Ambispective Observational Study to Evaluate the Incidence and Management of Aplastic Anemia in Spain

NCT04001686 | Unknown

• 1 other identifier: IIS-APM-2017-01
• Study Type: observational
• Target: 109
• Locations: 1 country
• Sites: 1

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.

Conditions

Aplastic Anemia

Outcome Measures

Primary Outcomes (1)

• To evaluate the incidence of aplastic anemia

  The primary endpoint of the study is the number of cases of aplastic anemia per year recorded in the databases of the participant hospitals from January 2010 and the date of the study initiation (retrospective analysis) and all new cases of aplastic anemia detected per year (prospective analysis) during an 18-month period since study initation.

  2010-2022

Secondary Outcomes (9)

• 1. Number of patients diagnosed with moderate and severe aplastic anemia.

  2010-2022

• 2. Number of patients diagnosed with aplastic anemia according to their age and sex.

  2010-2022

• 3. Type of treatment prescribed as first-line, second-line or further-line treatment for aplastic anemia

  2010-2022

• 4. Percentage of responder patients 90, 180, 270 and 360 days after each treatment initiation.

  2010-2022

• 5. Best hematological response during each treatment line (CR, PR, and NR).

  2010-2022

Interventions

incidence aplastic anemia OTHER

Ambispective chart review

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• Patients of both sexes and of any age
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Biogipuzkoa Health Research Institute: lead

Study Sites (1)

Asociación Instituto Biodonostia

San Sebastián, Guipuzcoa, 20014, Spain

Location

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: OTHER
• Target Duration: 6 Months
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 26, 2019
• First Posted: June 28, 2019
• Study Start: January 31, 2018
• Primary Completion: September 1, 2021
• Study Completion: September 30, 2022
• Last Updated: August 10, 2021

Record last verified: 2021-07

Locations

ES (1)