Costs, quality of life, treatment satisfaction and compliance in patients with β-thalassemia major undergoing iron chelation therapy: the ITHACA study

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

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Iron chelation therapy: Clinical effectiveness, economic burden and quality of life in patients with iron overload

Advances in Therapy ◽

10.1007/s12325-008-0085-z ◽

2008 ◽

Vol 25 (8) ◽

pp. 725-742 ◽

Cited By ~ 36

Author(s):

Krista A. Payne ◽

Diana Rofail ◽

Jean-François Baladi ◽

Muriel Viala ◽

Linda Abetz ◽

...

Keyword(s):

Quality Of Life ◽

Iron Overload ◽

Economic Burden ◽

Chelation Therapy ◽

Iron Chelation ◽

Clinical Effectiveness ◽

Iron Chelation Therapy

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PSY53 SATISFACTION WITH IRON CHELATION THERAPY IS ASSOCIATED WITH IMPROVED QUALITY OF LIFE IN PATIENTS WITH IRON OVERLOAD

Value in Health ◽

10.1016/s1098-3015(10)67103-3 ◽

2008 ◽

Vol 11 (6) ◽

pp. A646 ◽

Cited By ~ 1

Author(s):

D Rofail ◽

L Abetz ◽

R Heelis ◽

JF Baladi

Keyword(s):

Quality Of Life ◽

Iron Overload ◽

Chelation Therapy ◽

Iron Chelation ◽

Iron Chelation Therapy

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Impact of Educational Program about Iron Chelation Therapy on the Quality of Life for Thalassemic Children

Port Said Scientific Journal of Nursing ◽

10.21608/pssjn.2019.66511 ◽

2019 ◽

Vol 6 (3) ◽

pp. 33-49 ◽

Cited By ~ 1

Author(s):

Donia El-Said Zaghamir ◽

Rehab Hanie El-Kazaz ◽

Amal Ahmed Khalil Morsy ◽

Momahmed Ahmed Elmazahy

Keyword(s):

Quality Of Life ◽

Educational Program ◽

Chelation Therapy ◽

Iron Chelation ◽

Iron Chelation Therapy

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Cost-Effectiveness Analysis of Oral Iron Chelation Therapy with Deferasirox (Exjade®, ICL670) Versus Infusional Chelation Therapy with Deferoxamine in Patients with Transfusion-Dependent Myelodysplastic Syndrome.

Blood ◽

10.1182/blood.v106.11.5585.5585 ◽

2005 ◽

Vol 106 (11) ◽

pp. 5585-5585 ◽

Cited By ~ 3

Author(s):

Thomas E. Delea ◽

Simu K. Thomas ◽

Jean-Francois Baladi ◽

Pradyumna D. Phatak

Keyword(s):

