Iron Overload, Thalassemia
Conditions
Keywords
quality of life, iron overload, treatment satisfaction
Brief summary
This observational clinical study aims to evaluate the HRQoL of thalassemia patients with iron overload in Greece, who are under treatment with deferasirox based on standard clinical practice.
Detailed description
The assessment of health-related quality of life (HRQoL) in thalassemic patients with iron overload offers a holistic approach to the disease and leads to better communication between physicians and patients. This prospective, non-interventional, observational clinical study aims to evaluate the HRQoL, adherence to deferasirox treatment and treatment effectiveness as well as patients' satisfaction and safety with deferasirox. All participants will complete a set of questionnaires and scales for QoL, adherence to treatment, and satisfaction of the treatment for iron overload. Demographic and clinical characteristics will be recorded to define patient subgroups based on the study protocol.
Interventions
None listed
Sponsors
Elpen Pharmaceutical Co. Inc.
Aghia Sophia Children's Hospital of Athens
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 100 Years
Healthy volunteers
No
Inclusion criteria
* 1\. Adult patients (≥ 18 years). 2. Patients with major beta-thalassemia with iron overload due to frequent blood transfusions (≥7 ml/kg/month of packed red blood cells). 3\. Patients with chronic iron overload due to blood transfusions if deferoxamine therapy is contraindicated or insufficient in the following patient groups: * adult patients with major beta-thalassemia with iron overload due to infrequent blood transfusions (\<7 ml/kg/month of packed red blood cells). * adult patients with other types of anemia. 4\. Patients with non-transfusion-dependent thalassemia syndromes with chronic iron overload requiring chelation therapy when deferoxamine therapy is contraindicated or insufficient. 5\. Patients who were receiving deferasirox before their inclusion in the study (90, 180, 360, 900 mg) and at their inclusion require treatment with deferasirox at least 900 mg, as part of the treatment of chronic iron overload and according to the SPC. 6\. Consent and compliance of participants with the treatments and procedures of the study. 7\. Patients for whom data from the last six months from the date of inclusion (medical history, concomitant medication) are available to determine the endpoints.
Exclusion criteria
* 1\. Patients under 18 years of age. 2. Patients with myelodysplastic syndromes. 3. Patients with a contraindication to taking deferasirox 90, 180, 360, 900 mg according to the drug's SmPC. 4\. Patients receiving or expect to receive another chelating agent (e.g. deferoxamine, deferiprone) in addition to deferasirox, during the study. 5\. Patients with a history of mental illness, substance abuse, to a degree that may prevent their participation in the study. 6\. Patients are participating in another research protocol.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Assessment of health-related quality of life in patients with chronic iron overload | Score of the Short-Form 36-item Health Survey (SF-36v2) questionnaire at baseline, at 6 and at 12 months and the change from baseline and among at 6 and 12 months | Score of the Short-Form 36-item Health Survey (SF-36v2) questionnaire |
Contacts
CONTACTAlexandros Ginis, MD
CONTACTPolyanthi Papanastasiou, BSc, MSc
STUDY_DIRECTORAlexandros Ginis, MD
Elpen Pharmaceutical Co. Inc.
Outcome results
None listed