Plain English Summary
Background and study aims
Myelodysplastic syndrome (MDS) is a type of blood cancer that limits the body's ability to produce blood cells. Patients with MDS receive an especially large number of blood transfusions. It is well known that a significant fraction of all manufactured blood units are transfused to patients with blood disorders, such as leukemia. Many patients with MDS get very large numbers of transfusions, resulting in very large costs. However, previous studies have not been able to estimate the total costs for transfusion therapy for this patient group, and has not been able to look in detail at these costs.
In the proposed project researchers will calcluate how much blood patients with MDS receive and what transfusion therapy for these patients would cost.
Who can participate?
All adult patients with a confirmed histopathological diagnosis of MDS in the time period from 2008-2017, living in Sweden.
What does the study involve?
Patient records will be analysed to estimate the costs related to blood transfusions.
What are the possible benefits and risks of participating?
Where is the study run from?
Karolinska Institutet, Sweden
When is the study starting and how long is it expected to run for?
February 2020 to August 2020
Who is funding the study?
1. Celgene, USA
2. Vetenskapsrådet (Swedish Research Council, VR)
Who is the main contact?
Dr Gustaf Edgren
Transfusion use in patients with myelodysplastic syndromes (MDS): a nationwide, retrospective cohort study
The overarching aim of the study is to perform a detailed characterization of blood transfusion use -- incorporating ensuing direct and indirect costs -- for patients with myelodysplastic syndromes (MDS).
Approved 02/05/2019 Swedish Ethical Review Authority (Etikprövningsmyndigheten, Box 2110,
750 02, Uppsala, Sweden; +46 10-475 08 00; email@example.com), ref: 2018/167-31 and 2019-02636
Retrospective cohort study
Primary study design
Secondary study design
Patient information sheet
No participant information sheet available
The study will include all adult patients with a confirmed histopathological diagnosis of MDS in the time period from 2010-2017, living in Sweden. In these patients, researchers will use nationwide registers to identify all blood transfusions. By incorporating known direct costs, and projected indirect costs, for transfusion therapy, researchers will estimate both blood use and the overall total costs for transfusion therapy in this patient group. Analyses will be conducted by first computing a cumulative number of transfusions per patient as a function of time since diagnosis and then the cumulative average number of transfusions for each patient by dividing this number with the corresponding number of patients who remain alive and under follow-up at the same time. Using this adjusted model, researchers can derive blood utilization estimates which incorporate both mortality, cure following aggressive treatment such as transplantation, as well as transition between risk groups. Estimates will thus be more specific for the subgroup at hand. Confidence intervals for averages can be constructed using boot strap methods. All analyses will be stratified by patient IPSSR risk group.
Primary outcome measure
Total transfusion cost, incorporating both direct and indirect costs, related to blood transfusions measured using patient records
Secondary outcome measures
Measured using patient records:
1. Total direct cost for transfusion therapy
2. Total indirect for transfusion therapy
3. Total number of blood transfusions, overall, and divded by component type
Overall trial start date
Overall trial end date
Reason abandoned (if study stopped)
Participant inclusion criteria
Adult patients with a confirmed histopathological diagnosis of MDS in the time period from 2008-2017, living in Sweden
Target number of participants
Total final enrolment
Participant exclusion criteria
Reseachers will restrict the analyses to patients with known WHO-subgroup and IPSS/IPSS-R risk group respectively
Recruitment start date
Recruitment end date
Countries of recruitment
Trial participating centre
Funding Body Type
private sector organisation
Funding Body Subtype
For-profit companies (industry)
United States of America
Swedish Research Council, VR
Funding Body Type
Funding Body Subtype
Results and Publications
Publication and dissemination plan
Results will be published in an international peer reviewed journal.
IPD sharing statement:
The datasets generated during and/or analysed during the current study are not expected to be made available due to no IRB approval granted to share data.
Intention to publish date
Participant level data
Not expected to be available
Basic results (scientific)
- ISRCTN38059137_PROTOCOL_v4.0_.pdf uploaded 05/02/2020