BSH Crucible Prize
The Crucible Prize is an innovative trainee-led initiative with a £1000 prize. The prize is open to doctors in training, nurses and other healthcare professionals (at any stage of their career). The aim of this session is to encourage reflection on what we do as healthcare providers, and why we do it.
The best abstracts are selected for oral presentation at BSH ASM. Presentations are judged on the quality and originality of their content, presentation skills and response to questions.
The theme for the 2021 Crucible Prize was 'What lessons can haematology learn from others?' The winner was Amy Cooper.
On the day that you started on your training path to where you are today, you unwittingly started on a journey to learn the art of a new language – the language of medicine. From there you have now flourished into someone who uses this complicated language fluently and expertly. This skill took years, perhaps decades, to truly master. Medical jargon allows for efficient and clear discussions to be had between medical professionals and is critically important in this respect.
Now imagine that you are a person who has suddenly become very unwell and you are being immersed into this language with absolutely no prior training or interest in the subject. In fact, you wish you didn’t have to hear any of this. You feel really ill – the worst you’ve ever felt in your life - and just want to go to sleep and let this all wash over your head. You’re being hit with a barrage of scary-sounding words that you don’t know. They said “acute” - you think that means painful… or does it mean small? You don’t even have the energy to ask.
Linguistics theorises that jargon has two main effects, the first being to increase efficiency of communication. However, the second is to exclude outsiders who do not have prior understanding of the topic being discussed. This unfortunate side-effect is causing a rift between haematologists and patients which can only be bridged by conscious attempts to simplify communication based on our audience.
As haematological treatment options become more plentiful and advanced, the language we use becomes more complicated by the day. I propose that haematology learns this simple lesson of the unwanted effects of jargon to allow patients to join the conversation with us.
“It is trite law that in general a doctor is not entitled to treat a patient without the consent of someone who is authorised to give that consent”1. What constitutes valid, informed consent? In 2015, an obstetric case (Montgomery v Lanarkshire Health Board) redefined the law on consent and rejected previous case law and by inference medical paternalism2. Should haematologists take heed from this lesson learnt on the labour ward?
Within haematology, certain conditions carry such a high untreated morbidity and mortality that treatment is clinically imperative. However, equipoise exists in many circumstances, for example in the context of low-grade disorders, palliative treatment, or where research evidence does not provide clarity to guide therapeutic choice. Haematology is a field of rapidly evolving treatments, adding complexity to decision-making.
Fundamentally, informed consent requires knowledge of the indications and material risks of proposed interventions. The National Chemotherapy Board provides guidance3 relating to systemic anticancer treatment and has recently collaborated with Cancer Research UK to publish regimen-specific consent forms providing comprehensive patient information.
However, a thorough, signed consent form is simply one component. Patients vary in their desire for information and involvement in choices regarding their care, but evidence suggests one third of haemato-oncology patients are dissatisfied with their role in decision-making4,5. “Shared” or “supported” decision-making is increasingly recognised as crucial in promoting patient autonomy and enabling valid consent. Shared decision-making involves the collaborative consideration of evidence-based information alongside patients’ own priorities and values. It has been promoted by both the Royal Colleges of Obstetricians and Gynaecologists, and of Surgeons, in response to the Montgomery ruling6,7 and is highlighted in the current draft revision of GMC consent guidance. Should we follow in our surgical colleagues’ footsteps to focus training and resources to ensure that our patients have the best opportunity to be involved in decision-making?
- Re R (A Minor) (Wardship: Consent to Treatment)  Fam 11 CA
- Montgomery v Lanarkshire Health Board  UKSC 11
- UK Chemotherapy Board (2018). Consent for Systemic Anti‐Cancer Therapy (SACT). URL https://www.cancerresearchuk.org/sites/default/files/consent_guidance_doc_v2018-06.pdf
- Rood JA, Nauta IH, Witte BI, Stam F, van Zuuren FJ, Manenschijn A, Huijgens PC, Verdonck‐de Leeuw IM, Zweegman S. Shared decision‐making and providing information among newly diagnosed patients with haematological malignancies and their informal caregivers: Not “one‐size‐fits‐all”. Psycho‐Oncology. 2017 Dec;26(12):2040-7.
