],"clinical_trials":[],"software_tools":[],"database_accessions":[{"name":"nct"}],"source":"live","citation_network_status":"fetched"},"created_at":"2026-08-08T03:34:51.669986Z","pmid":null,"pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}