Clinical trial NCT06831799: ERN-EuroBloodNet Registry on Patients With Rare Red Blood Cell Defects and COVID-19
Official title: ERN-EuroBloodNet European Collaborative Platform to Share COVID-19 Data in Patients With Red Blood Cell Disorders in Real Time
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemoglobinopathies
Sickle Cell Disease
Sickle Cell Anemia
Sickle Beta Thalassemia
Sickle-Cell; Hemoglobinopathy
Sickle Cell Hemoglobin C
Sickle Cell Hemoglobin D
Sickle Cell-Hemoglobin E Disease
Beta-Thalassemia
Beta Thalassemia Major
Beta Thalassemia Intermedia
Dominant Beta-Thalassemia
Hereditary Persistence of Fetal Hemoglobin Thalassemia
Delta-Beta Thalassaemia
Haemoglobin C-Beta-Thalassaemia Syndrome
Hemoglobin E-Beta Thalassemia
Alpha-Thalassemia
Hemoglobin H Disease
Hemoglobin Barts Hydrops
Enzyme Disorder; Anemia
Membranes; Disorder
Elliptocytosis, Hereditary
Stomatocytosis
Spherocytosis, Hereditary
Keywords: COVID-19
Hemoglobinopathy
Sickle cell disease
Sickle Cell Anemia
Sickle cell-beta-thalassemia
Sickle cell-hemoglobin
Beta-Thalassemia
Beta Thalassemia Major
Beta Thalassemia Intermedia
Delta-Beta Thalassaemia
Alpha-Thalassemia
Hemoglobin
Rare hemolytic anemia
Hereditary Spherocytosis
Hereditary Elliptocytosis
Hereditary Stomatocytosis
Lead sponsor: Hospital Universitari Vall d'Hebron Research Institute
Sponsor class: OTHER
Collaborators: name: European Georges Pompidou Hospital
name: Hospital General Universitario Gregorio Marañon
name: Hospital Vall d'Hebron
name: Centro Hospitalar e Universitário de Coimbra, E.P.E.
name: University Hospital Freiburg
name: Azienda Ospedaliera di Padova
name: Hôpital Necker-Enfants Malades
name: Guy's and St Thomas' NHS Foundation Trust
name: Erasme University Hospital
name: Aghia Sophia Children's Hospital of Athens
name: Cyprus Institute of Neurology and Genetics
name: University Hospital Heidelberg
name: Children's Health Ireland
name: San Luigi Gonzaga Hospital
name: Oxford University Hospitals NHS Trust
name: UMC Utrecht
name: General Hospital of Athens Elpis
name: Amsterdam UMC
name: Brno University Hospital
name: Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello
name: University Hospital, Gentofte, Copenhagen
name: Institute of Hematology and Blood Transfusion, Czech Republic
name: Universitaire Ziekenhuizen KU Leuven
name: Palacky University
First posted: 2025-02-18
Enrollment: 684
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: OTHER
Sampling method: PROBABILITY_SAMPLE
Study population: Patients both pediatric and adults with confirmed COVID19 and affected by a rare anaemia disorder due to a red blood cell defect according to the following ORPHA codes for rare diseases:

* 68364 Hemoglobinopathy
* 275752 Sickle Cell Disease and related diseases
* 848 Beta-thalassemia
* 846 Alpha-Thalassaemia
* 98363 Rare hemolytic anemia
* 98369 Rare constitutional hemolytic anemia due to an enzyme disorder
* 98364 Rare constitutional hemolytic anemia due to a red cell membrane anomaly

Brief summary: Patients with red blood cell disorders (RBCDs), such as Sickle cell disease (SCD) and Thalassemia, are chronic, life-threatening conditions that can become multi-organ complications over time, and are likely at an increased risk of COVID-19-related complications. Patients at the highest risk include the elderly (\>50 in our population), those with a history of respiratory or cardiac disease and those with other comorbidities. Several patients affected by RBCDs undergo splenectomy as therapeutic option to improve their level of hemoglobin concentration. Splenectomized patients, or in the case of SCD with functional hyposplenism, are more vulnerable to bacterial infections / superinfections after viral infection. Acute pulmonary syndrome (ACS) is the main cause of morbidity in SCD in middle-high income countries, and is often triggered by infectious events. Currently, there is no literature on the subject. Thus, any recommendation available comes from the experience gained with previous Coronaviruses infections. Accordingly, the correct treatment and management of infection by Coronavirus SARS-COV-2 (COVID-19) in patients affected by RBCDs may be challenging given the rapid spread of the pandemic and limited literature so far, especially in some countries. Accordingly, there is an urgent need to pool evidence in a unique repository on patients affected by RBCDs and COVID-19 in order to reach critical numbers to facilitate the medical decision making process across Europe.

