Bone Marrow Transplantation in Paediatrics PSP protocol

  • Published: 21 October 2024
  • Version: V1 - July 2024
  • 22 min read

Purpose of the PSP and background

 

The purpose of this protocol is to clearly set out the aims, objectives and commitments of the Bone Marrow Transplantation in Paediatrics Priority Setting Partnership (PSP) in line with James Lind Alliance (JLA) principles. The Protocol is a JLA requirement and will be published on the PSP’s page of the JLA website. The Steering Group will review the Protocol regularly and any updated version will be sent to the JLA. 

The JLA is a not-for-profit initiative, established in 2004. It brings patients, caregivers, researchers, and clinicians together in PSPs. These PSPs identify and prioritise the evidence uncertainties that have not yet been addressed by research, also known as ‘unanswered questions’, that they agree are the most important for research in their topic area.  Traditionally PSPs have focused on uncertainties about the effects of treatments, but some PSPs, including the Bone Marrow Transplantation in Paediatrics PSP have extended their scope beyond this. 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. This project is the first time that adolescents and young adults and children living with conditions requiring a bone marrow transplantation (BMT) for curative therapy, their caregivers, bone marrow transplant physicians, haematologists, oncologists, immunologists, intensivists, support personnel, researchers, patient engagement specialists and/or members of national organisations involved in conditions requiring BMT have been involved working in a partnership to determine the profession’s future research priorities. The National Institute for Health and Care Research (NIHR) coordinates the infrastructure of the JLA to oversee the processes for PSPs, based at the NIHR Coordinating Centre (NIHRCC), University of Southampton in the UK.    

The purpose of the Bone Marrow Transplantation [BMT] in Paediatrics PSP is to engage with key stakeholders (including adolescents and young adults and children living with conditions requiring a bone marrow transplantation for curative therapy, their caregivers, bone marrow transplant physicians, haematologists, oncologists, immunologists, intensivists, support personnel, researchers, patient engagement specialists and/or members of national cancer or immunodeficiency organisations) to identify the BMT research priorities in Australia and New Zealand, the United Kingdom and Netherlands for children and adolescents requiring a BMT. Funding for the Bone Marrow Transplantation in Paediatrics PSP is being provided by Victorian Paediatric Cancer Consortium [VPCC]. The Victorian Paediatric Cancer Consortium is a collaboration of academic, research and clinical organisations advancing research for patients with cancer or bone marrow transplantation. A steering group consisting of adolescents and young adults and children living with conditions requiring a bone marrow transplantation for curative therapy, their caregivers, bone marrow transplant physicians, haematologists, oncologists, immunologists, intensivists, support personnel, researchers, patient engagement specialists and/or members of national cancer or immunodeficiency. Participants were purposefully chosen to have different areas of expertise and levels of experience, work in various sectors, and come from various geographical locations. This steering group will meet virtually every month to discuss and agree on the aims, objectives, and commitments of the BMT in Paediatrics PSP.

Aims, objectives and scope of the PSP

The aim of the BMT in Paediatrics PSP is to identify the unanswered questions about BMT and its resultant complications from conditioning until Day 180 post BMT from the shared perspectives of children and adolescents and young adults who have received BMT, their caregivers, physicians and support personnel, researchers, patient engagement specialists and/or members of patient organisations, and then prioritise those unanswered questions that these groups agree are the most important for research to address. 

The objectives of the PSP are to:

  • work with parents/carers of children who have received a BMT, or adolescents or young adults who have received a BMT, physicians and support personnel, researchers, patient engagement specialists and/or members of patient organisations
  • to agree by consensus on a prioritised list of those uncertainties for future research
  • to publicise the results of the PSP
  • to take the results to research commissioning bodies to be considered for funding.

The focus of the BMT in paediatrics PSP is on practice-based BMT therapy and the complications arising from BMT therapy up until six months (Day 180) post transplant. BMT is a curative life-saving therapy for many patients with high-risk or relapsed leukaemia, bone marrow failure disorders, primary immunodeficiencies or inborn errors of metabolism. It involves intensive myelosuppressive chemotherapy, radiotherapy and/or serotherapy. Morbidity and mortality after BMT are commonly secondary to graft versus host disease or diseases of endothelial injury [sinusoidal obstructive syndrome, TMA etc]. BMT is indicated across a broad range of conditions, all these conditions are within the scope of this project. The scope of the BMT in paediatrics Priority Setting Partnership will encompass:

  • perspectives gathered from across Australia and New Zealand, United Kingdom and Netherlands in English;
  • needs and perspectives of adolescents and young adults [13-25 years] who have received a BMT as a result of their condition, and their caregivers
  • caregivers of children [<13] who have received a BMT as a result of their condition;
  • perspectives reflective of the range of practice-based roles contributing to the delivery of BMT services to children, adolescents and young adults who require BMT to treat their condition such as BMT physicians, haematologists, oncologists, immunologists, pharmacists, allied health professionals and members of patient advocacy organisations.

