scholarly journals The Clinical, Economic, and Humanistic Burden of Patients with β-Thalassemia: A US-Centric Review of the Literature

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 5855-5855
Author(s):  
Elizabeth S. Mearns ◽  
Chuka Udeze ◽  
Ronda Copher ◽  
Sujit Sheth

Introduction: β-thalassemia is a genetic blood disease characterized by ineffective erythropoiesis due to defective hemoglobin production that manifests as potentially fatal anemia. Patients with β-thalassemia rely on regular, lifelong red blood cell transfusions (RBCTs) for survival and suffer from multi-system complications, physical manifestations, and decreased health-related quality of life (HRQoL) due to iron overload and continual RBCTs. This literature review aims to describe the disease burden associated with β-thalassemia. Methods: Electronic databases (MEDLINE®, Embase®, Cochrane Library) were searched to identify articles in the English language assessing the clinical, economic, and humanistic burden of β-thalassemia in North America and Europe. Articles were selected based on predefined criteria including appropriate study design, disease state, country, and outcome measures. Results: Patients with β-thalassemia who received regular RBCTs were significantly more likely to develop ≥ 1 iron overload-related comorbidity than matched, non-thalassemia controls (P < 0.0001). Other iron-related issues included cardiac disease (observed in 13-30% of patients), hypogonadism (17-22%), diabetes (10-18%), osteoporosis (16%), hypothyroidism (12%), liver disease (4%), and hypoparathyroidism (2%). RBCTs also introduced the risk of transfusion reactions (48-50%), infections (24-65%), and development of alloantibodies (19-23%). Mortality rate for the US population was 1.9%. The direct annual cost per regularly transfused patient with β-thalassemia was significantly higher than in matched controls (USD 128,062 vs USD 5,438; P < 0.001; 2016 USD). The main annual cost drivers per patient were iron chelation therapy (48%; USD 61,974) and RBCTs (31%; USD 39,723). Indirect costs for β-thalassemia were impacted by unemployment, reported at 33% among patients with β-thalassemia versus 24% for the general US population. Patients scored significantly worse on 5 subdomains (physical functioning, role-physical, general health, social functioning, and role-emotional) of the Short Form-36 (SF-36) HRQoL questionnaire compared with the general US population (all P < 0.0001) with the greatest impairments seen in the general health subdomain (P < 0.0001). Clinically meaningful reductions in physical functioning, role-physical, and general health scores were observed for patients with β-thalassemia aged < 65 years with transfusion-related iron overload. Bodily pain was reported by 56-69% of patients with β-thalassemia, and pain interfered with patient physical and emotional functioning HRQoL outcomes. Increasing age correlated with higher numbers of pain responses (P < 0.001). Psychiatric comorbidities were found in 24% of patients with β-thalassemia, and mood disorders, including depression and anxiety, were associated with overall significantly poorer physical and mental HRQoL (P < 0.001). Conclusions: β-thalassemia is associated with a substantial clinical, economic, and humanistic burden in the USA. Patients with β-thalassemia experience reduced life expectancy, significant comorbidities, considerable direct healthcare costs, unemployment, and impaired HRQoL. There is a need for more effective management and therapeutic options for these patients. Disclosures Mearns: Celgene Corporation: Employment. Udeze:Celgene Corporation: Employment. Copher:Celgene Corporation: Employment. Sheth:CRSPR/Vertex: Other: Clinical Trial Steering committee; Apopharma: Other: Clinical trial DSMB; Celgene: Consultancy.

2021 ◽  
pp. 194589242110016
Author(s):  
Tran B. Locke ◽  
Auddie M. Sweis ◽  
Jennifer E. Douglas ◽  
Kevin I. Ig-Izevbekhai ◽  
Elizabeth M. Stevens ◽  
...  