Quality Of Life ◽

Cost Effectiveness ◽

Chelation Therapy ◽

Iron Chelation ◽

Iron Chelator ◽

Iron Chelation Therapy ◽

The Difference ◽

One Year ◽

The Cost

Abstract Background. Patients with symptomatic myelodysplastic syndrome (MDS) frequently receive chronic transfusions, along with chelation therapy to prevent complications of iron overload. Deferoxamine (DFO) is an effective iron chelator, but must be administered as an 8–12 hour infusion 5–7 times per week, leading to poor compliance and/or reduced quality of life. Deferasirox (DSX) is an investigational once-daily oral iron chelator that has been shown to produce reductions in liver iron concentrations and serum ferritin similar to those obtained with DFO. The objective of this analysis was to evaluate from a US perspective the cost-effectiveness (CE) of DSX vs DFO in patients with transfusion-dependent MDS. Methods. Data from a variety of published and unpublished sources were used to estimate the CE of chelation therapy with DSX vs DFO in MDS patients receiving frequent transfusions (≥8 per year). As there are no long-term studies describing the complications of iron overload in MDS, we focused on the short-term (i.e., one year) cost and quality-of-life effects of chelation therapy. As comparative data for DSX vs DFO in MDS are unavailable, we estimated the average dose (mg/kg/d) of DSX based on results for MDS patients in a non-comparative Phase II study. The relative dose of DFO that would result in similar efficacy was based on data from comparative studies in other transfusion-dependent anemias. To be conservative, we assumed that all patients would be fully compliant with chelation therapy. CE was measured in terms of the ratio of the difference (DSX vs DFO) in costs of chelation therapy to the difference in quality adjusted life years (QALYs) over one year of treatment. Analyses were based on the wholesale acquisition cost of generic DFO and the anticipated cost of DSX in the US. Mean weight was estimated to be 70 kg, based on data for MDS patients in DSX clinical studies. The cost of DFO administration was based on analyses of health insurance claims data for patients with transfusion-dependent anemias. Utilities (weights representing patient quality of life) for MDS patients receiving transfusions were based on published data for patients with anemia from metastatic cancer. The difference in quality of life for DSX vs DFO was based on results of a study that used time-trade-off methods to estimate community-based preferences for oral vs infusional iron chelation therapy. Results. Total annual costs are estimated to be $7,679 greater with DSX ($35,672 vs $27,993 with DFO). Annual costs of DFO included $20,185 for drug acquisition and $7,808 for drug administration. One year of treatment with DSX is estimated to result in a gain of 0.23 QALYs (0.78 vs 0.55 with DFO). The CE of DSX vs DFO is therefore estimated to be $33,387 per QALY gained. CE of DSX vs DFO was sensitive to the assumed dosages of DSX and DFO, the cost of infusional therapy, and the decrement in quality of life associated with infusional therapy. Conclusion: The CE of DSX versus DFO in patient with transfusion-dependent MDS is favorable compared with that of other generally-accepted treatments for patients with hematologic/oncologic disorders. These results may be conservative, as they did not consider the potential benefits of improved compliance with DSX or side effects of infusion therapy.

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Health-related quality of life in transfusion-dependent patients with myelodysplastic syndromes: a prospective study to assess the impact of iron chelation therapy

BMJ Supportive & Palliative Care ◽

10.1136/bmjspcare-2014-000726 ◽

2014 ◽

Vol 6 (1) ◽

pp. 80-88 ◽

Cited By ~ 7

Author(s):

Fabio Efficace ◽

Valeria Santini ◽

Giorgio La Nasa ◽

Francesco Cottone ◽

Carlo Finelli ◽

...

Keyword(s):

Quality Of Life ◽

Chelation Therapy ◽

Iron Chelation ◽

Iron Chelation Therapy ◽

Health Related Quality ◽

Related Quality ◽

Health Related ◽

A Prospective Study ◽

The Impact

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329 Heart failure predictive value of the electric risk score in patients suffering from thalassemia major

European Heart Journal Supplements ◽

10.1093/eurheartj/suab142.054 ◽

2021 ◽

Vol 23 (Supplement_G) ◽

Author(s):

Elisa Tomarelli ◽

Federica Moscucci ◽

Anna Annunziata Losardo ◽

Pellegrina Pugliese ◽

Mauro Schina ◽

...

Keyword(s):

