Present Patient-reported Outcomes Research Articles

Health-related quality of life in transplant-eligible patients with newly diagnosed multiple myeloma treated with daratumumab, lenalidomide, bortezomib, and dexamethasone: Patient-reported outcomes from GRIFFIN.

In the phase 2 GRIFFIN trial (ClinicalTrials.gov identifier: NCT02874742), daratumumab added to lenalidomide, bortezomib, and dexamethasone (D-RVd) improved depth of response and progression-free survival (PFS) versus lenalidomide, bortezomib, and dexamethasone (RVd) alone in transplant-eligible (TE) patients with newly diagnosed multiple myeloma (NDMM). Here, we present patient-reported outcomes (PROs) collected using the European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30-item (QLQ-C30), EORTC Quality of Life Questionnaire Multiple Myeloma Module 20-item (QLQ-MY20), and EuroQol 5-Dimension 5-Level (EQ-5D-5L) tools on day 1 of cycles 1, 2, and 3; on day 21 of cycle 4 (end of induction therapy); on day 1 of cycle 5; on day 21 of cycle 6 (end of posttransplant consolidation therapy); and at months 6, 12, 18, and 24 of maintenance therapy. Meaningful improvements from baseline were seen in most of the PRO scales with both treatments after consolidation and were sustained for at least 2 years of maintenance treatment. Large reductions from baseline (~20 points) were especially observed in pain symptoms for both treatment groups, although these were numerically higher for patients receiving D-RVd during the majority of the time points. In addition, improvements in key scales, such as global health status, fatigue symptoms, and physical functioning, were also seen with both D-RVd and RVd. These improvements in health-related quality of life contribute to the totality of evidence supporting the improvement in clinical outcomes such as response rates and PFS with D-RVd in induction, consolidation, and maintenance therapy in TE patients with NDMM.

Patient-Reported Outcomes from the MCL Cohort of the Phase 1, Seamless Design TRANSCEND NHL 001 Study of Lisocabtagene Maraleucel in Patients with R/R B-Cell NHL

Background: MCL is an aggressive form of NHL. Despite high initial response rates to currently available frontline therapies for newly diagnosed MCL, patients will inevitably experience relapse. Survival outcomes are poor for patients with R/R MCL. Lisocabtagene maraleucel (liso-cel) is an autologous, CD19-directed, 4-1BB CAR T cell product administered at equal target doses of CD8 + and CD4 + CAR + T cells. In the primary analysis of the MCL cohort from the phase 1, seamless design TRANSCEND NHL 001 study (NCT02631044), liso-cel treatment resulted in a rapid, high ORR and high rate of durable CR with low incidences of grade ≥ 3 cytokine release syndrome, neurological events, and infections in heavily pretreated patients with high-risk, aggressive R/R MCL (Wang M, et al. Hematol Oncol 2023). Here, we present patient-reported outcomes (PRO) results from the MCL cohort of TRANSCEND NHL 001. Methods: Patients had R/R MCL after ≥ 2 lines of prior therapy, including a Bruton tyrosine kinase inhibitor, alkylating agent, and CD20-targeted agent. After leukapheresis and lymphodepleting chemotherapy (LDC), patients received liso-cel. Patients completed the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-30 items (EORTC QLQ-C30) and EQ-5D-5L utility index ≤ 7 days before LDC; before infusion on the day of liso-cel infusion (Day 1; baseline); then on Days 29, 60, 90, 180, 270, 365, 545, and 730 posttreatment; and at disease progression/relapse. Based on clinical relevance to MCL, the prespecified primary domains of interest were EORTC QLQ-C30 global health status/quality of life (QOL), physical functioning, fatigue, and pain. PRO analyses were conducted in all MCL patients treated with liso-cel who had an evaluable measure at baseline and at ≥ 1 postbaseline visit. The least squares (LS) mean change from baseline was estimated from a linear mixed-effects model for repeated measures at each time point above. Published minimally important difference thresholds (Cocks K, et al. Eur J Cancer 2012; Pickard AS, et al. Health Qual Life Outcomes 2007) were used to interpret whether LS mean changes from baseline were clinically meaningful. Proportions of patients with clinically meaningful change were calculated using published responder definitions of 5-30 points for EORTC QLQ-C30 domains (Cocks K, et al. Qual Life Res 2023), 0.08 for EQ-5D-5L utility index (Hernández-Alava M, et al. Pharmacoeconomics 2023), and 7 for EQ-5D-5L visual analog scale (EQ-VAS) (Pickard AS, et al. Health Qual Life Outcomes 2007). Results: Of 88 patients who received liso-cel, 66 were included in the PRO analyses (median age, 68.5 years [range, 36-86]; 76% male; 89% White; 94% not Hispanic or Latino). Baseline and clinical characteristics were comparable with those of the total liso-cel-treated population, including baseline ECOG PS scores and best response to any prior therapy. Baseline PRO scores for most primary domains were comparable with scores from a US general population norm; other domains fluctuated. Questionnaire completion rates ranged from 55% to 76% from Day 29 to Day 365 postinfusion. Overall LS mean changes from baseline showed significant and meaningful improvements for all primary domains except for pain, as well as for most other PRO domains. Although not showing meaningful improvement, pain scores remained largely unchanged through last assessment (Table 1). Across visits, most patients (60%-100%) experienced meaningful improvement or no meaningful change for all domains (primary domains shown in Table 2). More than 50% of patients achieved meaningful improvement in global health status/QOL, fatigue, social functioning, and EQ-VAS across visits after Day 29. In analyses evaluating the subset of patients in the MCL cohort with secondary CNS lymphoma (n = 5), improvements from baseline exceeded those of the non-secondary CNS population. Conclusions: Patients with R/R MCL who were treated with liso-cel in the MCL cohort of TRANSCEND NHL 001 showed meaningful improvements across relevant aspects of health-related QOL, including symptoms and functioning. Clinically meaningful improvements were observed for all primary domains except for pain, and for many of the other PRO domains. These findings complement the clinical efficacy and safety data from the primary analysis of the MCL cohort of TRANSCEND NHL 001, further supporting the benefit of liso-cel in patients with R/R MCL.

