Top 10 research priorities for paediatric haematopoietic stem cell transplant (HSCT)

Following the completion of our priority setting partnership, we have pleased to announce the top 10 research priorities for paediatric haematopoietic stem cell transplant.

1. How can a personalised approach to treatment and medication (e.g. genetic testing) reduce adverse effects and improve recovery and outcomes for transplant patients?
2. How can treatment related toxicities best be predicted and reduced in stem cell transplant patients?
3. What are the most effective ways to prevent, identify and treat Graft versus Host Disease (GvHD)?
4. How can immune function be restored more rapidly following stem cell transplantation?
5. What is the role of a diverse intestinal microbiome in transplant outcomes, and how can this diversity be achieved?
6. What are the psychological concerns and needs of patients and families at different times during stem cell transplant?
7. Sometimes, transplanted bone marrow doesn't work properly. How do you improve its function or manage graft failure?
8. What are the necessary infection prevention and isolation measures during stem cell transplant?
9. What are the best exercises to maintain muscle and manage fatigue. How can these be used by stem cell transplant patients?
10. How does a stem cell transplant impact cognition, memory and the way you think, and can this be improved or prevented?
    
    

Download the Top 10 Priorities for Paediatric HSCT7.20 MB

Research aims

We aimed to identify questions that are important to patients, families, healthcare workers and the community which are not currently addressed by research, also known as unanswered questions.

The results of this study will help ensure that future research focuses on the needs of children requiring a BMT.

Study methodology

This study follows the scientific methods of the James Lind Alliance (JLA). 

Research on the effects of treatments often overlooks the shared interests of patients, carers and clinicians. As a result, the questions that they consider important are not addressed equally, and many areas of potentially important research are therefore neglected. The JLA method aims to address this imbalance.

In this method, a range of representatives including patients who had a BMT as a child, their parents or carers, clinicians and allied health professionals make up a steering group guided by a neutral JLA adviser. Each representative has equal input on the direction of the study.

A JLA study brings patients, caregivers, researchers and clinicians together in priority setting partnerships (PSPs). The aim of a PSP is to help ensure that those who fund health research are aware of what really matters to patients, caregivers and clinicians.

More information

An introduction to James Lind Alliance Priority Setting Partnerships

Thank you for all participants in the study.

Our first survey closed in October 2024. Nearly 150 people responded to the survey which provided over 650 individual questions about transplant. These were combined to 80 questions and investigated to see if they had already been answered by research.

The final workshop was held at the end of June 2025. It was run online, across four countries. The workshop prioritised the Top 10 questions for research based on 19 questions from survey 2.

What happens next?

The top 10 research priorities are freely available to ensure researchers, organisations and funding bodies know what is important to those who are affected by paediatric stem cell transplant.

We are ensuring future research is directed to where it is needed the most.

Your rights

If you would like more information about your rights as a participant, please contact

The Director of Research Operations at The Royal Children’s Hospital
Phone: +61 3 9345 5044
Email: [email protected]

Lead researcher

Project members

Coordinators and information specialists

Steering group members

Funding

Funding for this project has been received from the Children’s Cancer Foundation and Children's Cancer CoLab. The Children's Cancer CoLab is a collaboration of academic, research and clinical organisations which advance research for patients with cancer. 

Collaborators

We extend a heartfelt thank you to all participants, key stakeholders, partners and funders for all involvement in this Bone Marrow Transplantation in Paediatrics Priority Partnership.

• Australian and New Zealand Children’s Oncology Group (ANZCHOG) and sub-group TACTIC (national malignant discussion BMT group)
• Zero Childhood Cancer Program Patient Advisory Group (ZERO PAG)
• Australian Society of Clinical Immunology and Allergy (ASCIA) and the sub-group TAPID (national immunology BMT discussion group)
• Australia and New Zealand Transplant & Cellular Therapies (The Blood and Marrow Transplant Advisory Committee)
• Immunodeficiency UK
• Anthony Nolan | Saving lives through stem cells
• British Society of Blood and Marrow Transplantation and Cellular Therapy
• Youth Cancer Advisory Board
• Childhood Cancer Association
• Arrow: The Bone Marrow Transplant Foundation
• Bubble Foundation
• Children's Cancers Foundation
• The Kids Cancer Project
• Leukaemia & Blood Cancer New Zealand
• Maddie Riewoldt's Vision
• Paediatric Study Group - ANZICS
• Young Lives Vs Cancer
• Children's Cancer CoLab

Contact us

If you would like more information about the project, please contact us.

Ms Roxanne Dyas or Ms Beth Williams 
Email: show email address
Phone: show phone number

Murdoch Children's Research Institute
Royal Children's Hospital 
50 Flemington Road 
Parkville
Victoria 3052 Australia