Health-Related Quality of Life In Patients with Chronic Myeloid Leukemia Undergoing First Line Treatment with Imatinib for at Least Three Years Compared with the General Population. A Multicenter Study Including 448 Patients

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 2273-2273
Author(s):  
Fabio Efficace ◽  
Michele Baccarani ◽  
Massimo Breccia ◽  
Giuliana Alimena ◽  
Gianantonio Rosti ◽  
...  

Abstract Abstract 2273 Background: While Imatinib (IM) has revolutionized treatment for chronic myeloid leukemia (CML), demonstrating outstanding survival figures, currently no data exist on mid to long term impact of disease burden and therapy from the patients’ perspective. Aim: The main objective of this study is to identify specific limitations of quality of life (QoL) in CML survivors who are undergoing first line treatment with IM in comparison with controls from the general population. Patient-reported symptom prevalence was also investigated. Patients and methods: Patients were recruited in 26 centers, randomly selected to geographically represent the whole study country. Patients selection criteria included: being in treatment with IM for at least three years and being in complete cytogenetic response at the time of study entry. All patients were invited by their treating physicians in the hospital to participate and all consenting patients were requested to complete a Health Survey Packet at home. Pre-paid reply envelopes were also provided with the request to send back completed Surveys to an independent National coordinating Data Center. Generic QoL was assessed with the SF-36 that consists of 36 items covering eight generic health status/QoL domains: physical functioning (PF), role limitations due to physical health (RP), bodily pain (BP), general health perceptions (GH), vitality (VT), social functioning (SF), role limitations due to emotional problems (RE), and mental health (MH). All scales ranging between 0 and 100 with higher scores representing better outcomes. A previously devised patient-reported CML Symptom Checklist was used to investigate 9 symptoms of possible major concern in these patients. Mean SF-36 scores were compared to available national general population reference values and all analyses were adjusted for age and gender. Statistical comparisons were all adjusted for multiple testing. Differences in mean scores were expressed in Cohen effect sizes (ES; with 0.2, 0.5, and 0.8 indicating small, medium, and large ES, respectively) and clinical significance. Results: Between March and December 2009, 448 patients were recruited in a large national-based survivorship project. Patients’ compliance was optimal with 94% of patients (N=421) returning a valid Health Survey Packet to the National coordinating Data center. At study participation, mean age of patients was 56 years (59% male and 41% female) and median time of IM therapy was 5 years. Seventy-seven percent of patients were receiving standard dose of 400 mg and 43% had at least one comorbidity. Age and gender adjusted comparisons with general population norms revealed worse outcomes for the following scales: RP (P<.001; ES=0.3), GH (P<.001; ES=0.4) and RE (P=.01; ES=0.2). The largest clinically meaningful difference (Δ=12.3 points) was found for the RP domain with mean scores of 61.4 vs. 73.7 respectively for the CML and the general population. Age specific comparisons, adjusted by gender, (55-64; 65–74 and >75 years) suggested an almost uniform pattern in all scales with worse outcomes between CML patients and population controls among the youngest groups. GH was significantly worse in younger patients (55-64) (P=.03; ES=0.4) and no differences were found in the older age groups compared with population norms. Prevalence of reported symptoms (with any level of concern) was: fatigue (82%); problems with muscular cramps (78%); problems with musculoskeletal pain (72%); problems with edema (70%); skin problems (47%); diarrhea (43%); headache (39%); abdominal discomfort and nausea (28%). Conclusion: This study suggests that while still being on treatment with IM for years, CML patients might expect to have a QoL profile broadly similar to that of general population in many areas. However, role limitations (i.e., in work or other regular daily activities) due to physical health seem the major constraint faced by these patients; there is also an indication that younger patients might be those experiencing major limitations. Additional analyses will be undertaken to ascertain the impact of symptoms and other laboratory and clinical data on specific QoL domains. Such unique patient-reported data supplements conventional information on clinical efficacy of IM and may support both clinicians and patients in making more informed treatment decisions in this area. Disclosures: Rosti: Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Bristol M. Squibb: Honoraria, Speakers Bureau; Roche: Speakers Bureau.

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 2056-2056
Author(s):  
Fabio Efficace ◽  
Paola Fazi ◽  
Francesco Rodeghiero ◽  
Gianluca Gaidano ◽  
Piercarla Schinco ◽  
...  