- Sullivan KH, Toscani M, Poretta T, Patzke C, Korattyil T, O’Malley D, Gonzalez V, Barone J, Evens AM. Shared decision making in cancer treatment: findings from a large survey of lymphoma patients and survivors. Abstract at: American Society of Hematology Annual Meeting; 2019 Dec 7-10; Orlando, FL
- Royal College of Obstetricians and Gynaecologists (2016). Providing Quality Care for Women: A Framework for Maternity Service Standards.URL https://www.rcog.org.uk/globalassets/documents/guidelines/working-party-reports/maternitystandards.pdf
- Royal College of Surgeons (2018). Consent: supported decision-making. A guide to good practice. URL https://www.rcseng.ac.uk/-/media/files/rcs/standards-and-research/standards-and-policy/good-practice-guides/new-docs-may-2019/rcs-_consent-supported-decision-making.pdf
Increasing awareness led to closer integration between clinical haematology and specialist palliative care (SPC) services. However, with <50% of haematology patients being referred to SPC, they remain less likely to receive palliative care or hospice services than those with other cancers. This is despite haemato-oncological patients having a substantial illness burden, poor quality of life (QoL), and aggressive care at the end of life with greater likelihood of dying in hospital.
Many barriers to timely SPC referrals have been identified, including unpredictable disease trajectories, inaccessibility to often fragmented services and ill-conceived physician perceptions. However, haematology can learn one thing from oncologists and palliative care physicians – that SPC is not synonymous with end-of-life care. Rather, SPC describes a philosophy of care, working with the other patients’ doctors to provide an extra layer of support. Aside from managing a variety of symptoms including: pain, mucositis, dyspnoea, fatigue, nausea, constipation, and diarrhoea, palliative care specialists also help patients engage in emotional work to facilitate coping, accepting, and planning.
The emphasis is on the well-being of patients and families at any point along their disease trajectory, regardless of their illness state. Mounting evidence suggests that earlier SPC input results in better outcomes. Three seminal, well-designed, randomised controlled trials have clearly established the feasibility and benefits of integrating early palliative care concurrently with standard oncology care2. Also, various other solid tumour studies documented: reduced symptom burden, better QoL, improved depression scores4,, higher mean scores for global health with better emotional and social functioning, and even significantly longer survival4,5. Early SPC referral decreased receipt of aggressive interventions at end-of-life and significantly lengthened duration of hospice care.
Haematology should take a page from Oncology and appreciate the scope for major research in this area. Hopefully, this will lead to earlier SPC intervention for our patients.
 Howell, D.A. et al., (2015) Variations in specialist palliative care referrals: findings from a population-based patient cohort of acute myeloid leukaemia, diffuse large B-cell lymphoma and myeloma. BMJ Supportive & Palliative Care. 5:496–502.
 Thienprayoon, R. and LeBlanc, T. (2015) When and why should patients with hematologic malignancies see a palliative care specialist? American Society of Hematology – Hematology 2015. 479-483.
 Jacobsen, J. et al., (2011) Components of early outpatient palliative care consultation in patients with metastatic non-small cell lung cancer. J Palliat Med. 14(4):459-464.
 Bakitas, M. et al. (2009) Effects of a palliative care intervention on clinical outcomes in patients with advanced cancer: The Project ENABLE II randomised controlled trial. JAMA. 302(7):741-749.
 Temel, J.S, et al. (2010) Early palliative care for patients with metastatic non-small-cell lung cancer. New England Journal of Medicine. 363(8):733-742.
