Pathology (February 2014) 46(2), pp. 160–163
SOFT TISSUE PATHOLOGY
An Australasian perspective on sarcoma research, translational biology and clinical trials: the Australasian Sarcoma Study Group (ASSG) SUSIE BAE1,2, DENISE CARUSO2
AND
JAYESH DESAI1,2,3
1Australasian Sarcoma Study Group (ASSG), 2Peter MacCallum Cancer Centre, and 3Royal Melbourne Hospital, Melbourne, Victoria, Australia
Summary Each year approximately 800 Australians are diagnosed with sarcoma, accounting for less than 1% of cancer diagnoses overall. A significant proportion of these sarcoma cases are in children and adolescents. The rarity and heterogeneity of this group of tumours, coupled with Australasia’s relative geographical isolation, pose significant challenges in developing locoregional basic, translational and clinical research. The Australasian Sarcoma Study Group (ASSG) was established in 2008 as a Cooperative Cancer Clinical Research Group and is now the peak body for sarcoma research in Australasia, providing a mechanism to drive and coordinate collaborative research, promote education and assist with advocating for sarcoma within the region. This paper describes the development of ASSG and examines the current state of play with regard to sarcoma research in Australasia. Key words: Cancer research, multidisciplinary collaboration, sarcoma. Received 14 November, accepted 15 November 2013
SARCOMA IN AUSTRALIA Sarcomas are a group of rare malignant mesenchymal tumours arising from bone and soft tissue, which are marked by their diversity and complexity in both histology and molecular biology. Each year approximately 800 Australians are diagnosed with sarcoma, accounting for less than 1% of cancer diagnoses overall, comparable to other countries with a similar demographic and ethnic base. A significant proportion of these sarcoma cases are in children and adolescents.1 The heterogeneity of this group of tumours and the rarity of each subtype, coupled with Australia’s relative geographical isolation, pose significant challenges in developing our own clinical research in search of effective therapeutic agents and improving overall outcome of sarcomas in the Australasian setting.
ASSG BACKGROUND The Australasian Sarcoma Study Group (ASSG) was established in 2008 as a Cooperative Cancer Clinical Research Group within Cancer Australia’s Support for Cancer Clinical Research programme. The ASSG is the peak body for sarcoma research in Australasia, with a mission to undertake national and international basic, translational, clinical and supportive care research into sarcoma and related tumours. The ASSG was Print ISSN 0031-3025/Online ISSN 1465-3931 DOI: 10.1097/PAT.0000000000000051
#
established to provide a coordinated mechanism to drive collaborative research, promote education and assist the sarcoma community in advocating for sarcoma within the Australasian region. Although there were mechanisms for professional exchange in Australia (via an annual educational forum bringing together individuals involved with sarcoma in Australia), a recognised organisational structure to enable a formal approach to enacting the broader goals of these individuals was lacking. In parallel with this, sarcoma research (basic, translational and clinical) output, sophistication in the level of organised multidisciplinary care and community enthusiasm to advocate for sarcoma as a disease were rapidly maturing, and the opportunity for us to benefit from these synergies was increasingly recognised. This was particularly noted with respect to the opportunities for collaboration in clinical trials being offered by colleagues from the two key international cooperative groups in sarcoma, the EORTC Bone and Soft Tissue Sarcoma Group in Europe and SARC in North America. Core funding to establish the ASSG came through Cancer Australia, and they have continued to provide core infrastructure funds on a 3-year grant cycle. The ASSG has since served as the central point for coordination of collaborations to expand research capacity for the sarcoma community. The role of the ASSG within the community therefore has been broader than its international counterparts (i.e., other cooperative groups such as the EORTC or SARC), in serving core functions that extend beyond research.
ASSG CORE FUNCTIONS AND ROLES WITHIN THE AUSTRALASIAN COMMUNITY The ASSG has a pivotal role in improving outcomes for patients diagnosed with sarcoma and their families. It has long been recognised internationally that the difficult nature of sarcoma demands a multi-disciplinary team (MDT) approach to treatment that includes clinicians (including pathologists and imaging specialists), researchers, allied health and psychosocial professionals to ensure best outcomes. Specialist sarcoma centres have been established and, importantly for prospective patients and referring clinicians, identified in the key capital cities in Australia to create cohesive MDTs for patient management. These MDTs are listed on the ASSG website, and with an increasing volume of patient-patient communication and direct communication with the ASSG office, provide a rapid mechanism for patients to seek expert help at a time of need. Although we have not demanded a formal accreditation process as yet, we do ask that for a multidisciplinary group to be listed
2014 Royal College of Pathologists of Australasia
Copyright © Royal College of pathologists of Australasia. Unauthorized reproduction of this article is prohibited.
AUSTRALASIAN SARCOMA STUDY GROUP AND SARCOMA RESEARCH
on the ASSG website they require broad clinical engagement as highlighted above, as well as active participation in clinical trials. The MDTs have in turn enabled a smoother engagement process with the ASSG, in particular around considering participation in research studies including clinical trials and the clinical database (see below).
CLINICAL TRIALS The ASSG acts as sponsor for local investigator-initiated trials, as local sponsor (if needed) for international cooperative group trials that we are participating in, and as a conduit for clinician engagement with industry clinical trials. Clinical trials are clearly a high priority endeavour for sarcoma patients, and a focus of Cancer Australia. The ASSG has sponsored and conducted 23 trials since its inception, and continues to seek important trials that fulfil treatment gaps and provide further treatment options for those patients that have failed standard therapies. Importantly, sarcoma clinical trials that have been conducted came from several sources including investigatorinitiated, industry, and collaborations with international cooperative groups. The development of investigator-initiated trials has been strongly supported through a partnership with the Centre for Biostatistics and Clinical Trials (BaCT). We have a formal engagement process in place, which provides core support for protocol and budget development, CRF development, trial coordination and biostatistical support during all stages of the trial. This essentially enables us to readily access these services on an as-needed basis, and has been a critical step in enabling us to allocate our resources efficiently in the short time we have been in existence. The financial support for BaCT services was initially provided by Cancer Australia, but we have since evolved to funding those services from a variety of sources. The relationship with BaCT remains fundamental to our ability to deliver coordinated clinical trials to our members, and over the last couple of years we have developed this further by co-locating the ASSG office to BaCT, as well as creating positions on the Board and Executive for the BaCT Director. This partnership model has served us well, and is certainly one that other smaller cooperative groups could consider adopting. In addition to investigator-initiated research, either developed locally or by other international cooperative group partners, the ASSG has taken on a role in helping facilitate its members’ participation in typical industry-sponsored trials, including registration studies. This has, for the most part, simply involved helping coordinate access for sponsors to key sarcoma centres/investigators in Australia. On occasion, we have also used this mechanism to develop parallel investigator-initiated translational studies that could be tagged onto the primary trial; e.g., adding in a specific quality-of-life substudy to address key issues unique to the Australian context. Although this is clearly not one of our key functions, we have felt that our commitment to facilitating access for all Australian sarcoma patients to a diverse range of clinical research studies was our first priority, and with the limited number of options typically on offer we felt that broad coordination of a ‘clinical trials portfolio’ was critically important. Additionally, by continuing to build our collective expertise and capacity for clinical research/clinical trials via these multiple mechanisms, our ability to attract future studies to our region, and meaningfully contribute to them, will continue to improve. We have also been fortunate in being provided with opportunities to participate in
161
internationally-led cooperative group trials, typically from groups based in Europe (including the EORTC, EURAMOS and country-led groups such as the French Sarcoma Group and Italian Sarcoma group) or North America (SARC), via an ‘Intergroup’ mechanism. Close international ties have been forged by multiple members of the ASSG in both the adult and paediatric domain, and with a mechanism in place to now take on the multiple responsibilities required, including acting as a local sponsor, we are now well placed to contribute to these international efforts.
