Paediatric haematopoietic stem cell transplantation research priorities: Results from an international multi‐stakeholder priority setting partnership

Co-designed research in paediatric HSCT is limited. We sought to determine research priorities which represent the shared priorities of patients, parents, carers and healthcare professionals (HCP) within Australia, New Zealand, the Netherlands and the United Kingdom. An international, multiphase priority-setting methodology was implemented in partnership with the James Lind Alliance and delivered over an 18-month period. Part 1: an international scoping survey asked respondents to submit their research uncertainties related to paediatric HSCT. Part 2: summarising and evidence-checking the submitted uncertainties. Part 3: interim prioritisation survey. Part 4: consensus workshop. In the first international scoping survey, 667 topic ideas were suggested (45% by consumers, 55% by HCP), which were categorised into 80 summary questions. After systematic literature review, 35 summary questions were judged to be true uncertainties (i.e. not answered by existing evidence). These 35 uncertainties were included in a second interim prioritisation survey and completed by 224 participants. From those, a shortlist of 19 questions was drawn. After a multistakeholder workshop, consensus was reached on the top 10 priorities. The PSP identified important research gaps in the management of paediatric HSCT. Priority areas included: implementing personalised medicine approaches, improving immune recovery and adjunct interventions such as exercise, nutrition and microbiome-directed strategies.

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Publication Details

Journal
British Journal of Haematology
Published
2026-10-16
DOI
https://doi.org/10.1111/bjh.70901
Primary Topic
Hematopoietic Stem Cell Transplantation
Type
article
Field-Weighted Citation Impact
0.00

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article

Paediatric haematopoietic stem cell transplantation research priorities: Results from an international multi‐stakeholder priority setting partnership

Tim D. Prestidge, M. Y. Eileen C. van der Stoep, Vanessa Clifford, Andrew Richard Gennery et al.
British Journal of Haematology
Hematopoietic Stem Cell Transplantation
article

Paediatric haematopoietic stem cell transplantation research priorities: Results from an international multi‐stakeholder priority setting partnership

Tim D. Prestidge, M. Y. Eileen C. van der Stoep, Vanessa Clifford, Andrew Richard Gennery, Katherine S. Colman, Theresa S. Cole, Suzannah Kinsella, Hilda Mekelenkamp, Amanda Gwee, Gabrielle M. Haeusler, Arjan C. Lankester, Lorna McLeman, Ben Gelbart, Shivanthan Shanthikumar, Mary Anne Slatter, Rachel K. Conyers, Adam S. Nelson, Amy Luisa Lovell, Roxanne Dyas, Elizabeth Williams, Lisa M. Ott de Bruin, Lori Chait-Rubinek, Kanchan Rao, Karen Nicholson
article en

Abstract

Co-designed research in paediatric HSCT is limited. We sought to determine research priorities which represent the shared priorities of patients, parents, carers and healthcare professionals (HCP) within Australia, New Zealand, the Netherlands and the United Kingdom. An international, multiphase priority-setting methodology was implemented in partnership with the James Lind Alliance and delivered over an 18-month period. Part 1: an international scoping survey asked respondents to submit their research uncertainties related to paediatric HSCT. Part 2: summarising and evidence-checking the submitted uncertainties. Part 3: interim prioritisation survey. Part 4: consensus workshop. In the first international scoping survey, 667 topic ideas were suggested (45% by consumers, 55% by HCP), which were categorised into 80 summary questions. After systematic literature review, 35 summary questions were judged to be true uncertainties (i.e. not answered by existing evidence). These 35 uncertainties were included in a second interim prioritisation survey and completed by 224 participants. From those, a shortlist of 19 questions was drawn. After a multistakeholder workshop, consensus was reached on the top 10 priorities. The PSP identified important research gaps in the management of paediatric HSCT. Priority areas included: implementing personalised medicine approaches, improving immune recovery and adjunct interventions such as exercise, nutrition and microbiome-directed strategies.

British Journal of Haematology
Leiden University (NL), Royal Children's Hospital (AU), University of Auckland (NZ), The University of Melbourne (AU), Leiden University Medical Center (NL), Great Ormond Street Hospital (GB), Great Ormond Street Hospital for Children NHS Foundation Trust (GB), Peter MacCallum Cancer Centre (AU), UNSW Sydney (AU), Centre for Medical Systems Biology (NL), Sydney Children's Hospital (AU), Willem-Alexander Kinderziekenhuis (NL), NIHR Evaluation Trials and Studies Coordinating Centre (GB), Great North Children's Hospital (GB), Starship Children's Health (NZ), Murdoch Children's Research Institute (AU), Newcastle University (GB)
Medical Research Future Fund
Good health and well-being
Openalex Percentile: Top 13%
Hematopoietic Stem Cell Transplantation
DOIOpen Access PDFOpenAlex
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