 Rugno, F.C., Paiva, B.S. and Paiva, C.E. (2014) Early integration of palliative care facilitates the discontinuation of anticancer treatment in women with advanced breast or gynecologic cancers. Gynecol Oncol. 135:249-254.
‘The mind is such a fragile thing. How can someone begin to heal physically if they are mentally broken?’
The haematology landscape has undergone revolutionary transformations; however, we often neglect the mental and social-health needs of our patients and carers throughout their journey from diagnosis to recovery and beyond.
The scale of the problem is significant. Anxiety and depression are 3-4 times more likely where physical health issues co-exist. 30% of patients with a long-term condition, including many with haematological conditions, will have a mental health problem1 and they have poorer outcomes when they have a blood cancer2. Evidence supporting psychological services within haemato-oncology is summarised in NICE guidance3 and more recent research4. Further, specialist psychiatric care is necessary to manage complex treatment effects and drug interactions, alongside other issues5.
Many haematological services already attempt integrated ‘Mind and Body’ care, with psychology or counselling services available for some groups.
Is this sufficient? Evidence suggests more is needed to care for mental and physical needs equally and simultaneously, developing integrated Mind and Body service ‘whole person’ models.
This necessary shift would see routine mental health and wellbeing screening for all patients on every haematology pathway, together with clinically appropriate, personalised responses.
An enhanced Mind and Body programme has potential to transform patient experience, enabling optimised psychological, social, and clinical care concurrently.
A compelling case supports transformation of physical, mental, and social care delivery across haematology pathways, but requires skills derived from a team of specialist psychiatrists, psychological therapists, and social workers. This multi-professional collaboration embedded within routine haematological care offers a unified focus to benefit patient experience, outcomes, and wellbeing.
Haematology has much to learn from others and embedding a Mind and Body team within haematology will create a culture shift, departing from conventional compartmentalised care to a truly holistic model.
- Albert L. Siu, MD, MSPH; and the US Preventive Services Task Force (USPSTF). Screening for Depression in Adults. USPSTF Statement. JAMA. 2016;315(4):380-387. doi:10.1001/jama.2015.18392
- Kisely S, Crowe E, Lawrence D (2013) Cancer-related mortality in people with mental illness. JAMA Psychiatry
- Improving Supportive and Palliative Care for Adults with Cancer (2004) NICE
- Patient Need: Mapping the needs, experiences and outcomes of people affected by blood cancer (April 2015), accessed https://bloodwise.org.uk/mail/files/patient_need_phase_one_findings_report.pdf on 24/11/15
- Hodgkiss A, (2016) Biological Psychiatry of Cancer and Cancer Treatment. OUP.
The theme for BSH Virtual 2020 was "What lessons learnt during the COVID-19 pandemic should change future practice in haematology?” The winner was Joshua Bomsztyk.
As we sit in the exacting classroom of COVID-19, with a ferocious and uncompromising viral teacher, the question of ‘lessons learnt’ might be considered premature. As we embark on a probable second wave and beyond, a particular lesson has stood out and should remain embedded in our psyche.
Undeniably, patient safety is the primary focus of our care. Haematologists are used to the idea of finely balancing the risk of treatment versus undesirable consequences. This pervades every decision surrounding chemotherapy, transfusion or anticoagulation. The benefit vs. risk conundrum.
The risk of a respiratory virus is also well recognised as the BSH guideline would attest[i]. With a mortality of up to 33%[ii] in the context of allogeneic transplant, the impact of community acquired respiratory viruses (CARV) should not be underestimated.
However, the concept of social distancing and hospital avoidance have not been considered as preventative strategies prior to COVID-19. The delivery of haematology services had shifted to large volumes of patients attending outpatient clinics and day units for review and treatment.
The lesson of COVID-19 ought to be a focus on hospital avoidance. The design of newer therapies should focus on this strategy. The development of oral and subcutaneous therapies should not be merely seen as a convenience but as a patient safety issue. COVID-19 has reminded us that hospitals are dangerous places. The challenge is to progress these novel working styles for the sake of patient safety.
