Socio-Economic Impact of Infused Iron Chelation Therapy in France: ISOSFER Study Results.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 3354-3354 ◽  
Author(s):  
Catherine Brun-Strang ◽  
Dora Bachir ◽  
Mariane De Montalembert ◽  
Isabelle Thuret
Keyword(s):  
Adverse Events ◽  
Chelation Therapy ◽  
Ferritin Level ◽  
Iron Chelation ◽  
Organ Damage ◽  
Total Direct Cost ◽  
Iron Chelation Therapy ◽  
Cross Sectional ◽  
Study Results ◽  
The Cost

Abstract Background: Patients suffering from β-thalassemia (TM), sickle cell disease (SCD), and myelodysplastic syndromes (MDS) undergoing chronic blood transfusions are at risk for iron overload which, if not treated by iron chelation therapy (ICT), can cause serious organ damage and reduce life expectancy. Deferoxamine (DFO) is the standard of care for the depletion of excess body iron. It has to be infused for 8–10 hours, 5–7 times a week. Although the clinical need for ICT is clearly established, less is known about the economic burden of DFO treatment. Aim: To estimate the total annual costs of DFO ICT in treatment centers in France. Methods: A cross-sectional study with a prospective recruitment. Among 278 consecutive patients receiving regular transfusions for TM, SCD or MDS who consulted between October 2005 and February 2006 in 24 French centers, 161 were on ICT. 124 patients were treated with DFO alone for more than 1 year. Among them, 67 aged 14 years or more agreed to participate. Resources used were collected through patient and physician questionnaires. Unit costs (2004/2005 €) were applied according to French economic guidelines. Results: 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. TM (n=24) SCD (n=17) MDS (n=26) *Cardiac, liver and endocrine diseases, lens opacities, osteoporosis Median age (min-max), years 30 (15–70) 32 (14–57) 69 (45–85) Sex, M/F 11/13 6/11 14/12 Organ dysfunction potentially related to hemosiderosis* (%) 75 47 54 Ferritin level (median), ng/mL 1049 2653 2627 DFO nb/week (mean) 3.7 4.5 4 Dose (mean) 40 17 43 For all patients, the estimated mean weighted annual cost of infusions is 16009 € (SD ± 13867). Costs are similar for the three diseases. ICT delivery equipment (infusion set and pump) and nursing administration, drug cost, DFO adverse events monitoring, periodic exams and treatment of infused ICT-related adverse events represent respectively 56.5%, 38.5%, 0.3%, 3.7% and 0.9% of total direct cost. The estimated annual mean cost of the drug alone was 6160 € (SD ± 4145). Average cost for DFO adverse events management is low at 151.5€ (SD ± 1224), essentially due to one patient complication. Costs of periodic exams are also low due to the fact that exams are not strictly performed annually as recommended. These estimates of the total annual costs of DFO ICT are likely to be underestimating the overall cost of DFO therapy because treatment costs of the clinical consequences of poor adherence to DFO and lost productivity were not collected in the study. Conclusions: ISOSFER demonstrated that total direct costs of ICT are substantial and well exceed the cost of DFO alone. The cost of DFO administration constitutes a significant portion of the total cost of iron chelation (54%). These data are comparable to other analyses published from US (43% of the total costs, n=155) and Swiss (45%, n=17) databases.

Ferritin Levels, Compliance and Adverse Events Related to Infused Iron Chelation Therapy in a Cohort of Patients from Usual Care Setting in the United States.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 3360-3360 ◽  
Author(s):  
K.A. Payne ◽  
M.-P. Desrosiers ◽  
I. Proskorovsky ◽  
K. Ishak ◽  
N. Lordan ◽  
...  
Keyword(s):  
Adverse Events ◽  
Iron Overload ◽  
Usual Care ◽  
Care Setting ◽  
Chelation Therapy ◽  
Ferritin Level ◽  
Iron Chelation ◽  
The United States ◽  
Iron Chelation Therapy ◽  
Available N