The PSP will exclude from its scope questions about:

  • BMT practice outside Australia and New Zealand, Netherlands and United Kingdom;
  • services with significant competing or commercial interests, such as pharmaceutical companies
  • adult BMT practice, including literature

The Steering Group will discuss the implications the PSP scope will have on the evidence-checking stage of the process. Resources and expertise will be put in place to do this evidence checking. 

The Steering Group

The Steering Group includes membership of adolescents and young adults living with conditions requiring a bone marrow transplantation for curative therapy, caregivers, bone marrow transplant physicians [either haematologists or immunologists], intensivists, support personnel, researchers, patient engagement specialists and/or members of national/international cancer or immunodeficiency organisations

The BMT in paediatrics PSP will be led and managed by a Steering Group involving:

A/Prof Rachel Conyers is a paediatric oncologist and BMT physician, a clinician scientist fellow through Murdoch Children’s Research Institute and Associate Professor through University of Melbourne. Her research includes toxicities of therapy and pharmacogenomics. This is the co-chair of the Zero Childhood Cancer Program Pharmacogenomics group and Principal Investigator for cardiotoxicity in the Ponte De Legno European Working Group.

A/Prof Theresa Cole is a BMT physician, immunologist and Associate Professor through University of Melbourne. Her research explores immunology and bone marrow transplantation. She is the current president of the Australasian Society of Clinical Immunology and Allergy [ASCIA] and facilitator of the Transplantation in Primary Immunodeficiency Group [TAPID]. 

Dr Katherine Colman is an advanced trainee in paediatric oncology with a clinical interest in bone marrow transplantation. She has been awarded a prestigious Melbourne Academic Centre for Health Track-PhD program to commence in 2024.

Ms Roxanne Dyas is a pharmacist with over 20 years of clinical paediatric pharmacy experience including 20 years in paediatric oncology and bone marrow transplant (BMT) pharmacy. She has helped to develop care therapy plans and guidelines relating to conditioning and supportive care in the BMT setting.  She is currently works with Murdoch Children’s Research Institute and The Royal Children’s Hospital studying the toxicities of therapies.

Professor Trevor Duke is a paediatrician and intensive care physician, clinical head of the General Intensive Care Unit at The Royal Children’s Hospital, Melbourne and a Professor at the University of Melbourne Department of Paediatrics.

Nader Eloshaiker is a parent of teenage girl who was diagnosed with pre-cursor B-ALL as a pre-teen. Unfortunately, his daughter was refractory to chemotherapy, she underwent CAR-T Cell immunotherapy followed by an allogeneic BMT. Nader is an active representative on the Victorian Paediatric Cancer Consortium Patient and Family Advisory Committee.

Dr Ben Gelbart is a paediatric intensive care specialist at the Royal Children’s Hospital Melbourne, clinician scientist fellow at the Murdoch Children’s Research Institute and honorary fellow of the Department of Paediatrics, University of Melbourne. Ben recently completed a PhD, which focused on quantifying oedema in critically ill children. He has led and collaborated on multicentre randomised trials within the Australian and New Zealand Intensive Care Society Paediatric Study Group.

Professor Andrew Gennery is a Professor in Paediatric Immunology and Haematopoetic stem cell transplantation and the Sir James Spence Professor of Child Health at Newcastle University, United Kingdom. He is an Honorary Consultant in Paediatric Immunology and Haematopoeitc stem cell transplantation at The Great North Children’s Hospital, Newcastle. Professor Gennery is a member of European Bone Marrow Transplantation [EBMT], European Society for Immunodeficiencies [ESID] and is the previous chair of the ESID/EBMT Inborn Errors Working party and ESID Clinical Working Party.

A/Prof Amanda Gwee is a paediatrician, infectious disease physician and pharmacologist. She is the group leader of the Antimicrobials Group at the Murdoch Children’s Research Institute. A/Prof Gwee research focuses on optimising antimicrobial use using international clinical trials, pharmacokinetic/pharmacodynamic studies. 

