Home > Patient Burden and Real-World Management of Chemotherapy-Induced Myelosuppression: An Expert Interview with Dr Robert S Epstein
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Patient Burden and Real-World Management of Chemotherapy-Induced Myelosuppression: An Expert Interview with Dr Robert S Epstein

Authors: Robert S Epstein, Epstein Health, LLC, Woodcliff Lake, NJ, USA Published Online: August 6th 2020


Chemotherapy-induced myelosuppression is one of the most common side effects of chemotherapy treatment, and is a result of the cytotoxic effect of chemotherapy on actively dividing hematopoietic stem and progenitor cells in the bone marrow. This may result in anaemia, neutropenia, thrombocytopenia, and/or lymphopenia, which can cause life-threatening infections and a range of symptoms that severely impact on the patient’s quality of life.1 Current management strategies include dose modifications of their chemotherapy treatment and the use of various rescue strategies to stimulate white cells (growth factors, such as granulocyte colony-stimulating factors), replenish red blood cells and platelets (transfusions); or stimulate red blood cell precursors (growth factors, such as erythropoiesis-stimulating agents).2–4 However, the patient burden of chemotherapy-induced myelosuppression has not been well documented.

In an expert interview, Dr Robert S Epstein discusses his recently published study on the real-world impact on patients of chemotherapy-induced myelosuppression.5

Q. What are the unmet needs and what challenges do physicians face when deciding a treatment strategy for patients with chemotherapy-induced myelosuppression?

Reactive rescue treatments are available for chemotherapy-induced myelosuppression but they all have side effects associated with their use. All the guidelines recommend waiting for the blood counts to be sufficiently low before using any of them.3,4 For example, in the case of erythropoiesis stimulating agents or blood transfusions, we usually wait until the haemoglobin is below 7–8 g/dl before using them because we are worried about their side effects like thrombotic events or transfusion reactions. As a result, the use of these agents is often more of an art than a science – balancing risks and benefits at the individual patient level.

Q. How important is patient-reported outcomes when deciding an appropriate treatment strategy?

They are very important as they give us context around the interpretation of blood counts and gauging what to do next. For example, if the patient has chemotherapy-induced anaemia but is asymptomatic it is possible to wait until the haemoglobin is below 7–8 g/dl before treating the patient according to various guidelines. However, if they are symptomatic or have cardiovascular disease and require the oxygen carrying capacity of red blood cells, guidelines suggest we may consider treating more aggressively if the haemoglobin is above 7-8 g/dl but below 10 g/dl. Patient-reported measures may add to the information we need to make treatment decisions.

Q. Please can you describe the aims and design of your patient survey that has been published in Advances in Therapy?

Many clinicians have felt they have had sufficient rescue agents to address myelosuppression but several members of our research team felt that this should be investigated from the patient’s perspective and wanted to explore the challenges faced by patients. We therefore conducted an online survey of around 300 patients with breast, lung or colorectal cancer who had at least one episode of chemotherapy-induced myelosuppression in the last year.

Q. What were the major findings of the study?

The top line finding was that 9 out of 10 patients (88%) said that the overall impact on their life was moderate to major, a higher figure than we were expecting. Furthermore, this was pervasive, including impacts on activities of daily living at home or out of the home, relationships with spouses and children, feelings of social isolation because of fear of infection, and depression. We also found that two-thirds of patients had to have chemotherapy dose reduction, delay or discontinuation because of myelosuppression. Around two-thirds of patients stated that they were happy to have received some treatment but 30% felt there had been a delay in getting to that point, and they had been miserable during the delay. Around 30% of patients said they hadn’t realised before initiation of chemotherapy that this could be an issue for them. So there appears to have been important communication challenges before starting treatment, but also along the treatment course as well. Caregiver issues were also an interesting area of concern for patients. They highlighted the fact that they often did not want to, or could not, drive themselves to centres for infusions and that level of dependency on caregivers was an issue in multiple ways.5

Q. How can these findings inform treatment strategies for chemotherapy-induced myelosuppression in the future?

Firstly, it is important to make patients more aware before initiating treatment that myelosuppression is a possible side effect of chemotherapy and explain it in a way that a patients will understand, mentioning symptoms such as fatigue and shortness of breath for anaemia, infection or fever for neutropenia, bruising, dark urine, blood in stool or bleeding gums for thrombocytopenia. Likewise, it is important to encourage patients to speak up if they are having these problems and in a timely manner because it can inform reactive treatment decisions. Lastly, in the light of the pandemic, many strategies have been implemented to avoid infection, such as having single rooms for infusions rather than infusion suites and home infusions. Infusions have even been carried out in patient’s cars. The important point is to ease patients’ fears of side effects like infection while addressing the discomfort brought upon by chemotherapy-induced myelosuppression.


  1. Barreto JN, McCullough KB, Ice LL, et al. Antineoplastic agents and the associated myelosuppressive effects: a review. J Pharm Pract. 2014;27:440–6.
  2. Kuter DJ. Managing thrombocytopenia associated with cancer chemotherapy. Oncology (Williston Park). 2015;29:282–94.
  3. Aapro M, Beguin Y, Bokemeyer C, et al. Management of anaemia and iron deficiency in patients with cancer: ESMO Clinical Practice Guidelines. Ann Oncol. 2018;29:iv96–iv110.
  4. Klastersky J, de Naurois J, Rolston K, et al. Management of febrile neutropaenia: ESMO Clinical Practice Guidelines. Ann Oncol. 2016;27:v111–v8.
  5. Epstein RS, Aapro MS, Basu Roy UK, et al. Patient burden and real-world management of chemotherapy-induced myelosuppression: Results from an online survey of patients with solid tumors. Adv Ther. 2020;37:3606–18.


Author profile: Dr Robert S Epstein is an epidemiologist with extensive expertise in pharmacoeconomics and health outcomes research, having served in academia and public health prior to joining the private sector. He is currently the chief executive officer and co-founder of Epstein Health LLC and has provided strategic consultancy services to life sciences companies since June 2013. From 2010 to 2012, Dr Epstein was president of Medco-UBC, a 2,400-person global organisation conducting personalised medicine, health economics, drug safety, outcomes and comparative effectiveness research for diagnostic, medical device and biopharmaceutical firms. From 1997 to 2010, he served as Medco’s chief medical officer where he led formulary and clinical guideline development, drug information services, personalised medicine program development and client analytics and reporting. Dr Epstein is the former president of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR), and has served on the boards of directors of the Drug Information Association (DIA) and the International Society of Quality of Life. He has also served on the Centers for Disease Control and Prevention’s Evaluation of Genomic Applications in Practice & Prevention Stakeholder Committee and the Agency for Healthcare Research and Quality Centers for Education and Research on Therapeutics Committee. He has published more than 100 peer-reviewed medical papers and book chapters, and serves as a reviewer for multiple medical journals. Dr Epstein serves on the board of directors of Illumina, Inc., Fate Therapeutics, Inc., Decipher Biosciences, and Veracyte, Inc. as well as Proteus Digital Health, Inc., a privately held digital healthcare technology company. Dr Epstein received his BSc in Biomedical Science and his MD from the University of Michigan, Ann Arbor, MI, US. In addition, he received his MSc and specialty training and certification in Preventive Medicine from the University of Maryland, College Park, MD, US.

Disclosures: Dr Epstein has no conflicts of interest to declare in relation to this article.

Support: Commissioned, edited and supported by Touch Medical Media, who commissioned the interview in liaison with W2O Group. Writing assistance was provided by Katrina Mountfort from Touch Medical Media.

Published: 6 August 2020


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