[i] Fiona L Dignan et al. BCSH/BSBMT/UK clinical virology network guideline: diagnosis and management of common respiratory viral infections in patients undergoing treatment for haematological malignancies or stem cell transplantation. April 2016
[ii] Versluys AB, Boelens JJ. Morbidity and Mortality Associated With Respiratory Virus Infections in Allogeneic Hematopoietic Cell Transplant: Too Little Defense or Harmful Immunity? Front Microbiol. 2018;9:2795. Published 2018 Nov 21. doi:10.3389/fmicb.2018.02795
Limitations of treatment and escalation decisions on admission during the COVID-19 outbreak were a pertinent discussion with haematology patients undergoing chemotherapy and urgent stem cell transplants. During the peak of COVID-19, it was made clear to patients that there may not be intensive care capacity if they become critically unwell. This was of course a very difficult conversation for patients and staff.
This poses the question – should we be having these conversations with patients during the treatment consent process, involving ceilings of care and DNA-CPR discussion and should it be routinely discussed to try and normalise the conversation to prevent the last minute panic when a patient deteriorates?
What would be the benefits of this?
- Prevent difficult conversations with family members should a patient deteriorate. Patient and families to have time to discuss wishes in the event of an emergency situation
- Staff caring for the patient would have a clear pathway for ceilings of care treatment and if a patient was to deteriorate out of hours, there would already be a plan of care in place
What would be the disadvantages?
- Taking hope away from patients
- So many patient cohorts – one rule doesn’t fit all
What process would need to take place?
- Absolute agreement by consultant teams
- CNS support
- Ward staff support
- Clear documentation coherent with inpatient and outpatient communication
- Consistency in care
Conversations with patients about haematological conditions tend to air on the way of positivity and often, we avoid the elephant in the room – Am I going to die from this disease? Often, patients look to us as health care professionals to be frank and truthful about the outlook and we believe that this is an area of practice that as a group of haematology professionals we can all learn from for future practice.
The COVID-19 pandemic has disrupted clinical research worldwide, affecting up to 732,176 participants across England. Many are haemato-oncology patients, who were shown to be particularly vulnerable to COVID-19 infection, and in need of shielding. Self-isolation and physical distancing measures inevitably led to most clinical trials halting recruitment, with actively enrolled patients either having their treatment modified, paused or stopped altogether due to difficulties in continuing under lockdown conditions. Various guidelines were issued at unprecedented speed,, to help stakeholders better manage trials during this pandemic – striking a balance between patient safety, maintaining trial integrity, and ensuring adherence to good clinical practice standards.
Remote healthcare access and service delivery was encouraged. Trial sponsors adapted protocols to facilitate remote assessment via telephone/video conferencing and performing investigations outside the site and in the patient’s local community with more flexible test scheduling. Despite reluctance in allowing patients to self-administer subcutaneous chemotherapy at home, investigational medicinal products were sometimes delivered through community facilities like Healthcare at Home, with courier services used to distribute oral medications, including cytotoxics. Wider adoption of electronic case report forms (eCRF) replacing paper versions enabled faster data collection, preserving data integrity through more efficient data cleaning and improved data management processes.
Although meant to be temporary, many physicians and patients hope that some of these adaptations continue beyond the pandemic. Also, longstanding concerns that clinical trial populations insufficiently resemble real-world patients can be addressed by having more inclusive patient-centric study designs that better reflect real-world treatment provision and maximise translatability to routine practice.
The risks posed to haematology patients by COVID-19 demand rapid structural changes in healthcare delivery, with positive trends encouraged to continue long-term. Given the considerable uncertainty around duration of restrictions, an immediate critical reassessment of trial methodology is essential to ensure resilience in clinical research and optimise patient-centred care.
