Monday June 19, 2023
Retinoblastoma is a rare cancer, posing major challenges to researchers everywhere. In part 1/2 of this article, Rb survivor and WE C Hope CEO, Abby White, explores the many ways multicentre research collaboration can overcome these challenges and change patient care and outcomes – for the child, survivor, family and professional.
For children with retinoblastoma and survivors navigating its lifelong consequences, deciding the best screening, treatment. Follow-Up, and supportive care can be the difference between life and death, saving some sight or none, good quality of life or long-lasting deep trauma. Research gives us all the evidence to decide what is best.
Patient involvement in research is now an almost universal prerequisite for funding and Human Research Ethics Committees (HRECs). Parents and survivors within the retinoblastoma community are becoming more involved in guiding research questions and design, and other areas of research advocacy.
As we do, it becomes more important that we understand the research process, and the things that help the best research to thrive. We can then make the most informed choices about how we engage with and support the research needed to improve care for ourselves, our loved ones, and future children with eye cancer.
This two-part blog explores multicentre collaboration for retinoblastoma research.
Part 1 (below) examines why it is needed, what it is, 12 ways it improves research, and 8 key areas in which it can positively change patient care.
Part 2, Multicentre Research Collaboration: the Challenge and the Light, focuses on the common challenges of multicentre research, with solutions for each, and the main ways we can encourage collaboration.
Together, Everyone Achieves More
Medical research is the systematic gathering of information about a specific condition. The data can then be analysed and reviewed to better understand the condition itself, how it affects people, and treatment responses, effects, and outcomes. This ongoing research process helps identify the safest and most effective care, including diagnosis, treatment, and patient support.
Retinoblastoma is the most common eye cancer of infancy and early childhood, but it is rare among cancers. This rarity poses major challenges to researchers everywhere.
Globally, there is uneven distribution of children with retinoblastoma and professionals equipped to treat and study the cancer. At well-resourced research centres scattered throughout the world, patients and survivors are treated and followed in small numbers.
In contrast, teaching hospitals with limited resources in developing countries are overwhelmed with high patient numbers. Many of those children have advanced cancer due to late diagnosis, and most families struggle to access treatment for complex socioeconomic reasons.
These imbalances make it difficult to save most children’s lives and conduct the high-quality research we need to develop safe, effective care for everyone.
Retinoblastoma also presents in diverse ways – children may have one or more tumours in one or both eyes, each needing different treatment and follow-up. Tailoring care to the child is key in retinoblastoma, but we need research evidence to guide and inform these critical clinical care decisions.
Chemotherapy is recognized globally as the best eye-salvage therapy. Systemic chemotherapy is also the best life-protecting or life-saving treatment when cancer may spread beyond the eye, or has already spread.
However, a range of delivery methods are used, and there are no internationally agreed-to regimens for treatment in any form. Elements of care may also differ between centres around the world, from dosage and timing of treatment to number of cycles, supportive medications, and concurrent therapies. Since different regimens are being used on a small number of children, one cannot accurately compare their outcomes.
Our understanding of retinoblastoma and advances in its treatment move fastest when healthcare professionals, scientists, and patient advocates (parents, survivors, and others) work together with the shared goal of improving patient care. When two or more treatment centres, research teams, and organizations work together on a research project, this is called multicentre collaborative research.
Collaboration plays a key role in research and management of rare cancers like retinoblastoma. Pooling expertise, resources, and patient data generates a broad perspective to inform patient care, and more robust study results that help develop reliable, safe therapies and patient-focused approaches to care.
Multicentre collaborative research can also lay the foundations of knowledge and partnership needed to develop new treatment programs in under-served areas – both in resource-rich and resource-limited countries.
Below, we look in more detail at the main reasons we need multicentre collaboration, how collaboration can benefit retinoblastoma research, and its impacts for patients, families, survivors, and medical professionals. In the second part of our deep-dive, we share the main challenges to multicentre collaboration, ways to overcome them, and how our community can encourage more collaboration.
Some of the participants at One Rb World 2017 in Washington D.C. – events like One Rb World nurture strong connections, important discussions, and patient focused research priorities.
Benefits of Research-Focused Collaborations
Multicentre research collaborations unite healthcare professionals, scientists, and patient advocates from different institutions, and often multiple countries. They offer many advantages over single-centre research, hastening our understanding of retinoblastoma, developing effective care faster, and guiding the best outcomes for children, adult survivors, and our families.
