scholarly journals Lenalidomide for the Treatment of Low- and Int-1-Risk MDS with Del(5q): Efficacy and Quality of Life Study.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 2763-2763 ◽  
Author(s):  
Esther Natalie Oliva ◽  
Roberto Latagliata ◽  
Fortunato Morabito ◽  
Antonella Poloni ◽  
Riccardo Ghio ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Performance Status ◽  
Cytogenetic Response ◽  
Study Entry ◽  
International Prognostic Scoring System ◽  
Ecog Performance Status ◽  
Cytogenetic Abnormalities ◽  
Preliminary Results ◽  
Transfusion Independence

Abstract Abstract 2763 Poster Board II-739 Introduction: Chronic anemia of myelodysplastic syndromes (MDS) is associated with poor quality of life (QoL) and an inferior clinical course. Transfusion dependence in lower-risk patients is associated with reduced survival as a result of iron overload, heart failure, and progression to acute myeloid leukaemia. Lenalidomide is approved for the treatment of transfusion-dependent anemia in patients with International Prognostic Scoring System (IPSS) Low- or Intermediate (Int)-1-risk MDS with deletion 5q [del(5q)]. Rapid and durable responses include transfusion independence with a rise in Hb, suppression of the del(5q) clone, and improvement in bone marrow morphological features. We present preliminary results of a prospective single-arm trial investigating the effect on QoL, efficacy, and safety of lenalidomide in the treatment of 49 adult patients with IPSS Low- and Int-1-risk MDS with del(5q) with/without additional cytogenetic abnormalities and Hb < 10 g/dL. Methods: Exclusion criteria include: ANC < 500/mm3; PLT count < 50,000/mm3; prior chemotherapy; and ongoing treatment with rHuEpo. Lenalidomide was administered orally at a starting dose of 10 mg/day. If necessary, dosing was reduced to 5 mg/day or 5 mg on alternate days. Treatment will be continued for a maximum of 12 months or until evidence of unacceptable non-hematological adverse events, lack of response, disease progression, or relapse following erythroid improvement. QoL was assessed at study entry and weeks 8, 12, and 24 using the QOL-E v.2 questionnaire. QoL scores are standardized in a 0–100 scale with lower scores representing a worse QoL. Response was evaluated according to the modified International Working Group (IWG) response criteria. Results: Twenty patients (5 M, 15 F, mean age 72 ± 10 years) are evaluable for erythroid responses and cytogenetic changes at 12 weeks and 13 patients have reached a 24-week follow-up. At baseline, mean disease duration was 3.4 ± 2.3 years. Seventeen patients were transfusion dependent (TD), 3 were transfusion free (TF). ECOG performance status was 0 in 14 patients and 1 in 6 patients. After 12 weeks from study entry, 17 (85%) patients obtained an erythroid response with a mean Hb level increase from baseline 8.6 ± 0.9 g/dL to 11.1 ± 2.4 (p=0.001). By 24 weeks, 11 of the 13 patients re-evaluated were erythroid responders obtaining transfusion independence and significant improvements in Hb (mean change from baseline 3.7 ± 2.7 g/dL, and increase to mean 11.1 ± 2.4 g/dL (p<0.001). Eight out of 20 cases (35%) reached normal Hb levels after 12 weeks and 8 out of 13 patients (62%) by 24 weeks. A cytogenetic response (at least 50% reduction in del[5q]) was observed in 5 responders out of 13 patients evaluated at 24 weeks. Additional cytogenetic abnormalities were observed in 4 responders. A progressive improvement in QoL was experienced in responders in the first 24 weeks of treatment. Physical QoL scores increased from 35 ± 9 at baseline to 69 ± 25 at week 24 (p = 0.086). Social-QoL scores significantly changed from 29 ± 20 at baseline to 83 ± 20 at week 12 (p = 0.021). Changes in physical QoL correlated with improvements in Hb (r = 0.768, p=0.001). Drug interruption followed by reduction to 5 mg/day was required in 16 patients within the first 8 weeks due to significant neutropenia, which was associated with thrombocytopenia in 3 patients and hospitalization because of infection in 2 patients. One patient withdrew from treatment because of progressive anemia. Conclusions: Preliminary results confirm that in Low- and Int-1-risk MDS patients with del(5q) lenalidomide induces clinically significant erythroid responses and transfusion independence. Most patients require a dose reduction mainly due to neutropenia. Responders experience improvements in physical and social QoL. Disclosures: Oliva: Celgene: Consultancy. Finelli:Celgene: Consultancy.

