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

• ClinicalTrials.gov Identifier: NCT03358498
• Recruitment Status: Unknown
• First Posted: November 30, 2017
• Last Update Posted: November 30, 2017
• Sponsor: Assiut University
• Information provided by (Responsible Party): Rania, Assiut University

Tracking Information

• First Submitted Date: November 27, 2017
• First Posted Date: November 30, 2017
• Last Update Posted Date: November 30, 2017
• Estimated Study Start Date: December 1, 2017
• Estimated Primary Completion Date: December 1, 2018 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: November 27, 2017)

The Medical Outcomes Short-Form 36-Item Health Survey (SF-36v2) [ Time Frame: Baseline ]

- 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

• Original Primary Outcome Measures: Same as current
• Change History: No Changes Posted
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Quality of Life and Treatment Satisfaction in β-Thalassemia Patients Receiving Deferasirox
• Official Title: Quality of Life and Treatment Satisfaction in β-Thalassemia Patients Receiving Deferasirox
• Brief 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

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
• Study Design: Observational Model: Cohort; Time Perspective: Cross-Sectional
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• 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.
• Condition: β-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.

.

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

Study Groups/Cohorts

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 :

1. full history and thorough clinical evaluation.
2. . Complete blood count. .3- Serum ferritin .

4-Renal function tests. 5-liver function tests.

Intervention: Other: The Medical Outcomes Short-Form 36-Item Health Survey (SF-36v2) It

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.

Recruitment Information

• Recruitment Status: Unknown status
• Estimated Enrollment (submitted: November 27, 2017): 75
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: March 1, 2020
• Estimated Primary Completion Date: December 1, 2018 (Final data collection date for primary outcome measure)

Eligibility Criteria

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 ,

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 16 Years to 65 Years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: Yes
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Egypt

Administrative Information

• NCT Number: NCT03358498
• Other Study ID Numbers: QOLATSIBTPRD
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Undecided

• Current Responsible Party: Rania, Assiut University
• Original Responsible Party: Same as current
• Current Study Sponsor: Assiut University
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Assiut University
• Verification Date: November 2017