Patient-reported outcomes with durvalumab, with or without tremelimumab, plus chemotherapy as first-line treatment for metastatic non-small-cell lung cancer (POSEIDON)

ObjectivesIn the phase 3 POSEIDON study, first-line tremelimumab plus durvalumab and chemotherapy significantly improved overall survival and progression-free survival versus chemotherapy in metastatic non-small-cell lung cancer (NSCLC). We present patient-reported outcomes (PROs). Patients and methodsTreatment-naïve patients were randomized 1:1:1 to tremelimumab plus durvalumab and chemotherapy, durvalumab plus chemotherapy, or chemotherapy. PROs (prespecified secondary endpoints) were assessed using the European Organisation for Research and Treatment of Cancer 30-item core quality of life questionnaire version 3 (QLQ-C30) and its 13-item lung cancer module (QLQ-LC13). We analyzed time to deterioration (TTD) of symptoms, functioning, and global health status/quality of life (QoL) from randomization by log-rank test and improvement rates by logistic regression. Results972/1013 (96 %) patients randomized completed baseline QLQ-C30 and QLQ-LC13 questionnaires, with scores comparable between treatment arms. Patients receiving tremelimumab plus durvalumab and chemotherapy versus chemotherapy had longer median TTD for all PRO items. Hazard ratios for TTD favored tremelimumab plus durvalumab and chemotherapy for all items except diarrhea; 95 % confidence intervals did not cross 1.0 for global health status/QoL, physical functioning, cognitive functioning, pain, nausea/vomiting, insomnia, constipation, hemoptysis, dyspnea, and pain in other parts. For durvalumab plus chemotherapy, median TTD was longer versus chemotherapy for all items except nausea/vomiting and diarrhea. Hazard ratios favored durvalumab plus chemotherapy for all items except appetite loss; 95 % confidence intervals did not cross 1.0 for global health status/QoL, physical functioning, role functioning, dyspnea, and pain in other parts. For both immunotherapy plus chemotherapy arms, improvement rates in all PRO items were numerically higher versus chemotherapy, with odds ratios > 1. ConclusionsTremelimumab plus durvalumab and chemotherapy delayed deterioration in symptoms, functioning, and global health status/QoL compared with chemotherapy. Together with significant improvements in survival, these results support tremelimumab plus durvalumab and chemotherapy as a first-line treatment option in metastatic NSCLC.

