scholarly journals Correlation between MPN-SAF TSS and EORTC QLQ-C30 Scores in Patients with PV: Data from the Reveal Study

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 2259-2259 ◽  
Author(s):  
Ivy Altomare ◽  
Aaron T. Gerds ◽  
David Lessen ◽  
Philomena Colucci ◽  
Shreekant Parasuraman ◽  
...  
Keyword(s):  
Health Status ◽  
Global Health ◽  
Research Funding ◽  
Symptom Burden ◽  
Global Health Status ◽  
Employment Equity ◽  
Eortc Qlq C30 ◽  
Equity Ownership ◽  
Eortc Qlq

Abstract Introduction Polycythemia vera (PV) is characterized by clonal proliferation of myeloid cells and erythrocytosis. Patients with PV often present with symptoms or develop symptoms that may negatively impact quality of life (QOL). In clinical trials, the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) and European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire C30 (EORTC QLQ-C30) have both been used to assess symptom burden in patients with PV. This analysis was conducted in patients with PV enrolled in REVEAL, a multicenter, prospective, observational trial, in an attempt to corroborate previous work by Emanuel et al (J Clin Oncol 2012;30:4098), which demonstrated associations between the MPN-SAF TSS and EORTC QLQ-C30. Methods Patients ≥ 18 years of age with PV were enrolled and followed during usual care visits for ≤ 36 months. Patient-reported outcomes, including the MPN-SAF TSS and EORTC QLQ-C30, were collected at enrollment and at approximate 3-month intervals; only the forms completed at the time of enrollment were included in this analysis. MPN-SAF TSS items are scored on a linear analog scale ranging from 0 (absent) to 10 (worst imaginable), and individual symptom scores were added together to calculate a TSS; higher scores represent worse symptom burden. In the EORTC QLQ-C30, 28 questions are scored using a 4-point scale indicating frequency: 1 (not at all), 2 (a little bit), 3 (quite a bit), and 4 (very much); this includes 6 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial difficulties). Two questions on overall health and QOL are rated on a 1 (very poor) to 7 (excellent) scale. Five multi-item functional scales (physical, role, cognitive, emotional, and social), 3 multi-item symptom scales (fatigue, nausea/vomiting, and pain), and a multi-item global health status/QOL scale are derived from the 30 questions. Linear transformation to 0-100 was applied to raw scores to obtain scores for each scale or single item. Higher scores for functional scales and global health status represent higher functioning and better health status/QOL, respectively. Higher scores for symptom scales/items represent higher symptom burden. Pearson correlation coefficient was used to assess correlations between MPN-SAF TSS and EORTC QLQ-C30 scales. Results As of data cutoff (April 30, 2018), 2,298 of 2,510 enrolled patients (91.6%) had completed both MPN-SAF TSS and EORTC QLQ-C30 forms at enrollment. Median age was 67 years (range, 22-97 years), 54.0% were male, and 89.7% were Caucasian. Median disease duration at the time of enrollment was 4.1 years. The majority (52.5%) of patients were treated with hydroxyurea (28.7%) or hydroxyurea with phlebotomy (23.8%). The mean MPN-SAF TSS was 18.7 (out of 100) compared to 21.8 reported by Emanuel et al 2012. The 4 symptoms with the highest mean scores were fatigue (3.5), early satiety (2.6), inactivity (2.5), and itching (2.3). The QLQ-C30 mean scores for overall QOL and health were 5.5 and 5.3, respectively. EORTC QLQ-C30 symptom scales were highest for fatigue (29.9), insomnia (28.7), and pain (20.0). Correlation between MPN-SAF TSS and EORTC QLQ-C30 results showed stronger associations between multiple items (Table). Calculated TSS had the strongest association with fatigue (r = 0.72), pain (r = 0.59), cognitive functioning (r = -0.58), and emotional functioning (r = -0.58). Problems with concentration in the MPN-SAF TSS was moderately correlated with cognitive functioning (r = -0.70) in the EORTC QLC-C30. Fatigue assessments were also moderately correlated (r = 0.65) between the MPN-SAF TSS and EORTC QLQ-C30. Conclusions In this analysis of prospectively gathered real-world data, the MPN-SAF TSS results confirm that patients with PV experience a recognizable constellation of symptoms, including fatigue, early satiety, inactivity, and itching. Not surprisingly, PV-related symptoms have a negative impact on QOL. There were moderate correlations (r = 0.5-0.75) between the MPN-SAF TSS and the EORTC QLC-C30 with respect to global health status/QOL, the 5 functional scales, and fatigue, pain, and dyspnea. Consistent with the previous analysis, this analysis provides further evidence that the MPN-SAF TSS represents an accurate, yet simple tool to assess PV-related symptoms and their potential impact on QOL. Disclosures Altomare: Novartis: Consultancy; Incyte: Consultancy; Amgen: Consultancy; Bayer: Consultancy; Genentech: Consultancy; Celgene: Other: Advisory Board Member; Ipsen: Other: Advisory Board Member. Gerds:Celgene: Consultancy; Apexx Oncology: Consultancy; Incyte: Consultancy; CTI Biopharma: Consultancy. Lessen:Abbvie: Honoraria; Teva: Honoraria, Speakers Bureau; Incyte: Honoraria, Research Funding, Speakers Bureau; Astellas: Research Funding; Bayer: Honoraria, Speakers Bureau; Amgen: Honoraria, Speakers Bureau; Portola: Honoraria, Speakers Bureau; Janssen: Research Funding. Colucci:Incyte: Employment, Equity Ownership. Parasuraman:Incyte: Employment, Equity Ownership. Paranagama:Incyte: Employment, Equity Ownership. Mesa:Pfizer: Research Funding; Incyte Corporation: Research Funding; Gilead: Research Funding; Promedior: Research Funding; NS Pharma: Research Funding; Celgene: Research Funding; Novartis: Consultancy; UT Health San Antonio - Mays Cancer Center: Employment; CTI Biopharma: Research Funding; Genentech: Research Funding.

