{"doi":"10.1177/26320843251396745","title":"Retrospective analysis of the TOPPIC trial using DOOR methodology","abstract":"<jats:sec>\n                    <jats:title>Background</jats:title>\n                    <jats:p>Established analysis methods for composite outcomes can be complex to interpret, clinically and statistically. To counter this, composite outcomes are frequently reduced to a binary outcome. This can simplify interpretation but there exists a danger that deeper understanding of patient experience is lost. Rank-based DOOR methodology is emerging as an attractive option, using all components of a composite outcome. DOOR methodology has been employed retrospectively to the TOPPIC trial (ISRCTN89489788) to determine if making fuller use of data could enhance study results.</jats:p>\n                  </jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods</jats:title>\n                    <jats:p>TOPPIC patients were assigned a clinical outcome rank (1 = most desirable to 8 = least desirable). DOOR methodology was applied, testing the null hypothesis of a DOOR probability of 50% (no difference). Additionally, smokers and non-smokers were considered separately. Win ratio and ordinal logistic regression provided supportive analyses.</jats:p>\n                  </jats:sec>\n                  <jats:sec>\n                    <jats:title>Results</jats:title>\n                    <jats:p>\n                      Employing DOOR methodology demonstrated the distribution of ranks differed between treatment groups - DOOR probability of a more desirable outcome with active treatment of 54.6% (95% CI 53.8% - 55.4%,\n                      <jats:italic toggle=\"yes\">p</jats:italic>\n                      &lt; 0.0001). Considering smokers and non-smokers separately, this difference was amplified in smokers (DOOR probability 66.1% (95% CI 62.7% - 69.4%,\n                      <jats:italic toggle=\"yes\">p</jats:italic>\n                      &lt; 0.0001). Win ratio methodology and ordinal logistic regression showed no difference between treatments.\n                    </jats:p>\n                  </jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions</jats:title>\n                    <jats:p>DOOR methodology was applied successfully to a previously published trial and shows potential to discriminate clinical outcomes effectively by deconstructing a binary endpoint of component parts into an ordinal outcome. This approach should be considered when designing trials, either as principal analysis of a rank-based outcome or as a confirmatory sensitivity analysis.</jats:p>\n                  </jats:sec>","journal":"Research Methods in Medicine &amp; Health Sciences","year":2026,"id":621716,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":0,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"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":1605639,"name":"Catriona Keerie","orcid":"0000-0001-5459-6178","position":0,"is_corresponding":false}],"reference_count":0,"raw_metadata":{"has_enrichment":true,"resolved":true,"title":"Retrospective analysis of the TOPPIC trial using DOOR methodology","abstract":"<jats:sec>\n                    <jats:title>Background</jats:title>\n                    <jats:p>Established analysis methods for composite outcomes can be complex to interpret, clinically and statistically. To counter this, composite outcomes are frequently reduced to a binary outcome. This can simplify interpretation but there exists a danger that deeper understanding of patient experience is lost. Rank-based DOOR methodology is emerging as an attractive option, using all components of a composite outcome. DOOR methodology has been employed retrospectively to the TOPPIC trial (ISRCTN89489788) to determine if making fuller use of data could enhance study results.</jats:p>\n                  </jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods</jats:title>\n                    <jats:p>TOPPIC patients were assigned a clinical outcome rank (1 = most desirable to 8 = least desirable). DOOR methodology was applied, testing the null hypothesis of a DOOR probability of 50% (no difference). Additionally, smokers and non-smokers were considered separately. Win ratio and ordinal logistic regression provided supportive analyses.</jats:p>\n                  </jats:sec>\n                  <jats:sec>\n                    <jats:title>Results</jats:title>\n                    <jats:p>\n                      Employing DOOR methodology demonstrated the distribution of ranks differed between treatment groups - DOOR probability of a more desirable outcome with active treatment of 54.6% (95% CI 53.8% - 55.4%,\n                      <jats:italic toggle=\"yes\">p</jats:italic>\n                      &lt; 0.0001). Considering smokers and non-smokers separately, this difference was amplified in smokers (DOOR probability 66.1% (95% CI 62.7% - 69.4%,\n                      <jats:italic toggle=\"yes\">p</jats:italic>\n                      &lt; 0.0001). Win ratio methodology and ordinal logistic regression showed no difference between treatments.\n                    </jats:p>\n                  </jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions</jats:title>\n                    <jats:p>DOOR methodology was applied successfully to a previously published trial and shows potential to discriminate clinical outcomes effectively by deconstructing a binary endpoint of component parts into an ordinal outcome. This approach should be considered when designing trials, either as principal analysis of a rank-based outcome or as a confirmatory sensitivity analysis.</jats:p>\n                  </jats:sec>","is_dataset_classified":null,"base_score":0.0,"endowment":0.0,"datacite_reuse_total":0,"file_count":0,"downloads":0,"views":0,"has_version_chain":false,"is_dataset":false,"is_oa":false,"pmid":null,"pmcid":null,"openalex_id":null,"authors":[],"funders":[],"total_grants":0,"fwci":null,"citation_percentile":null,"influential_citations":0,"citation_trend":[],"oa_status":"hybrid","license":"cc-by","oa_locations":[{"url":"https://doi.org/10.1177/26320843251396745","host_type":"publisher"},{"url":"https://journals.sagepub.com/doi/pdf/10.1177/26320843251396745","host_type":"publisher"},{"url":"https://journals.sagepub.com/doi/full-xml/10.1177/26320843251396745","host_type":"publisher"},{"url":"https://www.research.ed.ac.uk/en/publications/20cf63da-f20d-4a01-b8e2-56e85919ee3a","host_type":"repository"}],"fields_of_study":[],"mesh_terms":[],"keywords":[],"sdg_mappings":[],"linked_datasets":[],"clinical_trials":[],"software_tools":[],"database_accessions":[],"source":"live","citation_network_status":"fetched"},"created_at":"2026-08-03T15:42:12.220773Z","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":[]}