Patient-Reported Outcomes With Belantamab Mafodotin Treatment in Patients With Triple-Class Refractory Multiple Myeloma.

In the randomized phase II DREAMM-2 study, single-agent belantamab mafodotin demonstrated deep and durable responses and a manageable safety profile in triple-class refractory relapsed/refractory multiple myeloma (RRMM). We present patient-reported outcomes (PROs) from this study for patients treated with the approved dose of belantamab mafodotin (2.5 mg/kg q3w). Disease and treatment-related symptoms, health-related quality of life (HRQOL), functioning, and patient-reported ocular changes were assessed using questionnaires (European Organisation for Research and Treatment of Cancer Quality of Life questionnaires EORTC-QLQ-C30 and EORTC-QLQ-MY20, Ocular Surface Disease Index [OSDI], and the National Eye Institute Visual Functioning Questionnaire 25 [NEI VFQ-25]) at baseline, during treatment (every 3 or 6 weeks), and at the end of treatment (EOT). Eye examinations were conducted at baseline, prior to each treatment cycle, and at EOT. Patients reported ocular symptoms in the OSDI and NEI VFQ-25 questionnaires, with the median time to worst severity of 45 to 64 days depending on symptoms considered. Some limitations in driving and reading were reported. Ocular symptoms were improved and median time to recovery was 23.5 to 44.0 days. EORTC-QLQ-C30 data suggest core MM symptoms (including fatigue and pain), overall HRQOL, and patient functioning were maintained while patients continued belantamab mafodotin treatment, even if meaningful worsening of vision-related symptoms occurred. These PRO results, together with the clinical efficacy of belantamab mafodotin, support its use in patients with RRMM and further evaluation of its use at earlier lines of therapy.

Patient-reported outcomes of furmonertinib versus gefitinib in patients with locally advanced or metastatic, EGFR mutation-positive non-small cell lung cancer (FURLONG): A multicenter, double-blind, randomized phase 3 study.

9081 Background: Furmonertinib showed superior efficacy compared with gefitinib as first-line therapy in Chinese patients with EGFR mutation-positive non-small cell lung cancer (NSCLC), along with an acceptable safety profile without new signals in the FURLONG study. Here we present patient-reported outcomes (PROs). Methods: FURLONG is a multicenter, double-blind, double dummy, randomized phase 3 study done in 55 hospitals across mainland China. Eligible patients were randomly assigned (1:1) to receive either furmonertinib or gefitinib. The primary endpoint of IRC-assessed progression-free survival has been reported previously. PROs as prespecified secondary outcomes by the European Organization for Research and Treatment of Cancer (EORTC) Quality-of-Life Questionnaire Core 30 (QLQ-C30) and Quality-of-Life Questionnaire Lung Cancer 13 (QLQ-LC13), were analyzed in randomly assigned patients who had received at least one dose of study drug and completed at least one PRO assessment. Changes from baseline to randomized treatment discontinuation were analyzed with mixed model for repeated measures and time-to-event analyses. This study is ongoing for survival follow-up. Results: 357 patients received at least one dose of study drug, all of them completed at least one PRO assessment. Questionnaire completion rates were ≥95% at most time points. Baseline mean scores were similar in the furmonertinib and gefitinib groups. From baseline to randomized treatment discontinuation, statistically significant differences favoring furmonertinib for physical function, nausea/vomiting, appetite loss, diarrhea, alopecia, and other pain (any pain other than chest, arm, or shoulder pain) were observed. Time to deterioration in physical function, cognitive function, nausea/vomiting, appetite loss, diarrhea, cough, dyspnea, dysphagia, and alopecia were significantly longer with furmonertinib versus gefitinib (Table). Conclusions: Furmonertinib demonstrated favorable PROs as measured by the EORTC QLQ-C30 and QLQ-L13 versus gefitinib. These data presented the positive benefit risk profile of first-line furmonertinib and further support this novel third generation EGFR-TKI as a new standard of first line treatment in EGFR mutation-positive NSCLC. Clinical trial information: NCT03787992 . [Table: see text]

Patient-reported outcomes for the Intergroup Sentinel Mamma study (INSEMA): A randomised trial with persistent impact of axillary surgery on arm and breast symptoms in patients with early breast cancer.

