Evaluation of HRQoL in Adult With Immune Thrombocytopenia in China

Immune Thrombocytopenia Clinical Trial

— HRQoL  

Official title:

A Multi-Center Prospective Study of Evaluating Health Related Quality of Life in Adult With Immune Thrombocytopenia in China Using Real-world Data

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03324087
• Other study ID #: ITP-HRQoL
• Status: Recruiting
• Phase: N/A
• First received: August 21, 2017
• Last updated: October 26, 2017
• Start date: May 1, 2017
• Est. completion date: May 2018

Study information

• Verified date: October 2017
• Source: Shandong University
• Contact: Ming Hou
• Phone: +86-18560087007
• Email: houming[@]medmail.com.cn
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Immune thrombocytopenia (ITP) is a heterogeneous disorder with variable clinical symptomsHealth related quality of life (HRQoL) could be considered in ITP, as in numerous chronic diseases, as a method to provide information about the effects of medical interventions. Symptoms of ITP, such as spontaneous bruising, menorrhagia, mucosal bleeding and prolonged bleeding with injury, may significantly affect HRQoL in ITP subjects. Treatments for chronic ITP can also be associated with substantial side effects. Restoring and/or maintaining quality of life should be an important goal of treatment. We conducted a real-world multicenter study using SF-36 and ITP-PAQ questionnaires to assess the HRQoL in patients with ITP in the real world, and analyze the influencing factors of HRQoL so as to provide a sufficient basis for clinical decision making.

Description:

Immune thrombocytopenia (ITP) is an autoimmune thrombocytopenic syndrome characterized by decreased platelet count and an increased risk of bleeding.The pathogenesis of ITP has been considered as autoantibody-mediated and cell-mediated platelet over-destruction. The estimated prevalence for ITP in the China is 5-10/100,000. Adult women are disproportionately affected by the disorder, with a female to male ratio of nearly two to one. The disorder rarely remits spontaneously in adult subjects. The mortality rate is relatively low (< 1%) in adults less than 65 years of age. Morbidity increases above age 65, primarily as a result of an increase in age-related major bleeding events.

The decision to treat and the type of treatment are controversial, since among the different medications available, none has proven its superiority regarding reduction in bleeding complications or transition to chronic ITP. Furthermore, profound thrombocytopenia, the risk of intracranial bleeding, and the appearance of bruises may seriously influence lifestyle, school functioning and physical activities and frequently result in anxiety in children and adults. Therefore, since the benefit-risk ratio of current medications is unclear and as the psychological and the physical burden of the disease is highly variable, it appears necessary to develop new tools to better understand the exact impact of the disease on daily life. Although treatment decision making depends essentially on the physician's experience, it seems of interest to include in this reflection a measurement of quality of life. In this context, evaluation of health related quality of life (HRQoL) could be considered in ITP, as in numerous chronic diseases, as a method to provide information about the effects of medical interventions.

Symptoms of ITP, such as spontaneous bruising, menorrhagia, mucosal bleeding and prolonged bleeding with injury, may significantly affect HRQoL in ITP subjects. Treatments for chronic ITP can also be associated with substantial side effects. In addition, subjects who are resistant to current therapies are likely to experience an even greater decrement to their HRQoL than responders to treatment. Thus, restoring and/or maintaining quality of life should be an important goal of treatment.

HRQoL can be analyzed by universal or disease specific scales, the latter usually have better reactivity. Medical Outcomes Study Short Form 36 Health Survey (SF-36) is one of the most widely used universal HRQoL scales, and it is widely used in the HRQoL measurement of general population, evaluation of clinical trials and health policy evaluation. The ITP Participants Assessment Questionnaire (ITP-PAQ) was developed to assess disease-specific quality of life (QoL) in adults with ITP. It is a 44-item questionnaire that includes scales for physical health (symptoms, fatigue/sleep, bother, and activity), emotional health (psychological and fear), overall QoL, social activity, women's reproductive health, and work. The reliability and validity of these scales have been confirmed by a number of studies at home and abroad.

