An international survey of patients with thalassemia major and their views about sustaining life-long desferrioxamine use

Ward, Alexandra; Jaime, J.; Green, Traci Craig; Huybrechts, Krista F.; Arana, Alejandro; Wait, Suzanne; Eleftheriou, Androulla

doi:10.1186/1472-6904-2-3

Cited by 38 publications

(56 citation statements)

References 23 publications

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“…The association between ICT-related adverse events and non-compliance observed in this study is also consistent with the findings of other earlier studies. 42,43 Patients with thalassaemia, sickle cell disease, and MDS currently undergoing ICT had much lower HRQOL scores compared with population norms, and in particular for the General Health, Role Limitations -Physical, and Physical Functioning domains of the SF-36. The dif-f f ference between the SF-36 domain study scores and the population norms were of-f f ten 3 to 5 units.…”

Section: Discussionmentioning

confidence: 92%

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

Payne¹,

Rofail²,

Baladi

et al. 2008

Adv Therapy

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Generally, patients are not achieving target serum ferritin thresholds despite chronic treatment for iron overload. ICT appears to negatively impact HRQOL; compliance with ICT is poor; and, in the case of DFO, treatment costs well exceed the cost of DFO alone. These results suggest that current ICT in the real-world setting is suboptimal with respect to various clinical, HRQOL and economic outcomes.

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Section: Discussionmentioning

confidence: 92%

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

Payne¹,

Rofail²,

Baladi

et al. 2008

Adv Therapy

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“…Several papers have been published on the degree of compliance with iron chelation therapy (ICT). Seven studies examined deferoxamine only, [22,[96][97][98][99][100][101], six examined Deferiprone only [22,55,56,[102][103][104][105] and five compared Deferoxamine and Deferiprone [57,59,62,106]. Five studies were based on Italian patients and five were based on patients in Canada.…”

Section: Compliance To Iron Chelation Therapy and Cost Implicationsmentioning

confidence: 99%

Current Status in Iron Chelation in Hemoglobinopathies

Cappellini¹,

Piga

2008

CMM

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Although blood transfusions are important for patients with hemoglobinopathies, chronic transfusions inevitably lead to iron overload as humans cannot actively remove excess iron. The cumulative effects of iron overload lead to significant morbidity and mortality, if untreated. Desferrioxamine (DFO) is the reference-standard iron chelator whose safety and efficacy profile has been established through many years of clinical use. DFO side effects are acceptable and manageable however the prolonged subcutaneous infusion regimen of 5-7 days per week is very demanding and results in poor adherence to therapy. Deferiprone (Ferriprox, L1) is a bidentate molecule, orally administrable three-times/day, licensed in Europe and in other regions but in the USA and Canada, for the treatment of iron overload in patients for whom DFO therapy is contraindicated or inadequate. Preliminary evidences suggest that Deferiprone may be more effective than DFO in chelating cardiac iron. The side effects include gastrointestinal symptoms, liver dysfunction, joint pain, neutropenia and agranulocytosis. A weekly assessment of white blood cell counts is recommended because of the risk of agranulocytosis. Deferasirox is a new, convenient, once-daily oral iron chelator that has demonstrated in various clinical trials good efficacy and acceptable safety profile in adult and pediatric patients affected by transfusion-dependent thalassemia major and by different chronic anemias (SCD, BDA, MDS). The long half-life of Deferasirox (16-18 hours) provides sustained 24 hr iron chelation coverage. The efficacy and safety profile have been evaluated in more than 1000 patients in clinical trials allowing FDA registration. Patient satisfaction with Deferasirox was superior than with DFO therapy.

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“…The effectiveness of this parenterally administered chelator is limited partly by poor patient compliance (11). In addition, because cell membranes are relatively impermeable to DFO and DFO-iron complexes, serious cardiac, hepatic and endocrine complications continue to arise, even in compliant patients (12).…”

Section: Introductionmentioning

confidence: 99%

Thyroid dysfunction in thalassaemic patients: ferritin as a prognostic marker and combined iron chelators as an ideal therapy

Valeria

Lacquaniti

Salpietro

et al. 2013

European Journal of Endocrinology

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Objective: Endocrine complications characterised patients with b thalassaemia (bT). In particular, thyroid dysfunction occurs frequently in bT major, but its long-term natural history is poorly understood. Design: A total of 72 bT patients were followed for 8 years. The incidence of thyreopathies, defined as the primary study endpoint, was assessed. The aim of this study was to analyse the prognostic role of ferritin for thyreopathies in patients with major and intermedia bT. The power of different iron chelators to treat iron overload and to prevent or reverse thyreopathies was also assessed. Methods: Patients were treated with chelators with different chelation strategies during the study. Receiver operating characteristics analysis was employed to calculate the area under the curve for serum ferritin to find the best cutoff values capable of identifying thyroid dysfunction in thalassaemic patients. Kaplan-Meier curves were generated to assess incidence of thyreopathy. Adjusted risk estimates for thyreopathy were calculated using univariate followed by multivariate Cox proportional hazard regression analysis. Results: Patients with thyroid dysfunction were characterised by higher ferritin when compared with patients without thyreopathies (1500 (872-2336) vs 513 (370-698) mg/l; P!0.0001). Patients with ferritin values above 1800 mg/l experienced a significantly faster evolution to endpoint (log-rank (c 2 ): 7.7; PZ0.005). Ferritin predicted high risk of thyroid dysfunction independently of confounding factors (hazard ratio: 1.20; P!0.0001). The intensification of chelation therapy led to an amelioration of thyroid function. Conclusions: Ferritin represents a prognostic marker for bT patients and a predictive factor for progression to thyroid dysfunction. Intensive chelation therapy allows the prevention and reversibility of thyroid complications.

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An international survey of patients with thalassemia major and their views about sustaining life-long desferrioxamine use

Cited by 38 publications

References 23 publications

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

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

Current Status in Iron Chelation in Hemoglobinopathies

Thyroid dysfunction in thalassaemic patients: ferritin as a prognostic marker and combined iron chelators as an ideal therapy

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