Relationship between corneal exam findings, best-corrected visual acuity (BCVA), and ocular symptoms in patients with relapsed or refractory multiple myeloma (RRMM) receiving belantamab mafodotin (belamaf).

8033 Background: Belantamab mafodotin (GSK2857916; belamaf; BLENREP) is a B-cell maturation antigen (BCMA)-targeting antibody–drug conjugate approved in the US and EU as a monotherapy for the treatment of adult patients with RRMM. Ocular events (OEs) during the pivotal DREAMM-2 trial (NCT03525678) included corneal exam findings (punctate keratopathy and microcyst-like epithelial changes), BCVA changes, and ocular symptoms. Dose reductions or delays based on corneal exam findings and BCVA were used to manage OEs. Here we performed a post hoc investigation of relationships between corneal exam findings, BCVA changes, and patient-reported ocular symptoms to explore if BCVA changes and symptoms could guide dosing, rather than corneal exams. Methods: Eye evaluations (including a corneal exam and BCVA assessment of Snellen visual acuity) were performed on all patients receiving single-agent belamaf (2.5 mg/kg) by ophthalmologists at baseline and prior to each belamaf dose. Changes in the corneal epithelium (Ker) and BCVA were both assessed as per protocol-defined criteria and assessment of grade (Gr) was based on the worse eye. BCVA grading was relative to baseline. Patient-reported ocular symptoms were reported as per the Common Terminology Criteria for Adverse Events. Results: In 12.5% of eye evaluations Gr 3–4 Ker was associated with minimal or no (Gr ≤1) BCVA changes. When patient-reported ocular symptoms were also considered, only 7.5% of evaluations found Gr 3–4 Ker with Gr ≤1 BCVA changes or ocular symptoms. Mild or no (Gr ≤2) Ker was associated with Gr ≤1 BCVA changes in 59.5% of evaluations, or in 38.8% of evaluations with no ocular symptoms reported. Overall, Gr 3–4 Ker were found in 24.9% of evaluations; by contrast, patients had Gr 2–4 BCVA changes or ocular symptoms in 53.7% of evaluations. Association of corneal epithelium changes (Ker) with BCVA changes and ocular symptoms. Conclusions: These findings highlight that BCVA changes and ocular symptoms should be further investigated to determine if they can be used as alternatives (eg, frequency of eye examinations based on symptoms) for the management of belamaf dosing to potentially reduce the burden on patients and healthcare professionals. Clinical trial information: NCT03525678. [Table: see text]

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Exploring Alternative Dosing Regimens of Single-Agent Belantamab Mafodotin on Safety and Efficacy in Patients with Relapsed or Refractory Multiple Myeloma: DREAMM-14

Blood ◽

10.1182/blood-2021-152224 ◽

2021 ◽

Vol 138 (Supplement 1) ◽

pp. 1645-1645

Author(s):

Malin Hultcrantz ◽

David Kleinman ◽

Pavandeep Ghataorhe ◽

Astrid McKeown ◽

Wei He ◽

...

Keyword(s):