 National Institute for Research Health (NIRH) Annual Statistics. Available from: https://www.nihr.ac.uk/about-us/our-contribution-to-research/research-performance/annual-statistics.htm [Accessed 10th September 2020]
 The Association of the British Pharmaceutical Industry. Clinical Trials – How the UK is Researching Clinical Trials of the Future (Autumn 2019). Available from: https://www.abpi.org.uk/media/7607/rmi-0128-0919-clinical-trials-report.pdf [Accessed 10th September 2020]
 Williamson E.J., Walker A.J., Bhaskaran, K. et al. Factors associated with COVID-19-related death using OpenSAFELY. Nature. 2020; 584: 430–436. Available from: https://doi.org/10.1038/s41586-020-2521-4.
 Van Dorn, A. COVID-19 and readjusting Clinical Trials. The Lancet. 2020; 396(10250):523-524.
 European Medicines Agency and Heads of Medicines Agency. Guidance on the Management of Clinical Trials During the COVID-19 (Coronavirus) Pandemic. Version 3 (28/04/2020) Available from: https://ec.europa.eu/health/sites/health/files/files/eudralex/vol-10/guidanceclinicaltrials_covid19_en.pdf [Accessed 28th August 2020].
 European Medicines Agency. Points to consider on implications of Coronavirus disease (COVID-19) on methodological aspects of ongoing clinical trials. EMA/158330/2020 Rev. 1 3 (26 June 2020). Available from: https://www.ema.europa.eu/en/documents/scientificguideline/points-consider-implications-coronavirus-disease-covid-19-methodologicalaspects-ongoing-clinical_en-0.pdf. [Accessed 28th August 2020].
 Medicines and Healthcare products Regulatory Agency. Managing Clinical Trials During Coronavirus (COVID-19). Available from: https://www.gov.uk/guidance/managing-clinicaltrials-during-coronavirus-covid-19 [Accessed 28th August 2020].
 Doherty G.J., Goksu M. and de Paula B.H.R. Rethinking cancer clinical trials for COVID-19 and beyond. Nature Cancer. 2020; 1:568–572. Available from: https://doi.org/10.1038/s43018-020-0083-x
Responding to the difficult and challenging time that COVID-19 pandemic has brought to healthcare provision, such as difficulties to provides regular treatments to high risks and vulnerable transfusion dependent thalassemia patients during the national lockdown.
It became extremely concerned for our several thalassemia patients with complex medical problems that are considered high risk to travel to Whittington hospital clinics or outpatient Units for their regular scheduled transfusion. Many of these high risk patients were living far away from Whittington hospital, from 2 to 100 miles away. This became a serious problem for the haematology team to maintain the regular essentials treatment for these patients at this critical National lockdown time when they were instructed to be shielding at home from March 2020.
After a department meeting to discussed this problem, a contingency plans was developed and agreed to consider providing 'Home transfusion' in order to deliver the essential health care needs for the high risk shielding thalassemia patients.
It became fully cleared that administering blood at home to the high risk thalassemia patients may be possible but can also be challenging.
Home transfusion service is very uncommon practice in the United Kingdom. However, this practice has been taking place in other countries. Therefore, COVID-19 pandemic triggered the needs to establish home transfusion services at Whittington hospital in order to provide the necessary treatment for our high risk thalassemia patients shielding at home.
Fourteen patients were identified and enrolled on the service. Since 24th March 2020 almost 200 home transfusions had taken place uneventfully. Patient’s care was maintained in their safe environment and the level of patient’s satisfaction remained high.
There is good evidence to support home transfusion service may change future haematology practice.
The COVID-19 pandemic brings challenges to delivering clinical care due to a range of factors: the generally immunocompromised patients, the shielding requirements, the need to protect those who had to receive treatment in-person and the staff who continued working. As the pandemic first gathered momentum, so did emails communicating clinical decisions to rationalise care. One unsurprising decision was for treatments which only prolong progression-free survival to be stopped. A comment from a colleague struck a chord, “well at least now we will know if as haematologists we are doing more harm than good”.
