Health-related Quality Of Life Endpoints Research Articles

Health-related quality of life and quality-adjusted progression free survival for carfilzomib and dexamethasone maintenance following salvage autologous stem-cell transplantation in patients with multiple myeloma: a randomized phase 2 trial by the Nordic Myeloma Study Group

BackgroundDecisions regarding maintenance therapy in patients with multiple myeloma should be based on both treatment efficacy and health-related quality of life (HRQL) consequences. In the CARFI trial, patients with first relapse of multiple myeloma underwent salvage autologous stem cell transplantation (salvage ASCT) before randomization to carfilzomib-dexamethasone maintenance therapy (Kd) or observation. The primary clinical endpoint was time to progression, which was extended by 8 months by Kd. The aim of this paper is to present the all HRQL endpoints of the CARFI trial including the HRQL effect of Kd maintenance therapy relative to observation. The primary HRQL endpoint was assessed by EORTC QLQ-C30 Summary score (QLQ-C30-sum) at 8 months follow-up. A key secondary HRQL endpoint was quality-adjusted progression-free-survival (QAPFS).MethodsHRQL was assessed with EORTC QLQ-C30, EORTC QLQ-MY20 and FACT/GOG-Ntx at randomization and every second month during follow-up. HRQL data were analyzed with linear mixed effect models until 8 months follow-up. QAPFS per individual was calculated by multiplying progression-free survival (PFS) by two quality-adjustment metrics, the QLQ-C30-sum and EORTC Quality of Life Utility Measure-Core 10 dimensions (QLU-C10D). The QAPFS per treatment group was estimated with the Kaplan-Meier method. P < 0.05 was used for statistical significance, and a between-group minimal important difference of 10 points was interpreted as clinically relevant for the QLQ-C30-sum.Results168 patients were randomized. HRQL questionnaire compliance was 93%. For the QLQ-C30-sum, the difference of 4.62 points (95% confidence interval (CI) -8.9: -0.4, p = 0.032) was not clinically relevant. PFS was 19.3 months for the Kd maintenance group and 16.8 months for the observation group; difference = 2.5 months (95% CI 0.5; 4.5). QAPFS based on the QLQ-C30-sum for the Kd maintenance group was 18.0 months (95% CI 16.4; 19.6) and for the observation group 15.0 months (95% CI 13.5; 16.5); difference = 3.0 months (95% CI 0.8–5.3). QAPFS based on the QLU-C10D for the Kd maintenance group was 17.5 months (95% CI 15.9; 19.2) and 14.0 months (95% CI 12.4; 15.5) for the observation group; difference = 3.5 months (95% CI 1.1–5.9).ConclusionsKd maintenance therapy after salvage ASCT did not adversely affect overall HRQL, but adjustment for HRQL reduced the PFS compared to unadjusted PFS. PFS of maintenance therapy should be quality-adjusted to balance the benefits and HRQL impact.

A Phase 3 Study of Pembrolizumab versus Placebo for Previously Treated Patients from Asia with Hepatocellular Carcinoma: Health-Related Quality of Life Analysis from KEYNOTE-394

Introduction: KEYNOTE-394 showed pembrolizumab significantly improved overall survival, progression-free survival, and objective response rate with manageable safety versus placebo for patients from Asia with previously treated advanced hepatocellular carcinoma. We present results on health-related quality of life (HRQoL). Methods: HRQoL was evaluated using the EORTC Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30) and EuroQol-5D-3L (EQ-5D-3L) questionnaires. Key HRQoL endpoints were least squares mean (LSM) score changes from baseline to week 12 and time to deterioration (TTD) for EORTC QLQ-C30 global health status (GHS)/QoL. p values were one-sided and nominal without adjustment for multiplicity. Results: The HRQoL population included patients randomly assigned to pembrolizumab (n = 298) and placebo (n = 152). From baseline to week 12, a greater decline in EORTC QLQ-C30 GHS/QoL score was observed with placebo (LSM, −8.4; 95% CI: −11.7 to −5.1) versus pembrolizumab (−4.0; 95% CI: −6.4 to −1.6; difference vs. placebo: 4.4; 95% CI: 0.5–8.4; nominal p = 0.0142). Similarly, a greater decline in the EQ-5D-3L visual analog scale score was observed with placebo (−6.9; 95% CI: −9.4 to −4.5) versus pembrolizumab (−2.7; 95% CI: −4.5 to −1.0; difference vs. placebo: 4.2; 95% CI: 1.2–7.2; nominal p = 0.0030). TTD in EORTC QLQ-C30 GHS/QoL score was similar between arms (hazard ratio, 0.85; 95% CI: 0.58–1.25; nominal p = 0.1993). Conclusion: Patients receiving placebo showed a greater decline in HRQoL than those receiving pembrolizumab. Combined with efficacy and safety data from KEYNOTE-394 and the global KEYNOTE-240 and KEYNOTE-224 trials, our data support the clinically meaningful benefit and manageable tolerability of pembrolizumab as second-line therapy for patients with advanced hepatocellular carcinoma.

The Effects of Rilzabrutinib, an Oral Bruton Tyrosine Kinase Inhibitor, on Bleeding Symptoms and Health-Related Quality of Life in Patients with Immune Thrombocytopenia

Introduction: Patients with immune thrombocytopenia (ITP) have a high incidence of comorbid conditions, elevated risk for bleeding and thrombosis, and reduced health-related quality of life (HRQoL). Rilzabrutinib is a potent oral, reversible inhibitor of Bruton tyrosine kinase with the potential to modulate multiple immunological pathways. In part A of a phase 1/2 study (LUNA 2) of ITP patients, rilzabrutinib treatment led to rapid and durable platelet responses with a favorable safety profile. This abstract reports the effects of rilzabrutinib on bleeding and HRQoL on patients in part B of the LUNA 2 study. Methods: The multicenter, open-label, phase 1/2, part B study evaluated the efficacy and safety of rilzabrutinib 400 mg BID in relapsed ITP (NCT03395210). Adults aged 18-80 y were eligible with ≥2 baseline platelet counts &amp;lt;30x10 9/L no less than 7 days apart in the 15 days before the first dose. Eligible patients were required to have a past response (achievement of platelet count ≥50x10 9/L) to intravenous immunoglobulin (IVIg)/anti-D or corticosteroid (CS) that was not sustained, and have failed ≥1 other ITP therapy (not IVIg or CS). Stable doses of concomitant CS/thrombopoietin receptor agonists (TPO-RA) were allowed with rilzabrutinib. The primary endpoints were safety and durable platelet response defined as platelet counts ≥50x10 9/L on ≥8 of the last 12 weeks of rilzabrutinib without rescue medication. Change from baseline in the ITP Bleeding Scale (IBLS) score (0 none to 2 marked bleeding) was a secondary endpoint to assess bleeding across 9 anatomical sites (8 for male/postmenopausal women). An exploratory HRQoL endpoint evaluated the EuroQol-5 Dimensions 5-Level (EQ-5D-5L) + Visual Analog Scale (EQ-VAS) and ITP Patient Assessment Questionnaire (ITP-PAQ; scores 0 worst to 100 best HRQoL). Results: For 26 enrolled patients with data cut-off of 31Jan2023, 15 patients completed 24 weeks of treatment and 11 discontinued due to lack of response (n=5); adverse events (AEs, n=3); and n=1 each lack of response/AE, noncompliance, and erroneous enrollment. At baseline, patients had a median age of 57 y (range, 20-75) with median platelet count of 13x10 9/L (range, 2-24x10 9/L). Patients had a median duration of ITP of 10.3 y (range, 0.7-48.2) and had received a median of 6 prior unique ITP therapies (range, 3-19; 46% splenectomy). Prior ITP medication included CS (100%), TPO-RA (85%), immunosuppressants or IVIg (81%), and rituximab (50%). During the study, 17 patients (65%) received concomitant non-rescue CS and/or TPO-RA. The median compliance to rilzabrutinib for patients with 24 weeks of treatment was 99%. Nine patients (35%; 95% CI, 17-56%) achieved the primary endpoint of durable platelet response. Treatment-related AEs were mainly grade 1/2, with no treatment-related grade ≥2 bleeding/thrombotic events or infections, serious AEs, or deaths. At baseline, the IBLS average score across sites was a mean of 0.27 (SD, 0.27) and mean change from baseline at week 25 was -0.07 (SD, 0.12). The anatomical site with the highest proportion of patients having a baseline score of 2 was skin (n=7 per medical history, n=6 per physical examination), all of whom had a decrease at 25 weeks. At baseline, median EQ-VAS was 78 (range, 8-100) with a median change from baseline (N=26) to week 25 (n=15) of +7 (range, -5 to 77) indicating an improvement in HRQoL. Median changes in ITP-PAQ domain scores from baseline to week 25 (Figure 1) showed clinically meaningful improvement in women's reproductive health with a 21-point increase (range, 0-38; median baseline: 54); clinically meaningful improvement in fatigue/sleep, activity, and overall HRQoL scales with ~13-point increases (range, -23 to 75; median baseline: 53, 50, and 52, respectively); and improvement in psychological, symptoms, social activity, and bother-physical health domains with 4- to 8-point increases (range, -13 to 56; median baseline: 70, 60, 75, 69, respectively). Fear and work domains had high scores at baseline (≥90) with minimal changes over time. Conclusion: Overall, rilzabrutinib showed durable platelet responses, high compliance, and improvements on HRQoL measures in difficult to treat patients with relapsed ITP. There was no evidence of increased bleeding with rilzabrutinib. Clinically meaningful improvements in HRQoL were observed in multiple individual and overall HRQoL health domains following rilzabrutinib.

Impact of pembrolizumab versus chemotherapy on health-related quality of life in patients with metastatic triple-negative breast cancer: results from the phase 3 randomised KEYNOTE-119 study

In KEYNOTE-119 (ClinicalTrials.gov, NCT02555657), overall survival (primary end-point) was similar between pembrolizumab and chemotherapy in patients with previously treated metastatic triple-negative breast cancer (TNBC), although the pembrolizumab treatment effect increased with tumour PD-L1 expression. We report results of prespecified health-related quality of life (HRQoL) analyses from KEYNOTE-119. Eligible patients were randomised 1:1 to pembrolizumab 200mg Q3W intravenously for up to 35 cycles or treatment of physician's choice per local/country guidelines. Prespecified exploratory end-points were the change from baseline in HRQoL (EORTC QLQ-C30, QLQ-BR23) and to characterise utilities (EQ-5D-3L). Time to deterioration (TTD) was the time from start of treatment to first onset of a ≥10-point worsening from baseline. HRQoL analyses included 187 patients with tumour PD-L1 combined positive score (CPS) ≥10. Changes from baseline at 6 weeks (primary analysis time point) were directionally better with pembrolizumab versus chemotherapy for QLQ-C30 GHS/QoL (between-group difference in least-squares mean scores of 4.21 [95% CI, -1.38 to 9.80]), QLQ-C30 functional scales (physical, role, cognitive, social), QLQ-C30 symptom scales/items (fatigue, nausea/vomiting, dyspnoea, appetite loss), and QLQ-BR23 symptom scales/items (systemic therapy side-effects, upset by hair loss). Median TTD was directionally longer for pembrolizumab versus chemotherapy for QLQ-C30 QHS/QoL (4.3 versus 1.7 months), QLQ-C30 nausea/vomiting (7.7 versus 4.8 months), and QLQ-BR23 systemic therapy side-effects (6.1 versus 3.4 months). Minimal treatment differences were observed for other HRQoL end-points. HRQoL results were consistent with clinical outcomes and appeared to be driven by results for patients with tumour PD-L1 CPS ≥10.

423 Long-term efficacy of abrocitinib up to 96 weeks in adults with moderate-to-severe atopic dermatitis stratified by age: a post hoc analysis of the JADE EXTEND phase 3 trial