Risk Factors ◽

Risk Score ◽

Ventricular Hypertrophy ◽

Chelation Therapy ◽

Iron Chelation ◽

Myocardial Damage ◽

Thalassemia Major ◽

Left Ventricular ◽

P Value ◽

Iron Chelation Therapy

Abstract Aims Complications associated with iron accumulation were highly recurrent in thalassemia patients, who underwent frequent blood transfusions, in particular hemosiderotic cardiomyopathy which could lead to heart failure and arrhythmias. Nowadays, the better iron chelation therapy has improved cardiovascular morbidity in these patients; nevertheless, mild impairment should be seek for and eventually treated. The objective of our study was to evaluate the possibility of using early electrocardiographic markers of myocardial damage and predictors of mortality, such as the Electric Risk Score (ERS). Methods and results 73 patients with thalassemia major were enrolled in this study, which were divided into two groups, with 45 years old as cut off. Anamnestic, clinical, electrocardiographic, and echocardiographic data were collected. From ECG, ERS was obtained. over 45 yrs-old group of pts, in addition to a predictable increase in the prevalence of traditional cardiovascular risk factors and drug intake, an alteration of the QRS-T angle (14[30] vs. −4[28], p value: <0.0001) and an increased prevalence of left ventricular hypertrophy (2.88 ± 0.86 vs. 2.40 ± 0.57 p value: <0.05) were found. In patients taking drugs with possible interactions with the ventricular repolarization phase, there is a slight increase in the QT interval, left ventricular hypertrophy and a reduction in Tpeak-Tend (Table 1). Electrocardiographic values in groups of patients with different age groups who are taking therapies that can affect QT. The echocardiogram revealed an increase in the end-diastolic diameter of the right ventricle (26 ± 3 vs. 28 ± 3 mm, P-value: 0.05) in the group of patients over the age of 45, a decrease in the acceleration time of the pulmonary systolic flow (138 ± 25 vs. 125 ± 13 ms, P-value: 0.04) and TAPSE (25 ± 3 vs. 22 ± 4 mm, P-value: 0.002). Conclusions From the data in our study it emerged that an appropriate iron-chelation therapy is able to effectively counteract the hemosiderotic cardiomyopathy of thalassemic patients so as to detect electro- and echocardiographic anomalies only in patients of more advanced age, a result that we think both the consequence, not so much of iron overload, but of an increase in the prevalence of age- and gender-related cardiovascular risk factors. The initial changes in cardiac electromechanics, which can be assessed with the aforementioned methods, we believe, can become a very early sign of specific myocardial damage. 329 Figure 1Electrical risk score parameters.

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Testicular function in patients with regular blood transfusion for thalassemia major

Asian Biomedicine ◽

10.5372/1905-7415.0902.385 ◽

2015 ◽

Vol 9 (2) ◽

Cited By ~ 1

Author(s):

Sukumarn Siripunthana ◽

Taninee Sahakitrungruang ◽

Suttipong Wacharasindhu ◽

Darintr Sosothikul ◽

Vichit Supornsilchai

Keyword(s):

Blood Transfusion ◽

Chelation Therapy ◽

Cell Function ◽

Iron Chelation ◽

Thalassemia Major ◽

Delayed Puberty ◽

Control Group ◽

Blood Transfusions ◽

Testicular Function ◽

Iron Chelation Therapy

AbstractBackgroundRegular blood transfusion and iron chelation therapy have improved the quality of life of patients with thalassemia and increased their longevity, but transfusion also increases the frequency of endocrine complications, possibly because of iron deposition in the pituitary gland or the gonads, or both.ObjectiveTo evaluate testicular function in patients with thalassemia major by basal hormonal study, and identify risk factors for dysfunction.MethodsWe performed a cross-sectional study of 28 patients with thalassemia major aged 11.7 ± 1.8 (8–14.9) years (15 in prepuberty, 13 in puberty with no delayed puberty) who had regular blood transfusions. A normal control group comprised 64 boys who were matched for age and Tanner genital stage.ResultsThe mean level of serum ferritin in the previous year was 1,575 ± 642 ng/mL, and the onset of blood transfusion was at 3.8 ± 2.3 years and iron chelation therapy was 6.6 ± 2.8 years. The trend for anti-Müllerian hormone levels in patients and controls was similar with age, and although higher in the patients, particularly at Tanner stage II, was not significantly different. Testosterone levels were lower in the patients compared with controls; particularly at Tanner stages IV–V (290.88 vs. 537.4 ng/dL,ConclusionPatients who received regular blood transfusions had normal Sertoli cell function. Leydig cell dysfunction may occur, even though the patients had a normal pubertal onset.

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