The concept of progression-free survival remains a double-edged sword. It has driven the innovation for new treatments in haematological diseases, of which a number are chronic. On the other hand, it has made haematologists focus on intervention that can be offered, rather than whether we should. Intervention almost always appear more attractive than not. And this is inevitably what is needed to prolong progression-free survival. Interventions include increasing the visits we expect patients to attend both in clinics and treatment appointments. The pandemic is an unintended opportunity for us to re-evaluate what the definition of haematological care and quality of life is, weighing up the additional time, costs, travel and exposure our patients take in order to access the care we recommend. We should pause and consider the impact even one routine clinic appointment has on our patients; a whole day often, waiting, when they could have been picking up their grandchildren, helping their sister run their new business, hiking up Snowdonia or caring for their spouse with metastatic cancer. Only time will tell, in our strive to offer more, whether we are doing good and not harm. Perhaps our patients’ attitudes to the rationale of the treatment they receive will evolve too.
The theme for the 2019 Crucible Prize was 'How can haematology change the world?'. The winner was Jennifer Darlow.
Haematology, more than many specialities, manages patients with compromised immune systems (e.g. neutropenic, bone marrow transplant patients). These patients regularly contract infections and consequently receive broad spectrum antibiotics. This includes carbapenems, particularly in the context of rising antimicrobial resistance and recent piperacillin-tazobactam shortages.
In the last decade the emergence and spread of carbapenem resistance presents antimicrobial dependent medicine with an unprecedented crisis. Globally, carbapenem resistance is rising – in parts of India, resistance rates are above 50%. Even in the developed world, carbapenem resistance is a growing problem, costing millions and leading to patient mortality. This is compounded by the fact that the new antimicrobial pipeline is sparse.
Widespread use of carbapenems contribute to this problem, by providing selection pressure that allows resistant organisms to flourish. Although haematology uses a relatively small quantity of antibiotics, its intensive use of carbapenems drives resistance disproportionately. Haematologists are therefore contributing to the aforementioned antimicrobial resistance situation. This is self-defeating in the medium-to-long term. If carbapenem resistance continues to be encouraged, immunosuppressive therapeutic interventions for haematological conditions, such as bone marrow transplant, will be unviable. Haematology is changing the world by driving a global crisis from which it will be difficult to return.
There are strategies that we can adopt to help avoid this nightmare scenario:
- With close collaboration with infection specialist colleagues, we can alter our prescribing habits to reduce antimicrobial resistance selection pressure whilst still treat patients appropriately.
- Wider and more consistent use of diagnostics, along with development and adoption of new diagnostics, can allow targeting of patients who require antibiotics and reduce unnecessary prescriptions for those that don’t.
- We can encourage investigation into the use of broad-spectrum antibiotic in haematological patients, to determine where it is necessary and where narrow therapy can be safely used instead.
Anaemia represents a huge global burden of disease. In 2010, around one third of the world’s population was affected by anaemia, with an estimated 50% of those cases attributable to iron deficiency. The majority of these cases of iron deficiency anaemia (IDA) occur in low- and middle-income countries. IDA primarily affects children and women, due to increased iron requirements secondary to growth, pregnancy or menstrual loss. Iron deficiency in children can affect both physical growth and cognitive performance, which will potentially have a lifelong impact. Thus, iron supplementation is recommended to ensure children reach their full potential. Indiscriminate use of iron supplementation, however, has been linked to increased mortality from malaria and other infections, notably diarrhoeal disease.