BASIC AND TRANSLATIONAL RESEARCH Whilst clinical trials remain a very high priority for the ASSG, considerable efforts have also been placed into encouraging collaboration in basic and translational research, to some extent differentiating us from typical cooperative groups here and internationally, which tend to be almost exclusively trialsfocused. It was recognised early that a number of laboratory-based groups were focused on sarcoma around Australia, but often lacked an intimate knowledge of related work conducted by other groups, and hence collaborative opportunities. Additionally, clinical engagement was variable, and therefore translational opportunities were sometimes missed. The ASSG has deliberately identified and engaged with sarcoma-focused laboratory groups via invitations to our twice-yearly research meetings/workshops, with one of these meetings having a distinctive basic/translational research theme. Additionally, we have been very fortunate in receiving generous support from the philanthropic community in the form of seed grants to help support early research initiatives. Through this mechanism we have been able to offer ten Sarcoma Research Grants to basic science and allied health researchers to date, and have also awarded two basic science PhD scholarships. These grants and scholarships have provided excellent opportunities for broader engagement and collaboration, and have also provided an opportunity for new groups entering the sarcoma research space to have an initial opportunity to receive valuable funds. Additionally, our philanthropic community has seen tremendous value for their investment, as they have been able to support young investigators in sarcoma research. Of note, the translational research projects have not been limited to ‘benchto-bedside’ translation, but also in translating ideas into practice in relation to supportive care and allied-health led projects. Some of these early efforts have now been developed into investigator-initiated clinical trials, and provided further opportunities for ‘bedside-to-bench’ translation.
ADDITIONAL AREAS OF FOCUS The ASSG identifies gaps within sarcoma treatment and research that require future work and support. Sarcomas disproportionately affect the adolescent and young adult (AYA) population and therefore AYA patients have been a key focus of ASSG research and supportive care efforts. The ASSG is working with paediatric and adult sarcoma centres to identify treatment gaps and address the needs of these patients. Collaboration with other AYA groups internationally, as well as the large paediatric trials cooperative groups, continue to develop, with a number of trial opportunities now being made available. Additionally, other patient groups have more recently commanded specific focus. The gynaecological sarcoma population
Copyright © Royal College of pathologists of Australasia. Unauthorized reproduction of this article is prohibited.
162
BAE et al.
of patients has been recognised as underserved, given they are managed by either sarcoma-focused oncologists or gynaeoncologists. The World Sarcoma Network, of which the ASSG is a founding member, has developed initiatives to highlight this group of sarcomas for further awareness and research.2 Already, important differences have been identified in how our respective groups manage these patients (e.g., uterine leiomyosarcomas), perhaps reflecting underlying biases and varying levels of familiarity with using specific drugs in practice. The ASSG has sought to engage our gynae-oncologists, both directly and via the existing gynae-oncology cooperative groups in Australia. Ongoing collaboration and joint efforts in developing clinical trials and research initiatives have been enthusiastically embraced by both subspecialties.
BETTER UNDERSTANDING AND IMPROVING CLINICAL PRACTICE Rare and complex diseases such as sarcoma present significant challenges for evaluating the quality and consistency of care delivered to our patients. Two specific projects being developed to address this are (1) the development and publication of the National Sarcoma Treatment Guidelines, and (2) the National Sarcoma Database, both discussed further below. The first Australian Sarcoma Guidelines have been developed by the ASSG in partnership with Cancer Council Australia. After a rigorous appraisal of the literature by a large number of ASSG members to inform evidence-based best practice, the guidelines were developed over an 18-month period using a wiki platform, and were published online towards the end of 2013.3 It is envisaged that these will provide a consensus view on sarcoma management in Australia, and thereby help to standardise practice between different centres. During its formation, the ASSG recognised the importance of getting a clear indication of the impact of sarcoma on our community, both to better guide clinical research opportunities and to advocate for resources with government by providing accurate data. Established mechanisms such as cancer registries were not adequately equipped to collect accurate clinical data because of the complexity involved in capturing data from the limited information available to central registries (including an ability to verify revised histological diagnoses upon expert review, accurate staging and the like). The heterogeneous nature of sarcoma brings inconsistencies in sub-classifying what is essentially a collection of more than 80 different disease subtypes, into their specific entities. Accordingly, the collection of high-quality clinical data at a site level, using the same database, was recognised as a key goal of the ASSG. Currently, there are five major sarcoma services in Australia and sarcoma data collection with a dedicated data manager is active at all sites. Most centres use the same data collection tool and collect the same data fields, which cover information from the patients’ initial presentation, diagnosis and treatment details to followup data on recurrence and death. There is a system in place through local IT expertise, which can link all of the datasets for project purposes. Thus, many research opportunities can be created, with the ability to extract consistent data for analysis. Well-annotated datasets can be used in understanding and mapping current heath care practice across Australia, and can also provide important background clinical information for translational research studies conducted on tumour biospecimens.
