Post HSCT study for PID patients

Clinicians at the Royal Free & Great Ormond Street Hospitals have been awarded a grant from the National Institute for Health Research (NIHR), the research arm of the NHS. The grant will fund a research study to investigate the long-term physical, social and psychological outcomes for a growing population of adults who underwent haematopoietic stem cell transplant (HSCT; also referred to as a bone marrow transplant: BMT) for PID during childhood, and to identify factors associated with these long-term health outcomes. The project will run for three years. 

The project will examine the current outcome of adults who were treated with HSCT during childhood, and compare this with that of their siblings or a nominated friend (“healthy controls”) and averages taken from a large group of the general population (“population norms”). It is hoped that the information gathered will inform clinical practice, policy-making and service delivery.

Dr Mari Campbell, Clinical Psychologist, who leads the study with Dr Penny Titman, Consultant Clinical Psychologist, said ‘Due to improved techniques, more transplants are being done and patients are living longer. However, previous research has focused on short-term medical outcome and little is known about health outcomes in adulthood, or the long-term quality of life of this population. In our own clinic, we have noticed differences in the way that adults (previously treated with HSCT for PID in childhood) experience life in the long-term.

In developing the grant proposal we talked with patients and their families, professionals, and patient support groups (IPOPI, PID UK, Royal Free PID Patient group) about patient experiences before, during and after transplant. It became clear that patients and their families would like to know more about life after HSCT, not only medically, but also looking at other types of outcomes and what patients’ lives are like as a whole.’

Invitation to patients

The research team is inviting all patients, aged over 16, who were treated with HSCT for PID at Great Ormond Street Hospital, five or more years previously, to take part in the project. Each patient will be asked to nominate a sibling or close friend who will also be asked to participate. Those who agree to participate will be asked to complete questionnaires and practical tasks to assess their current functioning and circumstances. Information will also be gathered from medical notes. 

If you would like further information about the project you are welcome to contact the team at rf.clinicalimmunology@nhs.net.

Related information

The official title of the project is 'Investigating the long-term outcomes of adults patients who underwent Haematopoeitic Stem CellTransplant (HSCT) for Primary Immunodeficiency (PID) during childhood.'

The research team includes:

Dr Mari Campbell, Clinical Psychologist
Dr Penny Titman, Consultant Clinical Psychologist
Dr Siobhan Burns, Consultant Immunologist
Professor Bobby Gaspar, Consultant Immunologist
Professor Emma Morris, Conusltant Haematologist
Dr Austen Worth,  Consultant Immunologist
Christopher Sandford, Patient Advisor
Professor Stephen Morris, Health Economist
Deborah Ridout, Statistician
Bethany Nicholson, Assistant Psychologist

Further information can be found at www.isrctn.com/ISRCTN11966493.

Posted June 2017