S. Anguille MD, PhD

Venetoclax-Azacitidine, the new standard of care for AML patients unfit for intensive treatment: A guide for clinical practice

SUMMARY

Acute myeloid leukaemia is an aggressive form of bone marrow cancer with poor prognosis, especially in elderly, unfit patients. The VIALE-A study showed an impressive improvement in complete remission rate and overall survival with the addition of venetoclax, a BCL-2 inhibitor, to azacitidine. This combination therapy is now reimbursed in Belgium for newly diagnosed adult AML patients who are considered unfit for intensive chemotherapy based on age and/or comorbidities. In this article, we provide recommendations on the use of this new combination, as well as on prophylaxis and management of specific side effects.

(BELG J HEMATOL 2023;14(2):59–66)

A multicentric observational study on the management of hyperleukocytic acute myeloid leukaemia in Belgium

SUMMARY

INTRODUCTION

In hyperleukocytic acute myeloid leukaemia (AML) the risk of leukostasis is high due to the rapid increase in WBC count and the size of the myeloid blasts. It is associated with poor prognosis due to an increased risk of early death and relapse. Immediate initiation of cytoreductive treatment is essential to improve outcome, but evidence to prefer hydroxyurea, leukapheresis, intensive chemotherapy (IC) or a combination treatment, is lacking. Therefore, we decided to investigate the current approach of hyperleukocytic AML in Belgium.

METHODS

A brief questionnaire on the management of hyperleukocytic AML was sent to all Belgian centres currently treating AML with IC and was replied by ten centres. Four centres agreed to a more detailed retrospective analysis. All newly diagnosed AML patients presenting with hyperleukocytosis between January 2013 and April 2019 were included. Patient and disease characteristics were collected, as well as treatment choice and outcome parameters.

RESULTS

We included 121 patients with a median WBC count of 116,360/µL. Mortality at day 21 was 20% and overall mortality was 64% at a median follow-up of six months. Twenty percent received leukapheresis, which was started within 24 hours. There was no difference in age distribution, treatment intensity or time to start IC between patients receiving leukapheresis or not. Although the leukapheresis group had a more severe presentation with a higher median WBC and blast count and a worse performance status, there was no difference in response to therapy, early or long-term mortality. In a multivariate analysis, age at diagnosis and treatment modality (IC vs non-IC) were the only independent parameters that significantly affected early death.

CONCLUSION

Evidence on optimal treatment options in hyperleukocytic AML is lacking. We could not demonstrate any added value of leukapheresis. To improve the prognosis of this dramatic presentation, national or even European databases should be used to document and learn from the outcome of current practice.

(BELG J HEMATOL 2020;11(7):325-34)

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)

Highlights in acute leukemia

(BELG J HEMATOL 2019;10(5):195–200)

Targeted therapies in multiple myeloma: new antibodies and CAR-T cells

SUMMARY

New therapeutic antibodies and T cells redirected to specific antigen targets with engineered chimeric antigen receptors (CARs) are emerging as powerful therapies in haematologic malignancies and multiple myeloma (MM). Various designs, manufacturing processes, and study populations, among other variables, have been tested and reported in clinical trials in MM. Here, we review and compare ongoing trials and the results of the reported clinical trials. We also discuss the outlook for CAR-T cell therapies, including managing toxicities and expanding the availability of personalized cell therapy as a promising approach to all haematologic malignancies.

P35 PD-1 blockade in a patient with relapsed Hodgkin lymphoma post-allogeneic hematopoietic cell transplant: complete metabolic response without graft-versus-host-disease

Paroxysmal nocturnal haemoglobinuria

SUMMARY

Paroxysmal nocturnal haemoglobinuria is a rare, acquired haematological disease that manifests with haemolytic anaemia, thrombosis and impaired bone marrow function. The absence of two glycosylphosphatidylinositol-anchored proteins, CD55 and CD59, leads to uncontrolled complement activation that accounts for haemolysis and other paroxysmal nocturnal haemoglobinuria manifestations. Patients may present with a variety of clinical manifestations, such as anaemia, thrombosis, kidney disease, smooth muscle dystonias, abdominal pain, dyspnoea, and extreme fatigue. Delayed recognition of this condition is common due to the variable clinical presentation. This delay in diagnosis confers an increased risk of mortality and morbidity. Therefore, the purpose of this review is to raise awareness about this potentially life-threatening disease among haematologists and to provide a guide to diagnosis and treatment.

(BELG J HEMATOL 2017;8(7):259–64)