Pathology (2014), 46(2), February
Examples of research initiatives utilising the database Three projects which are currently in progress include: 1. A retrospective study on use of systemic therapies in the management of advanced soft tissue sarcoma. This project is designed to explore, in detail, what the current standard of care is in sarcoma centres in Australia, and therefore enable better planning for future trials as well as to serve as a baseline assessment prior to the national guidelines being implemented. 2. A pilot study to assess cost-effectiveness in surveillance imaging for patients who complete curative-intent treatment for localised extremity soft tissue sarcoma. Firstly, we are mapping how patients with soft tissue sarcomas are actually followed up, and will then model this to conduct a prospective study exploring the costeffectiveness of surveillance, particularly with imaging of patients. 3. A retrospective study on treatment pattern and outcome for patients with rare sarcoma subtypes, in this instance: localised subcutaneous soft tissue sarcomas. Apart from a primary aim of mapping changing practice at a site level with national initiatives such as the guidelines, long-term goals will also be to perform pooled or collaborative studies with other regional and international groups, including correlative molecularly-focused studies with tissue and annotated data.
REGIONAL EFFORTS The ASSG remains the only regional collaborative group for sarcoma, and therefore has the responsibility to encompass all aspects of research, community support, awareness and advocacy to provide access to expert resources. The ASSG has an important role in establishing international collaborations, both regionally and globally. Given the rare nature of sarcomas it is crucial to form collaborations in order to conduct clinical trials and to improve outcomes. Asian countries including Singapore, Hong Kong and South Korea have no formal sarcoma infrastructure and can serve as key future partners for sarcoma initiatives and research projects. A recent clinical trial exploring the role of hedgehog inhibitor in bone sarcoma is a good example whereby collaboration with Singapore was fruitful in launching this study at the National Cancer Centre, Singapore.4
COMMUNITY ENGAGEMENT As outlined above, the ASSG has taken on an important responsibility to the wider community, to sarcoma consumers and the sarcoma philanthropic community. The ASSG receives donations from the public under the pledge that 100% of donations will support sarcoma research, an approach that has been very successful to date. Through raising sarcoma awareness, engagement with patients and families who have a direct experience with sarcoma and working with philanthropic groups to achieve their goals, the ASSG serves as a supportive resource for these overlapping endeavours.
CONCLUSIONS Since ASSG’s formation the sarcoma community has been able to turn to the ASSG for all aspects of sarcoma: treatment, education, research and consumer outreach. The goals for the
Copyright © Royal College of pathologists of Australasia. Unauthorized reproduction of this article is prohibited.
AUSTRALASIAN SARCOMA STUDY GROUP AND SARCOMA RESEARCH
ASSG are by necessity multifaceted and thus the ASSG has a broad portfolio of activities with the single aim of improving outcomes for sarcoma patients and their families. Conflicts of interest and sources of funding: The authors state that there are no conflicts of interest to disclose. Address for correspondence: Dr J. Desai, Department of Medical Oncology, Peter MacCallum Cancer Centre, St Andrews Place, East Melbourne, Vic 3004, Australia. E-mail: [email protected]
163
References 1. Australian Institute of Health and Welfare (AIHW) and Australasian Association of Cancer Registries (AACR). Cancer in Australia: An Overview. Cancer series no. 46. Cat. no. CAN 42. Canberra: AIHW, 2008. 2. World Sarcoma Network. Cited Nov 2013. http://www.worldsarcomanet work.com/?cat=4 3. Australasian Sarcoma Study Group (ASSG). Clinical practice guidelines for the management of adult onset sarcoma. Cited Nov 2013. http://wiki. cancer.org.au/australia/Guidelines:Sarcoma 4. Australian New Zealand Clinical Trials Registry (ANZCTR). Cited Nov 2013. https://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?id= 362511.
Copyright © Royal College of pathologists of Australasia. Unauthorized reproduction of this article is prohibited.
