An expert panel recently examined unmet clinical needs (UCNs) for patients with Philadelphia-negative myeloproliferative neoplasms (Ph-negative MPNs), in terms of dealing with an increased risk of thrombotic events. The panel presented its findings in Blood Cancer Journal.
The panel identified 8 UCNs they deemed to be of the greatest relevance, and for each of these UCNs the panel provided recommendations for researchers and/or clinicians. Some UCNs related to the categorization of risk of thrombotic events. These included a need for more understanding of the connections between somatic gene mutations and thrombosis risk. The panel recommended a prospective study to identify genotypes associated with thrombosis risk.
Another risk-related UCN identified by the panel involved quantification of thrombosis risk in polycythemia vera, and the panel noted that elevated white blood cells may serve as a prognostic tool with this condition. Identification of the relationship between a mutated endothelium and thrombosis risk was another identified UCN, for which the panel recommended a case-control study.
Multiple UCNs involved assessing advantages and risks of treatments, such as those outcomes related to the use of direct oral anticoagulants and the use of cytoreduction for splanchnic vein thrombosis without hypercythemia. The panel suggested study designs to best address these UCNs.
Treatment-related UCNs also included uncertainty around the dosing of aspirin in essential thrombocythemia, the timing of anticoagulation prophylaxis in recurrent venous thromboembolism, and the need for more information regarding optimal antithrombotic treatment for women with Ph-negative MPNs during pregnancy. The panel made study recommendations to address these uncertainties in treatment.
“The hope is that this comprehensive overview will serve to inform the design and implementation of research in the field and will help the management of special clinical situations in these disorders,” wrote the authors in their report.
Barbui T, De Stefano V, Falanga A, et al. Addressing and proposing solutions for unmet clinical needs in the management of myeloproliferative neoplasm-associated thrombosis: A consensus-based position paper. Blood Cancer J. 2019;9(8):61.