Clinical trial NCT05914441: Spanish-Portuguese Thrombotic Thrombocytopenic Purpura Registry
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)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Purpura, Thrombocytopenic
Keywords: registry
Thrombotic thrombocytopenic purpura
real-world
observational
Lead sponsor: Fundación Española de Hematología y Hemoterapía
Sponsor class: OTHER
First posted: 2023-06-22
Enrollment: 300
Sex: ALL
Maximum age (years): 99
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients diagnosed with Thrombotic thrombocytopenic purpura (TTP)

Brief summary: 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.

Eligibility criteria: Inclusion Criteria:

1. Patients with diagnosis of TTP according to International Consensus criteria from centres in Spain and Portugal.
2. 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.
3. 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:

1. Inability to comply with study procedures and follow-up exams.
2. Patients with any type of alteration that compromises their ability to grant written informed consent.
3. Patients that do not consent to participate in the study and do not sign informed consent.
4. Patients that do not meet the criteria previously mentioned for TTP.

Primary outcomes:
measure: Incidence of TTP in Spain/Portugal
timeFrame: Throughout the study period, calculated for the 3 years of expected duration.
measure: Morbidity of TTP in Spain/Portugal
timeFrame: Throughout the study period, calculated for the 3 years of expected duration.
measure: Mortality of TTP in Spain/Portugal
timeFrame: Throughout the study period, calculated for the 3 years of expected duration.
Secondary outcomes:
measure: Clinical response to treatment rate
timeFrame: Throughout the study period, approximately 3 years per patient
measure: TTP Clinical remission rate
timeFrame: Throughout the study period, approximately 3 years per patient
measure: TTP Exacerbation rate
timeFrame: Throughout the study period, approximately 3 years per patient
measure: TTP Relapse rate
timeFrame: Throughout the study period, approximately 3 years per patient
measure: Refractory TTP rate
timeFrame: Throughout the study period, approximately 3 years per patient
measure: Time-to-response (TTR)
timeFrame: Throughout the study period, approximately 3 years per patient
measure: Duration of response (DoR)
timeFrame: Throughout the study period, approximately 3 years per patient
measure: Relapse-free survival (RFS)
timeFrame: Throughout the study period, approximately 3 years per patient
measure: Overall survival (OS)
timeFrame: Throughout the study period, approximately 3 years per patient
measure: Frequency of serious adverse events (SAEs)
timeFrame: Throughout the study period, approximately 3 years per patient
measure: Rate of complications associated with plasma exchange treatment
timeFrame: Throughout the study period, approximately 3 years per patient
measure: Frequency of complications associated with plasma exchange treatment
timeFrame: Throughout the study period, approximately 3 years per patient
Other outcomes:
Locations:
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocío
state: Andalucia
city: L'Hospitalet De Llobregat
country: Spain
facility: Hospital Universitario Bellvitge
state: Barcelona
city: Barcelona
country: Spain
facility: Hospital Clínic Barcelona
city: Guadalajara
country: Spain
facility: Hospital Universitario de Guadalajara
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Murcia
country: Spain
facility: Hospital Virgen de la Arrixaca
city: Valencia
country: Spain
facility: Hospital Clínico Universitario de Valencia
Study officials:
affiliation: Hospital Universitario Virgen del Rocío. Sevilla, España.
name: Dr. María Eva Mingot Castellano, M.D. Ph.D.
role: STUDY_CHAIR
References: