T. Feys MBA, MSc

New haematology reimbursements in Belgium

OVERVIEW OF BELGIAN REIMBURSEMENT NEWS

(BELG J HEMATOL 2020;11(2):82)

Journal Scan

The goal of this new section in the BJH is to provide a snapshot of pivotal studies published in recent issues of the most important international journals focusing on haematology. Importantly, the selection of the studies discussed here is the sole responsibility of the publisher and was not influenced by third parties. Do you miss an important study, or did you read a hidden jewel that deserves to be shared with your colleagues? Please, let us know (editor@bjh.be) and we will make sure to include it in the journal scan section of the next BJH issue.

(BELG J HEMATOL 2020;11(2):79–81)

Highlights in chronic myeloid leukaemia

Summary

ASH 2019 did not feature ground-breaking results in the field of CML. Nevertheless, some interesting abstracts deserve to be discussed. This includes a study looking at the potential of next-generation sequencing (NGS) and digital droplet PCR (ddPCR) in monitoring responses in patients treated with a BCR-ABL1 TKI. As in previous years, many abstracts in CML focused on the concept of treatment discontinuation and treatment-free remission (TFR). In this overview, one study will be discussed looking at TFR in children, while another abstract describes a predictive score for TFR. A final TFR abstract concerns a long-term follow-up analysis of patients who discontinued TKI therapy in a French institution. With respect to therapeutic advances, two phase III trials looked into the potential of adding pegylated interferon to nilotinib in the frontline treatment of CML, while a third abstract discusses the results of a phase I trial in which a new third-generation TKI (HQP1351) was evaluated in patients with relapsed/refractory CML.

(BELG J HEMATOL 2020;11(1):13–7)

Highlights in myelodysplastic syndromes

Summary

In this article, a selection of interesting abstracts related to myelodysplastic syndromes (MDS) will be discussed. In lower-risk MDS, most attention goes to therapies that address anaemia. In this respect updated results of a phase III trial evaluating luspatercept were presented, while also the hydroxylase inhibitor roxadustat showed promising results. In addition to this, interesting data were presented on the restoration of erythropoietin sensitivity by lenalidomide. In higher-risk MDS, an abstract will be discussed looking at the optimal schedule for the administration of hypomethylating agents (HMA) next to a study evaluating the safety and efficacy of an oral HMA formulation. In addition to this, several new therapeutic options are being explored in higher-risk MDS. This includes combinations of novel agents (e.g. venetoclax, rigosertib, telaglenastat) with azacitidine and the use of targeted agents, specifically directed against mutations in MDS (e.g. olutasidenib, APR-246 and enasidenib).

(BELG J HEMATOL 2020;11(1):27–34)

Miscellaneous news from ASH 2019

Summary

In addition to the plethora of abstracts in the larger haematological subdomains discussed in this special issue of the BJH, ASH 2019 also featured many interesting presentations that do not fall within one of these categories. In this article we would like to address some of this ‘miscellaneous news’ from ASH 2019. In the field of venous thromboembolism (VTE), bodyweight-adjusted rivaroxaban could provide a new alternative treatment option for paediatric patients. Also with respect to VTE, the Ottawa score failed to demonstrate its predictive value for VTE recurrence in cancer patients. In addition, interesting new data were presented on the prevention of graft-versus-host-disease (GVHD) after an allogeneic transplantation. At this year’s meeting, there was also a session dedicated to disorders in the number or function of platelets in which much attention went to novel drug targets and novel drug combinations for the treatment of immune thrombocytopenia. Finally, some interesting presentations on sickle cell disease, myelofibrosis-associated anaemia and cold agglutinin disease (CAD) will be discussed in this overview.

(BELG J HEMATOL 2020;11(1):35–40)

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)

Antibody-drug conjugates in the treatment of haematological malignancies

SUMMARY

Antibody-drug conjugates (ADCs) combine the specificity of monoclonal antibodies with biologically active cytotoxic molecules or drugs. As such, they can deliver cytotoxic agents specifically at the tumour site in a way that minimises systemic exposure and its associated toxicity. As of 2001, four ADCs have been approved by the European Medicines Agency for multiple human malignancies: gemtuzumab ozogamicin, brentuximab vedotin, trastuzumab emtansine, and inotuzumab ozogamicin. In addition to this, several new promising agents are under development. Although ADCs represent a new, effective class of therapeutics, the selection of the appropriate cytotoxin and linker remains challenging and systemic toxicity and rapid clearance should be monitored carefully. This review gives an overview on the safety and efficacy of ADCs in the treatment of haematological malignancies.

(BELG J HEMATOL 2019;10(8):311–9)