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Morris MJ, de Bono J, Nagarajah J, Sartor O, Wei XX, Nordquist LT, Koshkin VS, Chi KN, Krause BJ, Herrmann K, Rahbar K, Vickers A, Mirante O, Ghouse R, Fizazi K, Tagawa ST. Correlation analyses of radiographic progression-free survival with clinical and health-related quality of life outcomes in metastatic castration-resistant prostate cancer: analysis of the phase 3 VISION trial. Cancer. 2024 Jun 21. doi: 10.1002/cncr.35438
Forsythe E, Haws RM, Argente J, Beales P, Martos-Moreno GA, Dollfus H, Chirila C, Gnanasakthy A, Buckley BC, Mallya UG, Clement K, Haqq AM. Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet-Biedl syndrome: phase 3 trial results. Orphanet J Rare Dis. 2023 Jan 16;18(1):12. doi: 10.1186/s13023-022-02602-4
Kuhnen P, Wabitsch M, von Schnurbein J, Chirila C, Mallya UG, Callahan P, Gnanasakthy A, Poitou C, Krabusch PM, Stewart M, Clement K. Quality of life outcomes in two phase 3 trials of setmelanotide in patients with obesity due to LEPR or POMC deficiency. Orphanet J Rare Dis. 2022 Feb 5;17(1):38. doi: 10.1186/s13023-022-02186-z
Mareque M, Montesinos P, Font P, Guinea JM, de la Fuente A, Soto J, Oyaguez I, Brockbank J, Iglesias T, Llinares J, Sierra J. Cost-effectiveness analysis of gemtuzumab ozogamicin for first-line treatment of patients with Cd-33 positive acute myeloid leukaemia in Spain. Clinicoecon Outcomes Res. 2021 Apr 22;2021(13):263-77. doi: 10.2147/CEOR.S302097
Noel E, Dussol B, Lacombe D, Bedreddine N, Fouilhoux A, Ronco P, Genevaz D, Bekri S, Hagege A, Dupuis-Simeon F, Derrien Ansquer V, Germain DP, Lidove O. Treatment needs and expectations for Fabry disease in France: development of a new Patient Needs Questionnaire. Orphanet J Rare Dis. 2019 Dec 4;14(1):284. doi: 10.1186/s13023-019-1254-7
Crew KD, Silverman TB, Vanegas A, Trivedi MS, Dimond J, Mata J, Sin M, Jones T, Terry MB, Tsai W-Y, Kukafka R. Study protocol: randomized controlled trial of web-based decision support tools for high-risk women and healthcare providers to increase breast cancer chemoprevention. Contemp Clin Trials Commun. 2019 Aug 22;22(16):100433. doi: 10.1016/j.conctc.2019.10043
Paquin RS, Fischer R, Mansfield C, Mange B, Beaverson K, Ganot A, Martin AS, Morris C, Rensch C, Ricotti V, Russo LJ, Sadosky A, Smith EC, Peay H. Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best–worst scaling experiment in caregivers and adult patients. Orphanet J Rare Dis. 2019 May 9;14(1):102. doi: 10.1186/s13023-019-1069-6
Hunter JE, Zepp JM, Gilmore MJ, Davis JV, Esterberg EJ, Muessig KR, Peterson SK, Syngal S, Acheson LS, Wiesner GL, Reiss JA, Goddard KA. Universal tumor screening for Lynch syndrome: assessment of the perspectives of patients with colorectal cancer regarding benefits and barriers. Cancer. 2015 Sep 15;121(18):3281-9. doi: 10.1002/cncr.29470
Burris HA, Lebrun F, Rugo HS, Beck T, Piccart M, Neven P, Baselga J, Petrakova K, Hortobagyi GN, Komorowski A, Chouinard E, Young R, Gnant M, Pritchard KI, Bennett L, Ricci J-F, Bauly H, Taran T, Sahmoud T, Noguchi S. Health-related quality of life of patients with advanced breast cancer treated with everolimus plus exemestane versus placebo plus exemestane in the phase 3, randomized, controlled, BOLERO-2 trial. Cancer. 2013 Mar 15;119(10):1908-15.
Maurel J, Martins AS, Poveda A, Lopez-Guerrero JA, Cubedo R, Casado A, Martinez-Trufero J, Ramon Ayuso J, Lopez-Pousa A, Garcia-Albeniz X, Garcia del Muro X, de Alava E. Imatinib plus low-dose doxorubicin in patients with advanced gastrointestinal stromal tumors refractory to high-dose imatinib: a phase I-II study by the Spanish Group for Research on Sarcomas. Cancer. 2010 Aug 1;116(15):3692-701. doi: 10.1002/cncr.25111