Abstract Background: Deferoxamine (DFO) is an iron chelation therapy (ICT) agent administered to patients undergoing chronic blood transfusions to avoid toxic iron overload. Although efficacious, it is burdensome to patients due to the need for almost daily infusions lasting 8–10 hours each, and the occurrence of treatment-related adverse events (AEs). Purpose: To document ferritin levels, compliance and prevalence of AEs in a cohort of patients undergoing DFO ICT. Methods: A naturalistic cohort study of resource utilization and quality-of-life burden of infused ICT in the usual care setting (acute hospital and out-patient) was undertaken in four US treatment centers between September and December 2005. Patients aged ≥6 years with thalassemia or sickle cell disease (SCD) currently undergoing ICT were eligible to participate. This abstract refers only to patient compliance, ferritin levels and AEs related to infused ICT. Compliance (up to 7 days prior to the study) and AEs (up to 30 days prior to the study) were obtained from patient interviews. Ferritin data from these same patients during their initial and most recent year of ICT were collected from medical charts. Results: 49 patients on infused ICT (50% male; mean age: 28 ± 10 years) with thalassemia (n=40) or SCD (n=9) were recruited. Ferritin level test results obtained from charts indicate that, in general, average blood iron levels were high and remained stable or increased over time, despite ICT. During the initial year of ICT (n=35), mean ferritin level was 2687 ± 1535 ng/mL for thalassemia patients and 2088 ± 791 ng/mL for SCD patients (2519 ± 1382 overall). During the most recent year of ICT (n=45), thalassemia patients had a mean ferritin level value of 2496 ± 2556 ng/mL and SCD patients had a mean ferritin level value of 4108 ± 2030 ng/mL (2741 ± 2532 overall). For all patients in whom data from the most recent year and the initial year of ICT were available (n=29), mean ferritin level increased by 306 ± 2774 ng/mL over a mean period of 20 ± 9 years of therapy. In general, high mean ferritin level during the most recent year of ICT was associated with poor compliance reported over the previous 7-day period (Table). Seventy-seven percent of patients reported missing at least one DFO dose over the previous 4 weeks. Among these patients, 14% did so due to AEs. Over the previous 30 days, 55% suffered at least one AE; the most commonly reported were site soreness (85%), site irritation (74%), ringing in the ears (26%), temporary hearing loss (11%), blurred vision (11%) and abdominal pain (11%). Conclusions: Infused ICT may not provide adequate effectiveness in the real world. High ferritin levels seem to be associated with patient non-compliance to infused ICT, which may result from the occurrence of bothersome side effects and the burdensome mode of administration. In all patients, even those compliant, generally high ferritin levels highlight the risk for iron-overload complications. An ICT agent offering improved convenience and patient satisfaction could improve the clinical and economic outcomes of therapy. Compliance and ferritin levels associated with infused ICT Compliance (%) Patients, n (%) Mean ferritin level ± SD, ng/mL Thalassemia (n=39) 0–50 9 (23) 3615 ± 3522 51–80 14 (36) 2831 ± 2474 81+ 16 (41) 1573 ± 1694 SCD (n=6) 0–50 2 (33) 5637 ± 2850 51–80 1 (17) 3828 81+ 3 (50) 3840 ± 1965

Cardiovascular Complications Of Thalassemia Patients In The Iron Chelation Therapy Era

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 5602-5602
Author(s):  
Adisak Tantiworawit ◽  
Suebsakul Tapanya ◽  
Arintaya Phrommitikul ◽  
Lalita Norasetthada ◽  
Chatree Chai-adisaksopha ◽  
...  
Keyword(s):  
Pulmonary Hypertension ◽  
Cardiac Function ◽  
Serum Ferritin ◽  
Chelation Therapy ◽  
Conflicts Of Interest ◽  
Ferritin Level ◽  
Iron Chelation ◽  
Cardiac Complications ◽  
Iron Chelation Therapy ◽  
Cross Sectional

