Observational Study of Fostamatinib as Second Line Therapy in Adult Patients With Immune Thrombocytopenia (ITP) and Insufficient Response to a Prior Therapy

Study Overview
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Brief Summary
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Evaluate patient characteristics, treatment patterns, clinical outcomes and safety of fostamatinib as second-line therapy in real-world clinical practice, for the treatment of ITP in adult patients who have an insufficient response to prior therapy (steroids ± immunoglobulins).

Detailed Description
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Evaluate patient characteristics, treatment patterns, clinical outcomes and safety of fostamatinib as second-line therapy in real-world clinical practice, for the treatment of ITP in adult patients who have an insufficient response to prior therapy (steroids ± immunoglobulins).

Official Title
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Observational Study of Fostamatinib as Second Line Therapy in Adult Patients With Immune Thrombocytopenia (ITP) and Insufficient Response to a Prior Therapy (FORTE)

Conditions
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ITP, Immune Thrombocytopenia

Intervention / Treatment
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* Drug: Fostamatinib


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Willing and able to provide written informed consent Diagnosis of ITP in adult patients (≥ 18 years of age) with an insufficient response to steroids ± immunoglobulins and requiring a change in therapy For Cohort 1, intent to initiate fostamatinib at enrollment as change in therapy For Cohort 2, currently undergoing treatment with fostamatinib as second line therapy for at least 12 weeks, with the intent to continue, at the time of enrollment Exclusion Criteria: Any prior ITP therapy/treatment other than steroids ± immunoglobulins (e.g. rituximab, thrombopoietin receptor agonists (TPO), splenectomy) Participation in any interventional study during the course of this study Lack of historical platelet counts, at time of fostamatinib initiation for those entering Cohort 2 Pregnant and/or nursing

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 100 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Initiating treatment with fostamatinib as second-line therapy<br> | Drug: Fostamatinib<br>* Fostamatinib disodium<br>|
| Treated with fostamatinib for at least 12 weeks as second-line therapy<br> | Drug: Fostamatinib<br>* Fostamatinib disodium<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Fostamatinib dosing | Changes in fostamatinib dosing | 12-month observation period | 
| Platelet counts over time during the course of fostamatinib therapy | Platelet count and change from baseline in platelet count over time. | 12-month observation period | 
| Use of concomitant medications related to ITP | Use of concomitant medications related to ITP during the study. | 12-month observation period | 
| Use of ITP Rescue Medication | The number of patients with ITP rescue medication use at any time during the study. | 12-month observation period | 
| Safety of fostamatinib | Evaluate continued safety of fostamatinib in ITP patients through adverse events | 12-month observation period | 
| ITP-PAQ Quality of life measure | Use of the score from questionnaire to assess disease-specific quality of life in adults with ITP. | 12-month observation period | 
| Treatment satisfaction measure (MSQ) |  | 12-month observation period | 
| SF-36 Quality of life measure | Scale of functional health and well-being from the patient's point of view. | 12-month observation period | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Immune Thrombocytopenia, ITP