SOFT TISSUE PATHOLOGY
An Australasian perspective on sarcoma research, translational biology and clinical trials: the Australasian Sarcoma Study Group (ASSG) SUSIE BAE1,2, DENISE CARUSO2
AND
JAYESH DESAI1,2,3
1Australasian Sarcoma Study Group (ASSG), 2Peter MacCallum Cancer Centre, and 3Royal Melbourne Hospital, Melbourne, Victoria, Australia
Summary Each year approximately 800 Australians are diagnosed with sarcoma, accounting for less than 1% of cancer diagnoses overall. A significant proportion of these sarcoma cases are in children and adolescents. The rarity and heterogeneity of this group of tumours, coupled with Australasia’s relative geographical isolation, pose significant challenges in developing locoregional basic, translational and clinical research. The Australasian Sarcoma Study Group (ASSG) was established in 2008 as a Cooperative Cancer Clinical Research Group and is now the peak body for sarcoma research in Australasia, providing a mechanism to drive and coordinate collaborative research, promote education and assist with advocating for sarcoma within the region. This paper describes the development of ASSG and examines the current state of play with regard to sarcoma research in Australasia. Key words: Cancer research, multidisciplinary collaboration, sarcoma. Received 14 November, accepted 15 November 2013
SARCOMA IN AUSTRALIA Sarcomas are a group of rare malignant mesenchymal tumours arising from bone and soft tissue, which are marked by their diversity and complexity in both histology and molecular biology. Each year approximately 800 Australians are diagnosed with sarcoma, accounting for less than 1% of cancer diagnoses overall, comparable to other countries with a similar demographic and ethnic base. A significant proportion of these sarcoma cases are in children and adolescents.1 The heterogeneity of this group of tumours and the rarity of each subtype, coupled with Australia’s relative geographical isolation, pose significant challenges in developing our own clinical research in search of effective therapeutic agents and improving overall outcome of sarcomas in the Australasian setting.
ASSG BACKGROUND The Australasian Sarcoma Study Group (ASSG) was established in 2008 as a Cooperative Cancer Clinical Research Group within Cancer Australia’s Support for Cancer Clinical Research programme. The ASSG is the peak body for sarcoma research in Australasia, with a mission to undertake national and international basic, translational, clinical and supportive care research into sarcoma and related tumours. The ASSG was Print ISSN 0031-3025/Online ISSN 1465-3931 DOI: 10.1097/PAT.0000000000000051
#
established to provide a coordinated mechanism to drive collaborative research, promote education and assist the sarcoma community in advocating for sarcoma within the Australasian region. Although there were mechanisms for professional exchange in Australia (via an annual educational forum bringing together individuals involved with sarcoma in Australia), a recognised organisational structure to enable a formal approach to enacting the broader goals of these individuals was lacking. In parallel with this, sarcoma research (basic, translational and clinical) output, sophistication in the level of organised multidisciplinary care and community enthusiasm to advocate for sarcoma as a disease were rapidly maturing, and the opportunity for us to benefit from these synergies was increasingly recognised. This was particularly noted with respect to the opportunities for collaboration in clinical trials being offered by colleagues from the two key international cooperative groups in sarcoma, the EORTC Bone and Soft Tissue Sarcoma Group in Europe and SARC in North America. Core funding to establish the ASSG came through Cancer Australia, and they have continued to provide core infrastructure funds on a 3-year grant cycle. The ASSG has since served as the central point for coordination of collaborations to expand research capacity for the sarcoma community. The role of the ASSG within the community therefore has been broader than its international counterparts (i.e., other cooperative groups such as the EORTC or SARC), in serving core functions that extend beyond research.
ASSG CORE FUNCTIONS AND ROLES WITHIN THE AUSTRALASIAN COMMUNITY The ASSG has a pivotal role in improving outcomes for patients diagnosed with sarcoma and their families. It has long been recognised internationally that the difficult nature of sarcoma demands a multi-disciplinary team (MDT) approach to treatment that includes clinicians (including pathologists and imaging specialists), researchers, allied health and psychosocial professionals to ensure best outcomes. Specialist sarcoma centres have been established and, importantly for prospective patients and referring clinicians, identified in the key capital cities in Australia to create cohesive MDTs for patient management. These MDTs are listed on the ASSG website, and with an increasing volume of patient-patient communication and direct communication with the ASSG office, provide a rapid mechanism for patients to seek expert help at a time of need. Although we have not demanded a formal accreditation process as yet, we do ask that for a multidisciplinary group to be listed
2014 Royal College of Pathologists of Australasia
Copyright © Royal College of pathologists of Australasia. Unauthorized reproduction of this article is prohibited.
