Andalusian Experience in the Use of Fostamatinib in Patients With ITP. FOSTASUR Study

Description of the use of fostamatinib in patients with PTI in the Andalusian region.

Study Overview

• Status: Completed
• Conditions: Primary Immune Thrombocytopenia
• Intervention / Treatment: Drug: Fostamatinib

Detailed Description

Fostamatinib is a splenic tyrosine kinase (SYK) inhibitor whose indications include the treatment of chronic primary immune thrombocytopenia (PTI) refractory to first-line treatments. Data from two phase 3 studies in PTI, together with supporting safety data from a comprehensive program in 3,437 subjects with rheumatoid arthritis, led to the approval and registration of fostamatinib by health authorities in the US and in Europe.

Given the recent commercialization of fostamatinib for PTI, there is little data in real clinical practice that helps its general management: how to perform tapering, response rates in less refractory patients, experience in subjects with associated immunosuppressive therapies or thrombopoietin analogues, etc. These studies outside the clinical trial environment are relevant to plan needs, consumption, efficacy, and safety results in real clinical practice in our environment, the Andalusian region. The pivotal studies present experience on 102 cases and the real-life experience is of case series of less than 5 patients, with the population of patients treated in the Andalusian region as of December 31st being greater than 40 cases. For all these reasons the Andalusian Group of Congenital Coagulopathies proposes collecting what the experience has been like in their environment. Participation in this study is not intended to change the routine treatment patients receive as determined by their prescribing physicians. No clinic visits, procedures, evaluations, or tests will be required for the purposes of the study. This is multicenter retrospective data that will be collected from information generated during routine examinations and treatments performed by the investigator according to the standard of care.

• Study Type: Observational
• Enrollment (Actual): 50

Contacts and Locations

Study Locations

• Spain
  • Granada, Spain, 18014
    • Hospital Universitario Virgen de Las Nieves
  • Granada, Spain, 18016
    • Hospital Universitario San Cecilio
  • Huelva, Spain, 21005
    • Hospital Universitario Juan Ramon Jimenez
  • Jaén, Spain, 23007
    • Hospital Universitario de Jaen
  • Málaga, Spain, 29010
    • Hospital Regional Universitario de Malaga
  • Málaga, Spain, 29010
    • Hospital Virgen de la Victoria
  • Sevilla, Spain, 41009
    • Hospital Universitario Virgen Macarena
  • Sevilla, Spain, 41013
    • Hospital Universitario Virgen del Rocío
  • Cádiz
    • Cadiz, Cádiz, Spain, 11009
      • Hospital Puerta del Mar
    • Jerez De La Frontera, Cádiz, Spain, 11407
      • Hospital Universitario de Jerez de la Frontera
    • Puerto Real, Cádiz, Spain, 11510
      • Hospital de Puerto Real
  • Huelva
    • Minas de Riotinto, Huelva, Spain, 21660
      • Hospital de Riotinto
  • Málaga
    • Ronda, Málaga, Spain, 29400
      • Hospital de la Serranía de Ronda
  • Sevilla
    • Osuna, Sevilla, Spain, 41640
      • Hospital de la Merced de Osuna

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Patients treated with fostamatinib for ITP between October 2021 (marketing date in Spain) and December 2022 in the Andalusian region.

Description

Inclusion Criteria:

1. Patients aged 18 years or older.
2. Patients with ITP criteria according to clinical practice guidelines.

Exclusion Criteria:

1. Patients with any medical or psychological condition that prevents them from following the procedures of normal clinical practice as determined by the investigator.
2. Subjects with other concomitant hemostatic defects.
3. Documented history or clinical evidence of medical conditions (other than ITP) that consider the underlying cause of thrombocytopenia.
4. Diagnosis of secondary immune thrombocytopenia.
5. Subject has a known allergy to fostamatinib or any of the ingredients included in its formulation.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
PTI patients treated with fostamatinib; Patient with criteria of PTI who has been treated with fostamatinib in the time described	Drug: Fostamatinib; Patient treated with fostamatinib in the period described; Other Names: Andalusian PTI fostamatinib patients

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Description of fostamatinib use	Description of dose and treatment duration of fostamatinib in patients primary immune thrombocytopenia (PTI)	From fostamatinib prescription first date

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Response	Platelets count greater than 30x10^9/L and greater than 50X10^9/L at least once during treatment in the absence of combination treatment or rescue therapy.	Whenever it occurs after the start of treatment with fostamatinib
Response duration	Duration in weeks of response greater than 30x10^9/L, 50x10^9/L and 100x10^9/L.	Whenever it occurs after the start of treatment with fostamatinib
Response failure rate	Platelets count less than 30x10^9/L after four weeks of treatment.	Whenever it occurs after the start of treatment with fostamatinib
Efficiency	Identify situations that limits the effectiveness of fostamatinib.	Whenever it occurs after the start of treatment with fostamatinib
Rescue treatment	Percentage of patients who required rescue therapy.	Whenever it occurs after the start of treatment with fostamatinib
Associated therapies	Percentage of patients who have required therapy for ITP associated with fostamatinib.	Whenever it occurs after the start of treatment with fostamatinib
Bleeding	Description of hemorrhagic events.	Whenever it occurs after the start of treatment with fostamatinib
Side effects	Description of side effects associated with the use of fostamatinib.	Whenever it occurs after the start of treatment with fostamatinib
Security	Number of emergencies, unscheduled visits or hospitalizations for PTI related causes during treatment with fostamatinib.	Whenever it occurs after the start of treatment with fostamatinib

Collaborators and Investigators

• Sponsor: Fundación Pública Andaluza para la gestión de la Investigación en Sevilla
• Investigators: Principal Investigator: María Eva Mingot Castellano, MD-PhD, Universisty Hospital Virgen del Rocío

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2023
• Primary Completion (Actual): April 1, 2023
• Study Completion (Actual): June 30, 2023

Study Registration Dates

• First Submitted: October 3, 2023
• First Submitted That Met QC Criteria: October 3, 2023
• First Posted (Actual): October 6, 2023

Study Record Updates

• Last Update Posted (Actual): October 17, 2023
• Last Update Submitted That Met QC Criteria: October 13, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
• Other Study ID Numbers: FOSTASUR

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Summaries or oral presentations will be presented with descriptive, preliminary results, by subgroups and finals of the study at international and national congresses of Hematology Societies.

Drug and device information, study documents

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