Kim Cocks

Adelphi Values · Patient-Centred Outcomes

11068 Background: LIBRETTO-431 (NCT04194944), a randomized phase 3 trial, compared first-line selpercatinib to platinum-based chemotherapy +/- pembrolizumab (pembro). Selpercatinib, a highly selective and potent CNS penetrant RET inhibitor, is approved for the treatment of advanced RET fusion+ NSCLC. This analysis reported NSCLC symptoms and HRQoL...

Background PROs are valuable tools in clinical care to capture patients’ perspectives of their health, symptoms and quality of life. However the COVID-19 pandemic has had profound impacts on all aspects of life, in particular healthcare and research. This study explores the views of UK and Irish health professionals, third sector and pharmaceutical...

Hodgkin lymphoma (HL) has become one of the most curable cancers. Therefore, rigorous assessment of health-related quality of life (HRQoL) and symptom burden of these patients is essential to support informed clinical decisions. This paper reports the final results of an international study by the European Organisation for Research and Treatment of...

Objectives: Patient-reported outcome (PRO) data are critical in understanding treatments from the patient perspective in cancer clinical trials. The potential benefits and methodological approaches to the collection of PRO data after treatment discontinuation (eg, because of progressive disease or unacceptable drug toxicity) are less clear. The pu...

Patient-reported outcomes (PROs), such as symptoms, functioning, and other health-related quality-of-life concepts are gaining a more prominent role in the benefit-risk assessment of cancer therapies. However, varying ways of analysing, presenting, and interpreting PRO data could lead to erroneous and inconsistent decisions on the part of stakehold...

Background Health‐related quality of life (HRQOL) is a critical aspect to consider when making treatment decisions for patients with non‐Hodgkin‐lymphoma (NHL). This international study by the European Organisation for Research and Treatment of Cancer (EORTC) tested the psychometric properties of two newly developed measures for patients with high‐...

The European Organisation for Research and Treatment of Cancer Quality of Life Multiple Myeloma Questionnaire (EORTC QLQ-MY20) was developed in 1996 to assess health-related quality of life (HRQoL) in patients with multiple myeloma. Since its development new therapies have prolonged survival in patients with myeloma and new combination agents are l...

Background: Cytomegalovirus (CMV), a common post-transplant infection, is associated with increased healthcare resource utilization. In the Phase 3 SOLSTICE trial, maribavir was superior to investigator-assigned therapy (IAT; valganciclovir/ganciclovir, foscarnet, and cidofovir) for CMV viremia clearance at Week 8 in transplant recipients with con...

Introduction: Early guidelines for minimally important differences (MIDs) for the EORTC QLQ-C30 proposed ≥10 points change as clinically meaningful for all scales. Increasing evidence that MIDs can vary by scale, direction of change, cancer type and estimation method has raised doubt about a single global standard. This paper identifies MID patter...

Aim The European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (QLQ-C30) is among the most widely used patient-reported outcome measures in cancer research and practice. It was developed prior to guidance that content should be established directly from patients to confirm it measures concepts of interest a...

Purpose Treatment benefit as assessed using clinical outcome assessments (COAs), is a key endpoint in many clinical trials at both the individual and group level. Anchor-based methods can aid interpretation of COA change scores beyond statistical significance, and help derive a meaningful change threshold (MCT). However, evidence-based guidance on...

Aim The European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (QLQ-C30) is among the most widely-used patient-reported outcome measures (PROMs) in cancer research and practice. It was developed prior to guidance that content should be established directly from patients to confirm it measures concepts of in...

Background: The European Organisation for Research and Treatment of Cancer (EORTC) health-related quality of life (HRQoL) questionnaire for anal cancer (QLQ-ANL27) supplements the EORTC cancer generic measure (QLQ-C30) to measure concerns specific to people with anal cancer treated with chemoradiotherapy (CRT). This study tests the psychometric pro...

Background Incisional hernias cause morbidity and may require further surgery. HART (Hughes Abdominal Repair Trial) assessed the effect of an alternative suture method on the incidence of incisional hernia following colorectal cancer surgery. Methods A pragmatic multicentre single-blind RCT allocated patients undergoing midline incision for colore...