Visual Acuity ◽

Research Funding ◽

Progressive Disease ◽

Single Agent ◽

Publicly Traded ◽

Ocular Symptoms ◽

Patient Reported ◽

Dosing Regimens ◽

Dose Modifications

Abstract Introduction: Belantamab mafodotin is a first-in-class, monomethyl auristatin F (MMAF)-containing, B-cell maturation antigen (BCMA)-directed antibody-drug conjugate (ADC) that is approved in the United States and European Union for adult patients with relapsed/refractory multiple myeloma (RRMM). In the pivotal Phase II DREAMM-2 study, single-agent belantamab mafodotin (2.5 mg/kg administered intravenously every 3 weeks [Q3W]) demonstrated an objective response rate of 32% with a manageable safety profile in triple-class refractory adult patients with RRMM (Lonial et al. Lancet Oncol. 2020). At 13 months of follow-up, responses were durable, with a median duration of response of 11 months and an overall survival of 13.7 months (Lonial et al. ASH 2020, Poster 1417). Corneal events are common and expected with belantamab mafodotin and other MMAF-containing ADCs. In DREAMM-2, keratopathy (a pathological eye exam finding) presented as superficial punctate keratopathy and/or microcyst-like epithelial changes. Ocular symptoms, such as decline in best-corrected visual acuity or patient-reported adverse events (eg, blurred vision or dry eye), were also common during treatment. Corneal events with or without symptoms were managed with dose modifications (delays and reductions), and clinically meaningful responses were observed even with prolonged treatment-free intervals; this suggests that alternative dosing regimens of belantamab mafodotin may lower rates of corneal events without compromising efficacy. The goal of the DREAMM-14 study is to investigate whether an improved overall benefit/risk profile of single-agent belantamab mafodotin can be achieved by modifying the belantamab mafodotin dose, schedule, or both relative to the approved dosing regimen (2.5 mg/kg Q3W). Methods: This Phase II, 5-arm, randomized, parallel, open-label multicenter study will include patients with RRMM who have received at least 3 prior lines of therapy including an anti-CD38 monoclonal antibody (mAb), an immunomodulatory agent, and a proteasome inhibitor. Patients aged ≥18 years with Eastern Cooperative Oncology Group Performance Status ≤2 and who provide informed consent will be eligible. Patients with corneal epithelial disease (except mild punctate keratopathy) or prior exposure to BCMA-targeted therapies or ADCs will be excluded. Patients will be randomized into Arms A to D (n=40 each) and arm E (n=20) in parallel and stratified by the International Staging System (ISS) for MM (I vs II vs III) and prior lines of therapy (3 vs ≥4). Single-agent belantamab mafodotin will be administered at doses and schedules as follows-Arm A: 2.5 mg/kg Q3W (control); Arm B: 1.9 mg/kg Q3W; Arm C: 2.5 mg/kg Q6W; Arm D: 1.9 mg/kg Q6W; Arm E: 1.9 mg/kg Q6W with dose modifications based on oncology staff assessment of ocular symptoms (patient-reported symptoms using the Ocular Surface Disease Index) and visual acuity. Participants in all arms will have Q3W response assessments and Q3W ophthalmic examinations and monitoring by qualified eye care specialists. Ocular event-related dose modifications for all arms except Arm E will be guided by the Keratopathy and Visual Acuity (KVA) scale. Participants in all arms will be treated until progressive disease, unacceptable toxicity, or death. The primary endpoint will be incidence of Grade ≥2 ocular adverse events according to the KVA scale. Key secondary endpoints include ocular safety and tolerability, pharmacokinetics, and efficacy outcomes of belantamab mafodotin in all arms. Follow-up for progression-free survival will be Q3W until progressive disease, start of new anticancer therapy, withdrawal of consent, end of study, or death. Follow-up for overall survival will be Q12W from treatment discontinuation. The duration of this study will be approximately 22 months. The study is planned to start in the first quarter of 2022. Funding: GSK (Study 209628); drug linker technology licensed from Seagen; mAb produced using POTELLIGENT Technology licensed from BioWa. Disclosures Hultcrantz: GlaxoSmithKline: Membership on an entity's Board of Directors or advisory committees, Research Funding; Curio Science LLC: Consultancy; Intellisphere LLC: Consultancy; Amgen: Research Funding; Daiichi Sankyo: Research Funding. Kleinman: Calm Water Therapeutics LLC: Current Employment; GlaxoSmithKline: Consultancy; Eyeon Therapeutics Inc.: Current holder of individual stocks in a privately-held company; ONL Therapeutics: Consultancy, Current holder of individual stocks in a privately-held company; Triphase Accelerator: Consultancy; Helixmith Co., Ltd: Consultancy; Aprea Therapeutics: Consultancy; Editas Medicine, Inc.: Consultancy; Olema Pharmaceuticals, Inc.: Consultancy. Ghataorhe: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. McKeown: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company; AstraZeneca: Current equity holder in publicly-traded company; Novartis: Current equity holder in publicly-traded company. He: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Ling: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Jewell: Alcon: Current equity holder in publicly-traded company; Novartis: Current equity holder in publicly-traded company; GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Brunner: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Byrne: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company; Adaptimmune: Current equity holder in publicly-traded company; Novartis: Current equity holder in publicly-traded company. Eliason: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Scott: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company. Opalinska: GlaxoSmithKline: Current Employment, Current equity holder in publicly-traded company.

