Aplastic AnemiaIISAPM201701NCT04001686
Ambispective Observational Study to Evaluate the Incidence and Management of Aplastic Anemia in Spain
An observational study of Aplastic Anemia, run by Biogipuzkoa Health Research Institute at 1 site in Spain.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- incidence aplastic anemia
- How many people
- 109 participants
- Where
- 1 siteSpain
- Study dates
- 31 Jan 2018to 30 Sep 2022
- How long will I take part?
- 6 Months
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the 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.
- Conditions
- Aplastic Anemia
- Being tested
- incidence aplastic anemia
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
incidence aplastic anemiaOther
Ambispective chart review
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 2010-2022
Secondary outcomes (9)
1. Number of patients diagnosed with moderate and severe aplastic anemia.
- The number of patients diagnosed with moderate and severe aplastic anemia will calculated. The number of cases per one million people per year will be presented.
Measured: 2010-2022
2. Number of patients diagnosed with aplastic anemia according to their age and sex.
The number of males and females diagnosed with aplastic anemia per one million people per year will be calculated.
The number of patients diagnosed with aplastic anemia in each age category considered (in the statistical plan) per one million people per year will be calculated.
Measured: 2010-2022
3. Type of treatment prescribed as first-line, second-line or further-line treatment for aplastic anemia
The percentage of patients receiving each type of treatment for aplastic anemia (stem cell transplantation, immunosuppressive treatment [ATG + CSA, CSA alone], androgens, eltrombopag, etc.) for first-line, second and further lines of treatment will be calculated
Measured: 2010-2022
4. Percentage of responder patients 90, 180, 270 and 360 days after each treatment initiation.
Hematological response will be assessed on the basis of hemoglobin, platelet and absolute neutrophil count every 3 months (90 days) according to routine clinical practice.
Hematological response will be classified as response (CR and PR vs. NR). CR and PR to treatment will be defined as follows:
CR: haemoglobin levels ≥120 g/L, platelet count ≥100 x 109/L, and neutrophil count ≥1.5 x 109/L.
PR: haemoglobin levels ≥80 g/L, platelet count ≥20 x 109/L (transfusion independent), and neutrophil count ≥0.5 x 109/L.
Measured: 2010-2022
5. Best hematological response during each treatment line (CR, PR, and NR).
Percentage of patients with CR, PR and NR as the best hematological response during each treatment line and the 95% CI will be calculated.
Measured: 2010-2022
6. Overall survival, defined as the time elapsed since first-line treatment initiation until death from any cause.
The OS will be calculated as the time elapsed from first-line treatment initiation to death. Patients will be censored at the date of last follow-up if still alive at the time of the analysis. The probability of OS will be estimated using the Kaplan-Meier method. Median OS (range) and 95% CI will be calculated.
Measured: 2010-2022
7. Relapse-free survival, defined as the time elapsed since complete or partial remission achievement until relapse or death from any cause
RFS will be calculated as the time elapsed since complete or partial remission achievement until relapse or death from any cause. The probability of RFS will be estimated using the Kaplan-Meier method. Median RFS (range) and 95% CI will be calculated
Measured: 2010-2022
8. Progression-free survival, defined as the time elapsed since treatment initiation until disease progression or death from any cause.
PFS will be calculated as the time elapsed since treatment initiation until disease progression or death from any cause. Patients will be censored at the date of last follow-up if still alive or without disease progression at the time of the analysis. The probability of PFS will be estimated using the Kaplan-Meier method. Median PFS (range) and 95% CI will be calculated.
Measured: 2010-2022
9. Percentage of patients with bleeding events an infections leading to hospitalization or death during the follow-up period.
A descriptive analysis will be performed on the bleeding episodes and/or infection reported during each treatment line received, including the absolute and relative frequencies and the corresponding 95% CI.
Measured: 2010-2022
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2019
- Study start31 Jan 2018
- Primary completion1 Sep 2021 (estimated)
- Study completion30 Sep 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Other
- Enrolment
- 109 participants (actual)
- Target duration
- 6 Months
Sponsor and investigators
- Lead sponsor
- Biogipuzkoa Health Research Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04001686
- Sponsor's protocol ID
- IIS-APM-2017-01
- Acronym
- IISAPM201701
- Status last verified
- Jul 2021
- First submitted
- 26 Jun 2019
- Last updated
- 10 Aug 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 10 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ambispective Observational Study to Evaluate the Incidence and Management of Aplastic Anemia in Spain
- ClinicalTrials.gov ID
- NCT04001686
- Acronym / sponsor ID
- IISAPM201701 / IIS-APM-2017-01
- Conditions
- Aplastic Anemia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 109 participants (actual)
- Lead sponsor
- Biogipuzkoa Health Research InstituteUniversity, hospital or other
- Study start
- 31 Jan 2018 (actual)
- Primary completion
- 1 Sep 2021 (estimated)
- Study completion
- 30 Sep 2022 (estimated)
- First posted
- 28 Jun 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Other
- Time perspective
- Other
- Target follow-up
- 6 Months
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| incidence aplastic anemiaAmbispective chart review | Other | – |
Primary outcome
To evaluate the incidence of aplastic anemia
Time frame: 2010-2022
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.
9 secondary outcomes
1. Number of patients diagnosed with moderate and severe aplastic anemia.
Time frame: 2010-2022
2. Number of patients diagnosed with aplastic anemia according to their age and sex.
Time frame: 2010-2022
3. Type of treatment prescribed as first-line, second-line or further-line treatment for aplastic anemia
Time frame: 2010-2022
4. Percentage of responder patients 90, 180, 270 and 360 days after each treatment initiation.
Time frame: 2010-2022
5. Best hematological response during each treatment line (CR, PR, and NR).
Time frame: 2010-2022
6. Overall survival, defined as the time elapsed since first-line treatment initiation until death from any cause.
Time frame: 2010-2022
7. Relapse-free survival, defined as the time elapsed since complete or partial remission achievement until relapse or death from any cause
Time frame: 2010-2022
8. Progression-free survival, defined as the time elapsed since treatment initiation until disease progression or death from any cause.
Time frame: 2010-2022
9. Percentage of patients with bleeding events an infections leading to hospitalization or death during the follow-up period.
Time frame: 2010-2022
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Aug 2021.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Aplastic Anemia, run by Biogipuzkoa Health Research Institute at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 10 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the incidence of aplastic anemia (time frame: 2010-2022). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Aug 2021.