Brief Title
Ambispective Observational Study to Evaluate the Incidence and Management of Aplastic Anemia in Spain
Official Title
Ambispective Observational Study to Evaluate the Incidence and Management of Aplastic Anemia in Spain
Brief Summary
This is a national, multicenter, ambispective, observational post-authorization study (EPA-SP
for its acronym in Spanish) to describe the incidence, clinical management and outcome of
aplastic anemia in hospitals throughout Spain.
Detailed Description
This is a national, multicenter, ambispective, observational post-authorization study (EPA-SP
for its acronym in Spanish) to describe the incidence, clinical management and outcome of
aplastic anemia in hospitals throughout Spain.
The study includes a retrospective chart review to identify all cases of aplastic anemia
diagnosed between January 2010 and the date of the study initiation and a prospective study
to detect new cases of aplastic anemia during an 18-month period since the study initiation
in the participant hospitals. The study is also designed to collect both retrospective and
prospective data on clinical management and outcome of patients with confirmed aplastic
anemia. All patients included in the study, including those cases of aplastic anemia
identified since 2010 until the study initiation through the retrospective chart review that
are alive at the time of inclusion in the study, will be followed up until death or
lost-to-follow-up or until 6 months after the last patient is enrolled in the study.
The study consists of a baseline visit, follow-up visits every 6 months (± 15 days) until
death or lost-to follow-up or up to 6 months after the last patient is enrolled in the study,
and a final visit (final/premature withdrawal visit) which will be performed at the study end
(6 months after the last patient is enrolled) or in case of premature withdrawal, except for
patient withdrawal of consent, loss-to-follow-up or death.
Study Type
Observational [Patient Registry]
Primary Outcome
To evaluate the incidence of aplastic anemia
Secondary Outcome
1. Number of patients diagnosed with moderate and severe aplastic anemia.
Condition
Aplastic Anemia
Intervention
incidence aplastic anemia
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Other
Estimated Enrollment
109
Start Date
January 31, 2018
Completion Date
September 30, 2022
Primary Completion Date
September 1, 2021
Eligibility Criteria
Inclusion Criteria:
- Patients of both sexes and of any age
- Confirmed diagnosis of spinal cord aplasia, regardless of its severity (moderate,
severe or very serious), from January 1, 2010 (until the beginning of the study)
(retrospective study) and during the inclusion period of 18 months in hospitals
Participants (prospective study).
Aplasia is defined by a cellularity of the bone marrow <25% and the presence of less than
the following: (i) hemoglobin <100 g / l (ii) platelet count <50 x109 / l (iii) Neutrophil
count < 1.5 x 109 / l.
- Any severity of the disease according to hematological and spinal criteria (5):
moderate aplasia (absolute neutrophil count [ANC]> 0.5 x 109 / l), severe (ANC
0.2-≤0.5) x 109 / l) or very severe (RAN <0.2 x 109 / l).
- Patients who voluntarily understand and sign the informed consent (if it can be
provided), preferably in writing or orally before a witness, or will be obtained from
the legal representative of the patient (for children under 16 years of age) before
the start of the study. The deceased patients and the patients with whom they can not
be contacted or have lost their follow-up, who have been diagnosed since January 1,
2010 until the beginning of the study, are exempt from the requirement of consent.
Exclusion Criteria:
- Patients with any medical or psychological alteration that, in the opinion of the
investigator, could compromise the patient's ability to grant their informed consent.
- Evidence of Fanconi anemia, dyskeratosis congenita, congenital spinal cord syndrome,
and myelodysplastic syndrome.
Gender
All
Ages
N/A - N/A
Accepts Healthy Volunteers
No
Contacts
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Location Countries
Spain
Location Countries
Spain
Administrative Informations
NCT ID
NCT04001686
Organization ID
IIS-APM-2017-01
Responsible Party
Sponsor
Study Sponsor
Asociacion Instituto Biodonostia
Study Sponsor
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Verification Date
July 2021