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.

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.

Liberal red blood cell transfusions impair quality of life after cardiac surgery

2019 ◽  
Vol 43 (3) ◽  
pp. 156-164
Author(s):  
A. González-Pérez ◽  
J.Z. Al-Sibai ◽  
P. Álvarez-Fernández ◽  
P. Martínez-Camblor ◽  
M. Argüello-Junquera ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cardiac Surgery ◽  
Blood Cell ◽  
Red Blood Cell ◽  
Red Blood Cell Transfusions

scholarly journals Myelodysplastic syndrome: the other cause of anemia in end-stage renal disease patients undergoing dialysis

2020 ◽  
Vol 10 (1) ◽  
Author(s):  
Min-Yu Chang ◽  
Sheng-Fung Lin ◽  
Shih-Chi Wu ◽  
Wen-Chi Yang
Keyword(s):  
Blood Cell ◽  
Renal Disease ◽  
Myelodysplastic Syndrome ◽  
Red Blood Cell ◽  
End Stage Renal Disease ◽  
Refractory Anemia ◽  
Research Database ◽  
Stage Renal Disease ◽  
End Stage ◽  
Esrd Patients

Abstract In end-stage renal disease (ESRD) patients receiving dialysis, anemia is common and related to a higher mortality rate. Erythropoietin (EPO) resistance and iron refractory anemia require red blood cell transfusions. Myelodysplastic syndrome (MDS) is a disease with hematopoietic dysplasia. There are limited reports regarding ESRD patients with MDS. We aim to assess whether, for ESRD patients, undergoing dialysis is a predictive factor of MDS by analyzing data from the Taiwan National Health Insurance Research Database. We enrolled 74,712 patients with chronic renal failure (ESRD) who underwent dialysis and matched 74,712 control patients. In our study, we noticed that compared with the non-ESRD controls, in ESRD patients, undergoing dialysis (subdistribution hazard ratio [sHR] = 1.60, 1.16–2.19) and age (sHR = 1.03, 1.02–1.04) had positive predictive value for MDS occurrence. Moreover, more units of red blood cell transfusion (higher than 4 units per month) was also associated with a higher incidence of MDS. The MDS cumulative incidence increased with the duration of dialysis in ESRD patients. These effects may be related to exposure to certain cytokines, including interleukin-1, tumor necrosis factor-α, and tumor growth factor-β. In conclusion, we report the novel finding that ESRD patients undergoing dialysis have an increased risk of MDS.

Quality of Life and Economic Impact of Red Blood Cell (RBC) Transfusions on Patients with Myelodysplastic Syndromes (MDS).

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 4716-4716 ◽  
Author(s):  
S. Brechignac ◽  
E. Hellstrom-Lindberg ◽  
D. T. Bowen ◽  
T. M. DeWitte ◽  
M. Cazzola ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Blood Cell ◽  
Red Blood Cell ◽  
Chelation Therapy ◽  
Subcutaneous Administration ◽  
Iron Chelation ◽  
International Prognostic Scoring System ◽  
Blood Collection ◽  
Iron Chelation Therapy

Abstract Background: Supportive care with blood product transfusions is the primary management strategy for the majority of patients with MDS. Approximately 80% of MDS patients are anemic at the time of presentation and more than 40% require regular RBC transfusions at some stage of disease, while platelet transfusions are less often required. Methods: In an effort to systematically study quality of life and economic cost associated with transfusion dependency (especially RBC transfusions), The MDS Foundation has disseminated a practices and treatment survey to its Centers of Excellence and is also accumulating transfusion data. Retrospective and prospective data collected include hematologic parameters defining transfusion need; percentage (%) of MDS patients requiring transfusion; % of transfusion-dependent MDS patients by subtype and International Prognostic Scoring System (IPSS) risk group; per patient frequency of transfusions; % of patients requiring iron chelation therapy. Results: A total of 30 Centers have replied to the survey to date, and responses reveal that a substantial proportion of MDS patients receive multiple RBC transfusions with most of these patients needing chelation therapy with desferoxamine (generally subcutaneous administration, 4-times weekly): Table 1. In addition, detailed data are available from 4 European Centers that have provided transfusion records from randomly selected multiply-transfused MDS patients: 38 patients (median age: 73) received a median of 42 transfusions over the last 24 months (range: 11–207). The average per transfusion costs calculated from estimates provided by the 4 European centers is 436 euros or $ 526 ($1 US dollar = 0.83 euros), where the per transfusion cost includes 2 filtered red blood cell units, blood collection, administrative costs, and staff time, resulting in a median per patient cost over the last 24 months of 11,118 euros (range: 5668–21,800 euros). This does not include the cost of chelation therapy (300 euros/month for desferioxamine SC) and indirect costs (e.g., time spent at transfusion facility, travel time for patient to facility, travel and wait time for private caretaker or family member). Conclusion: Preliminary data analysis from the ongoing retrospective study suggests that the transfusion burden to MDS patients and to society, in terms of quality of life and cost, is much greater than generally appreciated. Updated data of this study will be presented. Table 1: RBC Transfusion-dependent MDS patients Mean % IPSS low risk 39 IPSS intermediate-1 risk 50 IPSS intermediate-2 risk 63 IPSS high risk 79 Iron chelation therapy 28

scholarly journals Measuring Health-Related Quality of Life in Sickle Cell Disease Patients Undergoing Automated Red Blood Cell Exchange in the USA, France and the UK