Because retinoblastoma is rare, research funding is limited. Working together maximises efficient use of research infrastructure, equipment, people, and funds. Collaborating centres can coordinate to avoid duplicating efforts, take on larger-scale projects and access resources that might not otherwise be available.
Combining data from multiple centres overcomes many challenges of rarity. This helps to identify patterns and trends early, and supporting the high-level evidence doctors, parents and survivors need to make the best medical care decisions.
Using a shared database specifically designed to handle the unique features of retinoblastoma (two eyes, multiple tumours, different treatments for each), will be a major step forward for multicentre collaboration. A shared secure database is the most practical way to manage research data, with the most positive impact for patient families and survivors.
Researchers can study retinoblastoma in greatest detail when collaborating centres are spread across geographic regions, or throughout the world. A broad perspective can highlight variations in incidence, risk factors, genetic profiles, diagnostic age and stage, treatment pathways and tumour response, family needs, supports and coping, and patient outcomes.
Identifying trends early is vital to meet the emerging need quickly. Understanding regional differences is crucial to tailor programs that meet the real needs of diverse populations.
Retinoblastoma varies in presentation, progression, and treatment response. That wide variation occurs among small patient numbers at individual treatment centres. By collaborating across centres, researchers can recruit more patients with a more similar or diverse range of experiences, depending on the study’s focus. They can create more comprehensive studies that generate more accurate results, and produce stronger evidence to guide patient care.
Defining the best care for any disease requires ongoing review of practices and response to treatment options. This process is called evidence-based research and involves people receiving care as part of a research study observing their treatment response, recovery or outcomes.
Clinical Trials study patients with exactly the same condition. The gold standard of research is to treat several groups or “arms” of the study population in different ways for comparison, such as comparing a new treatment and standard therapy, or different doses of a standard therapy.
Multicentre Clinical Trials allow researchers to work together to evaluate treatments, medical procedures, and supportive care approaches in a larger patient population. Due to its rarity, multicentre collaborations are particularly important for quality clinical studies in retinoblastoma.
Longitudinal Studies assess long-term patient outcomes over extended periods. Multicentre collaborations allow researchers to track progress in a greater number and broader range of patients and survivors. This generates more comprehensive, accurate findings about the effectiveness and outcomes of different treatments, and impacts of the cancer itself, over time. These insights can guide both retinoblastoma patient and survivor care, and further research in both areas.
By pooling resources, collaborators can conduct larger scale pre-clinical and genetic studies. This research helps increase our understanding of how retinoblastoma behaves, and identifies new ways to manage the cancer. Access to a broader range of patient data and tissue samples also increases the strength of evidence each study can produce, and the positive impact that study can have on patient care.
Collaboration motivates the centres to develop and implement consistent procedures in clinical care and research to minimize variability across their partnered sites. Consistency is vital to compare data, produce high-quality results, and draw meaningful conclusions that clinicians and parents can rely on when making medical care decisions.
This lies at the heart of gold standard clinical trials; investigating the same specified treatment and supportive care protocol across all involved centres.
Retinoblastoma research requires a multidisciplinary approach involving ophthalmology, oncology, genetics, pathology, nursing, child life, psychology, parent and survivor representatives, and others. Collaborating across centres enables researchers to tap into the collective knowledge and expertise of stakeholders with diverse backgrounds and experiences. Multicentre interdisciplinary collaborations foster the greatest innovation and advances in treatment and patient-focused care.
Researchers with different backgrounds and perspectives can review and critique each other’s work, creating a more thorough and nuanced understanding of the study’s findings. This collaborative internal review process improves the quality and validity of the final research paper. The group has more opportunity to identify and address potential biases than a single-centre study team, and to ensure comprehensive analysis before sending to publication.
Ethnographic research provides a comprehensive understanding of the cultural, social, and contextual factors that influence patient, survivor, and family experience of retinoblastoma, clinical care and outcomes. The knowledge and insight helps shape more relevant, effective, inclusive medical care and support. But this type of human research is complex, time consuming, and often hard to conduct in the rare landscape of retinoblastoma.
As well as many of the benefits described above, multicentre collaborations bring some unique opportunities to ethnographic research. Working together, we may learn more from the human story of this cancer to improve patient and survivor care.