Psychometric Performance Of The EORTC Quality-Of-Life Core Questionnaire (QLQ-C30) and QLQ-Multiple Myeloma (QLQ-MY20) In Relapsed/Refractory Multiple Myeloma (RRMM)

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 1721-1721 ◽  
Author(s):  
Xavier Leleu ◽  
Maria Teresa Petrucci ◽  
Manfred Welslau ◽  
Isabelle Vande Broek ◽  
Philip T Murphy ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Multiple Myeloma ◽  
Psychometric Properties ◽  
Performance Status ◽  
Line Treatment ◽  
Ecog Performance Status ◽  
Advisory Committees ◽  
Financial Difficulties ◽  
Equity Ownership

Abstract Introduction With the increased use of first generation novel therapeutic agents and extended life expectancy of multiple myeloma, patients’ Health-Related Quality of Life (HRQoL) is gaining considerable importance, including the relapsed/refractory multiple myeloma (RRMM) setting. So far, few studies have been conducted to appreciate the physical, psychological/cognitive, financial, social health impact of living with RRMM on longer term exposure to anticancer drugs. The objective of this study was to evaluate the psychometric properties of the EORTC Quality-of-Life Core Questionnaire (QLQ-C30) and QLQ-Multiple Myeloma (QLQ-MY20) in RRMM patients. Methods A European, multicenter, observational study is being conducted in RRMM patients starting 2nd or 3rd line treatment. Patients are asked to complete the QLQ-C30 and QLQ-MY20 at baseline, month 3, and month 6 or discontinuation visit. Both generic and specific modules of EORTC questionnaires are widely used to assess HRQoL and symptoms in cancer patients. The QLQ-C30 includes 15 domains (Global Health Status/QOL, Physical Functioning, Role Functioning, Emotional Functioning, Cognitive Functioning, Social Functioning, Fatigue, Nausea and Vomiting, Pain, Dyspnea, Insomnia, Appetite Loss, Constipation, Diarrhea and Financial Difficulties); and the QLQ-MY20 includes four domains (Disease Symptoms, Side-Effects of Treatment, Body Image and Future Perspective). Construct validity was evaluated at baseline by confirming the structure using multitrait analysis and by assessing clinical validity against ECOG performance status. Internal consistency reliability was evaluated at baseline using Cronbach’s alpha. Results As of June 2013, 206 patients have been enrolled in the study and included in this interim analysis. Mean age was 69 years, with 51% male, and with an average time since diagnosis of 3.4 years. A total of 90% of patients started 2nd line treatment and 10% started 3rd line treatment. Both EORTC questionnaires were well completed by patients, with 95% of patients responding to the QLQ-C30 and MY20 at baseline and 74% and 66% of patients, respectively, completing all QLQ-C30 and QLQ-MY20 items at baseline. Substantial percentages of patients reported the best possible level of HRQoL and symptoms at baseline, i.e. zero points for Appetite Loss (61%), Constipation (51%), Diarrhea (80%), Financial Difficulties (73%) and Nausea and Vomiting (73%), respectively 100 score points for Body Image (61%). The structure of multi-item QLQ-C30 and QLQ-MY20 domains was confirmed, as was the relevance of aggregation of items into domains (Table 1 ). Both QLQ-C30 and QLQ-MY20 scores were correlated to ECOG performance status (Figure 1). Conclusions This interim analysis confirmed in RRMM patients the satisfactory psychometric properties of the EORTC QLQ-C30 and QLQ-MY20 in terms of validity and reliability, already published in other cancer conditions. Psychometric properties of both questionnaires will be confirmed when repeating the analyses on the final dataset of this study. Disclosures: Leleu: Janssen: Consultancy, Honoraria; Celgene: Consultancy, Honoraria; Onyx: Consultancy, Honoraria; Leopharma: Consultancy, Honoraria; Millennium: Honoraria; Amgen: Honoraria; Novartis: Honoraria. Petrucci:Celgene: Consultancy, Honoraria; Janssen-Cilag: Consultancy, Honoraria; Bristol-Myers Squibb: Consultancy, Honoraria. Welslau:Celgene: Membership on an entity’s Board of Directors or advisory committees; Janssen: Membership on an entity’s Board of Directors or advisory committees. Bottomley:Celgene: Consultancy, Research Funding. Bacon:Celgene International: Employment, Equity Ownership. Lewis:Celgene GmbH: Employment, Equity Ownership. Gilet:Celgene: Consultancy. Arnould:Celgene: Consultancy.