Background Aspirin-exacerbated respiratory disease (AERD) is optimally managed by endoscopic sinus surgery (ESS) followed by aspirin therapy after desensitization (ATAD). Most AERD quality of life (QOL) studies use the 22-item Sinonasal Outcomes Test (SNOT-22), which focuses predominantly on sinonasal outcomes. Objective This study seeks to assess QOL outcomes in AERD patients after ESS and ATAD via the 12-item Short Form Survey (SF-12), a well-validated QOL measure for general health status of chronic conditions. Methods Retrospective review of 112 AERD patients who underwent ESS followed by ATAD at our institution between 2016 and 2019. SF-12 was collected preoperatively, postoperatively/pre-AD, and serially post-AD (1–3, 4–6, 7–12, and >12 months). Optum® PRO CoRE software was used to compare data to national norms. ANOVA was performed comparing physical component summary (PCS), mental component summary (MCS) and eight health domains (physical functioning, role physical, general health, bodily pain, vitality, social functioning, role emotional, and mental health). Results AERD patients showed improvement in PCS scores across all timepoints after ESS and ATAD (p = 0.004). When stratified by gender, women demonstrated an improvement in PCS scores (p = 0.004). Within the domains, there were significant improvements in social functioning (SF), role physical (RP), and bodily pain (BP) at all timepoints (SF: p = 0.006; RP: p = 0.005; BP: p < 0.001). Conclusions AERD patients undergoing ESS and ATAD show improvement in physical QOL and 3 of the 8 health domains as measured by the SF-12. Future studies can use the SF-12 to study the impact of AERD treatment versus other chronic diseases and health demographics.


Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 1307-1307 ◽  
Author(s):  
John B Porter ◽  
Donald Bowden ◽  
Arnold Ganser ◽  
Gabor Domokos ◽  
Adam Gater ◽  
...  

Abstract Introduction: Iron chelation therapy (ICT) is essential in removing excess iron deposited in body organs, ultimately preventing organ failure and extending the lives of patients (pts) with transfusion-dependent hematological disorders such as β-thalassemia and myelodysplastic syndromes (MDS). As a life-long treatment, traditional ICT (deferoxamine, Desferal®, DFO) is based on a burdensome regimen (subcutaneous delivery 5–7 times a week) that has been shown to negatively impact on pts’ health-related quality of life (HRQoL). The oral chelator deferasirox (Exjade®) is less burdensome to pts offering 24-hour ICT, 7 days a week. Methods: This substudy was part of a single arm, multicenter, 1-year open-label trial (the EPIC study) to investigate the efficacy/safety of deferasirox. The first 558 pts with a variety of hematological disorders were recruited. These pts came from sites in seven countries: Australia, Belgium, France, Germany, UK, Greece, and Italy. Treatment-naïve pts and those having previously received ICT (DFO or deferiprone [Ferriprox®] exclusively, or combined) participated (n=558). Pts were asked at baseline, week 4 and week 52 (end of study [EOS]) to complete the 36-item Short Form health survey (SF-36). The SF-36 is a self-administered questionnaire and measures eight HRQoL domains: physical functioning; role-physical; bodily pain; general health; vitality; social functioning; role-emotional; and mental health. Mean change in SF-36 domain scores were calculated for all pts who had completed data at baseline and week 4, as well all those with completed data at baseline and EOS. All domains are scored so that higher scores indicate a better QoL. Results: Overall, the mean age of the 558 pts (274 β-thalassemia, 168 MDS, 50 sickle cell disease and 66 other anemias) recruited to take part in this substudy was 40.8 years (SD=22.58); 51.5% of patients (n=289) were male and 48.5% (n=272) were female. Within this sample, 337 pts aged ≥16 years completed the SF-36 at baseline, 322 at week 4 and 277 at EOS. Mean domain scores for pts at baseline, week 4 and EOS are presented in Table 1. With the exception of role-emotional (mean=0.78, SD=40.56), mean change in SF-36 domain scores significantly improved (P&lt;0.05) for all domains between baseline and week 4: physical functioning (mean=2.42, SD=17.44); role-physical (mean=5.67, SD=41.70); bodily pain (mean=5.96, SD=24.15); general health (mean=0.33, SD=14.44); vitality (mean=2.54, SD=15.93); social functioning (mean=2.51, SD=23.38); mental health (mean=1.98, SD=14.71). At EOS, mean change in SF-36 domain scores improved for all domains with the exception of social functioning, role-emotional and mental health. However, unlike results at week 4, none of the mean change domain scores at EOS reached statistical significance, possibly due to sample size decrease between week 4 and EOS. Table 1. SF-36 domain scores at baseline, week 4 and EOS in pts aged 3 16 years and treated with deferasirox SF-36 domains Baseline mean (SD) Week 4 mean (SD) End of study mean (SD) Physical functioning 66.32 (25.98)&#x2028; n=336 69.70 (25.98)&#x2028; n=317 71.67 (26.95)&#x2028; n=275 Role-physical 54.33 (42.43) n=331 61.03 (42.69) n=315 62.38 (41.71) n=270 Bodily pain 65.80 (26.89)&#x2028; n=336 74.67 (26.16)&#x2028; n=322 73.11 (27.20)&#x2028; n=276 General health 46.62 (21.15)&#x2028; n=330 48.01 (22.32)&#x2028; n=311 48.42 (22.19)&#x2028; n=269 Vitality 51.12 (21.28)&#x2028; n=328 54.57 (22.06)&#x2028; n=316 55.80 (23.06)&#x2028; n=272 Social functioning 71.73 (25.74)&#x2028; n=336 74.77 (23.72)&#x2028; n=321 73.05 (24.71)&#x2028; n=276 Role-emotional 68.05 (40.92)&#x2028; n=326 69.60 (41.60)&#x2028; n=313 69.81 (40.44)&#x2028; n=270 Mental health 67.04 (19.85)&#x2028; n=328 70.40 (19.73)&#x2028; n=316 67.85 (20.19)&#x2028; n=272 Conclusions: Since mean change scores were often of the magnitude of 3 to 5 units for role-physical and bodily pain, these results indicate clinically meaningful improvement for pts with hematological disorders receiving deferasirox.