Abstract Background Cardiac complications are the most common cause of death in thalassemia, accounting for up to 71% in the past. Iron chelation therapy is given to patients with iron overload to prevent these complications. The cost effectiveness of iron chelation therapy was arguable. This study aims to evaluate the prevalence of cardiac complication and the correlation between risk factors in iron chelation therapy era. Method This is a cross sectional study from June 2011- May 2012. All thalassemia aged > 15 years old were enrolled. Clinical data and hemoglobin typing were reviewed. Echocardiography and CMR T2*, a technique represent cardiac iron deposition used to evaluate cardiac function, were used to evaluate cardiac complications. Results Ninety one patients were enrolled, 63.7% were females, median age of 31 years (16-75). There was 49.5 % homozygous β thalassemia, 31.9% β thalassemia/Hb E disease, 18.7% Hb H disease. Half of patients were transfusion dependent and 63.7% underwent splenectomy. Eighty four percent of patients received iron chelation therapy but few of them got their preferable choice in adequate dosage. Patients with serum ferritin levels more than 1,000 ng/ml. received deferoxamine, deferiprone or deferasirox. Even with the iron chelation therapy, mean serum ferritin level was still high at 3,820 ng/ml for the whole group. CMR T2* was more sensitive in detecting cardiac function. The CMR T2* showed shorter signal (≤ 20 msec) in 11.1%. Only 8.2% had impaired ejection fraction <55% by echocardiography. The CMR T2*  ≤ 20 msec was significant correlated with higher maximum ferritin 5,739.14 ng/ml compared to 3,614 ng/ml (p=0.001). Pulmonary hypertension was found 7 patients (12.7%) and 71.42% had underwent splenectomy. Conclusion From our study, the CMR T2* is the sensitive method for detecting cardiomyopathy and highly correlated with serum ferritin levels. Splenectomy remains the major risk factor for pulmonary hypertension. The incidence of cardiac complications has decreased with iron chelation therapy for maintaining acceptable serum ferritin levels but the problem with cardiomyopathy and pulmonary hypertension still exist. Early detection, more sensitive implementation and aggressive iron chelation therapy are necessary to prevent these complications. The majority of the patients in Thailand which are under universal health-care coverage scheme could not get access to more effective and expensive iron chelator. Regular and adequate chelation plays a major role in the prevention of cardiac complications and the achievement of better quality of life. Disclosures: No relevant conflicts of interest to declare.

scholarly journals From Biology to Clinical Practice: Iron Chelation Therapy With Deferasirox

2021 ◽  
Vol 11 ◽  
Author(s):  
Giuseppe A. Palumbo ◽  
Sara Galimberti ◽  
Wilma Barcellini ◽  
Daniela Cilloni ◽  
Nicola Di Renzo ◽  
...  
Keyword(s):  
Therapeutic Strategy ◽  
Chelation Therapy ◽  
Allogeneic Bone Marrow Transplantation ◽  
Iron Chelation ◽  
Organ Damage ◽  
Allogeneic Bone ◽  
Iron Chelation Therapy ◽  
Radical Oxygen Species ◽  
Hereditary Disorders ◽  
Near Future

Iron chelation therapy (ICT) has become a mainstay in heavily transfused hematological patients, with the aim to reduce iron overload (IOL) and prevent organ damage. This therapeutic approach is already widely used in thalassemic patients and in low-risk Myelodysplastic Syndrome (MDS) patients. More recently, ICT has been proposed for high-risk MDS, especially when an allogeneic bone marrow transplantation has been planned. Furthermore, other hematological and hereditary disorders, characterized by considerable transfusion support to manage anemia, could benefit from this therapy. Meanwhile, data accumulated on how iron toxicity could exacerbate anemia and other clinical comorbidities due to oxidative stress radical oxygen species (ROS) mediated by free iron species. Taking all into consideration, together with the availability of approved oral iron chelators, we envision a larger use of ICT in the near future. The aim of this review is to better identify those non-thalassemic patients who can benefit from ICT and give practical tips for management of this therapeutic strategy.

scholarly journals Red Blood Cell Transfusion Independence Following the Initiation of Iron Chelation Therapy in Myelodysplastic Syndrome.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 4844-4844
Author(s):  
Maha A Badawi ◽  
Linda M Vickars ◽  
Jocelyn M Chase ◽  
Heather A Leitch
Keyword(s):  
Platelet Count ◽  
Myelodysplastic Syndrome ◽  
Research Funding ◽  
Chelation Therapy ◽  
Ferritin Level ◽  
Iron Chelation ◽  
Iron Chelation Therapy ◽  
Transfusion Independence ◽  
Transfusion Requirements ◽  
Rbc Transfusion

Abstract Abstract 4844 Background Iron chelation therapy (ICT) is often used to treat iron overload (IOL) in patients (pts) requiring transfusion of red blood cells (RBC) for chronic anemia. In myelodysplastic syndrome (MDS), guidelines recommend consideration of ICT in pts with lower risk International Prognostic Scoring System (IPSS) and IOL as defined by a ferritin level >1000 ug/l; IOL related organ dysfunction; or receipt of ≥20 RBC units. During treatment of a pt with MDS and IOL with ICT, RBC transfusion requirement (TR) ceased. Here we report his course and review reported cases of RBC transfusion independence (TI) or decreased RBC TR in MDS pts receiving ICT. Methods The pt chart was reviewed and reported cases identified by PubMed search using the terms ‘MDS’ and ‘iron chelation’. The clinical characteristics and course of published cases were summarized. Case A 76 year (y) old man was referred in May 2004 for management of MDS diagnosed in 1997, when the white blood cell (WBC) count was 2.4 ×109/l; neutrophils, 0.7 ×109/l; hemoglobin (Hb), 133 g/l; platelets, 108 ×109/l. Bone marrow aspiration and biopsy showed refractory anemia (RA), karyotype analysis 46,X,-Y,+8, and the IPSS score was intermediate-1. The erythropoitin (epo) level was 148.3 mIU/ml and the stem cell assay showed no epo-independent colony growth. In 2004 the Hb dropped to 60 g/l prompting the initiation of RBC transfusion support. He required 3 RBC units every 4 weeks to maintain a Hb >90 g/l and complained of fatigue and functional limitation. Creatinine, bilirubin, TSH, reticulocyte count, B12 and folate levels were all normal. The ferritin level in 2004 was 1293 ug/l and 2197 ug/l in 2006. He declined ICT with deferoxamine (DFO) but in 2006 accepted deferasirox (DFX). He required several dose interruptions and adjustments for renal insufficiency; the current dose is 5mg/kg/d with a normal creatinine. Two months (mo) after starting ICT, the Hb increased spontaneously to 109 g/l and he has not required RBC transfusion since. The mean Hb since starting ICT was 122 g/l and the ferritin decreased to 1082 ug/l in 2009. The most recent neutrophil count was 3.5 ×109/l, platelets consistently clump and the MCV is unchanged at 120 fl. He reports excellent energy and an improved quality of life, and has remained clinically well and RBC transfusion independent to the present, 36 mo from the initiation of ICT. Literature review There are 18 published cases of MDS showing improvement in Hb with ICT; 9 became RBC transfusion independent. Characteristics of the 10 TI pts were: median age at MDS diagnosis 58 (range 18-74) y; male, n=5. MDS subtype: RA, n=5; RARS, n=2, RCMD, n=1; RAEB, n=2. IPSS (reported in 8): low, n=1; int-1, n=5; int-1 or 2, n=1; high, n=1. ICT was: DFO, n=7; DFX, n=3. Median time to RBC TI was 17.5 (1-24) mo and TI duration 13 (3-28) mo to date. Of pts who had decreased RBC transfusion requirements with ICT but did not achieve transfusion independence: median age (reported in 3) was 67 (45-78) y; gender (reported in 3) female, n=3; MDS subtype: RA, n=8; RAEB-t, n=1; IPSS: int-1, n=3; ICT: DFO, n=8; DFX, n=1. Median time to decreased TR was 14.4 (3-24) mo; median duration of decreased TR (reported in 3) 9 (6-32) mo; initial TR 50.9 (19.7-447) g Hb/mo; median decrease in TR 12.7 (0.1-88) g Hb/mo. In one report of 6 pts, 2 with pancytopenia showed improvement with ICT in WBC from 1.4 to 1.9 ×109/l (p<0.0001) and neutrophils from 0.51 to 0.94 ×109/l (p<0.001). The platelet count increased from 16.6 to 22.5 ×109/l (p<0.001) and 14.6 to 29.6 ×109/l (p<0.00001) within 3 mo and the MCV decreased significantly in 5 by a mean of 5.1 (2.1-11.7) fl, normalizing in 2. In a second report, neutrophils increased in 8 of 9 pts; in 4 the initial neutrophil count was <1 ×109/l, and platelet counts increased in 7 of 11 pts, in 4 the initial platelet count was <20 ×109/l. Conclusions In summary, our pt is the 19th patient with MDS reported to date in whom improved Hb followed the initiation of ICT; 9 had a decrease in RBC transfusion requirements, and RBC transfusion independence occurred in 10. The remarkable course of these pts adds to evidence that ICT may be of clinical benefit for selected patients with MDS and IOL. Although the improvement in WBC and platelet counts with ICT in some pts implies a suppressive effect of IOL on hematopoiesis that may be abrogated by ICT, the mechanism by which the effects of ICT on transfusion requirements occur, and the frequency with which they occur, remains an area for future investigation. Disclosures Off Label Use: This presentation discusses the use of iron chelation therapy deferoxamine and deferasirox in patients with myelodysplastic syndrome.. Vickars:Novartis Canada: Honoraria, Research Funding. Leitch:Novartis Canada: Honoraria, Research Funding, Speakers Bureau.