Annette Hill is an Advanced Nurse Practitioner and has worked in paediatric BMT for over 20 years. Annette is vice chair of the UK BMT national nursing group. Annette is involved with research projects including HLA typing and donor selection.   

Professor Arjan Lankester is Professor in Paediatrics and Paediatric Stem Cell Transplantation at Leiden University Medical Centre. He is also head of the JACIE accredited paediatric stem cell transplantation program. His primary research interest is to improve efficacy and safety of stem cell therapy with particular focus onoptimising conditioning regimens and immune reconstitution after BMT. He is PI of the ongoing RECOMB trial on lentiviral stem cell gene therapy in RAG1severe combined immunodeficiency. He is co-founder of the AGORA foundation (Access to Gene therapies fOr RAre disease), and the past chair of Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation and European Society for Immune Deficiencies.

Dr Lorna McLeman is a paediatric oncologist and Bone Marrow Transplant physician at The Royal Children’s Hospital, Melbourne. Dr McLeman is undertaking a PhD at St Vincent’s Research Institute and Murdoch Children’s Research Institute pursing prime editing as a mechanism for gene therapy in Fanconi Anaemia. Furthermore, Dr McLeman is leading an inquiry as part of the PREPARE ReNEW Stem Cell Medicine Consortium investigating consumer readiness for gene therapy in Australia.

Dr Adam Nelsen is a staff specialist in Bone Marrow Transplant and Oncology at the Kids Cancer Centre, Sydney Children’s Hospital. Dr Nelsen has trained broadly including 7 years at Cincinnati Children’s Hospital, a world leader in bone marrow failure and cellular therapy. Dr Nelsen is the current chair of the Australian National Transplant group [TACTIC]. 

Dr Timothy Prestidge is a transplant haematologist at the Blood and Cancer Center, Starship Children’s Hospital and chair of the Blood and Marrow Transplant Advisory Committee, Auckland City Hospital. He is a Hematopathologist at LabPlus Auckland City Hospital and a member of the NZ Ministry of Health transplant and cellular therapy special interest working group. Other transplant related activities include development of the haploidentical transplantation program, Cooperative development a coordinated clinical trial approach for Paediatric/AYA patients within New Zealand and Introduction of the Simulation Program for Crisis Resource Management. Ongoing research interests in transplantation beyond malignancy including autoimmune and other non-malignant conditions, as well and bone marrow failure syndromes.

Dr Kanchan Rao is an experienced BMT physician having worked in the BMT department at Great Ormond Street Hospital (GOSH), United Kingdom since 1999. Dr Rao is the Chair of the United Kingdom and Ireland BMT group and chairs monthly meetings discussing complicated cases nationally and internationally. Dr Rao research areas include Hemophagocytic lymphohistiocytosis and viral complications following BMT. 

Dr. Lori Chait-Rubinek is a Monash and Notre Dame University, Melbourne, graduate who commenced her paediatric training at The Royal Children's Hospital, Melbourne in 2018. Lori completed her first-class honours thesis in the late effects of malignancy treatments in the paediatric and young adult population. Little did she know how real life would mimic this research when her daughter was diagnosed with cancer. The journey from a doctor with an interest in oncology to a parent of a child with an oncological diagnosis has been traumatic and enlightening, driving Lori to use her experiences to improve outcomes of patients and families living with childhood cancer.  

Professor Mary Slatter is a consultant paediatrician in haematopoetic stem cell transplantation at the Great North Children’s Hospital in Newcastle, United Kingdom who specialises in transplant for Inborn Errors of immunity and related diseases. She is also an Honorary Professor at Newcastle University. Professor Slatter is currently Newcastle’s Paediatric Haematopoetic Stem Cell Transplant Clinical Program Director. 

Ms Beth Williams is a pharmacist with 10 years experience in oncology pharmacy. With a broad range of previous clinical experience, Beth is currently working at the Murdoch Children’s Research Institute as an academic pharmacist assessing the use of pharmacogenomic screening in paediatric oncology patients receiving polypharmacy, while working towards improving current pharmacogenetic guidelines and toxicity outcomes.

Dr Shivanthan Shanthikumar is a paediatric respiratory specialist and clinician-scientist fellow on the Melbourne Children's Campus. His clinical areas of expertise include cystic fibrosis, asthma, and respiratory complications of childhood cancer. His research spans laboratory profiling using novel single cell techniques, to clinical trials and evaluation of new models of care, all with the aim of facilitating a precision medicine approach to paediatric respiratory disease.

