{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,31]],"date-time":"2026-08-31T11:09:48Z","timestamp":1788174588978,"version":"build-2803163510"},"reference-count":133,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2024,5,21]],"date-time":"2024-05-21T00:00:00Z","timestamp":1716249600000},"content-version":"vor","delay-in-days":20,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["HemaSphere"],"published-print":{"date-parts":[[2024,5]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Notable treatment advances have been made in recent years for patients with myelodysplastic syndromes\/neoplasms (MDS), and several new drugs are under development. For example, the emerging availability of oral MDS therapies holds the promise of improving patients' health\u2010related quality of life (HRQoL). Within this rapidly evolving landscape, the inclusion of HRQoL and other patient\u2010reported outcomes (PROs) is critical to inform the benefit\/risk assessment of new therapies or to assess whether patients live longer and better, for what will likely remain a largely incurable disease. We provide practical considerations to support investigators in generating high\u2010quality PRO data in future MDS trials. We first describe several challenges that are to be thoughtfully considered when designing an MDS\u2010focused clinical trial with a PRO endpoint. We then discuss aspects related to the design of the study, including PRO assessment strategies. We also discuss statistical approaches illustrating the potential value of time\u2010to\u2010event analyses and their implications within the estimand framework. Finally, based on a literature review of MDS randomized controlled trials with a PRO endpoint, we note the PRO items that deserve special attention when reporting future MDS trial results. We hope these practical considerations will facilitate the generation of rigorous PRO data that can robustly inform MDS patient care and support treatment decision\u2010making for this patient population.<\/jats:p>","DOI":"10.1002\/hem3.69","type":"journal-article","created":{"date-parts":[[2024,5,21]],"date-time":"2024-05-21T11:04:49Z","timestamp":1716289489000},"source":"Crossref","is-referenced-by-count":9,"title":["Toward\u00a0a more patient\u2010centered drug development process in clinical trials for patients with myelodysplastic syndromes\/neoplasms (MDS): Practical considerations from the International Consortium for MDS (icMDS)"],"prefix":"10.1002","volume":"8","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5065-5166","authenticated-orcid":false,"given":"Fabio","family":"Efficace","sequence":"first","affiliation":[{"name":"Italian Group for Adult Hematologic Diseases (GIMEMA),\u00a0Health Outcomes Research Unit GIMEMA Data Center Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rena","family":"Buckstein","sequence":"additional","affiliation":[{"name":"Department of Medical Oncology\/Hematology Sunnybrook Health Sciences Centre Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gregory A.","family":"Abel","sequence":"additional","affiliation":[{"name":"Divisions of Population Sciences and Hematologic Malignancies Dana\u2010Farber Cancer Institute Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Johannes M.","family":"Giesinger","sequence":"additional","affiliation":[{"name":"University Hospital of Psychiatry II Medical University of Innsbruck Innsbruck Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierre","family":"Fenaux","sequence":"additional","affiliation":[{"name":"H\u00f4pital Saint Louis Assistance Publique H\u00f4pitaux de Paris and Paris Cit\u00e9 University Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jan Philipp","family":"Bewersdorf","sequence":"additional","affiliation":[{"name":"Leukemia Service, Department of Medicine Memorial Sloan Kettering Cancer Center New York New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrew M.","family":"Brunner","sequence":"additional","affiliation":[{"name":"Leukemia Program, Harvard Medical School Massachusetts General Hospital Cancer Center Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rafael","family":"Bejar","sequence":"additional","affiliation":[{"name":"Division of Hematology and Oncology, Moores Cancer Center UC San Diego La Jolla California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Uma","family":"Borate","sequence":"additional","affiliation":[{"name":"Ohio State University Comprehensive Cancer Center\/James Cancer Hospital Ohio State University Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amy E.","family":"DeZern","sequence":"additional","affiliation":[{"name":"Sidney Kimmel Comprehensive Cancer Center Johns Hopkins Hospital Baltimore Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter","family":"Greenberg","sequence":"additional","affiliation":[{"name":"Department