Condition category
Cancer
Date applied
13/01/2020
Date assigned
24/01/2020
Last edited
01/09/2020
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

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?
None.

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
gustaf.edgren@ki.se

Trial website

http://www.scandat.se

Contact information

Type

Public

Primary contact

Dr Gustaf Edgren

ORCID ID

http://orcid.org/0000-0002-2198-4745

Contact details

Department of Medicine Solna
Karolinska Institutet
Clinical Epidemiology Division T2
Karolinska University Hospital
Stockholm
171 76
Sweden
+46 708188662
gustaf.edgren@ki.se

Additional identifiers

EudraCT number

Nil known

ClinicalTrials.gov number

Nil known

Protocol/serial number

1

Study information

Scientific title

Transfusion use in patients with myelodysplastic syndromes (MDS): a nationwide, retrospective cohort study

Acronym

SCANDAT

Study hypothesis

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).

Ethics approval

Approved 02/05/2019 Swedish Ethical Review Authority (Etikprövningsmyndigheten, Box 2110,
750 02, Uppsala, Sweden; +46 10-475 08 00; registrator@etikprovning.se), ref: 2018/167-31 and 2019-02636

Study design

Retrospective cohort study

Primary study design

Observational

Secondary study design

Cohort study

Trial setting

Other

Trial type

Treatment

Patient information sheet

No participant information sheet available

Condition

Myelodysplastic syndromes

Intervention

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.

Intervention type

Procedure/Surgery

Phase

Drug names

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

01/01/2019

Overall trial end date

01/07/2020

Reason abandoned (if study stopped)

Eligibility

Participant inclusion criteria

Adult patients with a confirmed histopathological diagnosis of MDS in the time period from 2008-2017, living in Sweden

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

3,000

Total final enrolment

2858

Participant exclusion criteria

Reseachers will restrict the analyses to patients with known WHO-subgroup and IPSS/IPSS-R risk group respectively

Recruitment start date

01/01/2008

Recruitment end date

31/12/2017

Locations

Countries of recruitment

Sweden

Trial participating centre

Karolinska Institutet
Box 281
Stockholms
17177
Sweden

Sponsor information

Organisation

Karolinska Institute

Sponsor details

Department of Medicine Solna
Karolinska Institutet
Clinical Epidemiology Division T2
Karolinska University Hospital
Stockholm
17176
Sweden
+46 8512480000
info@ki.se

Sponsor type

University/education

Website

http://ki.se/en/startpage

Funders

Funder type

Not defined

Funder name

Celgene

Alternative name(s)

Celgene Corporation

Funding Body Type

private sector organisation

Funding Body Subtype

For-profit companies (industry)

Location

United States of America

Funder name

Vetenskapsrådet

Alternative name(s)

Swedish Research Council, VR

Funding Body Type

government organisation

Funding Body Subtype

National government

Location

Sweden

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

31/12/2020

Participant level data

Not expected to be available

Basic results (scientific)

Publication list

Publication citations

Additional files

Editorial Notes

01/09/2020: The following changes were made to the trial record: 1. Total final enrolment number added. 2. The intention to publish date was changed from 01/09/2020 to 31/12/2020. 05/02/2020: Uploaded protocol (not peer reviewed) as an additional file. 14/01/2020: Trial’s existence confirmed by Swedish Ethical Review Authority (Etikprövningsmyndigheten)