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Ocular Toxicity of Belantamab Mafodotin, an Oncological Perspective of Management in Relapsed and Refractory Multiple Myeloma

Frontiers in Oncology ◽

10.3389/fonc.2021.678634 ◽

2021 ◽

Vol 11 ◽

Author(s):

Ahsan Wahab ◽

Abdul Rafae ◽

Kamran Mushtaq ◽

Adeel Masood ◽

Hamid Ehsan ◽

...

Keyword(s):

Multiple Myeloma ◽

Visual Acuity ◽

Treatment Interruption ◽

Ocular Toxicity ◽

Antibody Drug Conjugate ◽

Blurred Vision ◽

Dry Eyes ◽

Refractory Multiple Myeloma ◽

Ocular Symptoms ◽

Increased Risk

Belantamab mafodotin (belamaf), an antibody-drug conjugate approved for the treatment of relapsed and refractory multiple myeloma (RRMM), is an anti B-cell maturation antigen (BCMA) agent. DREAMM-1, a first in-human trial of belamaf, reported several ocular toxicities requiring dose adjustments, dose delays and treatment discontinuations. In DREAMM-1, 53% of patients in part-1 and 63% of patients in part-2 had ocular toxicity. Similarly, 73% of patients in DREAMM-2 had keratopathy (71% in 2.5 mg/kg versus 75% in 3.4 mg/kg) with the most common symptoms being blurred vision and dry eyes. Ocular toxicity of belamaf is attributed to microtubule-disrupting monomethylauristatin-F (MMAF), a cytotoxic payload of the drug that causes an off-target damage to the corneal epithelial cells. Ocular adverse events (AEs) of belamaf are more frequent at higher doses compared with lower doses. Higher belamaf dose, history of dry eyes and soluble BCMA are associated with increased risk of corneal toxicity. Absence of ocular symptoms does not exclude the possibility of belamaf-induced ocular toxicity, so patients need slit lamp and Snellen visual acuity testing to detect microcytic-like epithelial changes and visual decline. Corticosteroid eyes drops for 4-7 days prior to belamaf dose do not prevent ocular AEs and may cause steroid-related AEs instead. Keratopathy and Visual Acuity scale (KVA) is recommended to document the severity of belamaf-induced ocular toxicity and make treatment adjustments. Management of toxicity includes dosage modifications, treatment interruption or discontinuations and preservative-free artificial tears along with close ophthalmology and hematology-oncology follow-ups.

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DREAMM-2: Single-agent belantamab mafodotin (GSK2857916) in patients with relapsed/refractory multiple myeloma (RRMM) and renal impairment.

Journal of Clinical Oncology ◽

10.1200/jco.2020.38.15_suppl.8519 ◽

2020 ◽

Vol 38 (15_suppl) ◽

pp. 8519-8519 ◽

Cited By ~ 2

Author(s):

Hans Chulhee Lee ◽

Adam D. Cohen ◽

Ajai Chari ◽

Malin Hultcrantz ◽

Ajay K. Nooka ◽

...

