2021 Innovation in hematology
Vekemans MC., Havelange V., Van Den Neste E., Bailly S., Lambert C., Straetmans N., Poiré X., Constantinescu SN., Hermans C.
The year 2021 was rich in innovations in regard to both malignant and benign hematological diseases, with the most relevant of which discussed below. • In recent years, the prognosis of multiple myeloma has been greatly improved, yet the disease remains as yet incurable. Current strategies aim to achieve optimal disease control at diagnosis using four-drug regimens designed to delay relapse, while in relapsed patients, innovative alternatives like immunotherapy are being employed to effectively re-arm the immune system, and this represents a real hope for patients suffering from this chronic disease. • The management of chronic myeloid leukemia is still evolving. Since 2001, tyrosine kinase inhibitors have revolutionized its prognosis, but they are also responsible for toxicities. One of the current therapeutic goals is to achieve a sufficiently deep and prolonged response, thereby enabling tyrosine kinase inhibitors to be discontinued and remission maintained. • Myelofibrosis is a myeloproliferative neoplasm with poor prognosis. The only curative treatment available to date is peripheral stem cell allograft, which can be applied at least for some patients. New therapeutic agents are currently being investigated, which are highly promising not only on account of their efficacy on symptoms and splenomegaly, but also for their potential anti-fibrotic and reducing effect on the mutated clone. Certain agents are in clinical assays in combination with JAK2 inhibitors. • Several recent studies have suggested that CAR-Ts were likely to emerge as the second-line treatment of choice for patients suffering from aggressive lymphomas, while secondarily reducing the number of autologous stem cell transplants. • Patients with acute myeloid leukemia and TP53-mutated myelodysplastic syndromes still represent a challenging population to treat with low and only shortlived response rates. The advent of APR-246, which restores TP53 activity, is likely to increase the number of responders among these very high-risk patients. • Beyond its well-known involvement in heparin thrombocytopenia, platelet factor 4 (PF4) plays a major role in the very rarely observed thrombosis with thrombocytopenia occurring in association with COVID-19 adenoviral vector vaccines. Inhibition of FXI is expected to emerge as a promising anticoagulation strategy with reduced bleeding risk, while induction of antithrombin deficiency by interfering RNA is likely to prevent bleeding in hemophilia A and B patients, either with and without inhibitors. • Concerning the management of immune thrombocytopenic purpura, targeted therapies are currently being favored owing to their lower toxicity and individualized platelet count targets.