Treatment of Patients with Low-Risk Myelodysplastic Syndromes Receiving Azacitidine Who Are Enrolled in AVIDA, a Longitudinal Patient Registry.

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 1646-1646 ◽  
Author(s):  
David L. Grinblatt ◽  
Mohit Narang ◽  
James M. Malone ◽  
David A. Sweet ◽  
Tim S. Dunne ◽  
...  
Keyword(s):  
Platelet Transfusion ◽  
Performance Status ◽  
Low Risk ◽  
Patient Registry ◽  
International Prognostic Scoring System ◽  
Community Based ◽  
Ecog Performance Status ◽  
Transfusion Independence ◽  
Risk Patients ◽  
Rbc Transfusion

Abstract With the development of new agents such as azacitidine, a hypomethylating agent approved for the treatment of all 5 MDS subtypes, chemotherapy for MDS patients has become more common. Nonetheless, the use of hypomethylating agents in patients with low-risk MDS in the community-based setting has not been well characterized. AVIDA is a longitudinal, multicenter patient registry designed to prospectively collect data from community-based hematology clinics on the natural history and management of patients with MDS and other hematologic disorders, including acute myeloid leukemia, who are treated with azacitidine. The characteristics and transfusion status of patients enrolled in AVIDA who had an International Prognostic Scoring System (IPSS) risk classification score of low/intermediate-1 at baseline are presented. As of August 1, 2008, 130 (91 males, 39 females) patients with an IPSS score of low/intermediate-1 have been enrolled in AVIDA; 45 (35%) with low and 85 (65%) with intermediate-1. Median age is 74.7 years (range, 41.4–91.4), and the majority (80%) had a baseline ECOG performance status of 0 or 1. Median time from first MDS diagnosis until azacitidine treatment was 7.5 months (range, 0 to 108), suggesting a delay in treatment of these patients compared with high risk patients (n = 55) in the registry (1 month). At baseline, 103/130 (79%) patients had &lt;5% bone marrow blasts and 109/130 (84%) had a ‘good’ karyotype. Cytopenias were reported in 0 or 1 lineage for 69/130 (53%) patients and in 2 or 3 lineages for 60/130 (46%) patients; number of cytopenias at baseline is unknown for 1 (1%) patient. A total of 483 cycles of azacitidine have been administered either by subcutaneous (43%) or intravenous (57%) infusion. A total of 126 patients with an IPSS score of low/intermediate-1 have received a median of 3 cycles (range, 1 to 15); 94/126 (75%) patients have received at least 2 cycles. Transfusion independence was defined as no transfusions for at least 56 days. The first day of the 56-day period with no transfusions was noted as the time at which patients first achieved transfusion independence. Transfusion data are available for 52 patients who were receiving RBC transfusions at baseline and have received at least 2 cycles. Of these patients, 24/52 (46%) have achieved RBC transfusion independence while receiving azacitidine; 16/24 (67%) achieved RBC transfusion independence during the first 2 cycles. Thirteen patients were receiving platelet transfusions at baseline and have received at least 2 cycles of therapy. Of those patients 8/13 (62%) have achieved platelet transfusion independence; 7/8 (88%) achieved platelet transfusion independence during the first 2 cycles. In these low-risk patients, azacitidine was generally well tolerated; the most common adverse events were anemia (20%), thrombocytopenia (13%), nausea (11%), constipation (10%), fatigue (10%), and neutropenia (10%). These data demonstrate that patients with an IPSS score of low/intermediate-1 are being treated in the community-based setting and can achieve transfusion independence while receiving azacitidine. AVIDA provides a unique opportunity to characterize the MDS patient population that is receiving chemotherapy in the community-based setting. The effect of azacitidine on the outcome and overall quality of life in this patient subpopulation will become clearer as more patients with low-risk MDS are treated.

