Paediatric haematopoietic stem cell transplantation research priorities: results from an international multi-stakeholder Priority Setting Partnership

Technical Deep Dive: Paediatric Haematopoietic Stem Cell Transplantation Research Priorities

Overview

This study aimed to identify research priorities for paediatric haematopoietic stem cell transplantation (HSCT) through an international, multi-stakeholder process involving patients, parents/carers, and healthcare professionals.

Methodology

• An 18-month priority setting partnership was implemented in collaboration with the James Lind Alliance.
• Part 1: International scoping survey collected 667 research ideas from patients, parents/carers (45%), and healthcare professionals (55%).
• Part 2: 80 summary questions were created from the submitted ideas, and a systematic literature review determined that 35 were true uncertainties not answered by existing evidence.
• Part 3: 224 participants completed an interim prioritization survey on the 35 uncertainties.
• Part 4: A multi-stakeholder consensus workshop finalized the top 10 research priorities.

Results

The top 10 research priorities identified were:

1. How can personalized medicine approaches (e.g. pharmacogenomics, minimal residual disease monitoring) improve outcomes for children undergoing HSCT?
2. What adjunct interventions (e.g. exercise, nutrition, microbiome-directed strategies) can improve immune recovery after paediatric HSCT?
3. What factors influence the development of chronic graft-versus-host disease (cGVHD) in children, and how can cGVHD be better prevented and managed?
4. How can we optimize the management of infections (viral, bacterial, fungal) in children undergoing HSCT to improve outcomes?
5. What are the long-term impacts of paediatric HSCT on growth, endocrine function, fertility, and quality of life, and how can these be mitigated?
6. How can we improve the effectiveness and safety of conditioning regimens prior to HSCT?
7. What are the optimal strategies for enhancing immune reconstitution and reducing complications after paediatric HSCT?
8. How can we improve the management of complications such as veno-occlusive disease, thrombotic microangiopathy, and sinusoidal obstruction syndrome in children undergoing HSCT?
9. What factors influence the risk of relapse after paediatric HSCT, and how can we better prevent and manage relapse?
10. How can we improve access to, and the equity of, paediatric HSCT globally?

Interpretation

This study provides an important consensus on key research priorities in paediatric HSCT from the perspective of patients, families, and healthcare providers across multiple countries. The priorities span areas such as personalized medicine, immune recovery, graft-versus-host disease, infections, long-term effects, and access to care.

Limitations & Uncertainties

• The study was limited to Australia, New Zealand, the Netherlands, and the United Kingdom, so the priorities may not fully represent global perspectives.
• It is unclear how the relative importance or impact of the priorities was assessed and weighted.
• The study did not provide details on how the specific priority areas were defined or the reasoning behind their selection.

What Comes Next

This set of research priorities can help guide future funding, study design, and collaboration in the field of paediatric HSCT. The next steps would be to develop specific research proposals and protocols to address these identified gaps and uncertainties.