Transfusion use in patients with rare blood cancer (myelodysplastic syndromes)

ISRCTN	ISRCTN38059137
DOI	https://doi.org/10.1186/ISRCTN38059137
Secondary identifying numbers	1

Submission date: 13/01/2020
Registration date: 24/01/2020
Last edited: 02/09/2021

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Cancer

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Plain English summary of protocol

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

Study website

Contact information

Dr Gustaf Edgren
Public

Department of Medicine Solna, Karolinska Institutet
Clinical Epidemiology Division T2
Karolinska University Hospital
Stockholm
171 76
Sweden

ORCID ID	0000-0002-2198-4745
Phone	+46 708188662
Email	gustaf.edgren@ki.se

Study information

Study design	Retrospective cohort study
Primary study design	Observational
Secondary study design	Cohort study
Study setting(s)	Other
Study type	Treatment
Participant information sheet	No participant information sheet available
Scientific title	Transfusion use in patients with myelodysplastic syndromes (MDS): a nationwide, retrospective cohort study
Study acronym	SCANDAT
Study objectives	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(s)	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
Health condition(s) or problem(s) studied	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
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 study start date	01/01/2019
Completion date	01/07/2020

Eligibility

Participant type(s)	Patient
Age group	Adult
Sex	Both
Target number of participants	3,000
Total final enrolment	2858
Key inclusion criteria	Adult patients with a confirmed histopathological diagnosis of MDS in the time period from 2008-2017, living in Sweden
Key exclusion criteria	Reseachers will restrict the analyses to patients with known WHO-subgroup and IPSS/IPSS-R risk group respectively
Date of first enrolment	01/01/2008
Date of final enrolment	31/12/2017

Locations

Countries of recruitment

Sweden

Study participating centre

Karolinska Institutet

Box 281
Stockholms
17177
Sweden

Sponsor information

Karolinska Institute
University/education

Department of Medicine Solna, Karolinska Institutet
Clinical Epidemiology Division T2
Karolinska University Hospital
Stockholm
17176
Sweden

Phone	+46 8512480000
Email	info@ki.se
Website	http://ki.se/en/startpage
"ROR"	https://ror.org/056d84691

Funders

Funder type

Not defined

Celgene
Private sector organisation / For-profit companies (industry)

Alternative name(s): Celgene Corporation
Location: United States of America

Vetenskapsrådet
Government organisation / National government

Alternative name(s): Swedish Research Council, VR
Location: Sweden

Results and Publications

Intention to publish date	31/12/2020
Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not expected to be made available
Publication and dissemination plan	Results will be published in an international peer reviewed journal.
IPD sharing plan	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.

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Protocol file	version v4.0		05/02/2020	No	No
Results article		11/05/2021	02/09/2021	Yes	No

Additional files

ISRCTN38059137_PROTOCOL_v4.0_.pdf: uploaded 05/02/2020

Editorial Notes

02/09/2021: Publication reference added.
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)