Abstract Abstract 2056 Background: Recent international guidelines (Rodeghiero F, et al. Blood 113:2386–93, 2009) emphasize the importance of health-related quality of life (HRQOL) in patients with primary immune thrombocytopenia (pITP) and are advocating for more research in this area. However, very little research has been conducted on HRQOL of these patients. Aim: The main objective of this study was to identify specific limitations of HRQOL in pITP patients (ie, chronic, persistent and newly diagnosed patients) in comparison with their peers from the general population. Also, socio-demographic and clinical factors were considered to evaluate HRQOL impairment. Patients and Methods: Data were gathered through an ongoing multicenter observational study that recruits p-ITP patients. HRQOL was the primary endpoint of the study and was assessed with Medical Outcomes Study 36-Item Short-Form Health Survey (SF-36) that consists of 36 items covering eight generic health status/QoL domains: physical functioning (PF), role limitations due to physical health (RP), bodily pain (BP), general health perceptions (GH), vitality (VT), social functioning (SF), role limitations due to emotional problems (RE), and mental health (MH). All scales ranging between 0 and 100 with higher scores representing better outcomes. Two summary scores, namely the physical component summary (PCS) and the mental component summary (MCS) are derived from a weighted combination of the eight scales. Mean SF-36 scores were compared to available national general population reference values and analyses were adjusted for age and gender. Based on previous research, eight points were considered to be a minimally important difference (MID) for the first eight SF-36 scales, while a difference of two points was judged as MID for the PCS and the MCS scores. A score difference at least equal to MID was considered as a clinically meaningful difference. Socio-demographic, clinical and laboratory data were also collected to investigate their association with HRQOL outcomes. Univariate and multivariate linear regression analyses were used. Results: To date of the 256 pITP patients included in this study, 69%, 16% and 15%, were diagnosed with chronic, persistent and newly diagnosed pITP respectively. Present analysis is based on 175 patients with HRQOL data currently available. At study participation, mean age of patients was 54 years (67% female and 33% male). At least one comorbidity was present in 53% of patients. The median time from initial diagnosis to study entry was 0.3, 8 and 77 months respectively for newly diagnosed, persistent and chronic patients. Age and gender adjusted comparisons with general population norms revealed worse outcomes for the following scales: RP (P<.001), GH (P=.01), SF (P=.002), RE (P=.002). The largest clinically meaningful difference (Δ=13.4 points) was found for the RP domain with mean scores of 58.3 vs. 71.7 respectively for pITP patients and the general population. Age specific comparisons, (18–54, 55–64 and >65 years) suggested an almost uniform pattern in all scales with worse outcomes between pITP patients and population controls among the youngest groups. Statistically and clinically meaningful differences (ie, >8 points) were found in five out of the eight scales of the SF-36 in the youngest group of patients (18–54 years), when compared with their peers, while these differences were not present in the other age group categories. Comorbidity was the main factor influencing HRQOL by independently predicting worse HRQOL outcomes across all domains of the SF-36. SF-36 mean score differences in patients with or without comorbidity, were more than twice the magnitude of a clinically meaningful difference for PF and RP, being respectively: 69.7 vs. 87 and 51.3 vs. 68 points. Conclusions: This study suggests that HRQOL of p-ITP patients is far from optimal. In particular, role limitations (i.e., in work or other daily activities) due to physical health seems the major constraint faced by these patients when compared to their peers in the general population. Comorbidity seems the major factor associated with a poorer HRQOL profile. These findings need to be confirmed with a larger sample size and will eventually help guide the development of patient-centered supportive care programs. Disclosures: No relevant conflicts of interest to declare.


Author(s):  
Tjaša Filipčič ◽  
Špela Bogataj ◽  
Jernej Pajek ◽  
Maja Pajek

Hemodialysis (HD) patients have lower functional abilities compared to healthy people, and this is associated with lower physical activity in everyday life. This may affect their quality of life, but research on this topic is limited. Therefore, the present study aimed to determine the relationship between habitual physical activity and quality of life in HD patients and healthy controls. Ninety-three HD patients and 140 controls participated in the study. Quality of life was assessed using a 36-item medical outcomes study short-form health survey (SF-36). Human Activity Profile (HAP) was used to assess habitual physical activity. The adjusted activity score (AAS) from HAP, age, gender, fat tissue index (FTI), lean tissue index (LTI), and Davies comorbidity score were analyzed as possible predictors of the Physical Component Summary (PCS) of the SF-36. Three sequential linear models were used to model PCS. In Model 1, PCS was regressed by gender and age; in Model 2 the LTI, FTI, and Davies comorbidity scores were added. Model 3 also included AAS. After controlling for age and gender (ModelHD 1: p = 0.056), LTI, FTI, and Davies comorbidity score effects (ModelHD 2: p = 0.181), the AAS accounted for 32% of the variation in PCS of HD patients (ModelHD 3: p < 0.001). Consequently, the PCS of HD patients would increase by 0.431 points if the AAS increased by one point. However, in healthy controls, AAS had a lower impact than in the HD sample (B = 0.359 vs. 0.431), while the corresponding effects of age and gender (ModelH 1: p < 0.001), LTI, FTI, and Davies comorbidity score (ModelH 2: p < 0.001) were adjusted for. The proportion of variation in PCS attributed to AAS was 14.9% (ModelH 3: p < 0.001). The current study results showed that physical activity in everyday life as measured by the HAP questionnaire is associated to a higher degree with the quality of life of HD patients than in healthy subjects. Routine physical activity programs are therefore highly justified, and the nephrology community should play a leading role in this effort.