Keyword(s):

Renal Function ◽

Renal Impairment ◽

Single Agent ◽

Similar Efficacy ◽

Poor Prognostic Factor ◽

B Cell Maturation ◽

Duration Of Response ◽

Function Table ◽

Mild Impairment ◽

Post Hoc

8519 Background: Renal impairment, a frequent complication and poor prognostic factor in RRMM, often leads to poor tolerability of standard regimens. We report outcomes in patients with renal impairment receiving single-agent belantamab mafodotin (2.5 or 3.4 mg/kg; B-cell maturation antigen targeting immunoconjugate not renally metabolized) from the DREAMM-2 post-hoc analysis (NCT03525678). Methods: Eligible patients with RRMM had no active renal conditions and adequate renal function (based on albumin/creatinine ratio [<500 mg/g] and eGFR [mL/min/1.73 m2]: normal [≥90], mild impairment [mild, ≥60≤90], moderate impairment [mod, ≥30≤60]). Results: Overall response rates (95% CI) in patients with mild/mod impairment (2.5 mg/kg: 32% [21.4–44.0]; 3.4 mg/kg: 36% [25.6–48.5]) were similar to those in the overall population ( Lancet Oncol.2020). The median duration of response (DoR) was not reached (NR) in 2.5 mg/kg mild/mod subgroup (95% CI estimate: 4.2 months–NR); median DoR was 7.5 months (4.9–NR) in 3.4 mg/kg mild/mod subgroup. Rates of keratopathy and albuminuria were similar regardless of renal function; rates of anemia, pyrexia, and thrombocytopenia were more frequent in patients with impaired renal function (Table). eGFR did not change or changed to normal in most patients. Conclusions: Following treatment with single-agent belantamab mafodotin, patients with mild/mod renal impairment achieved a similar efficacy and safety profile as patients with normal renal function. Funding: GlaxoSmithKline (205678). Drug linker technology licensed from Seattle Genetics; monoclonal antibody produced using POTELLIGENT Technology licensed from BioWa. Clinical trial information: NCT03525678 . [Table: see text]

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Landscape review of the patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE) in oncology: Adoption and recent learnings.

Journal of Clinical Oncology ◽

10.1200/jco.2021.39.15_suppl.e18587 ◽

2021 ◽

Vol 39 (15_suppl) ◽

pp. e18587-e18587

Author(s):

Antoine Regnault ◽

Stephane Quéré ◽

Boris Gorsh ◽

Sandhya Sapra ◽

Angély Loubert ◽

...

Keyword(s):

