D. Selleslag MD

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

Targeted next generation DNA sequencing for the detection of clonal haematopoiesis in idiopathic cytopaenia of undetermined significance (ICUS)

SUMMARY

Patients lacking diagnostic criteria for myelodysplastic syndrome, but who show an unexplained persistent cytopaenia are classified as patients suffering from idiopathic cytopaenia of undetermined significance (ICUS). A fraction of these patients carry somatic mutations in genes which are also mutated in myeloid neoplasms. The significance of these mutations in ICUS patients is not well known and only few research papers have tried to correlate them with clinical outcome. ICUS patients carrying somatic mutations seem to have a higher progression rate to myeloid malignancies compared to unmutated patients. Some mutation profiles also show lower overall survival, similar to patients with (low-risk) myelodysplastic syndrome. Therefore, it seems useful to screen for somatic mutations in cytopaenic patients. The goal of this paper is to review recent literature regarding the significance of somatic mutations in cytopaenic patients and propose a screening protocol by evaluating a test protocol at the AZ Sint-Jan hospital Brugge-Oostende.

(BELG J HEMATOL 2019;10(6):231–40)

Highlights in acute myeloid leukemia

The ASH 2018 conference in San Diego featured a number of exciting new studies with clinical relevance for the treatment of patients with acute myeloid leukemia (AML). The key studies that are discussed here are grouped in 3 broad treatment groups: targeted therapies, immunotherapy and allogeneic stem cell transplantation (allo SCT).

(BELG J HEMATOL 2019;10(1):36–40)

A Belgian consensus protocol for autologous haematopoietic stem cell transplantation in multiple sclerosis

SUMMARY

Multiple sclerosis is considered to be an immune mediated inflammatory disorder of the central nervous system. It mainly affects young, socioeconomic active patients. Although our armamentarium for this disease has significantly evolved in recent years some patients remain refractory to conventional therapies. In these cases, autologous haematopoietic stem cell transplantation can be considered as a therapeutic option. Decreasing morbidity, mortality and increasing patient awareness have led to rising inquiry by our patients about this treatment option. With the aim of a standardised protocol and data registration, a Belgian working party on stem cell therapy in multiple sclerosis was established. In this paper, we report the consensus protocol of this working party on autologous haematopoietic stem cell transplantation in multiple sclerosis.

(BELG J HEMATOL 2018;9(5):167–74)

Management of immune thrombocytopenia in multiple sclerosis patients treated with alemtuzumab: a Belgian consensus

SUMMARY

Alemtuzumab (Lemtrada®) is a humanised monoclonal antibody indicated for the treatment of adult patients with relapsing/remitting multiple sclerosis with active disease defined by clinical or imaging features. Alemtuzumab demonstrated superior efficacy over active comparator in both treatment naive patients and those with inadequate response to prior therapy. Alemtuzumab is associated with a consistent and manageable safety and tolerability profile. Treatment with alemtuzumab for multiple sclerosis increases the risk for autoimmune adverse events including immune thrombocytopenia. Complete blood counts with differential should be obtained prior to initiation of treatment and at monthly intervals thereafter for 48 months after the last infusion. After this period of time, testing should be performed based on clinical findings suggestive of immune thrombocytopenia. If immune thrombocytopenia onset is confirmed, appropriate medical intervention should be promptly initiated, including immediate referral to a specialist. This paper presents the consensus of Belgian multiple sclerosis specialists and haematologists to guide the treating physician with practical recommendations.

(BELG J HEMATOL 2018;9(3):118–23)

P07 Refractory mediastinal myeloid sarcoma preceding an acute myeloid leukemia with t(10;11) chromosome translocation

Is there a place for allogeneic stem cell transplantation in chronic lymphocytic leukaemia in the era of the new molecules?

SUMMARY

Allogeneic stem cell transplantation can cure about 40% of patients with chronic lymphocytic leukaemia. The early transplant related mortality with reduced intensity conditioning is low, but the late non relapse mortality is around 20% due to the high incidence of chronic graft versus host disease. The graft versus leukaemia effect is crucial for cure of chronic lymphocytic leukaemia after allogeneic stem cell transplantation and can be obtained by immune interventions. The place of allogeneic stem cell transplantation needs to be redefined in the era of novel targeted treatments (BCR pathway inhibitors and BCL2 inhibitors). Taking into consideration the promising results of BCR pathway inhibitors in genetically high-risk chronic lymphocytic leukaemia (with 17p deletion/TP53 mutation or complex karyotype) and fludarabine resistant chronic lymphocytic leukaemia, the current recommendation is to proceed with allogeneic stem cell transplantation in chronic lymphocytic leukaemia patients failing a BCR pathway inhibitor. The question when to proceed with allogeneic stem cell transplantation in responding patients remains unanswered. In the absence of randomised or prospective observational studies comparing novel agents to allogeneic stem cell transplantation the decision should be individualised and depend on the estimated transplantation risks and the patient’s desires.

(BELG J HEMATOL 2017;8(5):185–91)