Changes in Quality of Life and Disease-Related Symptoms in Patients with Polycythemia Vera Receiving Ruxolitinib or Best Available Therapy: RESPONSE Trial Results

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 709-709
Author(s):  
Ruben Mesa ◽  
Srdan Verstovsek ◽  
Jean-Jacques Kiladjian ◽  
Martin Griesshammer ◽  
Tamas Masszi ◽  
...  
Keyword(s):  
Global Health ◽  
Board Of Directors ◽  
Research Funding ◽  
Symptom Burden ◽  
Global Health Status ◽  
Employment Equity ◽  
Advisory Committees ◽  
Eortc Qlq C30 ◽  
Equity Ownership ◽  
Eortc Qlq

Abstract Background : Polycythemia vera (PV) is a myeloproliferative neoplasm driven by JAK/STAT activation and is associated with erythrocytosis and a broad symptom burden that may negatively impact patient quality of life (QoL). Hydroxyurea (HU) is often used as first-line therapy for high-risk patients but may not effectively control or reduce symptom burden. RESPONSE is a phase III trial comparing ruxolitinib (RUX) with best available therapy (BAT) in patients with PV who were intolerant of or resistant to HU according to modified European LeukemiaNet (ELN) criteria. The primary study endpoint (a composite of hematocrit control and ≥35% spleen volume reduction at Week 32) was achieved by 21% of patients in the RUX arm vs 1% in the BAT arm (P<0.0001); 77% of patients in the RUX arm achieved at least one component of the primary endpoint. The current analysis was conducted to evaluate the effect of RUX on PV-related symptoms and QoL measures in the RESPONSE trial. Methods : Patients with PV aged ≥18 years, resistant to or intolerant of HU (modified ELN criteria) with splenomegaly, and who required phlebotomy for hematocrit control were randomized 1:1 to receive open-label RUX 10 mg twice daily (BID) or BAT (administered based on investigator judgment). Dose adjustments were permitted (RUX, 5-mg BID increments [25 mg BID max]; BAT was adjusted per investigator judgment). Objectives of this analysis included assessment of improvement in symptom burden as assessed by patient-reported outcomes using the 14-item modified Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF), the Pruritus Symptom Impact Scale (PSIS), the European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30), and the Patient Global Impression of Change. The 14-item MPN-SAF (graded from 0 [absent] to 10 [worst imaginable]) comprised symptoms related to cytokines (tiredness, itching, muscle ache, night sweats, and sweats while awake), hyperviscosity (vision problems, dizziness, concentration problems, headache, numbness/tingling, ringing in ears, and skin redness), and splenomegaly (abdominal discomfort and early satiety). Changes in total symptom score (TSS; maximum score = 140) and individual symptom scores from baseline to Week 32 were summarized by treatment group. For the EORTC QLQ-C30 Global Health Status/QOL score, the percentage of patients with a minimally important difference (MID) from baseline (10-point change) at Week 32 was summarized. Results : Overall, 222 patients were randomized (RUX, 110; BAT, 112). Median age (range) was similar between arms (RUX, 62.0 [34.0–90.0]; BAT, 60.0 [33.0–84.0]); the RUX and BAT arms were 60% and 71% male, respectively. At Week 32, a higher proportion of patients in the RUX vs the BAT arms had a ≥50% improvement in MPN-SAF TSS (49% vs 5%, respectively) and MPN-SAF symptom cluster scores (cytokine, 64% vs 11%; hyperviscosity, 37% vs 13%; splenomegaly, 62% vs 17%). Median percentage changes in individual symptom scores are presented in the Table. Mean changes from baseline at Week 32 on the 5 items of the PSIS indicated that the severity of pruritus and its interference on daily life improved with RUX (range, −1.5 to −2.2) and was unchanged/worsened with BAT (range, −0.1 to 0.3). Treatment with RUX vs BAT was associated with improved mean changes from baseline at Week 32 on EORTC QLQ-C30 symptom subscales, functional subscales, and Global Health Status/QOL (Table); 46% of RUX patients versus 10% of BAT patients achieved an MID in Global Health Status/QOL (Figure). At Week 32, RUX patients were more likely to rate their global impression of symptom changes as “very much improved” or “much improved” (67%) vs BAT patients (13%). Conclusion : In patients with PV who were resistant to or intolerant of HU, treatment with RUX was associated with greater and clinically meaningful improvements in PV-related symptom burden and QoL measures compared with BAT. Figure 1 Figure 1. Figure 2 Figure 2. Disclosures Mesa: Incyte Corporation: Research Funding; CTI: Research Funding; Gilead: Research Funding; Genentech: Research Funding; Eli Lilly: Research Funding; Promedior: Research Funding; NS Pharma: Research Funding; Sanofi: Research Funding; Celgene: Research Funding. Off Label Use: Ruxolitinib is a JAK1/JAK2 inhibitor approved for the treatment of patients with intermediate or high-risk myelofibrosis, including primary myelofibrosis, post polycythemia vera myelofibrosis, and post-essential thrombocythemia myelofibrosis. Verstovsek:Incyte Corporation: Research Funding. Kiladjian:Novartis Pharmaceuticals: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding. Masszi:Novartis Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees. Durrant:Novartis Pharmaceuticals: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding. Harrison:Novartis Pharmaceuticals: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding. He:Incyte Corporation: Employment, Equity Ownership. Jones:Incyte Corporation: Employment, Equity Ownership. Parasuraman:Incyte Corporation: Employment, Equity Ownership. Li:Novartis Pharmaceuticals: Employment, Equity Ownership. Côté:Novartis Pharmaceuticals: Employment, Equity Ownership. Habr:Novartis Pharmaceuticals: Employment, Equity Ownership. Vannucchi:Novartis Pharmaceuticals: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding.

scholarly journals Patient-Reported Outcomes (PRO) Data from Patients (Pts) with Essential Thrombocythemia (ET) Enrolled in the MOST Study

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 1665-1665
Author(s):  
Ellen K. Ritchie ◽  
Anas Al-Janadi ◽  
Philomena Colucci ◽  
Patricia Kalafut ◽  
Dilan Paranagama ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Global Health ◽  
Research Funding ◽  
Symptom Burden ◽  
Advisory Board ◽  
Employment Equity ◽  
Scale Scores ◽  
Equity Ownership ◽  
Eortc Qlq