Consequently, there is a need to find a reliable diagnostic test to discriminate between IDA and anaemia secondary to other causes. While there are a variety of tests currently available to diagnose anaemia (including serum haemoglobin and ferritin), there remain challenges with their use and/or interpretation, especially in the low- and middle-income setting. Serum hepcidin has been posited as a potential alternative to current tests. Hepcidin plays a key role in iron regulation; low expression increases plasma iron concentrations while high expression decreases plasma concentrations. Recent research in both developed and developing countries has demonstrated that serum hepcidin concentration may potentially be able to differentiate IDA from anaemia of inflammation. Further research is still required before hepcidin becomes widely used as a marker for IDA. A reliable hepcidin point-of-care test still needs to be developed and there must be clinical trials comparing hepcidin to the currently used test., Hepcidin does, however, have the potential to represent the “holy grail” of iron deficiency diagnostics: the ability to identify children most likely to benefit from, and least likely to be harmed by, iron supplementation.
The United Nations’ Sustainable Development Goals (SDGs) set in 2015, include the attainment of universal health coverage, as well as a reduction in mortality from non-communicable diseases (NCDs) including cancer, and conditions relating to pregnancy and childbirth.1 These issues disproportionately affect Low and Middle Income Countries (LMICs) where 78% of deaths from NCDs2 and 99% of maternal deaths occur.3 Around 25% of these maternal deaths may be due to lack of blood transfusion4 and the provision of oncology treatment is often dependent on the availability of safe transfusion. There is huge inequality in blood availability worldwide. Donation rates in higher income countries are around 32 per 1000 population per year, compared to only 4 per 1000 population per year in lower income countries.5
Increasing investment and development of new technologies and drugs is significantly improving outcomes and survival in higher income countries. However, for these benefits to be realised, countries require functioning health systems with adequate access to reliable pathology and laboratory services. This is often lacking in lower resource settings.2 6 Haematology is an essential component of laboratory and pathology departments, enabling accurate diagnostics and safe transfusion.2
There is a need to advocate for the prioritisation of laboratory and transfusion services in health system planning and funding. Development of training programmes for biomedical scientists and post-graduate physicians will enable the improvement of laboratory and transfusion services to provide support for developing health systems.2 7 Haematology services can help reduce deaths from lack of blood, improve the process of accurate diagnostics and support developing oncology programmes. Haematologists can help through advocacy and support for training in low resource settings.
In order for the SDGs to be met, significant improvements in laboratory and transfusion services worldwide are essential. This is how haematology can help to change the world for the better.
- Publications UN. Sustainable Development Goals Knowledge Platform [Available from: sustainabledevelopment.un.org accessed 21/11/2018.
- Wilson ML, Fleming KA, Kuti MA, et al. Access to pathology and laboratory medicine services: a crucial gap. Lancet 2018;391(10133):1927-38. doi: 10.1016/s0140-6736(18)30458-6 [published Online First: 2018/03/20]
- WHO. Trends in maternal mortality: 1990 to 2015. Geneva, 2015.
- Bates I, Chapotera GK, McKew S, et al. Maternal mortality in sub-Saharan Africa: the contribution of ineffective blood transfusion services. BJOG : an international journal of obstetrics and gynaecology 2008;115(11):1331-9. doi: 10.1111/j.1471-0528.2008.01866.x [published Online First: 2008/10/01]
- WHO. Global status report on blood safety and availability 2016. Geneva, 2017.
- Bates I, Maitland K. Are Laboratory Services Coming of Age in Sub-Saharan Africa? Clinical Infectious Diseases 2006;42(3):383-84. doi: 10.1086/499368
- Horton S, Sullivan R, Flanigan J, et al. Delivering modern, high-quality, affordable pathology and laboratory medicine to low-income and middle-income countries: a call to action. Lancet 2018;391(10133):1953-64. doi: 10.1016/s0140-6736(18)30460-4 [published Online First: 2018/03/20]
“How can haematology change the world?! Devastatingly. It can take the world you thought you knew and give it back to you completely altered.