2017 ◽  
Vol 35 (15_suppl) ◽  
pp. e18525-e18525
Author(s):  
Bhavik J. Pandya ◽  
Anna Hadfield ◽  
Bruno C. Medeiros ◽  
Samuel Wilson ◽  
Cat N. Bui ◽  
...  

e18525 Background: There is currently limited data on the quality-of-life (QoL) of patients with acute myeloid leukemia (AML) in the real-world setting. The objective of this analysis was to understand the impact of AML on patients receiving first-line treatment vs those who were relapsed/refractory to first-line treatment and therefore on later lines of therapy. Methods: The Adelphi AML Disease-Specific Programme, a real-world, cross-sectional survey involving 61 US hematologists/hemato-oncologists and their consulting AML patients, was conducted between February–May 2015. Physicians provided details on patient demographics and clinical information. Each patient was asked to complete both the EQ-5D-3L and Functional Assessment of Cancer Therapy Leukemia (FACT-Leu). Scores range from −1.09–1 (EQ-5D-3L) and 0–176 (FACT-Leu), where a higher score indicates a better QoL. Data from physician-completed record forms and corresponding patient self-completion forms on a matched sample of 75 patients were analyzed. Results: Of the patients who took part in the survey, 75% (n = 56) were receiving first-line treatment for AML and 25% (n = 19) were relapsed/refractory to first-line treatment and had progressed to later lines of therapy. The first-line patients had a mean age of 56.6 years and an average of 2.1 symptoms whereas the relapsed/refractory patients had a mean age of 56.9 years and an average of 2.4 symptoms, according to the physician. First-line patients may have a directionally better QoL scores than those on later lines of therapy, according to both the EQ-5D (0.75 and 0.71 respectively, P= .51) and the FACT-Leu (103.7 and 92.5 respectively, P= .098) measures. Results from the FACT-Leu-Physical Well-Being sub-domain show that relapsed/refractory patients were significantly more likely than first-line patients to be affected physically by their AML condition (13.0 and 17.6 respectively, P= .005). Conclusions: AML patients who have relapsed or become refractory to first-line treatment report worse QoL than those still on first-line treatments. These observational data shows a need for effective and tolerable treatments that can maintain or improve patients’ QoL, especially for patients with relapsed or refractory disease.


2020 ◽  
Vol 20 (1) ◽  
Author(s):  
Vivienne Kahlmann ◽  
◽  
Montse Janssen Bonás ◽  
Catharina C. Moor ◽  
Coline H. M. van Moorsel ◽  
...  

Abstract Background Treatment of pulmonary sarcoidosis is recommended in case of significant symptoms, impaired or deteriorating lung function. Evidence-based treatment recommendations are limited and largely based on expert opinion. Prednisone is currently the first-choice therapy and leads to short-term improvement of lung function. Unfortunately, prednisone often has side-effects and may be associated with impaired quality of life. Methotrexate is presently considered second-line therapy, and appears to have fewer side-effects. Objective The primary objective of this trial is to investigate the effectiveness and tolerability of methotrexate as first-line therapy in patients with pulmonary sarcoidosis compared with prednisone. The primary endpoint of this study will be the change in hospital-measured Forced Vital Capacity (FVC) between baseline and 24 weeks. Secondary objectives are to gain more insights in response to therapy in individual patients by home spirometry and patient-reported outcomes. Blood biomarkers will be examined to find predictors of response to therapy, disease progression and chronicity, and to improve our understanding of the underlying disease mechanism. Methods/design In this prospective, randomized, non-blinded, multi-center, non-inferiority trial, we plan to randomize 138 treatment-naïve patients with pulmonary sarcoidosis who are about to start treatment. Patients will be randomized in a 1:1 ratio to receive either prednisone or methotrexate in a predefined schedule for 24 weeks, after which they will be followed up in regular care for up to 2 years. Regular hospital visits will include pulmonary function assessment, completion of patient-reported outcomes, and blood withdrawal. Additionally, patients will be asked to perform weekly home spirometry, and record symptoms and side-effects via a home monitoring application for 24 weeks. Discussion This study will be the first randomized controlled trial comparing first-line treatment of prednisone and methotrexate and provide valuable data on efficacy, safety, quality of life and biomarkers. If this study confirms the hypothesis that methotrexate is as effective as prednisone as first-line treatment for sarcoidosis but with fewer side-effects, this will lead to improvement in care and initiate a change in practice. Furthermore, insights into the immunological mechanisms underlying sarcoidosis pathology might reveal new therapeutic targets. Trial registration The study was registered on the 19th of March 2020 in the International Clinical Trial Registry, www.clinicaltrials.gov; ID NCT04314193.


