Paediatric haematopoietic stem cell transplantation research priorities: Results from an international multi‐stakeholder priority setting partnership
Abstract
Summary 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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Authors: Elizabeth Williams, Roxanne Dyas, Katherine S. Colman, Suzannah Kinsella, Amanda Gwee, Amy Luisa Lovell, Andrew Richard Gennery, Mary Anne Slatter, Lori Chait-Rubinek, M. Y. Eileen C. van der Stoep, Arjan C. Lankester, Hilda Mekelenkamp, Ben Gelbart, Karen Nicholson, Lorna McLeman, Shivanthan Shanthikumar, Vanessa Clifford, Theresa S. Cole, Gabrielle M. Haeusler, Lisa M. Ott de Bruin, Tim D. Prestidge, Adam S. Nelson, Kanchan Rao, Rachel K. Conyers
Institutions: Peter MacCallum Cancer Centre, Leiden University Medical Center, The University of Melbourne, UNSW Sydney, Royal Children's Hospital, Great Ormond Street Hospital, Newcastle University, Murdoch Children's Research Institute, University of Auckland, Leiden University, Sydney Children's Hospital, Great Ormond Street Hospital for Children NHS Foundation Trust, Willem-Alexander Kinderziekenhuis, NIHR Evaluation Trials and Studies Coordinating Centre, Great North Children's Hospital, Starship Children's Health, Centre for Medical Systems Biology