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

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

Study Overview

• Status: Unknown
• Conditions: β-thalassemia
• Intervention / Treatment: Other: The Medical Outcomes Short-Form 36-Item Health Survey (SF-36v2) It

Detailed 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

• Study Type: Observational
• Enrollment (Anticipated): 75

Contacts and Locations

• Study Contact: Name: Osama A Ibrahiem, Prof; Phone Number: 00201006372498; Email: oibrahiem@yahoo.com
• Study Contact Backup: Name: Rania M Hafez, doctor; Phone Number: 00201000019198; Email: raniahafez@ymail.com

Study Locations

• Egypt
  • Assiut, Egypt, Assiut university71515
    • Assiut University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 65 years (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

Seventy five thalassemic patients will be included in the study from haematology unit in internal medicine department in Assiut University Hospital.

Description

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 ,

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

β-thalassemia group; SICT It is a questionnaire to assess patient satisfaction with ICT regimens. It comprises 19 items assessing four domains: perceived effectiveness of ICT (PE), burden of ICT (BD), acceptance of ICT (AC), and side effects of ICT (SE). Patients rate all items on scale from 1 "very dissatisfied" to 5 "very satisfied". Lab methods : full history and thorough clinical evaluation.; . Complete blood count. .3- Serum ferritin . 4-Renal function tests. 5-liver function tests.

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. .; Other Names: Satisfaction with ICT Questionnaire (SICT) Complete blood count. - Serum ferritin . -Renal function tests. -liver function tests.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: Assiut University

Publications and helpful links

General Publications

• 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. doi: 10.1111/j.1537-2995.2007.01416.x.
• Bollig C, Schell LK, Rucker 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(8):CD007476. doi: 10.1002/14651858.CD007476.pub3.
• 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. doi: 10.1111/j.1537-2995.2007.01398.x.
• 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.
• 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.

Study record dates

Study Major Dates

• Study Start (ANTICIPATED): December 1, 2017
• Primary Completion (ANTICIPATED): December 1, 2018
• Study Completion (ANTICIPATED): March 1, 2020

Study Registration Dates

• First Submitted: November 27, 2017
• First Submitted That Met QC Criteria: November 27, 2017
• First Posted (ACTUAL): November 30, 2017

Study Record Updates

• Last Update Posted (ACTUAL): November 30, 2017
• Last Update Submitted That Met QC Criteria: November 27, 2017
• Last Verified: November 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Thalassemia; beta-Thalassemia
• Other Study ID Numbers: QOLATSIBTPRD

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No