Health Utility in Relapsed/Refractory Diffuse Large B-Cell Lymphoma (RR-DLBCL) Patients - Results of a Phase II Trial with ORAL Selinexor

Author(s)

Casasnovas RO¹, Daniele P², Tremblay G³, Maerevoet M⁴, Zijlstra J⁵, Follows G⁶, Vermaat JSP⁷, Kalakonda N⁸, Goy AH⁹, Choquet S¹⁰, Van Den Neste E¹¹, Hill BT¹², Thieblemont C¹³, Cavallo F¹⁴, de la Cruz F¹⁵, Kuruvilla J¹⁶, Hamad N¹⁷, Bouabdallah R¹⁸, Jäger U¹⁹, Caimi P²⁰, Gurion R²¹, Warzocha K²², Bakhshi S²³, Sancho JM²⁴, Schuster M²⁵, Egyed M²⁶, Offner F²⁷, Vasilakopoulos T²⁸, Samal P²⁹, Nagy A³⁰, Ku M³¹, Canales Albendea MÁ³²
¹CHU Dijon Bourgogne, Dijon, France, ²Purple Squirrel Economics, Montreal, QC, Canada, ³Purple Squirrel Economics, New York, NY, USA, ⁴Institut Jules Bordet, Brussels, Belgium, ⁵Amsterdam UMC, Amsterdam, Netherlands, ⁶Addenbrooke's Hospital, Cambridge, UK, ⁷Leiden University Medical Center, Leiden, Netherlands, ⁸Institute of Translational Medicine, Liverpool, UK, ⁹Hackensack University Medical Center, Hackensack, NJ, USA, ¹⁰Hôpital Pitié-Salpêtrière, Paris, France, ¹¹Université catholique de Louvain, Ottignies-Louvain-la-Neuve, Belgium, ¹²Cleveland Clinic, Cleveland, OH, USA, ¹³Hôpital Saint-Louis, Paris, France, ¹⁴University of Turin, Turin, Italy, ¹⁵Hospital Universitario Virgen del Rocío, Sevilla, Spain, ¹⁶University Health Network Research, Toronto, ON, Canada, ¹⁷St Vincent’s Hospital, Melbourne, Australia, ¹⁸Institut Paoli-Calmettes, Marseille, France, ¹⁹Medical University of Vienna, Vienna, Austria, ²⁰UH Cleveland Medical Center, Cleveland, OH, USA, ²¹Tel Aviv University, Tel Aviv, Israel, ²²Instytut Hematologii i Transfuzjologii, Warsaw, Poland, ²³All India Institute Of Medical Sciences, New Delhi, India, ²⁴Germans Trias i Pujol Hospital, Barcelona, Spain, ²⁵Stony Brook University, New York, NY, USA, ²⁶Somogy County Kaposi Mór Hospital, Kaposvár, Hungary, ²⁷Ghent University Hospital, Ghent, Belgium, ²⁸National and Kapodistrian University of Athens, Athens, Greece, ²⁹Institute of Hematology and Transfusion Medicine, Kolkata, India, ³⁰Semmelweis University, Budapest, Hungary, ³¹St Vincent’s Hospital, Melbourne, VIC, Australia, ³²Hospital Universitario La Paz, Madrid, Spain

OBJECTIVES : Well-tolerated oral therapies are essential for maintaining health utility in patients with RR-DLBCL. We assessed the impact of single-agent oral selinexor on health utility in patients with RR-DLBCL in a single arm, phase II, open-label, multicenter trial.

METHODS : EuroQol-5D-5L (EQ5D) was administered to patients at baseline and at each cycle until the end of treatment, and scored using the US value set. EQ5D health utility scores describe health-related quality of life on a scale ranging from 0 (dead) to 1 (full health). Patients within the modified intent-to-treat population with complete EQ5D at baseline and ≥1 follow-up were included (N=89/127). Multivariable mixed-effects models were used to estimate longitudinal health utility by treatment response, health state utility values, and disutility of grade ≥3 and serious adverse events (AEs).

RESULTS : Treatment responders had a higher EQ5D index score than non-responders by cycle 3 (mean difference: 0.075 [95%CI: 0.023, 0.126]; p<0.001), which was maintained through the end of treatment (mean difference: 0.249 [95%CI: 0.095, 0.402]; p<0.001). Health state utility values at the end of treatment were estimated as 0.669 (95%CI: 0.619, 0.719) for progressive disease, 0.721 (95%CI: 0.658, 0.784) for stable disease, and 0.739 (95%CI: 0.689, 0.790) for treatment response. Stable disease and treatment response were associated with higher health utility compared to progressive disease with mean differences of 0.052 (p=0.073) and 0.07 (p=0.001), respectively. Grade ≥3 and serious AEs were associated with significant declines in health utility of -0.038 (95%CI: -0.062, -0.013; p=0.002) and -0.036 (95%CI: -0.067, -0.006; p=0.02), respectively.

CONCLUSIONS : Patients with objective response to selinexor maintained significantly higher health utilities than non-responders. Observed differences based on treatment response exceeded the lower threshold for clinically meaningful differences (>0.06), while decrements associated with AEs did not. Health utilities derived from this study may be useful for future health economic evaluations of selinexor.

Conference/Value in Health Info

2020-11, ISPOR Europe 2020, Milan, Italy

Value in Health, Volume 23, Issue S2 (December 2020)

Code

PCN325

Topic

Clinical Outcomes, Economic Evaluation, Epidemiology & Public Health, Patient-Centered Research

Topic Subcategory

Clinician Reported Outcomes, Cost-comparison, Effectiveness, Utility, Benefit Analysis, Health State Utilities, Safety & Pharmacoepidemiology

Disease

Oncology

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