Abstract Oral abrocitinib 200 and 100 mg once daily provided short-term efficacy in a dose-dependent manner over 12 or 16 weeks in various age groups of patients with moderate-to-severe atopic dermatitis (AD). This study aims to evaluate the long-term efficacy of abrocitinib treatment for up to 96 weeks in adult patients with moderate-to-severe AD who enrolled in JADE EXTEND (NCT03422822). This planned interim analysis included adult patients from the JADE clinical trials MONO-1 (NCT03349060), MONO-2 (NCT03575871), COMPARE (NCT03720470) and DARE (NCT04345367) who subsequently enrolled into the ongoing phase 3 extension trial JADE EXTEND (data cutoff: September 25, 2021). Patients who were randomly assigned to abrocitinib 200 or 100 mg once daily in the qualifying trials continued to receive the same dose in JADE EXTEND with blinding maintained; those who received placebo were randomly assigned to abrocitinib 200 or 100 mg. All patients from JADE DARE received a known dose of abrocitinib 200 mg in JADE EXTEND. Patients previously randomly assigned to dupilumab in JADE DARE or JADE COMPARE were excluded from this analysis. Patients were grouped for analysis by age (18–50 years; &amp;gt;50 years) recorded at the screening visit of the respective qualifying trial. Assessments included the proportion of patients who achieved Investigator’s Global Assessment (IGA) score of 0 (clear) or 1 (almost clear) with a ≥2-grade improvement from baseline, ≥75% and ≥90% improvement from baseline on the Eczema Area and Severity Index (EASI-75 and EASI-90), a score of &amp;lt;2 or a ≥4-point improvement from baseline in Peak Pruritus Numerical Rating Scale score (PP-NRS 0/1 or PP-NRS4), and a score of &amp;lt;2 on the Dermatology Life Quality Index (DLQI 0/1). Data (as observed values) were evaluated up to 96 weeks of abrocitinib treatment. A total of 1309 patients [18–50 years (n = 1046); &amp;gt;50 years (n = 263)] were analysed. At Week 16 of abrocitinib treatment, patients achieved improvements with abrocitinib 200 or 100 mg in a dose-dependent manner in both age groups for IGA 0/1 [18–50 years, 55% (abrocitinib 200 mg)/34% (abrocitinib 100 mg); &amp;gt;50 years, 56%/38%], EASI-75 (79%/61%; 76%/69%) and PP-NRS4 (70%/51%; 76%/61%). Similar dose-dependent responses in both age groups were observed for the high-threshold clinical, patient-reported and health-related quality of life (HRQoL) endpoints of EASI-90 [18–50 years, 54% (abrocitinib 200 mg)/33% (abrocitinib 100 mg); &amp;gt;50 years, 59%/40%], PP-NRS 0/1 (35%/25%; 54%/30%) and DLQI 0/1 (38%/23%; 47%/24%). Regardless of age and dose, substantial proportions of patients were observed with responses at Weeks 24, 48 and 96 of abrocitinib treatment. At Week 96, dose-dependent responses remained for the younger subgroup of patients (including for high-threshold responses). For the older subgroup, responses were less clearly dose-dependent from Week 48. At Week 96, responder proportions were as follows: for IGA 0/1: 18–50 years, 55% (abrocitinib 200 mg)/44% (abrocitinib 100 mg); &amp;gt;50 years, 58%/51%; for EASI-75: 85%/73%, 89%/86%; for PP-NRS4: 66%/54%; 80%/79%; and for the high-threshold endpoints of EASI-90: 58%/45%; 73%/58%; for PP-NRS 0/1: 38%/26%; 44%/54%; and for DLQI 0/1: 41%/32%; 48%/51%. Regardless of age, substantial proportions of adults achieved clinical and patient-reported endpoints (IGA 0/1, EASI-75 or PP-NRS4) after short-term treatment with either abrocitinib dose. Similar trends were observed for high-threshold clinical, patient-reported and HRQoL endpoints (EASI-90, PP-NRS 0/1 or DLQI 0/1). Dose-dependent efficacy was observed short-term regardless of age, and up to 96 weeks in patients aged 18–50 years. Responder proportions of patients aged &amp;gt;50 years were less clearly dose-dependent from Week 48 of abrocitinib treatment. These findings may be informative for patients and healthcare providers when making treatment decisions. Further data from the ongoing JADE EXTEND trial may provide greater precision for long-term efficacy estimates.

Measuring What Really Matters: Disease Specific Health Related Quality of Life in Patients with Chronic Limb Threatening Ischaemia

Disease Specific Health Related Quality of Life in Patients With Chronic Limb Threatening Ischaemia Undergoing Revascularisation of Femoropopliteal LesionsEuropean Journal of Vascular and Endovascular SurgeryPreviewPatients with chronic limb threatening ischaemia (CLTI) suffer from pain and non-healing ulcers, which impact negatively on both their physical and mental health. While maintaining and improving quality of life is a principal aim with all treatments, little is known about the health related quality of life (HRQoL) of CLTI patients and how revascularisation procedures impact on HRQoL endpoints. The aim of this study was to investigate disease specific HRQoL before and after revascularisation in patients with CLTI undergoing femoropopliteal revascularisation. Full-Text PDF Open Access

Disease Specific Health Related Quality of Life in Patients With Chronic Limb Threatening Ischaemia Undergoing Revascularisation of Femoropopliteal Lesions

Patients with chronic limb threatening ischaemia (CLTI) suffer from pain and non-healing ulcers, which impact negatively on both their physical and mental health. While maintaining and improving quality of life is a principal aim with all treatments, little is known about the health related quality of life (HRQoL) of CLTI patients and how revascularisation procedures impact on HRQoL endpoints. The aim of this study was to investigate disease specific HRQoL before and after revascularisation in patients with CLTI undergoing femoropopliteal revascularisation. HRQoL was prospectively analysed in 190 CLTI patients with main atherosclerotic target lesions in the femoropopliteal segment, who were planned for endovascular or open revascularisation. The choice of revascularisation method was made by the vascular team, represented by both open and endovascular expertise. The Vascular Quality of Life (VascuQoL) questionnaire was used to assess disease specific HRQoL before revascularisation and one month, one year, and two years after the procedure. Main endpoints were mean VascuQoL score changes, effect sizes of observed changes and the proportion reaching a minimally important difference (half a standard deviation change from baseline) during two years after revascularisation. Patient reported VascuQoL scores were low at baseline (mean 2.68, 95% CI 1.18 - 4.17). After revascularisation, the mean VascuQoL score improved statistically significantly over time, with the largest improvement observed after one year (difference from baseline 2.02, 95% CI 1.75 - 2.29; p < .001). No differences in HRQoL change over time were observed between patients treated with endovascular approaches compared with bypass surgery. Approximately half the patients reached the minimally important threshold at one year (53%), which was largely maintained also at two years (41%). While CLTI profoundly affected HRQoL, a large and clinically meaningful HRQoL increase was observed after revascularisation. This confirms the value of CLTI revascularisation on HRQoL and underlines the importance of including patient reported outcomes when evaluating revascularisation procedures in CLTI patients.