scholarly journals Long-Term Quality of Life after Pancreatic Surgery for Intraductal Papillary Mucinous Neoplasm

2021 ◽  
pp. 1-8
Author(s):  
Helwig Valentin Wundsam ◽  
Christiane Sophie Rösch ◽  
Patrick Kirchweger ◽  
Ines Fischer ◽  
Michael Weitzendorfer ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Performance Status ◽  
Eastern Cooperative Oncology Group ◽  
Life Questionnaire ◽  
Ecog Performance Status ◽  
Long Term Follow Up ◽  
Eortc Qlq

<b><i>Introduction:</i></b> Intraductal papillary mucinous neoplasms (IPMNs) represent the most common precancerous cystic lesions of the pancreas. The aim of our study was to investigate if resection for non-invasive IPMNs alters quality of life (QoL) in a long-term follow-up. <b><i>Methods:</i></b> Patients (<i>n</i> = 50) included in the analysis were diagnosed and resected from 2010 to 2016. QoL was assessed at a median of 5.5 years after resection. At that point in time, the current QoL as well as the QoL before resection was evaluated retrospectively. The standardised European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire for Pancreatic Cancer (EORTC QLQ – PAN26) was applied for the QoL assessment. <b><i>Results:</i></b> After a median of 66 months postoperatively, the total QoL score significantly worsened (92.13 vs. 88.04, <i>p</i> = 0.020, maximum achievable score = 100) for patients (median age at surgery 68.0 years), mostly due to digestive symptoms. During the same follow-up period, median Eastern Cooperative Oncology Group (ECOG) performance status did not worsen (<i>p</i> = 0.003). <b><i>Conclusions:</i></b> Long-term QoL statistically significantly worsened after pancreatic resection for IPMN. The extent of worsening, however, was small, and QoL still remained excellent. Therefore, resection in cases of IPMN is appropriate, if indicated carefully.

scholarly journals Feasibility of Peri-Transfusion Quality of Life Assessment for Patients with Myelodysplastic Syndromes

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 25-26
Author(s):  
Gregory A. Abel ◽  
Heidi D. Klepin ◽  
Emily S. Magnavita ◽  
Tim Jaung ◽  
Rory M. Shallis ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Myelodysplastic Syndromes ◽  
Conflicts Of Interest ◽  
Performance Status ◽  
Life Assessment ◽  
Ecog Performance Status ◽  
Before And After ◽  
Ecog Ps ◽  
Rbc Transfusion

