Preface – EHA 2020

Portrait Tam

Constantine Tam, MB, BS (Hons), MD, FRACP, FRCPA, Peter MacCallum Cancer Centre, Royal Melbourne Hospital, and University of Melbourne Melbourne, Australia

Dear Colleagues,
Due to the circumstances brought about by the COVID-19 pandemic, the 25th European Hematology Association (EHA) Annual Congress had to take place as a virtual edition, although this raised new possibilities such as a 10-day program. Like its predecessors conducted onsite, the EHA25 Virtual Congress offered original unpublished scientific hematology data, hematological innovations, and evidence-based knowledge of primary clinical relevance.
This memo inHaematology publication summarizes content presented on the topics of B cell malignancies, paroxysmal nocturnal hemoglobinuria, and cold agglutinin disease.

Within the field of non-Hodgkin lymphoma, Bruton’s tyrosine kinase (BTK) inhibitors have emerged as important players in the management of patients with Waldenström’s macroglobulinemia and mantle cell lymphoma but were also shown to be active in diffuse large B cell lymphoma, follicular lymphoma, and marginal zone lymphoma. Next-generation agents with optimized features are under development. In patients with mantle cell lymphoma, promising results obtained with novel agents challenge the role of chemotherapy, particularly in the front-line setting. Both single-agent and combined regimens containing various classes of targeted agents might open up new dimensions and are being extensively evaluated in clinical trials.
BTK inhibition has also transformed the treatment of chronic lymphocytic leukemia and continues to be investigated alone and together with other agents. The advent of modern treatment options has given rise to an increasing demand for time-limited therapy in these patients. Fixed-duration approaches, possibly guided by the assessment of minimal residual disease, represent a feasible road ahead.
Although rare, paroxysmal nocturnal hemoglobinuria is a potentially life-threatening condition that calls for effective management. The range of available agents targeting the complement system is currently being expanded. Novel agents, by addressing various factors that enhance residual anemia, contribute to more complete disease control. Likewise, cold agglutinin disease shows a low prevalence but considerably affects patient prognosis and quality of life. As in paroxysmal nocturnal hemoglobinuria, inhibition of certain components of the complement system can lead to rapid and durable clinical improvements. Global registry data will elucidate clinical characteristics as well as the use of treatments, patient outcomes and the natural history of this disease.

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Patient and disease characteristics in a small CAD cohort

A retrospective analysis hints at the wide range of cold agglutinin disease (CAD) clinical behavior. Koudouna et al. investigated the characteristics of 8 patients with CAD at the time of diagnosis [1]. Median age was 62 years, and 5 patients were women. Hematologic malignancies constituted 50 % of underlying medical conditions; in 37 %, hepatitis B/C was the associated disease, and in 13 %, autoimmune disorders.

Cold agglutinin disease: on the road to new insights and potential treatment options

Cold agglutinin disease (CAD) is a rare type of autoimmune hemolytic anemia (AIHA) elicited by cold-sensitive antibodies including cold agglutinins. Ninety percent of cold agglutinins belong to the IgM kappa category and bind to red blood cell surface antigens at temperatures of ≤ 37 °C, thus inducing hemolysis.

Paroxysmal nocturnal hemoglobinuria: improving outcomes with novel strategies

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, potentially life-threatening clonal hematopoietic stem cell disorder characterized by hemolytic anemia, bone marrow failure, thrombosis, and peripheral blood cytopenia. The disease results from an acquired loss-of-function mutation of the PIGA gene involved in the synthesis of the glycosylphosphatidylinositol-anchored complement inhibitors CD55 and CD59.

Targeted approaches in various B-cell malignancies

BTK inhibitors are active in many B-cell malignancies such as mantle cell lymphoma, CLL and Waldenström’s macro­globulinemia, but also in diffuse large B-cell lymphoma (DLBCL), follicular lymphoma (FL), and marginal zone lymphoma (MZL). Zanubrutinib is currently being assessed in pivotal phase II and III studies in all of these indications.

Changing paradigms in the management of mantle cell lymphoma

Mantle cell lymphoma (MCL) is a rare, heterogenous and generally aggressive subtype of B-cell non-Hodgkin lymphoma that remains incurable in the majority of cases. Median survival in non-trial patients has been estimated at 3 to 5 years. First-line therapy usually consists of chemoimmunotherapy, while both immunochemotherapy and targeted agents are recommended in relapsed disease.

Optimizing timing, efficacy and tolerability in chronic lymphocytic leukemia

In both treatment-naïve and relapsed/refractory patients with chronic lymphocytic leukemia (CLL) and small lymphocytic leukemia (SLL), inhibition of Bruton’s tyrosine kinase (BTK) represents a treatment standard as it has improved clinical outcomes. Compared to the first-generation agent ibrutinib, the second-generation, highly selective BTK inhibitor acalabrutinib shows minimal off-target kinase inhibition, thus potentially offering an optimized safety profile.