Lisa Ott de Bruin is a paediatrician and fellow of paediatric infectious diseases and immunology, Leiden University Medical Centre

Dr Amy Lovell is a senior lecturer at the University of Auckland and paediatric oncology dietitian at the Starship Blood and Cancer Centre in Auckland, the largest primary treatment centre for children with cancer in Aotearoa, New Zealand. Reflecting her dual clinical-research roles, Dr Lovell’s research focuses on applying evidence-based nutrition models and methods to address the nutritional implications of a cancer diagnosis and its treatment for children and young people with cancer. Her research spans the cancer trajectory, from diagnosis to survivorship, and as an early career researcher, has begun to disseminate her research through peer reviewed publications. A strong emphasis of Dr Lovell’s research program is understanding the patient [and caregiver] cancer experience with nutrition [and lifestyle], with the goal of improving nutrition-related resources and interventions for patients, families/whānau, and health professionals

Vanessa Clifford works clinically (across infectious diseases, infection control and microbiology) at the Royal Children’s Hospital and the Women’s hospital and is also Medical Lead for the donor human milk program at Australian Red Cross Lifeblood, a senior research Fellow at the University of Melbourne and a clinician scientist fellow at Murdoch Children’s Research Institute. She completed a PhD on novel diagnostic tests for tuberculosis in 2018. Her diverse interests include infectious diseases in the immunocompromised, donor human milk research, sustainability, and antimicrobial stewardship.

Sister Karen Nicholson is a nurse practitioner and lead of the Children and Young People’s Stem Cell Transplant Service for Immunology at the Great North Children’s Hospital. Karen has worked in the service for 5 years with previous experience in paediatric oncology.

Aleksandra Pomper is the parent of a child who has needed two BMTs. 

Prof Robert Wynn is a paediatric transplanter in Manchester, UK, specialist in transplant for metabolic disorders

Lucy Staite and Neil Timmins are parents to Evie Timmins. Evie had her stem cell transplant at Great North Children's Hospital on October 2nd 2020. Evie is now 15 years old.

Abbie Young was diagnosed with Aplastic Anaemia at the start of 2020 following blood tests which required a bone marrow transplant and an extended stay in The Great North Children’s hospital in Newcastle. Abbie underwent chemotherapy and radiotherapy, received two bone marrow transplants and then spent eighteen months in isolation at home during Covid, to allow her immune system to recover. Abbie’s prolonged period in hospital has given her first-hand experience of front-line medical treatment.  

Hilda Mekelenkamp works as a specialised nurse in paediatric stem cell transplantation at the Leiden University Medical Centre in the Netherlands. She is also a postdoc researcher at the Department of Medical Ethics & Health Law. Her research focusses on stakeholder perspectives about the selection, decision-making, and consent process regarding hematopoietic stem cell transplantation and gene therapy. 

Kavitha Asokan graduated from Queen Mary University with a degree in biochemistry and microbiology. After working in the financial industry, Kavitha decided to dedicate her time to her children. Kavitha’s youngest daughter, Esha, was diagnosed with acute myeloid leukaemia at the age of 4. After unsuccessful chemotherapy she underwent a bone marrow transplant at Great Ormond Street Hospital.

Rish Nadeswaran is a chartered accountant and director in banking and financial services with over 25 years of experience. Rish is married with two daughters and resides in Redbridge, Greater London. Rish’s youngest daughter, Esha, was diagnosed with acute myeloid leukaemia (AML) at the age of 4. After unsuccessful chemotherapy she underwent a bone marrow transplant at Great Ormond Street Hospital.

Ms Eileen van der Stoep, PhD, hospital pharmacist with specific expertise in pharmacokinetics and ATMPs.

Suzannah Kinsella is a James Lind Alliance Adviser. As a facilitator and researcher, Suzannah has involved the public and stakeholders in health and social care policy and service discussions. She has worked with complex and sensitive issues such as genetic technologies, organ donation, changes to health services and data security. The discussions she designs help to generate constructive and insightful conversations that lead to better outcomes. She has facilitated and chaired a wide range of engagement sessions. During her career, Suzannah has been Head of Engagement at a social research agency and held the same position at the Cabinet Office agency the Central Office of Information (COI). Suzannah joined the JLA team as an adviser in April 2019.

The Steering Group will agree on the resources, including time and expertise that they will be able to contribute to each stage of the process, with input and advice from the JLA advisor. 