Researchers can work with local stakeholders in retinoblastoma care to deepen their individual and shared understanding, and engage in culturally sensitive research across different regions. Local collaborators provide key insights into cultural norms, beliefs, and practices, ensuring the research is respectful, relevant, and beneficial to the community being studied. The best studies will include parent and survivor advocates themselves from the planning stages.
Retinoblastoma care practices vary across different ethnic, cultural, and social contexts. Collaboration allows researchers to gather data from multiple centres, encompassing diverse populations and cultures. They can explore the many cultural variations influencing care and outcomes for the child and family, including beliefs, attitudes, and practices related to diagnosis, treatment recommendations, decision-making, and medical and supportive care.
Ethnographic research aims to understand the social and environmental factors that influence healthcare.
When multiple centres work together, researchers can study how retinoblastoma care and the family experience are shaped by various factors such as:
- Healthcare systems;
- Socioeconomic conditions (e.g. education, employment, income, community safety and resources); and
- Psychosocial factors (e.g. social network, support, coping, and quality of life).
Understanding the environment of the retinoblastoma story gives valuable insights into the challenges families experience, and opportunities to improve care in specific settings and circumstances.
Working across multiple centres, researchers can identify similarities and differences in retinoblastoma care practices, patient experiences, caregiver roles, and decision-making processes. By examining these variations together, they can draw out best practice, challenges, and areas needing improvement. Their wide lens approach leads to more culturally appropriate, patient-centred changes to care.
WE C Hope USA President and bilateral Rb survivor, Marissa Gonzalez, with Rb patient, Lucia Billings at One Rb World in Washington D.C.
Healthcare professionals who engage in multicentre research projects gain direct experience of advancing science for their patients, and deepen their individual understanding of retinoblastoma. Being actively involved in research activities enhances analytical skills, and encourages critical thinking and problem solving, all of which contribute to better patient care.
Collaborations foster a sense of collective purpose and shared responsibility among professionals who care for retinoblastoma patients and study the cancer. Multicentre partnerships encourage collaborators to share knowledge, develop skills, and build camaraderie – boosting individual, home team, and collaboration-wide morale.
Energy and motivation increase for the individual and collective when colleagues connect across the centres who share subspecialties, patient care interests, research focus, and well-combined personalities. When each person understands, enjoys, and feels more supported in their work, their wellbeing at work is better, and the quality of patient care is higher.
Pooled data provides accurate information about the incidence of retinoblastoma and second primary cancers, and age and cancer stage at diagnosis.
Retinoblastoma data can highlight countries and regions needing urgent awareness programs for early diagnosis, and detect changing clinical care processes and priorities early.
A documented down-trend in age and cancer stage at diagnosis will likely capture the success of awareness campaigns and screening programs, even if those campaigns are not being studied. An upward trend in age and stage of disease at diagnosis will identify serious emerging barriers to diagnosis – such as a pandemic restricting access to primary care and referral.
Second primary cancer data will provide accurate information about how these risks have changed with shifting patterns of retinoblastoma treatment – from radiotherapy to chemotherapy, and from primary enucleation to eye-salvage. They will highlight risks and priority needs for different age groups, and stimulate more survivor-focused research and investment in lifelong patient care.
By combining data and expertise from different centres, clinicians and researchers can more easily discover the vast array of genetic changes involved in the formation and growth of retinoblastoma in its various types. They can learn more, faster, about how these genes impact cancer progression and treatment response, develop and test new ways to gain accurate genetic information for every patient, and develop the most targeted therapies.
Collaborative awareness, screening, and surveillance studies can help us effectively promote early warning signs, develop sensitive screening methods, and improve surveillance protocols for at-risk children and adults. All resulting in timely detection of retinoblastoma and second cancers, and better lifelong cancer care for individuals with a known or suspected RB1 pathogenic allele.
By sharing data, expertise, and experience, collaborators can identify treatments and delivery methods that yield the best outcomes for the whole child and their eye. Through clinical trials, they can optimize existing therapies, and safely test novel approaches to care. The faster multicentre research findings translate into clinical practice, the sooner high-quality evidence will underpin doctors’ recommendations and parents’ decisions for their child’s care.
A shared data system can document side effects and adverse events, identify trends, and clearly establish treatment-related risks. Multicentre clinical trials will also record this data, assesses treatment efficacy, establish safety guidelines, and develop approaches to reduce risks to the child.