AUSTRALASIAN SARCOMA STUDY GROUP AND SARCOMA RESEARCH
on the ASSG website they require broad clinical engagement as highlighted above, as well as active participation in clinical trials. The MDTs have in turn enabled a smoother engagement process with the ASSG, in particular around considering participation in research studies including clinical trials and the clinical database (see below).
CLINICAL TRIALS The ASSG acts as sponsor for local investigator-initiated trials, as local sponsor (if needed) for international cooperative group trials that we are participating in, and as a conduit for clinician engagement with industry clinical trials. Clinical trials are clearly a high priority endeavour for sarcoma patients, and a focus of Cancer Australia. The ASSG has sponsored and conducted 23 trials since its inception, and continues to seek important trials that fulfil treatment gaps and provide further treatment options for those patients that have failed standard therapies. Importantly, sarcoma clinical trials that have been conducted came from several sources including investigatorinitiated, industry, and collaborations with international cooperative groups. The development of investigator-initiated trials has been strongly supported through a partnership with the Centre for Biostatistics and Clinical Trials (BaCT). We have a formal engagement process in place, which provides core support for protocol and budget development, CRF development, trial coordination and biostatistical support during all stages of the trial. This essentially enables us to readily access these services on an as-needed basis, and has been a critical step in enabling us to allocate our resources efficiently in the short time we have been in existence. The financial support for BaCT services was initially provided by Cancer Australia, but we have since evolved to funding those services from a variety of sources. The relationship with BaCT remains fundamental to our ability to deliver coordinated clinical trials to our members, and over the last couple of years we have developed this further by co-locating the ASSG office to BaCT, as well as creating positions on the Board and Executive for the BaCT Director. This partnership model has served us well, and is certainly one that other smaller cooperative groups could consider adopting. In addition to investigator-initiated research, either developed locally or by other international cooperative group partners, the ASSG has taken on a role in helping facilitate its members’ participation in typical industry-sponsored trials, including registration studies. This has, for the most part, simply involved helping coordinate access for sponsors to key sarcoma centres/investigators in Australia. On occasion, we have also used this mechanism to develop parallel investigator-initiated translational studies that could be tagged onto the primary trial; e.g., adding in a specific quality-of-life substudy to address key issues unique to the Australian context. Although this is clearly not one of our key functions, we have felt that our commitment to facilitating access for all Australian sarcoma patients to a diverse range of clinical research studies was our first priority, and with the limited number of options typically on offer we felt that broad coordination of a ‘clinical trials portfolio’ was critically important. Additionally, by continuing to build our collective expertise and capacity for clinical research/clinical trials via these multiple mechanisms, our ability to attract future studies to our region, and meaningfully contribute to them, will continue to improve. We have also been fortunate in being provided with opportunities to participate in
161
internationally-led cooperative group trials, typically from groups based in Europe (including the EORTC, EURAMOS and country-led groups such as the French Sarcoma Group and Italian Sarcoma group) or North America (SARC), via an ‘Intergroup’ mechanism. Close international ties have been forged by multiple members of the ASSG in both the adult and paediatric domain, and with a mechanism in place to now take on the multiple responsibilities required, including acting as a local sponsor, we are now well placed to contribute to these international efforts.
