International Myeloma Workshop in Boston productive for the Myeloma and Related Diseases Registry (MRDR)
The MRDR team presented at the recent International Myeloma Workshop (IMW) in Boston, attended by over 3200 delegates. In a special Nursing Symposium, Dr Elizabeth Moore, MRDR research fellow, presented results of “My-PROMPT”: a pilot randomised trial testing the feasibility of real-time reporting to clinicians of myeloma patient-reported outcome measures; and Prof Andrew Spencer, MRDR Coordinating Principal Investigator, gave an invited presentation on the role of myeloma registries in improving real-world outcomes. In a plenary session, Andrew Spencer and MRDR steering committee member Prof Joy Ho represented Australia in an update on clinical trials around the world.
MRDR posters included the impact of number of cycles of therapy on outcomes in patients treated with bortezomib induction (A/Prof Peter Mollee), and myeloma in Pacific Islanders in New Zealand (A/Prof Hilary Blacklock).
The inaugural Asia-Pacific MRDR (APAC MRDR: a sister registry of the MRDR) Investigators’ Meeting was held during IMW. It was well attended by investigators from Korea, China, Hong Kong, Taiwan, Singapore and Australia, who discussed progress with Andrew Spencer and APAC MRDR Project Manager Naomi Aoki.
Dr Andrew Flint presents at the Military Health System Research Symposium, Florida
Dr Andrew Flint, Monash PhD candidate, recently presented his work on the ACSePT score for predicting platelet transfusion in cardiac surgery, with an oral abstract entitled “Predictive model for platelet transfusion in patients with acute haemorrhage” at the Military Health System Research Symposium in Florida, USA.
Andrew is supervised by Prof Michael Reade, A/Prof Zoe McQuilten and Prof Erica Wood.
Using data from the Massive Transfusion Registry to understand and improve inventory management for major haemorrhage
Helen Haysom, transfusion scientist and project coordinator for the Massive Transfusion Registry (MTR), recently updated members of the Victorian Immunohaematology Discussion Group on two interesting MTR analyses focussing on (1) the use of group O, RhD negative red cells in urgent bleeding situations, and (2) supply of RhD compatible red cells to RhD negative females of child-bearing potential – for whom the two most common major bleeding contexts were trauma and obstetric bleeding. The data were originally presented at the International Society of Blood Transfusion international congress in Toronto in 2018. The MTR provides valuable data for a wide range of purposes, including understanding and improving inventory management.
ICAN study open to recruitment – Immunoglobulin use in Chronic lymphocytic leukaemia And Non-Hodgkin lymphoma
The ICAN project, led by A/Prof Zoe McQuilten, and funded by the National Blood Authority, is collecting data on immunoglobulin replacement and other infection prevention strategies, as well as patient-centred outcomes, in 500 patients with chronic lymphocytic leukaemia (CLL) and non-Hodgkin lymphoma (NHL). In a subgroup of participants, the study will also collect serial blood samples for novel immune-profiling studies. The project uses the network and infrastructure of the Lymphoma and Related Diseases Registry, which includes patients with (CLL), managed by TRU. It complements the work of the IMPROVE study in myeloma, also currently underway. Further information is available below, or by contacting the Lymphoma and Related Diseases Registry (firstname.lastname@example.org).
NHMRC Investigator Grant awarded to Professor Erica Wood
Congratulations to Erica Wood, who has been awarded an L2 Investigator Grant by the NHMRC. This prestigious award will support the work of Erica and the Transfusion Research Unit over the next 5 years.
3000th patient recruited to the Myeloma and Related Diseases Registry
Congratulations to the Myeloma and Related Diseases Registry (MRDR) team – the registry has now recruited more than 3000 patients! The MRDR captures and analyses data on diagnoses, treatment and outcomes for patients with multiple myeloma and monoclonal gammopathy of undetermined significance (MGUS), and supports a number of important linked research activities.
The MRDR is chaired by Professor Andrew Spencer (Alfred Hospital) and managed by the Transfusion Research Unit at SPHPM. Thirty-six sites are already participating across Australia and New Zealand, and more are joining. Congratulations to Northern Hospital in Melbourne who recruited the 3000th patient!