This data will empower multicentre working groups to assess and monitor risks and safety concerns over time, and develop recommendations to ensure the child’s life, potential sight, and wellbeing are protected. Collaborating with parent and survivor advocates, professionals can develop safer treatment, more effective, compassionate care, and the best outcomes possible for the whole family.
Working together, researchers are more able to assess medical and supportive approaches to retinoblastoma, and factors affecting care. By tracking patients over time, outcomes are more reliable, and patterns emerge about long-term effects that can guide improvements to clinical and supportive care. Those insights also guide new patient-focused research priorities.
Studying the retinoblastoma experience in diverse populations and cultural contexts, collaborators will identify important factors that influence care and outcomes. Their findings can help us all understand the barriers and facilitators of care and coping, especially at critical points in cancer journey such as diagnosis, treatment decision-making, and talking about vision loss and RB1 genetics.
With more understanding, care teams can clarify needs and develop targeted, relevant supports and policies to improve wellbeing and outcomes for each child, survivor, and their entire family.
The knowledge, insight, and strong evidence we gain from large-scale studies and diverse perspectives will empower our community to develop culturally sensitive care and practical retinoblastoma care guidelines. Informed care that can be tailored to the individual child, adult survivor, and family needs. Consensus best practice guidelines also highlight gaps in published evidence, motivating more collaborative research to improve care further.
The consistency required in multicentre research can rapidly benefit patient care. Following agreed care guidelines makes decisions easier and faster for both professionals and parents. Everyone involved can quickly establish a safe holistic care plan that offers the best outcomes possible. While a commonly used data system supports shared care and lifelong follow-up – no matter where the survivor lives, moves to, or receives care.
Multicentre collaboration thrives on the cooperation, open-minded innovation, and objective evaluation needed to translate evidence into patient care. Where they are encouraged and supported, visible changes to patient care will follow.
A breakout session at One Rb World 2017 in Washington D.C.
High quality research takes years to complete, going through multiple phases from the initial question to evidence that translates into a visible change in patient care. Sometimes there is a gap between one phase of research and translation of that knowledge into another phase due to challenges such as rarity.
For example, data collected by multiple centres worldwide has shown for years the high lifelong risk of certain primary cancers in carriers of an RB1 pathogenic allele. However, as the population of survivors is small and scattered widely, little research has been invested into screening, early diagnosis, and effective second cancer care. The evidence is barely useful as a tool for survivors to self-advocate for their healthcare.
Parents and survivors who actively engage in research can advocate for continued focus on the ultimate benefit and outcome for the patient throughout the years-long research process. We can ask tough questions and hold up lanterns to the most untrodden paths whose destinations promise more holistic care and more lives saved.
Below are some recent examples of collaborative research. We look forward to sharing an article later this year about the Global Retinoblastoma Study Group and the power of global research data to improve treatment, care, and patient outcomes.
Multicentre research collaborations hold huge potential to advance our knowledge and understanding of retinoblastoma, save more lives and improve care for all, for life. While collaborators can encounter many challenges, the benefits they bring to children, families and survivors are immense. We encourage all healthcare professionals, scientists, institutions, parents, and survivors to support and participate in multicentre collaborations to improve the patient experience.
If you would like to join a global community of clinicians, researchers, parents and survivors sharing knowledge and experience, and exploring priority subjects, we welcome you to One Retinoblastoma World, October 15-17 2024 in Honolulu, Hawaii. Community, conversation and collaboration for optimal care.
Visit our One Rb World 2024 Registration Portal to find all you need to know about the event, get your tickets, and book your group rate hotel room. We do hope very much to see you in Hawaii!
Sponsorship and partner opportunities are available for businesses, foundations and individuals. If you are interested in partnering with us to help host this important forum for global collaboration to improve patient care and cure, please email Marissa Gonzalez at marissa(at)wechope.org.
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About the Author
Abby’s father was diagnosed with bilateral retinoblastoma in Kenya in 1946. Abby was also born with cancer in both eyes. She has an artificial eye and limited vision in her left eye that is now failing due to late effects of radiotherapy in infancy.
Abby studied geography at university, with emphasis on development in sub-Saharan Africa. She co-founded WE C Hope with Brenda Gallie, responding to the needs of one child and the desire to help many in developing countries. After receiving many requests for help from American families and adult survivors, she co-founded the US chapter to bring hope and encourage action across the country.
Abby enjoys listening to audio books, creative writing, open water swimming and long country walks.