Introduction: ET is a chronic myeloproliferative neoplasm (MPN) characterized by thrombocytosis and an increased risk for thrombotic and hemorrhagic events. ET can be associated with substantial symptom burden, impaired quality of life (QoL), and reduced survival. PRO data pertaining to the impact of ET on QoL and symptom burden in these pts are limited. The ongoing Myelofibrosis and Essential Thrombocythemia Observational STudy (MOST) was designed to collect data about the demographics, disease burden, PROs, and management of pts with ET or myelofibrosis (MF) in clinical practices throughout the United States. This analysis describes PROs from pts with ET enrolled in MOST. Methods: MOST is a longitudinal, multicenter, noninterventional, prospective, observational study (NCT02953704). Eligible adults with ET were ≥60 years of age, had a history of thrombotic events, or were receiving ET-directed therapy. PROs were collected in conjunction with usual-care visits approximately every 6 months over a planned observation period of 36 months. Patient-reported symptom burden was assessed with the disease-specific MPN Symptom Assessment Form Total Symptom Score (MPN-SAF TSS), composed of 10 items (fatigue, early satiety, abdominal discomfort, inactivity, concentration problems, night sweats, itching, bone pain, fever [>100oF], weight loss). The MPN-SAF numbness/tingling item was also included in the questionnaire but was not included in the TSS calculation. Symptom severity was graded from 0 (absent) to 10 (worst imaginable), with a possible TSS ranging from 0 to 100. Health-related QoL was evaluated with the European Organization for Research and Treatment of Cancer Core Quality of Life Questionnaire (EORTC QLQ-C30 v3.0), composed of 5 functional scales, 3 symptom scales, 6 additional single-symptom items, and a global health/QoL scale. For functional and global health/QoL scales, higher scores indicate higher functioning and better global health/QoL, respectively. For symptom scales/items, higher scores indicate greater symptom burden. High-risk pts and low-risk pts receiving ET-directed therapy (excluding aspirin only) with baseline PRO data were included in this analysis. Data were summarized with descriptive statistics. Results: The MOST study enrolled 1234 pts with ET between Nov 29, 2016 and March 29, 2019 at 124 sites. Of these pts, 794 qualified for this analysis (data cut-off date, June 17, 2019); median age was 70 (range, 19-93) years, 80% were ≥60 years of age, 68% were women, 90% were white, 42% were working full or part-time, and 4% had a documented family history of MF, ET, or polycythemia vera. The majority of pts (87%) had high-risk ET. At enrollment, 768 pts completed the MPN-SAF. Mean (SD) TSS was 17.1 (15.6); 33% of pts had TSS ≥20. Women had higher mean (SD) TSS than men (18.5 [15.8] vs 14.2 [14.9]) and had higher mean individual symptom scores, except for weight loss and fever. The highest mean (SD) individual symptom scores were fatigue (3.4 [2.7]), numbness/tingling (2.3 [3.0]), inactivity (2.3 [2.8]), and early satiety (2.3 [2.7]) (Fig A). The most frequently reported severe symptoms (ie, score ≥7) were fatigue (17% [127/746]), numbness/tingling (14% [107/767]), and inactivity (11% [86/762]). At enrollment, 794 pts completed the EORTC QLQ-C30. The highest mean (SD) symptom scale scores (score ≥15) were fatigue (29.6 [25.8]), insomnia (28.6 [30.6]), pain (22.1 [27.9]), dyspnea (17.2 [25.5]), and constipation (15.7 [25.2]) (Fig B). The mean (SD) global health status/QoL score was 72.7 (21.9); functional scores ranged from 79.9 (21.9) for emotional functioning to 85.2 (24.1) for social functioning (Fig C). The average functional scale scores and symptom scale scores indicate higher functioning and less symptom burden, respectively, in men vs women. Conclusion: Pts with ET experienced a high symptom burden; fatigue was the most common and highest in severity. Symptom burden and quality of life scores in the current study were similar to prior reports (Emanuel J Clin Oncol 2012; Scherber Blood 2011). Women reported higher symptom burden than men in both the MPN-SAF and EORTC QLQ-30. Of note, numbness/tingling, which is not included in the MPN-SAF TSS calculation, was one of the most frequently reported severe symptoms for pts with ET in MOST. Future analyses from this trial will continue to increase understanding of the symptom burden and its impact on QoL in pts with ET. Disclosures Ritchie: Celgene, Incyte, Novartis, Pfizer: Consultancy; Genentech: Other: Advisory board; Tolero: Other: Advisory board; Pfizer: Other: Advisory board, travel support; agios: Other: Advisory board; Celgene: Other: Advisory board; Jazz Pharmaceuticals: Research Funding; Celgene, Novartis: Other: travel support; AStella, Bristol-Myers Squibb, Novartis, NS Pharma, Pfizer: Research Funding; Ariad, Celgene, Incyte, Novartis: Speakers Bureau. Al-Janadi:Incyte: Honoraria, Other: Travel, Accommodations, Expenses, Research Funding; Celgene: Honoraria, Other: Travel, Accommodations, Expenses, Research Funding, Speakers Bureau; Genentech/Abbvie: Honoraria, Other: Travel, Accommodations, Expenses, Speakers Bureau; Genentech/Roche: Honoraria, Other: Travel, Accommodations, Expenses, Speakers Bureau; Gilead Sciences: Consultancy, Honoraria, Other: Travel, Accommodations, Expenses; Sandoz-Novartis: Consultancy, Honoraria; Alexion Pharmaceuticals: Consultancy, Honoraria, Other: Travel, Accommodation, Expenses, Research Funding, Speakers Bureau; Takeda: Consultancy, Honoraria, Other: Travel, Accommodations, Expenses, Research Funding, Speakers Bureau; MEI Pharma: Research Funding; Seattle Genetics: Consultancy, Honoraria, Other: Travel, Accommodations, Expenses. Colucci:Incyte: Employment, Equity Ownership. Kalafut:Incyte: Employment, Equity Ownership. Paranagama:Incyte: Employment, Equity Ownership. Mesa:Genotech: Research Funding; Promedior: Research Funding; Sierra Onc: Consultancy; Celgene: Research Funding; AbbVie: Research Funding; Novartis: Consultancy; La Jolla Pharma: Consultancy; CTI Biopharma: Research Funding; Samus: Research Funding; Incyte: Research Funding.

scholarly journals Disease Burden Subgroup Analysis of Health-Related Quality of Life of Blinatumomab Versus Standard-of-Care Chemotherapy in Patients with Relapsed or Refractory Philadelphia Chromosome-Negative B-Cell Precursor Acute Lymphoblastic Leukemia in a Randomized, Open-Label Phase 3 Study (TOWER)

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 3967-3967
Author(s):  
Anthony S. Stein ◽  
Zachary F. Zimmerman ◽  
Paul Cannell ◽  
Hervé Dombret ◽  
Johan Maertens ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Health Status ◽  
Global Health ◽  
Research Funding ◽  
Disease Burden ◽  
Employment Equity ◽  
Eortc Qlq C30 ◽  
Equity Ownership ◽  
Eortc Qlq ◽  
Baseline Hrqol