More information on the MRDR and its projects: mrdr.net.au
Information on the Asia-Pacific expansion of the MRDR: apacmrdr.org
Professor Erica Wood delivers plenary lecture at the South African National Blood Transfusion Congress
Erica Wood was an invited international speaker at the recent South African National Blood Transfusion Congress, held in Sun City. Erica shared her experience in three invited presentations on patient blood management, international haemovigilance, and careers in transfusion medicine, and also participated in judging the congress singing competition!
Professor Johanna van der Bom visits the Transfusion Research Unit
Professor Johanna (Anske) van der Bom, Professor of Clinical Epidemiology at the University of Leiden in The Netherlands, and head of Sanquin's Center for Clinical Transfusion Research (CCTR) visited the TRU in July, while in Melbourne for the congress of the International Society of Thrombosis and Haemostasis. Anske shared some of her research group's activities, including the role of the CCTR’s "scientific integrity committee" -- a governance and quality review mechanism for research proposals, publications and data management. Anske was the primary PhD supervisor for Dr Nic Saadah, who re-joined the TRU earlier this year.
A/Prof Zoe McQuilten speaks at the CICM Trauma conference
A/Prof Zoe McQuilten presented an invited lecture on Innovations in Transfusion: Management of Traumatic Haemorrhage at the College of Intensive Care Medicine’s Annual Scientific Meeting in Cairns in June.
TRU at the International Society of Blood Transfusion congress in Basel
Dr Allison Mo and Prof Erica Wood represented TRU at the recent ISBT congress in Basel, Switzerland. Allison made an invited presentation on the multidisciplinary management of anaemia in a session focussed on the role of the transfusion practitioner, and participated in a panel discussion. Erica is president-elect of ISBT.
Photo: L-R: Linley Bielby, Rachel Moss and Allison Mo at the ISBT session. Photo with thanks to Dr Chris Hogan.
Aplastic Anaemia Registry and DIAAMOND study profiled at inaugural National Symposium on Bone Marrow Failure Syndromes, 25–26 May 2019
Data from the Aplastic Anaemia Registry were presented at the inaugural National Symposium on Bone Marrow Failure Syndromes on 25–26 May 2019. Supported by Maddie Riewoldt’s Vision, the symposium attracted nearly 200 participants, including clinicians, research scientists and patients from across Australia and New Zealand.
Dr Lucy Fox, TRU clinical research fellow, provided an overview of registry activities to date and plans for the future. A/Prof Zoe McQuilten presented a summary of the DIAAMOND study of avatrombopag, a second-generation thrombopoietin receptor agonist, which will be trialled in both treatment-naïve and relapsed/refractory adult patients with AA. DIAAMOND study is funded by the Medical Research Future Fund. Prof Erica Wood was a member of the symposium steering committee.
AAR investigators also recently published a review of AA in the Internal Medicine Journal: ncbi.nlm.nih.gov/pubmed/30324755
Celebrating 10 years of the Thrombotic Microangiopathies Registry with a symposium at the International Society on Thrombosis and Haemostasis and TTP practice survey
TRU is celebrating 10 years of the Thrombotic Thrombocytopenic Purpura (TTP)/Thrombotic Microangiopathies Registry, and hosting a one-day symposium on Friday 5 July 2019, as one of the pre-congress symposia ahead of the meeting of the International Society on Thrombosis and Haemostasis in Melbourne, 6–10 July 2019.
The interactive seminar, “Making sense of thrombotic microangiopathies”, features international and Australian invited speakers and interesting case discussions. All are welcome and the symposium is free to attend.
Program and registration information: isth2019.org/pre-congress-symposia
For more information, please contact us via email: Med-TTPRegistry@monash.edu
The TTP Registry is conducting a survey of practice in managing patients with TTP and other TMAs. This survey updates a similar survey performed in 2015, and seeks to determine how widely specialised testing (ADAMTS13 levels, anti-ADAMTS13 antibodies, genetic testing etc) is being performed, the range of therapies used in the management of TTP/TMAs and if, or how, this has changed over the last 4 years.
This survey should take only 15 to 20 minutes: monash.az1.qualtrics.com/jfe/form/SV_0PtGBZ9fqmJaZkV
International REDDS-2 trial – A feasibility randomised pilot trial of weekly-interval red cell transfusion schedule in myelodysplastic syndromes
Building on the success of the REDDS-1 trial of Hb thresholds in MDS, presented at the American Society of Hematology in 2018 (bloodjournal.org/content/132/Suppl_1/527) we are conducting a randomised pilot clinical trial to assess feasibility of delivery of a weekly red cell transfusion schedule using matched red cells, and impact on quality of life and physical function outcomes, in patients with transfusion-dependent myelodysplasia (MDS).