Socio-psychological impact of infused iron chelation therapy with deferoxamine in metropolitan France: ISOSFER study results

Hematology ◽  
2009 ◽  
Vol 14 (6) ◽  
pp. 315-322 ◽  
Author(s):  
Isabelle Thuret ◽  
Maya Hacini ◽  
Brigitte Pégourié-Bandelier ◽  
Martine Gardembas-Pain ◽  
Ségolène Bisot-Locard ◽  
...  
Keyword(s):  
Chelation Therapy ◽  
Psychological Impact ◽  
Iron Chelation ◽  
Iron Chelation Therapy ◽  
Study Results

Estimating the Total Cost of Infused Iron Chelation Therapy.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 5576-5576 ◽  
Author(s):  
Marie-Pierre Desrosiers ◽  
Krista A. Payne ◽  
Jean-Francois Baladi
Keyword(s):  
Cohort Study ◽  
Resource Use ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Cost Data ◽  
Iron Chelation Therapy ◽  
Cell Disease ◽  
Total Cost ◽  
Unit Costs ◽  
The Cost

Abstract Background: Patients suffering from β-thalassemia or sickle cell disease require on-going blood transfusions. Chronic transfusion, however, results in iron overload, which if not removed by iron chelation therapy (ICT), causes organ damage. Deferoxamine (DFO) is currently the standard of care for ICT, but many patients do not adhere to therapy possibly because of the need for almost daily infusions lasting 8 to 10 hours each. Rationale: While the impact of current care on clinical and patient outcomes is generally understood, less is known about the total cost of DFO therapy. Objectives: To identify a complete set of cost items to inform the development of an ICT related Resource Use Questionnaire (RUQ) for administration in an international cohort study of the actual cost of ICT in practice; and to obtain a preliminary, literature-based estimate of total annual per patient costs of ICT. Methods: A search of the literature (EMB Reviews; Scirus and Ovid Medline (1996+); PubMed (1995+) was performed using the following key words: thalassemia, sickle cell disease, myelodysplastic syndrome, cost, iron chelation, Desferal, deferoxamine, resource use, reimbursement and compliance. Cost items were extracted from eligible studies to create an aggregated, composite set of ICT-related variables to which unit costs (2004/2005 USD) were applied. Results: Of 396 abstracts obtained, all but 96 were excluded because ICT cost data were lacking. Of those retained, only 4 studies (1 Israël;1 US;2 UK) reported ICT-related costs (1 lifetime;3 annual). Cost variables differed markedly among studies each focusing on some specific aspect. The application of unit costs to the composite list of ICT-related variables and associated resource use profiles reveal that total annual per patient ICT costs may be as high as $7,487 to $15,836 (£4,191 to £8,865) depending on age. The cost of DFO accounts for only 16%–31% of these estimated total costs, with the balance accounted for by other annual ancillary expenditures such as equipment and supplies, monitoring, and home health care services. Total costs could well be underestimated given that component lifetime costs such as DFO treatment complications, the clinical sequelae of poor adherence to DFO, and the indirect costs of lost productivity were not included. Cost estimates will be supplemented and validated at the time of abstract presentation by the resource use and unit cost data generated by the RUQ employed in the aforementioned international cohort study. Conclusions: Estimated total costs of ICT are substantial and well exceed the cost of DFO alone. A paucity of published data related to the total costs of ICT underscores the need for additional ICT cost data from actual practice to better understand the economic impact of novel ICT agents.