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 5892-5892
Author(s):  
Koenraad Dierick ◽  
Joseph Roig
Keyword(s):  
Quality Of Life ◽  
Sickle Cell Disease ◽  
Blood Cell ◽  
Red Blood Cell ◽  
Blood Cells ◽  
Health Related Quality ◽  
Cell Disease ◽  
Related Quality ◽  
Health Related

Abstract Background Sickle cell disease (SCD) is a group of disorders that affects hemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. People with this disorder have atypical hemoglobin molecules called hemoglobin S, which can distort red blood cells into a sickle, or crescent, shape. Additionally, affected red blood cells have a high likelihood of stacking up and causing blockages in the small blood vessels. Both mechanisms of disease may cause damage to organs requiring oxygen, which causes pain and may be deadly. SCD and its complications reduce life expectancy and the nature of its complications and treatments may cause reduced quality of life. Although a rare disease, SCD may cause significant costs to healthcare and society. Depending on the indication, either hydroxyurea or chronic RBC transfusion is employed to treat SCD patients. RBC transfusion, simple or exchange, is first-line treatment for primary and secondary prevention of stroke. When the study was performed, no published research had been completed that compared patient outcomes in terms of Health-Related Quality of Life (HRQoL) for patients treated with automated red blood cell exchange versus simple transfusion. Objectives There are multiple objectives covered within the scope of this study:To quantify HRQoL as experienced by SCD patients in aRBCx versus simple transfusion.To determine the drivers of HRQoL amongst SCD patientsTo assess whether physicians and patients have a similar view on the impact of aRBCx on HRQoL amongst SCD patients. Methodology A cross-sectional study was performed amongst 40 SCD patients, 20 from the USA, 10 from France and 10 from the UK as well as amongst 40 SCD treating physicians with experience in both simple transfusion as well as aRBCX. The physicians had the same regional distribution as the patients. Results SCD patients undergoing aRBCX reported an HRQoL that was 25% higher compared to the period where they were treated with simple transfusion (0.70 vs. 0.55; p<0.01). The main drivers of HRQoL identified were (correlation efficient): pain reduction (0.57), improved social live (0.49), autonomy in terms of all day living activities and being independent from others (0.56), feeling energetic and physical functioning (0.57) and lastly emotional worry and mental health (0.56), all with p-values < 0.01. Together these variables explain 39% of the HRQoL experienced by SCD patients (R² = 0.39, p < 0.01). 80% of the patients preferred aRBCx over simple transfusion. 87% of the participating physicians believed that switching patients from simple transfusion to automated red blood cell exchange (aRBCX) positively affected the SCD patients' quality of life. Physicians identified the following factors being responsible for the improved HRQoL in patients on aRBCX. Those with an average score of 5.5 or greater on a 7-point scale were: less iron overload, RBCX effectiveness, reduced acute complications, reduced chronic complications, and superior mechanism of action during acute situations. Conclusion Sickle cell disease patients that require chronic blood transfusion experience better health-related quality of life when they are treated with automated red blood cell exchange versus simple transfusion. This observation is supported by the opinion of their treating physicians. Disclosures Dierick: Terumo BCT: Employment. Roig:Terumo BCT: Employment.

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.

Eltrombopag for the Treatment of Thrombocytopenia of Low and Intermediate-1 IPSS Risk Myelodysplastic Syndromes: Interim Results on Efficacy, Safety and Quality of Life of an International, Multicenter Prospective, Randomized, Trial

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 91-91 ◽  
Author(s):  
Esther Natalie Oliva ◽  
Valeria Santini ◽  
Caterina Alati ◽  
Antonella Poloni ◽  
Alfredo Molteni ◽  
...  
Keyword(s):  
Blood Loss ◽  
Study Design ◽  
Research Funding ◽  
Performance Status ◽  
Advisory Board ◽  
Refractory Anemia ◽  
Severe Thrombocytopenia ◽  
Ecog Performance Status ◽  
Ringed Sideroblasts

