Clinical trial NCT04001686: 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
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Aplastic Anemia
Lead sponsor: Biogipuzkoa Health Research Institute
Sponsor class: OTHER
First posted: 2019-06-28
Enrollment: 109
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population consists of all consecutive patients diagnosed with aplastic anemia between January 2010 and the date of finalization of the 18-month inclusion period

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.

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.

Primary outcomes:
measure: To evaluate the incidence of aplastic anemia
timeFrame: 2010-2022
Secondary outcomes:
measure: 1. Number of patients diagnosed with moderate and severe aplastic anemia.
timeFrame: 2010-2022
measure: 2. Number of patients diagnosed with aplastic anemia according to their age and sex.
timeFrame: 2010-2022
measure: 3. Type of treatment prescribed as first-line, second-line or further-line treatment for aplastic anemia
timeFrame: 2010-2022
measure: 4. Percentage of responder patients 90, 180, 270 and 360 days after each treatment initiation.
timeFrame: 2010-2022
measure: 5. Best hematological response during each treatment line (CR, PR, and NR).
timeFrame: 2010-2022
measure: 6. Overall survival, defined as the time elapsed since first-line treatment initiation until death from any cause.
timeFrame: 2010-2022
measure: 7. Relapse-free survival, defined as the time elapsed since complete or partial remission achievement until relapse or death from any cause
timeFrame: 2010-2022
measure: 8. Progression-free survival, defined as the time elapsed since treatment initiation until disease progression or death from any cause.
timeFrame: 2010-2022
measure: 9. Percentage of patients with bleeding events an infections leading to hospitalization or death during the follow-up period.
timeFrame: 2010-2022
Other outcomes:
Locations:
city: San Sebastián
country: Spain
facility: Asociación Instituto Biodonostia
state: Guipuzcoa
Study officials:
References: