Highlights from EHA

June 12, 2023

From 8th to 16th June, ALAN attended the European Hematology Association (EHA) 2023 congress, held for the hybrid, with a F2F part from 8th to 11th June and a virtual part from 14th to 16th June. We had the chance to be in Frankfurt for the F2F component as well as attending online when we came back.


Advancing Hematology Clinical Trials: Insights from the EHA-Patient Joint Symposium

At the 2023 EHA-Patient Joint Symposium, experts from various fields came together to address the pressing need for innovation in hematology clinical trials and the obstacles that hinder progress. The Symposium aimed to accelerate access to new treatments and improve patient outcomes by fostering collaboration among stakeholders.

Jan Geissler, a patient advocate and founder of Patvocates, emphasised the importance of patient-centric approaches in clinical trials, particularly in AML. Jan highlighted the need for rapid innovation and collecting patient-reported outcome data. Sharing data, involving patients in the research process, and designing trials that consider individual patient needs were stressed as crucial steps. Jan’s message emphasised the significance of placing patients at the heart of clinical research and promoting collaboration among all stakeholders.

Dr. Martin Kaiser, a myeloma specialist from the Institute of Cancer Research, UK, provided the physician’s perspective and discussed the ethical challenges of randomising patients to comparator arms that offer little improvement. Dr. Kaiser emphasised the importance of generating evidence through randomised clinical trials and cautioned against relying solely on unproven therapies. The difficulty in obtaining post-approval and real-world data was also highlighted. Dr. Kaiser presented an alternative trial design in myeloma that leveraged existing data as an external comparator arm, showcasing the potential to expedite drug development. Long-term planning and establishing trust among stakeholders were called for to ensure proper evidence generation.

Dr. Francesco Pignatti, representing the regulatory body of the European Medicines Agency (EMA), stressed the importance of collaboration among stakeholders. The speaker proposed solutions such as promoting collaboration and data sharing, utilising common ground and data to efficiently answer questions, and enhancing transparency in decision-making processes. Dr. Pignatti exemplified the use of alternate endpoints in diffuse large B-cell lymphoma (DLBCL) to expedite drug development and improve patient access.

Dr. Michelle Boyer, speaking from the industry perspective on behalf of Roche, highlighted the need for transformation in clinical trials to meet patient demands. They emphasised Roche’s commitment to patient-centric trials and strategies that utilise existing data and real-world evidence to expedite access to innovative treatments. Dr. Boyer discussed a pilot study conducted in collaboration with the U.S. Food and Drug Administration (FDA), where external comparator arm data were borrowed from an ongoing trial, aiming to reduce study time, costs, and patient exposure to standard care.

Dr. Camille Thomassin, from the French National Authority for Health, represented the health technology assessment (HTA) perspective.  Alternative trial designs and the use of real-world data were identified as key factors for enabling faster access to innovative treatments. Dr. Thomassin stressed the need for anticipating data availability and establishing registries before trial initiation. The successful chimeric antigen receptor therapy (CAR-T) cell registry initiative in France was highlighted as an example of generating comparative data after study initiation.

The session concluded with a panel discussion, where all stakeholders reiterated the significance of innovative clinical trials. Balancing risks, understanding the pros and cons of different trial designs, and providing checklists and guidance were highlighted. Early engagement and collaboration between stakeholders were seen as vital in de-risking trials and moving towards more innovative designs with a patient-centric focus.

What does this mean for patients?

The significance of patient-centric approaches and collaboration in hematology clinical trials is now widely accepted. The importance of innovation, use of alternative clinical trial designs, and generating evidence through collaboration with multiple healthcare stakeholders were also highlighted. This collective stakeholder approach that is now being pursued gives hope to patients with regards to expediting drug development, enhancing transparency, and meeting patient needs for innovative treatments and improved outcomes.