BACKGROUND: Decreased quality of life (QOL) due to fatigue in the setting of anemia is common with myelodysplastic syndromes (MDS), often leading to dependence on red blood cell (RBC) transfusions. Moreover, the COVID-19 pandemic has resulted in considerably reduced blood supply such that the importance of conserving donor RBCs for patients particularly in need is even more crucial (Shander, Anesth Analg 2020). While some patients with MDS experience improved QOL after transfusions, many do not, even at the same ECOG performance status (PS) and hemoglobin (Hb). In a pilot study, we sought to assess the feasibility of a peri-transfusion QOL assessment (PTQA; QOL assessed before and after transfusion) to determine if it could help inform future transfusion decisions for patients with MDS. METHODS: Starting in 2019, patients with MDS at Dana-Farber Cancer Institute, Yale School of Medicine, and Wake Forest University were screened for eligibility using clinic schedule reports in Epic; those 18 or older with biopsy-proven MDS presenting to clinic for RBC transfusions with no evidence of known CHF or unstable angina were eligible to participate. A Hb threshold of 7.5 g/dL or greater was required for participation (Tanasijevic, Leuk Lymph 2020). The QUALMS, a validated PRO for patients with MDS (Abel, Haematologica, 2016), was used to assess patients' QOL before and after RBC transfusion. At consent, patients were given a study packet including a copy of the QUALMS and were instructed to fill out the survey the day before their upcoming transfusion. One week after RBC transfusion, the patient completed the QUALMS again. Surveys were scored, compared, and compiled into a report that was sent to patients and providers (Figure). Based on the QUALMS validation study, we considered a change by 5 or more points to be potentially clinically significant. After descriptive statistics, two-sided Fisher's Exact tests assessed for associations between patient characteristics (ECOG PS and Hb) and post-transfusion increase in QOL. RESULTS: As of July 2020, a total of 57 patients had been enrolled. Of these, 28 (49%) have completed PTQA with both a pre-transfusion and post-transfusion QUALMS. Mean age was 72 years (standard deviation (SD)=11.6), 19% were female, and 89% had had one or more RBC transfusions within 8 weeks prior to enrollment. For the 29 patients who did not undergo PTQA, 11 are still awaiting their index transfusion; 4 passed away before the index transfusion; 4 were transplanted before the index transfusion; 2 developed AML; 7 later became ineligible or were lost to follow up; and 1 withdrew consent. Of the 28 who underwent PTQA, about half had an ECOG performance status of 1, and median Hb at transfusion was 8.05 g/dL (Table). The mean pre-transfusion QUALMS score was 56.1 (SD=15.3) and post-transfusion score was 59.3 (SD=18.0); overall, 35.7% experienced an increase in QOL. Patients with ECOG PS of 1 or 2 were no more likely to have an increase in QOL after RBC transfusion compared to those with PS of 0 (p=1.00). In contrast, 50% of patients with pre-transfusion Hb &lt; 8.0 g/dL had an increase in QOL compared to 25% of patients with Hb &gt;= 8.0 g/dL, although this difference also did not reach significance (p=0.24). CONCLUSIONS: Although PTQA was feasible for about half of patients enrolled, there were many barriers when working with this high-risk MDS population. Moreover, only about one-third of patients experienced an increase in QOL one week after RBC transfusion, arguing that, for some patients, a more limited transfusion schedule may be possible. Disclosures No relevant conflicts of interest to declare.

Quality of Life in Low-Risk Myelodysplastic Syndrome: A Cross-Sectional Study

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 5256-5256 ◽  
Author(s):  
Isik Kaygusuz-Atagunduz ◽  
Mirac Ozen ◽  
Tulin Firatli-Tuglular ◽  
Tayfur Toptas
Keyword(s):  
Quality Of Life ◽  
Blood Cell ◽  
Myelodysplastic Syndrome ◽  
Red Blood Cell ◽  
Performance Status ◽  
Low Risk ◽  
Refractory Anemia ◽  
Intermediate Risk ◽  
Ecog Performance Status

Abstract Myelodysplastic syndrome (MDS) is mainly a disease of elderly population. Chronic cytopenias, especially anemia, frailty comorbidities, and age may alter the physical status significantly. Only a few proportions of patients can achieve long-term cure. In particular, but not limited to patients with low risk MDS; palliative/supportive care is the mainstay of the treatment. Erythropoietin-stimulating agents (ESAs) and red blood cell transfusions are the treatment options for patients suffering from anemia. Except one study, ESA-responders had a better quality of life (QoL) in three studies. Hematologic improvements should be assesses by patient-reported outcomes (PROs) as well as objective measures. Validated PROs those were used to assess QOL in patients with MDS included Functional Assessment of Cancer Therapy- Anemia (FACT-An) and European Organization for Research and Treatment of Cancer QLQ-C30 (EORTC-QLQ C30) questionnaires and a MDS-specific questionnaire, QoL-E. All patients with MDS were screened. Patients with low-risk MDS were included in the study. One physician completed FACT-An, Hematopoietic Stem Cell Transplantation-Comorbidity Index (HCT-CI), and G8 frailty questionnaires in all patients. Demographic data were collected from patients' chart records. A total of 66 patients were screened. Fourteen patients were excluded due to high-risk MDS or indefinite diagnosis. In one patient, informed consent could not be obtained. Finally, 51 patients were included in the study. Median age was 66 years old (interquartile range [IQR]: 55-77). Twenty-one out of 51 patients (41.2%) were male. Most prevalent MDS subtype was MDS-refractory anemia (47%). All patients had very-low/low (86.3%) or intermediate-risk (13.7%) MDS according to age-adjusted IPSS-R (IPSS-RA). Median time from the diagnosis of MDS was 113 (IQR: 53-170) weeks. Twenty-eight patients (54.9%) were transfusion-dependent. Ten patients had a high transfusion burden, which was defined as transfusion requirement ≥4 units (U) over 8 weeks. Median transfusion duration was 112 (IQR: 31-173) weeks for transfusion-dependent patients. Median red blood cell transfusion during eight weeks was 1.5 (IQR: 0-4.5) U. Median hemoglobin concentration was 10.0 (7.9-11.3) g/dL for all patients. A total of nineteen patients (37.3%) were ESA-user/responder. Most of the patients (80.4%) had a low (<11 years) education level. Thirty-eight (74.5%) patients were living with their parents or partners. A half of the patients had an ECOG performance status ≤2. Sixty per cent were frail and 39% had significant (≥2) comorbidities. In univariate analyses, older age (β: -0.740, 95% CI: -1.138; -0.341, p<0.001), higher transfusion burden (β: -7.235, 95% CI: -14.279; -0.190, p=0.044), intermediate risk IPSS-RA (β: -8.113, 95% CI: -15.715; -0.511, p=0.037), lower educational status (β: -19.625, 95% CI: -32.565; -6.684, p=0.004), lower ECOG performance status (≥2) (β: -14.385, 95% CI: -24.805; -3.964, p=0.008), frailty (β: -13.740, 95% CI: -24.518; -2.962, p=0.014), and being ESA-user/responder (β: -15.431, 95% CI: -26.141; -4.722, p=0.006) were associated with worse FACT-An total scores. Multivariate analyses revealed that age (β: -0.738, %95 GA: -1.101; -0.374, p<0.001) and being ESA-user/responder (β: 15.368, %95 GA: 6.040; 24.697, p=0.002) were the only independent predictors of QoL in patients with low-risk MDS (Table 1, figure 1). Model stability was tested in 5000 bootstrap replicates of dataset. Age and being ESA-user/responder were included in 40.6% and 38.2% of all models (Table 1). These data indicates that age and ESA use are independent parameters of QoL in low-risk MDS. Impact of ESA use on QoL is independent from the hemoglobin level. Disclosures No relevant conflicts of interest to declare.

Use of palliative end points to evaluate the effects of mitoxantrone and low-dose prednisone in patients with hormonally resistant prostate cancer.

1994 ◽  
Vol 12 (4) ◽  
pp. 689-694 ◽  
Author(s):  
M J Moore ◽  
D Osoba ◽  
K Murphy ◽  
I F Tannock ◽  
A Armitage ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Prostate Cancer ◽  
Low Dose ◽  
Performance Status ◽  
Serum Testosterone ◽  
Life Questionnaire ◽  
Ecog Performance Status ◽  
European Organization ◽  
Dose Prednisone

PURPOSE This phase II study was designed to assess the effects of mitoxantrone with prednisone in patients with metastatic prostate cancer who had progressed on hormonal therapy. The methods of assessment included quality-of-life analyses, pain indices, analgesic scores, and the National Prostatic Cancer Project (NPCP) criteria. PATIENTS AND METHODS Patients received mitoxantrone 12 mg/m2 intravenously every 3 weeks plus prednisone 10 mg orally daily. All had a castrate serum testosterone and Eastern Cooperation Oncology Group (ECOG) performance status < or = 3, and had not received prior chemotherapy. Every 3 weeks, analgesic intake was scored, and a present pain intensity (PPI) record and visual analog scale (VAS) describing pain were collected. Every 6 weeks, the European Organization for Research and Treatment of Cancer (EORTC) core quality-of-life questionnaire plus a prostate-specific module were completed. A palliative response was defined as a decrease in analgesic score by > or = 50% or a decrease in PPI by > or = two integers without any increase in the other. RESULTS Twenty-seven patients were entered onto the study. Nine of 25 (36%) assessable patients achieved a palliative response maintained for > or = two cycles (range, two to eight or more). Improvements in mean PPI and VAS pain scores after each cycle of therapy (P < .05) were seen. Quality-of-life analysis showed improvements in social and emotional functioning, and in pain and anorexia. Using NPCP criteria, one patient achieved a partial response (PR) and 12 had stable disease; one of seven patients with measurable disease had a PR. No serious nonhematologic toxicity was experienced, and there were no episodes of febrile neutropenia. CONCLUSION Mitoxantrone with low-dose prednisone is a well-tolerated treatment regimen that has some beneficial effects on disease-related symptoms and quality of life for patients with advanced prostate cancer.