A collaboration between Australia and the UK, the aim of this study is to inform the design and conduct of a definitive international randomised trial to compare different strategies for RBC transfusion in outpatients with MDS. The Australian arm of the study is funded in part by a 2018 research grant from the Australian and New Zealand Society of Blood Transfusion.
A longitudinal population study of current transfusion needs and changes over time in elderly patients with myelodysplastic syndromes
Congratulations to TRU’s Dr Allison Mo (haematologist and PhD student), A/Prof Zoe McQuilten and Prof Erica Wood, and their collaborators A/Prof Jake Shortt of Monash University and Mr Ani Goswami and Ms Christine Quek of Western and Central Melbourne Integrated Cancer Services, who have been awarded 2019 research grant funding from the Australian and New Zealand Society of Blood Transfusion for this project. The research will analyse multiple linked statewide population-based datasets to investigate the current needs and changing trends of transfusion requirements in MDS patients in Victoria over 15 years.
The project will provide new information to clinicians, patients, health services and policy makers on current transfusion needs for MDS patients, and inform policy development and health resource allocation.
2000th patient recruited to the Lymphoma and Related Diseases Registry
Congratulations to the team at Peter MacCallum Cancer Centre, who recruited the 2000th patient to the Lymphoma and Related Diseases Registry (LaRDR) on 9th May 2019. Managed by TRU, and established with a pilot in 2016, LaRDR is now open at 17 sites across Australia, collecting data on non-Hodgkin and Hodgkin lymphoma and chronic lymphocytic leukaemia. Several collaborative research projects are underway using the registry, and more are planned.
Further information is available at lardr.org.
Myeloma and Related Diseases Registry participates in Myeloma UK patient workshop
As part of the Myeloma Patient Registry Stakeholder Workshop, held in London on 5th April 2019, the MRDR was pleased to be invited to provide information on our experience in developing a myeloma registry in Australia and New Zealand. Myeloma UK is scoping out a project to establish a registry, and MRDR Research Fellow Dr Elizabeth Moore recorded a narrated slide presentation to share at the workshop, covering research and operational aspects, including governance and funding.
Recruitment completed for the RATIONAL trial
Thanks and congratulations to all patients, site staff and investigators involved in the RATIONAL study. The study completed recruitment in March 2019.
Funded by the National Blood Authority, RATIONAL is a phase II, randomised controlled feasibility trial comparing the efficacy of prophylactic immunoglobulin with prophylactic antibiotics in patients with acquired hypogammaglobulinemia secondary to haematological malignancies. Patients enrolled in the trial are randomised to receive either immunoglobulin replacement therapy, or prophylactic oral antibiotics. The primary outcome is adherence to and acceptability of assigned treatment, with secondary outcomes including infectious outcomes, admissions, costs and quality of life being followed for 12 months.
More information is available at anzctr.org.au/Trial/Registration/TrialReview
2,500th patient recruited to the MRDR
Among its registry projects, TRU manages the Myeloma and Related Diseases Registry (MRDR). The MRDR, chaired by Professor Andrew Spencer of the Alfred Hospital, captures and analyses data on patterns of treatment and variation in patient outcomes (both survival and quality of life) for patients with multiple myeloma and monoclonal gammopathy of undetermined significance (MGUS). The first patients were enrolled in 2013. More than 30 sites from Australia and New Zealand are already participating and additional sites are continuing to join.
Congratulations to Christchurch Hospital in New Zealand who recruited the 2,500th patient!
Ultimately, MRDR data will help clinicians and hospitals to provide the best possible care to people with myeloma and MGUS and allow evaluation of the translation of advances in therapy (such as the introduction of new targeted therapies) into long-term outcomes, outside the setting of clinical trials.
400th patient recruited to the TREATT trial
TREATT is a randomised trial of tranexamic acid (TXA, an anti-fibrinolytic agent to prevent clot breakdown) or placebo to investigate whether TXA prevents bleeding in adult patients with a haematologic malignancy and profound thrombocytopenia.
TREATT is a collaboration between the NHS Blood and Transplant in the UK and Monash University. The Australian arm of the trial is funded by the NHMRC and the Australian and New Zealand Society of Blood Transfusion. Ten Australian hospitals are currently participating.