Partners

Organisations and individuals will be invited to be involved with the PSP as partners, particularly those organisations which can reach and advocate for the key groups involved in the PSP. Partners are organisations or groups who will commit to supporting the PSP, promoting the process and encouraging their represented groups or members to participate. Partners will represent the following groups:

  • adolescents and young adults who have received a BMT for inborn errors of metabolism, malignancy, bone marrow failure or primary immunodeficiency
  • caregivers of children, adolescents and young adults who have received a BMT for inborn errors of metabolism, malignancy, bone marrow failure or primary immunodeficiency
  • haematologists, oncologists, intensivists and immunologists with experience in bone marrow transplantation
  • pharmacists, dietitians, nurses, microbiologists and psychologists with expertise in BMT

Our current partners include: 

We are currently inviting individuals and organisations to be involved as partners to promote the BMT in Paediatrics PSP and encourage represented groups or members to participate. The goal is to help identify the BMT research priorities in Australia and New Zealand for patients who have received BMT and their carers/families. If you would like to become a partner, please contact the research coordinator.

Exclusion criteria

Some organisations may be judged by the JLA or the Steering Group to have conflicts of interest. These may be perceived to potentially cause unacceptable bias to the PSP process. As this is likely to affect the ultimate findings of the PSP, those organisations will not be invited to participate. It is possible, however, that interested parties may participate in a purely observational capacity when the Steering Group considers it may be helpful.

The methods the PSP will use

This section describes a schedule of proposed steps through which the PSP aims to meet its objectives. The process is iterative and dependent on the active participation and contribution of different groups. The methods used in any step will be agreed through consultation between the Steering Group members, guided by the PSP’s aims and objectives. More details of the method are in the Guidebook section of the JLA website where examples of the work of other JLA PSPs can be seen. 

Step 1: Identification and invitation of potential partners

Potential partner organisations will be identified through a process of peer knowledge and consultation, through the Steering Group members’ networks. Potential partners will be contacted and informed of the establishment and aims of the BMT in paediatrics PSP.

Step 2: Awareness raising 

Steering Group members of the BMT in Paediatrics PSP will need to raise awareness of their proposed activity among children, adolescents and young adults who have received a BMT, their caregivers, bone marrow transplant physicians, immunologists, intensivists, haematologists, pharmacists, and support personnel, researchers, patient engagement specialists and/or members of organisations for patients receiving BMT across different diseases, in order to secure support and participation. Depending on budget, this may be done by a face-to-face meeting, or there may be other ways in which the process can be launched, e.g., via social media. It may be carried out as part of steps 1 and/or 3. The Steering Group should advise on when to do this. Awareness raising has several key objectives:

  • To present the proposed plan for the PSP
  • To generate support for the process
  • To encourage participation in the process
  • To initiate discussion, answer questions and address concerns.

Step 3: Identifying evidence uncertainties

The BMT in paediatrics PSP will carry out a consultation to gather uncertainties from children, adolescents and young adults who have received a BMT, their caregivers, bone marrow transplant physicians, immunologists, intensivists, haematologists, pharmacists, and support personnel, researchers, patient engagement specialists and/or members of organisations for patients receiving BMT across different diseases. A period of 2 months will be given to complete this exercise (which may be revised by the Steering Group if required). 

The method of consultation must be transparent, inclusive and representative. The Steering Group must try to reach as representative a range of participants as feasible. Methods may include membership meetings, web-based questionnaires, or internet message boards.

Existing sources of evidence uncertainties may also be searched. This evidence can include research recommendations identified in scoping and other systematically conducted literature reviews, research reports/literature, clinical guidelines, protocols for systematic and scoping reviews being prepared, and registers of ongoing research in Australia and New Zealand.

Step 4: Refining questions and uncertainties

The consultation process will occur in English, will produce ‘unedited’ questions and comments indicating the areas of uncertainty from the perspectives of children, adolescents and young adults who have received a BMT, their caregivers, bone marrow transplant physicians, immunologists, intensivists, haematologists, pharmacists, and support personnel, researchers, patient engagement specialists and/or members of organisations for patients receiving BMT across different diseases. To clarify the precise uncertainty, which may have been submitted with a lot of narrative text, these unedited questions will be categorised and refined by the Information Specialist into summary questions that capture the meaning of the original submission. These summary questions are clear, addressable by research, and understandable to all. Similar or duplicate questions will be combined where appropriate. Submissions that can be answered by research, known as ‘answered’ submissions, and those that are out-of-scope will be compiled separately. The Steering Group will have oversight of this process to ensure that the raw data is being interpreted appropriately and that the summary questions are being worded in a way that is understandable to all audiences. The JLA Adviser will observe to ensure accountability and transparency. 