In clinically node-negative breast cancer patients, the INSEMA trial (NCT02466737) assessed the non-inferiority of avoiding sentinel lymph node biopsy (SLNB) or axillary lymph node dissection (ALND). Here we present patient-reported outcomes (PROs) as a secondary endpoint. PROs were assessed for patients with no axillary surgery, SLNB alone, and ALND. Quality of life (QoL) questionnaire EORTC QLQ-C30 and its breast cancer module (BR23) were used at baseline (pre-surgery) and 1, 3, 6, 12, and 18 months after surgery. The QoL scores were compared using repeated measures mixed models based on the safety set. Between 2015 and 2019, 5502 patients were recruited for the first randomization, and 5154 were included inthe intent-to-treat set (4124 SLNB versus 1030 no SLNB). In the case of one to three macrometastases after SLNB, 485 patients underwent second randomization (242 SLNB alone versus 243 ALND). Questionnaire completion response remained high throughout the trial: over 70% at all time points for the first randomization. There were significant differences for the BRBS (breast symptoms) and BRAS (arm symptoms) scores favoring the no SLNB group in all post-baseline assessments. Patients in the SLNB group showed significantly and clinically relevant higher scores for BRAS (differences in mean values ≥5.0 points at all times), including pain, arm swelling, and impaired mobility in all postoperative visits, with the highest difference at one month after surgery. Scoring of theQLQ-C30 questionnaire revealed no relevant differences between the treatment groups, although some comparisons were statistically significant. This is one of the first randomized trials investigating the omission of SLNB in clinically node-negative patients and the first to report comprehensive QoL data. Patients with no SLNB benefitted regarding arm symptoms/functioning, while no relevant differences in other scales were seen. Supported by German Cancer Aid (Deutsche Krebshilfe, Bonn, Germany), Grant No. 110580 and Grant No. 70110580 to University Medicine Rostock.

First-line nivolumab plus ipilimumab versus chemotherapy for the treatment of unresectable malignant pleural mesothelioma: patient-reported outcomes in CheckMate 743

ObjectiveIn CheckMate 743 (NCT02899299), nivolumab + ipilimumab significantly prolonged overall survival in patients with unresectable malignant pleural mesothelioma (MPM). We present patient-reported outcomes (PROs). Materials and MethodsPatients (N = 605) were randomized to nivolumab + ipilimumab or chemotherapy. Changes in disease-related symptom burden and health-related quality of life (HRQoL) were evaluated descriptively using the Lung Cancer Symptom Scale (LCSS)-Mesothelioma (Meso) average symptom burden index (ASBI), LCSS-Meso 3-item global index (3-IGI), 3-level EuroQol 5-dimensional (EQ-5D-3L) visual analog score (VAS), and EQ-5D-3L utility index. PROs were assessed at baseline and every 2 (nivolumab + ipilimumab) or 3 weeks (chemotherapy) through 12 weeks, every 6 weeks through 12 months, every 12 weeks thereafter, and at specified follow-ups. Mixed-effect model repeated measures (MMRM) and time to deterioration analyses were conducted. ResultsCompletion rates were generally >80%. LCSS-Meso ASBI mean changes from baseline trended to improve over time with nivolumab + ipilimumab and deteriorate with chemotherapy, but did not meet clinically important difference thresholds [±10 score change]. EQ-5D-3L VAS mean scores improved over time with nivolumab + ipilimumab; by week 60, patients had scores consistent with United Kingdom normal population values. MMRM analyses favored nivolumab + ipilimumab for all individual symptoms except cough. Nivolumab + ipilimumab delayed time to definitive deterioration in HRQoL (hazard ratio 0.52 [95% confidence interval 0.36–0.74]) and showed a trend in symptom delay versus chemotherapy. ConclusionsNivolumab + ipilimumab decreased the risk of deterioration in disease-related symptoms and HRQoL versus chemotherapy and maintained QoL in patients with unresectable MPM.

