Immune Thrombocytopenia, ITP
Conditions
Keywords
Immune Thrombocytopenia, ITP
Brief summary
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
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).
Interventions
DRUGFostamatinib
Fostamatinib disodium
Sponsors
Rigel Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 100 Years
Healthy volunteers
No
Inclusion criteria
1. Willing and able to provide written informed consent 2. Diagnosis of ITP in adult patients (≥ 18 years of age) with an insufficient response to steroids ± immunoglobulins and requiring a change in therapy 3. For Cohort 1, intent to initiate fostamatinib at enrollment as change in therapy 4. 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
1. Any prior ITP therapy/treatment other than steroids ± immunoglobulins (e.g. rituximab, thrombopoietin receptor agonists (TPO), splenectomy) 2. Participation in any interventional study during the course of this study 3. Lack of historical platelet counts, at time of fostamatinib initiation for those entering Cohort 2 4. Pregnant and/or nursing
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Fostamatinib dosing | 12-month observation period | Changes in fostamatinib dosing |
| Platelet counts over time during the course of fostamatinib therapy | 12-month observation period | Platelet count and change from baseline in platelet count over time. |
| Use of concomitant medications related to ITP | 12-month observation period | Use of concomitant medications related to ITP during the study. |
| Use of ITP Rescue Medication | 12-month observation period | The number of patients with ITP rescue medication use at any time during the study. |
| Safety of fostamatinib | 12-month observation period | Evaluate continued safety of fostamatinib in ITP patients through adverse events |
| ITP-PAQ Quality of life measure | 12-month observation period | Use of the score from questionnaire to assess disease-specific quality of life in adults with ITP. |
| Treatment satisfaction measure (MSQ) | 12-month observation period | — |
| SF-36 Quality of life measure | 12-month observation period | Scale of functional health and well-being from the patient's point of view. |
Countries
United States
Outcome results
None listed