Health-Related Quality of Life in Patients With Advanced Endometrial Cancer Treated With Lenvatinib Plus Pembrolizumab or Treatment of Physician’s Choice

PurposeLenvatinib and pembrolizumab (LEN+PEMBRO) demonstrated clinically meaningful and statistically significant improvements in efficacy versus treatment of physician’s choice (TPC) in patients with advanced endometrial cancer (aEC) in the phase 3 Study 309/KEYNOTE-775. Health-related quality-of-life (HRQoL) is reported. Patients and MethodsPatients were randomly assigned to receive LEN+PEMBRO (n = 411; LEN 20 mg/day; PEMBRO 200 mg Q3W) or TPC (n = 416; doxorubicin 60 mg/m2 Q3W or paclitaxel 80 mg/m2 [weekly, 3 weeks on/1 week off]). Impact of treatment on HRQoL assessed by the global health status/quality of life (GHS/QoL) score of the European Organisation for Research and Treatment of Cancer Quality-of-Life Questionnaire (EORTC QLQ-C30) was a secondary objective; other scales of the Quality-of-Life Questionnaire (QLQ-C30), EORTC QLQ-Endometrial, 24 questions (EORTC QLQ-EN24), and EuroQoL 5 dimensions, 5 levels (EQ-5D-5L) were exploratory objectives. HRQoL was assessed on day 1 of each cycle. Completion/compliance, change from baseline, time to first and definitive deterioration were assessed. No multiplicity adjustments were applied for HRQoL endpoints. ResultsThe latest timepoint at which the predefined rates of completion (≥60%) and compliance (≥80%) were met was week 12. HRQoL at week 12 between treatment groups was generally similar. Time to first deterioration symptom scales favoured LEN+PEMBRO for QLQ-C30 dyspnoea, and QLQ-EN24 for poor body image, tingling/numbness, and hair loss; and TPC was favoured for QLQ-C30 pain, appetite loss, and diarrhoea, and QLQ-EN24 muscular pain. While the QLQ-C30 physical functional scale favoured TPC, other functional scales were generally similar between arms. Time to definitive deterioration favoured LEN+PEMBRO on most scales. ConclusionHRQoL data from Study 309/KEYNOTE-775, with previously published efficacy and safety results, indicate that LEN+PEMBRO has an overall favourable benefit/risk profile versus TPC for the treatment of patients with aEC. ClinicalTrials.govNCT03517449.

Health-related quality of life (HRQoL) impact of lenvatinib (len) plus pembrolizumab (pembro) versus len plus placebo (pbo) as first-line (1L) therapy for advanced hepatocellular carcinoma (aHCC): Phase 3 LEAP-002 study.

506 Background: In LEAP-002 (NCT03713593), len + pembro achieved a median OS of 21.2 mo vs 19.0 mo with len + pbo with manageable safety in both arms in 1L aHCC, although the significance thresholds for OS and PFS were not met. We present here the results of prespecified exploratory patient (pt)-reported outcomes (PROs). Methods: 794 pts were randomized. PROs were assessed by EORTC QLQ-C30, EuroQol-5D5L (EQ5D-5L), and EORTC QLQ-HCC18 questionnaires. Analysis population for HRQoL endpoints included pts who received ≥1 dose of study Tx and completed ≥1 HRQoL assessment. Least squares mean (LSM) score changes from baseline (BL) to wk 27 were compared using a constrained longitudinal data analysis model (covariates: Tx, time, Tx by time interaction, and stratification factors). Kaplan-Meier method was used to estimate time to deterioration (TTD, time to 1st onset of ≥10 [out of 100] deterioration from BL in a given scale or subscale/confirmed by a 2nd adjacent ≥10 deterioration from BL) for EORTC QLQ-C30 global health status (GHS)/QoL, physical functioning (PF), and EORTC QLQ-HCC18 abdominal swelling, pain and fatigue, as reported by pts. Stratified Cox proportional hazards model was used to assess the magnitude of the Tx difference (HR) between arms in TTD. Results: Compliance with PRO assessments was &gt;91% from BL until wk 27 in both arms. From BL to wk 27, LSM changes in GHS/QoL, PF, role functioning (RF), QLQ-HCC18 functional/domain scores and EQ5D-5L VAS were generally similar between the two arms (Table). Treatment with len + pembro delayed deterioration of patient-reported quality of life vs len + pbo (median TTD, 11.5 vs 4.3 mo; HR, 0.80; 95% CI, 0.65-0.98). LSM score difference (95% CI) was 0.5 (-2.5, 3.4) for GHS/QoL, -1.7 (-4.5, 1.1) for PF, -1.0 (-4.9, 2.9) for RF, 1.0 (-2.2, 4.3) for abdominal swelling, 2.0 (-1.2, 5.2) for fatigue, 2.0 (-1.0, 5.0) for pain, and 0.6 (-2.0, 3.2) for EQ5D-5L VAS. Median TTD in QLQ-C30 PF (5.7 vs 9.1 mo; HR, 1.14; 95% CI, 0.93-1.41) and QLQ-HCC18 abdominal swelling (NR vs NR; HR, 1.19; 95% CI, 0.89-1.60) /fatigue (2.9 vs 2.8 mo; HR, 0.98; 95% CI, 0.81-1.19)/pain (9.6 vs 7.9 mo; HR, 0.93; 95% CI, 0.75-1.16) was similar between the arms. Conclusions: Len + pembro generally preserved HRQoL vs len + pbo in 1L for aHCC. Combined with efficacy and safety results from LEAP-002, these findings support continued development of len + pembro in HCC. Clinical trial information: NCT03713593 . [Table: see text]

Health-related quality of life in patients with RAS wild-type metastatic colorectal cancer treated with fluorouracil and folinic acid with or without panitumumab as maintenance therapy: An analysis of the Panama trial (AIO KRK0212).

