SPECIALS

CML: All needs met in 2021?

The therapeutic landscape of chronic myeloid leukaemia (CML) has changed dramatically over the past decades and the majority of patients with Philadelphia chromosome-positive (Ph+) CML in chronic phase (CP) now have a near-normal life expectancy.¹ Based on two recent published guidelines (European LeukemiaNet (ELN) guideline¹ and the British Society for Haematology guideline²) and the presentation by Prof. Jane Apperley during the 2021 BHS GAM, this article will give an overview of the latest developments in the therapeutic CML landscape and address a range of the persistent unmet needs in the diagnosis and treatment of CP-CML.

Paroxysmal nocturnal haemoglobinuria: from diagnosis to management

Paroxysmal nocturnal haemoglobinuria (PNH) is a rare hematopoietic stem cell disorder resulting from the somatic mutation of the X-linked PIGA gene. PNH has a very heterogeneous clinical presentation and the treatment for PNH patients depends on the severity of symptoms and the degree of hemolysis. In a special lecture at the 2021 BHS general annual meeting. Dr. Bérangère Devalet and Prof. Nicole Straetmans gave an overview of the current PNH landscape, from diagnosis to management.

The role of the pathologist in the diagnosis of lymphoma

The diagnosis of lymphomas requires multiple immunohistochemical (IHC) analyses in combination with additional tests, such as fluorescent in situ hybridization (FISH) and/or polymerase chain reaction (PCR) tests. During his lecture at the 2021 BHS general annual meeting, Dr. Thomas Touseyn discussed the contemporary role of the pathologist in the diagnostic work-up of aggressive lymphomas.

Advances in adult ALL therapy

Acute lymphoblastic leukaemia (ALL) is a rare disease with the highest incidence in childhood and patients older then 60 years. While the long-term outcomes of ALL have improved significantly in the paediatric population and to a lesser extent in young adults, elderly patients still have a very poor prognosis.^1,2 In fact, half of all deaths from ALL occur in patients older than 55 years and their 5-year overall survival (OS) rate ranges from 10–20%.^3,4 During the 36^th general annual meeting of the Belgian Haematology Society Prof. Anita Rijneveld, from the Erasmus Cancer Institute, Rotterdam, the Netherlands, gave an overview of recent advances in the treatment of adults ALL patients.

Sickle cell disease for the adult hematologist

In recent years we have witnessed a growing interest in the treatment of patients with sickle cell disease (SCD). Advances in general medical care, early diagnosis and comprehensive treatment have led to substantial improvements in the life expectancy of individuals with SCD in high-income countries. During her lecture at the 2021 annual meeting of the BHS, Prof. Veerle Labarque gave an overview on the current treatment landscape for adult SCD patients.

Chimeric antigen receptor T-cells: a new therapeutic option for relapsed/refractory B-cell malignancies and beyond

Chimeric antigen receptor (CAR) T-cell therapy is a new cancer immunotherapy targeting specific cell surface antigens. This type of adoptive cell immunotherapy has been a breakthrough in the treatment of aggressive B-cell lymphoma and B-cell precursor acute lymphoblastic leukaemia (ALL) and is currently also being studied in other cancer types, including multiple myeloma and chronic lymphocytic leukaemia. This review will discuss the recent clinical developments and future perspectives of CAR T-cell therapy, with a focus on the clinical trials that led to the FDA and EMA approval of tisagenlecleucel (Kymriah®, Novartis) and axicabtagene ciloleucel (Yescarta®, Gilead) for the treatment of childhood/adult relapsed/refractory (r/r) B-cell precursor ALL and aggressive B-cell non-Hodgkin lymphoma.

(BELG J HEMATOL 2019;10(8):301–10)

Bispecific T-cell engagers (BiTEs) in haematological malignancies

SUMMARY

Bispecific T-cell engagers (BiTEs) are a class of immunotherapeutics that can redirect T cells to haematological malignancies. A key advantage of BiTEs over adoptive T-cell therapies, consists of the fact that a BiTE is an “off the shelf” meaning that the same product can be given to all patients. In contrast, adoptive T-cell therapies must be made from cells taken from each patient and as a result this strategy is more time consuming and potentially more expensive. The most successful BiTE to date is blinatumomab. This agent is made up of CD3 and CD19 single-chain variable regions linked by a glycine–serine linker. It binds selectively to CD3 expressing T cells and CD19 expressing B cells, leading to the formation of immune synapses between T cells and B cells. In doing so, blinatumomab redirects unstimulated cytotoxic T cells to specifically target and lyse CD19-positive B cells. Blinatumomab is currently approved for patients with relapsed/refractory and minimal residual disease positive B-cell precursor acute lymphoblastic leukaemia (B-ALL). This review will discuss the pivotal trials with this agent and will touch upon some of the additional BiTEs that are under clinical evaluation in haematological malignancies. Finally, some remaining challenges with respect to optimising the efficacy and safety of BiTEs will be addressed.

(BELG J HEMATOL 2019;10(8):332–8)