Quality of Life and Treatment Satisfaction in β-Thalassemia Patients Receiving Deferasirox

ß-thalassemia Clinical Trial

Official title:

Quality of Life and Treatment Satisfaction in β-Thalassemia Patients Receiving Deferasirox

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT03358498
• Other study ID #: QOLATSIBTPRD
• Status: Not yet recruiting
• Phase: N/A
• First received: November 27, 2017
• Last updated: November 27, 2017
• Start date: December 1, 2017
• Est. completion date: March 1, 2020

Study information

• Verified date: November 2017
• Source: Assiut University
• Contact: Osama A Ibrahiem, Prof
• Phone: 00201006372498
• Email: oibrahiem[@]yahoo.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Regular blood transfusions are essential for the management of haematological conditions such as β-thalassemia major. As a result, however, patients with these conditions are susceptible to the development of transfusion-dependent iron overload (hemosiderosis or secondary iron overload

Description:

In the absence of a naturally occurring physiological mechanism for the removal of excess iron in the body, life-long treatment and adherence to iron chelation therapy (ICT) are necessary to prevent the morbidity and mortality that may result if excess iron is allowed to .

Deferasirox (DFO),is the oldest available form of ICT used by patients with transfusion-dependent disorders. Improvements in ICT administration convenience and tolerability are expected to improve patient's satisfaction with ICT and Health Related Quality of Life (HRQOL), thus promoting adherence to ICT regimens and potentially reducing iron overload-related morbidity/mortality and associated healthcare costs

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 75
• Est. completion date: March 1, 2020
• Est. primary completion date: December 1, 2018
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 16 Years to 65 Years

Eligibility

Inclusion Criteria:

1 - Age more than 16 years 2-transfusion related iron over load (serum ferritin more than 1000 ng/ml) 3-patients on oral iron chelation (deferasirox ) for one year or more

Exclusion

1. primary haemochromatosis

2. thalassemia minor patients

3. preseance of systemic disease that prevent patient from treatment ,

Related Conditions & MeSH terms

• beta-Thalassemia
• ß-thalassemia
• Thalassemia

Intervention

Other:
• The Medical Outcomes Short-Form 36-Item Health Survey (SF-36v2) It
SF-36v2 is questionnaire comprising 36items measuring eight dimensions of general HRQOL: physical functioning 10 items, physical health problems 4 items, bodily pain 2 items, general health perceptions 5 items, vitality 4 items, social functioning (2 items), role limitations due to emotional problems (3 items), and general mental health 5 items.

Locations

Country	Name	City	State
Egypt	Assiut University Hospital	Assiut

Sponsors (1)

Lead Sponsor	Collaborator
Assiut University

Country where clinical trial is conducted

Egypt, 

References & Publications (5)

Bollig C, Schell LK, Rücker G, Allert R, Motschall E, Niemeyer CM, Bassler D, Meerpohl JJ. Deferasirox for managing iron overload in people with thalassaemia. Cochrane Database Syst Rev. 2017 Aug 15;8:CD007476. doi: 10.1002/14651858.CD007476.pub3. Review. — View Citation

Delea TE, Edelsberg J, Sofrygin O, Thomas SK, Baladi JF, Phatak PD, Coates TD. Consequences and costs of noncompliance with iron chelation therapy in patients with transfusion-dependent thalassemia: a literature review. Transfusion. 2007 Oct;47(10):1919-29. Review. — View Citation

Fisher SA, Brunskill SJ, Doree C, Gooding S, Chowdhury O, Roberts DJ. Desferrioxamine mesylate for managing transfusional iron overload in people with transfusion-dependent thalassaemia. Cochrane Database Syst Rev. 2013 Aug 21;(8):CD004450. doi: 10.1002/14651858.CD004450.pub3. Review. — View Citation

Payne KA, Desrosiers MP, Caro JJ, Baladi JF, Lordan N, Proskorovsky I, Ishak K, Rofail D. Clinical and economic burden of infused iron chelation therapy in the United States. Transfusion. 2007 Oct;47(10):1820-9. — View Citation

Payne KA, Rofail D, Baladi JF, Viala M, Abetz L, Desrosiers MP, Lordan N, Ishak K, Proskorovsky I. Iron chelation therapy: clinical effectiveness, economic burden and quality of life in patients with iron overload. Adv Ther. 2008 Aug;25(8):725-42. doi: 10.1007/s12325-008-0085-z. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The Medical Outcomes Short-Form 36-Item Health Survey (SF-36v2)	- The Medical Outcomes Short-Form 36-Item Health Survey (SF-36v2) It is a self-administered questionnaire comprising 36-items measuring eight dimensions of general HRQOL: physical functioning (10 items), role limitation due to physical health problems (4 items), bodily pain (2 items), general health perceptions (5 items), vitality (4 items), social functioning (2 items), role limitations due to emotional problems (3 items), and general mental health (5 items). In addition to scores for individual dimensions, two summary scores assessing physical and mental dimensions of health and well-being can also be calculated: Physical Component Summary (PCS) score and the Mental Component Summary (MCS) score, respectively	Baseline