of Medicine, Division of Hematology, Cancer Institute Stanford University School of Medicine Stanford California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gail J.","family":"Roboz","sequence":"additional","affiliation":[{"name":"Weill Cornell Medical College and New York Presbyterian Hospital New York New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael R.","family":"Savona","sequence":"additional","affiliation":[{"name":"Department of Medicine, Division of Hematology\/Oncology Vanderbilt University Medical Center Nashville Tennessee USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1358-3095","authenticated-orcid":false,"given":"Francesco","family":"Sparano","sequence":"additional","affiliation":[{"name":"Italian Group for Adult Hematologic Diseases (GIMEMA),\u00a0Health Outcomes Research Unit GIMEMA Data Center Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jacqueline","family":"Boultwood","sequence":"additional","affiliation":[{"name":"Blood Cancer UK Molecular Haematology Unit, Radcliffe Department of Medicine Nuffield Division of Clinical Laboratory Sciences University of Oxford Oxford UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rami","family":"Komrokji","sequence":"additional","affiliation":[{"name":"Department of Malignant Hematology H. Lee Moffitt Cancer Center Tampa Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David A.","family":"Sallman","sequence":"additional","affiliation":[{"name":"Department of Malignant Hematology H. Lee Moffitt Cancer Center Tampa Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1266-9804","authenticated-orcid":false,"given":"Zhuoer","family":"Xie","sequence":"additional","affiliation":[{"name":"Department of Malignant Hematology H. Lee Moffitt Cancer Center Tampa Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Guillermo","family":"Sanz","sequence":"additional","affiliation":[{"name":"Health Research Institute La Fe, Valencia, Spain Hospital Universitario y Polit\u00e9cnico La Fe Valencia Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hetty E.","family":"Carraway","sequence":"additional","affiliation":[{"name":"Leukemia Program, Hematology and Medical Oncology Taussig Cancer Institute, Cleveland Clinic Cleveland Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Justin","family":"Taylor","sequence":"additional","affiliation":[{"name":"Sylvester Comprehensive Cancer Center University of Miami Miller School of Medicine Miami Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephen D.","family":"Nimer","sequence":"additional","affiliation":[{"name":"Sylvester Comprehensive Cancer Center University of Miami Miller School of Medicine Miami Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matteo Giovanni","family":"Della Porta","sequence":"additional","affiliation":[{"name":"Department of Biomedical Sciences IRCCS Humanitas Clinical and Research Center &amp; Humanitas University Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Valeria","family":"Santini","sequence":"additional","affiliation":[{"name":"Myelodysplastic Syndromes Unit, Department of Experimental and Clinical Medicine, Hematology, Azienda Ospedaliero Universitaria Careggi University of Florence Florence Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maximilian","family":"Stahl","sequence":"additional","affiliation":[{"name":"Department of Medical Oncology Dana\u2010Farber Cancer Institute and Harvard Medical School Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Uwe","family":"Platzbecker","sequence":"additional","affiliation":[{"name":"Department of Hematology and Cellular Therapy University Hospital Leipzig Leipzig Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mikkael A.","family":"Sekeres","sequence":"additional","affiliation":[{"name":"Sylvester Comprehensive Cancer Center University of Miami Miller School of Medicine Miami Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amer M.","family":"Zeidan","sequence":"additional","affiliation":[{"name":"Section of Hematology, Department of Internal Medicine Yale University School of Medicine and Yale Cancer Center New Haven Connecticut USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,5,21]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41375-022-01613-1"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2013-03-492884"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V89.6.2079"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2012-03-420489"},{"issue":"7","key":"e_1_2_9_6_1","article-title":"Molecular International Prognostic Scoring System for Myelodysplastic Syndromes","volume":"1","author":"Bernard E","year":"2022","journal-title":"N Engl J Med