J. Maertens MD, PhD

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

Defeating cytomegalovirus, the transplantation troll: can letermovir do the job?

Letermovir (AIC246, MK-8228) is a novel anti-cytomegalovirus (CMV) agent that inhibits CMV replication by targeting the viral terminase complex. In December 2017, letermovir was approved by the Food and Drug Administration (FDA) for the prophylaxis of CMV infection and disease in adult CMV-seropositive recipients of an allogenic haematological stem cell transplantation. Letermovir shows a favourable pharmacokinetic profile in haematological stem cell transplantation recipients after oral administration. The recommended dose for CMV-prophylaxis is once daily 480 mg (oral or intravenous). Letermovir demonstrated superiority in a placebo (plus polymerase chain reaction-monitoring and pre-emptive therapy)-controlled phase III randomised clinical trial. Letermovir is an inhibitor of the cytochrome P450 (CYP)3A family, CYP2B8 and an inducer of the CYP2C9/19. Dose-adjustments (240 mg/day) are necessary when letermovir is combined with cyclosporine. Combinations of letermovir with either voriconazole, midazolam and rosiglitazone require close monitoring of the plasma levels of the latter agents. Letermovir-resistant CMV mutants share mutations that are mostly located between the codon range 230–370 of the UL56 gene. Letermovir is not nephrotoxic nor myelotoxic, but slightly higher rates of atrial fibrillation and tachycardia have been described. In conclusion, letermovir is the first FDA approved anti-CMV agent for prophylaxis in haematological stem cell transplantation patients.

(BELG J HEMATOL 2019;10(4):136–45)

01 A pilot study to assess the feasibility of unrelated umbilical cord blood transplantation with coinfusion of third-party mesenchymal stromal cells after myeloablative or non-myeloablative conditioning in patients with haematological malignancies

Management of severe aplastic anaemia

SUMMARY

Aplastic anaemia is a rare condition characterised by pancytopenia and bone marrow hypocellularity and caused by the immune-mediated destruction of the haematopoietic precursors. The early complications are related to cytopaenias with infections being the major cause of morbi-mortality. The main long-term issue is clonal evolution to myelodysplastic syndrome or acute leukaemia. The diagnosis relies on exclusion of other causes of pancytopenia and characteristic pathologic findings. Severity is stratified according to peripheral blood counts. Nowadays, the survival of treated patients reaches 80–90%. The treatment of the severe form of aplastic anaemia consists on haematopoietic stem cell transplantation in eligible patients and immunosuppressive therapy in non-transplant candidates. Supportive therapy is an option in frail and/or elderly patients. Here, we define and briefly review the pathogenesis of aplastic anaemia. We propose a diagnostic and therapeutic strategy based on existing literature and experts’ recommendations. We finally report three cases illustrating particular clinical associations with pregnancy, hepatitis and paroxysmal nocturnal haemoglobinuria.

(BELG J HEMATOL 2018;9(3):76–85)

P37 Comparison of Multigam® IV (5% vs. 10%) in hematological patients with secondary immunodeficiencies to evaluate infusion time, tolerability and satisfaction. A monocentric observational Belgian study

O.1 Infusion of mesenchymal stem cells (MSC) as treatment for steroid refractory acute graft-versus-host study (aGVHD): a multicenter prospective phase II study of the BHS

PP3.3 The ‘EBMT GVHD App’ improves accuracy of graft versus host disease assessment according to NIH criteria compared to standard practice – A proof of concept