BASIC AND TRANSLATIONAL RESEARCH Whilst clinical trials remain a very high priority for the ASSG, considerable efforts have also been placed into encouraging collaboration in basic and translational research, to some extent differentiating us from typical cooperative groups here and internationally, which tend to be almost exclusively trialsfocused. It was recognised early that a number of laboratory-based groups were focused on sarcoma around Australia, but often lacked an intimate knowledge of related work conducted by other groups, and hence collaborative opportunities. Additionally, clinical engagement was variable, and therefore translational opportunities were sometimes missed. The ASSG has deliberately identified and engaged with sarcoma-focused laboratory groups via invitations to our twice-yearly research meetings/workshops, with one of these meetings having a distinctive basic/translational research theme. Additionally, we have been very fortunate in receiving generous support from the philanthropic community in the form of seed grants to help support early research initiatives. Through this mechanism we have been able to offer ten Sarcoma Research Grants to basic science and allied health researchers to date, and have also awarded two basic science PhD scholarships. These grants and scholarships have provided excellent opportunities for broader engagement and collaboration, and have also provided an opportunity for new groups entering the sarcoma research space to have an initial opportunity to receive valuable funds. Additionally, our philanthropic community has seen tremendous value for their investment, as they have been able to support young investigators in sarcoma research. Of note, the translational research projects have not been limited to ‘benchto-bedside’ translation, but also in translating ideas into practice in relation to supportive care and allied-health led projects. Some of these early efforts have now been developed into investigator-initiated clinical trials, and provided further opportunities for ‘bedside-to-bench’ translation.
ADDITIONAL AREAS OF FOCUS The ASSG identifies gaps within sarcoma treatment and research that require future work and support. Sarcomas disproportionately affect the adolescent and young adult (AYA) population and therefore AYA patients have been a key focus of ASSG research and supportive care efforts. The ASSG is working with paediatric and adult sarcoma centres to identify treatment gaps and address the needs of these patients. Collaboration with other AYA groups internationally, as well as the large paediatric trials cooperative groups, continue to develop, with a number of trial opportunities now being made available. Additionally, other patient groups have more recently commanded specific focus. The gynaecological sarcoma population
Copyright © Royal College of pathologists of Australasia. Unauthorized reproduction of this article is prohibited.
162
BAE et al.
of patients has been recognised as underserved, given they are managed by either sarcoma-focused oncologists or gynaeoncologists. The World Sarcoma Network, of which the ASSG is a founding member, has developed initiatives to highlight this group of sarcomas for further awareness and research.2 Already, important differences have been identified in how our respective groups manage these patients (e.g., uterine leiomyosarcomas), perhaps reflecting underlying biases and varying levels of familiarity with using specific drugs in practice. The ASSG has sought to engage our gynae-oncologists, both directly and via the existing gynae-oncology cooperative groups in Australia. Ongoing collaboration and joint efforts in developing clinical trials and research initiatives have been enthusiastically embraced by both subspecialties.
BETTER UNDERSTANDING AND IMPROVING CLINICAL PRACTICE Rare and complex diseases such as sarcoma present significant challenges for evaluating the quality and consistency of care delivered to our patients. Two specific projects being developed to address this are (1) the development and publication of the National Sarcoma Treatment Guidelines, and (2) the National Sarcoma Database, both discussed further below. The first Australian Sarcoma Guidelines have been developed by the ASSG in partnership with Cancer Council Australia. After a rigorous appraisal of the literature by a large number of ASSG members to inform evidence-based best practice, the guidelines were developed over an 18-month period using a wiki platform, and were published online towards the end of 2013.3 It is envisaged that these will provide a consensus view on sarcoma management in Australia, and thereby help to standardise practice between different centres. During its formation, the ASSG recognised the importance of getting a clear indication of the impact of sarcoma on our community, both to better guide clinical research opportunities and to advocate for resources with government by providing accurate data. Established mechanisms such as cancer registries were not adequately equipped to collect accurate clinical data because of the complexity involved in capturing data from the limited information available to central registries (including an ability to verify revised histological diagnoses upon expert review, accurate staging and the like). The heterogeneous nature of sarcoma brings inconsistencies in sub-classifying what is essentially a collection of more than 80 different disease subtypes, into their specific entities. Accordingly, the collection of high-quality clinical data at a site level, using the same database, was recognised as a key goal of the ASSG. Currently, there are five major sarcoma services in Australia and sarcoma data collection with a dedicated data manager is active at all sites. Most centres use the same data collection tool and collect the same data fields, which cover information from the patients’ initial presentation, diagnosis and treatment details to followup data on recurrence and death. There is a system in place through local IT expertise, which can link all of the datasets for project purposes. Thus, many research opportunities can be created, with the ability to extract consistent data for analysis. Well-annotated datasets can be used in understanding and mapping current heath care practice across Australia, and can also provide important background clinical information for translational research studies conducted on tumour biospecimens.