More information is available at treatt.org
Congratulations to the team at Peter MacCallum Cancer Centre in Melbourne who recruited the 400th patient!
TRU at the American Society of Hematology conference
Prof Erica Wood, A/Prof Zoe McQuilten and Dr Lucy Fox represented TRU at the recent ASH meeting in San Diego.
Data from the UK-Canada-Australian REDDS pilot study of red cell transfusion in patients with myelodysplasia were presented as an oral abstract by Dr David Bowen, with Zoe McQuilten as a co-author. See abstract 527: A Feasibility Randomized Trial of Red Cell Transfusion Thresholds in Myelodysplasia. Available at: https://ash.confex.com/ash/2018/webprogram/Paper112949.html
Lucy Fox and Erica Wood were co-authors on a poster presenting data from the Melbourne Genomics Health Alliance Bone Marrow Failure Flagship. See abstract 3867: Providing Diagnoses in Bone Marrow Failure Syndromes through Multimodal Comprehensive Genomic Evaluation and Multidisciplinary Care: The Melbourne Genomics Health Alliance Bone Marrow Failure Flagship. Available at https://ash.confex.com/ash/2018/webprogram/Paper114410.html
Zoe and Erica also met with colleagues for a steering committee meeting of the Asia-Pacific Myeloma and Related Diseases Registry, and made a presentation to the Myeloma Canada Research Network.
ICAN study – Immunoglobulin use in Chronic lymphocytic leukaemia And Non-Hodgkin lymphoma – funded by the National Blood Authority
Patients with certain types of non-Hodgkin lymphoma (NHL), including chronic lymphocytic leukaemia (CLL), are at increased risk of infections due to the effects of their disease and treatment on levels of protective antibodies. Immunoglobulin therapy (made from donated plasma) is commonly used to prevent infections by replacing protective antibodies in this patient group, and is one of the most common and fastest growing indications in Australia. Availability of novel treatments with profound and protracted immunosuppressive effects will likely mean that immunoglobulin demand will continue to grow.
However, there is limited evidence from clinical studies to guide clinicians on which patients are most likely to benefit, when to commence treatment, or for how long. The role of other strategies to prevent infection, such as prophylactic antibiotics and immunisations, have not been well studied.
Working with the Australian Lymphoma and Related Disease Registry and the newly established CLL Registry, both managed by TRU, the ICAN project will collect data on current immunoglobulin replacement and other infection prevention strategies, as well as patient-centred outcomes, in Australian patients with CLL and NHL. In a subgroup of participants, the study will also collect serial blood samples for novel immune-profiling studies.
The ICAN investigators are Zoe McQuilten, Erica Wood and Neil Waters from Monash University’s Transfusion Research Unit; other Monash University colleagues Orla Morrissey, Stephen Opat, Jonathan Wong and John Zalcberg; Philip Crispin from Canberra Hospital; Stephen Mulligan and Kyle Crassini from Royal North Shore Hospital/University of Sydney; and Monica Slavin and Benjamin Teh from Peter MacCallum Cancer Centre/ NHRMC National Centre for Infections in Cancer.
NHMRC success with the ASPREE-CHIP study for TRU’s A/Prof Zoe McQuilten and colleagues
A/Prof Zoe McQuilten and colleagues have been awarded $1.416 million for a study of clonal haematopoiesis of indeterminate potential (CHIP) in the elderly. This project will investigate CHIP in participants from the ASPirin in Reducing Events in the Elderly (ASPREE) study, a randomised controlled trial of daily low-dose aspirin or placebo in over 19,000 elderly but otherwise well individuals (www.aspree.org).
CHIP is the presence of a haematological malignancy-associated somatic mutation in blood or marrow without evidence of haematological disease. CHIP is increasingly common with increasing age, and is associated with risk of haematological cancers, coronary heart disease and ischemic stroke. Mouse models support a causative link between CHIP and development of atherosclerosis. However, very few studies have been performed in humans to date, and fundamental gaps remain in our understanding.
Using ASPREE trial data on incident haematological cancers, cardiovascular events, stroke, dementia and all-cause mortality, and biobanked samples at baseline and 3 years, we will study CHIP and its clinical consequences. We will also investigate the role of inflammation, and whether reducing inflammation through use of low-dose aspirin alters incidence, progression and consequences of CHIP.