“After the diagnosis our world centred on one idea: Cure the leukaemia. ‘Chemotherapy will put it into remission, but cannot cure it – she needs a bone marrow transplant.’ (Let’s go for it!) ’She needs a plastic line in her vein.’ (Sure, whatever you say!) ’She needs weekly blood tests.’ (Okay, no worries.) ’She needs platelets twice a week.’ (But it’s a two-hour round trip!) ’We need to admit her for intravenous antibiotics.’ (What, again?) ’She needs to take these tablets – sorry there are so many.’ (Her mouth is so dry she can hardly swallow!) ‘She seems to really benefit from having you here.’ (I know, I’m trying.) ‘Look after yourself! She needs you well-rested.’ (I know. I’m trying.)
“What have you done to the woman I married? Who is this fragile person I sit with, accompanying to blood tests and clinic appointments, visiting daily on the wards? What are we fighting for, her and me? My love, whose hand I hold while she fades away on an intensive care unit? Was it all for nothing?”
When it comes to allogeneic transplantation, haematology changes the world utterly. And as haematology doctors, we offer a cure, but we do not elaborate on the price that will be paid.
We need to understand how haematology can change the world of every one of our allograft patients: our therapies have physical, social and emotional impact, and we must characterise these better in order to mitigate against them. We need to better predict outcomes to minimise failed allografts and ultimately, we need cleaner interventions so that we can leave our patients’ worlds unchanged.
Genomics is changing the medical world, and as haematologists we are at the forefront of driving this field from the bench to the bedside bringing genomic era diagnosis and treatments to our patients around the globe.
Haematology Malignancy Diagnostic Service (HMDS) laboratories provide ever-increasing insight into the genomic complexity of our patient’s cancers, and allow us to prescribe new medication targeted to their cancer, for example, FLT-3 inhibitors in acute myeloid leukaemia (1).
In low- and middle-income countries (LMICs) GeneXpert PCR machines are enabling molecular diagnosis of CML (2). Therefore, drug funding can be released for patients who would have a prognosis of a few years without treatment and transform them into a functional chronic disease patient, with a life expectancy of near normal.
Genomics is also advancing the field of blood transfusion, where haematology has traditionally had the bulk of its global impact, by utilising next generation sequencing to identify extended red cell phenotypes. This technology has the potential to increase the efficiency of identification of rare blood types within our blood banks and save more patients with complex allo-antibodies (3).
Gene therapy has recently been used with success in Haemophilia B, sickle cell disease and β-thalassaemia. Liver-directed gene therapy has reduced the frequency of bleeds (4) in Haemophilia B. Sickle cell and β-thalassaemia patients receiving lentiviral transformed autologous transplantation of a normally functioning β-globin gene have experienced cessation of sickle crises (5) and achieved transfusion independence (6). These treatments could become a ‘one-hit-wonder’ curative therapy, which may also be useful in LMICs where access to chronic treatment can be logistically fraught, and associated with a high cumulative cost.
As haematologists we are the clinical leaders of the genomic era, and by pushing the genomic medicine frontier we can help create a world with a lower burden of life-limiting and chronic disease.
- NIHR Horizon Scanning Research and Intelligence Centre, Quizartinib for FLT3-ITD positive acute myeloid leukaemia – second line, June 2016, http://www.jo.nihr.ac.uk/wp-content/uploads/migrated/Quyizartinib-June16.pdf, accessed 30/11/2018.
- The Max Foundation, The Max Foundation-Cepheid Collaboration Agreement Improving clinical outcomes by strengthening diagnostics capacity, September 2015, https://themaxfoundation.org/wp-content/uploads/reports/The-Max-Foundation-Cepheid-Collaboration-Agreement-2015.pdf, accessed 30/11/2018
- Wu et al, Blood group genotyping goes next generation: featuring ABO, RH and MNS, ISBT Science Series, March 2018, 13(3):290-297.
- Miesbach et al, Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B, Blood 2018 131:1022-1031.
- Ribeil et al, Gene Therapy in a Patient with Sickle Cell Disease, The New England Journal of Medicine, March 2017, 376:848-855.
- Thompson et al, Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia, The New England Journal of Medicine, April 2018, 378:1479-1493