Health-related quality of life in patients with relapsed/refractory multiple myeloma treated with pomalidomide and dexamethasone ± subcutaneous daratumumab: Patient-reported outcomes from the APOLLO trial.

In the phase 3 APOLLO trial, daratumumab in combination with pomalidomide and dexamethasone (D-Pd) significantly reduced the rate of disease progression or death by 37% relative to Pd alone in patients with relapsed/refractory multiple myeloma (RRMM) who had received ≥1 prior line of therapy including lenalidomide and a proteasome inhibitor. Here, we present patient-reported outcomes (PROs) from APOLLO. Median treatment duration was 11.5months with D-Pd and 6.6months with Pd. PRO compliance rates were high and similar in both groups. No changes from baseline were observed for EORTC QLQ-C30 global health status scores in either group, while physical and emotional functioning, disease symptoms, and adverse effects of treatment remained at baseline levels with D-Pd but worsened with Pd. Reductions (p < 0.05) in pain and fatigue were seen at several time points with D-Pd versus Pd. Overall, these results suggest patients' health-related quality of life remained stable when daratumumab was added to Pd, with several results favoring D-Pd versus Pd. These findings complement the significant clinical improvements observed with D-Pd and support its use in patients with RRMM.

Development of ‘Core Outcome Sets’ for meningioma in clinical studies: The COSMIC project

Abstract Aims To date, meningioma clinical trial activity has been limited, but a number of high-quality studies are underway, with more in development. There is heterogeneity in the outcomes reported in meningioma clinical trials. The COSMIC Project will develop two ‘Core Outcome Sets’ (COS) through comprehensive, transparent, consensus methodology; to ensure outcomes relevant to key stakeholders are reported within and across future meningioma clinical studies. The first will be for use in clinical effectiveness trials (COSMIC: Intervention), the second will be for use in clinical studies of incidental meningioma (COSMIC: Observation). Method Three systematic literature reviews will be performed to generate a long-list of outcomes potentially relevant to meningioma patients, healthcare professionals, researchers, and other stakeholder groups. The first systematic review will present the outcomes reported in published and ongoing meningioma clinical effectiveness trials. The second systematic review will present patient-reported outcomes (PRO) from the measurement tools utilised in meningioma PRO studies. The third systematic review will present the outcomes reported in published and ongoing clinical studies of untreated meningioma. Outcomes will be deduplicated, unique outcomes categorised according to the taxonomy presented by COMET, and the lists combined. The long-list of outcomes will be prioritised through two, 2-round, modified eDelphi surveys including meningioma patients, healthcare professionals, researchers, and other stakeholder groups. Undecided outcomes from both eDelphi surveys will be ratified at two, one-day consensus meeting, with representation from all key stakeholder groups. Results We have formed a study advisory group with international representation from key organisations. The project already has confirmed support from the International Consortium on Meningioma (ICOM), the European Association of Neuro-Oncology (EANO), the Response Assessment in Neuro-Oncology Patient-Reported Outcome group (RANO-PRO), the Society for Neuro-Oncology (SNO), British Neuro-Oncology Society (BNOS), Society of British Neurological Surgeons (SBNS), The Brain Tumour Charity (TBTC), and Brainstrust. Conclusion Standardising minimum outcome reporting in meningioma clinical effectiveness trials and meningioma clinical studies, through the development of these two COS will ensure outcomes reported are relevant to key stakeholder groups, including patients, whilst reducing research waste for a disease with increasing clinical trial activity. We seek to raise awareness of this project and invite participation from a wide range of stakeholders to ensure that the final COS reflects the opinion of the neuro-oncology community. Registration will take place via the study website www.thecosmicproject.org between June-August 2021.