Investigators on the NHMRC-funded grant are: Zoe McQuilten and Erica Wood from Monash University’s Transfusion Research Unit, other Monash University colleagues David Curtis, Rory Wolfe, Paul Lacaze, John McNeil, Andrew Murphy, Moeen Riaz, Nick Wong and Robyn Woods, and Robert Sebra Icahn School of Medicine at Mount Sinai, USA.
CLIP-II study of frozen platelets funded by NHMRC
CLIP-II is a phase III, multicentre blinded randomised controlled clinical non-inferiority trial of cryopreserved platelets versus conventional liquid-stored platelets for the management of post-cardiac surgical bleeding.
The CLIP-II team have been awarded $1.825 million by the NHMRC for the project.
CLIP Chief Investigator Dr Michael Reade leads a team of experts from around Australia and New Zealand, including Drs Zoe McQuilten and Erica Wood of the Transfusion Research Unit at Monash University.
Sharing Massive Transfusion Registry data with transfusion practitioners
Dr Rosemary Sparrow and Mrs Helen Haysom provided an update on the Massive Transfusion Registry as part of the Blood Matters Transfusion Forum in November.
The MTR now has data on over 8000 cases of critical bleeding from 29 hospitals across all clinical contexts, including trauma, obstetrics, surgery and gastrointestinal haemorrhage. An additional 20 sites are in the process of joining. The MTR is proving valuable for both research and for clinical practice improvement and benchmarking, and data are provided to participating sites via hospital data reports, letters and presentations. Results have been presented this year at the BLOOD 2018 conference in Brisbane, the International Society of Blood Transfusion congress in Toronto, and the AABB conference in Boston, as well as through several publications.
Current MTR research projects include analyses of:
If you have an idea for a project using MTR data, please get in touch with us: email@example.com
TRU at BLOOD 2018 conference
The Transfusion Research Unit was well represented at the recent Australian and New Zealand annual haematology and transfusion conference, BLOOD 2018, held in Brisbane. Staff and collaborators contributed two invited presentations, nine oral abstracts and seven posters. Congratulations to Dr Elizabeth Moore who received a poster prize.
TRU’s exhibition booth was a great opportunity to profile the Unit’s research and hold informal discussions with delegates. Breakfast meetings for the lymphoma and myeloma registries included presentations by invited speakers as well as review of registry data and project updates, and were well attended by clinicians and industry partners.
Dr Rosemary Sparrow awarded honorary membership of BEST Collaborative
Dr Rosemary Sparrow represented the Transfusion Research Unit team at the Biomedical Excellence for Safer Transfusion (BEST) Collaborative meeting that was held in Newport, Rhode Island (USA). BEST is an international research group, with scientific membership drawn from individuals recognised as having high-level accomplishments in transfusion research. TRU is an institutional member of BEST.
Congratulations to Rosemary for being made an honorary member for her previous and ongoing contributions to BEST and transfusion research, and becoming the first Australian to be awarded this honour.
TRU at AABB 2018 meeting
The Transfusion Research Unit was well represented at the AABB (formerly American Association of Blood Banks) annual meeting in Boston, USA. TRU team members contributed two posters and one oral abstract. Congratulations to PhD student Phil Kiely who received a top poster award for 'Modelling the transfusion-transmission risk of West Nile Virus in Australia associated with travelling blood donors'. Dr Rosemary Sparrow (left) presented a poster on the use of group O negative red cells in critical bleeding, on behalf of the Massive Transfusion Registry investigators.
Prof Erica Wood and Dr Zoe McQuilten co-authored an abstract on an Australian clinical trial of cryopreserved platelets in cardiac surgery, presented in the Plenary Oral Abstract Session for the top six submitted abstracts.
ASPREE trial published in New England Journal of Medicine
The results of the Australia-US ASPREE trial of low-dose aspirin in the elderly have been published in three concurrent papers in the New England Journal of Medicine.
One of the publications focussed on clinically significant bleeding. Prof Erica Wood of Monash University’s Transfusion Research Unit chaired the ASPREE clinically significant bleeding panel and was one of the authors on this paper.
See: McNeil JJ, et al. Effect of aspirin on cardiovascular events and bleeding in the healthy elderly. N Engl J Med. 2018 Oct 18;379(16):1509-1518. doi: 10.1056/NEJMoa1805819.