The Palatability and Tolerability of Deferasirox (Exjade®) Taken with Meals, Different Liquids, or Crushed and Added to Food

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 5155-5155
Author(s):  
Stuart L Goldberg ◽  
Patricia Giardina ◽  
Joan Parkhurst Cain ◽  
Deborah Chirnomas ◽  
Jason Esposito ◽  
...  
Keyword(s):  
Adverse Events ◽  
Serum Ferritin ◽  
Research Funding ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Thalassemia Major ◽  
Iron Chelation Therapy ◽  
Age Group ◽  
Baseline Serum ◽  
Off Label

Abstract Abstract 5155 Introduction: Deferasirox (Exjade®, Novartis Pharmaceuticals) is an oral iron chelator indicated for the treatment of transfusional iron overload. The recommended mode of administration is to be taken on an empty stomach in water, apple juice or orange juice ≥30 minutes before food. However, there have been post-marketing reports of discontinuation or reduced compliance of deferasirox secondary to palatability and gastrointestinal adverse events. Registration trials with deferasirox did not evaluate different food combinations in an attempt to maintain predictable plasma levels. Early single dose studies suggested that the bioavailability of deferasirox is increased when administered with or before meals, and is positively influenced by fat content, but is not affected by degree of dispersion nor type of liquid. Long-term pharmacokinetic and tolerability studies involving a food effect have not been conducted to date, and the ability of alternate methods of administration to improve patient compliance with iron chelation therapy is unknown. Method: This is an ongoing single-arm, open-label, multi-center study designed to evaluate the palatability, safety, tolerability and pharmacokinetics of deferasirox when administered with food, dispersed in any liquid of choice, or crushed and added to food. The patient population includes patients with transfusional hemosiderosis (minimum entry serum ferritin ≥500 μ g/L) aged >2 years with thalassemia major, sickle cell disease (SCD), low or intermediate (INT-1) risk MDS or other anemias, who are on, starting, or resuming treatment with deferasirox. The study began with a 1-month run-in phase with deferasirox dosed according to prescribing information, then a 3-month assessment phase where subjects could choose each week from 5 general administration options including with or without meals, in the morning or evening, crushed and added to a soft food, or mixed in a liquid of choice. Subject diaries are used to record the meal and method of administration at the end of each week. Palatability is assessed with a modified facial hedonic scale, with additional directed questions capturing gastrointestinal side effects. This is a data analysis of the run-in phase. Result: Target enrollment has been met with 65 patients. Baseline data on the first 58 subjects include 8 in the 2 to <10 years of age group (median 7.5 years; range 3–9); 42 in the 10 to <60 years of age group (median 18.5 years; range 10–48); and 8 in the ≥60 years of age group (median 74 years; range71-83). Underlying hematologic diagnoses included SCD (41%), thalassemia major (29%), MDS (12%) and other anemias (17%). Sixty-nine percent of subjects were receiving deferasirox prior to entering the study. The median baseline serum ferritin level was 2405 μ g/L (range 560–8660) and was distributed as shown in Table 1. The most frequent adverse events were diarrhea (19%) and nausea (9%) (Table 2), which were more common in MDS (P=0.23 and P<0.01, respectively). Conclusion: This ongoing trial (NCT00845871) is evaluating whether alternative modes of administration improve palatability and tolerability while maintaining safety. Preliminary data from the assessment phase (deferasirox taken with meals, different liquids, or crushed and added to food) will be presented at the meeting. Disclosures: Goldberg: Novartis Oncology: Consultancy, Honoraria, Research Funding, Speakers Bureau. Off Label Use: Exjade, iron chelation therapy, off-label method of administration. Giardina:Novartis: Research Funding. Parkhurst Cain:Novartis: Research Funding. Chirnomas:Novartis: Research Funding. Esposito:Novartis: Employment. Paley:Novartis: Employment. Vichinsky:Novartis: Consultancy, Research Funding, Speakers Bureau; Hemaquest: Consultancy, Membership on an entity's Board of Directors or advisory committees; Apotex: Consultancy, Research Funding.

Application of Self-Efficacy Theory in Adherence to Iron Chelation Therapy: A Single-Center Cross-Sectional Study

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 5284-5284
Author(s):  
Kevin H.M. Kuo ◽  
Richard Ward
Keyword(s):  
Family Support ◽  
Self Efficacy ◽  
Chelation Therapy ◽  
Care Center ◽  
Insurance Coverage ◽  
Iron Chelation ◽  
Comprehensive Care ◽  
Iron Chelation Therapy ◽  
Cross Sectional ◽  
Significant Difference