Health-related quality of life in patients with newly diagnosed multiple myeloma ineligible for stem cell transplantation: results from the randomized phase III ALCYONE trial

BackgroundIn the phase III ALCYONE trial, daratumumab plus bortezomib/melphalan/prednisone (D-VMP) significantly improved overall response rate and progression-free status compared with VMP alone in transplant-ineligible patients with newly diagnosed multiple myeloma (NDMM). Here, we present patient-reported outcomes (PROs) from ALCYONE.MethodsThe European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30-item (EORTC QLQ-C30) and EuroQol 5-dimensional descriptive system (EQ-5D-5L) questionnaire were administered at baseline, every 3 months (year 1) and every 6 months (until progression). Treatment effects were assessed using a repeated-measures, mixed-effects model.ResultsCompliance with PRO assessments was comparable at baseline (> 90%) and throughout study (> 76%) for both treatment groups. Improvements from baseline were observed in both groups for EORTC QLQ-C30 Global Health Status (GHS), most functional scales, symptom scales and EQ-5D-5L visual analog scale (VAS). Between-group differences were significant for GHS (p = 0.0240) and VAS (p = 0.0160) at month 3. Improvements in pain were clinically meaningful in both groups at all assessment time points. Cognitive function declined in both groups, but the magnitude of the decline was not clinically meaningful.ConclusionsPatients with transplant-ineligible NDMM demonstrated early and continuous improvements in health-related quality of life, including improvements in functioning and symptoms, following treatment with D-VMP or VMP.Trial registrationClinicalTrials.gov identifier NCT02195479, registered September 21, 2014

Patient-Reported Outcomes from a Randomized, Active-Controlled, Open-Label, Phase 3 Trial of Burosumab Versus Conventional Therapy in Children with X-Linked Hypophosphatemia

Changing to burosumab, a monoclonal antibody targeting fibroblast growth factor 23, significantly improved phosphorus homeostasis, rickets, lower-extremity deformities, mobility, and growth versus continuing oral phosphate and active vitamin D (conventional therapy) in a randomized, open-label, phase 3 trial involving children aged 1–12 years with X-linked hypophosphatemia. Patients were randomized (1:1) to subcutaneous burosumab or to continue conventional therapy. We present patient-reported outcomes (PROs) from this trial for children aged ≥ 5 years at screening (n = 35), using a Patient-Reported Outcomes Measurement Information System (PROMIS) questionnaire and SF-10 Health Survey for Children. PROMIS pain interference, physical function mobility, and fatigue scores improved from baseline with burosumab at weeks 40 and 64, but changed little with continued conventional therapy. Pain interference scores differed significantly between groups at week 40 (− 5.02, 95% CI − 9.29 to − 0.75; p = 0.0212) but not at week 64. Between-group differences were not significant at either week for physical function mobility or fatigue. Reductions in PROMIS pain interference and fatigue scores from baseline were clinically meaningful with burosumab at weeks 40 and 64 but not with conventional therapy. SF-10 physical health scores (PHS-10) improved significantly with burosumab at week 40 (least-squares mean [standard error] + 5.98 [1.79]; p = 0.0008) and week 64 (+ 5.93 [1.88]; p = 0.0016) but not with conventional therapy (between-treatment differences were nonsignificant). In conclusion, changing to burosumab improved PRO measures, with statistically significant differences in PROMIS pain interference at week 40 versus continuing with conventional therapy and in PHS-10 at weeks 40 and 64 versus baseline.Trial registration: ClinicalTrials.gov NCT02915705

Health-Related Quality of Life in Patients with AL Amyloidosis Treated with Daratumumab, Bortezomib, Cyclophosphamide, and Dexamethasone: Results from the Phase 3 Andromeda Study

Health-Related Quality of Life in Patients with AL Amyloidosis Treated with Daratumumab, Bortezomib, Cyclophosphamide, and Dexamethasone: Results from the Phase 3 Andromeda Study

Patient-reported outcomes from the randomized phase III ALEX study of alectinib versus crizotinib in patients with ALK-positive non-small-cell lung cancer