2013 ◽  
Vol 154 (47) ◽  
pp. 1865-1872 ◽  
Author(s):  
Emese Pék ◽  
István Mártai ◽  
József Marton ◽  
József Betlehem

Introduction: The high work-related stress among ambulance workers is a widely known a phenomenon. Aim: The aim of the authors was to asses self-reported health status of ambulance workers. Method: An anonym self-fill-in questionnaire applying SF-36 was used among workers from the northern and western regions of Hungarian National Ambulance Service. Results: Based on the dimensions of the SF-36 questionnaire the responders considered their “Physical Functioning” the best, while “Vitality” was regarded the worst. The more time an employee have been worked at the Ambulance Service the worse his health was in the first four dimensions including “Physical Functioning”, “Role-Physical”, “Bodily Pain” and “General Health”: p<0.001. Those working in part-time jobs considered their health in all dimensions worse. The respondents who did some kind of sports hold their health in all dimensions better (p<0.001). The workers with higher body mass index regarded their health status worse, in four dimensions: “Physical Functioning” (p = 0.001), “Role-Physical” (p = 0.013), “General Health” (p<0.001) and “Role-Emotional” (p = 0.05). Conclusions: According to the subjective perception of health and measurable parameters of health status of workers proved to be insufficient. Poor physical health can lead indirectly to psychological problems, which may lower the quality of the work and can lead to high turn-over. Orv. Hetil., 154(47), 1865–1872.


2000 ◽  
Vol 6 (5) ◽  
pp. 338-342
Author(s):  
J HA Arnoldus ◽  
J Killestein ◽  
L EMA Pfennings ◽  
B Jelles ◽  
B MJ Uitdehaag ◽  
...  