Purpose The recommended method for establishing a meaningful threshold for individual changes in patient-reported outcome (PRO) scores over time uses an anchor-based method. The patients assess their perceived level of change and this is used to define a threshold on the PRO score which may be considered meaningful to the patient. In practice, such...

Purpose With higher efficacy of cancer therapies, the numbers and types of side effects experienced by patients have also increased, evidencing a need for brief assessments of side effect bother. The Functional Assessment of Cancer Therapy-General (FACT-G) includes the item “I am bothered by side effects of treatment” (GP5). This study aimed to con...

Selecting the most appropriate chronic lymphocytic leukaemia (CLL) treatment is challenging. Patient‐reported health‐related quality of life (HRQoL) is therefore a critical aspect to consider. This international study by the European Organization for Research and Treatment of Cancer (EORTC) tested the psychometric properties of a newly developed me...

Objectives A minimally important difference (MID) is the smallest difference in quality of life (QoL) perceived as relevant by patients or clinicians. MIDs aid interpretation of QOL data in research and clinical practice. We aimed to determine MIDs for the EORTC QLQ-C30 for patients with lung cancer or malignant pleural mesothelioma. Materials and...

Introduction Lors de l’étude de phase II CodeBreaK 100, le traitement par sotorasib obtient un TR de 37,1 % sur une durée médiane de 10,0 mois, une PFS médiane de 6,8 mois et un profil de tolérance acceptable chez des patients avec un CPNPC muté KRAS pG12 C prétraité. Les patients ont un nombre médian de 2 lignes de traitement antérieures. Cette an...

Objectives There is growing interest in condition-specific preference measures, including the European Organisation for Research and Treatment of Cancer Quality of Life Utility Measure-Core 10 Dimensions (QLU-C10D). This research assessed the implications of using utility indices on the basis of the EQ-5D-3L, a mapping of EQ-5D-3L to the EQ-5D-5L,...

Background The aim of the study was to estimate the minimally important difference (MID) for interpreting group-level change over time, both within a group and between groups, for the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) scores in patients with prostate cancer. Methods We...

Background The EORTC QLQ-MY20 is a widely used myeloma-specific patient-reported outcome measure originally developed in 1999. It consists of 20 items covering Disease Symptoms (DS), Side Effects (SE), Future Perspectives (FP) and Body Image (BI). Novel treatments and multiple lines of therapy mean the treatment for myeloma patients and life expect...

Objective: To evaluate psychometric performance of the NCCN-FACT Ovarian Cancer Symptom Index-18 (NFOSI-18) in advanced ovarian cancer. Methods: Cross-sectional, observational data from patients receiving treatment for ovarian cancer. Other measures included European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire co...

9057 Background: In the registrational phase 2 CodeBreaK 100 trial, sotorasib demonstrated a response rate of 37.1% with median duration of 10.0 months, a median progression-free survival of 6.8 months, and a tolerable safety profile in patients with pretreated KRAS p.G12C mutated NSCLC. Patients received a median of 2 prior lines of therapy. Here,...

Rationale Study 311 (E2007-G000-311; NCT02849626) was a Phase 3, multicenter, open-label single-arm study of adjunctive perampanel oral suspension in pediatric patients (aged 4 to <12 years) with partial-onset seizures (POS) (with/without secondarily generalized tonic-clonic seizures [SGTCS]) or primary generalized tonic-clonic seizures (PGTCS). He...

Purpose: Treatment benefit as assessed using clinical outcome assessments (COAs), is a key endpoint in many clinical trials at both the individual and group level. Anchor-based methods can aid interpretation of COA change scores beyond statistical significance, and help derive a meaningful change threshold (MCT). However, evidence-based guidance on...

Background: Minimally important differences (MIDs) allow interpretation of the clinical relevance of health-related quality of life (HRQOL) results. This study aimed to estimate MIDs for all EORTC QLQ-C30 scales for interpreting group-level results in brain tumor patients. Methods: Clinical and HRQOL data from three glioma trials were used. Clin...

Background Published in 2019, a new addendum to the ICH E9 guideline presents the estimand framework as a systematic approach to ensure alignment among clinical trial objectives, trial execution/conduct, statistical analyses, and interpretation of results. The use of the estimand framework for describing clinical trial objectives has yet to be exte...