51 Background: The PANAMA study demonstrated superior progression-free survival (PFS) with the addition of panitumumab (Pmab) to fluorouracil and folinic acid (FU/FA) as maintenance therapy following first-line induction therapy with FOLFOX/Pmab in patients with RAS wild-type metastatic colorectal cancer. We report health-related quality of life (HRQOL) analyses of the PANAMA study. Methods: HRQOL was assessed by European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) at every cycle of therapy until disease progression. All patients who received at least one dose of induction therapy and completed at least one HRQOL assessment were included into the analysis. HRQOL outcomes were mean changes in EORTC QLQ-C30 from baseline (prior to cycle 1 of induction therapy) to each cycle of treatment (both induction and maintenance therapy). The trial is registered with ClinicalTrials.gov (NCT01991873). Results: In total, 349/377 (93%) of the induction and 237/248 pts (96%) of the maintenance group completed at least one HRQOL assessment and were included in the HRQOL analysis population. There were no significant differences in any of the EORTC QLQ-C30 items between both treatment arms before induction therapy and at randomization. From baseline to cycle 6 of induction therapy there was significant improvement in mean EORTC QLQ-C30 global health status (GHS)/QOL, functioning (except for cognitive functioning) and symptom (except for nausea and vomiting, dyspnea, appetite loss, constipation, and financial difficulties) scores in the randomized population. During maintenance therapy, no significant differences between FU/FA plus Pmab and FU/FA alone were observed. In both arms of the trial, GHS/QOL scores were maintained or trended to improve from baseline (start of induction therapy) to cycle 10 of maintenance therapy (FU/FA plus Pmab: mean difference 9.48 [95% CI 1.96-17.00]; p=0.014); FU/FA arm (mean difference 6.52 [95% CI –1.9-14.95]; p=0.128). Conclusions: Using the established EORTC QLQ-C30 assessment, the addition of Pmab to FU/FA as maintenance therapy in patients with RAS wild-type metastatic colorectal cancer did not impair the HRQOL endpoints analyzed compared to FU/FA alone. These results, along with previously reported improvement in PFS, may support clinical decision-making concerning maintenance treatments. Clinical trial information: NCT01991873 .

Efgartigimod improved health-related quality of life in generalized myasthenia gravis: results from a randomized, double-blind, placebo-controlled, phase 3 study (ADAPT)

There are substantial disease and health-related quality-of-life (HRQoL) burdens for many patients with myasthenia gravis (MG), especially for those whose disease symptoms are not well controlled. HRQoL measures such as the Myasthenia Gravis Quality of Life 15-item revised (MG-QOL15r) and EuroQoL 5-Dimensions 5-Levels (EQ-5D-5L) are vital for evaluating the clinical benefit of therapeutic interventions in patients with MG, as they assess the burden of disease and the effectiveness of treatment, as perceived by patients. The phase 3 ADAPT study (NCT03669588) demonstrated that efgartigimod—a novel neonatal Fc receptor inhibitor—was well tolerated and that acetylcholine receptor antibody–positive (AChR-Ab+) participants who received efgartigimod had statistically significant improvements in MG-specific clinical scale scores. The ancillary data reported here, which cover an additional treatment cycle, show that these participants had similar significant improvements in HRQoL measures, the MG-QOL15r and EQ-5D-5L utility and visual analog scales, and that these improvements were maintained in the second treatment cycle. Positive effects on HRQoL were rapid, seen as early as the first week of treatment in both treatment cycles, and maintained for up to 4 weeks in the follow-up–only portion of treatment cycles. The pattern of improvements in HRQoL paralleled changes in immunoglobulin G level, and correlational analyses show that improvements were consistent across HRQoL measures and with clinical efficacy measures in the ADAPT study. The substantial and durable improvements in HRQoL end points in this study demonstrate the broader benefit of treatment with efgartigimod beyond relief of immediate signs and symptoms of gMG.

10-Day Decitabine Versus Intensive Chemotherapy Followed By Transplantation in Fit AML Patients Aged ≥60 Years: Health-Related Quality of Life Outcomes of the Randomized Phase III Trial AML21 of the EORTC Leukemia Group, Gimema, Celg, and Gmds-SG

Background To assess the optimal treatment strategy for fit older patients with acute myeloid leukemia (AML), we performed a large international phase III randomized controlled trial (RCT) in fit AML patients aged >60 years, comparing 10-day decitabine (DEC) followed by allografting (HSCT) versus intensive chemotherapy (IC, "3+7" regimen) followed by HSCT. This trial revealed comparable HSCT rates and survival outcomes, but a more favorable safety profile with DEC when compared to IC (Lübbert M. et al, HemaSphere, 6, 26-27, 2022), supporting the hypothesis that reduced toxicity translates into a better health-related quality of life (HRQoL). Objective We hereby report the HRQoL outcomes of this international RCT. Patients and Methods HRQoL was a secondary endpoint and was assessed by the EORTC QLQ-C30 and its validated module for evaluating HRQoL in older patients, namely the EORTC QLQ-ELD14. Key inclusion criteria were: newly diagnosed AML, age >60 years, eligibility for IC, and a WHO performance status of ≤2. Assessments of HRQoL were performed at baseline and then at 2 (at regeneration after two treatment cycles), 6 and 12 months. For patients undergoing a HSCT, HRQoL was assessed prior to the procedure and at day 100 after the transplant. Quality of life deterioration (QD) was defined as any of the following events: death, progression, and clinically meaningful deterioration from baseline in at least one of the five pre-selected primary HRQoL scales (i.e., physical functioning, role functioning, fatigue, pain, and illness burden). The two co-primary endpoints were: QD at 2 months and QD at 6 or 12 months. A difference of 10 points or more was considered clinically meaningful. We hypothesized better HRQoL outcomes for patients treated with DEC. The changes in the primary HRQoL scales from baseline to 2 months and from baseline to the average of 6 and 12 months were secondary HRQoL endpoints. HRQoL trajectories of patients undergoing HSCT on-study based on evaluations at baseline, prior to HSCT and at day 100 after HSCT were also described. This study was registered at ClinicalTrials.gov (NCT02172872). Results Between December 2014 and August 2019, 606 patients were randomized and a baseline HRQoL assessment was available for 549 patients (91%): 279 out of 303 in the DEC arm and 270 out of 303 in the IC arm. Compliance among alive patients at 2, 6 and 12 months was 57%, 57%, and 64%, respectively. The proportion of patients evaluable for QD at 2 months was 60%. The proportion of patients evaluable for QD at 6 or 12 months was 82%. Analysis of the primary HRQoL endpoint showed that the proportion of patients deteriorating at 2 months was 76% (95% CI, 69 to 82%) in the DEC group vs 88% (95% CI, 82 to 93%) in the IC group (odds ratio 0.43 [95% CI, 0.24 to 0.76], P=.003). Several sensitivity analyses, using multiple imputation to deal with missing values and predefined modifications of the definition of QD, confirmed this finding. QD analysis at 6 or 12 months indicated no statistically significant difference between both arms (odds ratio 0.83 [95% CI, 0.46 to 1.52]). At 2 months from randomization, the illness burden scale increased by 4.19 (95% CI, -0.44 to 8.83) points in the DEC and by 15.15 (95% CI, 9.88 to 20.42) points in the IC arm, indicating a clinically meaningful and statistically significant difference of -10.96 (95% CI, -17.94 to -3.97), P=.004. The changes in the primary HRQoL scales from baseline to 2 months and from baseline to the average of 6 and 12 months are presented in Table 1. Baseline data were available for 218 of the 240 patients who received an on-study HSCT. Clinically meaningful HRQoL deteriorations between baseline and post-HSCT in the IC group were observed in all primary HRQoL scales except for pain. The change from baseline to post-HSCT HRQoL in the DEC arm appeared less pronounced and did not reach the threshold of clinical relevance. Conclusions Following two months of treatment, there was a significantly lower percentage of patients treated with DEC who reported HRQoL deterioration, compared to those treated with IC. Additionally, patients treated with DEC did not experience any clinically meaningful deterioration in key pre-specified HRQoL domains post-HSCT, while this was the case for patients treated with IC. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Fitusiran Prophylaxis Improves Health-Related Quality of Life in People with Hemophilia a or B, with or without Inhibitors: Results of ATLAS-PPX Study