Clinical Trials ◽

Adverse Events ◽

Analytical Methods ◽

Linear Models ◽

Single Agent ◽

Patient Reported Outcome ◽

Item Selection ◽

Antibody Drug Conjugate ◽

Patient Reported ◽

Over Time

e18587 Background: The PRO-CTCAE is a patient-reported outcome measure of symptomatic toxicity in oncology trials designed to complement CTCAE criteria (clinician’s measure of adverse events [AEs]). Items for evaluation are selected from 78 symptomatic toxicities in the CTCAE. PRO-CTCAE uptake and use has not been uniformly assessed; understanding how to best analyze and present PRO-CTCAE data is important. We reviewed literature on clinical trials reporting the PRO-CTCAE to ascertain its use and current analytical methods to inform future analyses. Methods: A review of the literature and ongoing oncology trials using PubMed, oncology and outcomes research conferences (ASCO, ASH, ISOQOL, ISPOR), and ClinicalTrials.gov was completed August 2019; updated November 2020. Selected literature included guidance on: item selection; analytical methods; PRO-CTCAE data/endpoint use in clinical trials. Analytical and visualization methods from the literature were used to inform the specification of PRO-CTCAE analyses conducted on data from the DREAMM-2 (NCT03525678) trial of single-agent belantamab mafodotin (belamaf; BLENREP), a B-cell maturation antigen–binding antibody–drug conjugate, in relapsed/refractory multiple myeloma. Results: After screening (n = 197), 45 articles were reviewed: 13 related to recommendations for use/analysis, 7 to item selection, and 25 to examples of PRO-CTCAE use. 118 completed/ongoing trials reported PRO-CTCAE use (17 [14%] Phase I or I/II; 73 [62%] Phase II, II/III, or III; 3 [3%] Phase IV; 25 had no phase reported). Most (92/118, 78%) trials included the PRO-CTCAE as a secondary endpoint. The number of trials using the PRO-CTCAE increased from 3 in 2015 to 30 in 2020. PRO-CTCAE data in the literature were reported descriptively using tables (maximum post-baseline ratings; mean change over time; ratings higher than a predefined cutoff) and graphs (line graphs; stacked bar charts; heatmaps). These and more sophisticated analyses were applied to DREAMM-2 data to best capture the dynamics of patient-reported AEs over time, or control for baseline PRO-CTCAE ratings (eg, Toxicity over Time approach; ordinal log-linear models). Conclusions: The importance of tolerability and symptomatic AEs for patients is increasingly recognized, particularly for new therapies. This landscape review demonstrates recent growth in interest and adoption of PRO-CTCAE. There are few existing standards for analysis; this research increases understanding of existing methods and further evaluates ways of analyzing and presenting PRO-CTCAE data over time. Additional research exploring innovative methods to analyze PRO-CTCAE data and address its challenges is needed. Funding Source: GlaxoSmithKline (212225). Drug linker technology licensed from Seagen Inc.; mAb produced using POTELLIGENT Technology licensed from BioWa.

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Quality of life, effectiveness and patient reported outcomes of antiemetic prophylaxis with NEPA within the German NIS AkyPRO: post-hoc analysis of patients receiving carboplatin-based chemotherapy

10.1055/s-0040-1710726 ◽

2020 ◽

Author(s):

J Schilling ◽

R Lorenz ◽

D Rezek ◽

L Bauer ◽

D Hornung ◽

...

Keyword(s):

Quality Of Life ◽

Patient Reported Outcomes ◽

Life Effectiveness ◽

Post Hoc Analysis ◽

Antiemetic Prophylaxis ◽

Patient Reported ◽

Post Hoc

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Exploring the Relationship between Disease Awareness and Outcomes in Patients with Chronic Obstructive Pulmonary Disease

Respiration ◽

10.1159/000513953 ◽

2021 ◽

Vol 100 (4) ◽

pp. 291-297

Author(s):

Ilaria Baiardini ◽

Marco Contoli ◽

Angelo Guido Corsico ◽

Carla Scognamillo ◽

Fabio Ferri ◽

...

Keyword(s):

Chronic Obstructive Pulmonary Disease ◽

Pulmonary Disease ◽

Treatment Satisfaction ◽

Illness Perception ◽

Chronic Obstructive ◽

Obstructive Pulmonary Disease ◽

Post Hoc Analysis ◽

Disease Awareness ◽

Patient Reported ◽

Post Hoc

Background: Disease awareness is a challenge in the management of chronic obstructive pulmonary disease (COPD). Objectives: The aim of this analysis was to explore the association between COPD optimal and suboptimal awareness, clinical parameters, and the following patient-reported outcomes: modified Medical Research Council (mMRC), Treatment Satisfaction Questionnaire (TSQM-9), COPD Assessment Test (CAT), Morisky Medication-Taking Adherence Scale (MMAS-4), and Brief Illness Perception Questionnaire (B-IPQ). Methods: This post hoc analysis of the SAT study included all enrolled patients for whom awareness (Disease Awareness in COPD Questionnaire – DACQ) was assessed at baseline and 12 months. DACQ scores ≥80 were considered an indicator of an optimal awareness. Results: 367 patients (25.8% women, median age 72 years) were included in the analysis. At enrollment, 74 patients (20.2%) had a DACQ score ≥80. Patients with suboptimal awareness, compared to those in which awareness was optimal, had higher median scores for CAT (p = 0.0001) and mMRC (p = 0.0031), a lower median TSQM-9 global score (p < 0.0001), and higher median B-IPQ score (p < 0.0001). The proportion of patients who had exacerbations during the previous year was higher in patients with suboptimal COPD awareness than in those with DACQ score ≥80 (42.8 vs. 21.4%, p = 0.0009). During the 12-month observation period, illness perception, adherence, and treatment satisfaction were found to be independent factors significantly associated with level of disease awareness. Conclusion: The results of our post hoc analysis suggest that patients’ awareness of their COPD disease is related to both clinical outcomes and how they perceive and manage their condition.