2021 ◽  
Vol 15 (12) ◽  
pp. 3150-3151
Author(s):  
Farhana Memon ◽  
Ashfaque Ahmed ◽  
Saima Sagheer ◽  
Fariya Usmani ◽  
Muhammad Naveed ◽  
...  

Aim: Effect of age and gender on quality of life in patients of obstructive jaundice. Methodology: This was a case control study conducted in OPD of General Surgery, Unit-1, Civil Hospital, Karachi. It is non-probability, consecutive sampling technique. The sample size was 195 subjects. 87 patients had an age between 18-60 years whereas 108 patients had >60 years of age. Either gender was included. QoL was measured by using SF-36 based on 8 domains. Data were analyzed through SPSS version-21 Results: Significant effect of age in obstructive jaundice patients on their QOL (p<0.01) where patients aged from 18 to 60 years had significantly higher SF-36 quality of life scores, both overall and in all eight individual domains than patients aged >60 years. Gender of the obstructive jaundice patients did not have a significant effect on their QOL, though the female patients had higher SF-36 quality of life scores, both overall and in all eight individual domains. Conclusion: Quality of life of obstructive jaundice patients were more improved with the age between 20-60 years than age >60 years, whereas gender had no significant impact on the quality of life. Keywords: Obstructive jaundice, age, gender, quality of life


Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 770-770 ◽  
Author(s):  
Fabio Efficace ◽  
Alessandro Rambaldi ◽  
Eros Di Bona ◽  
Francesco Fabbiano ◽  
Francesco Cottone ◽  
...  

Abstract Background The combination of all-trans retinoic acid (ATRA) and anthracycline-based chemotherapy regimens is currently considered the standard of care for newly diagnosed acute promyelocytic leukemia (APL) patients. This combination has greatly contributed to convert APL from a frequently fatal disease to a highly curable one. However, there is lack of data on the impact of such therapies on patients’ health-related quality of life (HRQOL). Objective The main objective of this study was thus to investigate long-term HRQOL of APL patients previously treated with ATRA plus anthracycline-based chemotherapy. The physical and mental HRQOL profile of these patients was compared with that of matched control subjects from the general population to identify specific areas most in need of attention in long-term follow-up care. A secondary objective was to outline symptoms’ burden from the patients’ perspective. Patients and Methods Data were gathered through an ongoing multicenter survivorship study that recruits APL patients previously enrolled in two large GIMEMA trials (i.e., AIDA0493 and AIDA 2000). In both trials, APL patients were treated with ATRA plus Idrarubicin (AIDA). The main inclusion criterion was having survived the initial diagnosis for more than 5 years and being in complete remission (CR). Generic HRQOL was assessed with the SF-36 that consists of 36 items covering eight generic health status/QoL domains: physical functioning (PF), role limitations due to physical health (RP), bodily pain (BP), general health perceptions (GH), vitality (VT), social functioning (SF), role limitations due to emotional problems (RE) and mental health (MH). All scales ranged between 0 and 100, with the higher scores representing better outcomes. Clinical significance was evaluated and eight points were considered to be a minimally important difference for the eight SF-36 scales. Mean SF-36 scores were compared to available national general population reference values (i.e., 1997 subjects without cancer) and all analyses were adjusted for age and gender. Symptom burden was assessed according to the M.D. Anderson Symptom Inventory (MDASI). Symptom severity was assessed for the following symptoms: fatigue, pain, sleep disturbance, drowsiness, poor appetite, shortness of breath, nausea, vomiting, dry mouth, numbness, difficulty remembering, distress and sadness. All items were rated on a numeric rating scale from 0 to 10, with the higher scores indicating a higher level of symptoms. These were categorized as “mild” (ratings between 0 and 3) and “moderate to severe” (ratings between 4 to 10). Results Analysis is based on 136 adult APL patients who agreed to participate. At study participation, the mean age of patients was 52 years (55% males and 45% females) and the median time from diagnosis was 13 years (range: 4.5-20). Age and gender adjusted comparisons between APL patients and the general population norms revealed worse outcomes for the following scales: RP (P&lt;.001) and RE (P&lt;.001). Such differences were more than six times the magnitude of a clinically meaningful difference (i.e., at least 8 points), respectively for the physical (Δ=51 points) and the emotional scales (Δ=49 points) of the SF-36. Analysis within our patient cohort revealed that older APL patients (i.e., those aged more than 52 years) had a statically significant lower physical functioning (P&lt;.001) and higher pain severity (P=.0448) than younger ones. Investigation of the HRQOL profile of patients by trial participation (i.e., AIDA 0493 vs. AIDA 2000) or time since diagnosis (cut-off median time of 13 years) revealed no difference in all scales of the SF-36. Fatigue was the most burdensome symptom being reported as moderate to severe in 35% of patients. Other frequently reported moderate to severe symptoms were: difficulty in remembering (30%), sadness and distress (29%). Conclusions Although ATRA plus anthracycline-based chemotherapy regimens have greatly increased cure rates in APL, the HRQOL of these patients is heavily affected by the consequences of the disease and treatment, that persists many years after diagnosis and treatment. Disclosures: No relevant conflicts of interest to declare.


