SPECIALS

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.

Liquid biopsies for Hodgkin and DLBCL (differential) diagnosis

Cell-free plasma DNA (cfDNA) and circulating tumour DNA (ctDNA) represent potential alternative sources of diagnostic information. During his lecture at the 2021 BHS general annual meeting Dr. Jo Van Dorpe discussed the potential of these liquid biopsies in the diagnostic work-up of different lymohoma subtypes.

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.

Antibody-drug conjugates in the treatment of haematological malignancies

SUMMARY

Antibody-drug conjugates (ADCs) combine the specificity of monoclonal antibodies with biologically active cytotoxic molecules or drugs. As such, they can deliver cytotoxic agents specifically at the tumour site in a way that minimises systemic exposure and its associated toxicity. As of 2001, four ADCs have been approved by the European Medicines Agency for multiple human malignancies: gemtuzumab ozogamicin, brentuximab vedotin, trastuzumab emtansine, and inotuzumab ozogamicin. In addition to this, several new promising agents are under development. Although ADCs represent a new, effective class of therapeutics, the selection of the appropriate cytotoxin and linker remains challenging and systemic toxicity and rapid clearance should be monitored carefully. This review gives an overview on the safety and efficacy of ADCs in the treatment of haematological malignancies.

(BELG J HEMATOL 2019;10(8):311–9)

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)