Empowering Patients and Enhancing Healthcare: Insights from the 2023 EHA-Patient Joint Symposium

During the 2023 EHA-Patient Joint Symposium, experts and patients engaged in discussions on the importance of gathering real-world data and incorporating patient input into healthcare decisions. The attendees stressed the significance of innovative approaches in clinical trials and post-approval studies, within the field of hematology.

Zack Pemberton-Whiteley, the Chair of ALAN and CEO of Leukaemia Care, provided insights into current patient reported outcome (PRO) approaches that can provide a better understanding of how treatments impact patients’ lives. He pointed out that existing methods often fail to capture crucial aspects that affect patients’ quality of life. To address this, he suggested actively involving patients in the decision-making process, both in research studies and real-world scenarios. The overarching goal being to simplify patient access to appropriate treatments, while empowering them to actively participate in their own healthcare decisions.

Prof. Marie Jose Kersten, from the Amsterdam University Medical Center, The Netherlands, also emphasised the importance of PROs and patient-reported experience measures (PREMs) in cancer treatment. PROs capture symptoms and toxicities directly reported by patients, providing insights into their quality of life. PREMs focus on the patient’s overall treatment experience, including preferences and concerns. The speaker stressed that collaborating with patients is crucial to improve these measures and ensure they align with patients’ needs. She highlighted the importance of monitoring and managing treatment toxicities using these measures, as they can improve overall care and the need for specific PROs and PREMs tailored to different diseases.

Dr. Camille Thomassin, representing the French National Authority for Health, delved into the importance of incorporating PROs into real-world datasets and using them for treatment evaluation. The speaker emphasised that patient organisations and professional societies should be consulted during the selection of suitable PRO tools and that a variety of validated PRO tools should be considered, rather than relying solely on a single questionnaire/measure. The example of PROs in myeloma was highlighted, which take into consideration various aspects such as symptoms, impairment, and functioning, ultimately generating comprehensive data on quality of life.

Connie Berlin, the patient engagement lead at Novartis, spoke about how patient preference studies can improve drug development. She discussed six important questions that guide this process:

  1. What aspects of the disease or treatment matter most to patients?
  2. How can we measure what matters to patients and understand its importance?
  3. How well does the new treatment address patient needs, and how can we use patient-centered outcomes to make decisions?
  4. How do we effectively communicate this information to patients?
  5. What valuable insights can we gather from methods like social media listening, online discussions, and patient interviews?
  6. Which disease and treatment factors are most significant in preference studies, especially when considering trade-offs between benefits and risks?

Patient preference studies enable better decisions about treatments, assessment of the risks and benefits, and communication of important information to regulators and healthcare decision-makers. Regulatory agencies and health authorities are showing more interest in patient preferences, and early discussions with them are crucial to including patient perspectives into drug development.

The session also featured a patient who shared personal experiences, concerns, and frustrations related to accessing new treatments. A significant issue highlighted was the delay in patient access to approved medicines due to prolonged price negotiations between countries.

What does this mean for patients?

Patients’ perspectives, needs, and preferences are gaining recognition in healthcare decisions. These will lead to better treatment choices, improved risk assessment, and effective communication. Regulatory agencies are now considering more and more patient preferences and input, while advocacy efforts are gaining ground in addressing pricing disparities and promoting equitable and patient-centered healthcare.

How to Enhance Patient Engagement through Effective Communication

Effective patient communication is essential in healthcare to facilitate shared decision-making. This session, led by Dimitrios Kyrou and Nikolaos Vrontaras from the Aristotle University of Thessaloniki, emphasised the importance of involving patients in their treatment decisions through various strategies. By offering clear and concise information, healthcare providers can empower patients to actively participate in discussions about their care. Additionally, creating a supportive and respectful environment encourages patients to express their preferences and concerns.

The presentation included role-playing videos that illustrated the impact of both effective and ineffective communications, serving as powerful examples of how the way information is conveyed can significantly influence patient engagement.