Pathology (2014), 46(2), February
Examples of research initiatives utilising the database Three projects which are currently in progress include: 1. A retrospective study on use of systemic therapies in the management of advanced soft tissue sarcoma. This project is designed to explore, in detail, what the current standard of care is in sarcoma centres in Australia, and therefore enable better planning for future trials as well as to serve as a baseline assessment prior to the national guidelines being implemented. 2. A pilot study to assess cost-effectiveness in surveillance imaging for patients who complete curative-intent treatment for localised extremity soft tissue sarcoma. Firstly, we are mapping how patients with soft tissue sarcomas are actually followed up, and will then model this to conduct a prospective study exploring the costeffectiveness of surveillance, particularly with imaging of patients. 3. A retrospective study on treatment pattern and outcome for patients with rare sarcoma subtypes, in this instance: localised subcutaneous soft tissue sarcomas. Apart from a primary aim of mapping changing practice at a site level with national initiatives such as the guidelines, long-term goals will also be to perform pooled or collaborative studies with other regional and international groups, including correlative molecularly-focused studies with tissue and annotated data.
REGIONAL EFFORTS The ASSG remains the only regional collaborative group for sarcoma, and therefore has the responsibility to encompass all aspects of research, community support, awareness and advocacy to provide access to expert resources. The ASSG has an important role in establishing international collaborations, both regionally and globally. Given the rare nature of sarcomas it is crucial to form collaborations in order to conduct clinical trials and to improve outcomes. Asian countries including Singapore, Hong Kong and South Korea have no formal sarcoma infrastructure and can serve as key future partners for sarcoma initiatives and research projects. A recent clinical trial exploring the role of hedgehog inhibitor in bone sarcoma is a good example whereby collaboration with Singapore was fruitful in launching this study at the National Cancer Centre, Singapore.4
COMMUNITY ENGAGEMENT As outlined above, the ASSG has taken on an important responsibility to the wider community, to sarcoma consumers and the sarcoma philanthropic community. The ASSG receives donations from the public under the pledge that 100% of donations will support sarcoma research, an approach that has been very successful to date. Through raising sarcoma awareness, engagement with patients and families who have a direct experience with sarcoma and working with philanthropic groups to achieve their goals, the ASSG serves as a supportive resource for these overlapping endeavours.
CONCLUSIONS Since ASSG’s formation the sarcoma community has been able to turn to the ASSG for all aspects of sarcoma: treatment, education, research and consumer outreach. The goals for the
Copyright © Royal College of pathologists of Australasia. Unauthorized reproduction of this article is prohibited.
AUSTRALASIAN SARCOMA STUDY GROUP AND SARCOMA RESEARCH
ASSG are by necessity multifaceted and thus the ASSG has a broad portfolio of activities with the single aim of improving outcomes for sarcoma patients and their families. Conflicts of interest and sources of funding: The authors state that there are no conflicts of interest to disclose. Address for correspondence: Dr J. Desai, Department of Medical Oncology, Peter MacCallum Cancer Centre, St Andrews Place, East Melbourne, Vic 3004, Australia. E-mail: [email protected]
163
References 1. Australian Institute of Health and Welfare (AIHW) and Australasian Association of Cancer Registries (AACR). Cancer in Australia: An Overview. Cancer series no. 46. Cat. no. CAN 42. Canberra: AIHW, 2008. 2. World Sarcoma Network. Cited Nov 2013. http://www.worldsarcomanet work.com/?cat=4 3. Australasian Sarcoma Study Group (ASSG). Clinical practice guidelines for the management of adult onset sarcoma. Cited Nov 2013. http://wiki. cancer.org.au/australia/Guidelines:Sarcoma 4. Australian New Zealand Clinical Trials Registry (ANZCTR). Cited Nov 2013. https://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?id= 362511.
Copyright © Royal College of pathologists of Australasia. Unauthorized reproduction of this article is prohibited.