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POS0128 PHYSICIAN AND PATIENT PERCEPTIONS OF SURGICAL PROCEDURES FOR KNEE OA ACROSS JAPAN, THE US AND 5 EU COUNTRIES: RESULTS OF A REAL-WORLD STUDY

Annals of the Rheumatic Diseases ◽

10.1136/annrheumdis-2021-eular.2510 ◽

2021 ◽

Vol 80 (Suppl 1) ◽

pp. 275.2-276

Author(s):

N. Fukui ◽

P. G. Conaghan ◽

K. Togo ◽

N. Ebata ◽

L. Abraham ◽

...

Keyword(s):

Surgical Procedures ◽

Primary Care Physicians ◽

Total Joint Replacement ◽

Functional Improvement ◽

Published Data ◽

Knee Oa ◽

Obesity And Diabetes ◽

Patient Reported ◽

The Us ◽

Effective Option

Background:Patients with knee osteoarthritis (OA) who do not achieve adequate pain relief and functional improvement with a combination of non-pharmacologic and pharmacologic therapies are recommended an arthroplasty as an effective option to relieve severe pain and functional limitations. However, some patients are reluctant to undergo surgical interventions, and clinicians may choose to avoid or delay surgery due to safety risks and/or the financial cost. It is of interest to understand if the use and perception of surgery differs between countries, however, few published data exist.Objectives:To demonstrate how surgery and the use of surgical procedures differs across Japan, United States of America (US) and 5 major European countries (EU5) and to evaluate patient perception towards surgery.Methods:Data were drawn from the Adelphi OA Disease Specific Programme (2017-18), a point-in-time survey of primary care physicians (PCP), rheumatologists (rheums), orthopaedic surgeons (orthos) and their OA patients. Patients with physician-diagnosed knee OA were included and segmented into two categories: had previous surgery (PS) and never had surgery (NS). A Fisher’s exact test was performed on the two groups. Physicians reported on patient demographics; whether patients had undergone surgery; type of surgery; success of surgery; how success was defined; and reasons for wanting to delay surgery. Patients reported their willingness to undergo surgery; reasons for not wanting surgery; how successful their surgery was; and how they defined this success.Results:Physician/patient reported data were available for 302,230 (Japan), 527,283 (US) and 1487,726 (EU5) patients with diagnosed knee OA. Patients were categorised by their physicians as mild (40% Japan; 34% US; 24% EU5), moderate (49% Japan; 49% US; 56% EU5) or severe (9% Japan; 17% US; 19% EU5). Patients in Japan were more likely to be female (78% vs 54% US; 58% EU5), older (73 vs 65 US; 66 EU5) and have a lower BMI than patients in the US and EU5. Obesity and diabetes were much less prevalent among patients in Japan. One in ten patients in Japan had undergone a surgery (10%), far fewer than in the US (22%) or EU5 (17%). When surgery was performed, this was more likely to be a total joint replacement (TJR) in Japan, whereas in the EU and US, arthroscopic washout was more commonly performed.For over half of Japanese patients (56%), successful surgery was more likely to be defined as having no more pain (vs. 35% US; 14% EU5). Improved mobility and a reduction in pain were also commonly reported reasons. Physicians (in each region) were more likely to suggest pain reduction, rather than no pain, and improved mobility as markers of success. Patients in Japan were much more likely to say they would not agree to surgery if recommended by their doctor, or were unsure (84% vs. 68% US; 62% EU5). The main reason for patient reluctance in Japan was fear of surgery, whereas in the US and EU5 the main reason given was that surgery was not needed. This finding was also evident among physicians in Japan, who frequently reported that patient reluctance was a key reason for delaying surgery. Physicians in Japan, do however, report that patient request was one of their main triggers for recommending surgery (45% vs 20% US; 16% EU5).Conclusion:Although surgery can be an effective option for those with OA who have exhausted other treatment options, some patients are reluctant to undergo surgery out of fear, especially in Japan, possibly due to the higher patient age. Physicians aiming to delay surgery were driven by patient reluctance in Japan, whereas cost to patient was a bigger factor in the US and EU5. The higher level of TJR vs. other surgery options among patients in Japan may suggest physicians are looking for higher levels of efficacy.Disclosure of Interests:Naoshi Fukui Speakers bureau: Pfizer, Consultant of: Pfizer, Philip G Conaghan Speakers bureau: Abbvie, Novartis, Consultant of: AstraZeneca, BMS, Eli Lilly, EMD Serono, Flexion Therapeutics, Galapagos, Gilead, Novartis, Pfizer, Kanae Togo Shareholder of: Pfizer, Employee of: Pfizer, Nozomi Ebata Shareholder of: Pfizer, Employee of: Pfizer, Lucy Abraham Shareholder of: Pfizer, Employee of: Pfizer, James Jackson: None declared, Jessica Jackson: None declared, Mia Berry: None declared, Hemant Pandit Paid instructor for: Bristol Myers Squibb, Consultant of: Johnson and Johnson, Grant/research support from: GSK