Objectives: To determine the quality of life (QoL) of MS patients during the initial 6 months of treatment with interferon-b (IFN-b). Furthermore, to determine whether changes in QoL relate to disability, emotional state, therapeutic expectations or side effect profile. Background: IFN-b has been shown to have beneficial effects on the course of MS. Since the aim of IFN-b treatment is not to cure but to slow down the disease it is important to know how this treatment affects QoL. Surprisingly, the impact of treatment with IFN-b on QoL measures has not been extensively studied so far. Methods: Case report documentation, including EDSS, SF-36 and MADRAS scores, of 51 relapsing-remitting MS patients treated with IFN-b was obtained at baseline and at months 1, 3 and 6. Patients also filled in a form about their expectations of therapy and a questionnaire on side effects. Results: During treatment there was a significant linear trend indicating improvement in the role-physical functioning (RPF) scale of the SF-36 (F1,50=4.9, P=0.032). A transient decrease at month 1 was found in the scale for bodily pain, indicating more experienced pain (F1,50=19.8, P50.001). Subgroup analysis showed that patients with most depressive symptoms on the MADRAS at baseline contributed most to the increase in RPF scores over time (F1,24=5,6 P=0.026). Furthermore, we found associations between adverse event scores and several domains of QoL. Conclusions: Our findings suggest that IFN-b therapy has an impact on QoL of MS patients in that it improves role-physical functioning and transiently worsens experienced bodily pain. QoL during treatment with IFN-b is influenced by depressive symptoms at baseline as well as by treatment-associated side-effects.


F1000Research ◽  
2019 ◽  
Vol 8 ◽  
pp. 859 ◽  
Author(s):  
Lucia Giles ◽  
Caroline Freeman ◽  
Polly Field ◽  
Elisabeth Sörstadius ◽  
Bernt Kartman

Background: Heart failure (HF) is increasing in prevalence worldwide. This systematic review was conducted to inform understanding of its humanistic and economic burden. Methods: Electronic databases (Embase, MEDLINE®, and Cochrane Library) were searched in May 2017. Data were extracted from studies reporting health-related quality of life (HRQoL) in 200 patients or more (published 2007–2017), or costs and resource use in 100 patients or more (published 2012–2017). Relevant HRQoL studies were those that used the 12- or 36-item Short-Form Health Surveys, EuroQol Group 5-dimensions measure of health status, Minnesota Living with Heart Failure Questionnaire or Kansas City Cardiomyopathy Questionnaire. Results: In total, 124 studies were identified: 54 for HRQoL and 71 for costs and resource use (Europe: 25/15; North America: 24/50; rest of world/multinational: 5/6). Overall, individuals with HF reported worse HRQoL than the general population and patients with other chronic diseases. Some evidence identified supports a correlation between increasing disease severity and worse HRQoL. Patients with HF incurred higher costs and resource use than the general population and patients with other chronic conditions. Inpatient care and hospitalizations were identified as major cost drivers in HF. Conclusions: Our findings indicate that patients with HF experience worse HRQoL and incur higher costs than individuals without HF or patients with other chronic diseases. Early treatment of HF and careful disease management to slow progression and to limit the requirement for hospital admission are likely to reduce both the humanistic burden and economic impact of HF.


2019 ◽  
Vol 41 ◽  
pp. e45525
Author(s):  
Carlos Augusto Faria ◽  
Dayse Mary da Silva Correia ◽  
Karen Soto Perez Panisset ◽  
Maria Luiza Garcia Rosa

The objective of this study was to identify the factors associated with the impairment of quality of life (QoL) in community-dwelling individuals with LUTS. A randomized sample of the population registered in the Family Health Program - Niterói aged 45 years or over was selected. Information about demographic, socioeconomic and lifestyle factors, co-morbidities and nocturia was collected. The NANDA-I taxonomy was used to identify the other LUTS, and QoL evaluation was performed in accordance with the SF-36 Short Form questionnaire (SF36-SF). For the SF36-SF domains (outcome) associated with LUTS, multiple logistic models were tested including the urinary symptoms and the sociodemographic and associated clinical variables. Stress urinary incontinence was associated with white skin, female gender, obesity, smoking, alcohol intake, depression and low scores in all evaluated domains of QoL. Nocturia was associated with advanced age, low schooling level, higher BMI, hypertension, diabetes, health insurance and the lowest scores in all evaluated domains of Qol, except for the Role Emotional. According to multivariate analysis, stress incontinence and depression are associated with the highest risks of low scores in General Health, Physical Functioning and Vitality domains, while nocturia and obesity showed association with the highest risks of low scores in Physical Functioning, Bodily-Pain and Vitality domains.


Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 5513-5513 ◽  
Author(s):  
I. Thuret ◽  
C. Brun-Strang ◽  
D. Bachir ◽  
M. Hacini ◽  
B. Pegourie-Bandelier ◽  
...  

Abstract Purpose and Methods ISOSFER is an epidemiological cross-sectional study with prospective recruitment whose objectives are to evaluate patient characteristics, quality of life (QoL), compliance and patient satisfaction with deferoxamine (DFO) therapy. Results Among 278 consecutive patients receiving regular transfusions for thalassemia major (TM), sickle cell disease (SCD) or myelodysplastic syndromes (MDS) who consulted between October 2005 and February 2006 in 24 French centers, 161 were on chelation therapy. 124 patients were treated with DFO alone for more than 1 year. Among them, 67 aged 14 years or more agreed to participate. QoL was studied using the Short Form-36 scale. Compliance was assessed with the Morisky scale and a specific questionnaire. DFO was administered via subcutaneous (sc) infusion for 70% of patients, mainly nightly and with a mean duration of 10 hours. Other ways of administering DFO included intravenous (iv) infusion (15%), sc bolus (9%) and combined sc and iv treatment (5%). Patient characteristics are summarized in the table below. In comparison with the French general population, all dimensions of QoL are impaired in all three diseases and in all dimensions (physical activities, emotional problems, social activities, bodily pain, and vitality). TM and SCD patients had a better physical activities score than MDS patients but experienced more bodily pain than MDS patients. The global physical score was significantly influenced by age, marital status and occurrence of a co-morbidity related to hemosiderosis. Good compliance scores (0 and 1 on the Morisky scale) were found in only 74%, 67% and 87% of TM, SCD, MDS patients, respectively. In addition, DFO infusion was missed at least once a week during the past month in 17%, 44% and 4% of TM, SCD, MDS patients, respectively. Compliance was influenced by age, duration and number of weekly DFO infusions. Iron chelation with DFO was generally found to be inconvenient and more than half of the patients were unsatisfied with its parenteral mode of administration. Nonetheless, patients, particularly those with TM, were convinced that DFO was an effective treatment for iron overload and a very important drug for their health. Conclusions These results suggest that QoL is severely compromised in patient on DFO and that compliance to DFO is poor. These results could help ascertain the improvement in QoL and compliance, which would result from the use of oral chelation in patients with transfusional iron overload. TM (n=24) SCD (n=17) MDS (n=26) *Cardiac, liver and endocrine diseases, lens opacities, osteoporosis Median age in years (range) 30 (15–70) 32 (14–57) 69 (45–85) Sex, M/F 11/13 6/11 14/12 Geographic origin: EU/subS; Africa/North; Africa/others 16/0/6/2 1/12/3/1 26/0/0/0 Employment/university (%) 45 27 8 Marital status + (patients >18 yrs old) 9/21 7/15 25/26 Organ dysfunction potentially related to hemosiderosis* (%) 75 47 54 Median ferritin level (μg/L) 1049 2653 2627 Nb of TF >100 (%) 100 82 42 Mean DFO frequency/week 3.7 4.5 4 Mean dose 40 17 43


2005 ◽  
Vol 152 (5) ◽  
pp. 713-717 ◽  
Author(s):  
Salman Razvi ◽  
Lorna E Ingoe ◽  
Carolyn V McMillan ◽  
Jolanta U Weaver

Objective: Sub-clinical hypothyroidism (SCH) is a common disorder. People with this condition may have symptoms which could affect their perception of health. Therefore, the perceived health status of people with SCH was assessed and compared with population-matched norms. Design: A prospective cross-sectional survey. Methods: Seventy-one adults with SCH, age range 18–64 years were studied. Perceived health status was measured by the Short Form-36 (SF-36) version 2 questionaire, which has been validated in a UK population setting. The SF-36 has eight scales measuring physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional and mental health. Their SF-36 scores were compared with UK normative data after matching for age and sex and are reported as z-scores. Results: Scores of all eight SF-36 scales were significantly lower in people with SCH compared with the normative population. A negative score (compared with zero of the normative population) indicates worse health status. The most significantly impaired aspects of health status were vitality and role limitations due to physical problems (role physical scale) with z-scores (95% confidence intervals) of −1.01 (−0.74 to −1.29) and −0.73 (−0.43 to −1.04) respectively. Thyroid autoimmunity did not influence the results. Conclusion: Perceived health status is significantly impaired in people with SCH when compared with UK normative population scores. This needs to be taken into consideration by clinicians when managing patients with this disease.