Introduction: Minimal important differences (MIDs) are useful for interpreting changes or differences in health-related quality of life scores in terms of clinical importance. There are currently no MID guidelines for the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire core 30 (EORTC QLQ-C30) specific to ov...

Background The European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ‐C30) assesses health related quality of life of patients in cancer trials. There are currently no minimally important difference (MID) guidelines for the EORTC QLQ‐C30 for colorectal cancer (CRC). Methods The data were obtaine...

Health‐related quality of life (HRQoL) in patients with chronic lymphocytic leukemia (CLL) is important in guiding treatment decisions. However, the impact of CLL treatment initiation on HRQoL is unclear. We assessed HRQoL using the FACT‐Leu and EQ‐5D‐3L questionnaires in the Connect® CLL Registry, a large, US‐based, multicenter, prospective observ...

Background We aimed to estimate minimally important difference (MID) for interpreting group‐level change over time for European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire core 30 (EORTC QLQ‐C30) scores in head and neck cancer. Methods Data were derived retrospectively from two published EORTC trials. Clinical a...

Patient-reported outcomes (PROs), such as symptoms, function, and other health-related quality-of-life aspects, are increasingly evaluated in cancer randomised controlled trials (RCTs) to provide information about treatment risks, benefits, and tolerability. However, expert opinion and critical review of the literature showed no consensus on optima...

483 Background: SOR is approved as 1L therapy for pts with aHCC, but there is still an unmet need to help improve or maintain HRQoL. This phase 3 study compared HRQoL of NIVO vs SOR as 1L therapy in pts with aHCC as an exploratory endpoint. Methods: FACT-Hep was administered cycle 1, day 1 and every other cycle. The effect of NIVO vs SOR on HRQoL u...

Background: This study aims to evaluate the feasibility and acceptability of the PhunkyFoods Programme, a primary school-based intervention to promote healthy nutrition and physical activity knowledge and behaviours to assess outcomes to inform a phase 3 trial. Methods: The cluster randomised feasibility trial recruited eight primary schools fro...

We compared patient-reported outcomes (PROs) with once-weekly carfilzomib 70 mg/m2 (Kd70 mg/m2) vs. twice-weekly carfilzomib 27 mg/m2 (Kd27 mg/m2) plus dexamethasone in relapsed or refractory multiple myeloma (RRMM). Patient-reported convenience/satisfaction collected at Cycle 2, Day 1 was compared between groups using logistic regression. European...

Introduction: Novel triplet therapies (tx) for relapsed/refractory multiple myeloma (RRMM) have improved outcomes and extended survival. However, the use of multi-agent tx over an extended period increases the tx-related symptom burden and impacts health-related quality of life (HRQoL). Therefore, highly effective tx that preserve HRQoL are needed....

Introduction: The phase 3 international, randomized, double-blind ROBUST clinical trial (NCT02285062) compared progression-free survival between patients with previously untreated ABC-type DLBCL treated with placebo plus rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (placebo-R-CHOP) versus lenalidomide + R-CHOP (R2-CHOP) for...

Objectives Thresholds for the minimally important difference (MID) or responder definition (RD) in health‐related quality‐of‐life (HRQoL) scores are required to interpret the impact of an intervention or change in the trajectory of the condition which is meaningful to patients. This study aimed to establish MID and RD for the European Organisation...

Aims: To evaluate the accuracy of a continuous intracoronary (IC) adenosine infusion, administered through a novel HYPEREM™IC over-the-wire microcatheter, to measure fractional flow reserve (FFR). Methods and Results: The HYPEREMIC trial was a randomised, non-inferiority, crossover study in which patients with intermediate coronary lesions were en...

Background We aimed to estimate the minimally important difference (MID) for interpreting group-level change over time, both within a group and between groups, for European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire core 30 (EORTC QLQ-C30) scores in patients with advanced breast cancer. Patients and Methods Data...

Objectives In the CheckMate 141 trial (NCT02105636), nivolumab demonstrated survival, health-related quality of life, and healthcare resource utilization benefits vs single-agent therapy of investigator’s choice (IC) (methotrexate, docetaxel or cetuximab) in patients with platinum-refractory recurrent/metastatic squamous cell carcinoma of the head...

