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

2019 ◽  
Vol 6 (3) ◽  
pp. 33-49 ◽  
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

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

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

2008 ◽  
Vol 25 (8) ◽  
pp. 725-742 ◽  
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

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 ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 5585-5585 ◽  
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.

Once-Daily Oral Deferasirox (Exjade®, ICL670) Versus Infusional Deferoxamine as Iron Chelation Therapy in Patients with Sickle-Cell Disease Receiving Frequent Transfusions: A Cost-Effectiveness Analysis.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 5584-5584 ◽  
Author(s):  
Thomas E. Delea ◽  
Simu K. Thomas ◽  
Jean-Francois Baladi ◽  
Thomas D. Coates
Keyword(s):  
Quality Of Life ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Iron Chelator ◽  
Cell Disease ◽  
Once Daily ◽  
The Difference ◽  
One Year ◽  
The Cost

Abstract Background. Patients with sickle-cell disease (SCD) receiving chronic transfusions require chelation therapy to prevent complications from iron overload. Although deferoxamine (DFO) is an effective iron chelator, it 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 in magnitude to those obtained with DFO. Cost-effectiveness (CE) analysis is a technique used to determine whether the benefits of new therapies are worth their additional costs. The objective of this analysis was to evaluate from a US perspective the CE of DSX versus DFO in SCD patients receiving frequent transfusions. Methods. Data from a variety of published and unpublished sources were used to estimate the CE of chelation therapy with DSX versus DFO in SCD patients receiving frequent transfusions (≥8 per year). As there are no long-term studies describing the complications of iron overload in patients with SCD, we focused on the short term (i.e., one year) costs and quality-of-life effects of chelation therapy. We assumed that patients would receive dosages (mg/kg/d) of DSX and DFO that have been found to be similarly effective in patients with SCD. 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 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 52 kg, based on data from deferasirox 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) were based on results of a study that used time-trade-off methods to estimate community-based preferences for oral versus infusional iron chelation therapy. Results. Total annual costs were estimated to be $1,486 greater with DSX ($22,922 vs $21,436 with DFO). Annual costs of DFO included $13,628 for drug acquisition and $7,808 for drug administration. One year of treatment with DSX is estimated to result in a gain of 0.25 QALYs (0.82 vs 0.57 with DFO). The CE of DSX versus DFO is therefore estimated to be $5,944 per QALY gained. CE of DSX versus DFO was sensitive to the assumed dosages of DSX and DFO and the cost of infusional therapy. Conclusion. In patients with SCD receiving frequent transfusions, DSX versus DFO is highly cost-effective compared with other generally-accepted treatments for patients with hematologic and oncologic disorders. These results may understate the CE of DSX, as they did not consider the potential benefits of improved compliance or side effects of infusion therapy. Further research is needed to assess the potential implication of DSX on the risk-benefit profile of transfusion therapy in patients with SCD.

scholarly journals DEFERASIROX: OVER A DECADE OF EXPERIENCE IN THALASSEMIA

2018 ◽  
Vol 10 ◽  
pp. e2018066 ◽  
Author(s):  
Ali Taher
Keyword(s):  
Iron Overload ◽  
Chelation Therapy ◽  
Iron Concentration ◽  
Iron Chelation ◽  
Chelating Agent ◽  
Iron Chelation Therapy ◽  
Liver Iron ◽  
Adequate Treatment

Thalassemia incorporates a broad clinical spectrum characterized by decreased or absent production of normal hemoglobin leading to decreased red blood cell survival and ineffective erythropoiesis. Chronic iron overload remains an inevitable complication resulting from regular blood transfusions (transfusion-dependent) and/or increased iron absorption (mainly non-transfusion-dependent thalassemia), requiring adequate treatment to prevent the significant associated morbidity and mortality. Iron chelation therapy has become a cornerstone in the management of thalassemia patients, leading to improvements in their outcome and quality of life. Deferasirox, an oral iron chelating agent is approved for use in transfusion dependent and non-transfusion-dependent thalassemia and has shown excellent efficacy in this setting. We herein present an updated review of the role of deferasirox in thalassemia, exploring over a decade of experience, which has documented its effectiveness and convenience; in addition to its manageable safety profile. Keywords: iron overload, iron chelation therapy, transfusion-dependent thalassemia, non-transfusion dependent thalassemia, serum ferritin, liver iron concentration, deferasirox

scholarly journals Adherence to Iron Chelation Therapy and Its Determinants

2021 ◽  
Author(s):  
Sukhmani Sidhu ◽  
Shruti Kakkar ◽  
Priyanka Dewan ◽  
Namita Bansal ◽  
Praveen C. Sobti
Keyword(s):  
Iron Overload ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Iron Chelation Therapy ◽  
Liver Iron ◽  
Free Survival ◽  
Liver Iron Overload ◽  
The Impact ◽  
Study Questionnaire

Background: Thalassemia is a chronic disease requiring lifelong treatment. The adherence to regular iron chelation therapy is important to ensure complication-free survival and good quality of life. The study aim to assess the adherence to iron chelation therapy (ICT) in patients with transfusion-dependent thalassemia (TDT), evaluate various causes of non-adherence and study the impact of non-adherence on the prevalence of complications secondary to iron overload. Materials and Methods: Patients with TDT on ICT for > 6 months were enrolled in the study. Hospital records were reviewed for demographic details, iron overload status, treatment details, and the prevalence of complications. A study questionnaire was used to collect information on adherence to ICT, knowledge of patients, and the possible reasons for non-adherence. Results: A total of 215 patients with a mean age of 15.07+7.68 years and an M: F ratio of 2.2:1 were included in the study. Non-adherence to ICT was found in 10.7% of patients. Serum ferritin levels were significantly higher in the non-adherent group (3129.8+1573.2 µg/l) than the adherent population (2013.1+1277.1 µg/l). Cardiac as well as severe liver iron overload was higher in the non-adherent patients. No correlation was found between disease knowledge and adherence to ICT. Difficulties in drug administration and many medicines to be taken daily were statistically significant reasons for non-adherence. There was no difference in the co-morbidities arising due to the iron overload in the two groups. Conclusion: Nearly 11% of patients with TDT were non-adherent to ICT. Non-adherence results in higher iron overload.

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