One of the key takeaways from the session is the significance of addressing patient concerns. By actively listening to patients and acknowledging their fears, doubts, or questions, healthcare providers can establish trust and foster a collaborative relationship. Moreover, effective communication involves more than just speaking; it also requires actively listening to patients’ perspectives, validating their experiences, and responding empathetically.

Another crucial aspect highlighted in the session is the need to empower patients to make informed decisions. Providing comprehensive information about treatment options, risks, benefits, and alternatives equips patients with the knowledge necessary to actively participate in their own healthcare journey.

Ultimately, effective patient communication contributes to better healthcare outcomes and increased patient satisfaction. When patients are engaged, well-informed, and actively participate in their care, they are more likely to adhere to treatment plans, experience improved health outcomes, and report higher levels of satisfaction with their healthcare providers. By incorporating such strategies and principles, healthcare professionals can enhance their communication skills and foster stronger partnerships with their patients.

What does this mean for patients?

This session emphasised the crucial role of healthcare professionals (HCPs) in involving patients in their treatment decisions and fostering a supportive environment. By providing clear information, actively listening to patients’ concerns, and empowering them with comprehensive knowledge, HCPs can build trust, enhance patient engagement, and ultimately improve healthcare outcomes and patient satisfaction. These educational sessions for HCPs offer hope for a positive shift in HCP-patient communication, driven by HCPs themselves.

International Perspectives and Clinical Trial Breakthroughs in ALL and T-ALL

At the EHA 2023 conference, the European Working Group for Adult Acute Lymphoblastic Leukemia (EWALL) organised a session featuring researchers and clinicians from Finland, Italy, France, and North America. They shared significant research advancements from their respective national working groups.

In Finland, a unique system for ALL patient care has been implemented, ensuring national protocols and close monitoring of outcomes are used for all patients. While gathering real-world data presents challenges, efforts are underway to enhance data availability. Notably, Finland has established a remarkable hematological registry and biobank (FHRB – www.fhrb.fi) containing samples from over 500 ALL patients. Researchers worldwide are encouraged to apply for access to these materials. Studies utilising these biobank samples have compared drug effectiveness in different types of ALL and explored genetic predispositions, particularly the DNA repair gene CHEK2. Approximately one in ten patients exhibit a rare genetic predisposition to the disease, emphasising the need for further research before implementing genetic analysis in clinical practice.

The Italian perspective at the conference focused on T-cell ALL (T-ALL). While progress has been made in treating B-cell ALL, T-ALL does not respond as well to current treatments. Researchers in Italy have developed a genetic profiling test to identify patients who might respond well to specific drugs and to discover new targets for drug development. Through studying the reaction of T-ALL cells to different drugs, they identified potential treatment targets, including the protein G9a. This approach aids in personalised treatment selection based on each patient’s genetic mutations, allowing for tailored therapies. The speaker stressed the importance of consistent testing and collaboration with biotech and pharmaceutical companies to incorporate new or repurposed drugs. Ultimately, their research aims to enhance T-ALL treatment by finding effective therapies that align with each patient’s cancer cells.

The French experience in managing relapsed/refractory (R/R) T-ALL was also presented. Despite therapeutic advancements, R/R T-ALL patients face significant challenges. The speaker discussed the emergence of candidate targets and targeted therapies from basic research, emphasising the need to translate these findings into clinical practice. They described a new multi-strategy approach incorporating information from samples taken at diagnosis and relapse, as well as functional data from drug testing. Successful outcomes of this strategy were shared, leading to the validation of promising single-agent and combination therapies for specific T-ALL patient subgroups. The talk highlighted the importance of collaboration between diagnostic labs, research labs, and clinicians to improve personalised treatment options and identify biomarkers for predicting treatment response in T-ALL.