2020 ◽  
Vol 32 (10) ◽  
pp. 2041-2047
Author(s):  
Anna K. Ojala ◽  
Harri Sintonen ◽  
Risto P. Roine ◽  
Timo E. Strandberg ◽  
Camilla Schalin-Jäntti

Abstract Background Cardiovascular disease (CVD) and thyroid dysfunction are common in older people, but little is known about how they affect health-related quality of life (HRQoL). Methods We assessed HRQoL with the 15D instrument in 329 home-dwelling patients aged ≥ 75 years with stable CVD and compared the results to those of an age- and gender-matched general population (n = 103). We also studied the impact of age, BMI, number of medications, thyroid-stimulating hormone (TSH) concentration, levothyroxine (L-T4) substitution and Mini-Mental State Examination (MMSE) on HRQoL. Results Overall HRQoL was impaired in older people with stable CVD (mean 15D score 0.777 vs 0.801, p = 0.001), and also on single dimensions of breathing, sleeping, discomfort and symptoms, distress, vitality (all p < 0.001), and depression (p = 0.016) compared to the age- and gender-matched general population. Furthermore, in the patients, L-T4 substitution associated with impaired sleeping (p = 0.018) and sexual activity (p = 0.030). Moreover, MMSE points, number of medications used, age (all p < 0.001) and BMI (p = 0.009) predicted impaired HRQoL. Conclusions Older people with stable CVD are characterized by impaired HRQoL compared to age- and gender-matched controls. We demonstrate that this is the consequence of impaired breathing, sleeping, discomfort and symptoms, distress, vitality, and depression. L-T4 substitution has a negative impact on HRQoL in old patients with stable CVD. MMSE score, number of medications, age and BMI predict worse HRQoL.


2020 ◽  
Vol 7 (2) ◽  
pp. 205-211
Author(s):  
Kaynat Fatima ◽  
Syed Tasleem Raza ◽  
Ale Eba ◽  
Sanchita Srivastava ◽  
Farzana Mahdi

The function of protein kinases is to transfer a γ-phosphate group from ATP to serine, threonine, or tyrosine residues. Many of these kinases are linked to the initiation and development of human cancer. The recent development of small molecule kinase inhibitors for the treatment of different types of cancer in clinical therapy has proven successful. Significantly, after the G-protein-coupled receptors, protein kinases are the second most active category of drug targets. Imatinib mesylate was the first tyrosine kinase inhibitor (TKI), approved for chronic myeloid leukemia (CML) treatment. Imatinib induces appropriate responses in ~60% of patients; with ~20% discontinuing therapy due to sensitivity, and ~20% developing drug resistance. The introduction of newer TKIs such as, nilotinib, dasatinib, bosutinib, and ponatinib has provided patients with multiple options. Such agents are more active, have specific profiles of side effects and are more likely to reach the necessary milestones. First-line treatment decisions must be focused on CML risk, patient preferences and comorbidities. Given the excellent result, half of the patients eventually fail to seek first-line treatment (due to discomfort or resistance), with many of them needing a third or even further therapy lines. In the present review, we will address the role of tyrosine kinase inhibitors in therapy for chronic myeloid leukemia.


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