Y. Beguin MD, PhD

Front-line venetoclax monotherapy for blastic plasmacytoid dendritric cell neoplasm

SUMMARY

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare aggressive haematological disease with a grim prognosis. The majority of patients at diagnosis are elderly (typical age range: 60 to 70 years) and seldom fit enough to receive intensive induction therapy. We report here the case of a 76-year-old patient with significant cardiac comorbidity at the time of the diagnosis of BPDCN with bone marrow involvement. He achieved haematologic complete remission with venetoclax as first-line therapy with preservation of a good quality of life, an almost exclusively outpatient care and transfusion independency. The only adverse event was a grade 4 neutropenia without any infectious complication.

(BELG J HEMATOL 2024;15(3):125–9)

Primary immune thrombocytopenia in adults: Belgian recommendations for diagnosis and treatment anno 2021 made by the Belgian Hematology Society

SUMMARY

The Belgian Hematology Society (BHS) updated the 2013 guidelines for diagnosis and treatment of primary immune thrombocytopenia (ITP).¹ As knowledge about ITP pathophysiology is increasing, the mode of action of old therapies is better understood and novel drugs are introduced to target more specific pathways. Corticosteroids with or without intravenous immunoglobulins (IgIV) remain the first line treatment. According to the updated international guidelines, a short course of corticosteroids rather than a prolonged treatment has to be recommended. The same guidelines stress that consequent therapies as thrombopoietic agents (TPO-RAs) and rituximab should be available independent of duration of ITP. Although the majority of recommendations is based on very low-quality evidence, it is strongly advised to individualise the ITP management taking patient values and preferences in account. The main treatment goal in all ITP patients must be to maintain a safe platelet count to prevent or stop bleeding with a minimum of toxicity and not to normalise the platelet count.

(BELG J HEMATOL 2020;12(3):112-27)

Acute graft-versus-host disease: diagnosis, pathophysiology and prevention

SUMMARY

Acute graft-versus-host disease (aGVHD) remains a severe complication after allogeneic stem cell transplantation (alloHCT). It is a disregulated immune process, during which the immune cells of the donor attack the healthy tissues in the immunocompromised host. Over the past two decades, progress in understanding its pathophysiology have helped redefine aGVHD reactions and clinical presentations. Typically, the disease presents with serious inflammatory lesions mainly in the skin, gut and liver. Its severity is assessed by gathering clinical signs and dysfunctions of each organ. Despite standard prophylaxis regimens, aGVHD still occurs in approximately 30–60% of transplanted patients and remains a major cause of transplant-related morbidity and mortality. Hence, there is an urgent need for optimising preventive strategies. In this review, we give insights on how to make an accurate diagnosis and scoring assessment of aGVHD, propose a short overview of the current knowledge about its immunobiology and discuss the current and developing strategies for prevention.

(BELG J HEMATOL 2020;11(4):159–173)

PP.03.2 Improvement of antibody-induced cell death in multiple myeloma

O.6 Multipotent mesenchymal stromal cells for poor graft function after allogeneic hematopoietic cell transplantation – a multicenter prospective study

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)

Erythropoiesis and iron metabolism after haematopoietic stem cell transplantation

After haematopoietic stem cell transplantation (HCT), many patients present anaemia, which can persist for months due to an inadequate erythropoietin production for the degree of the anaemia. In this thesis, we performed two randomised studies with erythropoiesis-stimulating agents therapy after allogeneic (including myeloablative and non-myeloablative conditioning) and autologous transplantation. We showed a great efficacy of this growth factor to ensure full erythroid reconstitution when initiated soon after engraftment and not immediately after the transplant. Furthermore, as iron parameters are quite disturbed following HCT, we sought to study iron metabolism after HCT (which has not been much investigated), integrating the role of hepcidin, the key regulator in iron metabolism. Hence, we demonstrated that hepcidin levels prior to and following autologous HCT were influenced by iron stores and changes in erythropoietic activity.

(BELG J HEMATOL 2019;10(2):89–95)