Lastly, the session included the latest clinical trial results from the Children’s Oncology Group (COG) in North America, focusing on T-ALL and ALL in children and adolescent/young adults (AYA). The speaker emphasised the success achieved in curing over 90% of children with ALL through collaborative clinical trials. However, a significant number of patients still relapse, needing improved therapies. Findings from various clinical trials were presented, highlighting recent achievements such as improved disease-free survival in T-ALL patients treated with the combination of capizzi methotrexate and nelarabine. The addition of bortezomib and nelarabine showed good potential in paediatric and AYA T-cell lymphoblastic lymphoma and T-ALL. Ongoing trials explored targeted therapies, immunotherapeutic approaches, and reduced chemotherapy intensity to improve outcomes and reduce toxicity. The importance of precision medicine and matching patients with the appropriate drugs was further highlighted.

What does this mean for patients?

The research presented at EHA 2023 has important implications for patients. Finland’s ALL care system and biobank, Italy’s genetic profiling test for T-ALL, French strategies for R/R T-ALL, and North America’s Children’s Oncology Group’s clinical trial results offer improved care, personalised treatment, and hope for better outcomes for various ALL subgroups.

Advances in Treatment Approaches for AML: Immunotherapy, Targeted Combinations, and Maintenance Strategies.

This educational EHA 2023 session focused on new and innovative treatment approaches for AML.

Prof. Marion Subklewe, from the Ludwig Maximilian University, Germany, started the session by explaining that traditional chemotherapy is not always effective for patients with AML and that researchers are now exploring immunotherapy, which uses the body’s immune system to fight cancer. They talked about two types of immunotherapies. The first uses special proteins called bispecifics to activate immune cells, called T-cells, which attack AML cells. Clinical trials using bispecifics have shown some positive responses, but there can be side effects. The second approach involves genetically modified immune cells, called chimeric antigen receptor T-cells (CAR-T), to recognise and attack AML cells. Some patients in clinical trials have seen a good cancer reduction, but there are still challenges involving finding the right proteins to target and managing side effects. The speaker concluded by stating that immunotherapy shows great promise for AML, but there are still challenges to address and the understanding of the genetic makeup of AML is crucial, as some mutations can make the cancer cells less vulnerable to immune attacks. Ongoing clinical trials aim to find the most effective and safe ways to use immunotherapy for AML patients.

During the session, Prof. Paresh Vyas, from the University of Oxford, UK, discussed the importance of combining different targeted treatments to improve patient outcomes. Single target treatments have not been very effective as cancer cells find ways to resist them. Combination therapies are being evaluated for specific patient groups, but careful management of toxicity and evolving resistance is crucial. Drugs approved by the U.S. Food and Drug Administration (FDA), like ivosidenib, enasidenib, and gilteritinib were mentioned, but none provide a cure. Preliminary data on targeted combinations showed effectiveness, especially in specific subsets of patients but sustained responses and real-world outcomes require further study. Monitoring resistance in routine settings was recommended with bone marrow sampling at relapse. Collaboration and access to scientific knowledge were advocated for better outcomes.

Lastly, Dr. Bas Wouters, from the Erasmus MC Cancer Institute, The Netherlands, discussed the available maintenance treatments for AML that aim to prevent or delay relapse after initial therapy. Hypomethylating agents (HMAs), and especially oral azacytidine, have shown good potential for maintenance therapy. Another potential maintenance treatment, FLT3 inhibitors, targeting this common AML mutation, has yielded mixed results in clinical trials, thus requiring further research. The first generation FLT3 inhibitors, midostaurin and sorafenib, do not seem to improve patient outcomes. Nevertheless, the second-generation one, gilteritinib, has shown promise in relapsed/refractory AML potentially for a subset of patients (those with measurable residual disease [MRD]-positivity), but these still need to be further validated. Important questions regarding how to best select patients who benefit the most, whether we are preventing or delaying relapse with maintenance therapy, and how to position maintenance therapy in the era of venetoclax-based regimens, remain unanswered. Overall, the advancements show promise, but further research and well-designed clinical trials are needed to validate and improve AML treatment strategies.

What does this mean for patients?