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Linguistic validation of the simplified Chinese version of the US National Cancer Institute’s patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE™)

BMC Cancer ◽

10.1186/s12885-020-07631-5 ◽

2020 ◽

Vol 20 (1) ◽

Author(s):

Cheng KKF ◽

S. A. Mitchell ◽

N. Chan ◽

E. Ang ◽

W. Tam ◽

...

Keyword(s):

Adverse Events ◽

Cancer Patients ◽

Patient Reported Outcomes ◽

A Priori ◽

Cognitive Interviews ◽

Simplified Chinese ◽

Patient Reported ◽

The Us ◽

The Common ◽

Response Choices

Abstract Background The aim of this study was to translate and linguistically validate the U.S. National Cancer Institute’s Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE™) into Simplified Chinese for use in Singapore. Methods All 124 items of the English source PRO-CTCAE item library were translated into Simplified Chinese using internationally established translation procedures. Two rounds of cognitive interviews were conducted with 96 cancer patients undergoing adjuvant treatment to determine if the translations adequately captured the PRO-CTCAE source concepts, and to evaluate comprehension, clarity and ease of judgement. Interview probes addressed the 78 PRO-CTCAE symptom terms (e.g. fatigue), as well as the attributes (e.g. severity), response choices, and phrasing of ‘at its worst’. Items that met the a priori threshold of ≥20% of participants with comprehension difficulties were considered for rephrasing and retesting. Items where < 20% of the sample experienced comprehension difficulties were also considered for rephrasing if better phrasing options were available. Results A majority of PRO-CTCAE-Simplified Chinese items were well comprehended by participants in Round 1. One item posed difficulties in ≥20% and was revised. Two items presented difficulties in < 20% but were revised as there were preferred alternative phrasings. Twenty-four items presented difficulties in < 10% of respondents. Of these, eleven items were revised to an alternative preferred phrasing, four items were revised to include synonyms. Revised items were tested in Round 2 and demonstrated satisfactory comprehension. Conclusions PRO-CTCAE-Simplified Chinese has been successfully developed and linguistically validated in a sample of cancer patients residing in Singapore.

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