Evid"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41375-018-0367-7"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41375-018-0089-x"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.13138"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41375-020-0746-8"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1097\/HS9.0000000000000944"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2022009000"},{"key":"e_1_2_9_13_1","volume-title":"Guidance for Industry: Patient\u2010Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims","author":"US Food and Drug Administration","year":"2009"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2021004568"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1470-2045(15)00206-5"},{"key":"e_1_2_9_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/cncr.31193"},{"key":"e_1_2_9_17_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.26768"},{"key":"e_1_2_9_18_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.14033"},{"key":"e_1_2_9_19_1","doi-asserted-by":"publisher","DOI":"10.3390\/cancers15051388"},{"key":"e_1_2_9_20_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2020.10.017"},{"key":"e_1_2_9_21_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1470-2045(09)70200-1"},{"key":"e_1_2_9_22_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1470-2045(19)30656-4"},{"key":"e_1_2_9_23_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrclinonc.2015.29"},{"key":"e_1_2_9_24_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2007.13.3439"},{"key":"e_1_2_9_25_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.17537"},{"key":"e_1_2_9_26_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.blre.2021.100851"},{"key":"e_1_2_9_27_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.leukres.2018.06.006"},{"key":"e_1_2_9_28_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.critrevonc.2015.07.012"},{"key":"e_1_2_9_29_1","doi-asserted-by":"publisher","DOI":"10.2174\/1745017902117010307"},{"key":"e_1_2_9_30_1","doi-asserted-by":"publisher","DOI":"10.1586\/17474086.2.1.69"},{"key":"e_1_2_9_31_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.blre.2023.101114"},{"key":"e_1_2_9_32_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.critrevonc.2020.103067"},{"key":"e_1_2_9_33_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1908892"},{"key":"e_1_2_9_34_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(23)00874-7"},{"key":"e_1_2_9_35_1","doi-asserted-by":"publisher","DOI":"10.1097\/01.HS9.0000969832.59030.29"},{"key":"e_1_2_9_36_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(23)01724-5"},{"key":"e_1_2_9_37_1","doi-asserted-by":"publisher","DOI":"10.1080\/14737140.2023.2238897"},{"key":"e_1_2_9_38_1","doi-asserted-by":"publisher","DOI":"10.3390\/cancers14194941"},{"key":"e_1_2_9_39_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.xcrm.2023.100940"},{"key":"e_1_2_9_40_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-3026(18)30051-6"},{"issue":"17","key":"e_1_2_9_41_1","first-page":"2047","article-title":"Consensus proposal for revised International Working Group 2023 response criteria for higher\u2010risk myelodysplastic syndromes","volume":"141","author":"Zeidan AM","year":"2023","journal-title":"Blood"},{"key":"e_1_2_9_42_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2017-03-737403"},{"key":"e_1_2_9_43_1","doi-asserted-by":"publisher","DOI":"10.1158\/1078-0432.CCR-12-0653"},{"key":"e_1_2_9_44_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(22)02036-0"},{"key":"e_1_2_9_45_1","volume-title":"Guidance for Industry and FDA Staff Qualification Process for Drug Development Tools","author":"US Food and Drug Administration","year":"2020"},{"key":"e_1_2_9_46_1","volume-title":"Patient\u2010Focused Drug Development: Collecting Comprehensive and Representative Input. Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders","author":"US Food and Drug Administration","year":"2020"},{"key":"e_1_2_9_47_1","volume-title":"Patient\u2010Focused Drug Development: Methods to Identify What is Important to Patients. Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders","author":"US Food and Drug Administration","year":"2022"},{"key":"e_1_2_9_48_1","volume-title":"Patient\u2010Focused Drug Development: Selecting, Developing, or Modifying Fit\u2010for\u2010Purpose Clinical Outcome Assessments. Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders","author":"US Food and Drug Administration","year":"2022"},{"key":"e_1_2_9_49_1","volume-title":"Patient\u2010Focused Drug Development: Incorporating Clinical Outcome Assessments into Endpoints for Regulatory Decision\u2010Making. Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders","author":"US Food and Drug Administration","year":"2023"},{"key":"e_1_2_9_50_1","volume-title":"Oncology Working Party. Reflection Paper on the Use of Patient Reported Outcome (PRO) Measures in Oncology Studies","author":"European Medicine Agency","year":"2014"},{"key":"e_1_2_9_51_1","volume-title":"The Use of Patient\u2010reported Outcome (PRO) Measures in Oncology Studies. Appendix 2 to the Guideline on the Evaluation of Anticancer Medicinal Products in Man","author":"European Medicine Agency","year":"2016"},{"key":"e_1_2_9_52_1","volume-title":"General Methods (Version 6.1","author":"Institute for Quality and Efficiency in Health Care (IQWiG)","year":"2022"},{"key":"e_1_2_9_53_1","volume-title":"NICE Health Technology Evaluations: The Manual","author":"NICE","year":"2023"},{"key":"e_1_2_9_54_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2003.12.121"},{"key":"e_1_2_9_55_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ejca.2008.03.017"},{"key":"e_1_2_9_56_1","doi-asserted-by":"publisher","DOI":"10.1093\/jnci\/djz038"},{"key":"e_1_2_9_57_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2017.21903"},{"key":"e_1_2_9_58_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2013.879"},{"key":"e_1_2_9_59_1","doi-asserted-by":"publisher","DOI":"10.1136\/bmjopen-2020-045105"},{"key":"e_1_2_9_60_1","doi-asserted-by":"publisher","DOI":"10.1097\/00005650-199206000-00002"},{"key":"e_1_2_9_61_1","doi-asserted-by":"publisher","DOI":"10.1016\/0168-8510(90)90421-9"},{"key":"e_1_2_9_62_1","doi-asserted-by":"publisher","DOI":"10.1093\/jnci\/85.5.365"},{"key":"e_1_2_9_63_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.1993.11.3.570"},{"issue":"6","key":"e_1_2_9_64_1","first-page":"835","article-title":"Development and validation of QOL\u2010E\u00a9 instrument for the assessment of health\u2010related quality of life in myelodysplastic syndromes","volume":"8","author":"Oliva EN","year":"2013","journal-title":"Cent Eur J Med"},{"key":"e_1_2_9_65_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2015.140335"},{"key":"e_1_2_9_66_1","doi-asserted-by":"publisher","DOI":"10.1002\/cam4.5487"},{"key":"e_1_2_9_67_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jclinepi.2015.08.037"},{"key":"e_1_2_9_68_1","volume-title":"Core Patient\u2010Reported Outcomes in Cancer Clinical Trials. Guidance for Industry","author":"US Food and Drug Administration","year":"2021"},{"key":"e_1_2_9_69_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41467-022-33826-4"},{"key":"e_1_2_9_70_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41591-024-02827-9"},{"key":"e_1_2_9_71_1","doi-asserted-by":"publisher","DOI":"10.1111\/ejh.13389"},{"key":"e_1_2_9_72_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2020.11.004"},{"key":"e_1_2_9_73_1","doi-asserted-by":"publisher","DOI":"10.3390\/jcm11010027"},{"key":"e_1_2_9_74_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1470-2045(22)00654-4"},{"key":"e_1_2_9_75_1","doi-asserted-by":"publisher","DOI":"10.1186\/s41687-021-00334-w"},{"key":"e_1_2_9_76_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41591-022-01781-8"},{"key":"e_1_2_9_77_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2022.6421"},{"key":"e_1_2_9_78_1","doi-asserted-by":"publisher","DOI":"10.3390\/cancers14112742"},{"key":"e_1_2_9_79_1","doi-asserted-by":"publisher","DOI":"10.1201\/9781420061185"},{"key":"e_1_2_9_80_1","doi-asserted-by":"publisher","DOI":"10.1111\/trf.16584"},{"key":"e_1_2_9_81_1","doi-asserted-by":"publisher","DOI":"10.1002\/0470846283"},{"key":"e_1_2_9_82_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamaoncol.2016.6749"},{"key":"e_1_2_9_83_1","doi-asserted-by":"publisher","DOI":"10.1002\/(SICI)1097-0258(19980315\/15)17:5\/7<517::AID-SIM799>3.0.CO;2-S"},{"key":"e_1_2_9_84_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.clml.2017.12.004"},{"key":"e_1_2_9_85_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.16347"},{"key":"e_1_2_9_86_1","doi-asserted-by":"publisher","DOI":"10.2147\/PROM.S156279"},{"key":"e_1_2_9_87_1","doi-asserted-by":"publisher","DOI":"10.1136\/bmjopen-2015-010938"},{"key":"e_1_2_9_88_1","doi-asserted-by":"publisher","DOI":"10.1038\/clpt.2011.207"},{"key":"e_1_2_9_89_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2023.01.017"},{"key":"e_1_2_9_90_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamaoncol.2018.6205"},{"key":"e_1_2_9_91_1","doi-asserted-by":"publisher","DOI":"10.1093\/ije\/dyu115"},{"key":"e_1_2_9_92_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0159267"},{"key":"e_1_2_9_93_1","doi-asserted-by":"publisher","DOI":"10.7326\/0003-4819-157-6-201209180-00537"},{"key":"e_1_2_9_94_1","doi-asserted-by":"publisher","DOI":"10.1002\/cam4.3335"},{"key":"e_1_2_9_95_1","doi-asserted-by":"publisher","DOI":"10.1093\/jnci\/djab177"},{"key":"e_1_2_9_96_1","doi-asserted-by":"publisher","DOI":"10.1093\/jncics\/pkad002"},{"key":"e_1_2_9_97_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11136-021-02945-8"},{"key":"e_1_2_9_98_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamaoncol.2016.3328"},{"key":"e_1_2_9_99_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2020.12.015"},{"key":"e_1_2_9_100_1","volume-title":"Addendum