Abstract Background: In the phase 3 TOWER study, patients with relapsed or refractory (r/r) Philadelphia chromosome-negative (Ph-) B-cell precursor (BCP) acute lymphoblastic leukemia (ALL) who received bispecific T-cell engager (BiTE®) antibody construct blinatumomab had improved overall survival (OS; median, 7.7 vs 4.0 months; P=0.01;) and health-related quality of life (HRQoL) compared with those who received standard of care (SOC) chemotherapy (Kantarjian H, et al. N Engl J Med. 2017;376:836-847; Topp MS, et al. Blood. 2018;131:2906-2914). In this subgroup analysis of TOWER, we assessed the HRQoL between patients with low versus high baseline disease burden (low versus high bone marrow blast levels) who received blinatumomab or SOC chemotherapy. Methods: Patients (N=405) with r/r Ph- BCP ALL were randomized 2:1 to receive 2 cycles of induction blinatumomab by continuous intravenous infusion (n=271) or SOC (n=134). Those in remission could receive up to 3 consolidation cycles; 12 months of maintenance was allowed for those who received up to 3 consolidation cycles and had bone marrow response. HRQoL was assessed using the EORTC QLQ-C30 Questionnaire on days 1 (baseline), 8, and 15; on day 29 of cycle 1; days 1, 15, and 29 of consolidation; and at the safety follow-up. The questionnaire included 1 global health status scale, 5 functioning scales, 3 symptom scales, and 6 single-symptom items. For global health status and functioning scales, a higher score indicates better HRQoL; for symptom scales/items, a lower score indicates better HRQoL. A 10-point change was viewed as the minimum clinically important difference in EORTC QLQ-C30 (Zikos E, et al. EORTC. 2016). HRQoL was assessed in patient subgroups by screening the bone marrow aspirates for low blast levels (&lt;50% blasts) versus high blast levels (≥50% blasts). Although blast count was not a randomization stratification factor in TOWER, baseline HRQoL values were assessed for blinatumomab versus SOC in both subgroups; in the high blasts group, for blinatumomab versus SOC, the only differences were cognitive functioning and constipation scores, which were significantly higher, and for the diarrhea score, which was significantly lower. Between the subgroups, only physical functioning was significantly different (higher in the high blasts group). Analyses included patients with baseline and ≥1 postbaseline result of any multi-item scale or single-item measure. Mean change from baseline in scores for each scale/item were summarized for cycle 1. Time to deterioration (TTD) analyses assessed the treatment effect based on timing from the initiation of treatment to a 10-point deterioration from baseline. Results: In total, 342 patients (blinatumomab, n=247; SOC, n=95) had ≥1 HRQoL result: low blasts, n=87 (blinatumomab, n=64; SOC, n=23); high blasts, n=255 (blinatumomab, n=183; SOC, n=72). The EORTC QLQ-C30 analysis set included all randomized subjects with a nonmissing baseline result and at least 1 nonmissing post-baseline result of any EORTC QLQ-C30 scale/item. There was no statistically significant difference in baseline HRQoL scores between the high and low blasts groups; however, the high blasts group had worse HRQoL overall. Baseline HRQoL scores were also similar between blinatumomab arm and SOC arm for each group. Global health status was improved by blinatumomab regardless of baseline blast level; however, this effect was somewhat greater in the low blasts group. When the function scores worsened, the extent of worsening was almost always smaller for blinatumomab versus SOC, particularly in the high blasts group. Functioning status scores tended to stay the same or worsen with both blinatumomab and SOC regardless of blast level, except emotional scores, which improved with blinatumomab regardless of blast level (Figure 1). Symptom scores generally improved with blinatumomab but not with SOC, particularly in patients with high blasts (Figure 2). TTD analyses showed that hazard ratios favored blinatumomab over SOC, particularly in patients with high blasts (Table). Conclusions: Blinatumomab improved HRQoL in patients with r/r Ph- BCP ALL and delayed the time to clinically meaningful deterioration in HRQoL compared with SOC. The treatment effects of blinatumomab versus SOC on HRQoL were particularly larger among patients with high disease burden. Disclosures Stein: Amgen Inc.: Speakers Bureau; Celgene: Speakers Bureau. Zimmerman:Amgen Inc.: Employment, Equity Ownership. Dombret:Novartis: Consultancy, Honoraria, Research Funding; Agios: Consultancy, Honoraria; Sunesis: Consultancy, Honoraria; Ambit (Daiichi Sankyo): Consultancy, Honoraria; Karyopharm: Consultancy, Honoraria; Menarini: Consultancy, Honoraria; Astellas: Consultancy, Honoraria; Janssen: Consultancy, Honoraria; Servier: Consultancy, Honoraria; Seattle Genetics: Consultancy, Honoraria; Cellectis: Consultancy, Honoraria, Other: Travel expenses; Celgene: Consultancy, Honoraria, Other: Travel expenses, Speakers Bureau; Immunogen: Consultancy, Honoraria; Shire-Baxalta: Consultancy, Honoraria; Abbvie: Consultancy, Honoraria; Otsuka: Consultancy, Honoraria; Kite Pharma: Consultancy, Honoraria, Research Funding; Jazz Pharma: Consultancy, Honoraria, Research Funding; Ariad (Incyte): Consultancy, Honoraria, Other: Travel expenses, Research Funding, Speakers Bureau; Roche/Genentech: Consultancy, Honoraria; Pfizer: Consultancy, Honoraria, Research Funding, Speakers Bureau; Amgen: Consultancy, Honoraria, Other: Travel expenses, Research Funding, Speakers Bureau. Topp:F. Hoffmann-La Roche Ltd: Membership on an entity's Board of Directors or advisory committees, Research Funding; Regeneron Pharmaceuticals, Inc.: Honoraria, Research Funding; Amgen: Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Travel, Research Funding; Boehringer Ingelheim: Research Funding. Franklin:Amgen Inc.: Employment, Equity Ownership. Cong:Amgen, Inc.: Employment, Equity Ownership. Zhang:Amgen Inc.: Employment, Equity Ownership. Schuh:Pfizer: Consultancy; Novartis: Consultancy; Celgene: Consultancy; Teva: Consultancy; Amgen Inc.: Consultancy; Otsuka: Consultancy; Jazz: Consultancy; Shire: Consultancy.

FOENIX-CCA2 quality of life data for futibatinib-treated intrahepatic cholangiocarcinoma (iCCA) patients with FGFR2 fusions/rearrangements.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 4097-4097
Author(s):  
Juan W. Valle ◽  
Antoine Hollebecque ◽  
Junji Furuse ◽  
Lipika Goyal ◽  
Funda Meric-Bernstam ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Health Status ◽  
Global Health ◽  
Global Health Status ◽  
Phase 2 ◽  
Life Data ◽  
Eortc Qlq C30 ◽  
Eortc Qlq ◽  
Eq Vas

4097 Background: In FOENIX-CCA2 (NCT02052778), a pivotal phase 2 study among iCCA patients (pts) with FGFR2 fusions/rearrangements, the highly selective, irreversible FGFR1–4 inhibitor futibatinib demonstrated a confirmed objective response rate of 41.7%, with a 9.7-month median duration of response. Adverse events were manageable with dosing modifications that did not adversely impact on response. We report outcomes for the preplanned analysis of Patient-Reported Outcomes (PROs) during futibatinib treatment as a secondary objective of FOENIX-CCA2. Methods: Pts enrolled in FOENIX-CCA2 had locally advanced/metastatic unresectable iCCA with FGFR2 fusions/rearrangements, ≥1 prior line of therapy (including gemcitabine/cisplatin) and ECOG PS 0-1. Pts received oral futibatinib 20 mg continuous QD dosing per 21-day cycle. PRO measures included EORTC-QLQ-C30 (1 global health, 5 functional, 9 symptom scales), EQ-5D-3L, and EQ visual analogue scale (VAS). PROs were collected at screening, cycles 2 and 4, every 3 cycles thereafter, and end of treatment. PRO data were evaluated up to cycle 13, the last visit before data were missing for >50% of the PRO population (PRO primary assessment time point). Results: 92/103 (89.3%) pts enrolled had PRO completion data at baseline and a minimum of 1 follow-up assessment (median age 58 y, 56.5% female), with 48 pts having PRO data at cycle 13. At baseline, mean (SD) EORTC QLQ-C30 global health status score was 70.1 (19.4) and EQ VAS score 71.7 (20.3). Mean EORTC QLQ-C30 global health status scores were maintained from baseline to cycle 13, corresponding to 9.0 months on treatment, with no clinically meaningful (≥10-point) changes in individual functional measures (Table). EORTC QLQ-C30 scores across individual symptom measures were also stable from baseline through cycle 13; only constipation showed an average of 10.0-point worsening at only cycle 4. Mean EQ VAS scores were sustained from baseline to cycle 13 (mean change ranging -1.8 to +4.8 across cycles), with values maintained within the population norm range from across 20 countries. Conclusions: Quality of life data from the phase 2 FOENIX-CCA2 trial show that physical, cognitive and emotional functioning, and overall health status were maintained among pts with advanced iCCA receiving futibatinib. Clinical trial information: NCT02052778. [Table: see text]

