The impact of iron overload and its treatment on quality of life: results from a literature review
Linda Abetz1  , Jean-Francois Baladi2  , Paula Jones2 and Diana Rofail1
1Mapi Values Ltd, Adelphi Mill, Grimshaw Lane, Bollington, Cheshire, SK10 5JB, UK
2Novartis Pharmaceuticals Corporation, Global Marketing Oncology, 180 Park Avenue, Bldg. 105, Florham Park, NJ 07932-0675,
USA
Abstract
Background
To assess the literature for the impact of iron overload and infusion Iron Chelation Therapy
(ICT) on patients' quality of life (QoL), and the availability of QoL instruments for patients undergoing infusion ICT. Also, to obtain
patients' experiences of having iron overload and receiving infusion ICT, and experts' clinical opinions about the impact of treatment on
patients' lives.
Methods
A search of studies published between 1966 and 2004 was conducted using Medline and the
Health Economic Evaluation Database (HEED). Qualitative results from patient and expert interviews were analysed. Hand searching of
relevant conference abstracts completed the search.
Results
Few studies measuring the impact of ICT with deferoxamine (DFO) on patients QoL were located
(n = 15). QoL domains affected included: depression; fatigue; dyspnoea; physical functioning; psychological distress; decrease in QoL
during hospitalization. One theme in all articles was that oral ICT should improve QoL. No iron overload or ICT-specific QoL instruments
were located in the articles. Interviews revealed that the impact of ICT on patients with thalassemia, sickle cell disease, and
myelodysplastic syndromes is high.
Conclusion
A limited number of studies assessed the impact of ICT or iron overload on QoL. All
literature suggested a need for easily administered, efficacious and well tolerated oral iron overload treatments, given the impact of
current ICT on adherence. Poor adherence to ICT was documented to negatively impact survival. Further research is warranted to continue
the qualitative and quantitative study of QoL using validated instruments in patients receiving ICT to further understanding the issues
and improve patients QoL.
Background
Iron overload is caused by an increased absorption of iron over a long
period. Iron overload generally occurs as secondary to conditions that require repeated blood transfusions. Haemoglobinopathies such as,
thalassemia and Sickle Cell Disease (SCD), and dysfunctional bone marrow conditions such as Myelodysplastic Syndromes (MDS) are all
examples of diseases requiring chronic blood transfusion. If left untreated, iron overload may result in severe morbidity (such as cardiac
disease, diabetes, failure of sexual development, osteoporosis, liver damage) and early mortality [1]. However, no immediate iron overload symptoms are present until endocrinopathies and
cardiac/organ failure occurs. Given that iron overload occurs gradually, it is likely that the impact on Quality of Life (QoL) will not be
noticed until major complications begin to occur in the teens or early twenties for patients with thalassemia and SCD. Patients with MDS
face the same problem of gradual development of iron overload with the impact on QoL not noticed until complications begin to
occur.
Deferoxamine or desferal (DFO) has until now been considered the treatment choice for
patients with chronic iron overload due to blood transfusions. When it is adhered to by patients, it significantly reduces mortality and
has an acceptable safety profile. DFO is taken by infusion, often mixed by the patients (or their parents) and takes approximately 5–15
minutes to prepare. The preparation is infused with a thin needle into the arm or abdomen 5 nights per week, for 8 to 12 hours per night
[2,3] making it extremely burdensome for the patient. The site of the infusion must be rotated.
Pumps are available, with a range in convenience – older pumps tend to be large and noisy, while newer pumps tend to be smaller and
quieter. Although DFO is regarded as an effective and well tolerated drug, local injection site reactions that are generally not serious
but bothersome to patients include bumps, rashes and bruises, and infections [4,5]. Other side effects experienced by
patients on DFO treatment include: neutropenia; haematological toxicity; shortness of breath; headaches; and dizziness [6].
Given the inconvenience and side effects of the treatment, it is likely that iron overload
treatment with infusion limits QoL, thereby inhibiting adherence in patients already limited by thalassemia, SCD or MDS [7-10].
Health Related Quality of Life (HRQoL) is a multidimensional concept that represents the
patient's overall perception of the impact of illness and its treatment. An HRQoL measure captures at a minimum, physical, psychological
(including emotional and cognitive) and social functioning [11]. The measures are seen as methods of capturing patient's opinions and feelings regarding
their disease and treatment, their perceived need for healthcare and their preferences for treatment methods and disease outcomes. A
recent study in adults with thalassaemia suggested that treatment and cultural differences did not have a major effect on the QoL of
patients [12-14]. Pakbaz et al previously suggested that emotional functioning is one of the impaired
quality of life domains in patients affected by thalassaemia [15] and a further study showed that thalassaemia patients scored low in their emotional
functioning [16].
The objective of this study was to assess the literature for the impact of iron overload and
infusion Iron Chelation Therapy (ICT) on patients' QoL and to assess the availability of QoL instruments that have been used with iron
overload patients.
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