The Registry on patients with rare RBCDs and COVID-19 is an initiative conceived in the core of the European Reference Network on Rare Hematological Diseases (ERN-EuroBloodNet, FPA 739541, www.eurobloodnet.eu) aiming at supporting medical practice of COVID-19 in these patients by gathering evidence on pediatric and adult COVID-19 confirmed cases in RBCDs across Europe.

Detailed description: The Registry on patients with rare red blood cell disorders (RBCDs) and COVID-19 is an initiative conceived in the core of the European Reference Network on Rare Hematological Diseases (ERN-EuroBloodNet, FPA 739541, www.eurobloodnet.eu) aiming at supporting medical practice of COVID-19 in these patients by gathering evidence on pediatric and adult COVID-19 confirmed cases in RBCDs across Europe.

Primary objective of the registry is to pool evidence on the clinical management and outcomes of patients affected by red blood cell disorders and COVID-19 for supporting daily medical practice while enabling inter-professional consultation of complex cases. Secondary objective includes the performance of observational studies in the different cohorts of patients, including Sickle Cell Disease, Thalassaemia, Enzymopathies and Membranopathies patients in pediatric and or adult stages.

The network of hospitals that will be created from this registry will hold regular meetings to analyze the data that are being introduced and to discuss possible measures against COVID-19 based on them. The collaboration will continue with the development of observational studies that will give the necessary evidence to make recommendations for COVID-19 management in hematological patients.

METHODOLOGY

Inclusion criteria include both pediatric and adults patients with confirmed COVID-19 and affected by a rare anaemia disorder due to a red blood cell defect. Data set elements include:

* Demographics: country of living, sex, age (only year of birth)
* Data related to Red blood cell disorder: diagnosis, co-morbidities, treatments, splenectomy, blood transfusion requirement.
* Data related to COVID-19: date and method for diagnosis, severity grade, clinical manifestations i.e. pneumonia, symptoms days, acute events, treatments, days of hospitalization, days at intensive care unit, sequela, death.

The registry has been developed by Vall d'Hebron Research Institute using Redcap, a secure web application for building and managing online databases. Individual patients' data will be gathered in a codified way.

ETHICS AND GDPR COMPLIANCE

The processing of personal data is conducted fully respecting the Regulation (EU) 2016/679 (General Data Protection Regulation), including legal basis and special requirements and safeguards to ensure the safety and the confidentiality of the data subjects.

The Research Ethics Committee of the Vall d'Hebron's Hospital has confirmed that this exceptional case justifies the waiver of informed consent.

PROCESSING OF THE DATA

The patient's information included in the database is pseudonymised by single codification. The minimization principle of data protection is followed (i.e. only year of age is collected, no identified data is collected, and only clinical data of the health care is collected). The medical doctors at each center have a coding table in which the code can be linked to the patient's personal information. This table is safely guarded by the medical doctor and never leaves the center. All the information stored in the database is pseudonymised.

Eligibility criteria: Inclusion Criteria:

* Patients both pediatric and adults with confirmed COVID-19 and affected by a rare anaemia disorder due to a red blood cell defect

Primary outcomes:
measure: Clinical Management and Outcomes in Patients with Red Blood Cell Disorders and COVID-19
timeFrame: Through study completion, an average of 3 years
Secondary outcomes:
measure: Secondary Analysis in Different Cohorts of Patients with Red Blood Cell Disorders and COVID-19:
timeFrame: Through study completion, an average of 3 years
Other outcomes:
Locations:
city: Barcelona
country: Spain
facility: Vall d'Hebron Institut de Recerca
Study officials:
affiliation: Vall d'Hebron Institut de Recerca, Barcelona (Spain) and ERN-EuroBloodNet
name: María del Mar Mañú Pereira, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Vall d'Hebron University Hospital, Barcelona (Spain)
name: Pablo Velasco Puyó, MD
role: PRINCIPAL_INVESTIGATOR
References: