Spanish-Portuguese Thrombotic Thrombocytopenic Purpura Registry
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Standard clinical practice.
- Who it may be relevant to
- Registry conditions: Purpura, Thrombocytopenic. Basic parameters: up to 99 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Spain
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Development Of Spanish-Portuguese Thrombotic Thrombocytopenic Purpura Registry (REPTT): A Study Proposal Of The Spanish Society Of Hematology And Hemotherapy (SEHH) Whit The Portuguese Society Of Hematology (SPH)
Overview
REPTT is an observational, prospective, multi-country, multicentre and non-interventional registry in which at least 300 patients with Thrombotic thrombocytopenic purpura (TTP) in Spain and Portugal will be evaluated. The study will be carried out in the context of the usual clinical practice conditions, not imposing restrictions on the participating physician or influencing their normal clinical practice.
Detailed description
TTP is a rare life-threatening haematological disease characterised by thrombotic microangiopathy (TMA) with an average annual prevalence of approximately 10 cases/million people worldwide and an annual incidence between 1.5 and 6.0 cases per million according to different studies conducted in France, the United States and in the United Kingdom. In Spain the incidence is 2,67 cases / million population per year.
Acute TTP episodes cause sequelae like vascular disease or kidney damage along with other symptoms more subtle like small neurocognitive deficits and myocardial infarction.
Thus, prompt resolution of acute episodes along with a better understanding of the cardiac abnormalities may allow to prevent further complications, to develop targeted rehabilitation techniques for TTP patients and to improve their quality of life.
This project will collect a big database capable of providing better answers to questions related with treatment efficacy, associated morbidity and mortality, and the possible neurocognitive and cardiac sequelae derived from relapses and acute episodes. Additionally, this project will be linked to obtaining biological samples for a serum and DNA library from patients with TTP.
Patients will be recruited by medical researchers specialized in haematology or by other investigators specialized in thrombotic microangiopathies disease management. This recruitment will be performed in a competitive manner. The collection period will be at least 3 years with the possibility of extending it.
REPTT aims to evaluate new scores and prognostic factors of morbidity and mortality in TTP patients.
The final aim is to establish guidelines and recommendation to improve the global management, diagnosis and treatment of patients with TTP in real-life.
Interventions
- Other Standard clinical practice
The study will be carried out in the context of the usual clinical practice conditions, not imposing restrictions on the participating physician or influencing their normal clinical practice.
Primary outcome measures
- Incidence of TTP in Spain/Portugal [Time frame: Throughout the study period, calculated for the 3 years of expected duration.]
- Morbidity of TTP in Spain/Portugal [Time frame: Throughout the study period, calculated for the 3 years of expected duration.]
- Mortality of TTP in Spain/Portugal [Time frame: Throughout the study period, calculated for the 3 years of expected duration.]
Secondary outcome measures (12)
- Clinical response to treatment rate [Time frame: Throughout the study period, approximately 3 years per patient]
- TTP Clinical remission rate [Time frame: Throughout the study period, approximately 3 years per patient]
- TTP Exacerbation rate [Time frame: Throughout the study period, approximately 3 years per patient]
- TTP Relapse rate [Time frame: Throughout the study period, approximately 3 years per patient]
- Refractory TTP rate [Time frame: Throughout the study period, approximately 3 years per patient]
- Time-to-response (TTR) [Time frame: Throughout the study period, approximately 3 years per patient]
- Duration of response (DoR) [Time frame: Throughout the study period, approximately 3 years per patient]
- Relapse-free survival (RFS) [Time frame: Throughout the study period, approximately 3 years per patient]
- Overall survival (OS) [Time frame: Throughout the study period, approximately 3 years per patient]
- Frequency of serious adverse events (SAEs) [Time frame: Throughout the study period, approximately 3 years per patient]
- Rate of complications associated with plasma exchange treatment [Time frame: Throughout the study period, approximately 3 years per patient]
- Frequency of complications associated with plasma exchange treatment [Time frame: Throughout the study period, approximately 3 years per patient]
Eligibility criteria
Inclusion criteria
- Patients with diagnosis of TTP according to International Consensus criteria from centres in Spain and Portugal.
- Patients that voluntarily sign the informed consent. For subjects unable to provide informed consent, a fully recognized medical authority may be used according to local laws.
- Patients between 0 to 99 years old at the time of diagnosis.
Note: Decision was taken to treat the patient with an specific treatment prior and independently of patient inclusion in this non interventional study.
Exclusion criteria
- Inability to comply with study procedures and follow-up exams.
- Patients with any type of alteration that compromises their ability to grant written informed consent.
- Patients that do not consent to participate in the study and do not sign informed consent.
- Patients that do not meet the criteria previously mentioned for TTP.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Study design
- Observational model
- Cohort
Study locations
Spain · 7 centers
- Hospital Universitario Virgen del Rocío — Seville
- Hospital Universitario Bellvitge — L'Hospitalet de Llobregat
- Hospital Clínic Barcelona — Barcelona
- Hospital Universitario de Guadalajara — Guadalajara
- Hospital Universitario La Paz — Madrid
- Hospital Virgen de la Arrixaca — Murcia
- Hospital Clínico Universitario de Valencia — Valencia
Identifiers
NCT: NCT05914441 · RE-PTT