Dr Laura Raiti

Dr Laura Raiti

Children’s Cancer CoLab-Maddie Riewoldt’s Vision Clinician-Researcher Fellow

Dr Laura Raiti is a paediatric oncologist and cancer genetics fellow with a strong interest in hereditary cancer, cancer predisposition syndromes and inherited bone marrow failure syndromes. She completed her paediatric oncology fellowship training at The Royal Children’s Hospital, Melbourne, and has undertaken further clinical and research training through her Cancer Genetics Fellowship at The Hospital for Sick Children (SickKids), Toronto.

Dr Raiti’s research focuses on germline cancer predisposition, including genetic factors that contribute to haematological malignancies and solid tumours in children and young people. She will return to Melbourne to undertake the Children’s Cancer CoLab and Maddie Riewoldt’s Vision Clinician-Researcher Fellowship at Peter MacCallum Cancer Centre, continuing to build expertise across paediatric oncology, cancer genetics and inherited bone marrow failure syndromes.

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Dr Laura Raiti's research

Optimising treatment of bone marrow failure in children through genomic discovery and analysis of long-term outcomes

The challenge

Bone Marrow Failure Syndromes (BMFS) are rare conditions in which the bone marrow does not produce enough red blood cells, white blood cells and/or platelets. This can leave children and young people vulnerable to serious infections, bleeding and other complications. Some children with BMFS have an inherited genetic condition, known as an Inherited BMFS. Importantly, some inherited BMFS are also cancer-predisposition syndromes, increasing the risk of blood cancers and, in some cases, solid tumours.

This creates an important intersection between bone marrow failure, cancer genetics and paediatric oncology. For children with suspected inherited BMFS, an accurate genetic diagnosis can guide treatment decisions, inform cancer surveillance and help clinicians plan long-term care. However, many children and young people with suspected inherited BMFS remain without a diagnosis despite extensive genetic testing. This may occur because current tests cannot identify every genetic change, or because some genes that cause inherited BMFS have not yet been discovered. There is also limited long-term evidence about cancer development, treatment complications and outcomes for children and young people with these conditions. This project will address these gaps to help improve diagnosis, treatment and long-term care.

The research

Dr Raiti’s research combines advanced genomic testing with long-term clinical outcomes research in children, adolescents and young adults with inherited BMFS.

Aim 1: Improving genetic diagnosis

The first part of the project will apply advanced genomic technologies to improve the ability to diagnose children and young people with suspected inherited BMFS who remain undiagnosed despite previous genetic testing. This includes more comprehensive genetic testing of patients and, where relevant, their families, as well as re-analysis of existing genetic sequencing data. As knowledge of disease-causing genetic changes continues to grow, reviewing previous results using updated technologies and evidence may identify diagnoses that were not possible at the time of the original testing.

The research will also investigate potential new genes associated with inherited BMFS. Identifying these genes will expand understanding of the genetic causes of bone marrow failure and help improve diagnosis for future patients. For children and young people with inherited Bone Marrow Failure Syndromes, a definitive genetic diagnosis can be particularly important where there is an increased risk of cancer. It can help clinical teams make more informed decisions about treatment, cancer surveillance and long-term care.

Aim 2: Understanding cancer and treatment outcome

The second part of the project will undertake a comprehensive review of data from the Australian Aplastic Anaemia and other Bone Marrow Failure Syndromes Registry for paediatric, adolescent and young adult patients with inherited BMFS. The research will examine long-term clinical outcomes, including cancer development, treatment choices, treatment-related complications and patient outcomes. It will also examine the outcomes of treatments used for bone marrow failure, including stem cell transplantation where relevant.

This work will help address an important evidence gap at the intersection of inherited BMFS and paediatric oncology. By better understanding which patients develop cancer, how they respond to treatment and which complications they experience, clinicians will be better placed to guide monitoring, treatment and long-term care.

Together, these approaches will help identify new genetic causes of inherited Bone Marrow Failure Syndromes, improve diagnostic pathways and generate stronger evidence to support personalised care for children and young people at risk of bone marrow failure and cancer

The impact

Approximately 5-million people are affected by BMFS worldwide each year, with 160 new diagnoses of BMFS in Australia each year, of which 50% do not survive long-term. Many are young and otherwise previously well. Expansion of the known genetic causes of BMFS and improved treatment outcomes will have a profound effect on this population.

For children and families living with suspected inherited BMFS, a genetic diagnosis can provide clarity at a difficult time. It can help clinicians select the most appropriate treatment approach, plan cancer surveillance and identify whether other family members may benefit from genetic assessment. By identifying new genetic causes of BMFS and examining long-term outcomes, this research aims to improve care for children and young people with conditions that sit at the intersection of bone marrow failure and paediatric cancer. The findings will also contribute to a stronger evidence base for clinicians caring for these patients in Australia and internationally.

Lived experience collaboration

People with lived experience of BMFS will be involved throughout the project. Dr Raiti will seek to continue this involvement through consumer representatives who were involved in the earlier IBMDx study, or another consumer representative, ensuring the research remains relevant to the experiences and priorities of patients and families. Research findings will be shared with the scientific community through conference presentations and publications, and with the BMFS community through accessible forums and partner networks, including Maddie Riewoldt’s Vision.

Children’s Cancer CoLab Funding Information

Fellowship Awarded: $250,000 (Jointly supported by Children's Cancer CoLab and Maddie Riewoldt's Vision)

Fellowship Timeline: February 2027 to January 2029

‍Impact Program: Future Leaders

Lead Institution: Peter MacCallum Cancer Centre

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