Cardiopulmonary Assessment at Rest and during Exercise in patients with Sickle Cell Disease

ISRCTN ISRCTN63190358
DOI https://doi.org/10.1186/ISRCTN63190358
Secondary identifying numbers N/A
Submission date
02/01/2014
Registration date
22/01/2014
Last edited
02/02/2018
Recruitment status
No longer recruiting
Overall study status
Completed
Condition category
Haematological Disorders
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Record updated in last year

Plain English summary of protocol

Background and study aims
People with sickle cell disease (SCD) were unlikely to survive childhood in the past but treatment has improved. There are new challenges. It is known that ageing groups of SCD patients in the UK will have complications such as raised pressure in the lungs (pulmonary hypertension) and the same will happen in developing countries. Early intensive treatment of SCD (involving exchange transfusion and drugs designed to increase haemoglobin levels, such as 5-HU) may prevent such complications. The aim of the study is to assess this, by using exercise magnetic resonance imaging and echocardiography in patients undergoing intensive conventional therapy for SCD.

Who can participate?
All adults 18 years of age and older with homozygous SCD.

What does the study involve?
The study will include 3 visits over 12 months. Each visit is expected to last no more than 60 minutes.
Visit 1: patients will be asked to do a symptom limited exercise which will normally last no more than 10 minutes. They will have the following tests: cardiac magnetic resonance scan with exercise (cMR augmented CPEX), exercise echocardiography (ECHO) and Chester Step test with respiratory gas analysis (CPEX).
Visit 2 (after 6 months): ECHO and CPEX
Visit 3 (after 12 months): all tests
The data acquired at rest and during the exercise will be compared at each visit and across the duration of study.

What are the possible benefits and risks of participating?
This study will help the understanding of the effects of sickle cell disease on the heart and lungs of patients with this condition. Magnetic resonance and echocardiography have no proven side effect (no ionising radiation).

Where is the study run from?
This study is run from the University College London Hospital (UCLH), UK.

When is the study starting and how long is it expected to run for?
Recruitment starts on 13th January 2014 and is expected to run for 1 year.

Who is funding the study?
Biomedical Research Centre (BRC) Cardiometabolic programme (UK)

Who is the main contact?
Dr Emmanuel Ako; emmanuel.akoh@uclh.nhs.uk
Dr Malcolm Walker; malcolm.walker@ucl.ac.uk

Contact information

Dr Malcolm Walker
Scientific

The Hatter Cardiovascular Institute
67 Chenies Mews
London
WC1E 6HX
United Kingdom

Phone +44 (0)20 3447 9951
Email m.walker@ucl.ac.uk

Study information

Study designProspective longitudinal clinical observational study
Primary study designObservational
Secondary study designLongitudinal study
Study setting(s)Hospital
Study typeDiagnostic
Participant information sheet Not available in web format, please use the contact details to request a patient information sheet
Scientific titleProspective longitudinal clinical observational study in patients with homozygous sickle cell disease (SCD) to assess if conventional therapy alters cardiopulmonary complications
Study acronymCARE-SCD
Study objectivesIntensive conventional therapy can prevent the development of cardiopulmonary complications.
Ethics approval(s)West London & Gene Therapy Advisory Committee (GTAC) Health Research Authority, ref: 13/LO/1893
Health condition(s) or problem(s) studiedSickle cell disease
InterventionConventional therapy including hydroxyurea and exchange transfusion. This includes the use of 5-hydroxyurea or exchange transfusions as prescribed by haematology team caring for the patient. This is standard care for sickle cell patients. Patients will be followed up under haematology routinely but have cardiac scans at baseline, 6 months and 12 months.
Intervention typeOther
Primary outcome measure1. Cardiac Magnetic Imaging can be used to assess pulmonary vascular parameters in SCD patients
2. The pulmonary vascular responses to exercise are abnormal in patients with SCD

Timepoints of measurements: 0 months (baseline) and 12 months (end)
Secondary outcome measuresTreatment of SCD with transfusion therapy and 5-hydoxyurea, designed to reduce the proportion of sickle red cells, alter vascular responses to exercise

Timepoints of measurements: 0 months (baseline), 6 months (mid) and 12 months (end)
Overall study start date13/01/2014
Completion date18/01/2016

Eligibility

Participant type(s)Patient
Age groupAdult
Lower age limit18 Years
SexBoth
Target number of participants30 patients with homozygous sickle cell disease
Key inclusion criteria1. Patients with a diagnosis of homozygous sickle cell disease
2. Aged 18 and over
Key exclusion criteria1. Age outside inclusion criteria
2. Impaired left ventricle (LV) function
3. Valvular abnormalities
4. Sickle cell crisis within 2 weeks of recruitment.
5. Acute chest syndrome within 4 weeks of recruitment
6. Principal exclusion criteria to perform an MR scan:
6.1. Permanent pacemaker
6.2. Intracerebral aneurysm clip
6.3. Pregnancy
7. Physical diabilities which don't permit the patient to ride on a bike
8. Principal exclusion criteria to perform a cardiopulmonary exercise test:
8.1. Myocardial infarction (35 days)
8.2. Syncope
8.3. Uncontrolled heart failure
8.4. Uncontrolled Asthma
8.5. Respiratory failure
8.6. Resting saturations <85%
8.7. Uncontrolled arrythmias
8.8. Active endocarditis, myocarditis, pericarditis
Date of first enrolment13/01/2014
Date of final enrolment13/01/2015

Locations

Countries of recruitment

  • England
  • United Kingdom

Study participating centre

The Hatter Cardiovascular Institute
London
WC1E 6HX
United Kingdom

Sponsor information

University College London (UK)
University/education

c/o Mr David Wilson
Gower Street
London
WC1E 6BT
England
United Kingdom

Phone +44 (0)20 3447 5199
Email david.wilson@ucl.ac.uk
ROR logo "ROR" https://ror.org/02jx3x895

Funders

Funder type

Research organisation

Biomedical Research Centre (BRC) (UK), Ref: BRC72/CM/MW5982

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to shareNo
IPD sharing plan summaryNot provided at time of registration
Publication and dissemination planNot provided at time of registration
IPD sharing plan

Study outputs

Output type Details Date created Date added Peer reviewed? Patient-facing?
HRA research summary 28/06/2023 No No

Editorial Notes

02/02/2018: No publications found, verifying study status with principal investigator.