Abstract Background: About 10% of low risk patients with myelodysplastic syndromes (MDS) experience severe thrombocytopenia. Bleeding and the scarce efficacy of platelet (PLT) transfusions drive research in novel treatments. Eltrombopag is an oral agonist of the thrombopoetin-receptor (TPO-R). Its potential in increasing platelet (PLT) counts in low risk MDS has not been evaluated. We present interim results on the efficacy and safety of eltrombopag in inducing PLT responses in patients with low and intermediate-1 International Prognostic Scoring System (IPSS) risk MDS with severe thrombocytopenia in a Phase II, multicentre, prospective, placebo-controlled, single-blind study (EQoL-MDS). Methods: Primary endpoints are safety and efficacy of eltrombopag. Secondary endpoints include changes in quality of life (QoL), PLT transfusion requirement, incidence and severity of bleeding, and survival. Inclusion criteria are adult age; PLT<30 Gi/L; ECOG performance status < 4; ineligibility for, relapsed or refractory to other treatments; and naive to TPO-R agonists. Eltrombopag/placebo (2:1) is administered at a 50 mg daily starting dose with 50 mg increases every 2 weeks to maximum 300 mg to target PLT 100 Gi/L. Dose interruptions or reductions are required for PLT >200 Gi/L or adverse events. Study design is shown in the figure. PLT response, assessed at each visit, is defined as Response if: 1) baseline PLT>20 Gi/L: absence of bleeding and increase by at least 30 Gi/L from baseline; 2) baseline PLT<20 Gi/L: PLT>20 Gi/L and increase by at least 100%, not due to PLT transfusions; and Complete Response if PLT≥100 Gi/L and absence of bleeding. QoL scores are analysed by MDS-specific instrument, QOL-E v. 3. Results: Seventy patients (46 on eltrombopag - Arm A, 24 on placebo -Arm B) have been randomized at the time of this report. Mean age is 68.3 (SD 13.0) years, M/F 38/32. ECOG performance status was 0 in 47 cases, 1 in 16 cases, 2 in 7 cases. Ten patients had comorbidities. According to the WHO 2008 classification, 22 patients had refractory cytopenia with unilineage dysplasia, 9 had refractory anemia with ringed sideroblasts, 31 had refractory cytopenia with multilineage dysplasia (of which 15 with ringed sideroblasts), 6 had refractory anemia with excess blasts-1 and 2 were unclassified. IPSS score was low in 48 cases. Mean baseline platelet (PLT) count was 17.1 (SD 8.2) Gi/L, mean hemoglobin level 10.8 (SD 2.5) g/dL and mean white blood cell count was 5.0 (SD 3.8) Gi/L. Twenty-five (36%) patients were red blood cell transfusion-dependent. Thirty-three had a WHO bleeding scale of 1, 2 experienced mild blood loss, 4 a gross blood loss and 1 a debilitating blood loss. Fourteen patients in Arm A and 8 in Arm B had required PLT transfusions in the 8 weeks prior to randomization. Twenty-three cases (50%) in Arm A have responded versus 2 (8%) in Arm B (p=0.001). Thirty-three patients have completed at least 24 weeks of study. Median time to response was 14 days (IQR 7-46 days) at a median daily dose of 75 (IQR 50-162.5 mg). PLT count increased by mean 53.2 (SD 68.1) Gi/L (p=0.001) in Arm A versus no significant changes in Arm B by week 24. QOL-E scores at baseline and 12 weeks in 47 cases in both arms are shown in the table. There was an increase in treatment outcome index,mainly experienced in the first 3 weeks (p=0.034). Fatigue improved from baseline to 12 weeks associated with response (p=0.016). Related Grade III-IV adverse events (AE) occurred in 10 patients (22%) in Arm A and consisted in: nausea (4), hypertransaminasemia (3), hyperbilirubinemia (1), sepsis (1), pruritis (1), heart failure (1), asthenia (1), vomit (1), while in Arm B 1 patient (4 %) experienced grade 3 bone marrow fibrosis. MDS disease progression occurred in 5 (11%) in Arm A versus 2 (8%) in Arm B, p=ns. Conclusions: Preliminary data indicate that lower risk MDS patients with severe thrombocytopenia undergoing treatment with eltrombopag experience significant improvements in PLT counts accompanied by improvements in fatigue. The drug appears to be well-tolerated and not associated with MDS progression. Further follow-up is required to evaluate the impact on survival. Figure 1. Study design Figure 1. Study design Figure 2. QOL-E domains at baseline and 12 weeks between Arm A (eltrombopag) and Arm B (placebo) Figure 2. QOL-E domains at baseline and 12 weeks between Arm A (eltrombopag) and Arm B (placebo) Disclosures Oliva: Novartis: Speakers Bureau; Celgene: Other: Advisory Board, Speakers Bureau; Amgen: Consultancy. Santini:celgene, Janssen, Novartis, Onconova: Honoraria, Research Funding. Palumbo:Novartis: Honoraria, Other: Advisory Board. Fenaux:Novartis: Honoraria, Research Funding; Janssen: Honoraria, Research Funding; Celgene Corporation: Honoraria, Research Funding; Amgen: Honoraria, Research Funding.

Impact of a new dosing regimen of epoetin alfa on quality of life and anemia in patients with low-risk myelodysplastic syndrome

2004 ◽  
Vol 84 (3) ◽  
pp. 167-176 ◽  
Author(s):  
M. A. Aloe Spiriti ◽  
R. Latagliata ◽  
P. Niscola ◽  
A. Cortelezzi ◽  
M. Francesconi ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Myelodysplastic Syndrome ◽  
Low Risk ◽  
Epoetin Alfa ◽  
Dosing Regimen
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