Abstract 5284 Introduction: Poor adherence to iron chelation therapy (ICT) in beta-Thalassemia Major (TM) is associated with increased risk of cardiac complications and endocrinopathies, and lower survival, with substantial cost to the patient and the health care system. Canada is unique in that several predictors of non-adherence (Financial barriers to medical care, cost of medication and inadequate follow-up) are minimized due to the presence of universal health care, governmental subsidies for medications for patients with chronic disease, and the availability of comprehensive care center for most of the thalassemia patients in the country. Also, the availability of Deferiprone (DFP) via compassionate release program since July 2004 provides an alternative to patients intolerant or having suboptimal response to Deferoxamine (DFO) or Deferasirox (DFX). We hypothesize that the absence of these barriers improve adherence in the Canadian thalassemic population. We also explored self-efficacy as a concept of adherence behavior in our patient population, defined as “individuals' personal beliefs regarding their capabilities to carry out a specific task to achieve a desired outcome” (Bandura, 1989). Methods: A cross-sectional survey was conducted in June and July 2011 at a regional comprehensive care center for transfusion-dependent thalassemia patients. We assessed the age, sex, education, employment status, insurance coverage, types and dosage of ICT, self-reported level of adherence, and side effects. We adapted the Medication Adherence Self-Efficacy Scale (MASES) to assess self-efficacy (Ogedegbe, 2003). Results: Survey return rate was 45% (46/103), with each type of ICT proportionally represented (P = 0.6401). Eight surveys were discarded due to incompletion and 38 were analyzed. Thirty-two patients were on single agent ICT (6 on DFO, 23 on DFX, 3 on DFP) and 6 patients were on combination treatment (1 on DFO+DFX; 3 on DFO+DFP; 2 on DFX+DFP). Median duration of iron chelation was more than 10 years. All patients had either government (n = 10) or workplace (n = 28) coverage. Twenty-three patients (61%) were self-described as completely adherent and 15 were not completely adherent. Mean level of adherence is 90% (SD 16%), similar to those reported in the literature (Trachtenberg et al., 2011), with no significant difference between the different types of ICT (P = 0.1085). Half of the non-adherent patients (8/15, 53%) miss 1 prescribed day of medication per week. There was no significant difference between adherent and non-adherent patients in age (P = 0.1484), sex (P = 0.3764), type of insurance coverage (P = 4752), family support (P = 0.7190), type of ICT (P = 0.0611), participation and satisfaction with the Exjade Patient Support Program (P = 1.000 and 0.3012 respectively), duration of chelation (P = 0.3951), rate of side effects (P = 0.4167), or feelings of depression (P = 0.4780). There was a trend towards differences in education level (P = 0.0565) and a higher proportion of professionals in the non-adherent group. The mean self-efficacy score of patients self-described as completely adherent was significantly higher than the non-completely adherent group (2.66 vs 1.93, P<0.0001). Discussion: In this self-reported survey of patients on ICT in a Canadian regional comprehensive care center, age, presence of family support, and feelings of depression were not found to be a significant predictor of poor adherence, unlike previous studies. This could be because previous studies only examined certain types of ICTs whereas the present study examined all forms of chelation. Small sample sizes of patients on DFO and DFP is the main limitation of the study. This is also the first known application of self-efficacy theory in explaining adherence to ICT. Further studies are required to examine the internal consistency and test-retest reliability of MASES in evaluating self-efficacy in adherence to ICT. Disclosures: Kuo: Novartis Canada: Research Funding. Off Label Use: Deferiprone is an unlicensed drug in Canada and USA. It is an oral iron chelator.

Iron Overload and Iron Chelation Therapy in Pediatric Patients

2009 ◽  
Vol 02 ◽  
pp. 64
Author(s):  
Elliott Vichinsky ◽  
Keyword(s):  
Iron Overload ◽  
Patient Compliance ◽  
Pediatric Patients ◽  
Chelation Therapy ◽  
Growth Failure ◽  
Iron Chelation ◽  
Organ Damage ◽  
Clinical Consequence ◽  
Iron Chelation Therapy ◽  
Excess Iron

Iron overload is an unfortunate clinical consequence of repeated blood transfusions that can cause significant organ damage, morbidity, and mortality in the absence of proper treatment. Pediatric patients with transfusion-dependent pathologies face the additional risk of growth failure and poor sexual development owing to iron build-up in the anterior pituitary gland. Iron chelation therapy is necessary for the removal of excess iron, but treatment efficacy and success are highly dependent on patient compliance. Deferoxamine is a well-established but inconvenient therapy requiring parenteral administration over extended periods of time. Patient compliance can be improved with use of the oral iron chelators deferasirox and deferiprone. Long-term data have shown deferasirox to have a good safety and efficacy profile in pediatric patients.