More information is available at: aspree.org/aus
Frailty-stratified, randomised controlled Bayesian adaptive trial of bortezomib versus lenalidomide in transplant-ineligible myeloma – the FRAIL-M study – funded by the Medical Research Future Fund
TRU’s Zoe McQuilten, Erica Wood, Elizabeth Moore and Cameron Wellard are investigators on the FRAIL-M study. The project will utilise the infrastructure of the Myeloma and Related Diseases Registry (MRDR) managed by Monash University’s Transfusion Research Unit. Professor Andrew Spencer (Monash University/Alfred Hospital) is Chief Investigator A on FRAIL-M and chair of the MRDR Steering Committee. Funding of $1.642 million has been awarded by the MRFF through its call for applications for “Low Survival Cancers and Diseases”.
Current Australian guidelines recommend that up-front therapy for transplant-ineligible myeloma patients should be either bortezomib- or lenlidomide-based regimens, with dosing adjusted according to patient frailty. However, adapting treatment dose according to frailty assessment has not been tested in a clinical trial.
FRAIL-M is a stratified randomised controlled trial to identify which competing treatment options are more appropriate in transplant-ineligible myeloma patients according to frailty status. Participants will be stratified into three groups (fit, intermediate-fit or frail) based on a standardised and validated frailty assessment, and randomised to a bortezomib- vs a lenalidomide-based regimen, with dosing adjusted according to frailty stratum. A Bayesian Optimal Phase II design will allow us to jointly monitor efficacy and toxicity, with further adjustments if individual treatment arms appear either too toxic or ineffective.
The FRAIL-M investigators are Andrew Spencer, Zoe McQuilten, Hang Quach, Peter Mollee, Erica Wood, John Reynolds, Ruth Hubbard, Richard De Abreu Lourenco, Cameron Wellard, Elizabeth Moore, Madeleine King, Steve Roach and Tracy King.
TRU congratulates Dr Nicholas Saadah who was awarded his PhD from the University of Leiden on 11 September 2018
Nic’s thesis was entitled: Hemovigilance, heterogeneity, and hyperfibrinolysis; evaluating the Netherlands’ switch to solvent/detergent plasma". His studies focussed on the changing use of plasma transfusions at national level in The Netherlands, and internationally, including an analysis of information from the ISTARE database of the International Haemovigilance Network. He was supervised by Prof. J.G. (Anske) van der Bom and Dr Martin Schipperus.
Nic was an academic visitor to Monash during 2017, working on a range of transfusion research projects. Prof Erica Wood from the TRU was a member of the PhD defence panel for the University of Leiden.
The Asia-Pacific Myeloma and Related Diseases Registry has opened to recruitment at Samsung Medical Center in Korea!
The APAC MRDR is a clinical registry which collects health information on patients diagnosed with multiple myeloma, or related diseases such as plasmacytoma, plasma cell leukaemia, or monoclonal gammopathy of undetermined significance (MGUS) living in the Asia-Pacific region.
The registry was established in 2018 by Monash University, and in collaboration with participating pilot hospitals in Korea, Singapore, Hong Kong and Taiwan, and is affiliated with the Australian and New Zealand Myeloma and Related Diseases Registry (ANZ MRDR).
The APAC MRDR aims to provide health care services and researchers an important resource for investigating the best possible treatment options for people with these conditions. The Registry will tell us about:
The Registry will also form a network of clinical experts from the Asia-Pacific region with a special interest in myeloma and related diseases.
More information on the Australian and New Zealand MRDR and its projects is available at: mrdr.net.au
TRU awarded $1.75m for DIAAMOND PROJECT
Erica Wood and Zoe McQuilten of Monash University’s Transfusion Research Unit, in collaboration with colleagues from across Australia, have been awarded $1.75 million funding from the Medical Research Future Fund for the DIAAMOND project: Diagnosis of aplastic anaemia, management, and outcomes utilising a national dataset.
Aplastic anaemia is a life-threatening condition, and the only curative option, haematopoietic stem cell transplant, is not available to many patients. This study of avatrombopag (a second-generation thrombopoietin receptor agonist) in patients with treatment-naïve or relapsed/refractory aplastic anaemia, builds on promising results from recent studies with eltrombopag. The trial, using a novel Bayesian adaptive design, will be conducted using the Aplastic Anaemia Registry and is anticipated to open in early 2019.