Background: Fitusiran is a prophylactic investigational therapeutic comprised of synthetic small interfering ribonucleic acid (siRNA) that targets antithrombin to enhance thrombin generation and rebalance hemostasis in people with hemophilia A or B (PwHA/B), irrespective of inhibitor status. In previous Phase 3 trials, once-monthly fitusiran prophylaxis significantly improved HRQoL in PwHA/B, with or without inhibitors versus episodic/on-demand treatment Objective: We present health-related quality of life (HRQoL) results from the phase 3 ATLAS-PPX study (NCT03549871) comparing fitusiran prophylaxis versus prior prophylaxis with clotting factor concentrates (CFC) or bypass agents (BPA) in PwHA/B with or without inhibitors. Methods: This multicenter, multinational, open-label, Phase 3 study recruited male patients aged ≥12 years with severe hemophilia A or B, with or without inhibitors. Participants continued factor/BPA prophylaxis (6 months between month -6 and Day 0) before switching to once-monthly 80 mg SC fitusiran prophylaxis (7 months between Day 1 and month 7). Aside from annualized bleeding rates (ABR), the study assessed HRQoL at Month -6, Day 1 (baseline) and Month 7, during factor/BPA prophylaxis and during treatment with fitusiran using the following questionnaires: Hemophilia Quality of Life Questionnaire (Haem-A-QoL & Haemo-QoL; lower scores = improved HRQoL), Hemophilia Activity List (HAL & PedHal; range 0-100, higher scores = improved HRQoL), EuroQoL-5 Dimension 5-Level (EQ-5D-5L; index score ranges 0-1, higher scores = improved HRQoL), Treatment Satisfaction Questionnaire for Medication (TSQM-9), and the Hemophilia Joint Health Score (HJHS). Results: A total of 80 participants were enrolled and 65 were evaluable for efficacy analyses (inhibitor/non-inhibitor, n=19/46; hemophilia A/hemophilia B, n=50/15). At enrollment (Month -6), the mean (SD) age was 24.8 (11.2) years, transformed Haem-A-QoL physical health and total scores were 32.87 (23.68) and 31.63 (18.54) respectively, EQ-5D-5L index score was 0.83 (0.13), HAL total score was 79.11 (20.29) and TSQM-9 baseline score for effectiveness, convenience and satisfaction were 66.76 (18.06), 61.29 (17.97) and 69.35 (15.91), respectively. Fitusiran achieved the study primary endpoint and significantly reduced the frequency of bleeding episodes; observed median (IQR) ABR for treated bleeds was 0.0 (0.0; 2.3) in the fitusiran efficacy period and 4.35 (2.2; 10.9) in the factor /BPA prophylaxis period. The least squares (LS) mean difference between change from Month -6 to baseline (Day 1) and change from Month -6 to Month 7 in Haem-A-QoL scores demonstrated significant improvement in transformed total score with fitusiran treatment vs factor/BPA prophylaxis (-4.55 [95% CI: -7.56, -1.54]) and nominal improvement in physical health score in favor of fitusiran (-3.60 [95%-CI: -10.52, 3.33]). In addition to showing a good functional ability to perform activities of daily living, patients rated an even better physical ability over the two periods of assessment of the study as evidenced by changes in HAL scores: mean (SD) changes in total score were 3.05 (20.04) and 2.45 (19.42) for fitusiran and factor/BPA prophylaxis, respectively. Similar to the results from disease specific questionnaire, fitusiran also demonstrated nominal improvements in the EQ-5D-5L index score versus factor/BPA prophylaxis, with mean (SD) changes from Month -6 to Month 7/ Day 1of 0.04 (0.13) versus 0.0 (0.13). Overall results of the mean (SD) TSQM-9 score consistently favored fitusiran prophylaxis versus factor/BPA prophylaxis in all three domains of effectiveness, convenience, and global satisfaction (Table 1). Conclusions: Associated with a marked ABR reduction, PwHA/B with or without inhibitors on fitusiran prophylaxis consistently improved all HRQoL endpoints, demonstrating added value in PwHA/B beyond the improvement in bleeding phenotype. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Radiotherapy for elderly patients with glioblastoma: an assessment of hypofractionation and modern treatment techniques.

Glioblastoma (GBM) is a disease with a poor prognosis. For decades, radiotherapy has played a critical role in the management of GBM. The standard of care radiation prescription is 60 Gy in 30 fractions, but landmark trials have historically excluded patients older than 70 years. Currently, there is considerable variation in the management of elderly patients with GBM. Shortened radiation treatment (hypofractionated) regimens have been explored since conventional treatment schedules are lengthy and many elderly patients have functional, cognitive, and social limitations. Clinical trials have demonstrated the effectiveness of hypofractionated radiotherapy (40 Gy in 15 fractions) to treat elderly or frail patients with GBM. Although previous studies have suggested these unique hypofractionation prescriptions effectively treat these patients, there are many avenues for improvement in this patient population. Herein, we describe the unique tumor biology of glioblastoma, key hypofractionated radiotherapy studies, and health-related quality of life (HRQOL) studies for elderly patients with GBM. Hypofractionated radiation has emerged as a shortened alternative and retrospective studies have suggested survival outcomes are similar for elderly patients with GBM. Prospective studies comparing hypofractionation with conventional treatment regiments are warranted. In addition to evaluating survival outcomes, HRQOL endpoints should be incorporated into future studies.

Determining the relationship between preoperative mental health scores and postoperative outcomes in adult spinal deformity surgeries.