ObjectivesAlectinib demonstrated superior efficacy and a safety profile that compared favorably with crizotinib in treatment-naïve ALK+ non-small-cell lung cancer (NSCLC) in the phase III ALEX study. We present patient-reported outcomes (PROs) from ALEX to assess disease burden, treatment-related symptom tolerability, and health-related quality of life (HRQoL) with alectinib versus crizotinib. Materials and MethodsPatients were randomized to receive alectinib 600 mg or crizotinib 250 mg twice daily until disease progression, death, or withdrawal. Pre-specified PRO endpoints were: mean change from baseline in symptoms, HRQoL, and functioning; and time to deterioration (TTD) in cough, dyspnea, chest pain, arm/shoulder pain, fatigue, and a composite of three symptoms (cough, dyspnea, chest pain). PRO data were collected using EORTC QLQ-C30 and LC13 questionnaires. Raw scores were standardized to a 0–100-point range, with a ≥10-point score change defined as clinically meaningful. TTD was defined as the time from randomization until confirmed clinically meaningful deterioration (i.e., a ≥10-point score change from baseline). ResultsBaseline completion rates and characteristics were balanced in the PRO-evaluable population (alectinib n = 100, 66%; crizotinib n = 97, 64%). On average, alectinib-treated patients reported clinically meaningful improvements in lung cancer symptoms for longer than crizotinib-treated patients. Between-treatment differences in lung cancer symptoms tended to favor alectinib from 11.1 months (45 weeks) onwards, around the time of median PFS with crizotinib (11.1 months). TTD in lung cancer symptoms was similar between treatment arms, despite longer duration of symptom improvement with alectinib; composite symptom endpoint (hazard ratio 1.10 [95% confidence interval: 0.72–1.68]). Duration of clinically meaningful improvement in HRQoL was longer with alectinib versus crizotinib (Week 88 vs. Week 68, respectively). Better patient-reported tolerability was observed with alectinib versus crizotinib on common treatment-related symptoms. ConclusionPRO data support the superior efficacy and tolerability of alectinib relative to crizotinib demonstrated in the ALEX study.

1767P - Patient-reported outcome data during real-world use of NEPA for prevention of chemotherapy-induced nausea and vomiting in high-risk platin-receiving patients: A prospective multicenter trial

BackgroundNEPA is the first and only fixed oral combination of a long-acting NK1receptor antagonist (RA), netupitant, and a pharmacologically distinct 5-HT3RA, palonosetron. NEPA is approved for prevention of chemotherapy (ctx)-induced nausea and vomiting (CINV) in patients (pts) receiving cisplatin-based highly emetogenic ctx (HEC) or moderately emetogenic ctx (MEC). Here we present patient-reported outcomes (PRO) of patients (pts) receiving NEPA as an antiemetic prophylaxis during platin-based chemotherapy in a prospective non-interventional study. This group of patients presents a population at high risk for CINV. MethodsThe ongoing prospective AkyPRO study was designed to evaluate quality of life (QoL) in 2500 pts receiving NEPA for CINV prevention in single or two-day MEC or HEC over 3 cycles. The primary endpoint QoL was recorded by FLIE questionnaires. Secondary endpoints were efficacy, reported by pts and physicians on a 4-point scale; antiemetic rescue medication, and safety. ResultsIn this interims analysis 595 pts receiving platin-based ctx were evaluated. PRO data show that ctx-induced vomiting can be controlled very efficiently in the majority of platin-receiving pts. Between 80-90% of pts reported no impact on daily life due to vomiting and between 84-90% of pts had a complete response (no vomiting, no rescue medication) in the 3 analysed cycles, independently of which type of platin-ctx they received. Furthermore, the majority of pts (74%, 81% and 68%, respectively) reported no or mild nausea in the cisplatin-, carboplatin- and oxaliplatin-subgroup in cycle 1. However, even mild nausea seems to have a strong impact on daily life with 40% of pts receiving cisplatin- or carboplatin-based chemotherapy and 46% of oxaliplatin-receiving pts reporting an impact on daily life due to nausea in cycle 1. ConclusionsNEPA was proven highly effective in controlling both vomiting and nausea over three cycles of ctx independent of the type of platin administered. Despite the reported MEC classification of oxaliplatin in contrast to the HEC classification of cis- and carboplatin, nausea was more difficult to control in oxaliplatin-receiving patients. Legal entity responsible for the studyRiemser Pharma. FundingRiemser Pharma. DisclosureM. Karthaus: Advisory / Consultancy: Helsinn; Advisory / Consultancy: Riemser. J. Schilling: Advisory / Consultancy: Riemser. All other authors have declared no conflicts of interest.