Title: An evaluation of a new online patient reported outcome measure (PROM) within a rare cancer in the United Kingdom (UK): establishing a national validated PROM within the UK Gestational Trophoblastic Disease (GTD) Service to improve patient care and support a new era of virtual clinics for GTD patients.
Funder: Health Education England

Abstract: Background Gestational Trophoblastic Disease (GTD) is a rare complication of pregnancy that can present as, or develop into cancer1,2. Two regional specialist centres in the United Kingdom (UK) provide treatment and support where outcomes are measured only in the medical domain2,3. A systematic review of the literature suggests GTD patients experience a wide variety of distressing symptoms and conditions that are not fully understood. Holistic assessment and support is central to the care provided to patients who are receiving chemotherapy, but not available at the moment to any patients referred for monitoring of GTD remotely, or those who have completed chemotherapy. Around 2,000 patients are referred annually to GTD specialist centres, and only around 5%-10% will need chemotherapy treatment. An estimated 1800 GTD patients are managed remotely each year. A patient-reported outcome measure (PROM) is a subjective measure, based on the patient's perspective4. Policy leaders within cancer advocate the routine use of PROMs to measure the short and long-term effects of cancer and improve communication with patients5,6,7,8. Electronic Patient Assessment Questionnaire (ePAQ) technology enables a web-based clinical assessment, specifically designed to provide detailed, reliable and meaningful self-reported symptoms that can be used as part of a holistic assessment in routine care, and capture longitudinal cohort data. Recent Macmillan funding has supported the initial development of ePAQ-GTD but further research is required to validate and evaluate its use in clinical practice, and analyse the cohort data it will create. Research Question and Aims This study will validate ePAQ-GTD enabling it to be used nationally as a PROM embedded in routine practice. Subsequently, PROM data from ePAQ-GTD will be captured in a national cohort study to address identified gaps in our knowledge and understanding of GTD. We will evaluate the experience of using ePAQ-GTD as an outcome measure, and explore the benefits of a virtual clinic for GTD patients. Research Plan: The study will have three stages and use a mixed methods design. Stage 1: A cross-sectional survey of GTD patients in Sheffield in order to establish the psychometrics of ePAQ-GTD. Stage 2: A national cohort study using ePAQ-GTD PROM data alongside an in-depth evaluation of ePAQ-GTD as an outcome measure using qualitative methods in London and Sheffield Trophoblastic Centres. Stage 3: A national pilot study to evaluate the acceptability and feasibility of virtual clinics, based on ePAQ-GTD data, using qualitative methods. Anticipated impact and dissemination Establishment of a validated PROM for use in routine care throughout the UK Improved communication for GTD patients undergoing remote monitoring To give the patients the opportunity to discuss their condition and its impact on their quality of life with a specialist nurse The provision of information that could improve patients', the public's, NHS staff's and commissioners' understanding of GTD The provision of information of how best to address GTD patients' needs The provision of information that could change clinical practice, service provision and national guidelines. This may result in better outcomes for GTD patients and a more efficient use of NHS resources

Lay abstract: What is Gestational Trophoblastic Disease? When women become pregnant they are at risk of developing Gestational Trophoblastic Disease, or GTD. This occurs when the placenta, the organ that provides nutrients to an unborn baby, grows in an abnormal way. GTD is a rare condition. Around 2000 women each year are diagnosed and it happens in 1 in 750 pregnancies. However, for those women affected it can have a devastating impact upon their lives. GTD is not one condition, but a group of conditions. The most common type of GTD is the molar pregnancy. Molar pregnancies often lead to miscarriage, but are also linked with severe bleeding from the vagina, pain, and anxiety. Molar pregnancies are pre-cancerous but unfortunately, some women will develop cancer from the abnormal placental cells. Rarer types of GTD like choriocarcinoma are outright cancers, and can happen after any type of pregnancy. All women with GTD are referred to one of three national GTD centres. Signs for cancer are measured using samples sent from home. Only five to ten women in a 100 need cancer treatment. Treatment is a mixture of chemotherapy and surgery. The number of women who get better from GTD is very high, 98 women out of 100 will survive. However, survivors may face on-going side effects like tiredness, infertility and the risk and fear of the cancer returning. The Problem: Most GTD patients only send samples from home because they live a long way from a specialist centre. Within this group are new GTD patients being monitored for signs of cancer, and survivors being monitored for the cancer coming back. The GTD services do not routinely ask these women how they feel, emotionally or physically, or how GTD is affecting their life. Recent work found that little is known about the affect of GTD on women's lives. What we will do: We have developed a new electronic questionnaire with nine GTD patients (ePAQ-GTD). The questionnaire can be completed electronically at home or hospital, on a tablet, smartphone or computer. It asks women with GTD important questions about the effects of GTD and its treatment on their lives. For example, it asks about the anxiety caused by fertility issues. The aims of this research are to assess the quality of ePAQ-GTD as part of routine care. We also want to learn about the impact of GTD on women's lives. The research will involve three stages and use two different approaches (called quantitative and qualitative methods). In stage 1 the quality of ePAQ-GTD will be tested to make sure it has the right number of easy to understand questions. In stage 2, we plan to use the questionnaire in all three GTD centres. Our aim is that 3600 women out of 6000 will complete ePAQ-GTD at different time points (0, 3, 6, 12 and 24 months from diagnosis). We will also talk to women and GTD specialists about the experience of using ePAQ-GTD as part of routine care. The final part of the study (stage 3) will be a pilot study. This will test how practical it is to use ePAQ-GTD in a virtual clinic. GTD patients will not need to travel to access the 'virtual clinic'. Instead, their specialist will carry out a phone/video consultation with them. The results from blood tests/scans and the women's ePAQ-GTD answers will be used to guide the consultation. Dissemination and PPI: Our established PPI group will be involved throughout the study. Through co-ownership of results, we will together plan and distribute the study outcomes to patient and professional groups in open-access journals, newsletters, conferences, and social media.

Research activity codes: 7.1 INDIVIDUAL CARE NEEDS
Health categories: CANCER AND NEOPLASMS;REPRODUCTIVE HEALTH AND CHILDBIRTH