Patient-Reported Quality of Life In Elderly, Newly Diagnosed Multiple Myeloma Patients Treated with Bortezomib-Based Regimens: Results From the Phase 3b UPFRONT Study

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 3026-3026 ◽  
Author(s):  
Ruben Niesvizky ◽  
Ian W. Flinn ◽  
Robert M. Rifkin ◽  
Nashat Y Gabrail ◽  
Veena Charu ◽  
...  
Keyword(s):  
Health Status ◽  
Global Health ◽  
Treatment Period ◽  
Research Funding ◽  
Global Health Status ◽  
Status Score ◽  
Patient Reported ◽  
Equity Ownership ◽  
Eortc Qlq ◽  
Health Status Score

Abstract Abstract 3026 Background: UPFRONT is an ongoing US community-based phase 3b study designed to compare the safety and efficacy of three bortezomib (Velcade®, Vc)-based regimens, Vc-dexamethasone (VcD), Vc-thalidomide-dexamethasone (VcTD), and Vc-melphalan-prednisone (VcMP), followed by Vc maintenance therapy, in previously untreated multiple myeloma (MM) patients who were ineligible for high-dose therapy and autologous stem cell transplantation (HDT-SCT). Efficacy data have been presented elsewhere; here we present patient-reported quality of life (QoL) data after 300 patients had the opportunity to undergo the entire 13-cycle treatment period (8 Vc-based induction cycles and 5 Vc maintenance cycles). Methods: Patients with symptomatic, measurable MM were randomized (1:1:1) to receive 49 weeks of therapy: 24 weeks (eight 21-day cycles) of induction with VcD, VcTD, or VcMP (VcD: Vc 1.3 mg/m2, days 1, 4, 8, 11; D 20 mg, days 1, 2, 4, 5, 8, 9, 11, 12 [cycles 1–4]), days 1, 2, 4, 5 [cycles 5–8]); VcTD: Vc 1.3 mg/m2, days 1, 4, 8, 11; T 100 mg/d, d1–21; D 20 mg, days 1, 2, 4, 5, 8, 9, 11, 12 [cycles 1–4]), days 1, 2, 4, 5 [cycles 5–8]); VcMP: Vc 1.3 mg/m2, days 1, 4, 8, 11; M 9 mg/m2, and P 60 mg/m2, day 1–4, every other cycle), followed by 25 weeks (five 35-day cycles) of maintenance with Vc alone (1.6 mg/m2, days 1, 8, 15, 22). Patient-reported QoL was recorded using the EORTC QLQ-C30 questionnaire, which assesses global health status, physical, role, cognitive, emotional, and social functions, fatigue, nausea/vomiting, pain, dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial difficulties. Patients completed the questionnaire prior to dosing on cycle 1, day 1 (baseline), prior to dosing on day 1 of every odd cycle, at the end of treatment visit, and every 12 weeks thereafter. Here we present updated study results after 300 patients had the opportunity to undergo the entire 13-cycle treatment period, focusing on change in global health status score over time; no adjustment was made for missing cases or patient deaths. Results: Patient baseline characteristics were well balanced across the three treatment arms. Patients in the VcD, VcTD, and VcMP arms had median ages of 73.5, 73.0, and 72.0 years, respectively; 85%, 64%, and 74% had ISS stage II/III; 22%, 27%, and 28% were non-Caucasian; and 51%/25%/24%, 55%/30%/14%, and 62%/24%/15% had Charlson comorbidity index 0/1/≥2 in the VcD, VcTD, and VcMP arms, respectively. Patients received a median of 9 (VcD), 6 (VcTD), and 7 (VcMP) treatment cycles (induction + maintenance); 56%, 33%, and 43% of patients, respectively, received Vc maintenance. In the VcD, VcTD, and VcMP arms, Vc dose intensity (mean ratio of doses received/doses planned) was 76%, 63%, and 69% during induction, and 73%, 77%, and 85% during maintenance, respectively. After 13 treatment cycles, the rates of grade ≥3 adverse events (AEs) were 74%, 86%, and 80% in the VcD, VcTD, and VcMP arms. The incidence of serious AEs was highest in the VcTD arm (61%, vs 57% and 51% with VcD and VcMP), as was the rate of study drug discontinuation due to AEs (41%, vs 29% and 35% with VcD and VcMP). Global health status score by cycle is shown in the Figure, with number of patients with available QoL data in each arm indicated below. In all three arms, for those patients with available QoL data, global health status score at the end of cycle 12 was greater than at baseline and at the end of cycle 8 (end of Vc-based induction therapy). In the induction phase, global health status score remained stable in the VcD arm and transiently decreased in the VcMP arm at cycle 4, before increasing to above baseline levels. A similar trend was seen for the VcTD arm; global health status score started decreasing at cycle 2 but took slightly longer to increase, possibly due to the increased incidence of AEs in the VcTD arm. Similar trends were seen for other EORTC QLQ-C30 function and symptom scores. Conclusions: Although there was some variability during the study, by the end of the treatment period patients who received one of the three Vc-based regimens reported improvements in QoL compared with baseline values. The study is ongoing and patients continue to be followed for assessment of patient-reported QoL and long-term outcomes. Updated QoL data, including the correlation between change in QoL and best response achieved, will be presented. Disclosures: Niesvizky: Celgene: Consultancy, Research Funding; Millennium Pharmaceuticals, Inc.: Consultancy, Research Funding; Onyx: Consultancy, Research Funding. Off Label Use: Discussion of Velcade in a novel combination in frontline myeloma is included. Flinn:Millennium Pharmaceuticals, Inc.: Research Funding. Rifkin:Millennium Pharmaceuticals, Inc.: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Celgene: Speakers Bureau; Amgen: Speakers Bureau; Cephalon: Speakers Bureau; Dendreon: Speakers Bureau. Gabrail:Millennium Pharmaceuticals, Inc.: Research Funding. Charu:Amgen: Equity Ownership, Research Funding; Pfizer: Equity Ownership; GSK: Equity Ownership, Research Funding; Lilly: Equity Ownership, Research Funding; Millennium Pharmaceuticals, Inc.: Research Funding; Roche: Research Funding; Bristol-Myers Squibb: Equity Ownership. Neuwirth:Millennium Pharmaceuticals, Inc.: Employment. Corzo:Millennium Pharmaceuticals, Inc.: Employment. Reeves:Celgene: Equity Ownership.