This will result in a long list of in-scope summary questions. These are not research questions, as to try to word them as such may make them too technical for a non-research audience. The summary questions will instead be framed as researchable questions that capture the themes and topics that have been suggested. 

Systematic reviews, guidelines and large randomised controlled trials (RCT) will be identified and verified by the PSP Information Specialist to see to what extent these refined questions have, or have not, been answered by previous research. The PSP will complete the JLA Question Verification Form, which clearly describes the process used to verify whether the question has already been answered by research, before starting prioritisation. The Question Verification Form includes details of the types and sources of evidence used to verify uncertainty. The Question Verification Form will be published on the JLA website as soon as it has been agreed to enable researchers and other stakeholders to understand how the PSP has decided that its questions are unanswered, and any limitations of this process.

Sometimes, uncertainties are expressed that can in fact be resolved with reference to existing research evidence - i.e. they are "unrecognised knowns" and not unanswered questions. For example, if a question about interventions can be answered with existing information but this is not known, it suggests that information is not being communicated effectively to those who need it. Accordingly, the PSP will decide whether to keep the question as an unanswered question. 

Questions that are not adequately addressed by previous research will be collated and recorded on a standard JLA template by the PSP Information Specialist. This will show the checking undertaken to make sure that the uncertainties have not already been answered. The data should be submitted to the JLA for publication on its website on completion of the priority setting exercise, taking into account any changes made at the final workshop, in order to ensure that PSP results are publicly available. 

The Steering Group will also consider how it will deal with submitted questions that have been answered, and questions that are out of scope.

Step 5: Prioritisation – interim and final stages 

The aim of the final stage of the priority setting process is to prioritise through consensus the identified uncertainties about BMT in paediatrics PSP. This will involve input from children, adolescents and young adults who have received a BMT, their caregivers, bone marrow transplant physicians, immunologists, intensivists, haematologists, pharmacists, and support personnel, researchers, patient engagement specialists and/or members of organisations for patients receiving BMT across different diseases. We will encourage engagement from as wide a range of people as possible, including those who did and did not contribute to the first consultation. There are usually two stages of prioritisation:

  1. Interim prioritisation is the stage where the long list of questions is reduced to a shorter list that can be taken to the final priority setting workshop. This stage is aimed at a wide audience and will involve an on-line survey in English. With the JLA’s guidance, the Steering Group will need to consider how best to reach and engage a wide range of children, adolescents and young adults who have received a BMT, their caregivers, bone marrow transplant physicians, immunologists, intensivists, haematologists, pharmacists, and support personnel, researchers, patient engagement specialists and/or members of organisations for patients receiving BMT across different diseases in the process. The most highly ranked questions (approximately 25) will be taken to a final priority setting workshop. In the event that the interim prioritisation does not produce a clear ranking or cut off point, the Steering Group will decide which questions are taken forwards to the final prioritisation.
     
  2. The final priority setting stage is a virtual workshop over two half-days facilitated by the JLA. The workshop will take place in English. With guidance from the JLA and input from the Steering Group, up to 30 adolescents and young adults who have received a BMT, or their caregivers, bone marrow transplant physicians, immunologists, intensivists, haematologists, pharmacists, and support personnel, researchers, patient engagement specialists and/or members of organisations for children or young adults and adolescents who have received a BMT will be recruited to participate in a day of discussion and ranking, to determine the top 10 questions for research. All participants will declare their interests. The Steering Group will advise on any adaptations needed to ensure that the process is inclusive and accessible. 

Dissemination of results

The Steering Group will identify audiences with which it wants to engage when disseminating the results of the priority setting process, such as researchers, funders of research and the patient and clinical communities. Dissemination at the end of the PSP should be a consideration throughout the PSP process to maximise its success. They will need to determine how best to communicate the results and who will take responsibility for this step. Previous PSPs’ outputs have included academic papers, lay reports, infographics, conference presentations and videos for social media. Results will be available in English.

It should be noted that the priorities are not worded as research questions. The Steering Group will need to discuss how they will work with researchers and funders to establish how to address the priorities and to clarify the research questions that will address the issues that people have prioritised. The dissemination of the results of the PSP will be led by A/Prof Rachel Conyers, Ms Roxanne Dyas and Ms Beth Williams. 

The PSP will report back to the JLA about any activities that have come about because of the PSP, including funded research, by sending any details to the JLA Coordinating team.

Agreement of the Steering Group

The BMT in paediatrics PSP Steering Group agreed the content and direction of this Protocol on 05/03/2024.