Iron Overload and Haematopoiesis in MDS: Does Blood Transfusion Promote Progression to AML?

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 2685-2685 ◽  
Author(s):  
Lap Shu Alan Chan ◽  
Rena Buckstein ◽  
Marciano D. Reis ◽  
Alden Chesney ◽  
Adam Lam ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Dna Damage ◽  
Iron Overload ◽  
Serum Ferritin ◽  
Chelation Therapy ◽  
Bone Marrow Cells ◽  
Ferritin Level ◽  
Iron Chelation ◽  
Iron Chelation Therapy ◽  
Ferritin Concentration

Abstract Introduction: The biology of myelodysplastic syndrome (MDS) is poorly understood, and treatment options are limited. Thus, most MDS patients require chronic red blood cell transfusion, and many develop secondary iron overload. Although the pathophysiological consequences of iron overload to the heart, liver, and endocrine organs have been well characterized, its effects on haematopoiesis have not been studied. However, it has been observed that chelation therapy in iron-overloaded MDS patients may result in reduction of transfusion requirements, and recent studies have suggested a correlation between the use of iron chelation therapy and improvement in leukaemia-free survival in MDS. At the cellular level, iron toxicity is mediated in large part via the generation of reactive oxygen species (ROS). It has been shown in animal models that accumulation of ROS leads to senescence of haematopoietic stem cells, and that ROS cause DNA damage and promote the development of malignancy. These effects of ROS may be particularly important in MDS, in which haematopoiesis is already severely compromised and genetic instability is a striking feature. Hypothesis: We hypothesize that iron overload secondary to transfusion leads to increased levels of intracellular ROS in early haematopoeitic cells in MDS. The increase in intracellular ROS in MDS would be predicted to lead further impairment of haematopoiesis via stem cell exhaustion and while promoting accumulation of DNA damage by myelodysplastic stem cells and early progenitors, thus accelerating progression of MDS to acute leukaemia. Results: To test this hypothesis, we examined the relationship between transfusion-related iron overload and ROS content of CD34+ bone marrow cells in MDS. ROS content was measured in CD34+ cells by flow cytometry in bone marrow aspirates from 34 consecutive MDS patients (CMML=4, MDS/MPD=2, RA=4, RARS=3, RCMD=2, RAEB 1=6, RAEB 2=12, RAEB-t/AML=1). The patients represented a wide range of prior transfusion burden (0-&gt;300 units PRBC) and serum ferritin levels (11-&gt;10000 μg/L). ROS was strongly correlated with serum ferritin concentration for patients with iron overload (serum ferritin &gt;1000 μg/L; n=14, R=0.733, p&lt;0.005). The correlation between ROS and ferritin level was even stronger in the subset of patients with RAEB 1 or RAEB 2 and iron overload (n=11, R=0.838, p&lt;0.005). In contrast, no correlation between ROS and ferritin level was demonstrated for patients with serum ferritin &lt;1000 μg/L (n=20). Importantly, iron chelation therapy was associated with a reduction in CD34+ cell ROS content in one patient. To assess the effect of iron overload on normal stem cell and progenitor function, we established a mouse model of subacute bone marrow iron overload. B6D2F1 mice were loaded with iron dextran by intraperitoneal injection (150mg total iron load over 21 days), and sacrificed three days after the end of iron loading. Iron staining of tissue sections confirmed iron deposition in the bone marrow, liver, and myocardium. The development of splenomegaly was noted in iron-loaded animals. Flow cytometric analysis revealed increased apoptosis of bone marrow cells in iron loaded mice based on annexin V+/7 AAD-staining (6.26±0.96% versus 3.54±0.99% for control mice, paired student’s t-Test p&lt;0.005). However, ROS content in CD117+ progenitors of iron loaded mice was similar to control mice. Thus, subacute iron loading in mice increases apoptosis but does not alter the ROS content of HSCs; we postulate that chronic iron overload is required to achieve this effect. Conclusions: These results establish a relationship between CD34+ cell ROS content and serum ferritin concentration in MDS patients with iron overload, and indicate that iron chelation therapy in this patient population reverses this ROS accumulation. The physiological consequences of this relationship are currently being investigated in this patient set by haematopoietic colony assays and assessment of DNA damage in CD34+ cells. Nonethelesss, these data may have key implications for the deployment of iron chelation therapy in MDS patients, and may explain the association between the use of iron chelation and improved leukaemia-free survival in MDS.

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