Therefore, the investigators conducted a real-world multicenter study using SF-36 and ITP-PAQ questionnaires to assess the HRQoL in participants with ITP in the real world, and analyze the influencing factors of HRQoL so as to provide a sufficient basis for clinical decision making.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 1000
• Est. completion date: May 2018
• Est. primary completion date: May 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 14 Years to 90 Years

Eligibility

Inclusion Criteria:

1. Meet the diagnostic criteria for immune thrombocytopenia.

2. aged of 14-80 years.

3. Need or do not need treatments.

Exclusion Criteria:

1. Patients have any concomitant disease that could impact their quality of life significantly such as mental disorders

2. Severe medical condition (lung, hepatic or renal disorder) other than ITP. Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia)

3. Have a known diagnosis of other autoimmune diseases, established in the medical history and laboratory findings with positive results for the determination of antinuclear antibodies, anti-cardiolipin antibodies, lupus anticoagulant or direct Coombs test.

4. Patients who are deemed unsuitable for the study by the investigator.

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Diagnostic Test:
• HRQoL
use SF-36 and ITP-PAQ questionnaires to assess the HRQoL in patients with ITP in the real world, and analyze the influencing factors of HRQoL so as to provide a sufficient basis for clinical decision making.

Locations

Country	Name	City	State
China	Shandong University Qilu hospital	Jinan	Shandong

Sponsors (9)

Lead Sponsor	Collaborator
Shandong University	Binzhou Medical University, Jining Medical University, Liaocheng People's Hospital, Linyi People's Hospital, Qilu Hospital of Shandong University (Qingdao), Shengli Oilfield Hospital, Weifang People's Hospital, Yantai Yuhuangding Hospital

Country where clinical trial is conducted

China, 

References & Publications (5)

Efficace F, Mandelli F, Fazi P, Santoro C, Gaidano G, Cottone F, Borchiellini A, Carpenedo M, Simula MP, Di Giacomo V, Bergamaschi M, Vincelli ID, Rodeghiero F, Ruggeri M, Scaramucci L, Rambaldi A, Cascavilla N, Forghieri F, Petrungaro A, Ditonno P, Caocci G, Cirrincione S, Mazzucconi MG. Health-related quality of life and burden of fatigue in patients with primary immune thrombocytopenia by phase of disease. Am J Hematol. 2016 Oct;91(10):995-1001. doi: 10.1002/ajh.24463. Epub 2016 Jul 14. — View Citation

Flores A, Klaassen RJ, Buchanan GR, Neunert CE. Patterns and influences in health-related quality of life in children with immune thrombocytopenia: A study from the Dallas ITP Cohort. Pediatr Blood Cancer. 2017 Aug;64(8). doi: 10.1002/pbc.26405. Epub 2017 Jan 23. — View Citation

Grace RF, Neunert C. Second-line therapies in immune thrombocytopenia. Hematology Am Soc Hematol Educ Program. 2016 Dec 2;2016(1):698-706. Review. — View Citation

Lambert MP, Gernsheimer TB. Clinical updates in adult immune thrombocytopenia. Blood. 2017 May 25;129(21):2829-2835. doi: 10.1182/blood-2017-03-754119. Epub 2017 Apr 17. Review. — View Citation

Mathias SD, Li X, Eisen M, Carpenter N, Crosby RD, Blanchette VS. A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Determine the Effect of Romiplostim on Health-Related Quality of Life in Children with Primary Immune Thrombocytopenia and Associated Burden in Their Parents. Pediatr Blood Cancer. 2016 Jul;63(7):1232-7. doi: 10.1002/pbc.25984. Epub 2016 Apr 1. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	ITP-PAQ	ITP Patient Assessment Questionnaire (ITP-PAQ) was developed to assess disease-specific quality of life (QoL) in adults with ITP. It is a 44-item questionnaire that includes scales for physical health (symptoms, fatigue/sleep, bother, and activity), emotional health (psychological and fear), overall QoL, social activity, women's reproductive health, and work.	From date of randomization until the date of first documented progression,up to 12 months
Primary	SF-36	Medical Outcomes Study Short Form 36 Health Survey (SF-36) is one of the most widely used universal HRQoL scales, and it is widely used in the HRQoL measurement of general population, evaluation of clinical trials and health policy evaluation.	From date of randomization until the date of first documented progression,up to 12 months
Secondary	bleeding score		From date of randomization until the date of first documented progression,up to 12 months