The objective of this study was to examine the relationship between the mental health domain of the refined 22-item Scoliosis Research Society Outcome Questionnaire (SRS) and various postoperative outcome measures in the adult spinal deformity (ASD) population. Given the scale and involved nature of deformity surgery, some surgeons have proposed that preoperative mental health scores (MHSs) may assist in screening out poor surgical candidates. In this study, the authors aimed to further assess the SRS MHS as a preoperative metric and its association with postoperative outcomes and to comment on its potential use in patient selection and optimization for ASD surgery. The authors conducted a retrospective study of 100 consecutive patients who had undergone primary or revision ASD surgery at a single academic institution between 2015 and 2019. Each patient had a minimum 2-year follow-up. Patients were categorized on the basis of their baseline mental health per the SRS mental health domain, with a score < 4 indicating low baseline mental health (LMH) and a score ≥ 4 indicating high baseline mental health (HMH). Baseline and follow-up SRS and Oswestry Disability Index scores, surgical procedures, lengths of stay, discharge locations, intraoperative or postoperative complications, and other outcome metrics were then compared between the HMH and LMH groups, as well as these groups stratified by an age ≤ 45 and > 45 years. Among patients aged ≤ 45 and those aged > 45, the LMH group had significantly worse baseline health-related quality-of-life (HRQOL) metrics in nearly all domains. The LMH group also had an increased median estimated blood loss (EBL; 1200 vs 800 ml, p = 0.0026) and longer average surgical duration (8.3 ± 2.8 vs 6.9 ± 2.6 hours, p = 0.014). Both LMH and HMH groups had significant improvements in nearly all HRQOL measures postoperatively. Despite their worse preoperative HRQOL baseline, patients in the LMH group actually improved the most and reached the same HRQOL endpoints as those in the HMH group. While patients with lower baseline MHSs may require slightly longer hospital courses or more frequent discharges to rehabilitation facilities, these patients actually attain greater absolute improvements from their preoperative baseline and surprisingly have the same postoperative HRQOL metrics as the patients with high MHSs, despite their poorer starting point. This finding suggests that patients with LMH may be uniquely positioned to substantially benefit from surgical intervention and improve their HRQOL scores and thus should be considered for ASD surgery to an extent similar to patients with HMH.

P663: HEALTH-RELATED QUALITY OF LIFE OUTCOMES ASSOCIATED WITH ZANUBRUTINIB VS IBRUTINIB MONOTHERAPY IN PATIENTS WITH RELAPSED/REFRACTORY (RR) CLL/SLL: RESULTS FROM THE RANDOMIZED PHASE 3 ALPINE TRIAL

Background: In the phase 3 ALPINE trial (BGB-3111-305; NCT03734016), efficacy and safety of zanubrutinib, a highly selective, next-generation Bruton tyrosine kinase inhibitor (BTKi), were compared with the first-generation BTKi, ibrutinib, in adult patients with RR chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). Aims: This abstract, based on the interim analysis of the ALPINE trial, describes the effects of zanubrutinib monotherapy and ibrutinib monotherapy on the health-related quality of life (HRQoL). Methods: Health-related quality of life was examined by patient-reported outcomes (PROs) measures assessed by EORTC QLQ-C30 and EQ-5D-5L at baseline, Cycle 1, and then every 3rd cycle until end of treatment. Key PRO endpoints included global health status (GHS), physical and role functions, and fatigue, pain, diarrhea, and nausea/vomiting. Descriptive analysis on all the scales was conducted as was a mixed model repeated-measure (MMRM) analysis of the longitudinal QLQ-C30 data. Data presented are from key cycles (7 and 13), corresponding to 6 and 12 months of treatment, respectively. Results: In the intent-to-treat population (N=652; zanubrutinib, n=327; ibrutinib, n=325), adjusted completion rates were high (>85%) in both arms at Cycles 7 and 13. On the QLQ-C30, estimated mean treatment differences and 95% CI in key PRO endpoints demonstrated treatment differences, in favor of zanubrutinib, in GHS, physical functioning, and fatigue in Cycle 7, and diarrhea in Cycle 13 (Table). Mean change from baseline (SD) in EQ-5D-5L VAS showed consistently more improvement with zanubrutinib compared with ibrutinib at both Cycle 7 (8.4 [18.2] vs 4.0 [16.6]) and Cycle 13 (6.8 [18.8] vs 5.2 [17.5]). Image:Summary/Conclusion: In the ALPINE trial, patients with RR CLL/SLL who received zanubrutinib monotherapy reported improvements in key HRQoL endpoints compared with patients who received ibrutinib monotherapy.

Health-related quality of life (HRQoL) with enasidenib versus conventional care regimens in older patients with late-stage mutant-IDH2 relapsed or refractory acute myeloid leukemia (R/R AML).

7032 Background: Enasidenib (ENA) is an oral inhibitor of mutant-IDH2 (mIDH2) proteins. In the phase 3 IDHENTIFY trial, ENA improved event-free survival (EFS), overall response, and complete remission rate vs conventional care regimens (CCR) ( P &lt; 0.01 for all) in patients (pts) ≥ 60 years of age with m IDH2 R/R AML with 2 or 3 prior treatments (Tx) (DiNardo 2021). Pt-reported HRQoL was a secondary trial endpoint. Methods: IDHENTIFY is an open-label, randomized trial (NCT02577406). Pts were preselected to a CCR (SC azacitidine, intermediate- or low-dose Ara-C, or supportive care) and then randomized 1:1 to ENA 100 mg/d or CCR in 28-d cycles. Key HRQoL endpoints were mean changes from baseline (CFB) overall and by clinical response in the Global Health Status/QoL, Physical Functioning, Role Functioning, Fatigue, and Dyspnea domains of the EORTC QLQ-C30 questionnaire, and in EQ-5D-5L utility index (UI) and visual analogue scale scores. The QLQ-C30 and EQ-5D-5L were assessed on D1 of each Tx cycle (C) and at end of Tx. Minimally important differences (MIDs) in CFB scores within or between Tx arms were based on accepted thresholds. Sensitivity analysis using imputed data on CFB was conducted using pattern mixture modeling. Results: HRQoL-evaluable cohorts included 118/158 (74.7%) pts in the ENA arm and 80/161 (49.7%) in the CCR arm; 40 ENA pts and 81 CCR pts were not evaluable due to missing data at baseline (BL; 22 ENA and 51 CCR) and/or at ≥1 post-BL visit (26 ENA and 69 CCR). Pts ineligible for HRQoL analyses had lower response rates and worse EFS and overall survival than HRQoL-evaluable pts. Overall QLQ-C30 completion rates in the ENA and CCR arms were 79% and 65%, respectively ( P &lt; 0.001). While there was no meaningful improvement or worsening from BL (ie, exceeding MID) within either Tx arm in the key QLQ-C30 domains, scores worsened during initial Tx cycles and then improved with continued Tx. Mean EQ-5D-5L scores also worsened during early cycles in both Tx arms, with meaningful UI deterioration in the ENA arm from C2 through C7. However, between-group comparisons showed no consistent differences between ENA and CCR in mean CFB. Sensitivity analysis with imputation of missing CFB data showed worsened HRQoL compared with non-imputed data in the CCR arm but not with ENA. In the ENA arm, clinical responders reported relatively stable mean HRQoL scores over time, and non-responders showed no meaningful differences in CFB vs the CCR arm. Conclusions: HRQoL measures tended to worsen during early cycles in both Tx arms and gradually improved with continued Tx. Data should be interpreted with caution as only approximately one-half of pts in the CCR arm were HRQoL-evaluable. ENA improved clinical efficacy measures vs CCR without compromising HRQoL in older pts with R/R AML. Clinical trial information: NCT02577406.

