{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,21]],"date-time":"2026-08-21T13:29:37Z","timestamp":1787318977167,"version":"build-2736575974"},"reference-count":27,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2019,11,6]],"date-time":"2019-11-06T00:00:00Z","timestamp":1572998400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100000054","name":"National Cancer Institute","doi-asserted-by":"publisher","award":["P30\u2010CA008748\u201053"],"award-info":[{"award-number":["P30\u2010CA008748\u201053"]}],"id":[{"id":"10.13039\/100000054","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100002800","name":"Desmoid Tumor Research Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100002800","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["acsjournals.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Cancer"],"published-print":{"date-parts":[[2020,2]]},"abstract":"<jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Desmoid tumors (or aggressive fibromatosis) are locally infiltrative connective\u2010tissue tumors that can arise in any anatomic location; they can be asymptomatic, or they can result in pain, deformity, swelling, and loss of mobility and\/or threaten visceral organs with bowel perforation, hydronephrosis, neurovascular damage, and other complications. Existing clinical trial endpoints such as the Response Evaluation Criteria in Solid Tumors (version 1.1) and progression\u2010free survival are inadequate in capturing treatment efficacy. This study was designed to develop a novel clinical trial endpoint by capturing patient\u2010reported outcomes (PROs).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Following best practices in qualitative methodology, this study used concept elicitation (CE) interviews to explore desmoid patients' perspectives on key disease\u2010related symptoms and impacts. Qualitative analysis was performed to determine the relative frequency and disturbance of symptoms and impacts as well as other characteristics of these concepts. A draft PRO scale was then developed and tested with cognitive interviewing. Information from the interviews was subsequently incorporated into the refined PRO scale.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>CE interviews with desmoid patients (n\u00a0=\u00a031) helped to identify salient concepts and led to a draft scale that included symptom and impact scales. Cognitive interviews were completed with additional patients (n\u00a0=\u00a015) across 3 phases. Patient input was used to refine instructions, revise and\/or remove items, and modify the response scale. This resulted in an 11\u2010item symptom scale and a 17\u2010item impact scale.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>This is the first disease\u2010specific PRO instrument developed for desmoid tumors. The instrument is available as an exploratory endpoint in clinical trials. This study highlights the feasibility and challenges of developing PRO instruments for rare diseases.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/cncr.32555","type":"journal-article","created":{"date-parts":[[2019,11,6]],"date-time":"2019-11-06T04:04:00Z","timestamp":1573013040000},"page":"531-539","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":49,"title":["Prospective development of a patient\u2010reported outcomes instrument for desmoid tumors or aggressive fibromatosis"],"prefix":"10.1002","volume":"126","author":[{"given":"Mrinal M.","family":"Gounder","sequence":"first","affiliation":[{"name":"Memorial Sloan Kettering Cancer Center New York New York"},{"name":"Weill Cornell Medical College New York New York"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"LeAnne","family":"Maddux","sequence":"additional","affiliation":[{"name":"IQVIA New York New York"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean","family":"Paty","sequence":"additional","affiliation":[{"name":"IQVIA New York New York"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4965-3913","authenticated-orcid":false,"given":"Thomas M.","family":"Atkinson","sequence":"additional","affiliation":[{"name":"Memorial Sloan Kettering Cancer Center New York New York"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2019,11,6]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2017.75.8482"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1093\/annonc\/mdx323"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1805052"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2018.77.9819"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00520-018-4386-8"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrclinonc.2017.153"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMp1611252"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1470-2045(18)30141-4"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2017.7156"},{"key":"e_1_2_8_11_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2015.63.0830"},{"key":"e_1_2_8_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpainsymman.2010.01.017"},{"key":"e_1_2_8_13_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamaoncol.2015.4719"},{"key":"e_1_2_8_14_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11136-018-1931-3"},{"key":"e_1_2_8_15_1","doi-asserted-by":"publisher","DOI":"10.1093\/jnci\/85.5.365"},{"key":"e_1_2_8_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jclinepi.2010.04.011"},{"key":"e_1_2_8_17_1","doi-asserted-by":"publisher","DOI":"10.1002\/1097-0142(20001001)89:7<1634::AID-CNCR29>3.0.CO;2-V"},{"key":"e_1_2_8_18_1","unstructured":"US Food and Drug Administration.Patient\u2010Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. 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