Health-Related Quality of Life and Symptoms in Myelofibrosis Patients Treated with Ruxolitinib Versus Best Available Therapy

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 795-795
Author(s):  
Claire N. Harrison ◽  
Jean-Jacques Kiladjian ◽  
Haifa Kathrin Al-Ali ◽  
Heinz Gisslinger ◽  
Laurent Knoops ◽  
...  
Keyword(s):  
Research Funding ◽  
Treatment Phase ◽  
Well Being ◽  
Employment Equity ◽  
Advisory Committees ◽  
Eortc Qlq C30 ◽  
Health Related ◽  
Equity Ownership ◽  
Eortc Qlq

Abstract Abstract 795FN2 Background: Ruxolitinib (INC424), a potent and selective oral JAK1 and JAK2 inhibitor, demonstrated rapid and durable reductions in splenomegaly and improved disease-related symptoms, role functioning, and quality of life (QoL) in 2 phase 3 studies in patients with myelofibrosis (MF) (the COMFORT studies). Per-protocol, patient-reported, health-related QoL (HRQoL) and symptoms analyses in the COMFORT-II study are limited. Here we report on additional post hoc analyses of these outcomes. The COMFORT-II study includes 219 patients (ruxolitinib, n=146; best available therapy [BAT], n=73). At entry, all patients were classified into intermediate 2-risk or high-risk prognostic groups (Cervantes F, et al. Blood, 2009;113(13):2895-2901) and had palpable splenomegaly ≥5 cm below the costal margin. Patients could have received prior therapy for MF. On BAT, doses and schedules or no therapy were selected by the investigator; therapy adjustments were permitted during the randomized treatment phase at the investigator's discretion. Patients in both arms continued in the randomized treatment phase as long as there was no protocol-defined disease progression. Methods: The European Organisation for the Research and Treatment of Cancer (EORTC) QoL Questionnaire–Core 30 (QLQ-C30) and Functional Assessment of Cancer Therapy–Lymphoma (FACT-Lym) questionnaires were assessed at baseline and weeks 8, 16, 24, and 48. EORTC QLQ-C30 consists of 30 items combined into 15 subscales (Global Health Status/QoL, 5 functioning subscales, and 9 symptom subscales; scores range from 0 to 100, and higher scores indicate better HRQoL and functioning but a worsening of symptoms). FACT-Lym consists of 42 items combined into 8 subscales (4 well-being subscales; 1 symptom subscale [LymS]; and 3 total scale scores: FACT-General [G], FACT-Lym trial outcome index [TOI], and FACT-Lym total; scores for the different subscales vary from 0–28 to 0–168, and higher scores indicate better outcomes). This analysis includes evaluable patients in the randomized treatment phase and assesses changes from baseline in HRQoL and MF symptom scores, including the EORTC subscales, LymS, and FACT total scores, which incorporate well-being and/or symptom subscales. Mixed-model analyses, adjusted for age, sex, baseline score, and prognostic risk category, are used to evaluate treatment differences at each time point and overall across time. Results: HRQoL and MF symptoms, on average, improved compared with baseline for patients receiving ruxolitinib, but remained the same or worsened for patients receiving BAT. Based on the EORTC QLQ-C30, the treatment differences in physical functioning, role functioning, fatigue, and appetite loss significantly favored ruxolitinib starting at week 8 (P <.05) and remained significant at week 48 (P <.05). The overall between-treatment differences (on average across time) in adjusted mean change from baseline for MF symptom scores (95% confidence interval) were: Fatigue, –10.2 (–15.9, –4.5; P <.001); Dyspnea, –11.6 (–17.6, –5.6; P <.001); Appetite loss, –16.3 (–21.5, –11.1; P <.0001); Insomnia, –9.8 (–16.7, –3.0; P <.01); and Pain, –9.0 (–14.9, –3.0), P <.01); negative values favor ruxolitinib. Global Health Status/QoL (Figure) was significantly improved in the ruxolitinib arm compared with the BAT arm at weeks 8, 16, and 48. Scores on the LymS, which includes symptoms of pain, swelling, fever, night sweats, itching, trouble sleeping, fatigue, weight loss, loss of appetite, trouble concentrating, and other patient concerns, also improved significantly during treatment (Figure). Additionally, FACT-G, FACT-Lym TOI, and FACT-Lym total scores were all significantly (P <.05) improved for patients receiving ruxolitinib treatment compared with BAT. Most EORTC QLQ-C30 and FACT-Lym scores improved significantly on ruxolitinib compared with BAT. The treatment effect between the high-risk and intermediate 2-risk prognostic groups was not significantly different based on an analysis of the risk group–by–treatment interaction. Conclusions: These additional analyses from the COMFORT-II study further support that ruxolitinib significantly improves overall HRQoL and MF symptoms compared with BAT. Disclosures: Harrison: Novartis: Honoraria; Incyte: Honoraria; S*Bio: Honoraria; Celgene: Honoraria; Sanofi Aventis: Honoraria. Kiladjian:Novartis: Honoraria; Celgene: Honoraria. Gisslinger:Novartis: Speakers Bureau; Celgene Austria: Research Funding, Speakers Bureau; Aop-Orphan: Speakers Bureau. Knoops:Novartis: Consultancy. Waltzman:Novartis: Employment. Mendelson:Novartis: Employment, Equity Ownership. Zhou:RTI-HS: Employment; Novartis: Research Funding. Copley-Merriman:RTI-HS: Employment; Novartis: Research Funding. Hunter:Incyte Corporation: Employment, Equity Ownership. Levy:Incyte Corporation: Employment, Equity Ownership. Cervantes:Bristol-Myers-Squibb: Speakers Bureau; Novartis: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Passamonti:Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees. Vannucchi:Novartis: Honoraria. Barosi:Novartis: Consultancy.

An Evaluation of Half-Body Irradiation in the Treatment of Widespread, Painful Metastatic Bone Disease

2008 ◽  
Vol 94 (6) ◽  
pp. 813-821 ◽  
Author(s):  
Leszek Miszczyk ◽  
Andrzej Tukiendorf ◽  
Aleksandra Gaborek ◽  
Jerzy Wydmański
Keyword(s):  
Quality Of Life ◽  
Health Status ◽  
Global Health ◽  
Pain Intensity ◽  
Observation Time ◽  
Global Health Status ◽  
Pain Level ◽  
Eortc Qlq C30 ◽  
Eortc Qlq

Aims Evaluation of analgesic uptake, pain intensity, and quality-of-life changes after half-body irradiation of patients with bone metastases. Material and Methods Ninety-five patients (97 irradiations) were treated with single half-body irradiation fraction (3–8 Gy). Thirty-three patients had upper-half-body irradiation, 55 lower-half-body irradiation and 9 middle-half-body irradiation. The patients were examined on the day of irradiation, 2 and 4 weeks later, and then once a month. The intake of analgesics, pain level (from 0 to 10), and the quality of life (EORTC QLQ-C30) were evaluated. The fluctuations of pain levels and the particular scaling values of QLQ-C30 during a one-year period were analyzed (Kendall t correlation). Results Over the course of 5 months, the incidence of patients using strong opioids decreased from 43.8% to 33.3%, and the incidence of patients who did not need to resort to analgesics increased from 6.7% to 25%. The mean pain level decreased from 6.1 points (half-body irradiation) to 3.1 points 2 weeks later. An inverse correlation between pain level readings and time was statistically significant. An increase was observed in the values of the five functional scales as reflected on the EORTC QLQ-C30 questionnaire (four of which correlated significantly with the observation time). A similar situation prevailed with respect to global health status. A decrease was observed in most of the values on the symptoms scales; 6 saw a significant decrease, in correlation with the follow-up. Correlations were also found between pain intensity and functionality, and between symptoms scales readings and global health status. Conclusions Half-body irradiation of cancer patients suffering from painful multiple bone dissemination is an effective and simple treatment modality that affords significant quality-of-life improvement and pain relief, thus allowing for a reduction in the use of strong analgesics.

scholarly journals The Effect of Chemotherapy on Quality of Life of the Patients with Metastatic Gastric and Colorectal Cancer

2021 ◽  
Author(s):  
Selda Çakın Ünnü ◽  
Ilkay Tugba Unek ◽  
Ömercan Topaloğlu
Keyword(s):  
Quality Of Life ◽  
Colorectal Cancer ◽  
Health Status ◽  
Global Health ◽  
Palliative Chemotherapy ◽  
Global Health Status ◽  
Symptom Scores ◽  
Eortc Qlq C30 ◽  
Eortc Qlq

Objective: The self-administered questionnaires by the patients are among the most important methods to evaluate the patient’s health related quality of life. The objective of the study was to evaluate the effect of chemotherapy on quality of life of the patients receiving palliative chemotherapy with the diagnosis of metastatic gastric and colorectal cancer by using EORTC QLQ-C30. Methods: This study included 100 patients who were treated with palliative chemotherapy for the diagnosis of metastatic gastric or colorectal cancer in İzmir Tepecik Education and Research Hospital Department of Medical Oncology between 2011-2012. The EORTC QLQ-C30 questionnaire was filled twice by the patients before chemoterapy started and after chemotherapy completed. Results: When the two questionnaires were compared, it was found that global health status and physical functioning did not change after the chemotherapy. Role functioning, cognitive functioning, and social functioning impaired but emotional functioning improved (p<0.05). After the chemoterapy, scores of fatigue and constipation decreased but financial difficulties increased (p<0.05). The symptom scores of nausea-vomitting, pain, dyspnea, insomnia, anorexia, diarrhea did not change. Conclusion: The results of this study suggested that a quality of life assessment with the EORTC QLQ-C30 questionnaire would be beneficial in patients with metastatic gastric and colorectal cancer. In this way, impairments in functional scores, global health status and symptom scores that may occur after chemotherapy can be detected, clinicians can be helped to decide on the switch to chemotherapy regimens that are similar in effectiveness but have different side effects profile, the patients’ quality of life can be improved as a result of the application of the necessary palliative treatments.

scholarly journals Health-Related Quality of Life in Waldenstrom Macroglobulinemia (WM) and IgM Monoclonal Gammopathy of Undeterminated Significance (IgM-MGUS)

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 3593-3593
Author(s):  
Anna Maria Frustaci ◽  
Michele Nichelatti ◽  
Marina Deodato ◽  
Maddalena Mazzucchelli ◽  
Marco Montillo ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Health Status ◽  
Global Health ◽  
Global Health Status ◽  
Emotional Function ◽  
Vas Score ◽  
Eortc Qlq C30 ◽  
Eortc Qlq ◽  
The Impact

Abstract The clinical course of WM widely differs among patients, with some manifesting symptoms as a consequence of the monoclonal IgM component or lymphoma infiltration. IgM-MGUS is generally asymptomatic while, in some cases, paraprotein-related manifestations may occur. Patients with IgM-MGUS should perform a regular follow-up as they are at risk of developing WM or other B-cell lymphoproliferative disorders (1.5-2% per year). Although WM typically afflicts the elderly, there are no studies addressing the impact of ECOG performance status and comorbidities on patients' outcome. Furthermore, to our knowledge health-related quality of life (HRQOL) has never been evaluated in this category. The aim of this study is to analyze the impact of diagnosis and patients' characteristics on quality of life. From October 2017, HRQOL was assessed in 103 patients (37 WM with previous or ongoing treatment [tWM]; 29 asymptomatic MW [aWM]; 37 IgM-MGUS) by the administration of EORTC QLQ-C30, HADS, FACT-GOG neurotoxicity and EQ-5D-5L questionnaires. Demographic anamnestic and disease-related data were also collected for each patient. The same questionnaires continue to be administered every 6 months for 3 years, in order to capture changes in HRQOL over time. Patients features are reported in table 1. No significant differences in terms of age, sex distribution, age at diagnosis, months from diagnosis, ECOG performance status, CIRS or number of concomitant medications, were detected among the 3 groups (table 1). As regards CIRS, the organ systems mainly involved resulted: vascular and genitourinary for tWM, genitourinary for aWM and vascular, respiratory and genitourinary for IgM-MGUS. Among the 3 groups no statistical differences were reported when analyzing: EORTC QLQ-C30 global health status, functional scales (physical, role, emotional, cognitive and social functioning) and symptoms scale, EQ-5D VAS score, HADS anxiety and depression scores or FACT-GOG neurotoxicity score. Males had higher global health status and emotional function when compared to females both in IgM-MGUS and WM patients. Higher CIRS score and ECOG status negatively impacted on global health status, physical function, EQ-5D VAS score and anxiety both in WM and IgM-MGUS. WM patients with longer time from diagnosis showed a significantly worse emotional function. Patients-reported symptoms that could be referred to peripheral neuropathy (PN, 39 patients) resulted the only significant parameter negatively impacting on HRQOL (global health status, functional and symptoms scales according to EORTC QLQ-C30 and EQ-5D VAS score) and also affecting HADS anxiety score. The diagnosis of PN was confirmed by neurologic tests only in 16/39 subjects that, compared with the rest of the population, showed older age (p .019), older age at diagnosis (p . 015) and higher ECOG status (p .005). In these patients, EORTC QLQ-C30 detected a reduced cognitive function (p .0031), while HADS a greater perception of anxiety (p .0015). No differences were recorded for EQ-5D VAS score or HADS depression scale. In conclusion, in our series diagnosis per se didn't seem to affect HRQOL which was negatively influenced by high ECOG status and comorbidities. Emotional function meaningfully deteriorated as the time lapse from diagnosis became longer. Quality of life was significantly altered in patients reporting symptoms of PN and this was confirmed by all the questionnaires. Longer follow up is needed to confirm these preliminary data. Disclosures Montillo: Roche: Consultancy, Honoraria, Research Funding; Janssen: Consultancy, Honoraria; AbbVie: Consultancy, Honoraria, Speakers Bureau; Gilead: Consultancy, Honoraria, Speakers Bureau. Tedeschi:Janssen: Consultancy, Speakers Bureau; AbbVie: Consultancy; Gilead: Consultancy.