This session showcased new AML treatment approaches, such as immunotherapy, offering hope for patients resistant to traditional chemotherapy. Bispecifics and CAR-T cell trials showed positive responses, but challenges remain. Combining targeted treatments and exploring maintenance therapies aim to improve outcomes, requiring further research and clinical trials.

Optimizing AML Treatment Strategies: Patient Involvement, Molecular Testing and Combination Therapies

The session covered important aspects of acute myeloid leukemia (AML), such as involving patients in treatment decisions, timely molecular testing, and treatment debates based on recent evidence, all aimed at optimising AML treatment strategies.

Dr. Peter Valk, from the Erasmus MC University, The Netherlands, discussed the significance of timely molecular testing in AML patients, emphasising its role in identifying genetic mutations, guiding treatment decisions, and monitoring disease progression. Prioritising timely testing was shown to improve patient outcomes and enable effective treatment planning.

Dr. Eytan Stein, from the Memorial Sloan Kettering Cancer Center, U.S., emphasised the importance of shared decision making for borderline chemo-eligible AML patients. Involving patients in treatment decisions improves adherence, patient satisfaction, and overall outcomes. Encouraging meaningful discussions between physicians and patients empowers patients and optimises their treatment journey.

The session also featured a debate on the optimal treatment for elderly AML patients that are ineligible for intensive chemotherapy with an isocitrate dehydrogenase 1 (IDH-1) mutation. Two first line treatment options were presented: venetoclax + azacytidine, and ivosidenib + azacytidine. Both options showed very promising results in clinical trials, including response rates, overall survival, and transfusion independence. The speakers highlighted the distinct toxicity profiles of the treatments due to their different mechanisms of action, requiring differently tailored monitoring strategies. Both treatments are approved by the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) and offer hope for IDH-1 mutated AML patients. The debate concluded with the professional opinion of Dr. Andre Schuh, from the Princess Margaret Cancer Centre, Canada, that ivosidenib is the preferred choice over venetoclax for this patient population. This was based on ivosidenib’s overall response rates, potentially higher complete response rates, longer survival, better tolerability, and easier patient management.

The session ended with a case presentation on combination therapies by Dr. Daver DiNardo and Dr. Courtney DiNardo, focusing on the triplet combination of venetoclax, ivosidenib (IDH-1 inhibitor), and azacytidine (hypomethylating agent [HMA]) in relapsed AML patients. This triplet regimen showed impressive response rates in early small studies, particularly in high-risk myelodysplastic syndrome (MDS) patients. The speakers emphasised the importance of continuous molecular profiling, especially after a patient has experienced relapse, as mutational profiles can change. Such profiling plays a vital role in determining the most beneficial treatment options and enabling personalised medicine for AML patients. Further research is required to validate these findings and identify the optimal combination treatments and sequences. However, these results offer great hope for patients.

What does this mean for patients?

Patients must be given a greater role in their AML treatment decisions to lead to improved treatment adherence and satisfaction. Timely molecular testing enables better identification of genetic mutations and personalised treatment plans. The discussion on treatment options and combination therapies offers hope for patients, with potential for higher response rates, longer survival, and tailored approaches based on individual mutational profiles.


Top line results of FELIX pivotal Phase II study (Autolus) – Efficacy and safety of Obecabtagene autoleucel (OBE CEL, AUTO1), a fast off rate of CD19 CAR in R/R Adult B ALL –> FELIX study

Top line results of KOMET-001 study (Kura Oncology) – Activity, tolerability and resistance profile of the menin inhibitor ziftomenib in adults with relapsed or refractory NPM1-mutated AML –> KOMET-001 study

HARMONY Alliance research results in ALL and APL

ALL: Press release | HARMONY Acute Lymphoblastic Leukemia (ALL) abstract presented at EHA2023 – HARMONY Alliance (harmony-alliance.eu)

APL: Press release | HARMONY Acute Promyelocytic Leukemia (APL) abstract presented at EHA2023 – HARMONY Alliance (harmony-alliance.eu)