on Estimands and Sensitivity Analysis in Clinical Trials to the Guideline on Statistical Principles for Clinical Trials E9(R1","author":"ICH Harmonised Guideline","year":"2019"},{"key":"e_1_2_9_101_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1470-2045(19)30790-9"},{"key":"e_1_2_9_102_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11136-013-0583-6"},{"key":"e_1_2_9_103_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1470-2045(22)00021-3"},{"key":"e_1_2_9_104_1","doi-asserted-by":"publisher","DOI":"10.1186\/s12955-014-0192-2"},{"key":"e_1_2_9_105_1","volume-title":"ICH E9 (R1) Addendum on Estimands and Sensitivity Analysis in Clinical Trials to the Guideline on Statistical Principles for Clinical Trials. Step 5","author":"European Medicine Agency","year":"2020"},{"key":"e_1_2_9_106_1","volume-title":"Guidance for Industry: E9(R1) Statistical Principles for Clinical Trials: Addendum: Estimands and Sensitivity Analysis in Clinical Trials","author":"US Department of Health and Human Services Food and Drug Administration","year":"2021"},{"key":"e_1_2_9_107_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1470-2045(23)00157-2"},{"key":"e_1_2_9_108_1","doi-asserted-by":"publisher","DOI":"10.1080\/10543406.2023.2280628"},{"key":"e_1_2_9_109_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jclinepi.2023.08.009"},{"key":"e_1_2_9_110_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jclinepi.2020.06.016"},{"key":"e_1_2_9_111_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.blre.2018.09.001"},{"key":"e_1_2_9_112_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2010.033506"},{"key":"e_1_2_9_113_1","doi-asserted-by":"publisher","DOI":"10.1503\/cmaj.050051"},{"key":"e_1_2_9_114_1","doi-asserted-by":"publisher","DOI":"10.1046\/j.1532-5415.2001.49281.x"},{"key":"e_1_2_9_115_1","doi-asserted-by":"publisher","DOI":"10.1093\/annonc\/mdr587"},{"key":"e_1_2_9_116_1","doi-asserted-by":"publisher","DOI":"10.1182\/hematology.2019000041"},{"key":"e_1_2_9_117_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41375-019-0666-7"},{"key":"e_1_2_9_118_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41375-020-01026-y"},{"key":"e_1_2_9_119_1","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2022008360"},{"issue":"2","key":"e_1_2_9_120_1","first-page":"136","article-title":"Quality of life and physicians' perception in myelodysplastic syndromes","volume":"2","author":"Oliva EN","year":"2012","journal-title":"Am J Blood Res"},{"key":"e_1_2_9_121_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V120.21.699.699"},{"key":"e_1_2_9_122_1","doi-asserted-by":"publisher","DOI":"10.1186\/s12955-022-02032-7"},{"key":"e_1_2_9_123_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0189648"},{"key":"e_1_2_9_124_1","doi-asserted-by":"publisher","DOI":"10.1093\/jncics\/pkaa123"},{"key":"e_1_2_9_125_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-3026(20)30292-1"},{"key":"e_1_2_9_126_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.critrevonc.2022.103649"},{"key":"e_1_2_9_127_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.lungcan.2022.10.003"},{"key":"e_1_2_9_128_1","doi-asserted-by":"publisher","DOI":"10.1093\/annonc\/mdu489"},{"key":"e_1_2_9_129_1","doi-asserted-by":"publisher","DOI":"10.1136\/bmjspcare-2019-001902"},{"issue":"1","key":"e_1_2_9_130_1","doi-asserted-by":"crossref","DOI":"10.1136\/bmjonc-2022-000021","article-title":"Time trends in health\u2010related quality of life assessment and reporting within publications of oncology randomised phase III trials: a meta\u2010research study","volume":"2","author":"Marandino L","year":"2023","journal-title":"BMJ Oncol"},{"key":"e_1_2_9_131_1","doi-asserted-by":"publisher","DOI":"10.1093\/annonc\/mdv283"},{"key":"e_1_2_9_132_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11136-022-03119-w"},{"key":"e_1_2_9_133_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2023-174660"},{"key":"e_1_2_9_134_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.leukres.2012.11.017"}],"container-title":["HemaSphere"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/hem3.69","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,1,7]],"date-time":"2025-01-07T12:42:05Z","timestamp":1736253725000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/hem3.69"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,5]]},"references-count":133,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2024,5]]}},"alternative-id":["10.1002\/hem3.69"],"URL":"https:\/\/doi.org\/10.1002\/hem3.69","archive":["Portico"],"relation":{},"ISSN":["2572-9241","2572-9241"],"issn-type":[{"value":"2572-9241","type":"print"},{"value":"2572-9241","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,5]]},"article-number":"e69"}}