Pomalidomide (POM) Plus Low-Dose Dexamethasone (LoDEX) Improves Health-Related Quality Of Life (HRQoL) Vs High-Dose Dexamethasone (HiDEX) In Relapsed Refractory Multiple Myeloma (RRMM) Patients Enrolled In MM-003 Phase 3 Randomized Trial

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 2939-2939 ◽  
Author(s):  
Kevin W. Song ◽  
Meletios A. Dimopoulos ◽  
Katja Weisel ◽  
Michel Delforge ◽  
Lionel Karlin ◽  
...  
Keyword(s):  
Health Status ◽  
Global Health ◽  
Board Of Directors ◽  
Physical Functioning ◽  
Research Funding ◽  
Emotional Functioning ◽  
Global Health Status ◽  
Employment Equity ◽  
Advisory Committees ◽  
Equity Ownership

Abstract Background Pronounced symptoms, poor prognosis, and therapy exhaustion each affect HRQoL in RRMM patients. The MM-003, randomized, multicenter, open-label phase 3 trial reported that POM + LoDEX significantly extended median progression-free and overall survival vs HiDEX in RRMM patients who exhausted bortezomib (BORT) and lenalidomide (LEN), and progressed on their last treatment (San Miguel, EHA, 2013). Improved survival outcomes, treatment-related toxicity, and aging populations have placed an emphasis on HRQoL in RRMM. MM-003 is the first study to investigate HRQoL in RRMM patients treated with POM + LoDEX. Methods Patients were randomized 2:1 to receive 28-day cycles (C) of POM 4 mg D1-21 + LoDEX 40 mg (20 mg for patients aged > 75 years) weekly or HiDEX 40 mg (20 mg for patients aged > 75 years) D1-4, 9-12, and 17-20 until disease progression or unacceptable toxicity. HiDEX was chosen as the comparator to isolate the effects of POM as at the time of trial design, it was the standard salvage therapy for heavily pretreated patients. Time to meaningful worsening in HRQoL was analyzed for 5 predefined EORTC QLQ-C30 domains (Global Health Status, Physical Functioning, Fatigue, Emotional Functioning, and Pain) using the Kaplan-Meier method. A meaningful worsening was defined as a reduction in HRQoL equal to or greater than the domain-specific minimally important difference, calculated using the standard error of measurement. HRQoL was also evaluated cross-sectionally and longitudinally through a mixed effect model. Data from the September 7, 2012 data cut are presented below. Results 455 patients were randomized to POM + LoDEX (n = 302) or HiDEX (n = 153). 448 patients completed at least 1 QLQ-C30 questionnaire and were included in the HRQoL analysis. The median number of prior treatments was 5 (range, 2-17), and 82% were refractory to LEN and BORT. Median follow-up was 4.2 months. Analyses on time to clinically meaningful worsening showed that POM + LoDEX extended median time to meaningful worsening vs HiDEX for all the preselected domains (Table): Global Health Status (114 vs 85 days, P = .06), Physical Functioning (174 vs 106 days; P = .09), Fatigue (113 vs 60 days; P = .04), Emotional Functioning (190 vs 124 days; P = .02), and Pain (147 vs 113 days; P = .2). In the cross-sectional analysis, HRQoL scores were relatively better for POM + LoDEX vs HiDEX for all domains at every cycle. Statistically or marginally significant (P < .10) treatment differences were observed for Global Health Status (C2 and 4), Physical Functioning (C2, 3, and 4), Emotional Functioning (C3), and Fatigue (C2) domains. No statistically significant deteriorations from baseline were observed in the mean scores for the POM + LoDEX arm for any of the 5 domains at any treatment cycle. Such deteriorations were observed with HiDEX. These results were supported by a mixed effect model analysis. Data from the March 1, 2013 data cut will be presented at the meeting. Conclusions In heavily pretreated patients who exhausted BORT and LEN, POM + LoDEX resulted in better clinical outcomes and favorable HRQoL vs patients treated with HiDEX. POM + LoDEX should become a standard of care in relapsing patients as it has been proven to prolong and enhance the lives of RRMM patients. Disclosures: Song: Celgene: Honoraria, Membership on an entity’s Board of Directors or advisory committees, Research Funding, Speakers Bureau. Off Label Use: POM is approved in the US but not in Europe. Dimopoulos:Celgene: Honoraria, Membership on an entity’s Board of Directors or advisory committees. Weisel:Celgene: Consultancy, Honoraria, Research Funding; Janssen: Consultancy, Honoraria. Delforge:Celgene: Honoraria. Karlin:Celgene: Export board committee Other, Honoraria; Janssen: Honoraria. Goldschmidt:Celgene: Consultancy, Honoraria, Research Funding; Janssen: Consultancy, Honoraria, Research Funding; Novartis: Consultancy, Honoraria, Research Funding. Moreau:Celgene: Honoraria, Speakers Bureau. Oriol:Celgene: Consultancy. Spencer:Celgene: Honoraria, Membership on an entity’s Board of Directors or advisory committees. Knop:Celgene: Honoraria. Renner:Celgene: Consultancy, Honoraria, Travel support Other. Bahlis:Celgene: Consultancy, Honoraria, Research Funding. Amatya:Celgene: Consultancy. Yu:Celgene: Employment, Equity Ownership. Monzini:Celgene: Employment, Equity Ownership. Sternas:Celgene: Employment, Equity Ownership. Zaki:Celgene: Employment, Equity Ownership. Jacques:Celgene: Employment, Equity Ownership. San Miguel:Onyx: Consultancy, Honoraria, Membership on an entity’s Board of Directors or advisory committees; Novartis: Consultancy, Honoraria, Membership on an entity’s Board of Directors or advisory committees; Millenium: Consultancy, Honoraria, Membership on an entity’s Board of Directors or advisory committees; Janssen: Consultancy, Honoraria, Membership on an entity’s Board of Directors or advisory committees; Celgene: Consultancy, Honoraria, Membership on an entity’s Board of Directors or advisory committees.

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