scholarly journals Patient-Reported Outcomes Validation of the FACT-Leu in Acute Myeloid Leukemia: A Review of Baseline Characteristics in AML2002

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 3590-3590
Author(s):  
Jianming He ◽  
Renee Pierson ◽  
Christina Loefgren ◽  
David Cella
Keyword(s):  
Quality Of Life ◽  
Myeloid Leukemia ◽  
Research Funding ◽  
Performance Status ◽  
Well Being ◽  
Intensive Chemotherapy ◽  
Moderate Correlation ◽  
Ecog Performance Status ◽  
The Mean

Abstract Introduction: Acute Myeloid Leukemia (AML) is an aggressive disease associated with poor health related quality of life (HRQoL) and short overall survival (OS), particularly for patients' ineligible for intensive chemotherapy. The HRQoL was evaluated in a cohort of patients with AML who were not considered eligible for standard chemotherapy based on the Functional Assessment of Cancer Therapy - Leukemia (FACT-Leu) collected at baseline of AML2002 study (NCT02472145). Methods: This analysis was based on a randomized, phase 2/3, parallel design study conducted in patients with AML. Patients' perceptions to HRQoL were evaluated using a 44-item, self-reported leukemia-specific measure, FACT-Leu. It was assessed based on 5 subscales: physical well-being (PWB), social well-being (SWB), emotional well-being (EWB), functional well-being (FWB) and leukemia-specific concerns. Additionally, FACT-Leu was also evaluated based on the trial outcome index (TOI). European Quality of Life- 5 Dimension 5-Level (EQ-5D-5L) was reported based on index values and visual analogue scale (VAS). The summary statistics from the FACT-Leu was compared to the validation paper [1]. FACT-Leu by eastern cooperative oncology group (ECOG) performance status score was also assessed based on generalized linear model and the correlation among FACT-leu subscales was assessed using the Pearsons correlation coefficient Results: Of the 309 patients (mean age 74.9 years) enrolled, 46.3% were women, 87.3% were white (Caucasian) and 70.9% had de novo AML. For these patients, ECOG performance status distribution was 0 (18%), 1 (41.9%) and 2 (40.2%). At baseline, the mean index values for VAS of EQ-5D-5L were 0.68 and 62.5, respectively, and the mean FACT-Leu was 119.6. Except SWB, other FACT-Leu subscale and aggregated scores highly correlated with FACT-Leu (0.74-0.96; p<0.0001). Both index values (0.65) and VAS of EQ-5D-5L (0.57) showed moderate correlation with FACT-Leu. The EQ-5D-5L (0.71) and VAS (0.60) showed moderate correlation with FACT-TOI (p<0.0001). Except SWB and EWB, other FACT-Leu subscales and aggregated scores showed predicted differences in means based on the ECOG score, with higher scores associated with better ECOG status. However, compared to the results of the validation paper [1], the mean subscale scores of AML ineligible for intensive chemo therapy were lower. In addition to ECOG status, sex was also a significant predictor of FACT-Leu subscales as aggregated scores except SWB and EWB with men reporting better scores. Conclusions: FACT-Leu scores were significantly associated with PS and sex. The lower mean subscale scores in Patients with AML ineligible for intensive chemotherapy highlight the need for new therapies to improve patient HRQoL in this group of patients and suggested that there is a need for optimized instruments for women. The FACT-Leu tool could facilitate targeted population interventions, potentially improving quality of life. [1] Cella, Value in Health 15 (2012) 1051-1058 Disclosures He: Janssen Global services: Employment. Pierson:Janssen Global Services LLC: Employment. Loefgren:Janssen Global Services LLC: Employment. Cella:Pfizer: Consultancy, Research Funding; Novartis: Consultancy, Research Funding; Janssen Global services: Consultancy, Research Funding; Bristol-Myers Squibb: Consultancy, Research Funding.