2018 ◽  
Vol 1 (3) ◽  
Author(s):  
Andre Dharmawan Wijono

Pendahuluan: Epilepsi merupakan penyakit neurologis yang mempengaruhi kualitas hidup penderitanya. Hanya ada sedikit penelitian tentang kualitas hidup pasien epilepsi di Indonesia. Tujuan: Melihat hubungan antara usia, usia saat onset pertama muncul, dan durasi epilepsi terhadap kualitas hidup pasien epilepsi. Metode: Penelitian cross-sectional ini dilakukan di Departemen Neurologi, Rumah Sakit Bethesda, Yogyakarta, Indonesia pada November 2017 sampai Februari 2018. Pasien epilepsi yang berusia ≥ 18 tahun diikutkan dalam penelitian ini. Kualitas hidup diukur menggunakan instrument Short form 8 (SF-8). Hasil: Terkumpul 27 pasien yang memenuhi kriteria inklusi. Pasien dengan usia ≥ 60 tahun secara signifikan mengalami penurunan kualitas hidup dalam beberapa aspek, yaitu: physical functioning (42.44±8.243), general health (40.04±7.641), role emotional (39.60±7.638), mental health (43.50±10.347), dan mental component score (42.04±10.282). Pasien dengan usia saat onset pertama muncul ≥ 55 tahun secara signifikan memiliki role emotional (40.54±7.245) dan mental component score (42.98±10.155) yang lebih rendah. Durasi epilepsi tidak memiliki hubungan yang signifikan terhadap kualitas hidup pasien epilepsi. Kesimpulan: Penelitian ini menunjukkan bahwa usia dan usia saat onset epilepsi pertama muncul berhubungan dengan kualitas hidup pasien epilepsi.   Kata kunci: Epilepsi, faktor risiko yang berhubungan dengan usia, kualitas hidup, SF-8.


2006 ◽  
Vol 10 (01) ◽  
pp. 57-61 ◽  
Author(s):  
Fitnat Dinçer ◽  
Özlem Erol ◽  
Ayçe Atalay

Objective: To assess effect of physical therapy including both physical agents and exercise on pain, physical functioning and quality of life in patients with knee osteoarthritis. Methods: Twenty-seven patients (25 female and two male) with knee osteoarthritis were included in the study. Besides sociodemographic variables, presence of chronic diseases, analgesic use, recreational activites were recorded. A standard knee examination was performed and anteroposterior and lateral knee radiographs were obtained. Pain was measured using visual analogue scale (VAS), numeric scale and Likert scale. Pain, stiffness and physical function was assessed using Likert-scaled version of the Western Ontario McMaster Universities Osteoarthritis Index (WOMAC). For quality of life evaluation Medical Outcomes Study Short Form 36 (SF-36) was utilized. Evaluations were done at baseline and 1 month after completion of physical therapy sessions. Results: Significant reduction of pain measurements namely VAS, numeric scale and Likert scale was observed as a result of physical treatment (p = 0.0001, p = 0.0001 and p = 0.001 respectively). In all 3 subscores of WOMAC (pain, stiffness and physical function) significant improvement was noted due to treatment (p = 0.0001, p = 0.012 and p = 0.0001 respectively). Similarly, physical functioning, role-physical, bodily pain and vitality and social functioning subscores demonstrated significant reductions after therapy. (p = 0.039, p = 0.001, p = 0.0001, p = 0.001 and p = 0.035 respectively). Conclusion: Physical therapy including both physical agents and exercise leads to signifcant improvements in pain, physical functioning and quality of life. This change was demonstrated by both generic and disease specific outcome measures.


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