Background: Patients (pts) with classical Hodgkin lymphoma (cHL) frequently experience reduced health-related quality of life (HRQoL) (Oerlemans et al, Ann Hematol 2011). Nivolumab, a fully human IgG4 anti-programmed death-1 (PD-1) immune checkpoint inhibitor monoclonal antibody, demonstrated efficacy and clinically meaningful improvement in pt-rep...

We examined effects of carfilzomib-dexamethasone (Kd56) versus bortezomib-dexamethasone (Vd) on health-related quality of life (HR-QoL) in relapsed/refractory multiple myeloma (MM) patients from the ENDEAVOR study. HR-QoL was assessed by the European Organisation for Research and Treatment of Cancer QoL Questionnaire (QLQ-C30), MM-specific module (...

Introduction In addition to efficacy and safety, novel regimens for the treatment of multiple myeloma (MM) should be assessed in terms of disease-related symptoms, the adverse events patient's experience, and their effect on health-related quality of life (HRQOL). Patient-reported outcome (PRO) measures capture these experiences from the patient's...

Introduction: Novel therapies for relapsed/refractory multiple myeloma (RRMM) have demonstrated durable responses that prolong survival, thus assessing health-related quality of life (HRQoL) is becoming increasingly important in patient (pt) care. Long-term RRMM treatment may lead to a higher cumulative symptom burden and negatively affect HRQoL. I...

OBJECTIVES: Health-related quality of life (HRQOL) is increasingly incorporated into cancer clinical trials as an important endpoint. The notion of minimally important difference (MID) is relevant for interpreting differences and changes in HRQOL scores in terms of clinical meaningfulness. MIDs are useful for assessing the benefit of an interventio...

We compared patient-reported outcomes (PROs) with once-weekly carfilzomib 70 mg/m2 (Kd70 mg/m2) vs. twice-weekly carfilzomib 27 mg/m2 (Kd27 mg/m2) plus dexamethasone in relapsed or refractory multiple myeloma (RRMM). Patientreported convenience/satisfaction collected at Cycle 2, Day 1 was compared between groups using logistic regression. European...

Although patient-reported outcomes (PROs), such as health-related quality of life, are important endpoints in randomised controlled trials (RCTs), there is little consensus about the analysis, interpretation, and reporting of these data. We did a systematic review to assess the variability, quality, and standards of PRO data analyses in advanced br...

Background: There is currently a lack of consensus on how health-related quality of life and other patient-reported outcome measures in cancer randomized clinical trials are analyzed and interpreted. This makes it difficult to compare results across randomized controlled trials (RCTs) synthesize scientific research, and use that evidence to inform...

Introduction Up to 30% of hearing aids fitted to new adult clients are reported to be of low benefit and used intermittently or not at all. Evidence suggests that additional interventions paired with service-delivery redesign may help improve hearing aid use and benefit. The range of interventions available is limited. In particular, the efficacy o...

Introduction: Health-related quality of life (HRQOL) is increasingly recognised as an important end-point in cancer clinical trials. The concept of minimally important difference (MID) enables interpreting differences and changes in HRQOL scores in terms of clinical meaningfulness. We aimed to estimate MIDs for interpreting group-level change of E...

The majority of cancers are diagnosed using excised biopsy specimens. These are graded, using a gold-standard histopathology protocol based on haemotoxylin and eosin ('H + E') chemical staining. However the grading is done by eye and if the same biopsy is graded by different practitioners, they typically only agree ~70% of the time. The resulting o...

Purpose: This paper describes the international, cross-cultural development of four disease-specific EORTC QoL questionnaires, to supplement the EORTC QLQ-C30, for patients with Hodgkin lymphoma (HL), high- or low-grade non-Hodgkin lymphoma (HG/LG-NHL), and CLL. Methods: Questionnaire development was conducted according to guidelines from the EO...

Introduction As patient assessment of health-related quality of life (HRQOL) in cancer clinical trials has increased over the years, so has the need to attach meaningful interpretations to differences in HRQOL scores between groups and changes within groups. Determining what represents a minimally important difference (MID) in HRQOL scores is usefu...

Introduction: Assessing patients’ health-related quality of life (HRQOL) in cancer clinical trials is a well-established practice. Understanding the amount of change in HRQOL scores that is clinically relevant is crucial for interpretation. The concept of “minimal important difference (MID)” helps to attach meaningful interpretations to differences...