Transfusion Independence in Patients with Hematologic Disorders Receiving Azacitidine Who Are Enrolled in AVIDA, a Longitudinal Patient Registry

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 2683-2683
Author(s):  
David L. Grinblatt ◽  
Mohit Narang ◽  
James M. Malone ◽  
David A. Sweet ◽  
Tim S. Dunne ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Platelet Transfusion ◽  
Patient Registry ◽  
International Prognostic Scoring System ◽  
Community Based ◽  
Hematologic Disorders ◽  
Transfusion Independence ◽  
Transfusion Requirements ◽  
Rbc Transfusion

Abstract Patients with myelodysplastic syndromes (MDS) or other hematologic disorders experience anemia and/or thrombocytopenia at some point during the course of their disease. Current management of these cytopenias includes frequent red blood cell (RBC) and platelet transfusions, and use of other supportive therapy (eg, erythropoietin). Dependence on transfusions can be coupled with diminished quality of life, poorer outcomes, and increased economic burden. Azacitidine, a hypomethylating agent approved in the US for the treatment of all 5 MDS subtypes, is associated with transfusion independence in patients enrolled in clinical trials (Silverman, et al. J Clin Oncol.2006;24:3895). The establishment of transfusion independence in patients receiving azacitidine in the non-clinical trial/community-based setting is not well characterized. AVIDA is a longitudinal, multicenter patient registry designed to prospectively collect data from community-based hematology clinics on the natural history and management of patients with MDS and other hematologic disorders, including acute myeloid leukemia, who are treated with azacitidine. Baseline demographics and disease characteristics were obtained at enrollment. Transfusion requirements and onset of RBC and platelet transfusion independence were recorded. Transfusion independence was defined as no transfusions for at least 56 days. The first day of the 56-day period with no transfusions was noted as the time at which patients first achieved transfusion independence. As of August 1, 2008, 220 (154 males, 66 females; mean age, 73.5 yrs) have been enrolled in AVIDA. At baseline, the majority of patients had primary MDS (183 patients; 83%), an ECOG performance status of 0 or 1 (164 patients; 75%), and an International Prognostic Scoring System (IPSS) risk classification of Low/Intermediate-1 (130 patients; 59%); 55 (25%) had a Intermediate-2/high IPSS risk and 35 (16%) had unknown IPSS risk. Median time from first MDS diagnosis until azacitidine treatment was 3 months (range, 0 to 149). A total of 732 cycles of azacitidine have been administered either by subcutaneous (46%) or intravenous (54%) infusion; 203 patients have received a median of 3 cycles (range, 1–15). The most common dose and schedule is 75 mg/m2 (82%) at 5 days on treatment (51%). Transfusion data are available for 136 patients who have received at least 2 cycles of azacitidine. Eighty-three of 136 (61%) patients had received at least 1 RBC transfusion during the 6 months prior to AVIDA. Of these patients, 33/83 (40%) achieved RBC transfusion independence; 23/33 (70%) first achieved RBC transfusion independence during the first 2 cycles of azacitidine therapy. Among those patients who had received a platelet transfusion 6 months prior to AVIDA, 13/22 (59%) achieved platelet transfusion independence; 11/13 (85%) first achieved platelet transfusion independence within the first 2 cycles. Azacitidine was generally well tolerated; the most common adverse events were anemia (18%), thrombocytopenia (14%), fatigue (13%), nausea (13%), constipation (12%), and neutropenia (10%). These data demonstrate that in the community-based setting, patients with MDS or other hematologic disorders can achieve transfusion independence within the first 2 cycles. The full benefit of achieving transfusion independence on quality of life and clinical outcomes will be elucidated as more patients are enrolled in AVIDA.

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