Health-related quality of life (HRQoL) in previously untreated patients with advanced renal cell carcinoma (aRCC): CheckMate 9ER updated results.

323 Background: In CheckMate 9ER, patients (pts) with aRCC were randomized 1:1 to nivolumab 240 mg IV Q2W + cabozantinib 40 mg PO QD (N+C; n = 323) or sunitinib (S) 50 mg PO (4 weeks of 6-week cycles; n = 328). As reported previously, at a median follow-up of 18.1 months, N+C led to superior progression-free and overall survival and more favorable HRQoL than S. We report results of HRQoL analyses for 32.9-months follow-up. Methods: Disease-related symptoms (DRS) were evaluated using the FKSI-19 instrument and HRQoL with the EQ-5D-3L visual analogue scale (VAS). Change from baseline (BL) was assessed using mixed-model repeated measures (MMRM), adjusting for baseline scores and stratification factors, and included all common timepoints (at BL and every 6 wks) through week 151. Time to deterioration analysis was conducted for first (TFD), confirmed (TCD) and definitive (TDD) deterioration events using Kaplan-Meier estimates and Cox proportional hazards models. Bother with treatment side effects (item GP5 in FKSI-19) was assessed using a generalized estimating equations model with response dichotomized as minimal bother (“not at all” or “a little bit”) vs notable bother (“somewhat”, “quite a bit”, and “very much”). HRQoL endpoints were exploratory and P-values are nominal and descriptive. Results: Group mean scores over time show that HRQoL was maintained or improved from BL with N+C while reductions were observed with S. Overall changes from BL though week 151 favored N+C with nominal significant differences between treatments observed for all scores except functional well-being (FWB; Table). Similarly, decreased risk of deterioration was observed with N+C vs S for all scores except FWB, irrespective of the deterioration definition used (TFD, TCD, TDD). Pts in N+C arm were 48% (odds ratio: 0.52 [95% CI, 0.35-0.77]) less likely to be notably bothered by side effects than pts in S arm. Conclusions: Pts continued to report improved HRQoL with N+C compared to S. Treatment with N+C reduced the risk of meaningful deterioration in HRQoL scores, regardless of the definition of deterioration and showed a decreased risk of being bothered by treatment side effects in contrast to S. These results suggest that the superior efficacy of N+C over S with the additional benefit of improved HRQoL is maintained with longer follow-up. Clinical trial information: NCT03141177. [Table: see text]

Patient-reported health-related quality of life from a randomized phase II trial comparing standard-dose with high-dose twice daily thoracic radiotherapy in limited stage small-cell lung cancer

ObjectivesIn a randomized phase II trial, twice daily (BID) thoracic radiotherapy (TRT) of 60 Gy/40 fractions improved survival compared with 45 Gy/30 fractions in limited stage small-cell lung cancer (LS SCLC). Notably, the higher dose did not cause more toxicity. Here we present health related quality of life (HRQoL) reported by the trial participants during the first 2 years. Materials and methods170 patients were randomized 1:1 to TRT of 45 Gy or 60 Gy concurrently with cisplatin/etoposide chemotherapy. The 150 patients who commenced TRT and completed a minimum of one HRQoL-questionnaire were included in the present study. Patients reported HRQoL on the European Organization for Research and Treatment of Cancer Core 30 and Lung Cancer 13 Quality of Life Questionnaires. Questionnaires were completed weeks 0, 4 (before TRT), 8 (end of TRT), 12 (response evaluation after chemoradiotherapy) and 16 (end of prophylactic cranial irradiation), then every 10 weeks year one, and every 3 months year two. Primary HRQoL endpoints were dysphagia and dyspnea. A difference in mean score of ≥10 was defined as clinically significant. ResultsMaximum dysphagia was reported on week 8, with no significant difference between treatment arms (mean scores 45 Gy: 44.2, 60 Gy: 51.1). The 60 Gy arm had more dysphagia in the convalescence period, but dysphagia scores returned to baseline levels at week 16 in both arms. For dyspnea there were no significant changes, or differences between treatment arms, at any timepoint. There were no significant differences between treatment arms for any other HRQoL-scales. ConclusionTRT of 60 Gy did not cause significantly higher maximum dysphagia, though patients on the 60 Gy arm reported more dysphagia the first 8 weeks of convalescence. The higher dose was well tolerated and is an attractive alternative to current TRT schedules in LS SCLC.Trial reg Clinicaltrials.gov NCT0204184.

Health-Related Quality of Life Assessment in Patients with Myelodysplastic Syndromes: Evidence from Randomized Clinical Trials

Myelodysplastic syndromes (MDS) are characterized by ineffective hematopoiesis and blood cytopenia with a variable risk of progression to acute myeloid leukemia. The main goal of therapy for the large majority of patients is to improve health-related quality of life (HRQoL). Its rigorous assessment is now recommended in international MDS guidelines. Our review provides an overview of HRQoL results from randomized controlled trials (RCTs) in MDS patients. The literature search undertaken in PubMed identified 10 RCTs with HRQoL endpoints (all secondary) published between August 2008 and September 2020. These RCTs have helped to better understand the impact of therapies from the patient perspective and have generated valuable information that can be used to further support clinical decisions. However, the number of RCTs in MDS patients, including HRQoL endpoints, is still low. Given the importance of symptom relief and HRQoL improvement in the treatment of MDS patients, the assessment of the patient perspective in future RCTs is highly recommended to keep expanding the knowledge of the impact of new MDS therapies.